Eighteen-year-old Aisha Choudhary was just like any other adolescent eyes filled with dreams and a heart brimming with energy. The only difference was she was battling a rare genetic disease, Severe Combined Immune Deficiency (SCID). Diagnosed when she was six months old and undergoing medical treatment for years, she was iron-willed in playing the cards she was dealt.

Since one of the most effective cures for SCID is a stem cell transplant (grafting of the parent cells from which all blood cells develop), Aishas parents, Niren and Aditi, decided to opt for that treatment mode. But their cells were not a complete match with their daughters, and they had to look at external donors. However, due to a low number of voluntary, registered stem cell donors, Aisha could not get a compatible donor whose genetic markers were a close enough match to hers. With no other alternative treatment available, Aisha had a bone marrow transplant. But, it came with a side-effect that cost her life Pulmonary Fibrosis, a disease known to damage the lung tissues.

Aishas Choudhary's role has been played by Zaira Wasim in The Sky is Pink.

Aishas journey has been captured in The Sky is Pink, a recent Bollywood movie starring Priyanka Chopra, Farhan Akhtar, Zaira Wasim, and Rohit Saraf.

The 18-year-olds life story is mirrored in the experiences of many who await stem cell donation as treatment for blood-related illnesses likeleukemia, lymphoma, and sickle cell anemia every year. With very few individuals signing up as donors and the probability of finding a match being a dismal 0.0008 percent in India (against a lean 16 percent abroad), fatalities are mounting year on year.

However, in recent times, there has been one small break in the clouds a number of youngsters, non-governmental organisations, and medical professionals have come forward and are working to spread awareness about stem cell donation and motivate a larger number of people to register as donors.

The stem cells in a human body mainly comprise red blood cells, platelets, and white blood cells. These are found in the umbilical cord of newborns and in the peripheral or circulating blood and bone marrow.

A stem cell donation is as simple and painless as a blood donation.

Certain diseases like blood cancer and leukemia tend to destroy the bone marrow or affect its functioning.For these, treatments like chemotherapy and radiotherapy are tried initially. However, in some cases, they do not prove effective for a cure. The only recourse then is replacing the patients stem cells with those of a healthy person.

One of the main criteria for a successful transplant is a good match between the stem cells of the donor and those of the patient. Therefore, a donor registry will administer a cheek swab test (tissue samples extracted from the cheek) on all potential donors to match cell characteristics. This procedure of pairing generic markers is called Human Leukocyte Antigen (HLA) in medical terms.

A cheek swab test in progress.

Each potential donors tissue is entered in the registry and given an identification number after the test is done. If the registry finds a match at any point in time, the donor is contacted to initiate the transplant.

There are many organisations today that are leading the charge in saving the lives of people suffering from serious blood disorders like cancer, thalassemia, and anaemia.

For instance, Datri, an Ahmedabad-based NGO, is working to create a wide and diverse database of potential stem cell donors by organising donation drives. Founded in 2009 by two doctors and an engineer, the organisation focuses on conducting awareness campaigns and helping individuals sign up on its registry as a committed and voluntary benefactor.

The team of the NGO Datri.

The idea for Datri was initially born in the minds of doctors Nezih Cereb and Soo Young Yang, who run a laboratory, Histogenetics, for determining tissue matches between patients and donors. Since pairing tissue types is imperative for any stem cell transplant, and confronting a severe shortage of donors, the doctor duo would run from pillar to post to meet hospitals requirements. Working with a number of the hospitals in India, they realised just how acute the shortfall was in people willing to donate stem cells. They recognised the immediate need to create a donor registry here.

Soon after, Raghu Rajagopal, an engineer from BITS Pilani and Director of ready-to-eat venture Millets and More, connected with them and they decided to start Datri.

Today, the functioning of the registry, its maintenance, and even the substantial costs involved in conducting the HLA matching are taken care of by the lab. In the last 10 years, Datri has gotten over four lakh people to register as donors and has saved around 600 lives through successful transplantation.

Every day, about 40 people are diagnosed with blood disorders in India. Though these can be cured through a stem cell transplant from a genetically matched donor, there is only a 25 percent chance of finding a match from within the family. Others have no option but to rely on unrelated donors. But the chances of getting a match is anywhere between one in 10,000 and one in two million. There is an urgent need to rope in as many potential donors as possible, which is precisely what Datri is trying to do, Raghu explains.

Another organisation that is dedicated to fighting blood disorders with stem cell treatment is DKMS-BMST. It was formed through a joint venture between two renowned NGOs DKMS, which is one of the largest international blood stem cell donor centres globally, and the Bangalore Medical Services Trust (BMST).

The team of DKMS-BMST.

DKMS was founded in Germany in 1991 by businessman Dr Peter Harf, after he lost his wife to leukemia. BMST was born in 1984 from the vision of Dr Latha Jagannathan, a medical director and managing trustee. Since both organisations had a common goal to find a matching donor for every patient with a blood disorder, they decided to come together to achieve it.

A group of youngsters registering to be stem cell donors.

So far, more than 37,000 people in India have registered as potential donors after attending DKMS-BMSTs donor drives.

In highly populous countries like India, thousands of people are in need of stem cell transplants every year to survive. Though donating stem cells is a painless and non-invasive process, it remains a lesser-known medical concept in India, with only 3.6 lakh people willing to play a part in it. Besides, the chances of stem cells of people of the same ethnicity matching are higher than those of individuals from different ethnic backgrounds. But, it is due to sheer lack of awareness that India lags severely in stem cell donations, say experts.

Students taking a cheek swab test at one of the colleges in Bengaluru.

Dr Govind Eriat, a reputed hematologist and bone marrow transplant specialist, says,

With a major hurdle to stem cell donation in India proving to be the myths surrounding the subject, the youth are coming forward to deconstruct common misconceptions.

For instance, 21-year-old Tejaswini Patel, a student of Information Science at New Horizon College of Engineering, Bengaluru, has been busting the false ideas on stem cell donation, starting among her family and friends. She says,

She adds, with a notable sense of pride, In the last two years alone, around 400 students from my college have registered themselves as donors.

(Edited by Athirupa Geetha Manichandar)

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How Young India is fuelling the future of stem cell therapy and signing up to save lives - YourStory

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MANCHESTER Now that the world has the Impossible Whopper, will the impossible liver be far behind?

Such possibilities are being broached this week as inventor Dean Kamen, founder of the Advanced Regenerative Manufacturing Institute, hosts the fourth semi-annual summit since launching ARMI in July 2017.

The three-day conference started Tuesday with a members-only day for BioFab USA and ARMI. It included a dinner at Kamens Bedford home.

Two public days of speeches and workshops are scheduled through Thursday. A total of 150 have signed up for the event.

Guests listen to a speaker at the Meeting in the Millyard at ARMI in Manchester on Wednesday, Oct. 16, 2019.

The keynote speaker on Wednesday was Jason Kelly, co-founder and chief-executive of Ginkgo Bioworks, a synthetic biology company that programs cells for customers in the chemical, pharmaceutical, food and energy industries.

We program cells because they run on digital code in the form of DNA, Kelly told a crowd of scientists, entrepreneurs and regulators.

Much of ARMIs work has focused on the use of stem cells to generate replacements for human tissue, bones and organs. For example, one of ARMIs biggest accomplishments to date has been the Tissue Foundry; its first production was bone-ligament tissue grown together from bone-marrow stem cells.

But Kamen, who prides himself on introducing new technologies to a field, had Kelly speak about a different kind of cells synthetic cells.

Ginkgo Bioworks has partnered with Bayer to develop self-fertilizing crops, with Roche to develop antibiotics, and with Motif to produce animal-free protein ingredients. Kelly said synthetic cell production played a role in the Impossible Whopper, the plant-based patty that Burger King claims tastes like beef.

(According to the website of Impossible Food, the company that makes the patty, the company extracts DNA from soy plants and inserts it into genetically engineered yeast, which ferments to produce heme, the molecule that gives meat its taste.)

Ginkgo has made CNBCs Disruptor 50 List in the last three years and recently raised more than $430 million in venture capital. In doing so, Ginkgo has achieved what Kamen wants for ARMI to move from theoretical design and laboratory work to mass production.

Theyve learned how to scale it, Kamen said in his introduction. Kamen said he expects ARMI-linked production to start relatively quickly.

(Finding) talent is not the problem. Capital is the problem, Kelly said about tech startups. Many venture capitalists arent experts in the science-heavy world of what he calls tough tech. So they are wary about investing in something they cant grasp.

He advised startups to seek government grants Ginkgo would not have succeeded without them hustle the non-specialist investor and find third-party validation from agencies such as the FDA or the Standards Coordinating Body, a voluntary organization that sets standards for the regenerative medicine industry.

Kelly said the time is now for tough tech. He noted the work of SpaceX and Tesla, and he said Silicon Valley is embracing biotech.

People have run out of things to invent that end up as a square on your phone, he said.

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ARMI summit: With synthetic meat within grasp, why not synthetic liver? - The Union Leader

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Stories of using the little blue pill for bone marrow transplants, how pregnancy stress is related to the baby's sex, and a vaccine for breast cancer proliferated on the Internet this week. Here's why you didn't read about them on Medscape.

Researchers at the University of California, Santa Cruz, seem to think Viagra has more to offer in medicine. In a recent study of mice, they tested whether the vasodilator couldspeed up the migration of hematopoietic stem cells and progenitor stem cells from the bone to the blood, where the cells could be harvested noninvasively.

The standard protocol for preparing bone marrow donors for the harvesting procedure, a 5-day regimen of granulocyte-colony stimulating factor (G-CSF),is "complex, costly, unsuccessful in a significant proportion of donors," the study authors write, and typically results in fatigue, nausea, and bone pain. Using a two-drug strategy, oral Viagra and a single injection of the CXCR4 antagonist AMD3100 (plerixafor), elicited the same mobilization of stem cells in 2 hours.

We didn't cover the study because it's still too early to say whether this strategy might be effective in people. After this mouse study, the next step is testing the approach in larger animals before human clinical trials.

A study of 187 healthy pregnant women age 18 to 45 years suggests that preterm mental and physical stress may be related to the baby's sex and increase the risk for preterm birth. In the study, 16% of women were physically stressed, as measured by higher blood pressure and calorie intake; and 17% were mentally stressed with high levels of depressionand anxiety; 66% of the women were in the healthy (nonstressed) group.

Women who were stressed during pregnancy were more likely to give birth to a girl. Typically, 105 males are born for every 100 females, but the study authors found that the male-to-female ratio decreased to 2:3 in psychologically stressed patients and 4:9 in physically stressed patients. Physically stressed mothers also gave birth an average of 1.5 weeks earlier than mothers in the healthy group, with 22% giving birth preterm compared with 5% in the healthy group.

The study authors say the findings demonstrate the importance of maternal mental health. Medscape has covered the consequences of maternal stress extensively, including preterm birth, neurobehavioral risks, and potential links to hyperactivity during the offspring's teen years. However, the sample size in this study was small: the mentally and physically stressed groups combined only included about 60 women. That's not sufficient to inform clinical practice in counseling women who want to get pregnant about how stress may affect the sex of their baby, so we didn't cover it.

News spread this week that Floridian Lee Mercker became the first woman to "beat" breast cancer with the help of a new vaccine. The vaccine, which stimulates the immune system to fight off early-stage breast cancer, was developed and administered by researchers at the Mayo Clinic in Jacksonville, Florida. The vaccine is currently in an early trial.

Reports of Mercker's success raise hopes, but she's reportedly the first participant in the trial. The news report also says she underwent a double mastectomy after her diagnosis in March, so it's unclear what evidence of the vaccine's efficacy the researchers measured. Before this experimental vaccine is relevant to Medscape readers, we need to see additional detailed data from more patients in the clinical trial published in a peer-reviewed journal.

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The Week That Wasn't: Viagra BMTs, Pregnancy Stress, Breast Cancer Vaccine - Medscape

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BRIDGEPORT, Conn. (WTNH) The Gift of Life Marrow Registry organized the meeting Thursday between a bone marrow donor from Connecticut and the recipient whose life was saved by the donation.

Jennie Bunce, 25, of Redding donated her marrow. According to a representative for Gift of Life, Bunce was studying physical therapy and joined Gift of Life through a sorority event at North Carolinas High Point University in 2016.

I never win or get picked for anything, but it just felt like the right thing to do, Bunce told Gift of Life. Im just incredibly happy and grateful to be part of something so special. Its similar to holding the door open for someone or helping a friend in a time of need.

Across the country in Mesa, Arizona, father-of-6, Mark Roser, 33, was battling Acute Lymphoblastic Leukemia. He found out about the diagnosis after he broke a hip in 2018 and had continued weakness. Roser was told he needed a bone marrow transplant to survive.

The hardest part was knowing, no matter how hard I worked, that what I did would not be a deciding factor in my ability to receive this gift, said Roser.

