Regenerative Medicine Market: Snapshot

Regenerative medicine is a part of translational research in the fields of molecular biology and tissue engineering. This type of medicine involves replacing and regenerating human cells, organs, and tissues with the help of specific processes. Doing this may involve a partial or complete reengineering of human cells so that they start to function normally.

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Regenerative medicine also involves the attempts to grow tissues and organs in a laboratory environment, wherein they can be put in a body that cannot heal a particular part. Such implants are mainly preferred to be derived from the patients own tissues and cells, particularly stem cells. Looking at the promising nature of stem cells to heal and regenerative various parts of the body, this field is certainly expected to see a bright future. Doing this can help avoid opting for organ donation, thus saving costs. Some healthcare centers might showcase a shortage of organ donations, and this is where tissues regenerated using patients own cells are highly helpful.

There are several source materials from which regeneration can be facilitated. Extracellular matrix materials are commonly used source substances all over the globe. They are mainly used for reconstructive surgery, chronic wound healing, and orthopedic surgeries. In recent times, these materials have also been used in heart surgeries, specifically aimed at repairing damaged portions.

Cells derived from the umbilical cord also have the potential to be used as source material for bringing about regeneration in a patient. A vast research has also been conducted in this context. Treatment of diabetes, organ failure, and other chronic diseases is highly possible by using cord blood cells. Apart from these cells, Whartons jelly and cord lining have also been shortlisted as possible sources for mesenchymal stem cells. Extensive research has conducted to study how these cells can be used to treat lung diseases, lung injury, leukemia, liver diseases, diabetes, and immunity-based disorders, among others.

Global Regenerative Medicine Market: Overview

The global market for regenerative medicine market is expected to grow at a significant pace throughout the forecast period. The rising preference of patients for personalized medicines and the advancements in technology are estimated to accelerate the growth of the global regenerative medicine market in the next few years. As a result, this market is likely to witness a healthy growth and attract a large number of players in the next few years. The development of novel regenerative medicine is estimated to benefit the key players and supplement the markets growth in the near future.

Global Regenerative Medicine Market: Key Trends

The rising prevalence of chronic diseases and the rising focus on cell therapy products are the key factors that are estimated to fuel the growth of the global regenerative medicine market in the next few years. In addition, the increasing funding by government bodies and development of new and innovative products are anticipated to supplement the growth of the overall market in the next few years.

On the flip side, the ethical challenges in the stem cell research are likely to restrict the growth of the global regenerative medicine market throughout the forecast period. In addition, the stringent regulatory rules and regulations are predicted to impact the approvals of new products, thus hampering the growth of the overall market in the near future.

Global Regenerative Medicine Market: Market Potential

The growing demand for organ transplantation across the globe is anticipated to boost the demand for regenerative medicines in the next few years. In addition, the rapid growth in the geriatric population and the significant rise in the global healthcare expenditure is predicted to encourage the growth of the market. The presence of a strong pipeline is likely to contribute towards the markets growth in the near future.

Global Regenerative Medicine Market: Regional Outlook

In the past few years, North America led the global regenerative medicine market and is likely to remain in the topmost position throughout the forecast period. This region is expected to account for a massive share of the global market, owing to the rising prevalence of cancer, cardiac diseases, and autoimmunity. In addition, the rising demand for regenerative medicines from the U.S. and the rising government funding are some of the other key aspects that are likely to fuel the growth of the North America market in the near future.

Furthermore, Asia Pacific is expected to register a substantial growth rate in the next few years. The high growth of this region can be attributed to the availability of funding for research and the development of research centers. In addition, the increasing contribution from India, China, and Japan is likely to supplement the growth of the market in the near future.

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Global Regenerative Medicine Market: Competitive Analysis

The global market for regenerative medicines is extremely fragmented and competitive in nature, thanks to the presence of a large number of players operating in it. In order to gain a competitive edge in the global market, the key players in the market are focusing on technological developments and research and development activities. In addition, the rising number of mergers and acquisitions and collaborations is likely to benefit the prominent players in the market and encourage the overall growth in the next few years.

Some of the key players operating in the regenerative medicine market across the globe areVericel Corporation, Japan Tissue Engineering Co., Ltd., Stryker Corporation, Acelity L.P. Inc. (KCI Licensing), Organogenesis Inc., Medtronic PLC, Cook Biotech Incorporated, Osiris Therapeutics, Inc., Integra Lifesciences Corporation, and Nuvasive, Inc.A large number of players are anticipated to enter the global market throughout the forecast period.

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Regenerative Medicine Market Analysis Growth Demand, Key Players, Share Size, and Forecast To 2025 - 3rd Watch News

Cardiac Stem Cells Comments Off on Regenerative Medicine Market Analysis Growth Demand, Key Players, Share Size, and Forecast To 2025 – 3rd Watch News | June 26th, 2020

Eustasis Psychiatric & Addiction Health

417-322-6622| 3600 S. National Ave., Springfield

Eustasis Psychiatric & Addiction Health is pleased to announce construction of their brand new site in the heart of Medical Mile, opening in July! There is expanded access to medication management, psychotherapy, testing and advanced treatment options.

Dr. Alok Jain and his wife Breanna Jain started the clinic in 2018 with the mission of providing the highest quality psychiatric care to all patients. Since then they have put together an amazing team of board-certified providers and support staff who have tirelessly served the Springfield community.

We wanted to build something really vital for our patients. A place that everyone could come, regardless of age or diagnosis, says Dr. Jain.

Dr. Alok Jain has been honored as a 417 Top Doctor every year since 2007 and is 2020's top psychiatrist. He is a board-certified psychiatrist, member of the American Psychiatric Association and has an extensive background in consultation-liaison psychiatry and psychopharmacology.

Eustasiss immediate-access site has provided patients with an unprecedented way to receive psychiatric care without delay. Their walk-in and be seen model is changing the face of psychiatry. The new location has eight providers and room for growth! They are providing the most state of the art modalities, including ADHD testing and esketamine.

It is super exciting, says Breanna Jain, CEO, PMHNP-BC. We have patients who come in all hours of the day, pediatric or adult, all payor sources. They will ask, You mean I can really be seen right now? We can proudly tell them of course! This is just the way we believe mental health should be done.

Both Dr. Jain and Breanna know that people need options in this community. Patients struggles are numerous, ranging from addiction, bipolar, ADHD, trauma, anxiety and more. The Jains like to think of Eustasis as a one-stop-shop.

There shouldnt be high levels of bureaucracy. This is what overwhelms patients. Barriers have no place when it comes to mental health, the Jains explain.

Eustasis is committed to helping patients find the optimal balance of emotions during these difficult times. They have expanded their hours and have both in person and telemedicine options available. They are always accepting new patients!

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Medical Professionals in the Ozarks - 417mag

Cardiac Stem Cells Comments Off on Medical Professionals in the Ozarks – 417mag | June 26th, 2020

One day before Sila Jio Boonklomjit was set to donate desperately needed stem cells to save his sisters life, doctors made an alarming discovery: Hed contracted COVID-19.

The 5-year-old COVID-19 patient is now being credited with saving his big sisters life by going through with an experimental procedure to cure her of a genetic blood disorder without passing along the coronavirus.

Saying it was the first known case of such a procedure, Ramathibodi Hospital claimed victory yesterday after successfully transplanting bone marrow from Jio to his sister, Jintanakan Jean Boonklomjit, who was born with thalassemia and was in a severe condition.

Its as if my daughter is reborn and gets a new life, said the childrens father, Suchai Boonklomjit.

Thalassemia is a hereditary disorder that limits the bloods ability to carry oxygen and affects an estimated 1% of all Thais. Rather than being treated by ongoing blood transfusions, recent breakthroughs have shown it can be cured through gene therapy.

The procedure began in April but wasnt completed until yesterday by Suradej Hongeng of the hospitals pediatrics department.

Posted by onTuesday, June 23, 2020

According to Suradej, it was a long and uncertain road to this happy outcome. It had been difficult to find a donor compatible with Jean, leading them to settle on Jio as her best hope. After they were confirmed to be a genetic match in 2018, they prepared for the transplantation procedure.

Other difficulties followed, in part due to the young age of both patients. Moreover, Jeans immune system was compromised by chemotherapy while Jio had to be placed in quarantine on the eve of the procedure. Doctors believe he was likely infected by his mother, Sasiwimol Boonklomjit.

The case is believed to be the first successful stem cell transplant from a donor with active COVID-19. Both Jio and his mother have since recovered from the virus.

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This article, 5-year-old Thai boy with COVID-19 saves sisters life with his stem cells, originally appeared on Coconuts, Asia's leading alternative media company. Want more Coconuts? Sign up for our newsletters!

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5-year-old Thai boy with COVID-19 saves sisters life with his stem cells - Yahoo Singapore News

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Hematopoietic Stem Cell Transplantation (HSCT) Market report provides (6 Year Forecast 2020-2026) including detailed Coronavirus (COVID-19) impact analysis on Market Size, Regional and Country-Level Market Size, Segmentation Market Growth, Market Share, Competitive Landscape, Sales Analysis and Value Chain Optimization. This Hematopoietic Stem Cell Transplantation (HSCT) market competitive landscape offers details by topmost key manufactures (Regen Biopharma Inc, China Cord Blood Corp, CBR Systems Inc, Escape Therapeutics Inc, Cryo-Save AG, Lonza Group Ltd, Pluristem Therapeutics Inc, ViaCord Inc) including Company Overview, Company Total Revenue (Financials), Market Potential, Presence, Hematopoietic Stem Cell Transplantation (HSCT) industry Sales and Revenue Generated, Market Share, Price, Production Sites and Facilities, SWOT Analysis, Product Launch. For the period 2014-2020, this study provides the Hematopoietic Stem Cell Transplantation (HSCT) sales, revenue and market share for each player covered in this report.

Key Target Audience of Hematopoietic Stem Cell Transplantation (HSCT) Market: Manufacturers of Hematopoietic Stem Cell Transplantation (HSCT), Raw material suppliers, Market research and consulting firms, Government bodies such as regulating authorities and policy makers, Organizations, forums and alliances related to Hematopoietic Stem Cell Transplantation (HSCT) market.

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Synopsis of Hematopoietic Stem Cell Transplantation (HSCT) Market:In 2019, the market size of Hematopoietic Stem Cell Transplantation (HSCT) is million US$ and it will reach million US$ in 2025, growing at a CAGR of from 2019; while in China, the market size is valued at xx million US$ and will increase to xx million US$ in 2025, with a CAGR of xx% during forecast period.

In this report, 2018 has been considered as the base year and 2019 to 2025 as the forecast period to estimate the market size for Hematopoietic Stem Cell Transplantation (HSCT).

Based onProduct Type, Hematopoietic Stem Cell Transplantation (HSCT) market report displays the manufacture, profits, value, and market segment and growth rate of each type, covers:

Allogeneic Autologous

Based onend users/applications, Hematopoietic Stem Cell Transplantation (HSCT) market report focuses on the status and outlook for major applications/end users, sales volume, market share and growth rate for each application, this can be divided into:

Peripheral Blood Stem Cells Transplant (PBSCT) Bone Marrow Transplant (BMT) Cord Blood Transplant (CBT)

Hematopoietic Stem Cell Transplantation (HSCT) Market: Regional analysis includes:

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The Hematopoietic Stem Cell Transplantation (HSCT) Market Report Can Answer The Following Questions:

What are the Upstream Raw Materials And Manufacturing Equipment of Hematopoietic Stem Cell Transplantation (HSCT)? What is the manufacturing process of Hematopoietic Stem Cell Transplantation (HSCT)?

Who are the key manufacturers of Hematopoietic Stem Cell Transplantation (HSCT) market? How are their operating situation (Capacity, Production, Price, Cost, Gross and Revenue)?

Economic impact on Hematopoietic Stem Cell Transplantation (HSCT) industry and development trend of Hematopoietic Stem Cell Transplantation (HSCT) industry.

What is the (North America, South America, Europe, Africa, Middle East, Asia, China, Japan) Production, Production Value, Consumption, Consumption Value, Import And Export of Hematopoietic Stem Cell Transplantation (HSCT)?

What will the Hematopoietic Stem Cell Transplantation (HSCT) Market Size and The Growth Rate be in 2026?

What are the key market trends impacting the growth of the Hematopoietic Stem Cell Transplantation (HSCT) market?

What are the Hematopoietic Stem Cell Transplantation (HSCT) Market Challenges to market growth?

What are the types and applications of Hematopoietic Stem Cell Transplantation (HSCT)? What is the market share of each type and application?

What are the key factors driving the Hematopoietic Stem Cell Transplantation (HSCT) market?

What are the Hematopoietic Stem Cell Transplantation (HSCT) market opportunities and threats faced by the vendors in the Hematopoietic Stem Cell Transplantation (HSCT) market?

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Hematopoietic Stem Cell Transplantation (HSCT) Market Expand Their Businesses With New Investments In 2020 And Coming Future - Bulletin Line

Bone Marrow Stem Cells Comments Off on Hematopoietic Stem Cell Transplantation (HSCT) Market Expand Their Businesses With New Investments In 2020 And Coming Future – Bulletin Line | June 25th, 2020

Summary

A review of 423 patients treated at MSK finds that most people with cancer dont fare any worse if they get COVID-19 than other people who are hospitalized for that infection.

In the early days of the COVID-19 pandemic, many doctors worried that people undergoing treatment for cancer would do particularly poorly if they became infected with the virus that causes the disease. Thats because treatments for cancer, especially chemotherapy, can lower a persons immune defenses and put them at higher risk for all kinds of infections.

But according to a new study from Memorial Sloan Kettering published June 24 in Nature Medicine, most people in active cancer treatment dont fare any worse if they get COVID-19 than other people who are hospitalized with the infection. Further research is needed to look at the effects of certain drugs mainly immunotherapies called checkpoint inhibitors, which did seem to make COVID-19 worse. But the researchers say their findings suggest that no one should delay cancer treatment because of concerns about the virus.

If youre an oncologist and youre trying to figure out whether to give patients chemotherapy, or if youre a patient who needs treatment, these findings should be very reassuring, says infectious disease specialist Ying Taur, one of the studys two senior authors.

Infectious disease expert Ying Taur has cared for many MSK patients who were hospitalized with COVID-19.

The study looked at 423 MSK patients diagnosed with COVID-19 between March 10 and April 7, 2020. Overall, 40% were hospitalized for COVID-19, and 20% developed severe respiratory illness. About 9% had to be placed on a mechanical ventilator, and 12% died. The investigators found that patients taking immunotherapy drugs called immune checkpoint inhibitors were more likely to develop severe disease and require hospitalization. But other cancer treatments, including chemotherapy and surgery, did not contribute to worse outcomes.

The big message now is clear: People should stay vigilant but not stop or postpone checkpoint immunotherapy or any other cancer treatment.

Factors that did make COVID-19 worse were the same as those seen in studies of people who didnt have cancer. We found that being older, as well as preexisting conditions like heart disease and diabetes, are all drivers of severe COVID-19 illness, says MSK Chief Medical Epidemiologist Mini Kamboj, the studys other senior author. This wasnt surprisingbecause these connections are well established.

Although the study wasnt large enough to make determinations about every treatment and every cancer type, patterns did emerge. Dr. Taur says there was initially great concern about people receiving high doses of chemotherapy for leukemia, especially those who had recently undergone bone marrow or stem cell transplants. Thats because transplants require a persons entire immune system to be wiped out with chemotherapy before they receive new blood cells, leaving them susceptible to all kinds of infections.

Surprisingly, though, Dr. Taur cared for recent transplant recipients who were infected with COVID-19 but didnt have any symptoms. If you think about it more, it makes sense, he says. Most of the complications seen in people with COVID-19 seem to be caused by the bodys immune response to the virus.

On the other hand, immunotherapy drugs called checkpoint inhibitors work by freeing up the immune system to attack cancer. Patients receiving these agents may develop a more robust reaction to the virus that causes COVID-19. This may explain why this study observed higher rates of complications in people with COVID-19 infection who were treated with checkpoint inhibitors.

Even with immune checkpoint inhibitors, though, these findings should not affect whether patients get treated. Everyone who needs these drugs should still receive them, Dr. Kamboj says. Its just important for doctors to be extra vigilant about testing and monitoring for the virus and for people with cancer to take extra precautions to avoid infection.

