Thalassemia is a type of inherited blood disorder. It is passed from parents to children through genes. This disorder involves lack of oxygen-carrying protein called hemoglobin (an important part of red blood cells). When there is insufficiency of hemoglobin in the body, the red blood cells dont function properly. It also reduces the life of RBC, which means fewer healthy RBC travel in the blood.

RBC carries oxygen to all the cells of the body. Oxygen acts as food, which is used by cells to function. Shortage of healthy RBC means shortage in supply of oxygen to all other cells of the body. This may lead to lethargy in a person. The person may feel tired, weak or short of breath. This condition is termed as Anaemia.

People with thalassemia may suffer from mild or acute Anaemia. Acute Anaemia can be very severe and can lead to damage of major organs. It can even cause death.

Thalassemia major babies are born to parents who are carriers of thalassemia gene. According to rough estimates, each year some 10000 babies are born in India with thalassemia. Best way to prevent or eliminate thalassemia is screening of all pregnant women between 9 to 12 weeks.

Thalassemia is diagnosed through blood tests which include doing a complete blood count (CBC) and special hemoglobin tests. Through a sample of blood, CBC measures the amount of hemoglobin and the different kind of blood cells, such as red blood cells. Hemoglobin tests measure the types of hemoglobin in blood.

Moderate and acute thalassemia is usually diagnosed in childhood. This is because signs and symptoms, such as acute Anemia usually occur at an early age of 2 years. People who have mild form of thalassemia may get diagnosed after a routine blood test, as it will detect if they have anemia.

Here's Dr. Rahul Bhargava, Director and Head, Hematology, Haemato- Oncology and Bone Marrow Transplant, Fortis Memorial Research Institute, Gurugram has to say about the treatments:

Blood Transfusion

Treatment of thalassemia major relies on regular blood transfusion at regular intervals, to keep Hb above 9 gm. percent. It will help prevent form short stature and other skeletal and facial deformities. Recurrent lifelong blood transfusion since 6 months of birth is necessary.

Iron Chelation Therapy

With transfusion comes the problem of iron deposition, as each blood transfusion lead to incremental iron deposition in various tissues like pituitary gland liver and heart leading to early death. So along with transfusion patient also needs iron chelation therapy. It can be either oral (defriprone and defreseirox) or IV desferoxmine. Serum ferritin is one of the surrogate markers of iron overload in thalassemia patients. It needs to be done every 3 months. Gov.s efforts of providing free blood products and iron chelators is bearing fruits as life expectancy has shown an upward trend.

Bone Marrow or Stem Cell Transplant

As it is commonly known, bone marrow or stem cell transplant is the only curative modality for thalassemia. If done at an early age, 80 percent patients can be cured. Source of stem cell could be either brother or sister whose HLA is a complete match. Otherwise fully matched HLA donor can be tried in various international registries. This process is called as match unrelated donor transplant.

Gene Therapy

Gene therapy is gaining lot of traction in field of hemoglobinopathies. It has shown remarkable result with minimum toxicities and sustained haemoglobin production in various trials. There has been no major risk of cancer or other late effects.

We have come a long way and probably this decade will bring the much awaited cheers to thalassaemics. Till then in India, prevention is the only strategy to reduce the burden on already stretched health care system.

Better rate of blood transfusion

Regular Blood screening has significantly impacted reduction of infections due to blood transfusion

Significant improvement in treatment

Bone Marrow Transplant and Stem cell transplantation has led to patients having a good quality of life

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A 7-year-old boy in Marylandwho suffers from sickle cell anemiais on his way to a full recovery after being hospitalized with a case of the coronavirus, according to reports.

Nasir Striggs was first hospitalized at Sinai Hospital in Baltimore in early April. His mother, Deshannon Striggs, brought him in for an examination after she noticed her son was experiencing trouble breathing.

He tested positive for COVID-19 at the hospital. An X-ray revealed he also had pneumonia in both lungs. The child, diagnosed with sickle cell disease, an inherited red blood cell disorder, underwent several blood transfusions at the hospital before his release.

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Nasir Striggs, 7, who has sickle cell anemia, is home from the hospital after recovering from the coronavirus. (Courtesy: Deshannon Striggs)

He had to keep getting stuck by the needle because the needle kept coming out, the mother told WBAL. To watch him go through that, it was really scary.

After undergoing treatment for several days, his condition began to improve, Deshannon said. She said prayers and support, as well as the dedicated care from the hospitals medical team, have helped her sons recovery.

Just keep the faith. Thats the message: keep the faith, she said.

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Deshannon said doctors have been monitoringNasirs conditionvia virtual check-ups since he was discharged from the hospital. Photos she shared with Fox News show the boy at home smiling,his face mask pulled beneath his chin.

Sickle cell disease is usually diagnosed shortly after birth. The genetic disorder results in oxygen-carrying red blood cells taking on a C or sickle shape, instead of round,often getting stuck in small blood vessels and clogging blood flow, according to the Centers for Disease Control and Prevention.

Children with the disease are at an increased risk of infection and other health problems. The only known cure is a bone marrow or stem cell transplant.

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A Washington based biotechnology company developing leronlimab (PRO 140), a CCR5 antagonist with the potential for multiple therapeutic indications, announcedthe availability of thepre-print version of the manuscriptdescribing the immunological mechanism by whichleronlimabrestores immune function and impacts disease in COVID-19 patients.

This manuscript,Disruption of the CCL5/RANTES-CCR5 Pathway Restores Immune Homeostasis and Reduces Plasma Viral Load in Critical COVID-19, has been shared with the World Health Organization and is currently under peer review, said CytoDyn, Inc., as of May 6, 2020.

As described in thepre-print, in a cohort of 10critically ill patients, after treatment with leronlimab, these critically ill patients experienced reversed hyperimmune activation and inflammation, as well as reversed immunosuppression, therebyfacilitating a more effective immune responsecorrelated with decreases in SARS-CoV-2 level in blood.

These results demonstrate a novel approach to resolving unchecked inflammation whilerestoring immunologic deficiencies.

This is an important finding since according to various studies, a major driver of severe COVID-19 disease is excessive inflammation.

Nader Pourhassan, Ph.D., President and Chief Executive Officer of CytoDyn said in a press statement, We are now most hopeful the entire medical community will understand the potential benefit leronlimab can provide critically ill COVID-19 patients."

"Moreover, this discovery by Dr. Bruce Patterson that leronlimab decreases plasma viral load may have tremendous long-term positive ramifications to bring this pandemic under control. We are grateful that we are able to release this research at such a critical time for patients throughout the world.

Leronlimab (PRO 140) is being used as a treatment for severe COVID-19 under the emergency Investigational New Drug (IND) recently granted by the U.S. Food and Drug Administration (FDA).

Leronlimab is a drug candidate that is aCCR5 antagonist with the potential for multiple therapeutic indications.Leronlimab belongs to a group of HIV drugs calledCCR5antagonists.

The CCR5 receptor appears to play a central role in modulating immune cell trafficking to sites of inflammation. It may be crucial in the development of acute graft-versus-host disease (GvHD) and other inflammatory conditions.

Clinical studies by others further support the concept that blocking CCR5 using a chemical inhibitor can reduce the clinical impact of acute GvHD without significantly affecting the engraftment of transplanted bone marrow stem cells.

Previously, CytoDyn announced it is seeking a compassionate use designation for leronlimabfor the treatment of COVID-19 patients who are ineligible for participation in its two existing clinical trials. If this request is granted by the FDA, it will significantly expand the pool of patients who would be eligible to receive leronlimabtherapy.

CytoDyn is a late-stage biotechnology company developing innovative treatments for multiple therapeutic indications based on leronlimab, a novel humanized monoclonal antibody targeting the CCR5 receptor.

Precision Vaccinations publishes developing COVID-19 therapeutic drug news.

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VANCOUVER, Washington, May 08, 2020 (GLOBE NEWSWIRE) -- CytoDyn Inc. (OTC.QB: CYDY), (CytoDyn or the Company), a late-stage biotechnology company developing leronlimab (PRO 140), a CCR5 antagonist with the potential for multiple therapeutic indications, today further clarified the status of the Companys submission of its Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for leronlimab as a combination therapy with HAART for highly treatment experienced HIV patients, filed on April 27, 2020 with the FDA. The BLA will not be considered completed until the Company submits to the FDA clinical datasets required to address FDA comments it received in March 2020, as described in the Companys press releases on May 4 and May 6, 2020. CytoDyn expects to submit these clinical datasets on May 11, 2020.

After the BLA submission is deemed completed, FDA makes a filing decision and sets a PDUFA goal date. CytoDyn has Fast Track designation for leronlimab and a rolling review for its BLA, as previously assigned by the FDA and the Company plans to request a priority review for the BLA. A priority review designation, if granted, means the FDAs goal is to take action on the application within six months of receipt (compared with 10 months under standard review).

About Coronavirus Disease 2019CytoDyn is currently enrolling patients in two clinical trials for COVID-19, a Phase 2 randomized clinical trial for mild-to-moderate COVID-19 population in the U.S. and a Phase 2b/3 randomized clinical trial for severe and critically ill COVID-19 population in several hospitals throughout the country.

SARS-CoV-2 was identified as the cause of an outbreak of respiratory illness first detected in Wuhan, China. The origin of SARS-CoV-2 causing the COVID-19 disease is uncertain, and the virus is highly contagious. COVID-19 typically transmits person to person through respiratory droplets, commonly resulting from coughing, sneezing, and close personal contact. Coronaviruses are a large family of viruses, some causing illness in people and others that circulate among animals. For confirmed COVID-19 infections, symptoms have included fever, cough, and shortness of breath. The symptoms of COVID-19 may appear in as few as two days or as long as 14 days after exposure. Clinical manifestations in patients have ranged from non-existent to severe and fatal. At this time, there are minimal treatment options for COVID-19.

About Leronlimab (PRO 140) and BLA Submission for the HIV Combination TherapyThe FDA has granted a Fast Track designation to CytoDyn for two potential indications of leronlimab for deadly diseases. The first as a combination therapy with HAART for HIV-infected patients and the second is for metastatic triple-negative breast cancer.Leronlimab is an investigational humanized IgG4 mAb that blocks CCR5, a cellular receptor that is important in HIV infection, tumor metastases, and other diseases, including NASH.Leronlimab has completed nine clinical trials in over 800 people, including meeting its primary endpoints in a pivotal Phase 3 trial (leronlimab in combination with standard antiretroviral therapies in HIV-infected treatment-experienced patients).