The match was made by Gift of Life in about six months, and the transplant took place in Phoenix.

She is a hero to all the people in my life, said Roser.

She gave me life, she gave my children a future with their dad, she gave my wife a chance to hold her husband, to have someone hold her back. She allowed me to go to work, to play, to see things from a different perspective. I am grateful for every moment I have, and its because of her.

According to Gift a Life, medical privacy laws dictate that recipients and donors must remain anonymous and wait at least a year before meeting.

The two came face-to-face for the first time Thursday in Bridgeport at the Boca Oyster Bar.

Since its start in 1991, the Gift of Life Registry 349,000 individuals who have donated blood stem cells or bone marrow to save a life. The program has facilitated 16,800 matches and over 3,500 transplants.

To learn more about the organization and/or how to donate: https://www.giftoflife.org/.

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Bone marrow recipient comes face-to-face with CT donor for the first time - WTNH.com

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Company Announcement

Copenhagen, Denmark; October 18, 2019 Genmab A/S (Nasdaq:GMAB) announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion recommending broadening the existing marketing authorization for DARZALEX (daratumumab) in the European Union. The recommendation is for the use of DARZALEX in combination with lenalidomide and dexamethasone (Rd) as treatment for newly diagnosed adult patients with multiple myeloma who are ineligible for autologous stem cell transplant (ASCT). The Type II variation application, based on the Phase III MAIA (MMY3008) study, was submitted to the EMA by Janssen Pharmaceutica NV in March 2019. In August 2012, Genmab granted Janssen Biotech, Inc. an exclusive worldwide license to develop, manufacture and commercialize daratumumab.

We are encouraged that the CHMP are recommending a broadening of the current DARZALEX marketing authorization in the European Union to include DARZALEX in combination with lenalidomide and dexamethasone as a possible treatment for patients newly diagnosed with multiple myeloma. This would give patients another treatment option, in addition to the already approved combination of daratumumab plus bortezomib, melphalan and prednisone in this same setting, said Jan van de Winkel, Ph.D., Chief Executive Officer of Genmab.

About the MAIA (MMY3008) studyThe Phase III study (NCT02252172) is a randomized, open-label, multicenter study that includes 737 newly diagnosed patients with multiple myeloma who are not candidates for high dose chemotherapy and ASCT. Patients were randomized to receive either treatment with daratumumab in combination with lenalidomide (an immunomodulatory drug) and dexamethasone (a corticosteroid) or treatment with lenalidomide and dexamethasone alone. In the daratumumab treatment arm, patients received 16 milligrams per kilogram (mg/kg) weekly for first 8 weeks (Cycles 1 and 2), every other week for 16 weeks (Cycles 3 to 6) and then every 4 weeks (Cycle 7 and beyond) until progression of disease or unacceptable toxicity. Lenalidomide is administered at 25 mg orally on days 1 through 21 of each 28-day cycle, and dexamethasone is administered at 40 mg once a week for both treatment arms. Participants in both treatment arms will continue Rd until disease progression or unacceptable toxicity. The primary endpoint of the study is progression free survival.

About multiple myelomaMultiple myeloma is an incurable blood cancer that starts in the bone marrow and is characterized by an excess proliferation of plasma cells.1 Approximately 16,830 new patients were expected to be diagnosed with multiple myeloma and approximately 10,480 people were expected to die from the disease in the Western Europe in 2018.2 Globally, it was estimated that 160,000 people were diagnosed and 106,000 died from the disease in 2018.3 While some patients with multiple myeloma have no symptoms at all, most patients are diagnosed due to symptoms which can include bone problems, low blood counts, calcium elevation, kidney problems or infections.4

About DARZALEX (daratumumab)DARZALEX (daratumumab) intravenous infusion is indicated for the treatment of adult patients in the United States: in combination with bortezomib, thalidomide and dexamethasone as treatment for patients newly diagnosed with multiple myeloma who are eligible for autologous stem cell transplant; in combination with lenalidomide and dexamethasone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; in combination with bortezomib, melphalan and prednisone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for the treatment of patients with multiple myeloma who have received at least one prior therapy; in combination with pomalidomide and dexamethasone for the treatment of patients with multiple myeloma who have received at least two prior therapies, including lenalidomide and a proteasome inhibitor (PI); and as a monotherapy for the treatment of patients with multiple myeloma who have received at least three prior lines of therapy, including a PI and an immunomodulatory agent, or who are double-refractory to a PI and an immunomodulatory agent.5 DARZALEX is the first monoclonal antibody (mAb) to receive U.S. Food and Drug Administration (U.S. FDA) approval to treat multiple myeloma. DARZALEX is indicated in Europe in combination with bortezomib, melphalan and prednisone for the treatment of adult patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; for use in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for the treatment of adult patients with multiple myeloma who have received at least one prior therapy; and as monotherapy for the treatment of adult patients with relapsed and refractory multiple myeloma, whose prior therapy included a PI and an immunomodulatory agent and who have demonstrated disease progression on the last therapy6. The option to split the first infusion of DARZALEX over two consecutive days has been approved in both Europe and the U.S. In Japan, DARZALEX is approved in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for the treatment of adults with relapsed or refractory multiple myeloma and in combination with bortezomib, melphalan and prednisone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant. DARZALEX is the first human CD38 monoclonal antibody to reach the market in the United States, Europe and Japan. For more information, visit http://www.DARZALEX.com.

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Daratumumab is a human IgG1k monoclonal antibody (mAb) that binds with high affinity to the CD38 molecule, which is highly expressed on the surface of multiple myeloma cells. Daratumumab triggers a persons own immune system to attack the cancer cells, resulting in rapid tumor cell death through multiple immune-mediated mechanisms of action and through immunomodulatory effects, in addition to direct tumor cell death, via apoptosis (programmed cell death).5,6,7,8,9,10

Daratumumab is being developed by Janssen Biotech, Inc. under an exclusive worldwide license to develop, manufacture and commercialize daratumumab from Genmab. A comprehensive clinical development program for daratumumab is ongoing, including multiple Phase III studies in smoldering, relapsed and refractory and frontline multiple myeloma settings. Additional studies are ongoing or planned to assess the potential of daratumumab in other malignant and pre-malignant diseases in which CD38 is expressed, such as amyloidosis, NKT-cell lymphoma and B-cell and T-cell ALL. Daratumumab has received two Breakthrough Therapy Designations from the U.S. FDA for certain indications of multiple myeloma, including as a monotherapy for heavily pretreated multiple myeloma and in combination with certain other therapies for second-line treatment of multiple myeloma.

About Genmab Genmab is a publicly traded, international biotechnology company specializing in the creation and development of differentiated antibody therapeutics for the treatment of cancer. Founded in 1999, the company has two approved antibodies, DARZALEX (daratumumab) for the treatment of certain multiple myeloma indications, and Arzerra (ofatumumab) for the treatment of certain chronic lymphocytic leukemia indications. Daratumumab is in clinical development for additional multiple myeloma indications, other blood cancers and amyloidosis. A subcutaneous formulation of ofatumumab is in development for relapsing multiple sclerosis. Genmab also has a broad clinical and pre-clinical product pipeline. Genmab's technology base consists of validated and proprietary next generation antibody technologies - the DuoBody platform for generation of bispecific antibodies, the HexaBody platform, which creates effector function enhanced antibodies, the HexElect platform, which combines two co-dependently acting HexaBody molecules to introduce selectivity while maximizing therapeutic potency and the DuoHexaBody platform, which enhances the potential potency of bispecific antibodies through hexamerization. The company intends to leverage these technologies to create opportunities for full or co-ownership of future products. Genmab has alliances with top tier pharmaceutical and biotechnology companies. Genmab is headquartered in Copenhagen, Denmark with core sites in Utrecht, the Netherlands and Princeton, New Jersey, U.S.

Contact: Marisol Peron, Corporate Vice President, Communications & Investor Relations T: +1 609 524 0065; E: mmp@genmab.com

For Investor Relations: Andrew Carlsen, Senior Director, Investor RelationsT: +45 3377 9558; E: acn@genmab.com

This Company Announcement contains forward looking statements. The words believe, expect, anticipate, intend and plan and similar expressions identify forward looking statements. Actual results or performance may differ materially from any future results or performance expressed or implied by such statements. The important factors that could cause our actual results or performance to differ materially include, among others, risks associated with pre-clinical and clinical development of products, uncertainties related to the outcome and conduct of clinical trials including unforeseen safety issues, uncertainties related to product manufacturing, the lack of market acceptance of our products, our inability to manage growth, the competitive environment in relation to our business area and markets, our inability to attract and retain suitably qualified personnel, the unenforceability or lack of protection of our patents and proprietary rights, our relationships with affiliated entities, changes and developments in technology which may render our products or technologies obsolete, and other factors. For a further discussion of these risks, please refer to the risk management sections in Genmabs most recent financial reports, which are available on http://www.genmab.com and the risk factors included in Genmabs final prospectus for our U.S. public offering and listing and other filings with the U.S. Securities and Exchange Commission (SEC), which are available at http://www.sec.gov. Genmab does not undertake any obligation to update or revise forward looking statements in this Company Announcement nor to confirm such statements to reflect subsequent events or circumstances after the date made or in relation to actual results, unless required by law.

Genmab A/S and/or its subsidiaries own the following trademarks: Genmab; the Y-shaped Genmab logo; Genmab in combination with the Y-shaped Genmab logo; HuMax; DuoBody; DuoBody in combination with the DuoBody logo; HexaBody; HexaBody in combination with the HexaBody logo; DuoHexaBody; HexElect; and UniBody. Arzerra is a trademark of Novartis AG or its affiliates. DARZALEX is a trademark of Janssen Pharmaceutica NV.

1 American Cancer Society. "Multiple Myeloma Overview." Available at http://www.cancer.org/cancer/multiplemyeloma/detailedguide/multiple-myeloma-what-is-multiple-myeloma.Accessed June 2016.2 Globocan 2018. Western Europe Fact Sheet. Available at http://gco.iarc.fr/today/data/factsheets/populations/926-western-europe-fact-sheets.pdf Accessed March 20183 Globocan 2018. World Fact Sheet. Available at http://gco.iarc.fr/today/data/factsheets/populations/900-world-fact-sheets.pdf. Accessed December 2018.4 American Cancer Society. "How is Multiple Myeloma Diagnosed?" http://www.cancer.org/cancer/multiplemyeloma/detailedguide/multiple-myeloma-diagnosis. Accessed June 20165 DARZALEX Prescribing information, September 2019. Available at: https://www.accessdata.fda.gov/drugsatfda_docs/label/2019/761036s024lbl.pdf Last accessed September 20196 DARZALEX Summary of Product Characteristics, available at https://www.ema.europa.eu/en/medicines/human/EPAR/darzalex Last accessed October 20197De Weers, M et al. Daratumumab, a Novel Therapeutic Human CD38 Monoclonal Antibody, Induces Killing of Multiple Myeloma and Other Hematological Tumors. The Journal of Immunology. 2011; 186: 1840-1848.8 Overdijk, MB, et al. Antibody-mediated phagocytosis contributes to the anti-tumor activity of the therapeutic antibody daratumumab in lymphoma and multiple myeloma. MAbs. 2015; 7: 311-21.9 Krejcik MD et al. Daratumumab Depletes CD38+ Immune-regulatory Cells, Promotes T-cell Expansion, and Skews T-cell Repertoire in Multiple Myeloma. Blood. 2016; 128: 384-94.10Jansen, JH et al. Daratumumab, a human CD38 antibody induces apoptosis of myeloma tumor cells via Fc receptor-mediated crosslinking.Blood. 2012; 120(21): abstract 2974.

Company Announcement no. 50CVR no. 2102 3884LEI Code 529900MTJPDPE4MHJ122

Genmab A/SKalvebod Brygge 431560 Copenhagen VDenmark

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CHMP Issues Positive Opinion Recommending DARZALEX (Daratumumab) in Combination with Lenalidomide and Dexamethasone in Frontline Multiple Myeloma -...

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Results of an ex vivo study evaluating the phenotypic and growth characteristics of T cells collected by leukapheresis from cohorts of patients with newly diagnosed or relapsed/refractory multiple myeloma support use of chimeric antigen receptor (CAR)-T therapy earlier in course of the disease. The hypothesis-generating findings from this study were published in Blood Advances.

While CAR-T therapy targeted against the B-cellmaturation antigen (BCMA) has been associated with promising results inpatients with multiple myeloma, nearly all of the patients responding to thisapproach eventually develop progressive disease. Hence, strategies to optimizepatient selection for CAR-T therapy in the setting of multiple myeloma arebeing actively pursued.