A study published in May 2020 by MSK immunotherapy expert Matthew Hellmann focused exclusively on people with lung cancer who got COVID-19. The researchers didnt find the same risks from immune checkpoint drugs as this Nature Medicine study. But that study included data on far fewer patients treated at MSK, which could explain the difference.

Dr. Kamboj notes that one aspect of this research that sets it apart from other studies is that it included at least 30 days of follow-up after a COVID-19 diagnosis. Also, it reported severe respiratory illness as a main outcome rather than death.

Having that follow-up time is something that a lot of other studies have not included because everyone is in a rush to get their data out. In addition, reporting death rates can overestimate infection-related mortality, especially in the early phase of an epidemic, Dr. Kamboj says. Also, the clinical spectrum and course of this disease is still not fully understood, especially in people with cancer. We wanted to give patients enough time to recover and make sure they didnt need to be readmitted to the hospital.

Even with immune checkpoint inhibitors, though, these findings should not affect whether patients get treated. Everyone who needs these drugs should still receive them.

She adds that another strength of the study is that patient outcomes were not affected by constraints caused by a lack of space or supplies even though MSK is in the heart of the COVID-19 epicenter in New York City, where other hospitals faced overcrowding and other issues. This gave researchers a true picture of how cancer patients fare with COVID-19. We saw a surge during the peak of the epidemic in New York, but everyone got the care they needed, Dr. Kamboj explains. We had enough ventilators for everyone who needed them. We never had to make decisions about who to admit to intensive care because of a lack of critical equipment.

Drs. Taur and Kamboj agree that this is just one of many studies that will need to be done on the connections between cancer and COVID-19. We still need to find out more. We need to look at the connections between COVID-19 and particular types of cancer as well as outcomes related to specific chemotherapy drugs, Dr. Taur concludes. But the big message now is clear: People should stay vigilant but not stop or postpone checkpoint immunotherapy or any other cancer treatment.

Link:
Should You Delay Cancer Treatment Because of COVID-19? Study Says Most Treatments Dont Worsen Coronavirus Infection - On Cancer - Memorial Sloan...

Bone Marrow Stem Cells Comments Off on Should You Delay Cancer Treatment Because of COVID-19? Study Says Most Treatments Dont Worsen Coronavirus Infection – On Cancer – Memorial Sloan… | June 25th, 2020

Bone marrowaspiration and trephine biopsy are usually performed on the back of the hipbone, or posterior iliac crest. An aspirate can also be obtained from the sternum (breastbone). For the sternal aspirate, the patient lies on their back, with a pillow under the shoulder to raise the chest. A trephine biopsy should never be performed on the sternum, due to the risk of injury to blood vessels, lungs or the heart.

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The need to selectively isolate and concentrate selective cells, such as mononuclear cells, allogeneic cancer cells, T cells and others, is driving the market. Over 30,000 bone marrow transplants occur every year. The explosive growth of stem cells therapies represents the largest growth opportunity for bone marrow processing systems.Europe and North America spearheaded the market as of 2016, by contributing over 74.0% to the overall revenue. Majority of stem cell transplants are conducted in Europe, and it is one of the major factors contributing to the lucrative share in the cell harvesting system market.

In 2016, North America dominated the research landscape as more than 54.0% of stem cell clinical trials were conducted in this region. The region also accounts for the second largest number of stem cell transplantation, which is further driving the demand for harvesting in the region.Asia Pacific is anticipated to witness lucrative growth over the forecast period, owing to rising incidence of chronic diseases and increasing demand for stem cell transplantation along with stem cell-based therapy.

Japan and China are the biggest markets for harvesting systems in Asia Pacific. Emerging countries such as Mexico, South Korea, and South Africa are also expected to report lucrative growth over the forecast period. Growing investment by government bodies on stem cell-based research and increase in aging population can be attributed to the increasing demand for these therapies in these countries.

Major players operating in the global bone marrow processing systems market are ThermoGenesis (Cesca Therapeutics inc.), RegenMed Systems Inc., MK Alliance Inc., Fresenius Kabi AG, Harvest Technologies (Terumo BCT), Arthrex, Inc. and others

Covid 19 Impact[emailprotected] https://www.trendsmarketresearch.com/report/covid-19-analysis/3184

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Bone Marrow Processing Systems Market Business Analysis, New Innovation | Share, Revenue, And Sales Till 2025 - Cole of Duty

Bone Marrow Stem Cells Comments Off on Bone Marrow Processing Systems Market Business Analysis, New Innovation | Share, Revenue, And Sales Till 2025 – Cole of Duty | June 25th, 2020

Disruption of the TP53 gene and mutation of the KMT2D gene are predictive of poor outcomes in patients with mantle cell lymphoma (MCL) who are receiving high-dose therapy, according to a new study.

The findings, which build upon growing knowledge of the genetics of MCL, can be used to better classify patients into risk categories, the investigators said. The study was published in the journal Haematologica.1

Corresponding author Simone Ferrero, MD, of the University of Torino, in Italy, noted that the current treatment paradigm for patients with MCLcytarabine-based chemotherapy followed by autologous stem cell transplantation (ASCT)has led to dramatic advancements in the outcomes of patients who are young and healthy enough for the therapy. However, Ferrero said as many as one-quarter of those patients will experience early treatment failure.

So far, the best tool clinicians have had to identify patients at high risk of relapse has been the MCL international prognostic index (MIPI), and the Ki-67 proliferation index. The combination of the 2 is known as the MIPI-c score. Although the tool is of value, Ferrero and colleagues asserted that the resulting scores lack the precision necessary to develop tailored schedules specifically for high-risk patients.

In an effort to better elucidate the factors associated with a high risk of failure, the investigators used samples from the phase III FIL-MCL0208 trial, which is a prospective, open-label, multicenter study designed to evaluate lenalidomide (Revlimid) as a maintenance therapy versus observation in patients in MCL remission following high-dose chemotherapy including rituximab (Rituxan) followed by ASCT (NCT02354313).

Ferrero and colleagues performed targeted resequencing and DNA profiling on purified tumor samples of the patients in the study. Out of 300 patients enrolled in the study, samples from 186 patients were able to be evaluated for genetic mutations and abnormalities in copy numbers.

The analysis confirmed earlier reports2,3 that TP53 disruption is a significant prognostic factor. After 4 years, patients with mutations or deletions of TP53 had lower progression-free survival (PFS) and overall survival (OS) rates compared with patients without the disruptions.

However, the authors broke new ground by identifying KMT2D as another important genetic factor. In wild-type cases, those with KMT2D mutations had PFS rates of just 33.2%, versus 63.7% in those without the mutation after 4 years (P <.001). Overall survival was similarly affected; the 4-year OS rate among patients with KMT2D mutations was 62.3% versus 86.8% among those without the mutation (P = .002).

In the FIL-MCL0208 trial, KMT2D mutations emerged as a novel biomarker heralding chemo-immunotherapy failure, with a predictive value similar to that of TP53 aberrations, Ferrero and colleagues wrote.

The authors then used their findings to create a new scoring system to identify patients at the highest risk.

The independent adverse prognostic value of TP53 and KMT2D aberrations prompted us to integrate the molecular results into the MIPI-c, aiming at further improving its ability to discriminate high-risk patients, the authors said.

The model begins with MIPI-c score; those with low or intermediate risk scores under MIPI-c were given 0 points in the new model, and those placed in the high-risk category by the MIPI-c model were given one point. In addition, patients with TP53 disruptions were given 2 additional points, as were those with the KMT2D mutation. In this new scoring system, which the investigators dubbed MIPI-g, patients with a score of 0 were deemed low risk, patients with scores of 1 to 2 were deemed intermediate risk, and patients with scores of 3 or higher were categorized as high risk.

When investigators performed PFS and OS calculations based on their risk categories, they found PFS rates varied dramatically among the groups, from 72.0% in the low-risk group to 11.5% in the high-risk group after 4 years (P <.0001). Four-year OS rates similarly dropped from 94.5% in the low-risk group to 44.9% (P <.0001). Among patients in the intermediate group, the 4-year PFS rate was 42.2% and the OS rate was 65.8%.

In the Nordic validation series, patients with KMT2D mutations showed similar worse outcomes compared with wild-type patients (median OS, 8.4 vs 12.7 years). Among patients with TP53 mutations, the median OS was 2.0 years compared with 12.7 years for patients with wild-type TP53. The validation series also showed similar 4-year OS rates by risk groups: 91.3% for low-risk patients, 72.2% for intermediate risk, and 15.4% for high risk.

Among the studys limitations, the authors noted that their analysis was performed only on CD19-positive bone marrow cells. The investigators also said they do not yet have sufficient randomization data to know whether and to what extent lenalidomide maintenance affected the patients with these mutations within the broader FIL-MCL0208 trial. However, they said it is unlikely that full data will be able to offer clear takeaways, since only 27 patients with the TP53/KMT2D mutations were finally randomized in the study, due to a high rate of progressive disease among these patients. Of those 27, only 9 were started on lenalidomide maintenance.

In their conclusion, Ferrero and colleagues said that the ability to distinguish the highest-risk patients could be used by clinicians to identify high-risk patients for novel therapeutic approaches.

As in other lymphoid disorders, novel non-chemotherapeutic strategies specifically designed for [high-risk] patients need to be investigated in MCL, the authors said. Besides the approved drugs lenalidomide and ibrutinib [Imbruvica], new molecules such as the BCL-2 inhibitor venetoclax [Venclexta] might be very promising for these chemorefractory patients, especially for TP53 disrupted cases.

References:

1. Ferrero S, Rossi D, Rinaldi A, et al. KMT2D mutations and TP53 disruptions are poor prognostic biomarkers in mantle cell lymphoma receiving high-dose therapy: a FIL study. Haematologica. 2020;105(6):1604-1612. doi:10.3324/haematol.2018.214056

2. Nordstrm L, Sernbo S, Eden P, et al. SOX11 and TP53 add prognostic information to MIPI in a homogeneously treated cohort of mantle cell lymphoma--a Nordic Lymphoma Group study. Br J Haematol. 2014;166(1):98-108. doi:10.1111/bjh.12854

3. Halldrsdttir AM, Lundin A, Murray F, et al. Impact of TP53 mutation and 17p deletion in mantle cell lymphoma. Leukemia. 2011;25(12):1904-1908. doi:10.1038/leu.2011.162

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TP53, KMT2D Abnormalities Linked With Poor Prognosis in MCL - Targeted Oncology

Bone Marrow Stem Cells Comments Off on TP53, KMT2D Abnormalities Linked With Poor Prognosis in MCL – Targeted Oncology | June 25th, 2020

On February 67, 2020, the Simons Foundation Autism Research Initiative (SFARI) convened a two-day workshop to explore the possibility of gene therapies for autism spectrum disorder (ASD), a neurodevelopmental condition associated with changes in over 100 genes. Inspired by the recent, stunning successes of gene therapy for the fatal neuromuscular disorder spinal muscular atrophy (SMA)1, and by the accumulation of genes confidently associated with ASD2, SFARI welcomed a diverse collection of researchers to begin to think about whether a similar approach could be taken for ASD. Because gene therapy attempts to fix what is broken at the level of a causative gene, it would offer a more direct and imminent strategy than mitigation of the many and as yet mostly unclear downstream effects of a damaged gene.

The workshop was organized in 20 talks and several discussion panels, which tackled many outstanding issues, including how to choose candidate target genes and predict outcomes; how to optimize vectors for gene delivery; how to decide when to intervene; which animal models to develop; how to find appropriate endpoints for clinical trials and understand the available regulatory pathways. SFARI also raised the question of how its funding might best propel gene therapy efforts amid the emerging, complex ecosystem of academic laboratories, biotech companies, and pharmaceutical industries.

Even the opportunity to have this discussion is very rewarding, said SFARI Investigator Matthew State of the University of California, San Francisco (UCSF), one of the investigators who directed teams of geneticists to analyze the Simons Simplex Collection (SSC).

These efforts have offered up multiple potentially feasible therapeutic targets. Though rare, de novo disruptive mutations in the highest confidence ASD genes often result in severe impairment characterized not only by social difficulties, but also by intellectual disability and seizures. The combination of a single gene mutation of large effect coupled with particularly severe outcomes that include ASD are likely to offer the most immediate targets for gene therapy. For now, this leaves out a large number of individuals with autism for whom genetic causes are not yet known and are likely the result of a combination of many small effect alleles across a large number of genes.

Highlights from talks and discussion panel, chaired by Rick Lifton of Rockefeller University

In the first talk of the workshop, State brought the group up to speed on ASD genomics. The most recent tally from exome-sequencing in simplex cases of ASD highlighted 102 genes in which rare mutations confer individually large risks2. In contrast, the task of identifying common variants carrying very small risks remains quite challenging, with less than a half dozen alleles so far identified with confidence3. The rare, disruptive mutations that result in loss of function of one gene copy are an attractive focus for gene therapy because of the tractability of targeting a single spot in the genome per individual and because, in the vast majority of cases, there remains a single unchanged allele. This points to ways to boost gene and/or protein expression back toward the normal state by leveraging the unaffected copy. But both the limited number of cases known so far combined with the possibility that different mutations to the same gene may have different effects complicate thinking about how to prioritize targets for gene therapy.

State made several points that were continually touched on throughout the workshop. Many ASD genes are highly expressed during midfetal development in the cortex, and additional experiments will need to determine whether and how long a window of opportunity may be present for successful gene therapy postnatally. Given the relatively small number of people with these conditions, new clinical trial designs are needed that dont rely on comparisons between large control and intervention groups (see also Bryan Kings talk below).

Beyond the gene-crippling mutations found in the exome, disruptions to transcription may also dramatically raise risk for autism and may be corrected with a type of gene therapy using ASOs. SFARI Investigator Stephan Sanders of UCSF focused on the role of splicing, the process by which an initial transcript is turned into messenger RNA by removal of introns and joining together of exons. Splicing is disrupted in at least 1.5 percent of individuals with ASD4, and possibly many more, as suggested by transcript irregularities found in postmortem autism brain5. Sanders described Illuminas Splice AI project in which machine-learning helps predict noncoding variants that can alter splicing, including those beyond typical splice sites found near a gene6. As a result of incorporating sequence information around and between splice sites, this computational tool detected more mutations with predicted splice-altering consequences in people with ASD and intellectual disability than in those without the condition.

An ASO designed to bind specific portions of RNA could conceivably correct errors in transcription. ASOs have already been approved for use in other disorders in order to skip exons, retain exons or to degrade mRNA. Unlike other forms of gene therapy, ASOs do not permanently alter the genome, making it a kind of gene therapy lite. This reversibility has both disadvantages (having to re-infuse the ASO every few months) and advantages (multiple opportunities to optimize the dose and target; serious adverse effects are not permanent).

Jonathan Weissman of UCSF discussed the available toolbox for controlling gene expression developed by many different laboratories. To turn genes on or off, he has developed a method to combine CRISPR with an enzymatically inactive (dead) Cas9, which can then be coupled with a transcriptional activator (CRISPRa) or repressor (CRISPRi)7 (Figure 2). In the case of loss-of-function mutations, Weissman outlined strategies to make the remaining good allele work harder: increase transcription via CRISPRa, decrease mRNA turnover, increase translation of a good transcript via modification of upstream open reading frames (uORFs) or increase a proteins stability, possibly through small molecules acting on the ubiquitin system8. That said, the effects on a cell may be complicated. Using Perturb-Seq screens, Weissman described genetic interaction manifolds that show nonlinear mapping between genotype and single cell transcriptional phenotypes9. Additionally, Weissman summarized recent work from his laboratory that has identified large numbers of uORFs that result in polypeptides, some of which affect cellular function.

SFARI Investigator Michael Wigler of Cold Spring Harbor Laboratories echoed the idea of a gene-therapy strategy that increases expression of the remaining good copy of a gene, especially given that in his estimate, 45 percent of simplex cases of autism carried a de novo, likely disrupting variant. He also called attention to the uterine environment, especially the challenge posed by expression of paternally derived antigens in the fetus and the impact of a potential maternal immune response, and the need to understand how it interacts with de novo genetic events.