In the setting of HIV/AIDS, leronlimab is a viral-entry inhibitor; it masks CCR5, thus protecting healthy T cells from viral infection by blocking the predominant HIV (R5) subtype from entering those cells. Leronlimab has been the subject of nine clinical trials, each of which demonstrated that leronlimab could significantly reduce or control HIV viral load in humans. The leronlimab antibody appears to be a powerful antiviral agent leading to potentially fewer side effects and less frequent dosing requirements compared with daily drug therapies currently in use.

The Company filed its BLA for Leronlimab as a Combination Therapy for Highly Treatment Experienced HIV Patients to the FDA on April 27, 2020. The BLA will not be considered completed until the Company submits required clinical datasets to the FDA. The Company expects to submit the required datasets on May 11, 2020. After the BLA submission is considered completed, FDA will make a filing decision and set a PDUFA goal date. CytoDyn has Fast Track designation for leronlimab and a rolling review for its BLA, as previously assigned by the FDA and the Company plans to request a priority review for the BLA. A priority review designation means the FDAs goal is to take action on the marketing application within six months of receipt (compared with 10 months under standard review).

In the setting of cancer, research has shown that CCR5 may play a role in tumor invasion, metastases, and tumor microenvironment control. Increased CCR5 expression is an indicator of disease status in several cancers. Published studies have shown that blocking CCR5 can reduce tumor metastases in laboratory and animal models of aggressive breast and prostate cancer. Leronlimab reduced human breast cancer metastasis by more than 98% in a murine xenograft model. CytoDyn is, therefore, conducting aPhase 1b/2 human clinical trial in metastatic triple-negative breast cancer and was granted Fast Track designation in May 2019.

The CCR5 receptor appears to play a central role in modulating immune cell trafficking to sites of inflammation. It may be crucial in the development of acute graft-versus-host disease (GvHD) and other inflammatory conditions. Clinical studies by others further support the concept that blocking CCR5 using a chemical inhibitor can reduce the clinical impact of acute GvHD without significantly affecting the engraftment of transplanted bone marrow stem cells. CytoDyn is currently conducting a Phase 2 clinical study with leronlimab to support further the concept that the CCR5 receptor on engrafted cells is critical for the development of acute GvHD, blocking the CCR5 receptor from recognizing specific immune signaling molecules is a viable approach to mitigating acute GvHD. The FDA has granted orphan drug designation to leronlimab for the prevention of GvHD.

About CytoDynCytoDyn is a late-stage biotechnology company developing innovative treatments for multiple therapeutic indications based on leronlimab, a novel humanized monoclonal antibody targeting the CCR5 receptor. CCR5 appears to play a critical role in the ability of HIV to enter and infect healthy T-cells.The CCR5 receptor also appears to be implicated in tumor metastasis and immune-mediated illnesses, such as GvHD and NASH. CytoDyn has successfully completed a Phase 3 pivotal trial with leronlimab in combination with standard antiretroviral therapies in HIV-infected treatment-experienced patients. CytoDyn filed its BLA in April 2020 to seek FDA approval for leronlimab as a combination therapy for highly treatment experienced HIV patients, and plans to submit additional datasets needed to complete the BLA on May 11, 2020. CytoDyn is also conducting a Phase 3 investigative trial with leronlimab as a once-weekly monotherapy for HIV-infected patients. CytoDyn plans to initiate a registration-directed study of leronlimab monotherapy indication. If successful, it could support a label extension. Clinical results to date from multiple trials have shown that leronlimab can significantly reduce viral burden in people infected with HIV with no reported drug-related serious adverse events (SAEs). Moreover, a Phase 2b clinical trial demonstrated that leronlimab monotherapy can prevent viral escape in HIV-infected patients; some patients on leronlimab monotherapy have remained virally suppressed for more than five years. CytoDyn is also conducting a Phase 2 trial to evaluate leronlimab for the prevention of GvHD and a Phase 1b/2 clinical trial with leronlimab in metastatic triple-negative breast cancer. More information is atwww.cytodyn.com.

Forward-Looking StatementsThis press releasecontains certain forward-looking statements that involve risks, uncertainties and assumptions that are difficult to predict. Words and expressions reflecting optimism, satisfaction or disappointment with current prospects, as well as words such as believes, hopes, intends, estimates, expects, projects, plans, anticipates and variations thereof, or the use of future tense, identify forward-looking statements, but their absence does not mean that a statement is not forward-looking. Forward-looking statements specifically include statements about leronlimab, its ability to have positive health outcomes, the possible results of clinical trials, studies or other programs or ability to continue those programs, the ability to obtain regulatory approval for commercial sales, and the market for actual commercial sales. The Companys forward-looking statements are not guarantees of performance, and actual results could vary materially from those contained in or expressed by such statements due to risks and uncertainties including: (i)the sufficiency of the Companys cash position, (ii)the Companys ability to raise additional capital to fund its operations, (iii) the Companys ability to meet its debt obligations, if any, (iv)the Companys ability to enter into partnership or licensing arrangements with third parties, (v)the Companys ability to identify patients to enroll in its clinical trials in a timely fashion, (vi)the Companys ability to achieve approval of a marketable product, (vii)the design, implementation and conduct of the Companys clinical trials, (viii)the results of the Companys clinical trials, including the possibility of unfavorable clinical trial results, (ix)the market for, and marketability of, any product that is approved, (x)the existence or development of vaccines, drugs, or other treatments that are viewed by medical professionals or patients as superior to the Companys products, (xi)regulatory initiatives, compliance with governmental regulations and the regulatory approval process, (xii)general economic and business conditions, (xiii)changes in foreign, political, and social conditions, and (xiv)various other matters, many of which are beyond the Companys control. The Company urges investors to consider specifically the various risk factors identified in its most recent Form10-K, and any risk factors or cautionary statements included in any subsequent Form10-Q or Form8-K, filed with the Securities and Exchange Commission. Except as required by law, the Company does not undertake any responsibility to update any forward-looking statements to take into account events or circumstances that occur after the date of this press release.

CYTODYN CONTACTSInvestors: Dave Gentry, CEORedChip CompaniesOffice: 1.800.RED.CHIP (733.2447)Cell: 407.491.4498dave@redchip.com

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Korean researchers have found a signal transduction system that modulates the treatment of mesenchymal stem cells and immune control functions, opening the way for treating graft-versus-host disease treatment.

Mesenchymal stem cells divide into various cells, have immunomodulatory functions, and are the primary cell sources for stem cell therapy.

Graft-versus-host disease is a fatal disease that leads to death after an allogeneic blood transfusion or bone marrow transplantation. Although there are many clinical trials underway worldwide to treat the symptom, there are no applicable treatments besides alleviating symptoms with high-dose steroids.

The team, led by Professor Shin Dong-myeong of the Department of Biomedical Sciences at Asan Medical Center, discovered that the CREB1 (CAMP responsive element binding protein 1) signaling system activates the treatment and immune control functions of mesenchymal stem cells.

The team administered a therapeutic agent made by upgrading mesenchymal stem cells to graft-versus-host disease mice, and found that it alleviated anorexia symptoms and reduced the weight loss rate by 30 percent while increasing the survival rate by 30 percent.

When developing a cell therapy product, researchers have to cultivate the stem cells in vitro. Thus it is very likely that it will impair stem cell functions due to free radicals generated in the cells. To prevent the deterioration of stem cell function, it is necessary to improve the stem cell function in vitro culture, prevent stem cell oxidation, and increase the antioxidant capacity of the cell itself.

Until now, there was a lack of specific evidence and understanding of how stem cells regulate glutathione, an indicator of antioxidant capacity. Therefore, it was difficult to prevent stem cell dysfunction and oxidation.

Professor Shin's team developed experimental techniques that can monitor and quantify glutathione in real-time and confirmed that the CREB1 signaling system regulated the amount and activity of glutathione.

By activating the CREB1 signaling system, the team found that the process also activated nuclear factor erythroid 2-related factor 2 (NRF2) protein, which maintains the antioxidant capacity of mesenchymal stem cells and the increase of both the expression levels of peroxiredoxin-1 (PRDX1) and glutamate-cysteine ligase modifier subunit (GCLM) protein, which synthesize glutathione and are antioxidant activity indicators.

As a result, the team confirmed that its method was effective in treating the graft-versus-host disease.

"Based on this study, we have secured a technological foundation to advance stem cell treatment by controlling the antioxidant capacity of stem cells," Professor Shin said.

If this technology makes a high-purity and high-quality stem cell treatment, the team expects that it will be a step toward developing a graft-versus-host disease treatment and overcoming various intractable diseases such as nervous system diseases and inflammatory diseases with high medical demand, Shin added.

The results of the study were published in the journal, Science Advances.

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A group of UB researchers have published a paper that clarifies certain cellular mechanisms that could lead to improved outcomes in patients with globoid cell leukodystrophy, commonly known as Krabbe disease.

The paper, titled Macrophages Expressing GALC Improve Peripheral Krabbe Disease by a Mechanism Independent of Cross-Correction, was published May 5 in the journal Neuron.

The research was led by Lawrence Wrabetz and M. Laura Feltri. Wrabetz and Feltri head the Hunter James Kelly Research Institute and both are professors in the departments of Biochemistry and Neurology in the Jacobs School of Medicine and Biomedical Sciences at UB.

The institute is named for the son of former Buffalo Bills quarterback Jim Kelly. Hunter Kelly died at age 8 in 2005 from complications of Krabbe disease.

Krabbe disease is a progressive and fatal neurologic disorder that usually affects newborns and causes death before a child reaches the age of 2 or 3.

Traditionally, hematopoietic stem cell transplantation, also known as a bone marrow transplant, has improved the long-term survival and quality of life of patients with Krabbe disease, but it is not a cure.

It has long been assumed that the bone marrow transplant works by a process calledcross-correction, in which an enzyme called GALC is transferred from healthy cells to sick cells.

Using a new Krabbe disease animal model and patient samples, the UB researchers determinedthatin reality cross-correctiondoes not occur. Rather, the bone marrow transplant helps patients through a different mechanism.