Theratio of CD4 to CD8 T cells and/or the frequency of the CD81 CD45RO2 CD271 T-cell memory phenotype were usedin this study as surrogates for the clinical effectiveness of CAR-T therapysince previous studies ofCAR-T therapy in patients with chronic lymphocyticleukemia and multiple myeloma showed that of all baseline patient- anddisease-related characteristics considered, clinical response to CAR-T therapywas associated only with this T-cell ratio and/or the frequency of this subsetof memory T cells in the premanufacturing leukapheresis product.

Twocohorts of patients where compared in this study: 38 patients with newly diagnosedmultiple myeloma who had participated in clinical trials of induction therapy andon whom leukapheresis was performed before consolidation therapy and autologousstem cell transplantation (ASCT); and 25 patients with relapsed/refractorymultiple myeloma enrolled in a phase 1 clinical trial of anti-BCMA CAR-Ttherapy and on whom leukapheresis was performed during a washout period shortlyfollowing study enrollment.

Inboth patient cohorts, leukapheresis samples were exposed ex vivo to anti-CD3and anti-CD28 monoclonal antibodies covalently linked to magnetic beads toprovide stimulatory/costimulatory signals for T-cell proliferation and theexpansion of functional T cells.

The 2 patient cohorts were similar with respect to median age (ie, 55 years; 58 years [relapsed/refractory]), although the time from multiple myeloma diagnosis was 222 days for those treated with induction therapy and 4.6 years for those with relapsed/refractory disease.

Inaddition, differences in the median number of prior lines of therapy (1 vs 7),and bone marrow cellularity occupied by myeloma plasma cells (13% vs 65%) wereobserved when the former and latter cohorts were compared at the time thatleukapheresis was performed.

Akey finding from this study was a significantly higher frequency of T cellswith the CD81 CD45RO2CD271 T-cell memory phenotype(43.9% vs 29.0%; P =.001), as well asa significantly higher median CD4/CD8 ratio (2.6 vs 0.87; P <.0001) in the postinduction versus the relapsed/refractorypatient cohort.

Inaddition, the CD4/CD8 ratio was also significantly higher in the postinductioncohort compared with responders to anti-BCMA CAR-T therapy from the relapsed/refractorycohort (2.6 vs 1.3; P= .0009); however,while higher in the postinduction cohort, the difference in the frequency of Tcells with the CD81 CD45RO2CD271 T-cell memory phenotypewas not statistically significant when these 2 groups were compared.

Regardingcapacity for ex vivo proliferation during manufacturing, significantly highernumbers of population doubling by day 9 (PD9) were observed for thepostinduction cohort compared with either the overall relapsed/refractorycohort or the group of responders within the relapsed/refractory cohort.

Ourresults suggest that CAR T cells manufactured from leukapheresis samplesobtained after response to induction therapy would be, on average, moreclinically effective than those obtained from heavily relapsed/refractorymultiple myeloma patients, the study authors concluded.

Reference

Garfall AL, Dancy EK, Cohen AD, et al. T-cell phenotypes associated with effective CAR T-cell therapy in postinduction vs relapsed multiple myeloma. Blood Adv. 2019;3:2812-2815.

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CAR-T Therapy May Be More Effective When Administered Earlier in the Multiple Myeloma Treatment Continuum - Cancer Therapy Advisor

Bone Marrow Stem Cells Comments Off on CAR-T Therapy May Be More Effective When Administered Earlier in the Multiple Myeloma Treatment Continuum – Cancer Therapy Advisor | October 18th, 2019

Bhubaneswar: The advanced treatment of using stem cells for treating Autism and other neurological ailments have come as a ray of hope for the people living with some of these ailments. Medical experts working in the sector claim that the use of the technology improved the lives of many.

According to experts who practice stem cell therapy, the results have been overwhelming. Many of the patients have either been able to fight a deadly disease with the help of stem cells while many have been able to improve their quality of lives by using it. However, the technology is still not used widely in state hospitals.

Medical experts claim that stem cells could be used to treat neurological disorders like Autism, cerebral palsy, mental retardation, brain stroke, muscular dystrophy, spinal cord injury, head injury, cerebellar ataxia, dementia, motor neurone disease, multiple sclerosis while it has also been used to treat cancers like blood cancer with the help of bone marrow transplant when assisted by stem cell therapy.

However, treatment of Autism with stem cells is a new developing sector where visible changes are said to have been reported among children treated with this technology. However, the advanced technology which is now confined to only private sector is a bit expensive.

Autistic kids are usually treated with drugs for symptomatic relief, special education, occupational speech and behavioural therapies. In Autism, despite the best available medical and rehabilitative treatments satisfactory relief is still a far cry, said Dr Nandini Gokulchandran, Head Medical Services, NeuroGen Brain and Spine Institute, Mumbai.

Dr Gokulchandran claims that she has treated many cases of Autism in kids with stem cells which helped in overcoming their limited abilities. Under the treatment regime, an insertion procedure is undertaken followed by training to improve the skills and abilities of autistic kids.

Another neurologist, Dr Richa Bansod said that in India it has been reported that 1 in every 250 children have Autism and this number in increasing with better recognition and awareness of the condition. On the other hand, stem cells are now been used to fight deadly diseases.

Dr Joydeep Chakaborty, an oncologist and stem cell expert from HCG Cancer Hospital, Kolkata said, Stem cells and bone marrow transplants are now being used to cure blood cancer in many cases. It is also widely used to treat blood disorders like Thalassemia, Sickle Cell Anaemia and others.

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Stem cells treatment gives hope in fighting Autism, blood disorders - OrissaPOST

Spinal Cord Stem Cells Comments Off on Stem cells treatment gives hope in fighting Autism, blood disorders – OrissaPOST | October 18th, 2019

Cell therapy market size is estimated to grow significantly during the forecast period from 2019 to 2025. The cell therapy industry should witness substantial expansion over the forecast timeframe due to technological advancements coupled with increasing utilization of cell therapy along with gene therapy as treatment to several conditions.

Increasing funding from private organization as well as government to encourage cell therapy clinical trials, inclusion of varied cell types such as mesenchymal stem cells, skeletal muscle stem cells, lymphocytes, dendritic cells, hematopoietic stem cells (HSC) and pancreatic islet cells for cell therapy research will drive market size growth.

Bone marrow transplant or hematopoietic stem cell transplantation is most commonly performed cell therapy. Growing utilization of this therapy for treating conditions such as blood cancer and other hematologic conditions should drive industry growth. Potential application of cell therapy includes treatment for urinary problems, cancers, repairing spinal cord injuries, autoimmune disease, rebuilding damaged cartilage in joints, immune system improvement, neurological disorders and infectious disease. Such wide-ranging applications of cell therapy should drive business growth over the coming years.

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Increasing prevalence of chronic conditions, government assistance and numerous companies investing heavily in stem cell therapy research and development should stimulate industry growth. Proven effectiveness of cell therapy products coupled with rendering favorable guidelines pertaining to cell therapy manufacturing should positively impact industry growth. However, high cost and stringent regulations related to practicing of stem cell therapy will hamper industry growth to certain extent during the forecast period.

Based on therapy type, the industry is segmented into allogenic and autologous therapies. Autologous segment is anticipated to witness significant growth due to lower risk of fatal complications and graft failure resulting in high survival rate should drive segment growth. Moreover, significant investment in autologous cell therapy research by companies such as Vericel Corporation should positively impact growth.

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Applications of cell therapy include cardiovascular, ocular, musculoskeletal, gastrointestinal, oncology, neurological, dermatology, wounds and injuries, and others. Oncology segment is estimated to witness lucrative growth due to strong pipeline including Chimeric Antigen Receptor (CAR) T-cell therapy that has delivered promising results such as full recovery in end-stage patients suffering from acute lymphocytic leukemia during clinical trials.

Based on end-user, the industry is bifurcated into hospitals, diagnostic centers, clinics, research institutes, regenerative medicine centers and others. Stem cell is core component of regenerative medicine. Increasing research conducted by regenerative medicine researchers on variety of stem cell types such as multipotent adult stem cells (hematopoietic stem cells found in umbilical cord blood and mesenchymal stem cells [MSC] found in adipose tissue), along with pluripotent stem cells such as the bioengineered cells called induced pluripotent stem cells (iPSC) should drive segment growth.

North America cell therapy market will witness robust growth during the forecast period due to favorable regulatory framework to promote development of cellular therapy platform and presence of numerous companies engaged in cell therapy research. Rising awareness and growing healthcare expenditure should further propel industry growth.

Large patient population base suffering from chronic conditions along with development of healthcare infrastructure will drive Asia Pacific cell therapy market. Growing inclination towards advanced medicinal therapies will lead to growth of cell therapy market in the region.

Some notable industry players include, Vericel Corporation, Medipost, Cells for Cells, JCR Pharmaceuticals, Osiris Therapeutics, Kolon Tissuegene, NuVasive, Stemedica Cell Technologies, BioNTech IMFS, Anterogen, Pharmicell, Fibrocell Science, Novartis AG, Glaxosmithkline among others. Industry players are investing heavily in cell therapy research to develop innovative product and gain significant market share of lucrative market in future.

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Coronary Stents Market: https://www.marketwatch.com/press-release/coronary-stents-market-detailed-analysis-of-current-industry-figures-with-forecasts-growth-by-2025-2019-10-18

Companion Animal Diagnostic Market: https://www.marketwatch.com/press-release/companion-animal-diagnostic-market-industry-growth-analysis-forecast-by-2025-2019-10-18

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Cell Therapy Market outlook with industry review and forecasts - Technology Magazine

Spinal Cord Stem Cells Comments Off on Cell Therapy Market outlook with industry review and forecasts – Technology Magazine | October 18th, 2019

By Mitch LeslieOct. 17, 2019 , 2:00 PM

Mice put human runners to shame. Despite taking puny strides, the rodents can log 10 kilometers or more per night on an exercise wheel. But the mice that muscle biologist Eric Olson of the University of Texas Southwestern Medical Center in Dallas and colleagues unveiled in 2015 stood out. On a treadmill, the mice could scurry up a steep 10% grade for about 90 minutes before faltering, 31% longer than other rodents. Those iron mice differed from counterparts in just one small waythe researchers had genetically altered the animals to lack one muscle protein. That was enough to unleash superior muscle performance. "It's like you've taken the brakes off," Olson says.

Just as startling was the nature of the crucial protein. Muscles house some gargantuan proteins. Dystrophin, a structural protein whose gene can carry mutations that cause muscular dystrophy, has more than 3600 amino acids. Titin, which acts like a spring to give muscles elasticity, is the biggest known protein, with more than 34,000 amino acids. The protein disabled in the mice has a paltry 46. Although researchers have probed how muscles work for more than 150 years, they had completely missed the huge impact this tiny protein, called myoregulin, has on muscle function.

Olson and his colleagues weren't the only ones to be blindsided by Lilliputian proteins. As scientists now realize, their initial rules for analyzing genomes discriminated against identifying those pint-size molecules. Now, broader criteria and better detection methods are uncovering minuscule proteins by the thousands, not just in mice, but in many other species, including humans. "For the first time, we are about to explore this universe of new proteins," says biochemist Jonathan Weissman of the University of California, San Francisco.

Biologists are just beginning to delve into the functions of those molecules, called microproteins, micropeptides, or miniproteins. But their small size seems to allow them to jam the intricate workings of larger proteins, inhibiting some cellular processes while unleashing others. Early findings suggest microproteins bolster the immune system, control destruction of faulty RNA molecules, protect bacteria from heat and cold, dictate when plants flower, and provide the toxic punch for many types of venom. "There's probably going to be small [proteins] involved in all biological processes. We just haven't looked for them before," says biochemist Alan Saghatelian of the Salk Institute for Biological Studies in San Diego, California.

The venom of this predatory water bug has more than a dozen small proteins.

Small proteins also promise to revise the current understanding of the genome. Many appear to be encoded in stretches of DNAand RNAthat were not thought to help build proteins of any sort. Some researchers speculate that the short stretches of DNA could be newborn genes, on their way to evolving into larger genes that make full-size proteins. Thanks in part to small proteins, "We need to rethink what genes are," says microbiologist and molecular biologist Gisela Storz of the National Institute of Child Health and Human Development in Bethesda, Maryland.

Despite the remaining mysteries, scientists are already testing potential uses for the molecules. One company sells insecticides derived from small proteins in the poison of an Australian funnel-web spider. And a clinical trial is evaluating an imaging agent based on another minute protein in scorpion venom, designed to highlight the borders of tumors so that surgeons can remove them more precisely. Many drug companies are now searching for small proteins with medical potential, says biochemist Glenn King of the University of Queensland in St. Lucia, Australia. "It's one of the most rapidly growing areas."

Other short amino acidchains, often called peptides or polypeptides, abound in cells, but they are pared-down remnants of bigger predecessors. Myoregulin and its diminutive brethren, in contrast, are born small. How tiny they can be remains unclear. Fruit flies rely on a microprotein with 11 amino acids to grow normal legs, and some microbes may crank out proteins less than 10 amino acids long, notes microbial genomicist Ami Bhatt of Stanford University in Palo Alto, California. But even the largest small proteins don't measure up to average-size proteins such as alpha amylase, a 496amino-acid enzyme in our saliva that breaks down starch.