Highlights from talks and discussion panel, chaired by Arnon Rosenthal of Alector

The discussion turned to finding ways of getting genes into the central nervous system. The AAV is the darling of gene therapy, given that it does not replicate and is not known to cause disease in humans. A version that can cross the blood-brain barrier (AAV9) was used to deliver a gene replacement to children with SMA intravenously; though this effectively delivered the genetic cargo to ailing motor neurons in the spinal cord, it does not work that well at delivering genes throughout the brain.

Ben Deverman of the Stanley Center at the Broad Institute of MIT and Harvard detailed his efforts to optimize AAV for efficient transduction of brain cells through a targeted evolution process: his team engineers millions of variants in the capsid of the virus, then screens them for entry into the nervous system and transduction of neurons and glia. This has yielded versions (called AAV-PHP.B and AAV-PHP.eB) that more efficiently enter the brain10,11. One successfully delivered the MECP2 gene to the brain of a Rett syndrome mouse model, resulting in ameliorated symptoms and an extended lifespan12. Unfortunately, these viruses dont work in human cells or in all mouse strains. A quick mouse genome-wide association study (GWAS) revealed that the Ly6a gene mediates efficient blood-brain barrier crossing of AAV-PHP.B and AAV-PHP.eB13. Now his group has identified Ly6a-independent capsids that may translate better to humans. He also noted that the PHP.B vectors have tissue specificity for brain and liver.

With an estimated 87 percent of autism-associated genes raising risk through haploinsufficiency (having only one functional gene copy out of the two), SFARI Investigator Nadav Ahituv of UCSF made the case for approaches that boost expression of the remaining good copy of a gene through endogenous mechanisms a strategy he called cis-regulation therapy. This method also provides a way to work around the small four kb payload of AAV, which strains to contain cDNA of many autism genes. A recent study by his group used CRISPRa targeted at an enhancer or promoter of SIM1 and promoter of MC4R, both obesity genes, in mice. Using one AAV vector for a dCas9 joined to a transcription activator, and another AAV vector having a guide RNA targeting either a promoter or an enhancer, and a guide RNA targeting a promoter, the researchers injected the vectors together into the hypothalamus, which resulted in increased SIM1 or MC4R transcription and reversed the obesity phenotype brought on by loss of these genes14. Targeting regulatory elements had the added benefit of tissue specificity, and there seemed to be a ceiling effect for SIM1 expression, which suggested an endogenous safeguard against overexpression at work. He is now collaborating with SFARI Investigator Kevin Bender, also at UCSF, to apply this approach to the autism gene SCN2A.

Botond Roska of the Institute of Molecular and Clinical Ophthalmology in Basel, Switzerland pointed out that getting genes to the cells where they are needed is crucial when treating eye diseases. Off-target effects there can induce degeneration of healthy cells. For this reason, Roska and his group have created AAVs that target specific cell types in the retina by developing synthetic promoters that efficiently promote expression of the viruss cargo15. The promoters they designed were educated guesses based on four approaches: likely regulatory elements close to genes expressed with cell-type specificity in the retina, conserved elements close to cell typespecific genes, binding sites for cell typespecific transcription factors and open chromatin close to cell typespecific genes. Screening a library of these in mouse, macaque and human retina revealed some with high cell-type specificity (Figure 3). Importantly, macaque data predicted success in human retina much better than did mouse data. In preliminary experiments, and more relevant to gene therapy for ASD, these cell-specific vectors also had some success in mouse cortex, for example lighting up parvalbumin neurons or an apparently new type of astrocyte.

Roska also described new methods for delivery, in which nanoparticles are coated with AAV, then drawn into the brain using magnets16. This magnetophoresis technique allows a library of experimental AAVs to be tested at the same time in one monkey. Steering nanoparticles with magnets gives more control of vector placement and gene delivery. He argued that these in the future could access even deep structures of the brain.

Highlights from talks and discussion panel, chaired by Steven Hyman of the Broad Institute at MIT and Harvard

Kathy High of Spark Therapeutics reviewed the story of gene therapy for spinal muscular atrophy (SMA) type 1. Though she was not directly involved in that research, she is well aware of the regulatory atmosphere surrounding gene therapy, given that Spark Therapeutics developed the first approved AAV-delivered gene for a form of retinal dystrophy. The SMA story is a useful case study in that an ASO-based therapy (nusinersen, marketed as Spinraza), approved in 2016, set the stage for a gene-replacement therapy, marketed as Zolgensma (onasemnogene abeparvovec). Ultimately, the amount of data supporting Zolgensmas approval was modest: a Phase one dose study of 15 infants1, and an ongoing Phase three trial of 21 infants and safety data from 44 individuals. Yet the approval was helped by the dramatic results and clear endpoints: those receiving a single intravenous infusion of an AAV9 vector containing a replacement gene all remained alive at 20 months of age, whereas only 8 percent survived to that age in the natural history data, which compiles the diseases untreated course. High mentioned that maintaining product quality for gene therapeutics may prove trickier than for typical medications.

The attractive, highly customizable nature of gene therapy might have a regulatory downside in that different vector payloads, even when designed to do the same thing, could invite separate approval processes. Though not knowing how regulatory agencies would view this, High said that their perspectives are bound to evolve as more gene therapy trials are completed.

Getting to ASD-related syndromes, Bender talked about SCN2A, which encodes the sodium channel Nav1.2. SCN2A mutations in humans can be gain of function or loss of function; gain-of-function mutations are associated with early onset epilepsy, and loss-of-function mutations with intellectual disability and ASD. In a mouse model missing one copy of SCN2A, Bender and his group have discovered a role for SCN2A in action potential generation in the first week after birth, and in synaptic function and maturation afterward through regulation of dendritic excitability18 (Figure 4). Using AAV containing CRISPRa constructs developed with the Ahituv lab, the researchers successfully increased SCN2A expression, and recovered synapse function and maturity, even when done several weeks postnatally. Getting the appropriate dosage is critical since gain-of-function mutations are linked to epilepsy. However, Bender reported even when SCN2A expression increased to double normal levels, no hints of hyperexcitability appeared. We might be able to overdrive this channel as much as we want and actually may not have risk of producing an epileptic insult, he said. Next steps are to figure out the developmental windows for intervention, evaluate changes in seizure sensitivity and extend this kind of cis-regulatory approach to other ASD genes.

Angelman syndrome is another condition that attracts interest for gene therapy, in part because neurons already harbor an appropriate replacement gene. Angelman syndrome stems from mutations to the maternally inherited UBE3A gene, which is particularly damaging to neurons because they only express the maternal allele, while the paternal allele is silenced by an antisense transcript. SFARI Investigator Mark Zylka of the University of North Carolina and colleagues showed in 2011 that this paternal allele could be unsilenced with a cancer drug in a mouse model of Angelman syndrome19. Since then, three companies have built ASOs to do the same thing, and these are going into clinical trials. To get a more permanent therapeutic, Zylka has been developing CRISPR/Cas9 systems to reactivate paternal UBE3A, and preliminary experiments show that injecting this construct into the brains of embryonic mice, and then again at birth, results in brain-wide expression of paternal UBE3A and is long-lasting (at least 17 months). Zylka is now making human versions of these constructs. He later noted rare cases of mosaicism for the Angelman syndrome mutation people with 10 percent normal cells in blood have a milder phenotype20, which suggests that even inefficient transduction of a gene vector could help.

Zylka also made a case for prenatal interventions in Angelman syndrome: studies of mouse models indicate that early reinstatement of UBE3A expression in mouse embryos rescues multiple Angelman syndrome-related phenotypes, whereas later postnatal interventions rescue fewer of these21; for humans, a diagnostic, cell-based, noninvasive prenatal test will be available soon22; ultrasound-guided injections into fetal brain of nonhuman primates have been developed23; prenatal surgeries are now standard of care for spinal bifida; and intervening prenatally decreases the risk of an immunogenic response to an AAV vector or its cargo. During the discussion, it was noted that another benefit of acting early was that less AAV would be needed to transduce a much smaller brain; however, a drawback is the lack of data on Angelman syndrome development from birth to one year of age. This natural history would be necessary for understanding whether a prenatal therapy is more effective than treatment of neonates.

SFARI Investigator Guoping Feng of the Massachusetts Institute of Technology has been investigating SHANK3, a high-confidence autism risk gene linked to a severe neurodevelopmental condition called Phelan-McDermid syndrome, which is marked by intellectual disability, speech impairments, as well as ASD. SHANK3 is a scaffold protein important for organizing post-synaptic machinery in neurons. Mouse studies by Feng have shown that SHANK3 re-expression in adult mice that have developed without it can remedy some, but not all, of their phenotypes, including dendritic spine densities, neural function in the striatum and social interaction24. Furthermore, early postnatal re-expression rescued most phenotypes. This makes SHANK3 a potential candidate for gene therapy; however, it is a very large gene 5.2kb as a cDNA that is difficult to fit into a viral vector. To get around this, Fengs group has designed a smaller SHANK3 mini-gene as a substitute for the full-sized version. Preliminary experiments show that AAV delivery of the mini-gene can rescue phenotypes like anxiety, social behavior and corticostriatal synapse function in SHANK3 knockout mice. Feng also discussed his success in editing the genome in marmosets and macaques using CRISPR/Cas9 technology and showed data from a macaque model of SHANK3 dysfunction25. These models may help test gene therapy approaches and identify biomarkers of brain development closely related to the human disorder.

For people with rare conditions brought on by even rarer mutations, individualized gene therapies can provide a pathway for treatment. SFARI Investigator Timothy Yu of Boston Childrens Hospital/Harvard described his N-of-1 study in treating a girl with Batten disease, a recessive disorder in which a child progressively loses vision, speech and motor control while developing seizures. In a little over a year, an ASO that targeted her unusual splice-site mutation in the CLN7 gene was designed, developed and given intrathecally to the girl26. The lift was in negotiating with the FDA and working with private organizations, not just in the science, Yu said. After a year of treatment with the ASO (dubbed milasen after the girl, Mila), there were no serious adverse events; seizure frequency and duration had decreased (Figure 5); and possibly her decline had slowed. Though she remains blind, without intelligible speech and unable to walk on her own, she was still attentive and could respond happily to her familys voices. The highly personalized framework for this drugs approval is completely different from how medications meant for populations are approved, and it opens a regulatory can of worms, Yu said, though he added that the regulators were willing to countenance drug approval for an individuals clinical benefit.

Rett syndrome is a neurodevelopmental condition caused by mutations to the MECP2 gene that has a substantial research base in mouse models. Over 10 years ago, mouse models highlighted the possibility for therapeutics in this condition when Rett-associated phenotypes were rescued by adding back MECP2, even in adulthood27. This reversibility has spurred interest in gene therapy for Rett syndrome, but getting the MECP2 dose right is critical, said Stuart Cobb of the University of Edinburgh and Neurogene: just as too little MECP2 leads to Rett syndrome, too much also results in severe phenotypes. For this reason, it would be nice to package a replacement MECP2 gene with other regulatory elements to control its expression, but this results in constructs that do not fit into viral vectors. To make more room, Cobb and his colleagues have been able to chop away two-thirds of the MECP2, reserving two domains that interact to make a complex on DNA (Figure 6). Mice with this mini-gene are viable and have near normal phenotypes; likewise, injecting this mini-gene into MECP2-deficient mice extended their survival28. Doubling the dose, however, substantially lowered survival. Putting in safety valves to prevent overexpression is going to be quite important, he said. One idea is to add back a construct containing only the last two exons of MECP2, which is where most Rett mutations land. These would then be spliced into native transcripts (called trans-splicing), and thus their expression controlled by endogenous regulatory elements.

Underscoring the double-edged sword of MECP2 dosage, Yingyao Shao from Huda Zoghbis lab at Baylor described an MECP2 duplication syndrome (MDS) in humans, which features hypotonia, intellectual disability, epilepsy and autism. Experiments in an MDS mouse model, which carries one mouse version and one human version of MECP2, recapitulates some of the phenotypes of the human condition and can be rescued by an ASO targeting the human allele29. Shao described work to optimize the ASO for translation into humans, which involved developing a more humanized MDS model that carries two human MECP2 alleles. An acute injection of the ASO was able to knock down MECP2 expression in a dose-dependent manner in these mice, and RNA levels dropped a week after injection, with protein levels falling a week later. MECP2 target genes also normalized their expression level, and one maintained this for at least 16 weeks post-injection. The ASO also rescued behavioral phenotypes of motor coordination and fear conditioning, but not of anxiety; these corrections followed the molecular effects, and these timelines would be important to keep in mind while designing clinical trials. Shao also noted that overtreatment with the ASO resulted in Rett-associated phenotypes, but that this was reversible, which suggests that some fine-tuning of dosing in humans might be possible.

To avoid overtreatment and toxicity of any MDS-directed therapy, Mirjana Maletic-Savatic, also at Baylor, is leaving no stone unturned in a hunt for MDS biomarkers that can predict, in each individual, the safety of a particular dose and regimen. Such biomarkers would also help monitor individuals during treatment, give information about target engagement and identify candidates for a particular treatment. Anything found to be sensitive to expression levels of MECP2 could also be useful for Rett, though she noted that MECP2 levels measured in blood do not track linearly with gene copy number. Thus, because of interindividual variability, her approach is to collect a kitchen sink of data deriving composite biomarkers that accurately reflect the stage and severity of disease in a given case. She and her colleagues are collecting clinical, genetic, neurocircuitry (such as EEG and sleep waves), immunology and molecular data detected in blood, urine and CSF. These measures are also being explored in induced neurons derived from skin samples of people with MDS. She highlighted two interrelated potential biomarkers in the blood of those with this condition; both measures are downstream targets of MECP2 and are responsive to ASO treatment.

Highlights from Early detection and clinical trial issues talks and panel discussion, chaired by Paul Wang of SFARI

Coming up with objective measures of a persons status either their eligibility for a treatment, or whether the treatment has engaged with its target or even whether the treatment is effective is a real necessity in autism-related conditions, which comprise multiple interrelated behaviors. Eye-tracking methodology may provide such a marker, argued SFARI Investigator Ami Klin of Emory University. Focusing on the core social challenges of autism, Klin, Warren Jones and colleagues have been studying children as they view naturalistic social scenes to quantify their social attention patterns. This has revealed how remarkably early in development social visual learning begins and that this process is disrupted in infants later diagnosed with ASD prior to features associated with the condition appearing. By missing social cues, autism in many ways creates itself, moment by moment, Klin said. In considering gene therapy, it may be useful to know that eye looking (how much a subject looks at a persons eyes, an index of social visual engagement) in particular and social visual engagement in general are under genetic control30; that eye-tracking differences emerge as early as 26 months of age; and that homologies in social visual engagement exist between human babies and nonhuman infant primates.

In getting to a point to test gene therapies, identifying those who need them is essential. Wendy Chung of Columbia University and the Simons Foundation illustrated how diagnosis is yoked closely to therapy. To illustrate this, she described her pilot study of newborn blood spots to screen for SMA; at the start, no treatment was available, but the screen identified newborns for a clinical trial of nusinersin. Notably, the screen only cost an additional 11 cents per baby. In the three years since her pilot screen began, the FDA approved two gene therapies for SMA and the SMA screen was adopted for nationwide newborn screening. Currently she is piloting a screen for Duchenne muscular dystrophy and plans to develop a platform that will allow researchers to add other conditions. In prioritizing genetic conditions for gene therapy, she outlined some ideas for focus, such as genes resulting in phenotypes that would not be identified early without screening, those that are relatively frequent, those that are lethal or neurodegenerative, those with a treatment in clinical trials or with FDA-approved medications, and those conditions that are reversible.

In the meantime, Chung also outlined SFARIs involvement in establishing well-characterized cohorts of individuals with autism, which can help lay a groundwork for gene therapy. People with an ASD diagnosis can join SPARK (Simons Foundation Powering Autism Research for Knowledge), which collects medical, behavioral and genetic information (through analysis of DNA from saliva, at no cost to the participant). If a de novo genetic variant is found in one of ~150 genes, that person is referred to Simons Searchlight, which fosters rare conditions communities and which is also compiling natural history data on people with these mutations.