The researchers first determined which cells are involved in Krabbe disease and by which mechanism. They discovered that both myelin-forming cells, or Schwann cells, and macrophages require the GALC enzyme, which is missing in Krabbe patients due to genetic mutation.

Schwann cells require GALC to prevent the formation of a toxic lipid called psychosine, which causes myelin destruction and damage to neurons. Macrophages require GALC to aid with the degradation of myelin debris produced by the disease.

The research showed that hematopoietic stem cell transplantation does not work bycross-correction, but by providing healthy macrophages with GALC.

According to Feltri, the data reveal that improvingcross-correctionwould be a way to makebone marrow transplants and other experimental therapies such as gene therapy more effective.

Bone marrow transplantation and other treatments for lysosomal storage disorders, such as enzyme replacement therapy, have historically had encouraging but limited therapeutic benefit, says study first author Nadav I. Weinstock, an MD-PhD student in the Jacobs School. Our work defined the precise cellular and mechanistic benefit of bone marrow transplantation in Krabbe disease, while also shedding light on previously unrecognized limitations of this approach.

Future studies, using genetically engineered bone marrow transplantation or other novelapproaches,may one day build on our findings and eventually bridge the gap for effectively treating patients with lysosomal disease, he continues.

UB investigators included Daesung Shin, research assistant professor at the Hunter James Kelly Research Institute; Nicholas Silvestri, clinical associate professor of neurology, Jacobs School; Narayan Dhimal, PhD student; Chelsey B. Reed, MD-PhD student; and undergraduate student Oliver Sampson.

Also participating in the research were Eric E. Irons, MD-PhD student, and Joseph T.Y. Lau, a distinguished faculty member from the Department of Molecular and Cellular Biology at Roswell Park Comprehensive Cancer Center.

The research was funded by multiple grants from the National Institutes of Health awarded to Weinstock, Shin, Wrabetz and Feltri, and also supported by Hunters Hope.

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Avrobio is working to make conditioning, a necessary step for some gene therapies, safer. But its not stopping at improving current approachesthe company is teaming up with Magenta Therapeutics to see whether an antibody-drug conjugate (ADC) can do the job.

Under the deal, the duo will test Magentas lead conditioning program, MGTA-117, alongside at least one of Avrobios gene therapies. Each company will hold onto the rights for their respective programs, but Avrobio will pick up the tab for clinical trials involving MGTA-117.

We believe targeted ADCs represent the next generation of medicines to prepare patients for gene therapy or transplant in a targeted, precise way This partnership will allow Magenta to validate our conditioning platform in lentiviral gene therapy applications, said Magenta CEO Jason Gardner, D.Phil., in a statement.

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Avrobios lead program is a gene therapy for Fabry disease dubbed AVR-RD-01. It is based on CD34+ stem cells that have been modified using a lentiviral vector to carry and express the gene that codes for the enzyme that is missing in Fabry disease. It is also working on treatments for Gaucher disease, Cystinosis and Pompe disease.

RELATED: Avrobio posts encouraging update for Fabry gene therapy phase 1, 2 trials

Patients undergoing lentiviral gene therapies must first take the chemotherapy drug busulfan in a process called conditioning, which helps the gene-modified stem cells take root in their bone marrow. Avrobio uses therapeutic drug monitoring to tailor busulfan dosing to each patient, to improve the odds of success for its gene therapies while tamping down on side effects. Some patients may be more susceptible to infection and bleeding after conditioning, and they may suffer side effects like nausea, hair loss and mouth sores.

MGTA-117 is made up of an anti-CD117 antibody linked to amanitin, a cell-killing toxin. It is designed to target only hematopoietic, or blood-forming, stem cells and progenitor cells. Animal studies suggest it could clear space in bone marrow for gene-modified stem cells to take root, Magenta said in the statement. The company plans to wrap IND-enabling studies for the antibody-drug conjugate this year.

The deal comes on the heels of a busulfan-focused one for Avrobio. The company joined forces with Saladex Biomedical on Monday to develop a rapid blood test that monitors how quickly patients metabolize the drug. The hope is to get results in minutes, rather than the hours that current methods take, so dosing can be adjusted quickly.

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Developing new stem cell therapies requires more than a solo biologist having a eureka moment alone in the lab. Real progress relies on collaborations between biologists, engineers and physicians. Thats why The Eli and Edythe Broad Foundation has continued its support of two strategic initiatives: innovation awards bringing together teams of engineers and scientists from USC and Caltech, and clinical research fellowships for physician-scientists.

Engineering new approaches: The Broad Innovation Awards

For the fifth consecutive year, the Broad Innovation Awards are providing critical funding to USC-affiliated faculty members pursuing multi-investigator research collaborations related to stem cells. For the first year, these collaborations are also drawing on the expertise of biomedical engineers from Caltech. Each award provides $200,000 of funding for a one-year project.

Were very excited to be joining our colleagues at USC in pioneering new approaches to advancing stem cell research, said Stephen L. Mayo, chair of the Division of Biology and Biological Engineering at Caltech. Were thankful to The Broad Foundation for supporting cross-town collaborations between scientists with different expertise but common goals.

With support from a Broad Innovation Award, Andy McMahon, the director of the Eli and Edythe Broad Center for Regenerative Medicine and Stem Cell Research at USC, is collaborating with Caltech biomedical engineer Long Cai to leverage a new technology for understanding chronic kidney disease. The technology, called seqFISH, provides information about genetic activity taking place in intact tissueenabling the study of the interactions between cells in their native environments.

Dr. Cais seqFISH technology will provide an unprecedented insight into the cellular interplay underlying chronic kidney disease caused by a maladaptive response to acute kidney injury, said McMahon, who is the W.M. Keck Provost and University Professor of Stem Cell Biology and Regenerative Medicine, and Biological Sciences, as well as the chair of the Department of Stem Cell Biology and Regenerative Medicine at USC. We aim to better understand this maladaptive responsewhich is more common in malesin order to find new targets for preventing the progression to chronic kidney disease.

A second Broad Innovation Award brings together USC Stem Cell scientist Rong Lu and Caltech synthetic biologist Michael Elowitz. Their team will study the spatial organization of blood-forming stem and progenitor cells, also called hematopoietic stem and progenitor cells (HSPCs), which reside in the bone marrow. By pinpointing the locations of specific HSPCs, the scientists may find clues to explain why certain HSPCs are so dominantreplenishing the majority of the bodys blood and immune cells after a disruption such as a bone marrow transplantation.

Spatial advantages may be the primary drivers of what we refer to as the clonal dominance of certain HSPCs, said Lu, a Richard N. Merkin Assistant Professor of Stem Cell Biology and Regenerative Medicine, Biomedical Engineering, Medicine, and Gerontology at USC. Understanding the spatial competition between HSPCs could help improve bone marrow transplantation and provide new insights into aging and the development of diseases such as leukemiawhich are associated with clonal dominance.

Elowitz added: Thanks to the Broad Innovation Award and this exciting collaboration with Rong Lu, we will be able to bring a new, synthetic biology approach to record cell histories and read them out in individual cells within their native spatial context, providing new insights into fundamental questions in blood stem cell development.

A third Broad Innovation Award brings together three collaborators at USC: Michael Bonaguidi, an assistant professor of stem cell biology and regenerative medicine, biomedical engineering, and gerontology; Robert Chow, a professor of physiology and neuroscience, and biomedical engineering; and Jonathan Russin, an assistant professor of neurological surgery and associate surgical director for the USC Neurorestoration Center. Their project focuses on finding new approaches to treating epilepsy by studying neural cells called astroglia. These cells perform a variety of key functions that support the health of neurons in the brain, and they may also play a role in modulating epileptic seizures.

Although adults dont tend to generate many new brain cells, humans do produce a limited number of new astroglia, said Bonaguidi. We will examine these newborn astroglia at the single-cell level to better understand their role in epileptic patients, and to lay the groundwork for identifying new treatments.

The doctors are in: The Broad Clinical Research Fellowships

The Broad Clinical Research Fellowships are also entering their fifth consecutive year. These fellowships support stem cell research by physician-scientists and residents who intend to practice medicine in California.

These fellowships provide a very special opportunity for our medical residents to engage deeply in laboratory research, as a complement to their extensive training in patient care, said Laura Mosqueda, Dean of the Keck School of Medicine of USC. This valuable research experience gives them a much more complete perspective on how to meet the challenges of finding the best possible treatments for their patients.

A USC resident physician in general surgery, Kemp Anderson will spend his fellowship studying necrotizing enterocolitis, a very serious intestinal infection that affects nearly 10 percent of premature infants. Specifically, he will explore how a molecule involved in cellular communication, called farnesoid X receptor, or FXR, might contribute to this disease.

If FXR plays a role in compromising intestinal barrier function in these premature infants, then altering the activity of FXR could potentially yield treatment modalities for necrotizing enterocolitis, avoiding the morbidity and mortality associated with surgical intervention, said Anderson, who is performing the research under the mentorship of Christopher Gayer and Mark Frey at Childrens Hospital Los Angeles (CHLA). Im deeply appreciative of the benefactors and the selection committee for awarding me the Broad Clinical Fellowship, as it is allowing me devoted time to focus on this important project, and to become a more well-rounded physician through this academic pursuit.

Brittany Rocque, a resident physician in general surgery, will use her fellowship to seek better ways to predict, detect and diagnose immune rejection in patients who have undergone liver transplantation. Nearly 60 percent of pediatric patients and at least 15 percent of adult patients reject their liver transplants, and this can currently only be confirmed through an invasive surgical biopsy. Rocque is utilizing the technology Imaging Mass Cytometry to identify and analyze the types of immune cells involved in rejection.

My project has the potential to provide a noninvasive option to assess rejection in transplanted patients, and to expand our understanding of immune rejection, said Rocque, who is being co-mentored by Juliet Emamaullee and Shahab Asgharzadeh at CHLA. Im greatly looking forward to applying my passion for transplantation surgery in the context of basic science, and enhancing my appreciation for the nuances of research, thanks to the Broad Clinical Research Fellowship.

A hematology-oncology fellow who will be transitioning to a junior faculty position at USC next year, Caitlin ONeill will study a condition known as clonal hematopoiesis or CH, a phenomenon common in the aging population. CH involves genetic mutations that cause the expansion of a particular population of blood cells without leukemia or related malignancies. CH increases risks for certain health conditions including heart disease.