Few small proteins came to light until recently because of a criterion for identifying genes set about 20 years ago. When scientists analyze an organism's genome, they often scan for open reading frames (ORFs), which are DNA sequences demarcated by signals that tell the cell's ribosomes, its proteinmaking assembly lines, where to start and stop. In part to avoid a data deluge, past researchers typically excluded any ORF that would yield a protein smaller than 100 amino acids in eukaryotes or 50 amino acids in bacteria. In yeast, for example, that cutoff limited the list of ORFs to about 6000.

Relaxing that criterion reveals that cells carry vastly more ORFs. Earlier this year, Stanford postdoc Hila Sberro Livnat, Bhatt, and colleagues trawled genome fragments from the microbes that inhabit four parts of the human body, including the gut and skin. By searching for small ORFs that could encode proteins between five and 50 amino acids long, the researchers identified about 4000 families of potential microproteins. Almost half resemble no known proteins, but the sequence for one small ORF suggested that a corresponding protein resides in ribosomesa hint that it could play some fundamental role. "It's not just genes with esoteric functions that have been missed" when scientists overlooked small ORFs, Bhatt says. "It's genes with core functions."

For the first time, we are about to explore this universe of new proteins.

Other cells also house huge numbers of short ORFsyeast could make more than 260,000 molecules with between two and 99 amino acids, for example. But cells almost certainly don't use all those ORFs, and some of the amino acid strings they produce may not be functional. In 2011, after finding more than 600,000 short ORFs in the fruit fly genome, developmental geneticist Juan Pablo Couso of the University of Sussex in Brighton, U.K., and colleagues tried to whittle down the number. They reasoned that if a particular ORF had an identical or near-identical copy in a related species, it was less likely to be genomic trash. After searching another fruit fly's genome and analyzing other evidence that the sequences were being translated, the group ended up with a more manageable figure of 401 short ORFs likely to yield microproteins. That would still represent a significant fraction of the insects' protein repertoirethey harbor about 22,000 full-size proteins.

Weissman and colleagues found microproteins a second way, through a method they invented to broadly determine which proteins cells are making. To fashion any protein, a cell first copies a gene into messenger RNA. Then ribosomes read the mRNA and string together amino acids in the order it specifies. By sequencing mRNAs attached to ribosomes, Weissman and his team pinpoint which ones cells are actually turning into proteins and where on the RNAs a ribosome starts to read. In a 2011Cellstudy, he and his team applied that ribosome profiling method, also called Ribo-seq, to mouse embryonic stem cells and discovered the cells were making thousands of unexpected proteins, including many that would fall below the 100amino-acid cutoff. "It was quite clear that the standard understanding had ignored a large universe of proteins, many of which were short," Weissman says.

Saghatelian and his colleagues adopted a third approach to discover a trove of microproteins in our own cells. The researchers used mass spectrometry, which involves breaking up proteins into pieces that are sorted by mass to produce a distinctive spectrum for each protein. Saghatelian, his then-postdoc Sarah Slavoff, and colleagues applied the method to protein mixtures from human cells and then subtracted the signatures of known proteins. That approach revealed spectra for 86 previously undiscovered tiny proteins, the smallest just 18 amino acids long, the researchers reported in 2013 inNature Chemical Biology.

Being small limitsa protein's capabilities. Larger proteins fold into complex shapes suited for a particular function, such as catalyzing chemical reactions. Proteins smaller than about 50 to 60 amino acids probably don't fold, says chemist Julio Camarero of the University of Southern California in Los Angeles. So they probably aren't suited to be enzymes or structural proteins.

However, their diminutive size also opens up opportunities. "They are tiny enough to fit into nooks and crannies of larger proteins that function as channels and receptors," Olson says. Small proteins often share short stretches of amino acids with their larger partners and can therefore bind to and alter the activity of those proteins. Bound microproteins can also shepherd bigger molecules to new locationshelping them slip into cell membranes, for instance.

A microprotein in the poison of the deathstalker scorpion has been fused to a fluorescent dye to make tumors emit near-infrared light. (1) A tumor seen in visible light (2)Same tumor in visible and near-infrared light

Because of their attraction to larger proteins, small proteins may give cells a reversible way to switch larger proteins on or off. In a 2016 study inPLOS Genetics, plant developmental biologist Stephan Wenkel of the University of Copenhagen and colleagues genetically alteredArabidopsisplants to produce extra amounts of two small proteins. The plants normally burst into flower when the days are long enough, but when they overproduced the two microproteins, their flowering was postponed. The small proteins caused that delay by blocking a hefty protein called CONSTANS that triggers flowering. They tether CONSTANS to other inhibitory proteins that shut it down. "A cell uses things that help it survive. If a short protein does the job, that's fine," Saghatelian says.

Those jobs include other key tasks. In 2016, Slavoff, Saghatelian, and colleagues revealed that human cells manufacture a 68amino-acid protein they named NoBody that may help manage destruction of faulty or unneeded mRNA molecules. NoBody's name reflects its role in preventing formation of processing bodies (P-bodies), mysterious clusters in the cytoplasm where RNA breakdown may occur. When the protein is missing, more P-bodies form, thus boosting RNA destruction and altering the cell's internal structure. "It shows that small proteins can have massive effects in the cell," Slavoff says.

Muscles appear to depend on a variety of microproteins. During embryonic development, individual muscle cells merge into fibers that power contraction. The 84amino-acid protein myomixer teams up with a larger protein to bring the cells together, Olson's team reported in 2017 inScience. Without it, embryonic mice can't form muscles and are almost transparent.

Later in life, myoregulin steps in to help regulate muscle activity. When a muscle receives a stimulus, cellular storage depots spill calcium, triggering the fibers to contract and generate force. An ion pump called SERCA then starts to return the calcium to storage, allowing the muscle fibers to relax. Myoregulin binds to and inhibits SERCA, Olson's team found. The effect limits how often a mouse's muscles can contractperhaps ensuring that the animal has muscle power in reserve for an emergency, such as escaping a predator. Another small protein, DWORF, has the opposite effect, unleashing SERCA and enabling the muscle to contract repeatedly.

Even extensively studied organisms such as the intestinal bacteriumEscherichia coliharbor unexpected small proteins that have important functions. Storz and her team reported in 2012 that a previously undiscovered 49amino-acid protein called AcrZ helps the microbe survive some antibiotics by stimulating a pump that expels the drugs.

And the venom produced by a variety of organismsincluding spiders, centipedes, scorpions, and poisonous mollusksteems with tiny proteins. Many venom components disable or kill by blocking the channels for sodium or other ions that are necessary for transmission of nerve impulses. Small proteins "hit these ion channels with amazing specificity and potency," King says. "They are the major components of venoms and are responsible for most of the pharmacological and biological effects."

Australia's giant fish-killing water bug, for instance, doesn't just rely on sharp claws and lancelike mouthparts to subdue prey. It injects its victims with a brew of more than 130 proteins, 15 of which have fewer than 100 amino acids, King and colleagues reported last year.

Unlike hulking proteinssuch as antibodies, microproteins delivered by pill or injection may be able to slip into cells and alter their functions. Captopril, the first of a class of drugs for high blood pressure known as angiotensin-converting enzyme inhibitors was developed from a small protein in the venom of a Brazilian pit viper. But the drug, which the Food and Drug Administration approved for sale in the United States in 1981, was discovered by chance, before scientists recognized small proteins as a distinct group. So far, only a few microproteins have reached the market or clinical trials.

Cancer researchers are trying to capitalize on a microprotein in the poison of the deathstalker scorpion (Leiurus quinquestriatus) of Africa and the Middle East. The molecule has a mysterious attraction to tumors. By fusing it to a fluorescent dye, scientists hope to illuminate the borders of brain tumors so that surgeons can safely cut out the cancerous tissue. "It lights up the tumor. You can see the margins and if there are any metastases," King says. A clinical trial is now evaluating whether the dual molecule can help surgeons remove brain tumors in children.

How important small proteins will be for medicine is still unknown, but they have already upended several biological assumptions. Geneticist Norbert Hbner of the Max Delbrck Center for Molecular Medicine in Berlin and colleagues found dozens of new microproteins in human heart cells. The group traced them to an unexpected source: short sequences within long noncoding RNAs, a variety that was thought not to produce proteins. After identifying 169 long noncoding RNAs that were probably being read by ribosomes, Hbner and his team used a type of mass spectrometry to confirm that more than half of them yielded microproteins in heart cells, a result reported earlier this year inCell.

Bacteria such as Escherichia coli also churn out many microproteins, although their functions remain unclear in many cases.

The DNA sequences for other tiny proteins also occur in unconventional locations. For example, some lie near the ORFs for bigger proteins. Researchers previously thought those sequences helped manage the production of the larger proteins, but rarely gave rise to proteins themselves. Some coding sequences for recently discovered microproteins are even nested within sequences that encode other, longer proteins.

Those genomic surprises could illuminate how new genes arise, says evolutionary systems biologist Anne-Ruxandra Carvunis of the University of Pittsburgh in Pennsylvania. Researchers had thought most new genes emerge when existing genes duplicate or fuse, or when species swap DNA. But to Carvunis, microproteins suggest protogenes can form when mutations create new start and stop signals in a noncoding portion of the genome. If the resulting ORF produces a beneficial protein, the novel sequences would remain in the genome and undergo natural selection, eventually evolving into larger genes that code for more complex proteins.

In a 2012 study, Carvunis, who was then a postdoc in the lab of Marc Vidal at the Dana-Farber Cancer Institute in Boston, and colleagues found that yeast translate more than 1000 short ORFs into proteins, implying that these sequences are protogenes. In a new study, Carvunis and her team tested whether young ORFs can be advantageous for cells. They genetically altered yeast to boost output of 285 recently evolved ORFs, most of which code for molecules that are smaller than the standard protein cutoff or just over it. For almost 10% of the proteins, increasing their levels enhanced cell growth in at least one environment. The results, posted on the preprint server bioRxiv, suggest these sequences could be on their way to becoming full-fledged genes, Carvunis says.

Slavoff still recalls being astonished when, during her interview for a postdoc position with Saghatelian, he asked whether she would be willing to go hunting for small proteins. "I had never thought that there could be this whole size of proteins that was dark to us until then."

But the bet paid offshe now runs her own lab that is searching for microproteins. Recently, she unleashed some of her postdocs and graduate students on one of the most studied organisms, the K12 strain ofE. coli.The team soon uncovered five new microproteins. "We are probably only scratching the surface," she says.

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New universe of miniproteins is upending cell biology and genetics - Science Magazine

Skin Stem Cells Comments Off on New universe of miniproteins is upending cell biology and genetics – Science Magazine | October 18th, 2019

For the very first time, scientists have made artificial embryos from scratch, without sperm or egg, and implanted them into female mice. The embryos developed into live fetuses, but these exhibited major malformations.

The team at the University of Texas Southwestern Medical Center used extended pluripotent stem cells, which are cells that have the potential, like an embryo, to develop into any type of tissue in the body. These master cells are able to form all three major types of cell groups (ectoderm, endoderm, and mesoderm). Unlike simple pluripotent stem cells, the extended variety can develop into tissues that support the embryo, such as the placenta.Without this type of stem cells, embryos cannot develop and grow properly.

The researchers coaxed stem cells to form into all the cells required for the development of an embryo by bathing them into a solution made of nutrients, growth stimulants, and signaling molecules. The cells assembled into embryo-like structures, including placental tissue.

Next, the artificial embryos were implanted into the uteruses of female mice. Only 7% of the implants were successful but those embryos that did work actually started developing early fetal structures. There were major malformations, however, as the tissue structure and organization did not closely resemble that of a normal embryo.

Previously, other research groups had managed to grow artificial embryos but this was the first time that they were successfully implanted and developed placental cells.

In the future, the University of Texas researchers plan on refining their method in order to grow fetuses that are indistinguishable from normal ones. The goal is to replace real embryos and make artificial ones at scale. These embryo models could then be grown in dishes to study early mammalian development and accelerate drug development.

Some of the cells that the researchers used to grow into embryos originally came from the ear of a mouse. Theoretically, the same should be possible for human embryos, but why would we? Besides testing drugs, artificial embryos could be grown from the skin cells of an infertile person. Then, in the lab, these embryos could be studied in order to identify potential genetic defects that might cause infertility.

Even if such stem cell-derived embryos do not completely mimic normal embryo growth, there is still a lot we can learn about mammalian development. But, as is always the case with research that breaks the frontiers of what was once thought possible, our policies havent yet kept up with advances. There are serious ethical considerations to possibly making a person from a synthetic embryo. Although such a prospect is still science fiction, rapid developments such as the present study suggest that it is not impossible and we better prepare.