Bryan King of UCSF discussed how current trial designs for ASD were inadequate for gene therapy trials. As ASD prevalence has grown, parallel design trials with one group receiving an experimental medicine and the other a placebo are the standard, but these wont be possible for the rare conditions that are candidates for gene therapy. Also, change is hard to capture, given the malleable nature of ASD: with no intervention, diagnosis can shift between ASD and pervasive developmental disorder-not otherwise specified (PDD-NOS) in 1284 months (as defined by the DSM-IV). Current scales are subjective and may miss specific items of clinical significance. (Last year, SFARI funded four efforts to develop more sensitive outcome measures.) King outlined other pitfalls in ASD clinical trials, including significant placebo responses, inadequate sample sizes and not being specific enough when asking about adverse effects. King also mentioned improvements that may arise from just enrolling in a study, which could prompt previously housebound families to venture out with their child, which could kick off a cascade of positive effects. He reiterated how, for gene therapy, a natural history comparison group may be more appropriate, combined with solid outcome measures.

SFARI Investigator James McPartland of Yale University then underlined the need for objective biomarkers for clinical trials, for which there are currently none that are FDA qualified for ASD. As the director of the Autism Biomarkers Consortium for Clinical Trials (ABC-CT), he works with other scientists to develop reliable biomarkers that can be scaled for use in large samples across different sites. McPartland noted a biomarker studied in the ABC-CT: an event-related potential (N170) to human faces, which is on average slower in ASD than in typically developing children. He is working on ways to make it easier for people with ASD and intellectual disabilities to participate in biomarker studies and to make them more socially naturalistic. In discussion, he mentioned he thought it would be possible to look for these kinds of biomarkers in younger children.

SFARI Investigator Shafali Jeste of the University of California, Los Angeles recounted her experience in working with children with genetic syndromes associated with neurodevelopmental conditions. Though she is asked to participate in clinical trials for these conditions, she senses the field has some work to do to be ready for these trials, particularly in those with additional challenges such as epilepsy and intellectual disability. Meaningful and measurable clinical endpoints are still insufficient, and there needs to be more ways to improve accessibility of these trials for these rare conditions. This means developing new measures, such as gait-mat technology that senses walking coordination, or EEG measures in waking and sleep, which have been applied to people with chromosome 15q11.2-13.1 duplication (dup15q) syndrome, who have severe intellectual disability and motor impairments. Jeste also emphasized that increasing remote access to some measures can make a big difference for a trial; for example, a trial of a behavioral intervention for tuberous sclerosis complex that required weekly lab visits was disappointingly under-enrolled until researchers revamped it so most of the intervention could be done remotely31.

By grappling with the challenges to gene therapy for ASD, the workshop marked out a faint road map of a way forward. As the scientific questions are answered, the regulatory and clinical trial infrastructure will need to develop apace, and coordination between private, academic and advocacy sectors will be essential. But as gene therapy for diverse human conditions continues to be explored and gene discovery in ASD continues, there is reason to believe that some forms of ASD can eventually benefit from this strategy.This workshop provided a terrific discussion about the challenges in developing targeted gene interventions and their potentially transformative effects as therapies, said John Spiro, Deputy Scientific Director of SFARI. We are grateful to all theparticipants, and SFARI looks forward to translating these discussions into focused funding decisions in the near future.

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KENILWORTH, N.J.--(BUSINESS WIRE)--Merck (NYSE: MRK), known as MSD outside the United States and Canada, announced today that the U.S. Food and Drug Administration (FDA) has approved KEYTRUDA, Mercks anti-PD-1 therapy, as monotherapy for the treatment of patients with recurrent or metastatic cutaneous squamous cell carcinoma (cSCC) that is not curable by surgery or radiation. This approval is based on data from the Phase 2 KEYNOTE-629 trial, in which KEYTRUDA demonstrated meaningful efficacy and durability of response, with an objective response rate (ORR) of 34% (95% CI, 25-44), including a complete response rate of 4% and a partial response rate of 31%. Among responding patients, 69% had ongoing responses of six months or longer. After a median follow-up time of 9.5 months, the median duration of response (DOR) had not been reached (range, 2.7 to 13.1+ months).

Cutaneous squamous cell carcinoma is the second most common form of skin cancer, said Dr. Jonathan Cheng, vice president, clinical research, Merck Research Laboratories. In KEYNOTE-629, treatment with KEYTRUDA resulted in clinically meaningful and durable responses. Todays approval is great news for patients with cSCC and further demonstrates our commitment to bringing new treatment options to patients with advanced, difficult-to-treat cancers.

Immune-mediated adverse reactions, which may be severe or fatal, can occur with KEYTRUDA, including pneumonitis, colitis, hepatitis, endocrinopathies, nephritis and renal dysfunction, severe skin reactions, solid organ transplant rejection, and complications of allogeneic hematopoietic stem cell transplantation (HSCT). Based on the severity of the adverse reaction, KEYTRUDA should be withheld or discontinued and corticosteroids administered if appropriate. KEYTRUDA can also cause severe or life-threatening infusion-related reactions. Based on its mechanism of action, KEYTRUDA can cause fetal harm when administered to a pregnant woman. For more information, see Selected Important Safety Information below.

Data Supporting Approval

The efficacy of KEYTRUDA was investigated in patients with recurrent or metastatic cSCC enrolled in KEYNOTE-629 (NCT03284424), a multi-center, multi-cohort, non-randomized, open-label trial. The trial excluded patients with autoimmune disease or a medical condition that required immunosuppression. The major efficacy outcome measures were ORR and DOR as assessed by blinded independent central review (BICR) according to Response Evaluation Criteria in Solid Tumors (RECIST) v1.1, modified to follow a maximum of 10 target lesions and a maximum of five target lesions per organ.

Among the 105 patients treated, 87% received one or more prior lines of therapy and 74% received prior radiation therapy. Forty-five percent of patients had locally recurrent only cSCC, 24% had metastatic only cSCC and 31% had both locally recurrent and metastatic cSCC. The study population characteristics were: median age of 72 years (range, 29 to 95); 71% age 65 or older; 76% male; 71% White; 25% race unknown; 34% Eastern Cooperative Oncology Group (ECOG) Performance Status (PS) of 0 and 66% ECOG PS of 1.

KEYTRUDA demonstrated an ORR of 34% (95% CI, 25-44) with a complete response rate of 4% and a partial response rate of 31%. Among the 36 responding patients, 69% had ongoing responses of six months or longer. After a median follow-up time of 9.5 months, the median DOR had not been reached (range, 2.7 to 13.1+ months).

Patients received KEYTRUDA 200 mg intravenously every three weeks until documented disease progression, unacceptable toxicity or a maximum of 24 months. Patients with initial radiographic disease progression could receive additional doses of KEYTRUDA during confirmation of progression unless disease progression was symptomatic, rapidly progressive, required urgent intervention, or occurred with a decline in performance status. Assessment of tumor status was performed every six weeks during the first year and every nine weeks during the second year.

Among the 105 patients with cSCC enrolled in KEYNOTE-629, the median duration of exposure to KEYTRUDA was 5.8 months (range, 1 day to 16.1 months). Patients with autoimmune disease or a medical condition that required systemic corticosteroids or other immunosuppressive medications were ineligible. Adverse reactions occurring in patients with cSCC were similar to those occurring in 2,799 patients with melanoma or non-small cell lung cancer (NSCLC) treated with KEYTRUDA as a single agent. Laboratory abnormalities (Grades 3-4) that occurred at a higher incidence included lymphopenia (11%).

About KEYTRUDA (pembrolizumab) Injection, 100 mg

KEYTRUDA is an anti-PD-1 therapy that works by increasing the ability of the bodys immune system to help detect and fight tumor cells. KEYTRUDA is a humanized monoclonal antibody that blocks the interaction between PD-1 and its ligands, PD-L1 and PD-L2, thereby activating T lymphocytes which may affect both tumor cells and healthy cells.

Merck has the industrys largest immuno-oncology clinical research program. There are currently more than 1,200 trials studying KEYTRUDA across a wide variety of cancers and treatment settings. The KEYTRUDA clinical program seeks to understand the role of KEYTRUDA across cancers and the factors that may predict a patient's likelihood of benefitting from treatment with KEYTRUDA, including exploring several different biomarkers.

Selected KEYTRUDA (pembrolizumab) Indications

Melanoma

KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic melanoma.

KEYTRUDA is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph node(s) following complete resection.

Non-Small Cell Lung Cancer

KEYTRUDA, in combination with pemetrexed and platinum chemotherapy, is indicated for the first-line treatment of patients with metastatic nonsquamous non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.

KEYTRUDA, in combination with carboplatin and either paclitaxel or paclitaxel protein-bound, is indicated for the first-line treatment of patients with metastatic squamous NSCLC.

KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with NSCLC expressing PD-L1 [tumor proportion score (TPS) 1%] as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations, and is stage III where patients are not candidates for surgical resection or definitive chemoradiation, or metastatic.

KEYTRUDA, as a single agent, is indicated for the treatment of patients with metastatic NSCLC whose tumors express PD-L1 (TPS 1%) as determined by an FDA-approved test, with disease progression on or after platinum-containing chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving KEYTRUDA.

Small Cell Lung Cancer

KEYTRUDA is indicated for the treatment of patients with metastatic small cell lung cancer (SCLC) with disease progression on or after platinum-based chemotherapy and at least 1 other prior line of therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

Head and Neck Squamous Cell Cancer

KEYTRUDA, in combination with platinum and fluorouracil (FU), is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent head and neck squamous cell carcinoma (HNSCC).

KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent HNSCC whose tumors express PD-L1 [combined positive score (CPS) 1] as determined by an FDA-approved test.

KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent or metastatic head and neck squamous cell carcinoma (HNSCC) with disease progression on or after platinum-containing chemotherapy.

Classical Hodgkin Lymphoma

KEYTRUDA is indicated for the treatment of adult and pediatric patients with refractory classical Hodgkin lymphoma (cHL), or who have relapsed after 3 or more prior lines of therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Primary Mediastinal Large B-Cell Lymphoma

KEYTRUDA is indicated for the treatment of adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma (PMBCL), or who have relapsed after 2 or more prior lines of therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials. KEYTRUDA is not recommended for treatment of patients with PMBCL who require urgent cytoreductive therapy.

Urothelial Carcinoma

KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who are not eligible for cisplatin-containing chemotherapy and whose tumors express PD-L1 [combined positive score (CPS) 10], as determined by an FDA-approved test, or in patients who are not eligible for any platinum-containing chemotherapy regardless of PD-L1 status. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who have disease progression during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.

KEYTRUDA is indicated for the treatment of patients with Bacillus Calmette-Guerin (BCG)-unresponsive, high-risk, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors who are ineligible for or have elected not to undergo cystectomy.

Microsatellite Instability-High (MSI-H) Cancer

KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR)

This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with MSI-H central nervous system cancers have not been established.

Gastric Cancer

KEYTRUDA is indicated for the treatment of patients with recurrent locally advanced or metastatic gastric or gastroesophageal junction (GEJ) adenocarcinoma whose tumors express PD-L1 (CPS 1) as determined by an FDA-approved test, with disease progression on or after two or more prior lines of therapy including fluoropyrimidine- and platinum-containing chemotherapy and if appropriate, HER2/neu-targeted therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Esophageal Cancer

KEYTRUDA is indicated for the treatment of patients with recurrent locally advanced or metastatic squamous cell carcinoma of the esophagus whose tumors express PD-L1 (CPS 10) as determined by an FDA-approved test, with disease progression after one or more prior lines of systemic therapy.

Cervical Cancer

KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy whose tumors express PD-L1 (CPS 1) as determined by an FDA-approved test. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Hepatocellular Carcinoma

KEYTRUDA is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Merkel Cell Carcinoma

KEYTRUDA is indicated for the treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC). This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Renal Cell Carcinoma

KEYTRUDA, in combination with axitinib, is indicated for the first-line treatment of patients with advanced renal cell carcinoma (RCC).

Tumor Mutational Burden-High Cancer

KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic tumor mutational burden-high (TMB-H) [10 mutations/megabase (mut/Mb)] solid tumors, as determined by an FDA-approved test, that have progressed following prior treatment and who have no satisfactory alternative treatment options.

This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with TMB-H central nervous system cancers have not been established.

Cutaneous Squamous Cell Carcinoma

KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cutaneous squamous cell carcinoma (cSCC) that is not curable by surgery or radiation.

Selected Important Safety Information for KEYTRUDA

Immune-Mediated Pneumonitis

KEYTRUDA can cause immune-mediated pneumonitis, including fatal cases. Pneumonitis occurred in 3.4% (94/2799) of patients with various cancers receiving KEYTRUDA, including Grade 1 (0.8%), 2 (1.3%), 3 (0.9%), 4 (0.3%), and 5 (0.1%). Pneumonitis occurred in 8.2% (65/790) of NSCLC patients receiving KEYTRUDA as a single agent, including Grades 3-4 in 3.2% of patients, and occurred more frequently in patients with a history of prior thoracic radiation (17%) compared to those without (7.7%). Pneumonitis occurred in 6% (18/300) of HNSCC patients receiving KEYTRUDA as a single agent, including Grades 3-5 in 1.6% of patients, and occurred in 5.4% (15/276) of patients receiving KEYTRUDA in combination with platinum and FU as first-line therapy for advanced disease, including Grades 3-5 in 1.5% of patients.

Monitor patients for signs and symptoms of pneumonitis. Evaluate suspected pneumonitis with radiographic imaging. Administer corticosteroids for Grade 2 or greater pneumonitis. Withhold KEYTRUDA for Grade 2; permanently discontinue KEYTRUDA for Grade 3 or 4 or recurrent Grade 2 pneumonitis.

Immune-Mediated Colitis

KEYTRUDA can cause immune-mediated colitis. Colitis occurred in 1.7% (48/2799) of patients receiving KEYTRUDA, including Grade 2 (0.4%), 3 (1.1%), and 4 (<0.1%). Monitor patients for signs and symptoms of colitis. Administer corticosteroids for Grade 2 or greater colitis. Withhold KEYTRUDA for Grade 2 or 3; permanently discontinue KEYTRUDA for Grade 4 colitis.

Immune-Mediated Hepatitis (KEYTRUDA) and Hepatotoxicity (KEYTRUDA in Combination With Axitinib)

Immune-Mediated Hepatitis

KEYTRUDA can cause immune-mediated hepatitis. Hepatitis occurred in 0.7% (19/2799) of patients receiving KEYTRUDA, including Grade 2 (0.1%), 3 (0.4%), and 4 (<0.1%). Monitor patients for changes in liver function. Administer corticosteroids for Grade 2 or greater hepatitis and, based on severity of liver enzyme elevations, withhold or discontinue KEYTRUDA.

Hepatotoxicity in Combination With Axitinib

KEYTRUDA in combination with axitinib can cause hepatic toxicity with higher than expected frequencies of Grades 3 and 4 ALT and AST elevations compared to KEYTRUDA alone. With the combination of KEYTRUDA and axitinib, Grades 3 and 4 increased ALT (20%) and increased AST (13%) were seen. Monitor liver enzymes before initiation of and periodically throughout treatment. Consider more frequent monitoring of liver enzymes as compared to when the drugs are administered as single agents. For elevated liver enzymes, interrupt KEYTRUDA and axitinib, and consider administering corticosteroids as needed.

Immune-Mediated Endocrinopathies

KEYTRUDA can cause adrenal insufficiency (primary and secondary), hypophysitis, thyroid disorders, and type 1 diabetes mellitus. Adrenal insufficiency occurred in 0.8% (22/2799) of patients, including Grade 2 (0.3%), 3 (0.3%), and 4 (<0.1%). Hypophysitis occurred in 0.6% (17/2799) of patients, including Grade 2 (0.2%), 3 (0.3%), and 4 (<0.1%). Hypothyroidism occurred in 8.5% (237/2799) of patients, including Grade 2 (6.2%) and 3 (0.1%). The incidence of new or worsening hypothyroidism was higher in 1185 patients with HNSCC (16%) receiving KEYTRUDA, as a single agent or in combination with platinum and FU, including Grade 3 (0.3%) hypothyroidism. Hyperthyroidism occurred in 3.4% (96/2799) of patients, including Grade 2 (0.8%) and 3 (0.1%), and thyroiditis occurred in 0.6% (16/2799) of patients, including Grade 2 (0.3%). Type 1 diabetes mellitus, including diabetic ketoacidosis, occurred in 0.2% (6/2799) of patients.