During her Broad Clinical Research Fellowship, ONeill will look at one mutation seen in patients with CH: a mutation to the gene called Tet methylcytosine dioxygenase 2, or TET2. ONeill will explore if this mutation promotes blood clots, inflammation and heart disease.

The goal is to inform therapies to prevent heart disease and leukemic progression in aging patients with CH, said ONeill, who is working with co-mentors Casey OConnell and Rong Lu at USC. Im very happy to be working on this project, with support from the Broad Clinical Research Fellowship, during my transition to becoming a faculty member at USC.

More:
Broad Foundation brings together stem cell scientists, engineers and physicians at University of Southern - Mirage News

Bone Marrow Stem Cells Comments Off on Broad Foundation brings together stem cell scientists, engineers and physicians at University of Southern – Mirage News | May 7th, 2020

One of the most talked-about topics today happens to be Mike Tyson appearing in shape and ready to appear in a boxing ring again. The former heavyweight champion of the world is gearing up for charity exhibition bouts thanks to Kings MMA coach Rafael Cordeiro.

Tyson (50-6, 2NC) wants to help those who are going through a tough time, like drug addicts. Relating to their situation, The 53-year-old is looking to make a difference while staying in shape. How exactly did he quickly get into fighting shape?

You know what I had done? I had stem-cell research therapy, Tyson ended up stating to Shaquille ONeal on Instagram Live, via The Sun. I feel like a different person but I cant comprehend why I feel this way. Its really wild what scientists can do.

Stem-cell therapy is the use of stem cells to treat or prevent a disease or condition. The process to carry it out is via bone marrow transplantation. Several athletes from all sports have undergone the treatment, including Hines Ward, Alex Rodriguez, Kobe Bryant, Tiger Woods, and Rafael Nadal. There are specific types of therapy treatments, but it is unknown what Tyson went through.

Tyson made his professional debut in 1985 and quickly climbed up the ranks in the boxing world. In his 28th fight, Tyson knocked out Trevor Berbick in the second round to win the WBC Heavyweight Title. He became the youngest world heavyweight champion of all time at 20. Following his last fight in 2005, he ballooned up to 325 pounds.

When Shaq talked about hurting himself while hanging and working out with his kids, Tyson helped explain why. With his new regiment, Tyson is lifting weights constantly and sparring multiple times a day.

Thats just because you havent done it for a while, Tyson went on to say. If you continue to do it consistently youll be back to normal. Its just like me, I havent boxed or hit the bag for 15 years it has been three days so far and I feel incredible.

It is unknown who Tyson will be facing in his return. He has been offered, however, $1 million to fight in Australia to face some of the top stars in rugby and Australian football.

See the rest here:
Tyson Reveals What Helped Him Train For Exhibition Bouts | FIGHT SPORTS - FIGHT SPORTS

Bone Marrow Stem Cells Comments Off on Tyson Reveals What Helped Him Train For Exhibition Bouts | FIGHT SPORTS – FIGHT SPORTS | May 7th, 2020

"global Stem Cell Therapy market"

A new market study, titled Covid-19 Impact on Global Stem Cell Therapy Market Size, Status and Forecast 2020-2026 has been featured on WiseGuyReports.

The global Stem Cell Therapy market research offers a comprehensive overall market analysis focused on the latest findings. The introduction portion includes a brief overview of the industry, along with the product and service descriptions. This also includes the main applications for all end-user industries. The report also presents market prospects along with the forecast, with the study covering the period 2020-2026. The report includes an in-depth analysis of the major factors that could decide the market's trajectory in the coming years.

Stem-cell therapy is the use of stem cells to treat or prevent a disease or condition. Bone marrow transplant is the most widely used stem-cell therapy, but some therapies derived from umbilical cord blood are also in use.

In the last several years, global stem cell therapy market developed fast at a average growth rate of 46.81%.

Competition Analysis

In the competitive analysis section of the report, leading as well as prominent players of the global Stem Cell Therapy market are broadly studied on the basis of key factors. The report offers comprehensive analysis and accurate statistics on revenue by the player for the period 2015-2020. It also offers detailed analysis supported by reliable statistics on price and revenue (global level) by player for the period 2015-2020.

The following players are covered in this report:

Osiris Therapeutics

NuVasive

Chiesi Pharmaceuticals

JCR Pharmaceutical

Pharmicell

Medi-post

Anterogen

Molmed

Takeda (TiGenix)

This report also analyses the impact of Coronavirus COVID-19 on the Stem Cell Therapy industry.

Regional and Country-level Analysis

The report offers an exhaustive geographical analysis of the global Stem Cell Therapy market, covering important regions, viz, North America, Europe, China, Japan, Southeast Asia, India and Central & South America. It also covers key countries (regions), viz, U.S., Canada, Germany, France, U.K., Italy, Russia, China, Japan, South Korea, India, Australia, Taiwan, Indonesia, Thailand, Malaysia, Philippines, Vietnam, Mexico, Brazil, Turkey, Saudi Arabia, UAE, etc.

The report includes country-wise and region-wise market size for the period 2015-2026. It also includes market size and forecast by each application segment in terms of revenue for the period 2015-2026.

Stem Cell Therapy Breakdown Data by Type

Autologous

Allogeneic

Stem Cell Therapy Breakdown Data by Application

Musculoskeletal Disorder

Wounds & Injuries

Cornea

Cardiovascular Diseases

Others

Request Free Sample Report athttps://www.wiseguyreports.com/sample-request/5252275-covid-19-impact-on-global-stem-cell-therapy

Table of Contents

1 Report Overview

2 Global Growth Trends by Regions

3 Competition Landscape by Key Players

4 Breakdown Data by Type (2015-2026)

5 Stem Cell Therapy Breakdown Data by Application (2015-2026)

6 North America

7 Europe

8 China

9 Japan

10 Southeast Asia

11 India

12 Central & South America

13 Key Players Profiles

14 Analyst's Viewpoints/Conclusions

NOTE: Our team is studying Covid-19 and its impact on various industry verticals and wherever required we will be considering Covid-19 footprints for a better analysis of markets and industries. Cordially get in touch for more details.

About Us:

Wise Guy Reports is part of the Wise Guy Research Consultants Pvt. Ltd. and offers premium progressive statistical surveying, market research reports, analysis & forecast data for industries and governments around the globe.

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Impact of Covid-19 Outbreak on Global Stem Cell Therapy Market Future Opportunities and Forecast Analysis 2020-2026 - Press Release - Digital Journal

Bone Marrow Stem Cells Comments Off on Impact of Covid-19 Outbreak on Global Stem Cell Therapy Market Future Opportunities and Forecast Analysis 2020-2026 – Press Release – Digital Journal | May 7th, 2020

FDA Approves AstraZenecas Farxiga for Heart Failure in Adults with Reduced Ejection Fraction

The U.S. Food and Drug Administration (FDA) announced on Tuesday that it has approved dapagliflozin, also known under the brand name Farxiga, for the treatment of heart failure in adults with reduced ejection fraction. The drug can potentially reduce the risk of cardiovascular death and hospitalization for heart failure.

AstraZenecas Farxiga is now the first in its drug class of sodium-glucose co-transporter 2 (SGLT2) inhibitors to be approved to treat adults with the New York Heart Associations functional class II-IV heart failure with reduced ejection fraction. AstraZeneca was granted with the approval of Farxiga related to heart failure by the FDA.

In a clinical trial, Farxiga appeared to improve survival and reduce the need for hospitalization in adults with heart failure and reduced ejection fraction.

To determine the efficacy of the drug, researchers looked at the number of instances of cardiovascular death, hospitalization for heart failure and urgent heart failure visits. Some trial participants were given a once-daily dose of 10mg of Farxiga, while others were given a placebo. After approximately 18 months, those who were given Farxiga had fewer cardiovascular deaths, hospitalizations for heart failure and urgent heart failure visits compared to their counterparts.

Heart failure is a serious health condition that contributes to one in eight deaths in the U.S. and impacts nearly 6.5 million Americans, said Norman Stockbridge, M.D., Ph.D., director of the Division of Cardiology and Nephrology in the FDAs Center for Drug Evaluation and Research. This approval provides patients with heart failure with reduced ejection fraction an additional treatment option that can improve survival and reduce the need for hospitalization.

Farxiga can cause side effects including dehydration, urinary tract infections and genetical yeast infections. It can also potentially result in serious cases of necrotizing fasciitis of the perineum in people with diabetes and low blood sugar when combined with insulin.

On Tuesday, BioCardia, Inc. also announced positive preclinical datasupporting its new drug application for anti-inflammatory cell therapy for heart failure. BioCardias allogenic neurokinin 1 receptor positive mesenchymal stem cell (NK1R+ MSC) therapy appeared to improve heart function in a study. NK1R+ MSC is being marketed under the name CardiALLO.

Alexanderstock23 / Shutterstock

Researchers looked at 26 animals treated with both low dose and high dose CardiALLO in their study. Echocardiographic measures of cardiac ejection fraction, fractional shortening and cardiac outflow all notably improved in the animals.

In light of these positive data on our allogenic NK1R+ MSC therapy, we expect to meet our internal timeline to complete our submission to the FDA for our first indication for CardiALLO, and potentially receive IND acceptance by the end of the second quarter, said BioCardia Chief Scientific Officer Ian McNiece, PhD. The MSCs that were studied are subtypes of MSC that we have delivered previously in our co-sponsored trials, which we believe have enhanced potency over MSC generated from unselected bone marrow cells. We look forward to seeing additional data from this animal study that are currently being analyzed, including histology and pathology of the heart and lungs.

BioCardia also intends to submit an IND for the use of NK1R+ MSC delivered via intravenous infusion for the treatment of Acute Respiratory Distress Syndrome caused by COVID-19.

Approximately 6.5 million adults in the U.S. are living with heart failure, according to the Centers for Disease Control and Protection. In 2017, it was a contributing cause of death in one out of eight people.

See the original post:
FDA Approves AstraZeneca's Farxiga for Heart Failure in Adults with Reduced Ejection Fraction - PharmaLive

Bone Marrow Stem Cells Comments Off on FDA Approves AstraZeneca’s Farxiga for Heart Failure in Adults with Reduced Ejection Fraction – PharmaLive | May 7th, 2020

Laila Anderson continues to make an impact in the lives of others one year after she became part of the Blues run to the 2019 Stanley Cup title.