The findings were reported in the journal Cell.

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Artificial embryo without sperm or egg forms live fetus - ZME Science

Skin Stem Cells Comments Off on Artificial embryo without sperm or egg forms live fetus – ZME Science | October 18th, 2019

publication date: Oct. 18, 2019

The UCLA Jonsson Comprehensine Cancer Center has launched a CAR T-cell immunotherapy trialthat will attack cancer cells by simultaneously recognizing two targetsCD19 and CD20that are expressed on B-cell lymphoma and leukemia.

By launching a bilateral attack instead of using the conventional single-target approach, researchers are hoping to minimize resistance and increase the life expectancy for people diagnosed with these cancers.

One of the reasons CAR T cell therapy can stop working in patients is because the cancer cells escape from therapy by losing the antigen CD19, which is what the CAR T cells are engineered to target, Sarah Larson, a health sciences clinical instructor in hematology/oncology at UCLA Health and the principal investigator on the trial, said in a statement One way to keep the CAR T cells working is to have more than one antigen to target. So, by using both CD19 and CD20, the thought is that it will be more effective and prevent the loss of the antigen, which is known as antigen escape, one of the common mechanisms of resistance.

Up to two-thirds of the patients who experience relapse after being treated with the FDA-approved CD19 CAR T-cell therapy develop tumors that have lost CD19 expression. UCLA researchers are identifying and testing new strategies like this one so many more patients can benefit from the therapy.

In preclinical studiesled byYvonne Chen, an associate professor of microbiology, immunology, and molecular genetics at UCLA and the sponsor of the trial, the team was able to show that by simultaneously attacking two targets, the engineered T cells developed in her lab could achieve a much more robust defense compared to conventional, single-target CAR T cells against tumors in mice.

Chens team designed the CARs based on the molecular understanding of the CARs architecture, the antigen structure and the CAR/antigen binding interaction to achieve optimal T cell function. This design helps the T cells have dual-antigen recognition to help prevent antigen escape.

Based on these results, were quite optimistic that the bispecific CAR can achieve therapeutic improvement over the single-input CD19 CAR thats currently available, said Chen, who is also the co-director of the Jonsson Cancer Centers Tumor Immunology Program and a member of the UCLA Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research.

This first-in-humans study will evaluate the therapy in patients with non-Hodgkins B-cell lymphoma or chronic lymphocytic leukemia that has come back or has not responded to treatment. The goal is to determine a safe therapeutic dose.

Patients enrolled in the trial will have their white blood cells (T cells) collected intravenously then reengineered in the laboratory so the T cells can produce tumor-specific receptors (CARs), which allow the T cells to recognize and attack the CD19 and CD20 proteins on the surface of tumor cells. The new smarter and stronger T cells are then infused back into the patient and primed to recognize and kill cancer cells.

The trial is currently only offered at UCLA.

Results from STELLAR trial in MPM published in The Lancet Oncology

Novocure said the results from the STELLAR trial were published inThe Lancet Oncology.

The STELLAR trial was a prospective, single-arm trial including 80 patients that studied the use of Tumor Treating Fields, delivered via the NovoTTF-100L System, in combination with pemetrexed plus cisplatin/carboplatin as a first-line treatment for patients with unresectable, locally advanced or metastatic malignant pleural mesothelioma.

Data showed a median overall survival of 18.2 months (95 percent CI, 12.1 months-25.8 months) for patients treated with NovoTTF-100L and pemetrexed plus cisplatin or carboplatin. One- and two-year survival rates were 62.2 percent (95 percent CI, 50.3 percent-72.0 percent) and 41.9 percent (95 percent CI, 28.0 percent-55.2 percent), respectively. No serious systemic adverse events were considered to be related to the use of NovoTTF-100L. The most common mild to moderate adverse event was skin irritation beneath the transducer arrays.

The STELLAR trial demonstrated encouraging overall survival results with no increase in systemic toxicity observed in MPM patients treated with Tumor Treating Fields and standard chemotherapy, Giovanni Luca Ceresoli, head of pulmonary oncology at the Humanitas Gavazzeni Hospital in Bergamo, Italy, and principal investigator in the STELLAR trial, said in a statement. The median overall survival of 18.2 months is impressive given that MPM is a tumor with a dismal prognosis and few effective therapeutic options.

Median progression free survival was 7.6 months (95 percent CI, 6.7 percent-8.6 percent) for patients treated with NovoTTF-100L and pemetrexed plus cisplatin or carboplatin. There was a 97 percent disease control rate in patients with at least one follow-up CT scan performed (n=72). 40 percent of patients had a partial response, 57 percent had stable disease and 3 percent had progressive disease.

IASLC invites comments on Multidisciplinary Recommendations for Pathologic Assessment of Lung Cancer Resection Specimens Following Neoadjuvant Therapy

The International Association for the Study of Lung Cancer announced an open comment period for the IASLC Multidisciplinary Recommendations for Pathologic Assessment of Lung Cancer Resection Specimens Following Neoadjuvant Therapy paper.

The paper has been made available hereto provide an opportunity for public review of new draft recommendations. The open comment period runs from Oct. 14 to Nov. 7.

With the recent growing number of neoadjuvant therapy clinical trials for non-small cell lung cancer, there is a great need for standardization of specimen processing since major pathologic response has consistently been shown to be an important prognostic indicator.

The purpose of the paper is to outline detailed recommendations on how to process lung cancer resection specimens and to define pathologic complete response including major pathologic response and pathologic complete response following neoadjuvant therapy.

Currently there is no established guidance on how to process and evaluate resected lung cancer specimens following neoadjuvant therapy in the setting of clinical trials and clinical practice, Giorgio Scagliotti, past president of the IASLC and co-author of the paper, said in a statement. There is also a lack of precise definitions on the degree of pathologic response, including MPR or pCR.

IASLC is making an effort to collect such data from existing and future clinical trials. These recommendations are intended as guidance for clinical trials, although it is hoped they can be viewed as suggestions for good clinical practice outside of clinical trials, to improve consistency of pathologic assessment of treatment response.

The recommendations were developed by the IASLC Pathology Committee in collaboration with an international multidisciplinary group of experts in medical oncology, thoracic surgery and radiology.

We are crossing an exciting period of preclinical and clinical research around thoracic oncology. Targeted therapies and immunotherapy have greatly improved survival expectations in advanced disease and we believe they can equally generate benefit in the systemic therapy of earlier stages of the disease, Scagliotti said in a statement. Our initiative aims to use rigorous experimental conditions to analyze tissue specimens, collected in the context of already performed or ongoing neoadjuvant studies with targeted therapies and immunotherapy, to generate a diagnostic algorithm to be used in all subsequent studies in order to accelerate the scientific information about the clinical benefit produced by the neoadjuvant approach.

Expert second opinion improves reliability of melanoma diagnoses

Getting a reliable diagnosis of melanoma can be a significant challenge for pathologists.The diagnosis relies on a pathologists visual assessment of biopsy material on microscopic slides, which can often be subjective.

Of all pathology fields, analyzing biopsies for skin lesions and cancers has one of the highest rates of diagnostic errors, which can affect millions of people each year.

Now, a study led by UCLA researchers, has found that obtaining a second opinion from pathologists who are board certified or have fellowship training in dermatopathology can help improve the accuracy and reliability of diagnosing melanoma, one of the deadliest and most aggressive forms of skin cancer.

A diagnosis is the building block on which all other medical treatment is based,Joann Elmore, a professor of medicine at the David Geffen School of Medicine at UCLA and researcher at the UCLA Jonsson Comprehensive Cancer Center, said in a statement.All patients deserve an accurate diagnosis. Unfortunately the evaluation and diagnosis of skin biopsy specimens is challenging with a lot of variability among physicians.

In the study, led by Elmore and colleagues, the value of a second opinion by general pathologists and dermatopathologists were evaluated to see if it helped improve thecorrect diagnostic classification.

To evaluate the impact of obtaining second opinions, the team used samples from the Melanoma Pathology Study, which comprises of 240 skin biopsy lesion samples. Among the 187 pathologists who examined the cases, 113 were general pathologists and 74 were dermatopathologists.

The team studied misclassification rates, which is how often the diagnoses of practicing US pathologists disagreed with a consensus reference diagnosis of three pathologists who had extensive experience in evaluating melanocytic lesions. The team found that the misclassification of these lesions yielded the lowest rates when first, second and third reviewers were sub-specialty trained dermatopathologists. Misclassification was the highest when reviewers were all general pathologists who lacked the subspecialty training.

Our results show having a second opinion by an expert with subspecialty training provides value in improving theaccuracy of thediagnosis, which is imperative to helpguide patients to the most effective treatments, said Elmore, whois also the director of the UCLA National Clinician Scholars Program.

Elmore is now studying the potential impact of computer machine learning as a tool to improve diagnostic accuracy. She is partnering with computer scientists who specialize in computer visualization of complex image information, as well as leading pathologists around the globe to develop an artificial intelligence (AI)-based diagnostic system.

Michael Piepkorn of the University of Washington School of Medicine is the studys first author. Raymond Barnhill of the Institut Curie is the co-senior author.

The study was published in JAMA Network Open and supported by NCI.

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UCLA opens CAR T-cell trial focused on the most common types of lymphoma, leukemia - The Cancer Letter Publications

Skin Stem Cells Comments Off on UCLA opens CAR T-cell trial focused on the most common types of lymphoma, leukemia – The Cancer Letter Publications | October 18th, 2019

WILMINGTON, Del.--(BUSINESS WIRE)--Incyte Corporation (Nasdaq:INCY) today announced positive results from the Novartis-sponsored pivotal Phase 3 REACH2 study evaluating ruxolitinib (Jakafi) in patients with steroid-refractory acute graft-versus-host disease (GVHD). The study met its primary endpoint of improving overall response rate (ORR) at Day 28 with ruxolitinib treatment compared to best available therapy. No new safety signals were observed, and the ruxolitinib safety profile in REACH2 was consistent with that seen in previously reported studies in steroid-refractory acute GVHD.

Further analysis of the safety and efficacy data is ongoing. Novartis expects to initiate discussions with ex-U.S. regulatory authorities in 2020, and to submit REACH2 results for presentation at an upcoming scientific meeting.

GVHD is a challenging and serious disease, and physicians around the world need access to therapies that can improve outcomes for patients, said Peter Langmuir, M.D., Group Vice President, Targeted Therapies, Incyte. This positive result of the REACH2 study is excellent news for patients as it further reinforces the potential of ruxolitinib as a treatment option that can provide meaningful results for patients with steroid-refractory acute GVHD.

GVHD is a condition that can occur after an allogeneic transplant (the transfer of stem cells from a donor) where the donated cells initiate an immune response and attack the transplant recipients organs, leading to significant morbidity and mortality. There are two major forms of GVHD, acute and chronic, that can affect multiple organ systems including the skin, gastrointestinal (digestive) tract and liver.

Earlier this year, Jakafi was approved by the U.S. Food and Drug Administration (FDA) for the treatment of steroid-refractory acute GVHD in adult and pediatric patients 12 years and older based on results of the REACH1 trial. Jakafi is marketed by Incyte in the U.S.; ruxolitinib (Jakavi) is licensed to Novartis ex-U.S.

In addition, the pivotal REACH3 trial evaluating ruxolitinib in patients with steroid-refractory chronic GVHD is ongoing. A recent interim efficacy and safety analysis conducted by an Independent Data Monitoring Committee has recommended that REACH3, which is co-sponsored by Incyte and Novartis, should continue without modification. The results of the REACH3 trial are expected to be available in 2020.

About REACH2

REACH2 (NCT02913261) is a randomized, open-label, multicenter Phase 3 study sponsored by Novartis, evaluating safety and efficacy of ruxolitinib compared with best available therapy in patients with steroid-refractory acute GVHD.

The primary endpoint was overall response rate (ORR) at Day 28, defined as the proportion of patients demonstrating a best overall response (complete response or partial response). Secondary endpoints include durable ORR at Day 56, ORR at Day 14, duration of response, overall survival and event-free survival, among others. For more information about the study, please visit https://clinicaltrials.gov/ct2/show/NCT02913261.

About REACH

The REACH clinical trial program is evaluating Jakafi in patients with steroid-refractory GVHD and includes the collaborative Novartis-sponsored randomized pivotal Phase 3 trials: REACH2 and REACH3. The ongoing REACH3 trial is evaluating patients with steroid-refractory chronic GVHD with results expected next year. For more information about the REACH3 study, please visit https://clinicaltrials.gov/ct2/show/NCT03112603.

The REACH program was initiated with the Incyte-sponsored REACH1 trial, a prospective, open-label, single-cohort, multicenter, pivotal Phase 2 trial (NCT02953678) evaluating Jakafi in combination with corticosteroids in patients with steroid-refractory grade II-IV acute GVHD. For more information about the study, including trial results, please visit https://clinicaltrials.gov/show/NCT02953678.