Monitor patients for signs and symptoms of adrenal insufficiency, hypophysitis (including hypopituitarism), thyroid function (prior to and periodically during treatment), and hyperglycemia. For adrenal insufficiency or hypophysitis, administer corticosteroids and hormone replacement as clinically indicated. Withhold KEYTRUDA for Grade 2 adrenal insufficiency or hypophysitis and withhold or discontinue KEYTRUDA for Grade 3 or Grade 4 adrenal insufficiency or hypophysitis. Administer hormone replacement for hypothyroidism and manage hyperthyroidism with thionamides and beta-blockers as appropriate. Withhold or discontinue KEYTRUDA for Grade 3 or 4 hyperthyroidism. Administer insulin for type 1 diabetes, and withhold KEYTRUDA and administer antihyperglycemics in patients with severe hyperglycemia.

Immune-Mediated Nephritis and Renal Dysfunction

KEYTRUDA can cause immune-mediated nephritis. Nephritis occurred in 0.3% (9/2799) of patients receiving KEYTRUDA, including Grade 2 (0.1%), 3 (0.1%), and 4 (<0.1%) nephritis. Nephritis occurred in 1.7% (7/405) of patients receiving KEYTRUDA in combination with pemetrexed and platinum chemotherapy. Monitor patients for changes in renal function. Administer corticosteroids for Grade 2 or greater nephritis. Withhold KEYTRUDA for Grade 2; permanently discontinue for Grade 3 or 4 nephritis.

Immune-Mediated Skin Reactions

Immune-mediated rashes, including Stevens-Johnson syndrome (SJS), toxic epidermal necrolysis (TEN) (some cases with fatal outcome), exfoliative dermatitis, and bullous pemphigoid, can occur. Monitor patients for suspected severe skin reactions and based on the severity of the adverse reaction, withhold or permanently discontinue KEYTRUDA and administer corticosteroids. For signs or symptoms of SJS or TEN, withhold KEYTRUDA and refer the patient for specialized care for assessment and treatment. If SJS or TEN is confirmed, permanently discontinue KEYTRUDA.

Other Immune-Mediated Adverse Reactions

Immune-mediated adverse reactions, which may be severe or fatal, can occur in any organ system or tissue in patients receiving KEYTRUDA and may also occur after discontinuation of treatment. For suspected immune-mediated adverse reactions, ensure adequate evaluation to confirm etiology or exclude other causes. Based on the severity of the adverse reaction, withhold KEYTRUDA and administer corticosteroids. Upon improvement to Grade 1 or less, initiate corticosteroid taper and continue to taper over at least 1 month. Based on limited data from clinical studies in patients whose immune-related adverse reactions could not be controlled with corticosteroid use, administration of other systemic immunosuppressants can be considered. Resume KEYTRUDA when the adverse reaction remains at Grade 1 or less following corticosteroid taper. Permanently discontinue KEYTRUDA for any Grade 3 immune-mediated adverse reaction that recurs and for any life-threatening immune-mediated adverse reaction.

The following clinically significant immune-mediated adverse reactions occurred in less than 1% (unless otherwise indicated) of 2799 patients: arthritis (1.5%), uveitis, myositis, Guillain-Barr syndrome, myasthenia gravis, vasculitis, pancreatitis, hemolytic anemia, sarcoidosis, and encephalitis. In addition, myelitis and myocarditis were reported in other clinical trials, including classical Hodgkin lymphoma, and postmarketing use.

Treatment with KEYTRUDA may increase the risk of rejection in solid organ transplant recipients. Consider the benefit of treatment vs the risk of possible organ rejection in these patients.

Infusion-Related Reactions

KEYTRUDA can cause severe or life-threatening infusion-related reactions, including hypersensitivity and anaphylaxis, which have been reported in 0.2% (6/2799) of patients. Monitor patients for signs and symptoms of infusion-related reactions. For Grade 3 or 4 reactions, stop infusion and permanently discontinue KEYTRUDA.

Complications of Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)

Immune-mediated complications, including fatal events, occurred in patients who underwent allogeneic HSCT after treatment with KEYTRUDA. Of 23 patients with cHL who proceeded to allogeneic HSCT after KEYTRUDA, 6 (26%) developed graft-versus-host disease (GVHD) (1 fatal case) and 2 (9%) developed severe hepatic veno-occlusive disease (VOD) after reduced-intensity conditioning (1 fatal case). Cases of fatal hyperacute GVHD after allogeneic HSCT have also been reported in patients with lymphoma who received a PD-1 receptorblocking antibody before transplantation. Follow patients closely for early evidence of transplant-related complications such as hyperacute graft-versus-host disease (GVHD), Grade 3 to 4 acute GVHD, steroid-requiring febrile syndrome, hepatic veno-occlusive disease (VOD), and other immune-mediated adverse reactions.

In patients with a history of allogeneic HSCT, acute GVHD (including fatal GVHD) has been reported after treatment with KEYTRUDA. Patients who experienced GVHD after their transplant procedure may be at increased risk for GVHD after KEYTRUDA. Consider the benefit of KEYTRUDA vs the risk of GVHD in these patients.

Increased Mortality in Patients With Multiple Myeloma

In trials in patients with multiple myeloma, the addition of KEYTRUDA to a thalidomide analogue plus dexamethasone resulted in increased mortality. Treatment of these patients with a PD-1 or PD-L1 blocking antibody in this combination is not recommended outside of controlled trials.

Embryofetal Toxicity

Based on its mechanism of action, KEYTRUDA can cause fetal harm when administered to a pregnant woman. Advise women of this potential risk. In females of reproductive potential, verify pregnancy status prior to initiating KEYTRUDA and advise them to use effective contraception during treatment and for 4 months after the last dose.

Adverse Reactions

In KEYNOTE-006, KEYTRUDA was discontinued due to adverse reactions in 9% of 555 patients with advanced melanoma; adverse reactions leading to permanent discontinuation in more than one patient were colitis (1.4%), autoimmune hepatitis (0.7%), allergic reaction (0.4%), polyneuropathy (0.4%), and cardiac failure (0.4%). The most common adverse reactions (20%) with KEYTRUDA were fatigue (28%), diarrhea (26%), rash (24%), and nausea (21%).

In KEYNOTE-002, KEYTRUDA was permanently discontinued due to adverse reactions in 12% of 357 patients with advanced melanoma; the most common (1%) were general physical health deterioration (1%), asthenia (1%), dyspnea (1%), pneumonitis (1%), and generalized edema (1%). The most common adverse reactions were fatigue (43%), pruritus (28%), rash (24%), constipation (22%), nausea (22%), diarrhea (20%), and decreased appetite (20%).

In KEYNOTE-054, KEYTRUDA was permanently discontinued due to adverse reactions in 14% of 509 patients; the most common (1%) were pneumonitis (1.4%), colitis (1.2%), and diarrhea (1%). Serious adverse reactions occurred in 25% of patients receiving KEYTRUDA. The most common adverse reaction (20%) with KEYTRUDA was diarrhea (28%).

In KEYNOTE-189, when KEYTRUDA was administered with pemetrexed and platinum chemotherapy in metastatic nonsquamous NSCLC, KEYTRUDA was discontinued due to adverse reactions in 20% of 405 patients. The most common adverse reactions resulting in permanent discontinuation of KEYTRUDA were pneumonitis (3%) and acute kidney injury (2%). The most common adverse reactions (20%) with KEYTRUDA were nausea (56%), fatigue (56%), constipation (35%), diarrhea (31%), decreased appetite (28%), rash (25%), vomiting (24%), cough (21%), dyspnea (21%), and pyrexia (20%).

In KEYNOTE-407, when KEYTRUDA was administered with carboplatin and either paclitaxel or paclitaxel protein-bound in metastatic squamous NSCLC, KEYTRUDA was discontinued due to adverse reactions in 15% of 101 patients. The most frequent serious adverse reactions reported in at least 2% of patients were febrile neutropenia, pneumonia, and urinary tract infection. Adverse reactions observed in KEYNOTE-407 were similar to those observed in KEYNOTE-189 with the exception that increased incidences of alopecia (47% vs 36%) and peripheral neuropathy (31% vs 25%) were observed in the KEYTRUDA and chemotherapy arm compared to the placebo and chemotherapy arm in KEYNOTE-407.

In KEYNOTE-042, KEYTRUDA was discontinued due to adverse reactions in 19% of 636 patients with advanced NSCLC; the most common were pneumonitis (3%), death due to unknown cause (1.6%), and pneumonia (1.4%). The most frequent serious adverse reactions reported in at least 2% of patients were pneumonia (7%), pneumonitis (3.9%), pulmonary embolism (2.4%), and pleural effusion (2.2%). The most common adverse reaction (20%) was fatigue (25%).

In KEYNOTE-010, KEYTRUDA monotherapy was discontinued due to adverse reactions in 8% of 682 patients with metastatic NSCLC; the most common was pneumonitis (1.8%). The most common adverse reactions (20%) were decreased appetite (25%), fatigue (25%), dyspnea (23%), and nausea (20%).

Adverse reactions occurring in patients with SCLC were similar to those occurring in patients with other solid tumors who received KEYTRUDA as a single agent.

In KEYNOTE-048, KEYTRUDA monotherapy was discontinued due to adverse events in 12% of 300 patients with HNSCC; the most common adverse reactions leading to permanent discontinuation were sepsis (1.7%) and pneumonia (1.3%). The most common adverse reactions (20%) were fatigue (33%), constipation (20%), and rash (20%).

In KEYNOTE-048, when KEYTRUDA was administered in combination with platinum (cisplatin or carboplatin) and FU chemotherapy, KEYTRUDA was discontinued due to adverse reactions in 16% of 276 patients with HNSCC. The most common adverse reactions resulting in permanent discontinuation of KEYTRUDA were pneumonia (2.5%), pneumonitis (1.8%), and septic shock (1.4%). The most common adverse reactions (20%) were nausea (51%), fatigue (49%), constipation (37%), vomiting (32%), mucosal inflammation (31%), diarrhea (29%), decreased appetite (29%), stomatitis (26%), and cough (22%).

In KEYNOTE-012, KEYTRUDA was discontinued due to adverse reactions in 17% of 192 patients with HNSCC. Serious adverse reactions occurred in 45% of patients. The most frequent serious adverse reactions reported in at least 2% of patients were pneumonia, dyspnea, confusional state, vomiting, pleural effusion, and respiratory failure. The most common adverse reactions (20%) were fatigue, decreased appetite, and dyspnea. Adverse reactions occurring in patients with HNSCC were generally similar to those occurring in patients with melanoma or NSCLC who received KEYTRUDA as a monotherapy, with the exception of increased incidences of facial edema and new or worsening hypothyroidism.

In KEYNOTE-087, KEYTRUDA was discontinued due to adverse reactions in 5% of 210 patients with cHL. Serious adverse reactions occurred in 16% of patients; those 1% included pneumonia, pneumonitis, pyrexia, dyspnea, GVHD, and herpes zoster. Two patients died from causes other than disease progression; 1 from GVHD after subsequent allogeneic HSCT and 1 from septic shock. The most common adverse reactions (20%) were fatigue (26%), pyrexia (24%), cough (24%), musculoskeletal pain (21%), diarrhea (20%), and rash (20%).

In KEYNOTE-170, KEYTRUDA was discontinued due to adverse reactions in 8% of 53 patients with PMBCL. Serious adverse reactions occurred in 26% of patients and included arrhythmia (4%), cardiac tamponade (2%), myocardial infarction (2%), pericardial effusion (2%), and pericarditis (2%). Six (11%) patients died within 30 days of start of treatment. The most common adverse reactions (20%) were musculoskeletal pain (30%), upper respiratory tract infection and pyrexia (28% each), cough (26%), fatigue (23%), and dyspnea (21%).

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FDA Approves Merck's KEYTRUDA (pembrolizumab) for the Treatment of Patients with Recurrent or Metastatic Cutaneous Squamous Cell Carcinoma (cSCC) that...

Cardiac Stem Cells Comments Off on FDA Approves Merck’s KEYTRUDA (pembrolizumab) for the Treatment of Patients with Recurrent or Metastatic Cutaneous Squamous Cell Carcinoma (cSCC) that… | June 25th, 2020

Researchers have known for quite some time that HD causes a progressive loss of neurons. But what if we could find a way to fill their place? In a new report, researchers used an intriguing strategy in living mice to do just that they converted a different type of brain cell into neurons, with very promising results.

In HD research we spend a lot of time talking about neurons. And understandably so! Neurons are the cell type in the brain most affected by HD, and they are the cells that exchange messages to drive our movements, moods, and memories. You can think of neurons like the computer programmers of the brain they convert information into action.

In particular, neurons in an area of the brain called the striatum striatal neurons tend to be most vulnerable to the mutation that causes HD. Right now no one knows exactly why those cells are especially vulnerable. But researchers know that many of the symptoms of HD are related to the loss of neurons in this area of the brain.

But there are lots of different types of cells in the brain. In fact, the most abundant cell type in the brain isnt neurons its a cell type called glia. Glia is a general term that describes several kinds of cells in the brain and spinal cord that provide support, insulation, and protection. You can think of glia like the body guard of the brain they make sure other cell types have the support they need to function.

One type of glia are brain cells called astrocytes. A lot of the nervous system is made up of astrocytes 30% in fact! Because astrocytes are everywhere in the brain, theyre also present in the areas where neurons degenerate due to HD the striatum. And unlike neurons that stop dividing when theyre fully mature, glia continue to divide.

Recently, scientists took advantage of the abundance of glia in the brain and their ability to reproduce. They used an experimental technique in the brains of mice to turn astrocytes into new, functioning neurons. So to stick with our analogy, they encouraged the body guards of the brain to change jobs and become computer programmers.

The work was led by Dr. Gong Chen, a former professor at Penn State University, who is now leading the Institute of CNS Regeneration at Jinan University in China. His team took advantage of a technique to turn cells that arent neurons into neurons something called direct conversion.

This technique allows researchers to coax different cell types, such as astrocytes, into becoming neurons, by adding chemical cocktails to boost the action of genes that influence a cells role. This is a bit like changing the job description of a certain cell type - but this has been done before. Many times in fact. Its old news that scientists can take one cell type grown in a laboratory dish and directly turn it into a neuron.

So what did this report add, and why was it worthy of publication in the prestigious journal Nature Communications? Because these authors did direct conversion inside the brains of living mice! They used a harmless virus to deliver their chemical cocktail that gave a genetic nudge to the astrocytes, encouraging them to change jobs and become neurons. In this way, they were able to turn abundant astrocytes into potentially valuable striatal neurons a very cool accomplishment!

We know what you may be thinking Did you just say virus?! We all get a little weary when we hear that word, especially in the days of COVID-19! But rest assured, this is a very harmless method used frequently in biology.

Its actually just the outside of the virus thats used, without any of the inside bits that typically make viruses so harmful. Similar to a letter in an envelope researchers here are repurposing an envelope and adding something new inside. So the old message is removed, and the envelope is sent with new instructions that body guards should change jobs and become computer programmers!

An important finding from the paper was that the overall number of astrocytes didnt decrease over time. This is related to the point we made above about astrocytes they continue to divide. So even though the researchers turned some of the astrocytes into neurons, the astrocytes that remained produced more astrocytes to replace them. This approach provided a source of new striatal neurons for these HD mice without affecting the astrocyte population! And because these astrocytes are already located in the striatum, the intervention occurs in the exact area of the brain that could use more neurons.

Chen and colleagues also showed that these new neurons in the striatum fired signals just like native neurons. They also connected with other areas of the brain, just like native neurons. Most compellingly, with the addition of these new neurons in the striatum, the HD mice performed better on movement tests and had an extended lifespan. All very exciting and promising results!

The idea of adding back lost neurons in HD isnt new. The big difference is that previous studies have added new cells through surgery, performing whats called cell transplantation. So while direct conversions, like the experiments performed by Chen and his team, are like changing jobs within the same company, cell transplantations are like getting a job at a new company.

