Of late, there has been an increasing awareness regarding the therapeutic potential of stem cells for management of diseases which is boosting the growth of the stem cell therapy market. The development of advanced genome based cell analysis techniques, identification of new stem cell lines, increasing investments in research and development as well as infrastructure development for the processing and banking of stem cell are encouraging the growth of the global stem cell therapy market.

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One of the key factors boosting the growth of this market is the limitations of traditional organ transplantation such as the risk of infection, rejection, and immunosuppression risk. Another drawback of conventional organ transplantation is that doctors have to depend on organ donors completely. All these issues can be eliminated, by the application of stem cell therapy. Another factor which is helping the growth in this market is the growing pipeline and development of drugs for emerging applications. Increased research studies aiming to widen the scope of stem cell will also fuel the growth of the market. Scientists are constantly engaged in trying to find out novel methods for creating human stem cells in response to the growing demand for stem cell production to be used for disease management.

It is estimated that the dermatology application will contribute significantly the growth of the global stem cell therapy market. This is because stem cell therapy can help decrease the after effects of general treatments for burns such as infections, scars, and adhesion. The increasing number of patients suffering from diabetes and growing cases of trauma surgery will fuel the adoption of stem cell therapy in the dermatology segment.

Global Stem Cell Therapy Market: Overview

Also called regenerative medicine, stem cell therapy encourages the reparative response of damaged, diseased, or dysfunctional tissue via the use of stem cells and their derivatives. Replacing the practice of organ transplantations, stem cell therapies have eliminated the dependence on availability of donors. Bone marrow transplant is perhaps the most commonly employed stem cell therapy.

Osteoarthritis, cerebral palsy, heart failure, multiple sclerosis and even hearing loss could be treated using stem cell therapies. Doctors have successfully performed stem cell transplants that significantly aid patients fight cancers such as leukemia and other blood-related diseases.

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Global Stem Cell Therapy Market: Key Trends

The key factors influencing the growth of the global stem cell therapy market are increasing funds in the development of new stem lines, the advent of advanced genomic procedures used in stem cell analysis, and greater emphasis on human embryonic stem cells. As the traditional organ transplantations are associated with limitations such as infection, rejection, and immunosuppression along with high reliance on organ donors, the demand for stem cell therapy is likely to soar. The growing deployment of stem cells in the treatment of wounds and damaged skin, scarring, and grafts is another prominent catalyst of the market.

On the contrary, inadequate infrastructural facilities coupled with ethical issues related to embryonic stem cells might impede the growth of the market. However, the ongoing research for the manipulation of stem cells from cord blood cells, bone marrow, and skin for the treatment of ailments including cardiovascular and diabetes will open up new doors for the advancement of the market.

Global Stem Cell Therapy Market: Market Potential

A number of new studies, research projects, and development of novel therapies have come forth in the global market for stem cell therapy. Several of these treatments are in the pipeline, while many others have received approvals by regulatory bodies.

In March 2017, Belgian biotech company TiGenix announced that its cardiac stem cell therapy, AlloCSC-01 has successfully reached its phase I/II with positive results. Subsequently, it has been approved by the U.S. FDA. If this therapy is well- received by the market, nearly 1.9 million AMI patients could be treated through this stem cell therapy.

Another significant development is the granting of a patent to Israel-based Kadimastem Ltd. for its novel stem-cell based technology to be used in the treatment of multiple sclerosis (MS) and other similar conditions of the nervous system. The companys technology used for producing supporting cells in the central nervous system, taken from human stem cells such as myelin-producing cells is also covered in the patent.

Global Stem Cell Therapy Market: Regional Outlook

The global market for stem cell therapy can be segmented into Asia Pacific, North America, Latin America, Europe, and the Middle East and Africa. North America emerged as the leading regional market, triggered by the rising incidence of chronic health conditions and government support. Europe also displays significant growth potential, as the benefits of this therapy are increasingly acknowledged.

Asia Pacific is slated for maximum growth, thanks to the massive patient pool, bulk of investments in stem cell therapy projects, and the increasing recognition of growth opportunities in countries such as China, Japan, and India by the leading market players.

Global Stem Cell Therapy Market: Competitive Analysis

Several firms are adopting strategies such as mergers and acquisitions, collaborations, and partnerships, apart from product development with a view to attain a strong foothold in the global market for stem cell therapy.

Some of the major companies operating in the global market for stem cell therapy are RTI Surgical, Inc., MEDIPOST Co., Ltd., Osiris Therapeutics, Inc., NuVasive, Inc., Pharmicell Co., Ltd., Anterogen Co., Ltd., JCR Pharmaceuticals Co., Ltd., and Holostem Terapie Avanzate S.r.l.