About Jakafi (ruxolitinib)

Jakafi is a first-in-class JAK1/JAK2 inhibitor approved by the U.S. FDA for treatment of steroid-refractory acute GVHD in adult and pediatric patients 12 years and older.

Jakafi is also indicated for treatment of polycythemia vera (PV) in adults who have had an inadequate response to or are intolerant of hydroxyurea as well as adults with intermediate or high-risk myelofibrosis (MF), including primary MF, post-polycythemia vera MF and post-essential thrombocythemia MF.

Jakafi is marketed by Incyte in the United States and by Novartis as Jakavi (ruxolitinib) outside the United States. Jakafi is a registered trademark of Incyte Corporation. Jakavi is a registered trademark of Novartis AG in countries outside the United States.

Important Safety Information

Jakafi can cause serious side effects, including:

Low blood counts: Jakafi (ruxolitinib) may cause your platelet, red blood cell, or white blood cell counts to be lowered. If you develop bleeding, stop taking Jakafi and call your healthcare provider. Your healthcare provider will perform blood tests to check your blood counts before you start Jakafi and regularly during your treatment. Your healthcare provider may change your dose of Jakafi or stop your treatment based on the results of your blood tests. Tell your healthcare provider right away if you develop or have worsening symptoms such as unusual bleeding, bruising, tiredness, shortness of breath, or a fever.

Infection: You may be at risk for developing a serious infection during treatment with Jakafi. Tell your healthcare provider if you develop any of the following symptoms of infection: chills, nausea, vomiting, aches, weakness, fever, painful skin rash or blisters.

Skin cancers: Some people who take Jakafi have developed certain types of non-melanoma skin cancers. Tell your healthcare provider if you develop any new or changing skin lesions.

Increases in cholesterol: You may have changes in your blood cholesterol levels. Your healthcare provider will do blood tests to check your cholesterol levels during your treatment with Jakafi.

The most common side effects of Jakafi include: for certain types of MF and PV - low platelet count, low red blood cell count, bruising, dizziness, and headache; and for acute GVHD low red blood cell counts, low platelet counts, low white blood cell counts, infections and fluid retention.

These are not all the possible side effects of Jakafi. Ask your pharmacist or healthcare provider for more information. Tell your healthcare provider about any side effect that bothers you or that does not go away.

Before taking Jakafi, tell your healthcare provider about: all the medications, vitamins, and herbal supplements you are taking and all your medical conditions, including if you have an infection, have or had tuberculosis (TB), or have been in close contact with someone who has TB, have or had hepatitis B, have or had liver or kidney problems, are on dialysis, have a high level of fat in your blood (high blood cholesterol or triglycerides), had skin cancer or have any other medical condition. Take Jakafi exactly as your healthcare provider tells you. Do not change or stop taking Jakafi without first talking to your healthcare provider.

Women should not take Jakafi while pregnant or planning to become pregnant. Do not breast-feed during treatment with Jakafi and for 2 weeks after the final dose.

Full Prescribing Information, which includes a more complete discussion of the risks associated with Jakafi, is available at http://www.jakafi.com.

About Incyte

Incyte Corporation is a Wilmington, Delaware-based biopharmaceutical company focused on the discovery, development and commercialization of proprietary therapeutics. For additional information on Incyte, please visit the Companys website at http://www.incyte.com.

Follow @Incyte on Twitter at https://twitter.com/Incyte.

Forward Looking Statements

Except for the historical information set forth herein, the matters set forth in this press release, including statements regarding whether and when the REACH2 data will be presented, when results from the REACH3 study will be available, and the effect of the REACH2 results on patients with GVHD, contain predictions, estimates and other forward-looking statements.

These forward-looking statements are based on the Companys current expectations and subject to risks and uncertainties that may cause actual results to differ materially, including unanticipated developments in and risks related to: unanticipated delays; further research and development and the results of clinical trials possibly being unsuccessful or insufficient to meet applicable regulatory standards or warrant continued development; the ability to enroll sufficient numbers of subjects in clinical trials; determinations made by the FDA; the Companys dependence on its relationships with its collaboration partners; the efficacy or safety of the Companys products and the products of the Companys collaboration partners; the acceptance of the Companys products and the products of the Companys collaboration partners in the marketplace; market competition; sales, marketing, manufacturing and distribution requirements; greater than expected expenses; expenses relating to litigation or strategic activities; and other risks detailed from time to time in the Companys reports filed with the Securities and Exchange Commission, including its Form 10-Q for the quarter ended June 30, 2019. The Company disclaims any intent or obligation to update these forward-looking statements.

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Incyte Announces that the REACH2 Pivotal Trial of Ruxolitinib (Jakafi) Meets Primary Endpoint in Patients with Steroid-Refractory Acute...

Skin Stem Cells Comments Off on Incyte Announces that the REACH2 Pivotal Trial of Ruxolitinib (Jakafi) Meets Primary Endpoint in Patients with Steroid-Refractory Acute… | October 18th, 2019

All-natural grooming product labels are starting to read like grocery shopping lists. Thats because fruit is more than a healthy snack. Many of them possess skin-saving properties that eliminate the need for lab-made chemicals. Heres what were slathering on.

(1) Brandless Avocado Basil Hand Cream ($4) rejuvenates dry paws with a blend of avocado (yes, its a fruit) and almond oils, plus shea butter.

(2) Citrus is a natural stimulant, so a swipe of Way of Will 02 Lime + Black Spruce Deodorant ($13) perks you up, while geranium extract nixes body odor.

(3) For city dwellers, Malin+Goetz Advanced Renewal Moisturizer ($76) uses antioxidant-rich apple stem cells to protect the face from urban grime.

(4) Cold-pressed oils from apricot kernels, sunflower seeds, sage leaves, and more in Caldera Labs The Good Serum ($97) are so moisturizing that a few drops can sub in for face lotion. Use twice daily to help with fine lines, too.

(5) Nondrying Ye Ol Goat Soap Lemon + Verbena ($14) mixes olive oil and goat milkfor skin elasticitywith antibacterial citrus extract.

(6) Lucky Bastard Co. Premium Lip Balm ($8) combines fruit oils (coconut, avocado, raspberry seed) with beeswax to create a hydrating seal. And the flat slider container wont bulk up your front pocket.

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The Best Fruit-Based Skincare Products You Need This Season - Men's Journal

Skin Stem Cells Comments Off on The Best Fruit-Based Skincare Products You Need This Season – Men’s Journal | October 18th, 2019

A recent study looked at funding rates for R01 grant applications, which are designed to support "health-related research and development based on the mission of the NIH." In general, population-based projects were less likely to be funded than explorations of cellular mechanisms, the study found. Will & Deni McIntyre/Science Source hide caption

A recent study looked at funding rates for R01 grant applications, which are designed to support "health-related research and development based on the mission of the NIH." In general, population-based projects were less likely to be funded than explorations of cellular mechanisms, the study found.

Black applicants to a prestigious research grant program at the National Institutes of Health are awarded funding at a significantly lower rate than their white peers. The NIH has been intensively investigating this funding gap since a 2011 report revealed the extent of the problem, looking for underlying mechanisms to use as opportunities for corrective intervention.

NIH's latest finding, described in a study released this month in the open-access journal Science Advances, reveals that part of the gap can be attributed to differences in the types of topics scientists propose studying and how those topics are valued by grant reviewers.

The study of grant applications submitted between 2011 and 2015 suggests African American scientists may be more likely to pursue research in topic areas such as community-oriented research on disease prevention, for example, versus more microscopic-level research on cellular mechanisms or the basics of genetics. Those population-based topics aren't being funded as readily.

And that's a problem with the system, some outside researchers point out not with the choice of research topic.

"I do think that the areas of research that apparently are being funded at a lower rate are important," says David Asai, senior director for science education at Howard Hughes Medical Institute and an advocate for diversity in STEM, who was not involved in the NIH analysis. "This study might prompt the community to think about the underlying biases we might have in deciding what sorts of research deserve greater attention."

The NIH study looked at funding rates in the form of successful applications for R01 grants, which are designed to support "health-related research and development based on the mission of the NIH."

Despite NIH efforts to diversify the pool of scholars doing medical research, white applicants for these grants continue to receive funding at nearly twice the rate of black applicants 17.7% of white applicants were approved in fiscal years 2011-2015 compared with 10.7% of black applicants.

The researchers analyzed keywords in the topics of 157,549 grant applications and found that some topics were close to four times more likely to gain funding support.

"Among the less favored [topics] are areas that include study of groups of people," says Dr. James Anderson, deputy director for program coordination, planning and strategic initiatives at the NIH and one of the authors of the paper.

"These topics are are described by words like socioeconomic status, physical activity, pregnancy," Anderson says. "The ones that did best were really about molecular mechanisms cells, or parts of cells. Words like cilium, DNA polymerase, chimeral chemistry, ribosome. It's not absolute, but it's really quite a striking distinction." The success rates by topic ranged from about 29% to 7.5%.

The researchers used self-reported demographic data in an optional portion of the application one that was not visible to the grant reviewers to identify each applicant's race. They found that over a third of the applications from black scientists were tied to just eight of the 150 topic clusters.

Six of those eight topics involved "communities, or health disparities, and so on," says Anderson, "and those were in the topics that didn't do quite as well" in the funding process.

This difference in topic preference can account for 20% of the overall funding gap for black applicants, the study found, after controlling for other variables such as the applicant's prior academic and professional experience and accomplishments.

Dr. Hannah Valantine, director of the Office of Scientific Workforce Diversity at the NIH and another author on the paper, says black scientists might be more drawn to certain topic areas at the population level because "connection to one's community, and seeing the disparities, drives people to go into science to create a better environment for their community."

"It's critically important that African American scientists are able to advance their career and stay in academia, not only for their own success, but for enhancing the diversity of the biomedical workforce," Valantine says. "Because we know already that when we have a diverse scientific enterprise, we come up with more creative solutions to the problems that we seek to solve."

That concern resonates with Stephani Page, a postdoctoral fellow in biophysics at Duke University Molecular Physiology Institute and initiator of the Twitter hashtag #BLACKandSTEM, even though her field of study lies on the more statistically successful end of the grant-getting spectrum.

"For me, personally," Page says, "the science that gets me really excited, and I get tingles about, tends to be more quantitative, mechanistic science. But I also have the experience of coming up growing up and being a mom as a black woman in this skin. So when I think about what I want my career to be, it's difficult for me to detach from my career meaning something to my community more broadly."

Page says she is losing hope that she can have the community impact she wants helping black scientists feel affirmed while working in her current field. "I don't want to be a scientist who can't be committed and devoted to changing the system," she says.

One underlying cause of the disparity this study documented, Page says, might be that many of the NIH reviewers who evaluate grant proposals only 2.4% of whom were black in this study lack a certain lens when evaluating what research topics deserve priority.

"If you haven't grown up with inequity as deeply ingrained in your lived experience, it's not going to be as important a lens in your life decisions," she says. "The fact that there's data behind it now gives us a space to talk about it differently. Now we can begin to say that the lens makes a difference."

Valantine says the NIH is also actively evaluating whether the disparity is partly due to racial bias by reviewers. A study to be published early next year, she says, "will tell us whether, if we anonymize an application, we can close this gap."

Whatever the causes of the diversity gap, she says, the NIH is committed to closing it, and the study's results suggest several areas of intervention that could help. For one, the NIH has already begun mentoring programs aimed at increasing the diversity of the grant applicant pool.

"Black applicants submitted only 1.5% of the total applications for these R01s," Valantine says, adding that "we must do all we can" to increase that percentage.

In the meantime, the underfunded topics that the study identified are " 'mission critical' areas of NIH," Valantine says. "The solution is figuring out, within NIH, how we can make sure that those areas are funded."

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Racial Disparities In NIH R01 Funding May Be Partly Caused By Topic Choice : Shots - Health News - NPR

Skin Stem Cells Comments Off on Racial Disparities In NIH R01 Funding May Be Partly Caused By Topic Choice : Shots – Health News – NPR | October 18th, 2019

For anyone who has had hip replacement surgery, Im sure they will agree that it is better to get hit by a bus than to undergo another one. Last year after several years of suffering, I decided to take the leap and go for the hip replacement that my specialist recommended. I was told that it was a common surgery and that it was the best solution for me. Between us; it was probably the most painful thing I have ever gone through. So much so, that at the time, I just wanted to die. Not only did the pain persist for several weeks after the operation, but I was on painkillers for days, which eventually added to my suffering. I had to use a walker for the first 2 weeks and then depended on a cane for over 2 months before I could walk on my own.