Several research groups have experimented with cell transplantation as a therapy for HD, and some of these options are moving toward clinical trials. More recently, cell transplantations have been done with immature cells known as stem cells or neural progenitor cells that havent fully committed to becoming a specific cell type yet. The benefit of using immature cells is that they can obtain cues from the surrounding environment, letting them know what cell type is needed.

Cell transplantations have shown promise, but can come with some risks. Theres no guarantee that the cells will become exactly the type of neuron you want. And theres no guarantee that the cells will survive long-term because thats not their native environment.

Chens group got around these issues by triggering specific biological machinery to convert astrocytes into striatal neurons. The researchers knew exactly what type of neuron they were going to get in the end. And because the astrocytes they targeted were already present in the striatum, they knew the new neurons would be in exactly the right place!

One thing to keep in mind with this approach is that the astrocytes used to make the neurons come from the HD mouse. That means the new striatal neurons also contain the genetic error (mutation) that causes HD. Researchers dont yet know what that means for the lifespan of those neurons.

While the results from this study are very exciting and potentially provide another tool in our belt to combat HD, this study was done as a proof-of-concept and still has a long way to go before it reaches the clinic. But so far, even though the new neurons carry the HD mutation, the direct conversion technique seems to improve HD-related symptoms in the mice.

Follow up studies are likely to try this technique in larger animals or to test it in combination with huntingtin lowering, which will undoubtedly provide interesting results. Well be eagerly waiting!

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Changing jobs: converting other cell types into neurons - HDBuzz

Spinal Cord Stem Cells Comments Off on Changing jobs: converting other cell types into neurons – HDBuzz | June 24th, 2020

Stem Cell Assay Market: Snapshot

Stem cell assay refers to the procedure of measuring the potency of antineoplastic drugs, on the basis of their capability of retarding the growth of human tumor cells. The assay consists of qualitative or quantitative analysis or testing of affected tissues andtumors, wherein their toxicity, impurity, and other aspects are studied.

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With the growing number of successfulstem cell therapytreatment cases, the global market for stem cell assays will gain substantial momentum. A number of research and development projects are lending a hand to the growth of the market. For instance, the University of Washingtons Institute for Stem Cell and Regenerative Medicine (ISCRM) has attempted to manipulate stem cells to heal eye, kidney, and heart injuries. A number of diseases such as Alzheimers, spinal cord injury, Parkinsons, diabetes, stroke, retinal disease, cancer, rheumatoid arthritis, and neurological diseases can be successfully treated via stem cell therapy. Therefore, stem cell assays will exhibit growing demand.

Another key development in the stem cell assay market is the development of innovative stem cell therapies. In April 2017, for instance, the first participant in an innovative clinical trial at the University of Wisconsin School of Medicine and Public Health was successfully treated with stem cell therapy. CardiAMP, the investigational therapy, has been designed to direct a large dose of the patients own bone-marrow cells to the point of cardiac injury, stimulating the natural healing response of the body.

Newer areas of application in medicine are being explored constantly. Consequently, stem cell assays are likely to play a key role in the formulation of treatments of a number of diseases.

Global Stem Cell Assay Market: Overview

The increasing investment in research and development of novel therapeutics owing to the rising incidence of chronic diseases has led to immense growth in the global stem cell assay market. In the next couple of years, the market is expected to spawn into a multi-billion dollar industry as healthcare sector and governments around the world increase their research spending.

The report analyzes the prevalent opportunities for the markets growth and those that companies should capitalize in the near future to strengthen their position in the market. It presents insights into the growth drivers and lists down the major restraints. Additionally, the report gauges the effect of Porters five forces on the overall stem cell assay market.

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Global Stem Cell Assay Market: Key Market Segments

For the purpose of the study, the report segments the global stem cell assay market based on various parameters. For instance, in terms of assay type, the market can be segmented into isolation and purification, viability, cell identification, differentiation, proliferation, apoptosis, and function. By kit, the market can be bifurcated into human embryonic stem cell kits and adult stem cell kits. Based on instruments, flow cytometer, cell imaging systems, automated cell counter, and micro electrode arrays could be the key market segments.

In terms of application, the market can be segmented into drug discovery and development, clinical research, and regenerative medicine and therapy. The growth witnessed across the aforementioned application segments will be influenced by the increasing incidence of chronic ailments which will translate into the rising demand for regenerative medicines. Finally, based on end users, research institutes and industry research constitute the key market segments.

The report includes a detailed assessment of the various factors influencing the markets expansion across its key segments. The ones holding the most lucrative prospects are analyzed, and the factors restraining its trajectory across key segments are also discussed at length.

Global Stem Cell Assay Market: Regional Analysis

Regionally, the market is expected to witness heightened demand in the developed countries across Europe and North America. The increasing incidence of chronic ailments and the subsequently expanding patient population are the chief drivers of the stem cell assay market in North America. Besides this, the market is also expected to witness lucrative opportunities in Asia Pacific and Rest of the World.

Global Stem Cell Assay Market: Vendor Landscape

A major inclusion in the report is the detailed assessment of the markets vendor landscape. For the purpose of the study the report therefore profiles some of the leading players having influence on the overall market dynamics. It also conducts SWOT analysis to study the strengths and weaknesses of the companies profiled and identify threats and opportunities that these enterprises are forecast to witness over the course of the reports forecast period.

Some of the most prominent enterprises operating in the global stem cell assay market are Bio-Rad Laboratories, Inc (U.S.), Thermo Fisher Scientific Inc. (U.S.), GE Healthcare (U.K.), Hemogenix Inc. (U.S.), Promega Corporation (U.S.), Bio-Techne Corporation (U.S.), Merck KGaA (Germany), STEMCELL Technologies Inc. (CA), Cell Biolabs, Inc. (U.S.), and Cellular Dynamics International, Inc. (U.S.).

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TMR Research is a premier provider of customized market research and consulting services to business entities keen on succeeding in todays supercharged economic climate. Armed with an experienced, dedicated, and dynamic team of analysts, we are redefining the way our clients conduct business by providing them with authoritative and trusted research studies in tune with the latest methodologies and market trends.

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Stem Cell Assay Market In-Depth Analysis and Forecast 2017-2025 - Daily Veterans

Spinal Cord Stem Cells Comments Off on Stem Cell Assay Market In-Depth Analysis and Forecast 2017-2025 – Daily Veterans | June 24th, 2020

The Autologous Stem Cell Based Therapies Market globally is a standout amongst the most emergent and astoundingly approved sectors. This worldwide market has been developing at a higher pace with the development of imaginative frameworks and a developing end-client tendency.

Autologous Stem Cell Based Therapies market reports deliver insight and expert analysis into key consumer trends and behaviour in marketplace, in addition to an overview of the market data and key brands. Autologous Stem Cell Based Therapies market reports provides all data with easily digestible information to guide every businessmans future innovation and move business forward.

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The worldwide Autologous Stem Cell Based Therapies market is an enlarging field for top market players,

The key players covered in this studyRegeneusMesoblastPluristem Therapeutics IncU.S. STEM CELL, INC.Brainstorm Cell TherapeuticsTigenixMed cell Europe

Market segment by Type, the product can be split intoEmbryonic Stem CellResident Cardiac Stem CellsUmbilical Cord Blood Stem Cells

Market segment by Application, split intoNeurodegenerative DisordersAutoimmune DiseasesCardiovascular Diseases

Market segment by Regions/Countries, this report coversUnited StatesEuropeChinaJapanSoutheast AsiaIndiaCentral & South America

The study objectives of this report are:To analyze global Autologous Stem Cell Based Therapies status, future forecast, growth opportunity, key market and key players.To present the Autologous Stem Cell Based Therapies development in United States, Europe and China.To strategically profile the key players and comprehensively analyze their development plan and strategies.To define, describe and forecast the market by product type, market and key regions.

In this study, the years considered to estimate the market size of Autologous Stem Cell Based Therapies are as follows:History Year: 2014-2018Base Year: 2018Estimated Year: 2019Forecast Year 2019 to 2025For the data information by region, company, type and application, 2018 is considered as the base year. Whenever data information was unavailable for the base year, the prior year has been considered.

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This Autologous Stem Cell Based Therapies report begins with a basic overview of the market. The analysis highlights the opportunity and Autologous Stem Cell Based Therapies industry trends that are impacted the market that is global. Players around various regions and analysis of each industry dimensions are covered under this report. The analysis also contains a crucial Autologous Stem Cell Based Therapies insight regarding the things which are driving and affecting the earnings of the market. The Autologous Stem Cell Based Therapies report comprises sections together side landscape which clarifies actions such as venture and acquisitions and mergers.

The Report offers SWOT examination and venture return investigation, and other aspects such as the principle locale, economic situations with benefit, generation, request, limit, supply, and market development rate and figure.

Quantifiable data:-

Geographically, this report studies the top producers and consumers, focuses on product capacity, production, value, consumption, market share and growth opportunity in these key regions, covering North America, Europe, China, Japan, Southeast Asia, India

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Finally, the global Autologous Stem Cell Based Therapies market provides a total research decision and also sector feasibility of investment in new projects will be assessed. Autologous Stem Cell Based Therapies industry is a source of means and guidance for organizations and individuals interested in their market earnings.

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Insight on the Growth of Autologous Stem Cell Based Therapies Market Growth with Challenges, Standardization, Competitive Market Share and Top Players...

Cardiac Stem Cells Comments Off on Insight on the Growth of Autologous Stem Cell Based Therapies Market Growth with Challenges, Standardization, Competitive Market Share and Top Players… | June 24th, 2020

LOS ANGELES, United States: QY Research has recently published a report, titled Global Autologous Stem Cell Based Therapies Market Size, Status and Forecast 2020-2026. The market research report is a brilliant, complete, and much-needed resource for companies, stakeholders, and investors interested in the global Autologous Stem Cell Based Therapies market. It informs readers about key trends and opportunities in the global Autologous Stem Cell Based Therapies market along with critical market dynamics expected to impact the global market growth. It offers a range of market analysis studies, including production and consumption, sales, industry value chain, competitive landscape, regional growth, and price. On the whole, it comes out as an intelligent resource that companies can use to gain a competitive advantage in the global Autologous Stem Cell Based Therapies market.

Key companies operating in the global Autologous Stem Cell Based Therapies market include , Regeneus, Mesoblast, Pluristem Therapeutics Inc, US STEM CELL, INC., Brainstorm Cell Therapeutics, Tigenix, Med cell Europe, Autologous Stem Cell Based Therapies

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Segmental Analysis

Both developed and emerging regions are deeply studied by the authors of the report. The regional analysis section of the report offers a comprehensive analysis of the global Autologous Stem Cell Based Therapies market on the basis of region. Each region is exhaustively researched about so that players can use the analysis to tap into unexplored markets and plan powerful strategies to gain a foothold in lucrative markets.

Global Autologous Stem Cell Based Therapies Market Segment By Type:

, Embryonic Stem Cell, Resident Cardiac Stem Cells, Umbilical Cord Blood Stem Cells Autologous Stem Cell Based Therapies

Global Autologous Stem Cell Based Therapies Market Segment By Application:

, Neurodegenerative Disorders, Autoimmune Diseases, Cardiovascular Diseases

Competitive Landscape

Competitor analysis is one of the best sections of the report that compares the progress of leading players based on crucial parameters, including market share, new developments, global reach, local competition, price, and production. From the nature of competition to future changes in the vendor landscape, the report provides in-depth analysis of the competition in the global Autologous Stem Cell Based Therapies market.

Key companies operating in the global Autologous Stem Cell Based Therapies market include , Regeneus, Mesoblast, Pluristem Therapeutics Inc, US STEM CELL, INC., Brainstorm Cell Therapeutics, Tigenix, Med cell Europe, Autologous Stem Cell Based Therapies

Key questions answered in the report:

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TOC

1 Report Overview1.1 Study Scope1.2 Key Market Segments1.3 Players Covered: Ranking by Autologous Stem Cell Based Therapies Revenue1.4 Market by Type1.4.1 Global Autologous Stem Cell Based Therapies Market Size Growth Rate by Type: 2020 VS 20261.4.2 Embryonic Stem Cell1.4.3 Resident Cardiac Stem Cells1.4.4 Umbilical Cord Blood Stem Cells1.5 Market by Application1.5.1 Global Autologous Stem Cell Based Therapies Market Share by Application: 2020 VS 20261.5.2 Neurodegenerative Disorders1.5.3 Autoimmune Diseases1.5.4 Cardiovascular Diseases1.6 Study Objectives1.7 Years Considered 2 Global Growth Trends2.1 Global Autologous Stem Cell Based Therapies Market Perspective (2015-2026)2.2 Global Autologous Stem Cell Based Therapies Growth Trends by Regions2.2.1 Autologous Stem Cell Based Therapies Market Size by Regions: 2015 VS 2020 VS 20262.2.2 Autologous Stem Cell Based Therapies Historic Market Share by Regions (2015-2020)2.2.3 Autologous Stem Cell Based Therapies Forecasted Market Size by Regions (2021-2026)2.3 Industry Trends and Growth Strategy2.3.1 Market Top Trends2.3.2 Market Drivers2.3.3 Market Challenges2.3.4 Porters Five Forces Analysis2.3.5 Autologous Stem Cell Based Therapies Market Growth Strategy2.3.6 Primary Interviews with Key Autologous Stem Cell Based Therapies Players (Opinion Leaders) 3 Competition Landscape by Key Players3.1 Global Top Autologous Stem Cell Based Therapies Players by Market Size3.1.1 Global Top Autologous Stem Cell Based Therapies Players by Revenue (2015-2020)3.1.2 Global Autologous Stem Cell Based Therapies Revenue Market Share by Players (2015-2020)3.1.3 Global Autologous Stem Cell Based Therapies Market Share by Company Type (Tier 1, Tier 2 and Tier 3)3.2 Global Autologous Stem Cell Based Therapies Market Concentration Ratio3.2.1 Global Autologous Stem Cell Based Therapies Market Concentration Ratio (CR5 and HHI)3.2.2 Global Top 10 and Top 5 Companies by Autologous Stem Cell Based Therapies Revenue in 20193.3 Autologous Stem Cell Based Therapies Key Players Head office and Area Served3.4 Key Players Autologous Stem Cell Based Therapies Product Solution and Service3.5 Date of Enter into Autologous Stem Cell Based Therapies Market3.6 Mergers & Acquisitions, Expansion Plans 4 Market Size by Type (2015-2026)4.1 Global Autologous Stem Cell Based Therapies Historic Market Size by Type (2015-2020)4.2 Global Autologous Stem Cell Based Therapies Forecasted Market Size by Type (2021-2026) 5 Market Size by Application (2015-2026)5.1 Global Autologous Stem Cell Based Therapies Market Size by Application (2015-2020)5.2 Global Autologous Stem Cell Based Therapies Forecasted Market Size by Application (2021-2026) 6 North America6.1 North America Autologous Stem Cell Based Therapies Market Size (2015-2020)6.2 Autologous Stem Cell Based Therapies Key Players in North America (2019-2020)6.3 North America Autologous Stem Cell Based Therapies Market Size by Type (2015-2020)6.4 North America Autologous Stem Cell Based Therapies Market Size by Application (2015-2020) 7 Europe7.1 Europe Autologous Stem Cell Based Therapies Market Size (2015-2020)7.2 Autologous Stem Cell Based Therapies Key Players in Europe (2019-2020)7.3 Europe Autologous Stem Cell Based Therapies Market Size by Type (2015-2020)7.4 Europe Autologous Stem Cell Based Therapies Market Size by Application (2015-2020) 8 China8.1 China Autologous Stem Cell Based Therapies Market Size (2015-2020)8.2 Autologous Stem Cell Based Therapies Key Players in China (2019-2020)8.3 China Autologous Stem Cell Based Therapies Market Size by Type (2015-2020)8.4 China Autologous Stem Cell Based Therapies Market Size by Application (2015-2020) 9 Japan9.1 Japan Autologous Stem Cell Based Therapies Market Size (2015-2020)9.2 Autologous Stem Cell Based Therapies Key Players in Japan (2019-2020)9.3 Japan Autologous Stem Cell Based Therapies Market Size by Type (2015-2020)9.4 Japan Autologous Stem Cell Based Therapies Market Size by Application (2015-2020) 10 Southeast Asia10.1 Southeast Asia Autologous Stem Cell Based Therapies Market Size (2015-2020)10.2 Autologous Stem Cell Based Therapies Key Players in Southeast Asia (2019-2020)10.3 Southeast Asia Autologous Stem Cell Based Therapies Market Size by Type (2015-2020)10.4 Southeast Asia Autologous Stem Cell Based Therapies Market Size by Application (2015-2020) 11 India11.1 India Autologous Stem Cell Based Therapies Market Size (2015-2020)11.2 Autologous Stem Cell Based Therapies Key Players in India (2019-2020)11.3 India Autologous Stem Cell Based Therapies Market Size by Type (2015-2020)11.4 India Autologous Stem Cell Based Therapies Market Size by Application (2015-2020) 12 Central & South America12.1 Central & South America Autologous Stem Cell Based Therapies Market Size (2015-2020)12.2 Autologous Stem Cell Based Therapies Key Players in Central & South America (2019-2020)12.3 Central & South America Autologous Stem Cell Based Therapies Market Size by Type (2015-2020)12.4 Central & South America Autologous Stem Cell Based Therapies Market Size by Application (2015-2020) 13 Key Players Profiles13.1 Regeneus13.1.1 Regeneus Company Details13.1.2 Regeneus Business Overview13.1.3 Regeneus Autologous Stem Cell Based Therapies Introduction13.1.4 Regeneus Revenue in Autologous Stem Cell Based Therapies Business (2015-2020))13.1.5 Regeneus Recent Development13.2 Mesoblast13.2.1 Mesoblast Company Details13.2.2 Mesoblast Business Overview13.2.3 Mesoblast Autologous Stem Cell Based Therapies Introduction13.2.4 Mesoblast Revenue in Autologous Stem Cell Based Therapies Business (2015-2020)13.2.5 Mesoblast Recent Development13.3 Pluristem Therapeutics Inc13.3.1 Pluristem Therapeutics Inc Company Details13.3.2 Pluristem Therapeutics Inc Business Overview13.3.3 Pluristem Therapeutics Inc Autologous Stem Cell Based Therapies Introduction13.3.4 Pluristem Therapeutics Inc Revenue in Autologous Stem Cell Based Therapies Business (2015-2020)13.3.5 Pluristem Therapeutics Inc Recent Development13.4 US STEM CELL, INC.13.4.1 US STEM CELL, INC. Company Details13.4.2 US STEM CELL, INC. Business Overview13.4.3 US STEM CELL, INC. Autologous Stem Cell Based Therapies Introduction13.4.4 US STEM CELL, INC. Revenue in Autologous Stem Cell Based Therapies Business (2015-2020)13.4.5 US STEM CELL, INC. Recent Development13.5 Brainstorm Cell Therapeutics13.5.1 Brainstorm Cell Therapeutics Company Details13.5.2 Brainstorm Cell Therapeutics Business Overview13.5.3 Brainstorm Cell Therapeutics Autologous Stem Cell Based Therapies Introduction13.5.4 Brainstorm Cell Therapeutics Revenue in Autologous Stem Cell Based Therapies Business (2015-2020)13.5.5 Brainstorm Cell Therapeutics Recent Development13.6 Tigenix13.6.1 Tigenix Company Details13.6.2 Tigenix Business Overview13.6.3 Tigenix Autologous Stem Cell Based Therapies Introduction13.6.4 Tigenix Revenue in Autologous Stem Cell Based Therapies Business (2015-2020)13.6.5 Tigenix Recent Development13.7 Med cell Europe13.7.1 Med cell Europe Company Details13.7.2 Med cell Europe Business Overview13.7.3 Med cell Europe Autologous Stem Cell Based Therapies Introduction13.7.4 Med cell Europe Revenue in Autologous Stem Cell Based Therapies Business (2015-2020)13.7.5 Med cell Europe Recent Development 14 Analysts Viewpoints/Conclusions 15 Appendix15.1 Research Methodology15.1.1 Methodology/Research Approach15.1.2 Data Source15.2 Disclaimer15.3 Author Details