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Stem Cell Therapy Market Estimated to Expand at a Robust CAGR over 2025 - The Monitor

Cardiac Stem Cells Comments Off on Stem Cell Therapy Market Estimated to Expand at a Robust CAGR over 2025 – The Monitor | December 30th, 2020

Shahid Akhter, editor, ETHealthworld, spoke to Mayur Abhaya, MD & CEO, LifeCell International, to know more about the latest advancements in Stem cell industry and how it has recovered from the Covid challenges.Impact and challenges of Covid-19 on the Stem cell industry ?One of the biggest issues faced by the stem cell industry during the pandemic was the transport of the cells after collection at birth. It needs to reach the lab within 72 hours and in the case of bone marrow stem cell from donation all the way until it reaches the patient that also has to be completed within 72 hours. The bone marrow cells cannot be handed over in courier. It has to be manually hand carried and that created a huge logistics hurdle were transplants significantly reduced in numbers because of the availability of donors and the transport issues around it.

What are the current global trends in the Stem Cell Industry at large?The recent clinical progress that has been made in the medical space of stem cell transplantation is that now cord blood is considered as a better source than bone marrow cells. This was found in a research study based in US, published in 2020. They have also shown that stem cells from the cord blood can now be used across many conditions with the same treatment protocol. Besides that, the preparation of the patient is different in different conditions but now they have simplified that and reduced the risk of death to a very, very low number. So the cord blood is preferred, as the outcomes are improving.How has Life Cell managed the scenario during the pandemic? Do let us know your challenges and the way forward plan?One of the biggest issues during the pandemic was transport, especially the flight operations because we heavily depend on them for moving the samples across the country. We had to revert to an alternate plan where we had to transport these samples through a relay network from one city to another, through a road network. Luckily LifeCell has operations across the country covering more than 250 cities. So still we had the ability to ensure that our commitment of getting the samples to the lab within those 72 hours was very much possible.

Another major milestone during this pandemic that we were able to help was to support a transplant were a child having Aplastic Anaemia needed not one but two cord blood units for the transplant and within the family they couldnt find a match. Luckily because of the LifeCell network and the inventory size of 50,000 units we were able to meet the requirements of the transplant and happy to share the outcome was very successful. So LifeCell ensures that we have appropriate training for its paramedical staff and they are also provided with the appropriate personal protective gears. There are restrictions on the entry of the team inside during the collection we work with the medical staff in the collection rooms, in the operation theatres to ensure a smooth and a well organised collection and even at the lab we have protocols that ensures hygiene and safety within the team and, the operating rooms we have for processing are also well managed.

Your future plans to ensure the smooth collection of Cord Blood?To ensure business continuity we have our teams located very close to our lab itself, you know, so about 100+ member team are placed within a Kilometer of the operating facility. We have adequate stocks, lots of the testing and the processing, consumables that we use are imported. We, at least, maintain 3 month inventory. We also have onsite power back up systems which include a month of diesel supply, month of liquid nitrogen supply and the teams also have a plan that we have a back site also with arrangements done. If for any reason we have cut off of the Chennai centre we have arrangements with an alternate lab to ensure the continuity of the operations

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Stem cells from cord blood can now be used across many conditions: Mayur Abhaya, MD & CEO, LifeCell Internat.. - ETHealthworld.com

Bone Marrow Stem Cells Comments Off on Stem cells from cord blood can now be used across many conditions: Mayur Abhaya, MD & CEO, LifeCell Internat.. – ETHealthworld.com | December 28th, 2020

Siblings Nabila Nakigozi and Fauzan Ssebaggala were diagnosed with sickle cell disease at birth, and with time running out they are taking action.

20-year old Nabila was also diagnosed with avascular necrosis the collapse of bones and as a result is losing her left hip.

She said: Sickle cell is very unpredictable. You can go from happy, to in pain and crying in a matter of minutes. Pain has stolen my childhood and now my adulthood.

Sickle cell disease (SCD) is a group of inherited red blood cell disorders. Those with SCD red blood cells are crescent or sickle shape. This can block blood flow through the body and can lead to many health problems including strokes and episodes of pain.

Fauzan, 24, is now disabled also due to avascular necrosis which has led to the collapse of his spine and hips.

He said: I spent last night crying in pain, every breath I take is painful.

There are two known cures for sickle cell, bone marrow and stem cell transplants.

After extensive research, the siblings have located a private doctor in India who specialises in sickle cell and are fundraising for the expensive but life- saving bone marrow transplant.

Nabila said: Discussions about this procedure is what should be happening within the NHS. We have waited for action but there is always an excuse.

On 11 November, The Parliaments Joint Committee released a report titled Black People, Racism and Human Rights.

The report found that more than 60% of Black people in the UK do not believe their health is as equally protected by the NHS compared to white people.

SCD commonly affects people of African and Caribbean descent.

Fauzan said: The NHS as a whole needs to re-evaluate its position on sickle cell because they can be very insensitive towards us.

There are assumptions that we are not really in pain and just addicted to morphine.

For Nabila, going through this with her brother makes every day easier.

She said: It might sound odd, but its probably the best thing that could have happened to both of us. We lean on each other for support.

But for Fauzan, seeing his sister suffer is difficult to watch.

He said: As an older brother, Im meant to be looking after her. Its heart-breaking knowing there are things I cant do for her.

Nabila is in hospital 80% of the time. I struggle looking into her eyes and confidently saying, tomorrow will be fine.