My entire demeanor changed, as well as the way I dealt with what once were minor things in life. I feared slipping in the shower, going down the stairs or walking my dogs. No one had prepared me for this. Ive had my share of surgeries including a double mastectomy when I was diagnosed with breast cancer but pain wise; this one was by far the worse. I was hoping after a very long recovery that I would never have to face this situation again. Unfortunately, a year later, I am starting to feel pain on the other side and dread the re-experience of my nightmare.

Although, I heard about Stem Cell, I did not know much about it. So I started to investigate for myself, speak to people, enquire about the procedure and look for a doctor in my area who specialized in Stem Cell. I was willing to do just about anything before considering another hip replacement. After extensive research, I came across Dr. Raj, a Double-Board Certified Orthopedic doctor in Beverly Hills, CA. Going to his website; I learned that he has been in private practice for 10 years. He has been named as one of Americas Top Orthopedists, been featured on the Best of LA and has received numerous other accolades and awards as one of the Top Orthopedic doctors. Providing the ultimate in state-of-the-art orthopedic care, Dr. Rajs practice is always on the cutting-edge of surgical and nonsurgical technologies, such as PRP (Platelet Rich Plasma) injections, stem cell injections for tendinitis and arthritis, minimally invasive surgery and more.

He is Board Certified as a Medical Legal Specialist in America, as well as, Canada and Dubai (Trial, Testimony, Deposition, IME) with a Subspecialty in Hip and Knee Surgery in Los Angeles, including Sports Surgeries.

He is also an Undergraduate from Dalhousie University in Halifax and Canada. He pursued his medical education at Memorial University PGME, before doing his internship and residency in the Department of Orthopedic Surgery. Now that I had found Dr. Raj, all I needed was to get myself educated. So lets start by what are stem cells? This is what I read: Mesenchymal stem cells (MSCs), commonly called stem cells, are precursor cells that havent decided yet what they are going to be in the body. They can differentiate into multiple forms including bone, cartilage, fat and other connective tissues. They play a significant role in the reparative processes throughout the human body.

Where do we find stem cells?

They may be harnessed from fat tissue, bone marrow, synovial tissue or umbilical cord tissue. While stem cell therapy is a promising technology, there is much we are still learning about the causes and pathways that lead to symptomatic osteoarthritis. We have not optimized the factors found in stem cell therapies. To be sure, only the good cells and growth factors are injected into a specific joint. And that is why further research is necessary before being approved by the FDA.

My next move would be to consult with Dr. Raj who would tell me the medical truth, beginning with this question:

What is the current state of Stem Cells and its success rate?

It's relatively new. It's been popular for about 20 years, internationally. In areas like Germany and Korea, it was utilized a lot more. It became popular here when athletes like Kobe Bryant started going to Germany for modified versions of PRP, which led on to regenerative technologies. We have a stigma correlating stem cells with abortions and issues like that. This in itself is completely different. We are not utilizing amniotic stem cells or placenta stem cells. We're utilizing your own stem cells. For issues such as a hip replacement, the most powerful stem cells are the ones in your body. Bone marrow stem cells work well on joints. Joints have zero blood supply. So, if God or the higher power created us where we had blood supply going through our joints, like a cut in our skin - we would constantly replenish or repair. A break in our bone would repair. If you get stem cells and you're in decent enough shape, you will heal no matter what because these stem cells will deposit. Will you heal straight? Probably not - that's where we come into play.

The reason why joints; hips, knees and shoulders degenerate is because there is no blood supply. So, if you have a cut or a loss of cartilage, it stays like that and accumulates overtime. The only way you can control it is externally. You get stronger, you lose weight and you increase your range of motion. But you can't control anything internally.

So regenerative technology is basically utilizing these cells to regenerate cartilage and repair. These are the same cells that flow through our body - and upon signal of an injury will heal skin to skin, bone to bone, tendon to tendon, muscle to muscle. Our joints are just an alcove of joint fluid and no blood supply. The whole concept is - throughout the years, we did steroid injections - they're like band aids. Basically they mask pain. What does masking pain do? It propagates injury. Because we put the band aid on, we don't feel it and we do more. We take this little cut or loss of cartilage and we make it even more over time.

Why is it that specialists do not recommend seeing a surgeon at a certain stage?

There are a lot of people who think one way and everyone is entitled to their own opinions. You can't change opinions.

Are people afraid of stem cells?

Some people are afraid because of stem cells causing cancer. But that's embryonic stem cells.

What is the process?

Bone marrow stem cells are the best because there is a higher chance of live stem cells. Less manipulation, meaning that - in a Mayo Clinic study 4 or 5 years ago, which has a two year follow through on people who are ready to get replacements for joint or knee - they had an 80% success rate where they didn't need it. I do replacements and I do stem cells.

How do you determine what's better for the patient?

My knowledge and years of experience. Also, my knowledge with fitness and being athletic myself. Understanding at a certain point, someone is mechanically compromised. Bone on bone is a term that's been used for years. There are a lot of people who think they are 'bone on bone." Coming from Canada, the US is notorious for doing unnecessary surgeries and replacements. It's the highest rate of replacements in the world. I do not like the term 'bone on bone' because a surgeon will look at an x-ray and say you're bone on bone because that's all they do: replacements. They become a 7-11 or 99 Cents store, lining up 21 people a day. That's not the right way to do things. You don't want to be one of those 21 people getting a replacement because you're not getting that surgeon's full attention. The reality is - you have a PA or an old plastic surgeon who's doing most of your surgery and there is more likelihood of issues. Amongst every specialty there is a lot of ignorance. The whole concept is - you preserve what you have for as long as you can. You have beauty on the outside; you need beauty on the inside too. What's beauty on the inside? Feeling good, you're less inflamed and your joints are healthy.

How does it work with a stem cell procedure?

I extract bone marrow from your pelvis. Take approximately 6 ccs. Under slight sedation, it takes about 5 minutes to take it. Then we separate it via an FDA approved technique. Per FDA, we cannot add anything to it, nor would I want to. We cannot harvest it because the longer it's outside of the body, the better it is. Basically, we then inject those pure cells right away into the joint. It's a four month process for an 80% of regeneration. So, it's not just reduction of inflammation, it's regeneration. It will be a year for a 100% effect. I've had probably about 20% of patients who have taken 6 months+. I've had over a 95% success rate with this technology.

Are you one of the only doctors doing this in LA?

I'm one of them. There are some family and pain management doctors who are doing it. I'm the only Orthopedic surgeon doing it. I'm sure different practitioners are starting to.

Dr. Raj and patient Paula Abdul

How often do you do the stem cell procedure?

You do it one time. It's a powerful injection and there are people Ihave 6 years out who are doing well.

Does it hurt after the fact?

No, not at all. You can walk and move. For example, with your hip - I would combine it with physical therapy to increase your range of motion. Once you have the anti-inflammatory effect, you have to take advantage of it. If you don't increase your range of motion - what happens is - you're walking on one nail vs. 100 nails. You want to dissipate the force over a greater area so that there's a higher chance of external success. Then you strengthen the muscles.

Are there people who are not good candidates for it?

Yes, when it's too far gone. Like I said, people are told they're bone on bone when they're not. They show you different views. It's a marketing gimmick. That person is lined up and ready to sell. Age is relative. There's physiologic age. It really depends on the person. Hypothetically, if you're an inflamed mess, a drinker and abusive to your body, then nothing is going to work. If you take care of yourself and you're motivated with the right protoplasm, then it's going to work.

What about the skeptics or the ones who think it's bad for you?

Don't get me wrong; amniotic stem cells are good for certain situations. Embryonic is bad. It means that it's too far gone. You want live stem cells in an area that does not have blood supply. The data is out there. How can you argue against a Mayo Clinic study with an 80% success rate? How can you argue against the hospitals for special surgery in New York that's doing it, or the Steadman Hawkins Clinic, I'm doing it. Top facilities in the world are doing it and a number of top athletes who are getting it done with success rates. Who's ignorant? Is it that one surgeon or everyone else?

Does insurance cover it?

No, not yet. Insurances are very backwards in terms of their understanding. They would rather cover a replacement.

Is it expensive?

If you break it down par and par and avoid a replacement, not really. On average, you're talking about $7,000, versus hospital, surgeon, facility fees+++,which can be about $25,000.

You're very progressive.

There are a lot of things that I do to try and reduce pain significantly.When I use screws, I use screws that are made out of calcium so they dissolve in your body. Some of my colleagues use tourniquet, I don't use one. I control bleeding and do it in less than an hour. The whole concept is, you don't have atourniquetsqueezing your leg and toxins causing significant pain.

And there you have it. Everything is a risk in life, we do not know if we will wake up tomorrow or if you will get hit by a car and so on so why not try this procedure. I believe that I am lucky enough to have met Dr. Raj. I have taken the decision to undergo the stem cells therapy FDA approved or not, anything before going under the knife one more time. Stay tuned, I will give you a report on the progress.

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Dr. Raj & Stem Cell Therapy Innovation - LATF USA

Skin Stem Cells Comments Off on Dr. Raj & Stem Cell Therapy Innovation – LATF USA | October 18th, 2019

Global Cosmetic Skin Care Market report covers the present scenario and the growth prospects of the global market and includes a discussion of the key vendors operating in the market. It intends to supply an entire 360-degree perspective of this market concerning cutting edge technology, key advancement, drivers and restraints and prospective trends with impact analysis. This study also analyzes the market status, market share, growth rate, future trends, market drivers, opportunities and challenges, risk and entry barriers. This Global Cosmetic Skin Care Market report presents the market competitive landscape and a corresponding detailed analysis of the major vendor/key players in the market.

Global cosmetic skin care market is set to witness a substantial CAGR of 5.5% in the forecast period of 2019- 2026. The report contains data of the base year 2018 and historic year 2017. Increasing self-consciousness among population and rising demand for anti- aging skin care products are the factor for the market growth.

Key Market Competitors:

Few of the major competitors currently working in the global cosmetic skin care market are LOral, Unilever, New Avon Company, Este Lauder Companies, Espa, Kao Corporation, Johnson & Johnson Services, Inc., Procter & Gamble, Beiersdorf, THE BODY SHOP INTERNATIONAL LIMITED, Shiseido Co.,Ltd., Coty Inc., Bo International, A One Cosmetics Products, Lancme, Clinique Laboratories, llc., Galderma Laboratories, L.P., AVON Beauty Products India Pvt Ltd, Nutriglow Cosmetics Pvt. Ltd, Shree Cosmetics Ltd among others.

Market Definition:

Cosmetic skin care is a variety of products which are used to improve the skins appearance and alleviate skin conditions. It consists different products such as anti- aging cosmetic products, sensitive skin care products, anti- scar solution products, warts removal products, infant skin care products and other. They contain various ingredients which are beneficial for the skin such as phytochemicals, vitamins, essential oils, and other. Their main function is to make the skin healthy and repair the skin damages.

Segmentation:Global Cosmetic Skin Care Market

Global Cosmetic Skin Care Market By Product (Anti-Aging Cosmetic Products, Skin Whitening Cosmetic Products, Sensitive Skin Care Products, Anti-Acne Products, Dry Skin Care Products, Warts Removal Products, Infant Skin Care Products, Anti-Scars Solution Products, Mole Removal Products, Multi Utility Products), Application (Flakiness Reduction, Stem Cells Protection against UV, Rehydrate the skins surface, Minimize wrinkles, Increase the viscosity of Aqueous, Others), Gender (Men, Women), Distribution Channel (Online, Departmental Stores and Convenience Stores, Pharmacies, Supermarket, Others), Geography (North America, Europe, Asia-Pacific, South America, Middle East and Africa) Industry Trends and Forecast to 2026

Competitive Analysis:

Global cosmetic skin care market is highly fragmented and the major players have used various strategies such as new product launches, expansions, agreements, joint ventures, partnerships, acquisitions, and others to increase their footprints in this market. The report includes market shares of cosmetic skin care market for Global, Europe, North America, Asia-Pacific, South America and Middle East & Africa.

Table Of Content: Cosmetic Skin Care Market

Part 01: Executive SummaryPart 02: Scope Of The ReportPart 03: Cosmetic Skin CarePart 04: Global Cosmetic Skin Care Market SizingPart 05: Global Cosmetic Skin Care Market Segmentation By ProductPart 06: Five Forces AnalysisPart 07: Customer LandscapePart 08: Geographic LandscapePart 09: Decision FrameworkPart 10: Drivers And ChallengesPart 11: Market TrendsPart 12: Vendor LandscapePart 13: Vendor Analysis

Continue. .

For Detailed TOC @ https://www.databridgemarketresearch.com/toc/?dbmr=global-cosmetic-skin-care-market

Market Drivers:

Market Restraints:

Key Developments in the Market:

Key Insights in the report:

The report provides insights on the following points:

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Global Cosmetic Skin Care Market 2019: Industry Analysis and Detailed Profiles of Top Industry Players LOral, Unilever, New Avon Company, Este Lauder...