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Trending: Autologous Stem Cell Based Therapies 2020: Global Size, Supply-Demand, Product Type and End User Analysis To 2026 - Weekly Wall

Cardiac Stem Cells Comments Off on Trending: Autologous Stem Cell Based Therapies 2020: Global Size, Supply-Demand, Product Type and End User Analysis To 2026 – Weekly Wall | June 24th, 2020

The world is increasingly becoming aware of the various kinds of damages that the SARS-CoV-2 can cause. However, researchers from Virginia Tech have found that the relatively harmless Adenovirus can cause heart conditions, which can be as life-threatening as the one induced by COVID-19.

According to the first-of-its-kind study, adenovirus can hamper the electrical signaling pathways between cells in the heart and also impair the ability of the cell to make new communication channels. The scientists exposed heart cells to the virus and learned of the potent effects it had on them.

"This is the first time we're putting this human virus on human heart cells to see what it does in the context of infected heart muscle cells. That's the real power of this," James Smyth, lead author of the study, said.

Adenoviruses belong to a class of common viruses that cause infections in the lining of the lungs, eyes, nervous system, and urinary tract. They often give rise to coughs, fever, pink eye, and sore throats, among others. While it generally affects children, all are prone to it.

The communication between heart muscles takes place through channels called gap junctions. They are formed by proteins known as connexins. Creating a bridge between two cells, gap junctions leads to the sharing of electrical signals that aid in the rhythmic contraction of the heart muscle cells. However, gap junctions can also alert neighboring cells about viral attacks.

Through the study, the researchers intended to demonstrate that the virus hijacks gap junctions, and when it does, it can decrease the production of connexin43(a component of a gap function). This in turn interrupts the electrical system that enables regular functioning of the heart, leading to arrhythmias (irregular heartbeat), and in extreme cases, cardiac death.

The researchers designed a diagnostic technique that employed pluripotent stem cell derived-cardiomyocytes, which are skin cells that have been made to convert to heart cells. The adenovirus was then applied to the cardiomyocytes and the resulting interactions were observed.

As expected, the virus hijacked the gap junctions in order to facilitate its own replication. However, the scientists also observed something that they had not anticipated. It was noted that two distinct processes were being carried out by the virus and that it inflicted dual damage to the cell's capacity to communicate with their neighbors. "Firstly, it was rapidly closing existing channels, and secondly it was shutting down the cells' ability to make new ones," explained Patrick Calhoun, co-author of the study.

Another aspect that caught the eye of the authors was the manner in which the virus prevented the creation of connexin43 and the formation of gap junctions. A protein pathway that is conventionally associated with the making of fresh connexin, was instead made to suppress its production by the virus. "We might learn something very new about the molecular biology there that's causing that switch," Smyth said

Smyth admits that the research is bound by the limitations of extending the results to a living heart while the experiment was conducted in vitro. However, highlighting the potential value of the findings, he asserted, "Fundamental studies provide the footing for the translational research that discovers therapeutics and diagnostic methods that improve people's health."

Going beyond the sheer understanding of viral infection, the research, Calhoun emphasized, can generate new therapeutic interventions for diseased hearts. "We're essentially learning from adenovirus to find the most efficient ways to stop, rather than cause, arrhythmias," he stressed.

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Scientists Find This Relatively Harmless Virus Can Attack and Damage Human Heart - International Business Times, Singapore Edition

Cardiac Stem Cells Comments Off on Scientists Find This Relatively Harmless Virus Can Attack and Damage Human Heart – International Business Times, Singapore Edition | June 24th, 2020

Bone marrowaspiration and trephine biopsy are usually performed on the back of the hipbone, or posterior iliac crest. An aspirate can also be obtained from the sternum (breastbone). For the sternal aspirate, the patient lies on their back, with a pillow under the shoulder to raise the chest. A trephine biopsy should never be performed on the sternum, due to the risk of injury to blood vessels, lungs or the heart.

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The need to selectively isolate and concentrate selective cells, such as mononuclear cells, allogeneic cancer cells, T cells and others, is driving the market. Over 30,000 bone marrow transplants occur every year. The explosive growth of stem cells therapies represents the largest growth opportunity for bone marrow processing systems.Europe and North America spearheaded the market as of 2016, by contributing over 74.0% to the overall revenue. Majority of stem cell transplants are conducted in Europe, and it is one of the major factors contributing to the lucrative share in the cell harvesting system market.

In 2016, North America dominated the research landscape as more than 54.0% of stem cell clinical trials were conducted in this region. The region also accounts for the second largest number of stem cell transplantation, which is further driving the demand for harvesting in the region.Asia Pacific is anticipated to witness lucrative growth over the forecast period, owing to rising incidence of chronic diseases and increasing demand for stem cell transplantation along with stem cell-based therapy.

Japan and China are the biggest markets for harvesting systems in Asia Pacific. Emerging countries such as Mexico, South Korea, and South Africa are also expected to report lucrative growth over the forecast period. Growing investment by government bodies on stem cell-based research and increase in aging population can be attributed to the increasing demand for these therapies in these countries.

Major players operating in the global bone marrow processing systems market are ThermoGenesis (Cesca Therapeutics inc.), RegenMed Systems Inc., MK Alliance Inc., Fresenius Kabi AG, Harvest Technologies (Terumo BCT), Arthrex, Inc. and others

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Bone Marrow Processing Systems Market Insights on Challenges & Opportunities by 2025 - 3rd Watch News

Bone Marrow Stem Cells Comments Off on Bone Marrow Processing Systems Market Insights on Challenges & Opportunities by 2025 – 3rd Watch News | June 24th, 2020

The global Stem Cell Banking market was valued at USD 1.52 billion in 2016 and is projected to reach USD 7.94 billion by 2025, growing at a CAGR of 20.17% from 2017 to 2025.

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Viral Inactivation MarketVirus Filtration MarketViral Clearance MarketVeterinary-Animal Vaccines Market Vaccine Adjuvants MarketTerahertz and Infrared Spectroscopy MarketTangential Flow Filtration MarketSterile Filtration MarketStem Cell Banking Market

Stem Cell banking involves preservation of new born placental stem cells or amniotic stem cells as well as adult bone marrow stem cells. The concept ensures health safety in case of a major surgery or organ regeneration needs for the patient. With increasing awareness regarding the practice, the market is expected to boost in near future.

The Final Report will cover the impact analysis of COVID-19 on this industry:

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Sample Infographics:

Market Dynamics:1. Market Drivers1.1 Easy Extraction Methods1.2 High birth ratio1.3 Increase in GDP and Disposable income in developing nations1.4 Increasing support from public and private sector1.5 Scope of stem cell usage in therapeutics and treatment2. Market Restraints2.1 Large number of players in the market2.2 High Cost of technology2.3 Lack of awareness2.4 Regulatory restrains

Market Segmentation:1. By Application:1.1 Cerebral Palsy1.2 Thalassemia1.3 Leukemia1.4 Diabetes1.5 Autism1.6 Others

2. By Services:2.1 Collection & Transportation2.2 Processing2.3 Analysis2.4 Storage

3. By Bank Type:3.1 Cord Blood3.2 Cord Tissue

4. By Region:4.1 North America (U.S., Canada, Mexico)4.2 Europe (Germany, UK, France, Rest of Europe)4.3 Asia Pacific (China, India, Japan, Rest of Asia Pacific)4.4 Latin America (Brazil, Argentina, Rest of Latin America)4.5 Middle East & Africa

Competitive Landscape:The major players in the market are as follows:1.CBR Systems, Inc.2. Cordlife3. Cryo-Cell4. Cryo-Save AG (A Group of Esperite)5. Lifecell6. Stemcyte7. Viacord8. Smart Cells International Ltd.9. Cryoviva India10. Cordvida11. China Cord Blood CorporationThese major players have adopted various organic as well as inorganic growth strategies such as mergers & acquisitions, new product launches, expansions, agreements, joint ventures, partnerships, and others to strengthen their position in this market.

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RESEARCH METHODOLOGY OF VERIFIED MARKET INTELLIGENCE:Research study on the Stem Cell Bankingmarketwas performed in five phases which include Secondary research, Primary research, subject matter expert advice, quality check and final review.The market data was analyzed and forecasted using market statistical and coherent models. Also market shares and key trends were taken into consideration while making the report. Apart from this, other data models include Vendor Positioning Grid, Market Time Line Analysis, Market Overview and Guide, Company Positioning Grid, Company Market Share Analysis, Standards of Measurement, Top to Bottom Analysis and Vendor Share Analysis.

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Stem Cell Banking Market Report (2020-2025) | The Demand For The Market Will Drastically Increase In The Future - Jewish Life News

Bone Marrow Stem Cells Comments Off on Stem Cell Banking Market Report (2020-2025) | The Demand For The Market Will Drastically Increase In The Future – Jewish Life News | June 24th, 2020

Leukemia are a heterogeneous group of cancers affecting the bone marrow and White Blood Cells (WBC). Leukemia is characterized by the rapid increase of abnormal blood cells growth or blasts, resulting in a decrease in the numbers of healthy, normal fully modified blood cells, leading to the typical symptoms of bleeding, anemia, and high risk of infection. Leukemia can grow along either the myeloid or lymphoid stem cell lines, it depends on the effect of genetic and epigenetic mutations on the progression of pluripotent stem cells to the various lines of mature cells which then pass into the blood. The effected line, combined with the rate of action and growth of disease reflects the four types of leukemias- Acute Myeloid Leukemia (AML), chronic lymphoblastic leukemia, chronic myeloid leukemia, acute lymphoblastic leukemia. AML: Acute Myeloid Leukemia, is a serious condition, its the most common leukemia suffered by adult people. According to a report from American Cancer Society, the average age for first diagnostic for AML is 64. With few days without treatment, AML develops fast, in duration of few weeks, the patient becomes severely ill. Due to its fast onset and acuteness in nature, there is no staging system for Acute Myeloid Leukemia (AML).The treatment for Acute Myeloid Leukemia (AML) has changed in last 4 decades.

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The drug approval process is difficult in AML, (many drugs have not been approved by USFDA, for instance Laromustine, Dacogen and Tipitarnib) efforts have been made to introduce new therapies in the AML market.

Primary drivers boosting the growth of acute myeloid leukemia (AML) therapeutics market are minimal but increased prevalence of acute myeloid leukemia (AML), increased drug approval rate for AML, classification of acute myeloid leukemia (AML) as an orphan disease. Over the forecast period, population of people over 65 year is anticipated to increase, which is another key driver for acute myeloid leukemia (AML) therapeutics market.

However, lack of targeted therapies in current acute myeloid leukemia (AML) therapeutics landscape, the drug difficult approval process in AML can hinder the growth of acute myeloid leukemia (AML) therapeutics market, but this restraint has opened an opportunity for key players to innovate acute myeloid leukemia (AML) therapeutics market.

The global acute myeloid leukemia (AML) therapeutics market is segmented on the basic of disease subtype, treatment type, end user and region.

Based on the disease subtype, the acute myeloid leukemia (AML) therapeutics market is segmented into the following:

Based on treatment type, the acute myeloid leukemia (AML) therapeutics market is segmented into the following:

Based on end user, the acute myeloid leukemia (AML) therapeutics market is segmented into the following:

The global acute myeloid leukemia (AML) therapeutics market is anticipated to show lucrative growth owing to increased investment in innovative technologies by key players. Players in this market using various strategies to fuel their global footprint and to gain a competitive edge. Product pipelines, new product launches, agreements and collaborations, acquisitions, mergers and clinical trials are some key strategies applied from global players in recent years are anticipated to give a robust hike to the market in the forecast period.

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Geographically, acute myeloid leukemia (AML) therapeutics market is segmented into regions viz. North America, Latin America, Europe, Asia Pacific and Japan, Middle East and Africa. North America is anticipated to be major contributor to this market accounting maximum percent of share in AML therapeutics market followed by Europe. Slow but constant growth in prevalence for AML in North America is anticipated to fuel the growth in acute myeloid leukemia (AML) therapeutics market. In Asia pacific region, China and India are anticipated to show high growth in acute myeloid leukemia (AML) therapeutics market due to new developments in healthcare infrastructure in the region.