The east London duo are passionate about highlighting the pain those with SCD go through beyond the surface.

Nabila said: We were born in Uganda and within the African community it isnt always acknowledged that the disease affects us mentally too.

Fauzan added: Ive really struggled with my mental health, and I felt a lot of pressure as a result of not working.

Despite their struggles, the siblings hope to be a voice for those with SCD through their charitable fashion brand, Werent Born Rich.

They make streetwear clothing, with a portion of the profits going to sickle cell patients.

Nabila said: I want people to look at Werent Born Rich and be inspired. If I can be this sick and continue to fight, so can you.

Tracy Williams, project manager for blood donation at the Sickle Cell Society, has advocated through multiple projects for more Black and mixed heritage blood donors.

She manages two projects South London Gives and Give Blood, Spread Love.

Tracy said: Only 1% of people who give blood are of Black heritage. This number needs to increase to help those with sickle cell with Exchange Blood Transfusions.

We advocate for the patient voice, and of that of families affected by SCD. Our goal is to improve the overall wellbeing for people with sickle cell by accessing excellent treatment, mental health support and easily accessible ethnically matched blood.

Despite SCD being the fastest growing genetically inherited condition in the UK, research remains limited.

She said: We have seen positive changes however we would like to see more research for widely accessible treatments. We also continue to campaign for their exemption from prescription charges.

Williams message for the Black community is clear.

She said: Your blood is needed. Donating blood is safe and the process takes half an hour. Its your chance to literally save someones life.

If you are not from our target communities be an ally and share our message too.

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'We had to take our health into our hands' - Meet the siblings fighting for their lives with sickle cell disease - South West Londoner

Bone Marrow Stem Cells Comments Off on ‘We had to take our health into our hands’ – Meet the siblings fighting for their lives with sickle cell disease – South West Londoner | December 28th, 2020

The vape flavorings so popular with kids and young adults are cardiotoxic and disrupt the hearts normal electrical activity, a University of South Florida Health preclinical study finds.

The appealing array of fruit and candy flavors that entice millions of young people take up vaping can harm their hearts, a preclinical study by University of South Florida Health (USF Health) researchers found.

Mounting studies indicate that the nicotine and other chemicals delivered by vaping, while generally less toxic than conventional cigarettes, can damage the lungs and heart. But so far there has been no clear understanding about what happens when the vaporized flavoring molecules in flavored vaping products, after being inhaled, enter the bloodstream and reach the heart, said the studys principal investigator Sami Noujaim, PhD, an associate professor of molecular pharmacology and physiology at the USF Health Morsani College of Medicine.

In their study published on November 20, 2020, in the American Journal of Physiology- Heart and Circulatory Physiology, Dr. Noujaim and colleagues report on a series of experiments assessing the toxicity of vape flavorings in cardiac cells and in young mice.

The flavored electronic nicotine delivery systems widely popular among teens and young adults are not harm-free, Dr. Noujaim said. Altogether, our findings in the cells and mice indicate that vaping does interfere with the normal functioning of the heart and can potentially lead to cardiac rhythm disturbances.

Dr. Noujaims laboratory is among the first beginning to investigate the potential cardiotoxic effects of the many flavoring chemicals added to the e-liquids in electronic nicotine delivery systems, or ENDS. He recently received a five-year, $2.2-million grant from the NIHs National Institute of Environmental Health Sciences to carry out this laboratory research. Commonly called e-cigarettes, ENDS include different products such as vape pens, mods, and pods.

Sami Noujaim, PhD, associate professor of molecular pharmacology and physiology at the University of South Florida Health (USF Health) Morsani College of Medicine, has begun investigating preclinically the potential cardiotoxic effects of many flavoring chemicals added to the e-liquids in electronic nicotine delivery systems. Credit: Photo courtesy of USF Health

Vaping involves inhaling an aerosol created by heating an e-liquid containing nicotine, solvents such as propylene glycol and vegetable glycerin, and flavorings. The vaping devices battery-powered heat converts this e-liquid into a smoke-like aerosolized mixture (e-vapor). Manufacturers tout e-cigarettes as a tool to help quit smoking, but evidence of their effectiveness for smoking cessation is limited, and they are not FDA approved for this use. E-cigarettes contain the same highly addictive nicotine found in tobacco products, yet many teens and young adults assume they are safe.

Among the USF Health study key findings:

Whether the mouse findings will translate to people is unknown. Dr. Noujaim emphasizes that more preclinical and human studies are needed to further determine the safety profile of flavored ENDS and their long-term health effects.

A partial government ban on flavored e-cigarettes aimed at stopping young people from vaping focused on enforcement against flavored e-cigarettes with pre-filled cartridges, like those produced by industry leader JUUL. However, teens quickly switched to newer disposable e-cigarettes still sold in a staggering assortment of youth-appealing fruity and dessert-like flavors.

Our research matters because regulation of the vaping industry is a work in progress, Dr. Noujaim said. The FDA needs input from the scientific community about all the possible risks of vaping in order to effectively regulate electronic nicotine delivery systems and protect the publics health. At USF Health, in particular, we will continue to examine how vaping may adversely affect cardiac health.