On Tuesday, Anderson, who has battled HLH, a disease that causes the body to make too many immune cells, took part in an all-day livestream event called Couch2Cure to benefit Be the Match. It served as not only a fundraiser, but also a call for people to become donors for patients seeking blood stem cell matches. She was joined by FOX play-by-play man Joe Buck and Blues PA announcer Tom Calhoun.

The event was a success, raising$1.45 million and resulting in 36,000 registries to the Be the Match program.

Laila spoke with NBC Sports Kathryn Tappen on Wednesday to talk about the Couch2Cure event, the importance of the Be the Match registry, and how shes doing one year after the Blues triumph.

MORE LAILA ANDERSON: Laila introduces Blues All-Stars with gusto Blues superfan enjoying life one year after bone marrow transplant Laila meets bone marrow donor Laila gets moment with Stanley Cup

Sean Leahy is a writer for Pro Hockey Talk on NBC Sports. Drop him a line at phtblog@nbcsports.com or follow him on Twitter @Sean_Leahy.

British Columbia Premier John Horgan has offered the NHL a place to play if the league can find a way to resume the season.

Speaking at a COVID-19 media briefing Wednesday, Horgan said he has written a letter to both NHL Commissioner Gary Bettman and NHL Players Association head Donald Fehr to let them know B.C. is a place to potentially restart the NHL assuming the games would be played without audiences, but instead played for television.

The NHL suspended its season March 12 with 189 regular-season games left.

Dr. Bonnie Henry, British Columbias provincial health officer, was asked Monday about Vancouver hosting NHL games with no fans and said: These are the types of things that we need to think about.

Ontario Premier Doug Ford said Tuesday the Maple Leafs parent company, MLSE, has been in contact with the province about the possibility of Toronto serving as a hockey pod for teams as well. Alberta Premier Jason Kenney and Bettman spoke last month about Edmonton as another potential hub city.

Welcome to the PHT Morning Skate, a collection of links from around the hockey world. Have a link you want to submit? Email us atphtblog@nbcsports.com.

A really nice read about ex-Blackhawks president John McDonoughs friendship with 11-year-old Cammy Babiarz, who is unable to walk or talk because of Rett Syndrome a rare developmental disorder. [Midway Minute]

It didnt last very long, but at one point in time Michael Jordan was a minority owner of the Capitals. [ESPN]

Economies are beginning to open up again, so too are hockey rinks in the U.S. [The Hockey News]

Georges Laraque opens about his up and down relationship with his father. [Vice]

How the 2011-12 Kings became unlikely Stanley Cup champions. [The Score]

Comparing Brady Tkachuks early days in the NHL to that of Mark Stones. [Silver Seven Sens]

What if some of NHLs all-time best hadnt run into historic dynasties? [Sportsnet]

Looking ahead to whats expected to be an intriguing 2022 NHL draft class. [Stephen Ellis]

Finally, the Wild music video you were looking for:

Sean Leahy is a writer for Pro Hockey Talk on NBC Sports. Drop him a line at phtblog@nbcsports.com or follow him on Twitter @Sean_Leahy.

After pulling the plug on the 2019-20 season in March, the KHL has decided there will be no champion and the Gagarin Cup will not be awarded for the first time in league history.

Due to this decision, the league has equally ranked the eight teams that advanced to the second round of the playoffs:Ak Bars Kazan,Barys Nur-Sultan,CSKA Moscow,Dynamo Moscow,Jokerit Helsinki,Salavat Yulaev Ufa,Sibir Novosibirsk, and SKA St. Petersburg.

From the KHL:

With the season incomplete, there is no way that a Gagarin Cup winner and other prize winners can be fairly chosen based on the results of the regular season. To announce a champion based on the regular season and one round of the playoffs would violate the sporting integrity of the competition.

The Russian Hockey Federation has drawn up a separate procedure to determine the Russian Champion for the 2019-20 season, and to award silver and bronze medals to the second and third-placed teams. This proposal will be submitted to the KHL Board of Directors for approval.

The Gagarin Cup playoffs were halted in the conference semifinals after Jokerit and Barys pulled out due to the coronavirus pandemic. Originally, the KHL was planning for a one-week break to come up with a new format for the four remaining teams. They later chose to end the season completely.

Im sure that the league has taken a fair and balanced decision in this difficult situation, said KHL president Alexei Morozov. This was the only choice that respects our sporting principles. For the first time in history, the KHL season had to be interrupted and ultimately curtailed. That was a tough, but essential decision, dictated by the need to protect the health of the nation.

MORE: Bill Peters signs two-year deal to coach KHLs Avtomobilist

Sean Leahy is a writer for Pro Hockey Talk on NBC Sports. Drop him a line at phtblog@nbcsports.com or follow him on Twitter @Sean_Leahy.

Brendan Leipsic of the Capitals has apologized for comments made on social media that were leaked online Wednesday. Panthers prospect Jack Rodewald was also in the Instagram group chat where remarks were made about the appearances of Meaghan Pearson, whose husband, Tanner, plays for the Canucks, Lauren Kyle, the girlfriend of Oilers forward Connor McDavid, and other women.

Yesterday my friends Instagram account was hacked and an individual circulated images that are representative of private conversations I was a part of, Leipsic wrote in an apology note posted on Twitter. I fully recognize how inappropriate and offensive these comments are and sincerely apologize to everyone for my actions. I am committed to learning from this and becoming a better person by taking time to determine how to move forward in an accountable, meaningful way. I am truly sorry.

The NHL released a statement of their own stating they will address this with the players involved.

The National Hockey League strongly condemns the misogynistic and reprehensible remarks made by players Brendan Leipsic and Jack Rodewald in a private group chat that has surfaced on social media. There is no place in our League for such statements, attitudes and behavior, no matter the forum. We will address this inexcusable conduct with the clubs and players involved.

Leipsic, who has played 61 games with the Capitals this season, is on his fifth team in five years since entering the NHL. His current team wrote in a statement,We are aware of the unacceptable and offensive comments made by Brendan Leipsic in a private conversation on social media. We will handle this matter internally.

Sean Leahy is a writer for Pro Hockey Talk on NBC Sports. Drop him a line at phtblog@nbcsports.com or follow him on Twitter @Sean_Leahy.

See original here:
Laila Anderson on Couch2Cure event, importance of Be the Match registry - NBCSports.com

Bone Marrow Stem Cells Comments Off on Laila Anderson on Couch2Cure event, importance of Be the Match registry – NBCSports.com | May 7th, 2020

The global Cell Therapy Manufacturing Market research report thoroughly explains each and every aspect related to the Cell Therapy Manufacturing Market, which facilitates the reports reader to study and evaluate the upcoming market trend and execute the analytical data to promote the business. The growth trend forecasted on account of thorough examination offers in-depth information regarding the global Cell Therapy Manufacturing Market. A pathway of development is offered by the market to the several connected networks of businesses under it, which include different firms, industries, organizations, vendors, distributors, and local manufacturers too. All the key Cell Therapy Manufacturing Market players compete with each other by offering better products and services at a reasonable price in order to grab significant share at the regional and global level market.

Cell therapy is one of the most promising healthcare procedure for restoration of damaged tissue. Cell therapies have huge potential for the wide range of disease treatment including tissue degradation, immune deficiency, metabolic disorders, and cancer. Cell therapy are categorized into two types allogeneic (cells from third party donor) and autologous (cells from ones own body). Cell therapy has gained significant traction in recent past and currently it under commercial development. Main objective of cell therapy is to re-establish the lost function of tissues and cells rather than to produce a new organ. Some cell therapies have been established and permitted for clinical trial. For instance, in 2013 a stage 2 clinical trial was completed for transplantation of bone marrow derived stem cells in affected knee by rheumatoid arthritis. The growth of this technique is also increased due to involvement of the government agencies. For instance, innovate U.K. 2014 report showed that government agreed to fund US$15.3 million in cell therapy manufacturing market.ription

Get a Sample Copy of this Report: https://www.coherentmarketinsights.com/insight/request-sample/1728

This report sample includesBrief Introduction to the research report.Table of Contents (Scope covered as a part of the study)Top players in the marketResearch framework (presentation)Research methodology adopted by Coherent Market Insights

The report incorporates an estimated impact of strict standards and regulations set by the government over the market in the upcoming years. The market report also comprises exhaustive research done using several analytical tools such as SWOT analysis to identify the market growth pattern.

Top Manufacturers in GlobalCell Therapy ManufacturingMarket Includes:Pharmicell, Merck Group, Dickinson and Company, Thermo Fisher, Lonza Group, Miltenyi Biotec GmBH, Takara Bio Group, STEMCELL Technologies, Cellular Dynamics International, Becton, Osiris Therapeutics, Bio-Rad Laboratories, Inc., Anterogen, MEDIPOST, Holostem Terapie Avanazate, Pluristem Therapeutics, Brammer Bio, CELLforCURE, Gene Therapy Catapult EUFETS, MaSTherCell, PharmaCell, Cognate BioServices and WuXi AppTec.

Regions & Countries Mentioned In The Cell Therapy Manufacturing Market Report:

North America ( United States)

Europe ( Germany, France, UK)

Asia-Pacific ( China, Japan, India)

Latin America ( Brazil)

The Middle East & Africa

Key Highlights of the Table of Contents:

Cell Therapy Manufacturing Market Study Coverage: It includes key manufacturers covered, key market segments, the scope of products offered in the global market, years considered, and study objectives. Furthermore, it tuches the segmentation study provided in the report on the basis of the type of product and applications.

Cell Therapy Manufacturing Market Executive Summary: This section emphasizes on the key studies, market growth rate,Competitive landscape, market drivers, trends, and issues.

Cell Therapy Manufacturing Market Production by Region: The report provides information related to import and export, production, revenue, and key players of all regional markets studied are covered in this section.

Cell Therapy Manufacturing Market Profile of Manufacturers: Analysis of each market player profiled is detailed in this section. This also provides SWOT analysis, products, production, value, capacity, and other vital factors of the individual player.

Buy This Complete A Business Report: https://www.coherentmarketinsights.com/insight/buy-now/1728

Table of Contents

Report Overview:It includes the Cell Therapy Manufacturing market study scope, players covered, key market segments, market analysis by application, market analysis by type, and other chapters that give an overview of the research study.