Skin Stem Cells Comments Off on Global Cosmetic Skin Care Market 2019: Industry Analysis and Detailed Profiles of Top Industry Players LOral, Unilever, New Avon Company, Este Lauder… | October 18th, 2019

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Analysis on Worldwide Autologous Stem Cell Based Therapies Market Inclinations Exhibit Growing Demand During The Period Until 2025 - Wheel Chronicle

Cardiac Stem Cells Comments Off on Analysis on Worldwide Autologous Stem Cell Based Therapies Market Inclinations Exhibit Growing Demand During The Period Until 2025 – Wheel Chronicle | October 18th, 2019

When young athletes experiences sudden cardiac death as they run down the playing field, it's usually due to arrhythmogenic cardiomyopathy (ACM), an inherited heart disease. Now, Johns Hopkins researchers have shed new light on the role of the immune system in the progression of ACM and, in the process, discovered a new drug that might help prevent ACM disease symptoms and progression to heart failure in some patients.

"We realized that heart muscle inflammation in ACM is much more complicated than we thought, but also might provide a therapeutic strategy," saysStephen Chelko, Ph.D., assistant professor of medicine at the Johns Hopkins University School of Medicine and senior author of the new paper, inSept. inCirculation.

In ACM, patients often harbor mutations in any of the five genes that make up the cardiac desmosome -- the gluelike material that holds heart cells together and helps coordinate mechanical and electrical synchronization of heart cells. Because of this, it's often called "a disease of the cardiac desmosome." In patients with ACM, heart cells pull apart over time, and these cells are replaced with damaged and inflamed scar tissue. These scars can increase risk of instances of irregular heart rhythms and lead to sudden cardiac death if the scar tissue causes the heart wall to stiffen and renders it unable to pump.

If a person is aware they carry an ACM-causing genetic mutation, doctors help them avoid cardiac death through lifestyle changes, such as exercise restriction, and medications that keep their heart rate low. However, there are currently no drugs that treat the underlying structural defects of the desmosome. People who live for many years with ACM still accumulate scar tissue and inflammation in their hearts, leading to chronic heart disease.

"We tended in the past to view ACM as something that kills due to a sudden arrhythmic event," said Chelko. "But now we're starting to also see it as a chronic inflammatory disease that can progress more slowly over time, leading to heart failure."

Chelko and his colleagues wanted to determine the molecular cause of inflammation in the hearts of people with ACM. So they studied mice with an ACM-causing mutation, as well as heart muscle cells generated from stem cells isolated from an ACM patient. They found that the inflammation associated with the disease arose from two separate causes. First, they noticed high levels of macrophages, a type of immune cell that's normally found at sites of inflammation, such as around cuts or scrapes that are healing.

"Macrophages are usually the good guys who help heal a wound and then leave," said Chelko. "But in ACM they're permanently setting up shop in the heart, which, over time, reduces its function."

Chelko's team also found that in ACM, the heart cells themselves are triggered by a protein known as nuclear factor kappa-light-chain-enhancer of activated B cells (NF-B) to produce chemicals called cytokines, which act as homing beacons for other inflammatory cells and molecules. When the researchers treated mice or isolated cells with a drug blocking NF-B, heart cells stopped producing many of these cytokines, leading to decreased inflammation and infiltration of inflammatory cells. In mouse models of ACM, animals treated with the NF-B-blocking drug Bay-11-7082 had a twofold increase in heart function, measured by how much blood their hearts could pump over time compared with untreated ACM animals. They also had a twofold reduction of damaged and inflammatory scar tissue in the heart.

More than one-third of patients with ACM who die of sudden cardiac death have no previous cardiac symptoms, so wouldn't ever know to seek treatment. However, for relatives of these people who discover that they carry a genetic mutation causing ACM -- or those who discover the mutation for other reasons -- a drug could help stave off long-term heart disease, Chelko said.

While the Bay-11-7082 drug is currently only used in the lab for experimental purposes, the U.S. Food and Drug Administration has approved canakinumab, a drug that targets the same inflammatory pathway, for use in juvenile arthritis and a collection of rare auto-inflammatory syndromes. Canakinumab is also being studied for use in coronary artery disease. Chelko's group is now investigating whether this drug would have the same effect as Bay-11-7082 in ACM.

"We're very excited to have found an FDA-approved drug that can reduce heart inflammation in ACM, and we're eager to do more research to ultimately help those who carry these genetic mutations," said Chelko.

Reference:Chelko, et al. (2019) Therapeutic Modulation of the Immune Response in Arrhythmogenic Cardiomyopathy. Circulation. DOI:https://doi.org/10.1161/CIRCULATIONAHA.119.040676

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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Drug Treats Inflammation Related to Genetic Heart Disease - Technology Networks

Cardiac Stem Cells Comments Off on Drug Treats Inflammation Related to Genetic Heart Disease – Technology Networks | October 18th, 2019

Mediating systemic health

Sphingosine 1-phosphate (S1P) is an important circulating lipid mediator that is derived from the metabolism of cell membranes. Its diverse homeostatic roles, particularly in immunology and vascular biology, can go awry in numerous diseases, including multiple sclerosis, cardiovascular diseases, and fibrosis. The centrality of S1P signaling has led to the development of several drugs, including two approved for treatment of multiple sclerosis. In a Review, Cartier and Hla discuss the current understanding of how one mediator can carry out so many signaling roles in different tissues, how these become dysregulated in disease, and efforts in drug development to target S1P signaling.

Science, this issue p. eaar5551

Sphingosine 1-phosphate (S1P), a product of membrane sphingolipid metabolism, is secreted and acts through G proteincoupled S1P receptors (S1PRs) in vertebrates. S1PR isoforms mediate complex cellular actions either alone or in combination in most organ systems. This stable lysolipid circulates as a complex with protein chaperones that not only enables aqueous solubility but also helps facilitate specific modes of receptor signaling. However, differential concentration gradients of S1P are normally present in various compartments and are perturbed under disease conditions. The abundance of circulatory S1P and the high expression of S1PRs in exposed cellsthat is, vascular and hematopoietic cellsposes a key question of how this signaling axis is regulated. This question is of clinical relevance because the first S1PR-targeted drug, fingolimod, has been approved for the treatment of multiple sclerosis since 2010. Recent findings from basic research as well as insights gleaned from clinical and translational studies have enriched our understanding of how this simple lysolipid evolved as a complex regulator of multiple physiological systems and, when dysregulated, contributes to numerous diseases.

Extracellular spatial gradients of S1P, demonstrated by using S1P reporters, are tightly regulated and control fundamental processes such as hematopoietic cell trafficking, immune cell fate, and vascular integrity. The gradients are formed through location-specific function of metabolic enzymes, S1P transporters, and chaperones. Such physiological S1P gradients are altered in diseases, thus contributing to conditions such as inflammation, autoimmunity, and vascular dysfunction. S1P complexed to chaperone proteinsfor example, high-density lipoproteinbound apolipoprotein Mmediate distinct modes of receptor activation, resulting in biased receptor signaling and specific biological outcomes. S1PRs are also regulated tightly through endocytic mechanisms and receptor modulators that enhance or inhibit signal strength and duration. Various signaling mechanisms of this simple lysolipid mediator has helped reveal its multiple actions in the immune system, which include adaptive immune cell localization in various compartments (egress versus retention), fate switching, survival, and activation that influences both cell-mediated and humoral immunity. In the cardiovascular system, high expression of multiple S1PR isoforms in various cell types regulate development, homeostasis, and physiology. Current S1PR-targeted drugs that aim to tame autoimmunity exhibit considerable cardiovascular-adverse events. In the central nervous system (CNS), widespread application of S1PR-targeted drugs in autoimmune neuroinflammatory diseases has stimulated research that revealed the broad but poorly understood effects of S1P signaling in neurodevelopment, the neurovascular unit, neurons, and glia. Furthermore, in addition to the involvement of pathological S1P signaling in acute ischemic conditions of various organs, chronic dysregulated S1P signaling has been implicated in fibrotic diseases of lung, heart, liver, and kidney.

Considerable challenges remain to fully harness the new knowledge in S1P pathobiology to translational utility in clinical medicine. Approaches that mimic S1P chaperones, S1P neutralizing agents, modulation of transporters, biased agonists and antagonists of S1PR isotypes, and sphingolipid metabolic enzyme modulators provide viable pathways to therapy. Focusing on the immune system, such approaches may widen the autoimmunity therapeutic landscape and provide new directions in cancer and chronic inflammatory diseases. For cardiovascular diseases, ischemic conditions as well as chronic heart failure are likely candidates for future translational efforts. Although further work is needed, S1P-targeted approaches may also be useful in regenerative therapies for the aging and diseased myocardium. The CNS-targeted efforts may cross into neurodegenerative diseases, given the success with S1PR-targeted drugs in reducing brain atrophy in multiple sclerosis. Other potential applications include approaches in pain management and neurodevelopmental disorders. Such strategies, although challenging, are greatly helped by findings from basic research on S1P pathobiology as well as pharmacological and clinical insights derived from the application of S1P-targeted therapeutics.

Extracellular S1P gradients created by transporters, chaperones (ApoM+HDL), and metabolic enzymes (LPP3) interact with S1PRs on the cell surface. Receptor activity, transmitted by means of G proteins, is regulated by multiple mechanisms, including -arrestin coupling, endocytosis, and receptor modulators. The resultant cellular changes influence multiple organ systems in physiology and disease.

Sphingosine 1-phosphate (S1P), a metabolic product of cell membrane sphingolipids, is bound to extracellular chaperones, is enriched in circulatory fluids, and binds to G proteincoupled S1P receptors (S1PRs) to regulate embryonic development, postnatal organ function, and disease. S1PRs regulate essential processes such as adaptive immune cell trafficking, vascular development, and homeostasis. Moreover, S1PR signaling is a driver of multiple diseases. The past decade has witnessed an exponential growth in this field, in part because of multidisciplinary research focused on this lipid mediator and the application of S1PR-targeted drugs in clinical medicine. This has revealed fundamental principles of lysophospholipid mediator signaling that not only clarify the complex and wide ranging actions of S1P but also guide the development of therapeutics and translational directions in immunological, cardiovascular, neurological, inflammatory, and fibrotic diseases.

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Sphingosine 1-phosphate: Lipid signaling in pathology and therapy - Science Magazine

Cardiac Stem Cells Comments Off on Sphingosine 1-phosphate: Lipid signaling in pathology and therapy – Science Magazine | October 18th, 2019

Sudden Infant Death Syndrome (SIDS), sometimes known as crib death, occurs when an infant under the age of one dies inexplicably.The typically healthy child will often die while sleeping and is the leading cause of death of children between the ages of one month and one year, claiming approximately 3000 lives a year. There has been little known about the cause of SIDS but new research is now showing that some form of SIDS could be linked to a genetic inability to digest milk.

A study out of theUniversity of Washington School of Medicine focused on the "mitochondrial tri-functional protein deficiency, a potentially fatal cardiac metabolic disorder caused by a genetic mutation in the gene HADHA."

It found that newborns with had the genetic mutation are unable toproperly digest some of the fats found in breastmilk, resulting in cardiac arrest. It found that "the heart cells of affected infants do not convert fats into nutrients properly," and once these fats build up they can cause serious heart and heart health issues.

There are multiple causes for sudden infant death syndrome, said Hannele Ruohola-Baker, who is also associate director of the UW Medicine Institute for Stem Cell and Regenerative Medicine. There are some causes which are environmental. But what were studying here is really a genetic cause of SIDS. In this particular case, it involves a defect in the enzyme that breaks down fat.

Lead author on the study Dr. Jason Miklassaid that it was his experience researching heart disease that prompted him to look at the possible link with SIDS. There was one particular study that had noted a link between children who had problems processing fats and who also had cardiac disease that caused him to delve a little deeper.

Miklas andRuohola-Baker teamed up to begin their own research study.If a child has a mutation, depending on the mutation the first few months of life can be very scary as the child may die suddenly,Miklas noted. An autopsy wouldnt necessarily pick up why the child passed but we think it might be due to the infants heart-stopping to beat.

Were no longer just trying to treat the symptoms of the disease, Miklas added. Were trying to find ways to treat the root problem. Its very gratifying to see that we can make real progress in the lab toward interventions that could one day make their way to the clinic.

Ruohola-Baker says their findings are a big breakthrough in understanding SIDS. There is no cure for this, she said. But there is now hope because weve found a new aspect of this disease that will innovate generations of novel small molecules and designed proteins, which might help these patients in the future.

Read Next:Babies May Not Be 'Designed' For Sleeping, According To SIDS Expert

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SIDS May Be Linked To A Genetic Inability To Digest Milk, Study Finds - Moms

Cardiac Stem Cells Comments Off on SIDS May Be Linked To A Genetic Inability To Digest Milk, Study Finds – Moms | October 18th, 2019