In 2020, 3.6 million U.S. youths still used e-cigarettes, and among current users, more than eight in 10 reported using flavored varieties, according to the Centers for Disease Control and Prevention.

Reference: In Vitro and In Vivo Cardiac Toxicity of Flavored Electronic Nicotine Delivery Systems by Obada Abou-Assali, Mengmeng Chang, Bojjibabu Chidipi, Jose L. Martinez-de-Juan, Michelle Reiser, Manasa Kanithi, Ravi Soni, Thomas Vincent McDonald, Bengt Herweg, Javier Saiz, Laurent Calcul and Sami F. Noujaim, 20 November 2020, American Journal of Physiology-Heart and Circulatory Physiology.DOI: 10.1152/ajpheart.00283.2020

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Vape Flavorings Are Cardiotoxic and Can Damage the Heart - SciTechDaily

Cardiac Stem Cells Comments Off on Vape Flavorings Are Cardiotoxic and Can Damage the Heart – SciTechDaily | December 28th, 2020

A U.S. study has shown for the first time that enhancing signals from the nociceptive nervous system could provide new approaches to improve the collection of hematopoietic stem cells (HSCs) for treating cancers of the blood and bone marrow, the authors reported in the December 23, 2020, edition of Nature.

The findings may help cancer treatments, as "in a meaningful fraction of leukemia, multiple myeloma and lymphoma patients, particularly those who have received anticancer treatment, the HSC mobilization yield can be insufficient," said study leader Paul Frenette.

"Therefore, more efficient HSC mobilization methods would allow the harvest of sufficient HSCs for life-saving transplantation," said Frenette, a professor and director of the Ruth L. and David S. Gottesman Institute for Stem Cell and Regenerative Medicine Research at Albert Einstein College of Medicine in New York.

HSCs characteristically migrate to different hematopoietic sites during embryonic development and continue to be released from the adult bone marrow.

Most hematopoiesis occurs in the bone marrow, where different types of blood cells are derived from limited numbers of HSCs, which are multipotent and capable of extensive renewal.

The HSC migratory properties have facilitated their collection from the blood and transplantation as regenerative cancer treatment, although the mechanisms by which HSCs migrate out of the bone marrow remain largely unknown.

In tissues exposed to the external environment, such as the skin, lung and gut, nociceptors enable the rapid detection of external insults, including pain, cold or heat, in order to avoid organ damage.

Nociceptor sensory neurons detect these harmful stimuli and can regulate the immune response to them by releasing neurotransmitters and other regulatory molecules.

However, other than for pain perception, the role of nociceptors in a deep tissue, such as the bone marrow, is poorly understood.

In bone marrow, HSCs are found in specialized microenvironments or niches, which are complex regulatory environments influenced by multiple cellular constituents, including nerves.

Although sympathetic nerves are known to regulate the HSC niche, the role of nociceptive neurons in the bone marrow in this regard remains unclear.

"We have previously shown that nerves from the sympathetic nervous system promoted HSC mobilization in bone marrow, so we were curious to see whether nociceptive neurons might also be involved," Frenette told BioWorld Science.

In their new Nature study, the Frenette and his team showed that nociceptive neurons are required for HSC mobilization and that they interact with sympathetic nerves to maintain HSCs in the bone marrow.

Notably, nociceptor neurons were demonstrated to drive granulocyte colony-stimulating factor (G-CSF)-induced HSC mobilization via secretion of calcitonin gene-related peptide (CGRP).

"Our findings may lead to more efficient mobilization methods in [blood and bone marrow cancer] patients in whom existing methods are insufficient," Frenette said.

For example, "we have shown that the CGRP pathway synergizes with G-CSF and plerixafor [(Mozobil, Genzyme), an immunostimulant used to mobilize HSCs into the bloodstream in cancer patients]." Unlike sympathetic nerves, which regulate HSCs indirectly via the bone marrow niche, CGRP was shown to act directly on HSCs via receptor activity modifying protein 1 (RAMP1) and the calcitonin receptor-like receptor to promote HSC egress via Galphas/adenyl cyclase/cAMP signaling pathway activation.

This research identifies potential treatment targets for boosting HSC mobilization, since "we have shown that increasing the downstream signaling of the CGRP receptor RAMP1, by increasing cyclic AMP can enhance HSC mobilization," said Frenette.

Importantly, the Einstein researchers then showed that ingestion of food containing capsaicin, a natural component of chili peppers that can trigger the activation of nociceptive neurons, significantly enhanced HSC mobilization in mice.

"Capsaicin tablets are currently sold over-the-counter to 'help support cardiovascular and digestive function' at higher doses than that predicted by our studies in mice for stimulating HSC mobilization in humans," said Frenette.

"These relatively low concentrations of capsaicin have biological effects without seemingly causing obvious pain," he said.

Collectively, these findings therefore suggest that targeting the nociceptive nervous system could represent a novel strategy by which to improve the yield of HSCs for stem cell-based therapies. (Gao, X. et al. Nature 2020, Advanced publication).