Executive Summary:This section of the report gives information about Cell Therapy Manufacturing market trends and shares, market size analysis by region and analysis of global market size. Under market size analysis by region, analysis of market share and growth rate by region is provided.

Profiles of International Players:Here, key players of the Cell Therapy Manufacturing market are studied on the basis of gross margin, price, revenue, corporate sales, and production. This section gives a business overview of the players and shares their important company details.

Regional Study:All of the regions and countries analyzed in the Cell Therapy Manufacturing market report is studied on the basis of market size by application, the market size by product, key players, and market forecast.

An Overview of the Impact of COVID-19 on this Market:

The pandemic of COVID-19 continues to expand and impact over 175 countries and territories. Although the outbreak appears to have slowed in China, COVID-19 has impacted globally. The pandemic could affect three main aspects of the global economy: production, supply chain, and firms and financial markets. National governments have announced largely uncoordinated, country-specific responses to the virus. As authorities encourage social distancing and consumers stay indoors, several businesses are hit. However, coherent, coordinated, and credible policy responses are expected to offer the best chance at limiting the economic fallout.

National governments and international bodies are focused on adopting collaborative efforts to encourage financial institutions to meet the financial needs of customers and members affected by the coronavirus. However, there are some sectors that have remained unscathed from the impact of the pandemic and there are some that are hit the hardest.

We, at Coherent Market Insights, understand the economic impact on various sectors and markets. Using our holistic market research methodology, we are focused on aiding your business sustain and grow during COVID-19 pandemics. With deep expertise across various industries-no matter how large or small- and with a team of highly experienced and dedicated analysts, Coherent Market Insights will offer you an impact analysis of coronavirus outbreak across industries to help you prepare for the future.

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Cell Therapy Manufacturing Market 2020 Coronavirus (Covid-19) Business Impact Size Will Escalate Rapidly in the Near Future 3w Market News Reports -...

Bone Marrow Stem Cells Comments Off on Cell Therapy Manufacturing Market 2020 Coronavirus (Covid-19) Business Impact Size Will Escalate Rapidly in the Near Future 3w Market News Reports -… | May 7th, 2020

British Columbia Premier John Horgan has offered the NHL a place to play if the league can find a way to resume the season.

Speaking at a COVID-19 media briefing Wednesday, Horgan said he has written a letter to both NHL Commissioner Gary Bettman and NHL Players Association head Donald Fehr to let them know B.C. is a place to potentially restart the NHL assuming the games would be played without audiences, but instead played for television.

The NHL suspended its season March 12 with 189 regular-season games left.

Dr. Bonnie Henry, British Columbias provincial health officer, was asked Monday about Vancouver hosting NHL games with no fans and said: These are the types of things that we need to think about.

Ontario Premier Doug Ford said Tuesday the Maple Leafs parent company, MLSE, has been in contact with the province about the possibility of Toronto serving as a hockey pod for teams as well. Alberta Premier Jason Kenney and Bettman spoke last month about Edmonton as another potential hub city.

Laila Anderson continues to make an impact in the lives of others one year after she became part of the Blues run to the 2019 Stanley Cup title.

On Tuesday, Anderson, who has battled HLH, a disease that causes the body to make too many immune cells, took part in an all-day livestream event called Couch2Cure to benefit Be the Match. It served as not only a fundraiser, but also a call for people to become donors for patients seeking blood stem cell matches. She was joined by FOX play-by-play man Joe Buck and Blues PA announcer Tom Calhoun.

The event was a success, raising$1.45 million and resulting in 36,000 registries to the Be the Match program.

Laila spoke with NBC Sports Kathryn Tappen on Wednesday to talk about the Couch2Cure event, the importance of the Be the Match registry, and how shes doing one year after the Blues triumph.

MORE LAILA ANDERSON: Laila introduces Blues All-Stars with gusto Blues superfan enjoying life one year after bone marrow transplant Laila meets bone marrow donor Laila gets moment with Stanley Cup

Sean Leahy is a writer for Pro Hockey Talk on NBC Sports. Drop him a line at phtblog@nbcsports.com or follow him on Twitter @Sean_Leahy.

Welcome to the PHT Morning Skate, a collection of links from around the hockey world. Have a link you want to submit? Email us atphtblog@nbcsports.com.

A really nice read about ex-Blackhawks president John McDonoughs friendship with 11-year-old Cammy Babiarz, who is unable to walk or talk because of Rett Syndrome a rare developmental disorder. [Midway Minute]

It didnt last very long, but at one point in time Michael Jordan was a minority owner of the Capitals. [ESPN]

Economies are beginning to open up again, so too are hockey rinks in the U.S. [The Hockey News]

Georges Laraque opens about his up and down relationship with his father. [Vice]

How the 2011-12 Kings became unlikely Stanley Cup champions. [The Score]

Comparing Brady Tkachuks early days in the NHL to that of Mark Stones. [Silver Seven Sens]

What if some of NHLs all-time best hadnt run into historic dynasties? [Sportsnet]

Looking ahead to whats expected to be an intriguing 2022 NHL draft class. [Stephen Ellis]

Finally, the Wild music video you were looking for:

Sean Leahy is a writer for Pro Hockey Talk on NBC Sports. Drop him a line at phtblog@nbcsports.com or follow him on Twitter @Sean_Leahy.

After pulling the plug on the 2019-20 season in March, the KHL has decided there will be no champion and the Gagarin Cup will not be awarded for the first time in league history.

Due to this decision, the league has equally ranked the eight teams that advanced to the second round of the playoffs:Ak Bars Kazan,Barys Nur-Sultan,CSKA Moscow,Dynamo Moscow,Jokerit Helsinki,Salavat Yulaev Ufa,Sibir Novosibirsk, and SKA St. Petersburg.

From the KHL:

With the season incomplete, there is no way that a Gagarin Cup winner and other prize winners can be fairly chosen based on the results of the regular season. To announce a champion based on the regular season and one round of the playoffs would violate the sporting integrity of the competition.

The Russian Hockey Federation has drawn up a separate procedure to determine the Russian Champion for the 2019-20 season, and to award silver and bronze medals to the second and third-placed teams. This proposal will be submitted to the KHL Board of Directors for approval.

The Gagarin Cup playoffs were halted in the conference semifinals after Jokerit and Barys pulled out due to the coronavirus pandemic. Originally, the KHL was planning for a one-week break to come up with a new format for the four remaining teams. They later chose to end the season completely.

Im sure that the league has taken a fair and balanced decision in this difficult situation, said KHL president Alexei Morozov. This was the only choice that respects our sporting principles. For the first time in history, the KHL season had to be interrupted and ultimately curtailed. That was a tough, but essential decision, dictated by the need to protect the health of the nation.

MORE: Bill Peters signs two-year deal to coach KHLs Avtomobilist

Sean Leahy is a writer for Pro Hockey Talk on NBC Sports. Drop him a line at phtblog@nbcsports.com or follow him on Twitter @Sean_Leahy.

Brendan Leipsic of the Capitals has apologized for comments made on social media that were leaked online Wednesday. Panthers prospect Jack Rodewald was also in the Instagram group chat where remarks were made about the appearances of Meaghan Pearson, whose husband, Tanner, plays for the Canucks, Lauren Kyle, the girlfriend of Oilers forward Connor McDavid, and other women.

Yesterday my friends Instagram account was hacked and an individual circulated images that are representative of private conversations I was a part of, Leipsic wrote in an apology note posted on Twitter. I fully recognize how inappropriate and offensive these comments are and sincerely apologize to everyone for my actions. I am committed to learning from this and becoming a better person by taking time to determine how to move forward in an accountable, meaningful way. I am truly sorry.

The NHL released a statement of their own stating they will address this with the players involved.

The National Hockey League strongly condemns the misogynistic and reprehensible remarks made by players Brendan Leipsic and Jack Rodewald in a private group chat that has surfaced on social media. There is no place in our League for such statements, attitudes and behavior, no matter the forum. We will address this inexcusable conduct with the clubs and players involved.

Leipsic, who has played 61 games with the Capitals this season, is on his fifth team in five years since entering the NHL. His current team wrote in a statement,We are aware of the unacceptable and offensive comments made by Brendan Leipsic in a private conversation on social media. We will handle this matter internally.

Sean Leahy is a writer for Pro Hockey Talk on NBC Sports. Drop him a line at phtblog@nbcsports.com or follow him on Twitter @Sean_Leahy.

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NHL offered place to play in British Columbia - NBCSports.com

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A recent market study on the global Hematopoietic Stem Cell Transplantation (HSCT) market reveals that the global Hematopoietic Stem Cell Transplantation (HSCT) market is expected to reach a value of ~US$ XX by the end of 2029 growing at a CAGR of ~XX% during the forecast period (2019-2029). The impact of the COVID-19 pandemic on the global Hematopoietic Stem Cell Transplantation (HSCT) market is discussed in the presented study.

The Hematopoietic Stem Cell Transplantation (HSCT) market study encloses a thorough analysis of the overall competitive landscape and the company profiles of leading market players involved in the global Hematopoietic Stem Cell Transplantation (HSCT) market. Further, the presented study offers accurate insights pertaining to the different segments of the global Hematopoietic Stem Cell Transplantation (HSCT) market such as the market share, value, revenue, and more.

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The following doubts are addressed in the market report:

Key Highlights of the Hematopoietic Stem Cell Transplantation (HSCT) Market Report

The presented report segregates the Hematopoietic Stem Cell Transplantation (HSCT) market into different segments to ensure the readers gain a complete understanding of the different aspects of the Hematopoietic Stem Cell Transplantation (HSCT) market.

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Segmentation of the Hematopoietic Stem Cell Transplantation (HSCT) market

Competitive Outlook

This section of the report throws light on the recent mergers, collaborations, partnerships, and research and development activities within the Hematopoietic Stem Cell Transplantation (HSCT) market on a global scale. Further, a detailed assessment of the pricing, marketing, and product development strategies adopted by leading market players is included in the Hematopoietic Stem Cell Transplantation (HSCT) market report.