The players in acute myeloid leukemia (AML) therapeutics market include Ambit Biosciences Corporation, Celgene Corporation, Cephalon Inc., Clavis Pharma ASA, Eisai Co. Ltd, Genzyme Corporation, and Sunesis Pharmaceuticals Inc., Abbvie Inc., Astellas Pharma Inc, CTI Biopharma Corp etc.

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Acute Myeloid Leukemia (AML) Therapeutics Market Promising Growth Opportunities over 2017 2025 - 3rd Watch News

Bone Marrow Stem Cells Comments Off on Acute Myeloid Leukemia (AML) Therapeutics Market Promising Growth Opportunities over 2017 2025 – 3rd Watch News | June 24th, 2020

The Maryland Stem Cell Research Commission (The Commission) recently announced over $7M in Maryland Stem Cell Fund (MSCF) grant awards for its second round of 2020 MSCF fund recipients. The MSCF, which is a program of the Maryland Technology Development Corporation (TEDCO), has awarded $157M in funding to BioHealth Capital Region (BHCR) companies seeking to accelerate stem cell research, therapies and commercialization of products since 2007.

The $7M in new funding follows MSCFs announcement in September 2019 of over $1.3M in grants for the first cohort of 2020 recipients, bringing the total 2020 MSCF award tally to approximately $8.3M for the year. The financial awards are delivered across a wide range of areas, including clinical, commercialization, validation, launch, discovery, and post-doctoral fellowships. The first cohort of funding included three commercialization and two validation awards; the second, larger recipient pool included one clinical, one commercialization, one validation, four launches, 11 discovery, and five post-doctoral awards.

Notable BHCR MSCF recipients included:

Dr. Luis Garza of Johns Hopkins University (JHU) received a clinical grant to support clinical trials for his autologous volar fibroblast injection into the stump site of amputees. The trials are exploring ways to make the skin where a prosthetic limb meets the stump site tougher and less irritable to the wearer. Skin irritation is a major issue for those with prosthetic limbs and is often a cause for individuals to stop wearing their prosthesis.

Vita Therapeutics, a company that spun out of JHU, was awarded a 300K MSCF grant to support the commercialization of the companys satellite stem cell therapy for limb-girdle Muscular Dystrophy. According to the National Organization for Rare Disorders (NORD), Limb-girdle muscular dystrophies (LGMD) are a group of rare progressive genetic disorders that are characterized by wasting (atrophy) and weakness of the voluntary muscles of the hip and shoulder areas (limb-girdle area). Vita Therapeutics is led by CEO Douglass Falk, who is a JHU alum.

Jamie Niland, VP of Baltimore, Marylands Neoprogen Inc. received part of $892,080K in funding that was part of MSCFs first 2020 grant round. Jamie is the son of Bill Niland, Neoprogens current CEO and the former leader of Baltimore, Maryland life science community anchor Harpoon Medical, which was acquired by Edwards Scientific in 2017. The award was for Neoprogens neonatal cardiac stem cells for the heart tissue regeneration program.

Dr. Brian Pollok of Rockville, Marylands Propagenix, Inc., was also the recipient of a commercialization award for his Apical Surface-Outward (ASO) airway organoids, which is a potential novel cell system for drug discovery and personalized medicine. Propagenix develops innovative new technologies that address unmet needs in epithelial cell biologyfor applications in life science research as well as in precision diagnostics, and next-generation therapeutics such as immune-oncology, tissue engineering, and regenerative medicine, according to the companys website.

In addition, Dr. Ines Silva, R&D Manager of REPROCELL, USA received an MSCF commercialization grant for its work on building a commercial neural cell bank from patient-derived induced pluripotent stem cells. REPROCELL was founded in Japan in 2003 and acquired BioServe in Beltsville, Maryland in 2014.

Dr. Sashank Reddy, the founder of JHU startup LifeSprout and Medical Director, Johns Hopkins Technology Ventures Johns Hopkins University, received a portion of the $1,334,462 distributed for launch grants in 2020. The grant will go to support the launch of regenerative cell therapies for soft tissue restoration. LifeSprout recently closed a $28.5M seed round.

Past MSCF grant recipients include Frederick, Marylands RoosterBio, Inc. and Theradaptive, Inc., and Baltimore, Marylands Gemstone Biotherapeutics and Domicell, Inc., among others.

TEDCOs MSRF program continues to lend its deep support and ample funding to build and grow Marylands burgeoning and exciting regenerative medicine industry. Well be keeping a close eye on these companies as they grow and make future contributions to the thriving BHCR biocluster.

Steve has over 20 years experience in copywriting, developing brand messaging and creating marketing strategies across a wide range of industries, including the biopharmaceutical, senior living, commercial real estate, IT and renewable energy sectors, among others. He is currently the Principal/Owner of StoryCore, a Frederick, Maryland-based content creation and execution consultancy focused on telling the unique stories of Maryland organizations.

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Over $8M in 2020 Stem Cell Funding Awards Continue to Fuel Marylands Leading Cell Therapy Industry - BioBuzz

Skin Stem Cells Comments Off on Over $8M in 2020 Stem Cell Funding Awards Continue to Fuel Marylands Leading Cell Therapy Industry – BioBuzz | June 24th, 2020

Introduction

Breastfeeding is the cornerstone of infant and young child survival, nutrition and development and maternal health. The World Health Organization recommends exclusive breastfeeding for the first 6 months of life, followed by continued breastfeeding with appropriate complementary foods for up to 2 years and beyond.1 Early and uninterrupted skin-to-skin contact, rooming-in2 and kangaroo mother care3 also significantly improve neonatal survival and reduce morbidity and are recommended by WHO.

However, concerns have been raised about whether mothers with COVID-19 can transmit the SARS-CoV-2 virus to their infant or young child through breastfeeding. Recommendations on mother-infant contact and breastfeeding must be based on a full consideration of not only of the potential risks of COVID-19 infection of the infant, but also the risks of morbidity and mortality associated with not breastfeeding, the inappropriate use of infant formula milks, as well as the protective effects of skin-to-skin contact. This scientific brief examines the evidence to date on the risks of transmission of COVID-19 from an infected mother to her baby through breastfeeding as well as evidence on the risks to child health from not breastfeeding.

WHO recommends that mothers with suspected or confirmed COVID-19 should be encouraged to initiate or continue to breastfeed. Mothers should be counselled that the benefits of breastfeeding substantially outweigh the potential risks for transmission.4

Mother and infant should be enabled to remain together while rooming-in throughout the day and night and to practice skin-to-skin contact, including kangaroo mother care, especially immediately after birth and during establishment of breastfeeding, whether they or their infants have suspected or confirmed COVID-19.

A living systematic review of evidence that followed the procedures of the Cochrane handbook for systematic reviews of interventions was carried out with the latest search done on 15 May 2020 to identify studies including mothers with suspected or confirmed COVID-19 and their infants or young children.5 The search was conducted on Cochrane Library, EMBASE (OVID), PubMed (MEDLINE), Web of Science Core Collection (Clarivate Analytics) and the WHO Global Database. A total of 12,198 records were retrieved, 6945 were screened after removing duplicates, and 153 records with mother-infant dyads in which the mother had COVID-19 were included in full-text review.

A total of 46 mother-infant dyads had breastmilk samples tested for COVID-19. All mothers had COVID-19, while 13 infants tested COVID-19 positive. Breastmilk samples from 43 mothers were negative for the COVID-19 virus while samples from 3 mothers tested positive for viral particles by RT-PCR. Among the 3 infants whose mothers breastmilk tested positive for virual RNA particles, not live virus, one infant tested positive for COVID-19 but infant feeding practices were not reported. The two other infants tested negative for COVID-19; one was breastfed, and the other newborn was fed expressed breast milk after viral RNA particles were no longer detected. In the single child with COVID-19, it was unclear through which route or source the infant became infected, i.e. through breastmilk or droplet from a close contact with the infected mother.

A preprint article reported secretory immunoglobulin A (sIgA) immune response against the COVID-19 virus found in 12 of 15 breastmilk samples from mothers with COVID-19.6 The implications of this finding on the effect, duration and protection against COVID-19 for the child was not addressed.

To date, studies of mother-infant dyads with data on feeding practices and COVID-19 infection have come from case reports, case series or a report of a family cluster. Other study designs such as cohort studies or case-control studies were eligible for inclusion, but none were identified. We are thus unable to measure and compare risks of infection based on feeding practices.

Although 1 of the 3 infants of mothers with viral particles in breast milk had COVID-19, it was unclear through which route or source the infant was infected, i.e., through breastfeeding or close contact with the mother or other infected person. RT-PCR detects and amplifies viral genetic material in samples, such as breastmilk, but does not provide information on viability or infectivity of the virus. Documented presence of replicative COVID-19 virus in cell culture from breast milk and infectivity in animal models are needed to consider breast milk as potentially infectious.

The presence of IgA in breast milk is one of the ways in which breastfeeding protects infants against infection and death. IgA antibodies with reactivity to the COVID-19 virus have been detected in breastmilk of mothers previously infected with COVID-19 but their strength and durability have not yet been adequately studied to address protection from COVID-19 among breastfed infants.

Detection of COVID-19 viral RNA in breastmilk is not the same as finding viable and infective virus. Transmission of COVID-19 would need replicative and infectious virus being able to reach target sites in the infant and also to overcome infant defense systems. If in the future COVID-19 virus from breastmilk were shown to be replicative in cell culture it would need to reach target sites in the infant and overcome infant defense systems for transmission of COVID-19 to occur.

The implications of transmission risk need to be framed in terms of COVID-19 prevalence in breastfeeding mothers and the scope and severity of COVID-19 infection in infants when transmission occurs compared to the adverse consequences of separation and using breastmilk substitutes and also separation of newborns and young infants from mothers.

Children appear to be at low risk of COVID-19. Among the cases of confirmed COVID-19 in children, most have experienced only mild or asymptomatic illness.7,8 This is also the case with other zoonotic coronaviruses (SARS-CoV and MERS-CoV), which seem to affect children less commonly and to cause fewer symptoms and less severe disease compared with adults.9

Secretory IgA have been detected in breastmilk of mothers with previous COVID-19 infection. Although the strength and durability of sIgA reactive to COVID-19 have not yet been determined, multiple bioactive components have been identified in breastmilk that not only protect against infections but improve neurocognitive and immunologic development of the child since Lars A Hanson first described sIgA in breastmilk in 1961.10-12

Skin-to-skin contact and kangaroo mother care facilitate breastfeeding as well as improve thermoregulation, blood glucose control, and maternal-infant attachment, and decrease the risk in mortality and severe infection among low birth weight infants.13,14 Beyond the neonatal period, positive effects of mother-infant holding include improved sleep patterns, lower rates of behavioural problems in the child and higher quality parental interaction.15,16

Exclusively breastfed infants, the risk of mortality is 14-fold higher in infants who are not breastfed.17 Over 820 000 childrens lives could be saved every year among children under 5 years, if all children 0-23 months were optimally breastfed. For mothers, breastfeeding protects against breast cancer and may protect against ovarian cancer and type 2 diabetes.18 On the other hand, children are at low risk of COVID-19.

It is still not clear whether the virus can or cannot be transmitted though breast milk. Risk of transmission based on feeding practices have not been quantified, compared, or modelled against the benefits of breastfeeding and nurturing mother-infant interaction.

At present, data are not sufficient to conclude vertical transmission of COVID-19 through breastfeeding. In infants, the risk of COVID-19 infection is low, the infection is typically mild or asymptomatic, while the consequences of not breastfeeding and separation between mother and child can be significant. At this point it appears that COVID-19 in infants and children represents a much lower threat to survival and health than other infections that breastfeeding is protective against. The benefits of breastfeeding and nurturing mother-infant interaction to prevent infection and promote health and development are especially important when health and other community services are themselves disrupted or limited. Adherence to infection prevention and control measures is essential to prevent contact transmission between COVID-19 suspected or confirmed mothers and their newborns and young infants.

Based on available evidence, WHO recommendations on the initiation and continued breastfeeding of infants and young children also apply to mothers with suspected or confirmed COVID-19.

WHO continues to monitor the situation closely for any changes that may affect this interim guidance. Should any factors change, WHO will issue a further update. Otherwise, this scientific brief will expire 2 years after the date of publication.

Continued here:
Breastfeeding and COVID-19 - World Health Organization

Skin Stem Cells Comments Off on Breastfeeding and COVID-19 – World Health Organization | June 24th, 2020

The unwanted side effects include blisters, scabbing, hyperpigmentation, and hypopigmentation. Of some comfort: Pigmentation issues aren't always permanent. If you get it, let your doctor know right away and use a little cortisone 1% cream on the area, says Marmur. Keep it out of the sun and heat, and apply a cool compress ASAP.

If you're predisposed to hyperpigmentation, your dermatologist may even make a preemptive strike. For patients with darker skin types, we apply over-the-counter hydrocortisone 1% cream in the office to minimize inflammation and the risk of darkening of skin, or post-inflammatory hyperpigmentation," says Kim. "We recommend applying the cream twice daily to the affected areas for three to five days.

It largely varies, since the lasers themselves have gotten better over the years. I've had patients who were able to tolerate the treatment without any numbing, and patients who experienced some pain even with topical anesthesia, says Kim. (I, a baby, prefer to spend an hour with numbing cream and have never felt a thing.)

Marmur compares the laser beam to a zinging feeling, similar to a needle prick. She's a fan of contact cooling systems, as they blunt the heat created as the laser beam (which is light energy) converts into heat. Plus, they offer enough cooling to minimize any damage caused to surrounding skin, reducing the risk of hyperpigmentation.

Certain pain-reducing methods, like suctions and contact cooling, are often built into the lasers. There is a new laser by Lumenis called Splendor that is very effective and significantly more comfortable than other existing lasers, and I have had great success with it, says Kim. Because this laser is much more comfortable, I have been able to treat almost all patients without any topical numbing cream which significantly reduces the waiting time for the patients as well.

Since laser hair removal heats up your skin as it blasts your hair follicles, it's important to cool it back down afterwards to avoid side effects like redness. We often give people cold gauze in Ziploc bags, says Marmur. If you're getting in a car, put on the air conditioning and stay in a cool place for a bit, or take a cool shower afterwards.

Marmur sends her patients home with a cooling serum, the Marmur Metamorphosis MMRevive Serum. You could also try Avne Cicalfate Restorative Protective Cream, which soothes with a combination of barrier-repairing ingredients and probiotics.

Avoiding sun exposure and wearing sunscreen is also a must, as sunlight can kickstart hyperpigmentation. Kim recommends wearing a minimum of SPF 30. Got another session coming up? Patients should not wax, pluck, or thread the treated areas in between treatments, because it's essential for the hair follicles to be intact in order for the treatments to be effective at the next session," he says.

If we're being technical, laser hair removal is something of a misnomer. It's more like laser hair reduction, says Marmur. That's because you have two types of hair: vellus hairs, which are fine baby hairs, and terminal hairs, which are more coarse. The vellus baby hairs get affected by hormones and convert to terminal hairs throughout your life, she says.

So, you may do laser hair removal at 18, but by 30, you might have new growth coming in. It's just nature doing its thing. That being said, once a hair follicle root is dead, it's dead forever.

Always make sure you're going to a board certified dermatologist or reputable practitionerthis isn't a procedure you want to cut corners on just because you found a good discount online. And don't be afraid to ask for a consultation ahead of an appointment to discuss the procedure. As for during your appointment, you'll want to make sure both you and your practitioner have safety goggles on while the laser is in process.

At-home laser hair removal devices also exist, but they're generally less effective (meaning it will take much longer to see results), and theres more room for error as the beam is less specific. This is why experts generally suggest going in-office for the procedure.

Laser hair removal costs an average of $285 for one session, according to the latest stats from the American Society of Plastic Surgeons, but some treatments can run up to $1,500 per session. That's because the cost varies widely according to a number of factors, such as the size of the area you're treating, the provider's expertise, and where you're located. Just remember: Any treatment that seems too affordable to be true, often is.

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Is Laser Hair Removal Worth the Cost & Hassle? What to Know - Glamour

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