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Nociceptive neurons shown to boost hematopoiesis | 2020-12-28 - BioWorld Online

Skin Stem Cells Comments Off on Nociceptive neurons shown to boost hematopoiesis | 2020-12-28 – BioWorld Online | December 28th, 2020

A group of scientists in the US, including an Indian-American from the prestigious Cleveland Clinic, have identified a potential new class of drugs that may prove effective in treating certain common types of blood and bone marrow cancers.

First published in the latest edition of Blood Cancer Discovery, the decade long research which reports that a new pharmacological strategy to preferentially target and eliminate leukemia cells with TET2 mutations, was carried out by Jaroslaw Maciejewski and his collaborator Babal Kant Jha from the Cleveland Clinic Department of Translational Hematology & Oncology Research.

Myeloid leukemias are cancers derived from stem and progenitor cells in the bone marrow that give rise to all normal blood cells.

One of the most common mutations involved in driving myeloid leukemias are found in the TET2 gene, which has been investigated for the last decade by Maciejewski and Jha.

In preclinical models, we found that a synthetic molecule called TETi76 was able to target and kill the mutant cancer cells both in the early phases of disease--what we call clonal hematopoiesis of indeterminate potential, or CHIP--and in fully developed TET2 mutant myeloid leukemia, said Dr Maciejewski.

According to a media release, the research team designed TETi76 to replicate and amplify the effects of a natural molecule called 2HG (2-hydroxyglutarate), which inhibits the enzymatic activity of TET genes.

The TET DNA dioxygenase gene family codes for enzymes that remove chemical groups from DNA molecules, which ultimately changes what genes are expressed and can contribute to the development and spread of disease.

While all members of the TET family are dioxygenases, the most powerful enzymatic activity belongs to TET2, the press release said.

Even when TET2 is mutated, however, its related genes TET1 and TET3 provide residual enzymatic activity. While significantly less, this activity is still enough to facilitate the spread of mutated cancer cells.

Maciejewskis and Jhas new pharmacologic strategy to selectively eliminate TET2 mutant leukemia cells centers on targeting their reliance on this residual DNA dioxygenase activity, it said.

We took lessons from the natural biological capabilities of 2HG, explained Jha, a principal investigator.

We studied the molecule and rationally designed a novel small molecule, synthesized by our chemistry group headed by James Phillips, PhD. Together, we generated TETi76a similar, but more potent version capable of inhibiting not just TET2, but also the remaining disease-driving enzymatic activity of TET1 and TET3, Jha said.

Cleveland Clinic said that the researchers studied TETi76s effects in both preclinical disease and xenograft models (where human cancer cells are implanted into preclinical models). Additional studies will be critical to investigate the small molecules cancer-fighting capabilities in patients.

We are optimistic about our results, which show not just that TETi76 preferentially restricts the growth and spread of cells with TET2 mutations, but also gives survival advantage to normal stem and progenitor cells, Jha said.

(This story has been published from a wire agency feed without modifications to the text.)

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New class of drugs to treat blood and bone marrow cancers: Research - Hindustan Times

Bone Marrow Stem Cells Comments Off on New class of drugs to treat blood and bone marrow cancers: Research – Hindustan Times | December 26th, 2020

Cade Cridland thought a lot about fate as he sat tethered to a machine that drained blood from one arm and pumped it back into his body through the other arm.

After four hours, blood stem cells processed by the machine would be flown thousands of miles to a young child he has never met. A child whose name he does not know.

A child battling blood cancer.

This story unfolded in a Denver hospital in September. But it began 14 years earlier with a split-second decision in Las Vegas when Cridland was 24.

Before the child was born.

Im not a religious person by any means, Cridland, now 38, said. But I do believe that theres a lot of fate that takes place in our actions on a day-to-day basis.

The year is 2006. George W. Bush is president.

And Cridland, a recent UNLV graduate with a bachelors degree in journalism and media studies, had just left his part-time job with Vegas PBS for a full-time job with the local chapter of the Leukemia and Lymphoma Society.

Through his new job, Cridland soon found himself at a donation drive, hosted by a charity called Be the Match, for a family desperately searching for a bone marrow donor.

For patients diagnosed with leukemia or lymphoma, a bone marrow or cord blood transplant may be their only hope for a cure.

Cridland was there to work the event, but he drew inspiration from those around him getting swabbed. Whats the harm, he thought.

That day, his DNA was packaged and shipped off to be entered in the National Bone Marrow Registry.

He wasnt a match. Life went on, and eventually Cridland forgot about the swab.

In the meantime, he moved on to a job with the Clark County School District, got married, adopted a dog, had two children, bought a house, got another dog.

Fourteen years came and went, and now it was 2020.

The phone call

The call came at the best time, during arguably one of the worst years in modern history. The 2020 pandemic was in full swing. Protests over racism and police brutality had taken hold of a divided nation.

Cridland, a spokesman for the school district, needed something good to focus on.

But when that something good came calling earlier this year, he almost didnt answer. A toll-free number lit up the screen. A telemarketer maybe, or a scam?