Companies Mentioned in the Report

The report profiles key manufacturers in the hematopoietic stem cell transplantation (HSCT) Market based on various attributes such as company details, SWOT analysis, strategic overview, financials, and business overview. Major players profiled in this report include Regen Biopharma, Inc., Escape Therapeutics, Inc., Lonza Group Ltd., and Pluristem Therapeutics Inc.

The global hematopoietic stem cell transplantation (HSCT) Market has been segmented as follows:

By Transplant Type

By Disease Indication

By Application

By Region

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Global trade impact of the Coronavirus Hematopoietic Stem Cell Transplantation (HSCT) Market Shares, Strategies and Forecast Worldwide, 2019-2027 -...

Bone Marrow Stem Cells Comments Off on Global trade impact of the Coronavirus Hematopoietic Stem Cell Transplantation (HSCT) Market Shares, Strategies and Forecast Worldwide, 2019-2027 -… | May 6th, 2020

As the world continues to battle the coronavirus pandemic, scientists are looking towards gene therapy to find ways to develop vaccines for the Covid-19 virus. Gene therapy itself was developed based on how viruses work.

When a virus attacks a host, it introduces its genetic material into the host cell as part of its replication cycle. The genetic material serves as an instruction manual on how to produce more copies of the virus, hijacking the host body's normal production machinery to serve the needs of the virus. The host cells then produce additional copies of the virus, leading to more host cells being infected.

Like animals, humans have found a way to domesticate viruses as well, i.e., direct the virus's function to achieve favorable results, which is prominent in gene therapy. Such viruses which physically insert their genes into the host's genome could instead be used to carry "good" genes into a human cell. Scientists would first remove the genes in the virus that cause diseases, and replace those genes with genes encoding the desired effect.

All of this sounds quite sci-fi but it has been done numerous times in the past. Peter Kolchinsky, a virologist and a biotechnology investor, compiled how different viruses have been used for gene therapy in the past.

Kolchinsky tweeted, "SARS2 is a scary menace, but did you know that we've domesticated viruses? Like wolves vs dogs, we've tamed them, including some deadly ones, to perform many useful functions (and may help us stop SARS2)."

The human immunodeficiency virus (HIV) has killed millions of people. It works by disabling the host body's immune system until it can't defend the person against common, normally mild pathogens. Kolchinsky explained that HIV's special trick is to integrate its genome into that of the host body's cells.

This feature of HIV is used for gene therapy, as explained before, by replacing a chunk of the virus's genome with the hemoglobin gene to insert it into bone marrow stem cells of patients with sickle cell anemia, whose hemoglobin genes are malfunctioning.

Kolchinsky also tweeted, "Adenoviruses typically cause mild infections, including common colds. These, too, we are trying to use for gene therapies, particularly when we just want to temporarily make a protein in cells. One company is developing such an adenovirus gene therapy for heart disease to induce growth of new blood vessels when old ones are clogged. Another is using this virus to make oral vaccines that would otherwise require injection (eg flu vaccine pill). When we use a virus to deliver code for making something in cells, we call that a virus vector."

There is now a wealth of clinical experience with numerous vector types that include primarily vaccinia, measles, vesicular stomatitis virus (VSV), polio, reovirus, adenovirus, lentivirus, -retrovirus, adeno-associated virus (AAV) and herpes simplex virus (HSV).

However, as with all other procedures, viral vector-gene therapy has associated risks. Viruses can usually infect more than one type of cell, so, when viralvectorsare used to carrygenesinto the body, they might infect healthy cells as well as cancer cells.

Another danger is that the new gene might be inserted in the wrong location in the DNA, possibly causing harmful mutations to the DNA or even cancer. Moreover, when viruses are used to deliver DNA to cells inside the patient's body, there is a slight chance that this DNA could unintentionally be introduced into the patients reproductive cells. If this happens, it could produce changes that may be passed on if a patient has children after treatment.

One study to help find a vaccine for Covid-19 aims to use the principles behind gene therapy to get the vaccine ready. The researchers' method uses a harmless virus as a vector to bring DNA into the patient's cells. The DNA should then instruct the cells to make a coronavirus protein that would stimulate the immune system to fight off future infections.

While a mass-produced vaccine may still take a while, this study is one of at least 90 vaccine projects around the world trying to find a cure for Covid-19. However, some experts are worried that a vaccine may never be available. According to our previous report, Dr David Nabarro, a professor of global health at Imperial College London, who also serves as a special envoy to the WHO on Covid-19, said, "There are some viruses that we still do not have vaccines against."

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Can gene therapy help develop coronavirus vaccine? Researchers banking on this technology for breakthrough - MEAWW

Bone Marrow Stem Cells Comments Off on Can gene therapy help develop coronavirus vaccine? Researchers banking on this technology for breakthrough – MEAWW | May 6th, 2020

MIKE TYSON revealed how he has started training again having not thrown a punch for a staggering 15 years - and is being aided by stem-cell research therapy.

The Baddest Man on the Planet, who hung up his gloves in 2005 following defeat by Kevin McBride, wouldn't elaborate on why he was having the treatment but added that it was 'really wild what scientists can do'.

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Tyson was having an Instagram chat with basketball legend Shaquille O'Neal when he revealed that he had been training for the previous three days after 15 years away - and his new health regime.

Iron Mike said: "You know what I had done? I had stem-cell research therapy.

"I feel like a different person but I can't comprehend why I feel this way. It's really wild what scientists can do."

Stem-cell therapy is the use of stem cells to treat or prevent a disease or condition that usually takes the form of a bone marrow transplantation.

He did not reveal what the exact condition was that was being treated.

Despite letting the gloves gather dust in the corner, it didn't take long for Iron Mike to show off that lethal punching power.

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The 53-year-old delighted fans in lockdown by uploading a viral video of him laying into a punchbag with his trademark speed and power.

Tyson's weight ballooned after retiring following battles with drug addiction and depression.

But he has since partnered with a new trainer, MMA coach Rafael Cordeiro, to kick-start his training after sensationally revealing his 15-year break.

During their chat, basketball legend O'Neal revealed how he ached for three days after playing with his sons.

Tyson responded: "That's just because you haven't done it for a while.

WHAT IS STEM CELL TREATMENT USED FOR?

Stem cell transplants are carried out when bone marrow is damaged or isnt able to produce healthy blood cells.

It can also be used to replace damaged blood cells as the result of intensive cancer treatment.

Here are conditions that stem cell transplants can be used to treat:

"If you continue to do it consistently you'll be back to normal.

"It's just like me, I haven't boxed or hit the bag for 15 years - it has been three days so far and I feel incredible."

Tyson, who has a 50-6 record, is reportedly gearing up for a sensational return amid plans to compete in exhibition bouts for charity later this year.

He told rapper T.I. last month: "I've been hitting the mitts for the last week.

"That's been tough, my body is really jacked up and really sore from hitting the mitts.

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"I've been working out, I've been trying to get in the ring, I think I'm going to box some exhibitions and get in shape.

"I want to go to the gym and get in shape to be able to box three or four-round exhibitions for some charities and stuff.

"Some charity exhibitions, make some money, help some homeless and drug-affected motherf****er like me."

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Mike Tyson, 53, reveals he had stem cell research therapy and hadnt hit bags for 15 YEARS before returning t - The Sun

Bone Marrow Stem Cells Comments Off on Mike Tyson, 53, reveals he had stem cell research therapy and hadnt hit bags for 15 YEARS before returning t – The Sun | May 5th, 2020

Veteran Bollywood actor Rishi Kapoor, who was battling leukemia for the last two years, died at Mumbais Sir HN Reliance Foundation Hospital on Thursday (April 30) morning. He was 67. Rishi Kapoor got treated for leukemia in the US for a year and had returned to India in September 2019.

Leukemia is a blood cancer caused by a rise in the number of white blood cells in your body. Those white blood cells crowd out the red blood cells and platelets that your body needs to be healthy. The extra white blood cells dont work right. A cancer of blood-forming tissues, hindering the body's ability to fight infection.

Dr Rahul Bhargava, Director, Haematology, Haemato Oncology and Bone Marrow Transplant, Fortis Memorial Research Institute, Gurugram says:

Leukemia, often fatal, is a blood cancer. As with cancer, it is associated with an exponential rise in the number of white blood cells. Symptoms begin to manifest slowly and can often be mistaken for something else sweating uncontrollably, shaking, fever, vomiting. It is caused by huge was is reduced focus on ones own health.

Intake of processed food, soda, artificial and synthetic sweets and preservatives, pesticide use in farms, lack of physical exercise, excessive tobacco and drug consumption, excessive alcohol intake, caffeine consumption are the main culprits causing cancer. Family history also plays an important role in a leukemia diagnosis. Only through screening can it be identified and if caught early managed. One needs to be mindful and ensure that they are employing good lifestyle habits.

This means eating green leafy vegetables and fruits, going for a 30 minutes walk every day or engaging in any other form of exercise for 30 minutes a day, drinking adequate amount of water to keep flushing out the toxins, controlling or completely eliminating our use of tobacco and other unnatural substances. Processed food and sugars should be consumed in control, they are the single largest cause of mutation to cells.

Leukemia symptoms vary, depending on the type of leukemia. Common leukemia signs and symptoms include:

Leukemia can also cause symptoms in organs that have been infiltrated or affected by the cancer cells. For example, if the cancer spreads to the central nervous system, it can cause headaches, nausea and vomiting, confusion, loss of muscle control, and seizures.

Leukemia can also spread to other parts of your body, including:

The major types of leukemia are:

Blood has three types of cells: white blood cells that fight infection, red blood cells that carry oxygen, and platelets that help blood clot. Every day, your bone marrow makes billions of new blood cells, and most of them are red cells. When you have leukemia, your body makes more white cells than it needs. These leukemia cells cant fight infection the way normal white blood cells do. And because there are so many of them, they start to affect the way your organs work. Over time, you may not have enough red blood cells to supply oxygen, enough platelets to clot your blood, or enough normal white blood cells to fight infection.