Cridland surprised himself and took the call.

I dont know if you remember this, Cridland recalled a woman on the other end explaining. But you gave a cheek swab at one of our events, and theres a possibility that this cheek swab is a match for a child in need of stem cells or marrow to help them fight blood cancer.

The woman, an employee of Be the Match, ended the call with a question: Would you be willing to donate?

She gave Cridland the weekend to think it over. But for Cridland and his wife, who have two young children, it was a no-brainer.

Not long after, a second cheek swab confirmed what the woman had told Cridland during their phone call.

He was a match for the patient. In this case, that meant at least eight specific genetic markers in Cridlands DNA, called human leukocyte antigens, matched the patients DNA.

Every one individual has their own unique genetic DNA code, said Erica Sevilla, a spokeswoman for Be the Match. What were looking to attach to that code is protein markers that tell your body what cells belong in the body and what cells do not. Essentially, youre looking to match immune systems between the donor and the patient.

From there, the patients doctor will decide the best course for treatment.

According to Be the Match, many believe that the only way to donate blood stem cells is through a surgical procedure, during which the donor receives anesthesia and a needle is used to extract liquid marrow from the pelvic bone. But 79 percent of donations are done through a nonsurgical procedure known as a peripheral blood stem cell donation.

The doctor in Cridlands donation case chose the nonsurgical route.

During the week leading up to the donation, Be the Match sent a nurse to Cridlands home in Henderson once a day for five consecutive days to administer injections of a medication that increased the number of blood-forming cells in Cridlands bloodstream.

Cridland and his wife were flown to Denver for the procedure.

At times throughout the donation process, which spanned about five months from the first phone call to the day of the procedure, Cridland would find himself moved to tears, overcome by his gratitude for the chance to help save a young child.

To me, the crazy thing about all this is that my actions from 14 years ago have had a dramatic effect on how somebody else is living in 2020, he said in an interview this month at his home. With all the negativity weve seen this year, this one family may look at 2020 as the best year of their lives because of this one specific moment in my life that took place 14 years ago.

Even now, three months removed from donation day, Cridland at random will break down in tears.

Sometimes he thinks about his blood pumping through the childs body. How a piece of him will always be with that child. How someone he has never met, and may never meet, could be such a close genetic match to him.

How were all more alike than we think.

Epilogue

I wish I could take this feeling, put it in a can and throw a lid on top, Cridland said of his experience donating stem cells.

If that were possible, Cridland said, he would pass it around like a party favor but he cant.

So instead, hes hoping his story will inspire others, especially people of color, to join the registry and give the gift of a cure to another patient in need.

Currently, there is a severe shortage of diverse donors in the national registry, which consists of 22 million donors. More than 13 percent of the American population is Black, for example, yet only about 4 percent of registered donors are Black.

And the disparity is costing lives.

Matching is based on genetic markers that can be traced back to your grandparents and your great-grandparents, said Sevilla, the spokeswoman for Be the Match. Theyre traced back to the very origins of your family, so thats why people who are of European descent have an easier time finding a match, whereas people who descended from slavery, for example, have a harder time.

To join the National Bone Marrow Registry and request a cheek swab kit, visit http://www.join.bethematch.org.

Meanwhile, as of mid-December, Cridland knew only that the patient had received his stem cells. He wasnt sure, even, if hed ever meet the child.

Both parties must consent to meeting, and different countries operate under varied cooling-off periods. For some countries, a patient and a donor must wait two years before they can meet or even speak.

As a donor, Cridland was told the patients age, specific diagnosis and city and country of residence. But he is not allowed to disclose that information to protect the patients privacy.

According to Be The Match, 50 percent of all marrow or stem cell donations are international.

We are either exporting cells or importing cells, said Sevilla, the charity spokeswoman.

Cridland gave his consent for the patients family to reach out to him in the future. For now, its a waiting game.

If the moment comes that they try to connect, Ill be there, he said.

Contact Rio Lacanlale at rlacanlale@reviewjournal.com or 702-383-0381. Follow @riolacanlale on Twitter.

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He got his cheek swabbed at 24. Nothing happened for 14 years. - Las Vegas Review-Journal

Bone Marrow Stem Cells Comments Off on He got his cheek swabbed at 24. Nothing happened for 14 years. – Las Vegas Review-Journal | December 26th, 2020

By shifting mouse elderly hematopoietic stem cells (aged HSCs) to the environment of young mice (bone marrow niche), it was shown that the pattern of stem cell gene expression was rejuvenated to that of young hematopoietic stem cells. On the other hand, the function of elderly HSCs failed to recover in the young bone marrow niche. The epigenome (DNA methylation) of aged HSCs didnt change significantly even in the young bone marrow niche, and DNA methylation profiles were found to be a better index than the gene expression pattern of aged HSCs.

A research group headed by Professor Atsushi Iwama in the Division of Stem Cell and Molecular Medicine, The Institute of Medical Science, The University of Tokyo (IMSUT) declared these world-first Outcomes and was published in the Journal of Experimental Medicine (online) on November 24th.

The results will contribute to the development of treatments for age-related blood diseases.

Professor Atsushi Iwama, Lead Scientist, IMSUT

The research group investigated whether rejuvenating aged HSCs in a young bone marrow market environment would rejuvenate.

Tens of thousands of elderly hematopoietic stem/progenitor cells gathered from 20-month-old mice were transplanted into 8-week-old young mice without pretreatment like irradiation. After two months of follow-up, they collected bone marrow cells and performed flow cytometric analysis.

The research team also transplanted 10-week-old young mouse HSCs for comparison. In addition, engrafted aged HSCs were fractionated and RNA sequence analysis and DNA methylation analysis were conducted.

They discovered that engrafted elderly HSCs were less capable of producing hematopoietic cells compared to younger HSCs. They also showed that differentiation of aged HSCs into multipotent progenitor cells was persistently impaired even in the young bone marrow market, and that the direction of differentiation was biased. It was found that the transfer of aged HSCs into the young bone marrow market does not enhance their stem cell function.

A more detailed analysis may reveal mechanisms that irreversibly affect aged HSC functionAging studies focusing on HSCs have been chased in mice with a bone marrow transfer model. However, the effect of aging on HSCs remains to be clarified.

Professor Iwama says as follows. This analysis has a substantial impact because it clarified the effect of aging on HSCs. Our results are expected to contribute to further elucidation of the mechanism of aging in HSCs and comprehension of the pathogenic mechanism of age-related blood disorders.

Source:

Journal reference:

Kuribayashi, W.,et al.(2020) Limited rejuvenation of aged hematopoietic stem cells in young bone marrow niche.Journal of Experimental Medicine.doi.org/10.1084/jem.20192283.

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Study clarifies the impact of getting old on hematopoietic stem cells - Microbioz India

Bone Marrow Stem Cells Comments Off on Study clarifies the impact of getting old on hematopoietic stem cells – Microbioz India | December 26th, 2020

A group of scientists in the US, including an Indian-American from the prestigious Cleveland Clinic, have identified a potential new class of drugs that may prove effective in treating certain common types of blood and bone marrow cancers.

First published in the latest edition of Blood Cancer Discovery, the decade long research which reports that a new pharmacological strategy to preferentially target and eliminate leukemia cells with TET2 mutations, was carried out by Jaroslaw Maciejewski and his collaborator Babal Kant Jha from the Cleveland Clinic Department of Translational Hematology & Oncology Research.

Myeloid leukemias are cancers derived from stem and progenitor cells in the bone marrow that give rise to all normal blood cells.

One of the most common mutations involved in driving myeloid leukemias are found in the TET2 gene, which has been investigated for the last decade by Maciejewski and Jha.

In preclinical models, we found that a synthetic molecule called TETi76 was able to target and kill the mutant cancer cells both in the early phases of disease--what we call clonal hematopoiesis of indeterminate potential, or CHIP--and in fully developed TET2 mutant myeloid leukemia," said Dr Maciejewski.

According to a media release, the research team designed TETi76 to replicate and amplify the effects of a natural molecule called 2HG (2-hydroxyglutarate), which inhibits the enzymatic activity of TET genes.

The TET DNA dioxygenase gene family codes for enzymes that remove chemical groups from DNA molecules, which ultimately changes what genes are expressed and can contribute to the development and spread of disease.

While all members of the TET family are dioxygenases, the most powerful enzymatic activity belongs to TET2, the press release said.

Even when TET2 is mutated, however, its related genes TET1 and TET3 provide residual enzymatic activity.

While significantly less, this activity is still enough to facilitate the spread of mutated cancer cells.

Maciejewski's and Jha's new pharmacologic strategy to selectively eliminate TET2 mutant leukemia cells centers on targeting their reliance on this residual DNA dioxygenase activity, it said.

We took lessons from the natural biological capabilities of 2HG, explained Jha, a principal investigator.

We studied the molecule and rationally designed a novel small molecule, synthesized by our chemistry group headed by James Phillips, PhD. Together, we generated TETi76a similar, but more potent version capable of inhibiting not just TET2, but also the remaining disease-driving enzymatic activity of TET1 and TET3, Jha said.

Cleveland Clinic said that the researchers studied TETi76's effects in both preclinical disease and xenograft models (where human cancer cells are implanted into preclinical models). Additional studies will be critical to investigate the small molecule's cancer-fighting capabilities in patients.

We are optimistic about our results, which show not just that TETi76 preferentially restricts the growth and spread of cells with TET2 mutations, but also gives survival advantage to normal stem and progenitor cells, Jha said.

(Only the headline and picture of this report may have been reworked by the Business Standard staff; the rest of the content is auto-generated from a syndicated feed.)

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Scientists find new class of drugs that may treat blood, bone marrow cancer - Business Standard

Bone Marrow Stem Cells Comments Off on Scientists find new class of drugs that may treat blood, bone marrow cancer – Business Standard | December 26th, 2020

Multiple chimeric antigen receptor (CAR) T-cell therapies for the treatment of lymphomas and multiple myeloma have moved forward in the regulatory process, with 1 new FDA approval in 2020 and others anticipated in the near future.