Leukemia is usually treated by a hematologist-oncologist. These are doctors who specialize in blood disorders and cancer. The treatment depends on the type and stage of the cancer. Some forms of leukemia grow slowly and don't need immediate treatment. However, treatment for leukemia usually involves one or more of the following:

Source: Information has been taken from various health website

Read more| National Honesty Day 2020: Date, history, significance and quotes on the value of honesty

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What Is Leukemia? Here's all you need to know - India Today

Bone Marrow Stem Cells Comments Off on What Is Leukemia? Here’s all you need to know – India Today | May 5th, 2020

The first-in-human, universal chimeric receptor antigen (CAR) T-cell (CAR-T) therapy GC027 was tolerable and resulted in antileukemic responses among patients with relapsed/refractory T-cell acute lymphoblastic leukemia (T-ALL), according to results from a phase 1 trial presented at the American Association for Cancer Research (AACR) Virtual Annual Meeting I 2020.1

Theuniversal CAR T cells target CD7, which, according to Xinxin Wang, PhD, ofGracell Biotechnologies Co, Ltd, in China, and lead author and presenter of thestudy, is a good target for T-ALL because it is expressed by more than 95% ofT-ALL patients.

GC027 isallogeneic, which may prevent the development of graft-versus-host disease. Theproduct is introduced using lentivirus for rapid elimination of T-ALL cells. Preclinicalstudies showed efficacy in a T-ALL xenograft model, and this prospective studyevaluated the safety and efficacy in humans.

Thesingle-arm, open-label study treated 5 adult patients with relapsed/refractoryCD7-positive T-ALL with a single infusion of 1 of 3 different dose levels ofG027: 0.6 x 107/kg, 3 x 107/kg, and 1.5 x 107/kg.Lymphodepletion therapy was administered prior to the G027 infusion. Theprimary endpoint was safety and the secondary endpoints included objectiveresponse rate (ORR) within 3 months after G027 infusion.

Patientswith extramedullary or central nervous system disease were excluded. Atbaseline, the median age was 24 (range, 19-38). Patients were heavilypretreated, with 5 median number of prior therapies (range, 1-9). Two patientshad high-risk disease and the median bone marrow tumor burden was a median of38.2% of blasts. None of the patients had undergone a prior allogeneic hematopoieticstem cell transplant.

Allpatients developed cytokine release syndrome (CRS), 4 of which were grade 3 and1 was grade 4. All cases were manageable and resolved with treatment andsupportive care. None of the patients developed neurotoxicity.

The completeremission (CR)/CR with incomplete hematologic recovery was 100%. By day 28, 4patients achieved a CR with negative for minimal residual disease (MRD) and 3of these patients remained MRD negative up to day 161. One patient achieved CRbut was MRD positive, and relapsed by day 29.

Peak CART-cell expansion in peripheral blood occurred between week 1 and 2.

As the first-in-human, universal CAR T-cell therapy for adult relapsed/refractory T-ALL, Dr Wang said, GC027 has demonstrated superior clinical efficacy and induced deep response in patients with acceptable safety profile. She added that trial enrollment is ongoing.

Reference

Wang X, Li S, Gao L, et al. Clinical safety and efficacy study of TruUCAR GC027: The first-in-human, universal CAR-T therapy for adult relapsed/refractory T-cell acute lymphoblastic leukemia (r/r T-ALL). Presented at: American Association for Cancer Research (AACR) Virtual Annual Meeting I; April 27-28, 2020. Abstract CT052.

This article originally appeared on Cancer Therapy Advisor

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First-in-Human Universal CAR-T Therapy Found Active in Relapsed/Refractory T-ALL - Oncology Nurse Advisor

Bone Marrow Stem Cells Comments Off on First-in-Human Universal CAR-T Therapy Found Active in Relapsed/Refractory T-ALL – Oncology Nurse Advisor | May 5th, 2020

Most doctors are prisoners of their education and shackled by their profession.

- Richard Diaz

A month ago we offered up the analysis below on this 'watch item' biotech stock from across the pond exclusively to Biotech Forum members. The stock is up over 40% since then but the concern is still intriguing. Today, we update our original thesis to account for all company-specific news and analyst ratings on this name since our original research was posted.

Orchard Therapeutics (ORTX) is a London, United Kingdom-based biopharmaceutical company that IPOd in 2018. The companys mission is to help patients with rare conditions by leveraging its understanding of gene therapy. The companys approach is to take a patients own blood stem cells and insert into them a working copy of the missing or malfunctioning gene. The companys approach circumvents the need for a bone marrow transplant by taking advantage of blood stem cells' intrinsic capacity to self-renew in a patients bone marrow and produce new blood cells of all types. The ultimate goal is to permanently correct genetic disorders through the use of a single treatment. The companys pipeline is broken down into three overarching programs: neurometabolic disorders, primary immune deficiencies, and blood disorders. The companys lead product candidates are OTL-200, OTL-101 and OTL-103. The companys only commercial product is Strimvelis, which has been approved by the EMA but not by the FDA. Orchard Therapeutics has a market capitalization of just over $1 billion and trades for around $11 a share.

Pipeline:

Source: Company Presentation

OTL-200:

OTL-200 is an ex vivo autologous gene therapy in development to treat Metachromatic leukodystrophy. The drug uses a modified virus to insert an operational copy of the ARSA gene into a patients cells. OTL-200 has received rare pediatric disease designation from the FDA. MLD is a rare and deadly inherited disease. The disease is characterized by the accumulation of fats called sulfatides, which causes a breakdown in the protective fatty layer surrounding nerves in the central and peripheral nerve systems. It is estimated that 1 in 40,000 to 1 in 160,000 people have the disease worldwide. OTL-200 was developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy.

Source: Company Presentation

On December 2nd, 2019, under accelerated assessment status, The EMA accepted the review for a marketing application of OTL-200 for MLD. Under accelerated assessment, the review period is 150 days, compared to the normal 210 days. The action date should occur very shortly.

Source: Company Presentation

Looking ahead, the company expects to obtain approval and launch the drug in Europe in the second half of 2020. The company is currently preparing for a commercial launch. Furthermore, the company expects a U.S. regulatory filing, a BLA, in late 2020 to early 2021.

Source: Company Presentation

OTL-101:

OTL-101 is an ex vivo autologous gene therapy in development to treat adenosine deaminase severe combined immunodeficiency. The drug uses a modified virus to insert an operational copy of the ADA gene into a patients cells. OTL-101 has received both Breakthrough Therapy designation and Rare Pediatric Disease designation from the FDA. ADA-SCID is a rare, inherited, pediatric disorder that is commonly fatal when undiagnosed or left untreated. The disorder is the result of a deficiency in adenosine deaminase. The disease culminates in a complete lack of/minimal immune system development. It is estimated that the diseases annual incidence is between 1 in 200,000 and 1 in 1 million live births.

Looking ahead, Orchard Therapeutics will initiate a rolling BLA filing in the U.S. for OTL-101 in ADA-SCID in the first half of 2020 with an anticipated completion of the filing within 12 months.

Source: Company Presentation

OTL-103:

0TL-103 is an ex vivo autologous gene therapy in development to treat Wiskott Aldrich syndrome. The drug uses a modified virus to insert a working copy of the WAS gene into a patients cells. WAS is a rare, X-linked, recessive, inherited immune disorder, which is characterized by reoccurring severe infections, autoimmunity, eczema and severe bleeding episodes. The company has received Rare Pediatric Disease designation and Regenerative Medicine Advanced Therapy designation from the FDA. OTL-103 is being developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy.

Looking ahead, the company is preparing to file a BLA in the U.S. and an MAA in the EU for OTL-103 in WAS in 2021.

Source: Company Presentation

As of December 31st, 2019, the company had cash and cash equivalents of over $300 million including marketable securities. Research and development expenses for the fourth quarter were $30.8 million, compared to $17.4 million in Q4 of 2018. Selling, general and administrative expenses were $18.5 million in the quarter, compared to $11.9 million in the same quarter of 2018. Overall, the company had a net loss of $45.4 million in the quarter, compared to a net loss of $25 million in the same period of 2018. The company believes that its current balance sheet will allow it to fund all anticipated operations and capital expenditures into the second half of 2021.

Despite a sizable market cap, Orchard gets little in the way of attention from analysts. That is probably because it is headquartered overseas. The current median analyst price target on Wall Street is just over $25.00 a share.

Oppenheimer reissued its Buy rating and $28 price target on April 1st. In mid-March, Cowen & Co. did the same with an identical price target in Mid-March. This follows a similar call at Cowen in December with strongly bullish commentary:

Orchard Therapeutics has a differentiated and promising lentiviral, ex vivo pipeline with gene therapies for ADA-SCID, WAS and MLD likely to get approved in 2021-22. With a deep early stage pipeline focused on validated targets, we anticipate strong stock appreciation as drugs are approved and early stage programs yield positive clinical results and advance into pivotal studies in 2020/21."

On September 17th, 2019, Guggenheim initiated coverage with a buy rating and a $31 a share price target. The analyst at Guggenheim views the company's ex vivo, lentiviral gene therapy platform as differentiated and de-risked. Furthermore, three drug approvals within the next 24 months bode well for share appreciation. Finally, in early September of last year, Barclays (NYSE:BCS) initiated coverage with an overweight rating and a $21 price target. The analyst at Barclays sees a company that strategically targets genetic diseases that have validated approaches with hematopoietic stem cell transplant, a fairly de-risked pipeline and multiple catalysts over the next 24 months.

Orchard has multiple shots on goal and several definable milestones in the coming quarters. Given some of the recent turmoil in the credit markets, anything that will need to raise capital in the foreseeable future is taken out and shot when markets have deep down days.

Given that, I still have Orchard as a solid 'watch item' holding and added several hundred shares in my personal account in late March. The shares appear to have an attractive risk/reward profile on a longer term basis, but I would accumulate on dips given the shares run up in April. Once volatility ebbs and we have more confidence in the economy and markets, this is a name we will revisit and maybe upgrade to a larger recommended holding.

The securest place is a prison cell, but there is no liberty

- Ben Franklin

Bret Jensen is the Founder of and authors articles for the Biotech Forum, Busted IPO Forum, and Insiders Forum

Live Chat on The Biotech Forum continues to be very active with new trade and the lucrative covered call ideas available thanks to the spike in market volatility throughout the trading day. If you join the The Biotech Forum today by clicking HERE you will automatically get access to our model portfolio, Live Chat, investment archives and our next 'option play of the week.'

Disclosure: I am/we are long ORTX. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

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Orchard Therapeutics: Updating The Investment Thesis - Seeking Alpha

Bone Marrow Stem Cells Comments Off on Orchard Therapeutics: Updating The Investment Thesis – Seeking Alpha | May 5th, 2020