In July, brexucabtagene autoleucel (Tecartus; KTEX19) received accelerated approval for the treatment of adult patients with relapsed or refractory mantle cell lymphoma (MCL) based on the results of the phase 2 ZUMA-2 trial (NCT02601313), bringing the treatment landscape of this hematologic malignancy into a new era.1

This approval is only the very beginning, and we are walking into a sophisticated CAR T-cell therapy era with many constructs being designed with [different mechanisms of action], Michael Wang, MD, said in an interview with Targeted Therapies in Oncology (TTO).

Additional actions by the FDA this year included granting priority review designations to lisocabtagene maraleucel (liso-cel) for the treatment of adult patients with relapsed or refractory (R/R) large B-cell lymphoma, after at least 2 prior therapies,2 as well as to idecabtagene vicleucel (ide-cel; bb2121)as treatment of adult patients with multiple myeloma who have received at least 3 prior therapies, including an immunomodulatory drug (IMiD), a proteasome inhibitor (PI), and an anti-CD38 antibody.3

The approval of brexucabtagene autoleucel, an antiCD19 CAR T-cell product, in MCL was based on objective response rate (ORR) data from patients treated on a single-arm trial who had previously received anthracycline- or bendamustine-containing chemotherapy, an anti-CD20 antibody, and a Bruton tyrosine kinase inhibitor (n = 74).2,4 Eligible patients received leukapheresis and optional bridging therapy, followed by conditioning chemotherapy and a single infusion of brexucabtagene autoleucel 2 106CAR T cells/kg.

The results of ZUMA-2 were published in the New England Journal of Medicine in April and demonstrated a 93% (95% CI, 84%-98%) ORR in 60 response-evaluable patients, 67% (95% CI, 53%-78%) of whom had a complete response (CR). ORRs were consistent across key patient subgroups. Two patients (3%) each had stable and progressive disease.

Progression-free and overall survival (OS) rates at 12 months were 61% and 83%, respectively, and 57% of patients remained in remission at the 12.3-month median follow-up.4 Cytokine release syndrome (CRS) was the most concerning adverse event, occurring in 91% of patients; grade 3 or higher CRS occurred in 15%.

Notably, the patient cohort comprised patients with a median of 3 prior lines of therapy (range, 1-5) and more than half (56%) were considered to have intermediateor high-risk features by the simplified Mantle Cell Lymphoma International Prognostic Index at baseline.

Before CAR T-cell therapy, we did not have any effective means [of getting patients with high-risk MCL into remission]. We used allogeneic transplantation [and] were able to put some of the patients into a long-term remission, but at a heavy price of mortality, said Wang, a professor in the Department of Lymphoma & Myeloma, Division of Cancer Medicine at The University of Texas MD Anderson Cancer Center in Houston. Overall, this brings hope to the high-risk patient population. It looks as though fewer patients are relapsing.

Lisocabtagene Maraleucel In February, the FDA granted liso-cel a priority review designation, an action supported by the safety and efficacy findings of the phase 1 TRANSCEND-NHL-001 trial (NCT02631044).2

Histologic subtypes eligible for treatment included diffuse large B-cell lymphoma (DLBCL); high-grade double- or triple-hit B-cell lymphoma; transformed DLBCL from indolent lymphoma; primary mediastinal B-cell lymphoma; and grade 3B follicular lymphoma. Patients were administered 2 sequential infusions of CD8+ and CD4+ CAR T cells following optional bridging therapy and lymphodepleting chemotherapy and were assigned to 1 of 3 target dose levels: 50 106 (1 or 2 doses), 100 106 , or 150 106 CAR-positive T cells. Investigators determined that the recommended target dose was 100 106 CAR-positive T cells.

In the 256 patients who received at least 1 dose of liso-cel and were included in the efficacy-evaluable group, the ORR was 73% (95% CI, 67%-78%), with 53% (95% CI, 47%-59%) achieving a CR. Investigators observed all-grade CRS (42%) and neurological events (30%), but most cases were grade 1 or 2 in severity.

Due to relatively low rates of CRS and neurological events, the administration of liso-cel has been explored in both the inpatient and outpatient settings. One that included a cohort of patients treated in the outpatient setting with proper monitoring versus the traditional inpatient setting demonstrated consistent safety.6

Based on these results, the indication is that you can deliver [liso-cel] in the outpatient setting and the outcomes are good compared with those treated in the inpatient setting, explained study author Carlos R. Bachier, MD, the director of cellular research at Sarah Cannon in Nashville, Tennessee, in an interview with TTO. Aside from that, they also showed that liso-cel could be safely administered outside of university programs and in more community-based programs, most of them being aligned [with] or part of stem cell and bone marrow transplant programs.

The target action date for a decision on the biologics license application (BLA) for liso-cel was extended twice in 2020 and remains under review. In May, the FDA moved the Prescription Drug User Fee Act (PDUFA) goal date out 3 months from its original August deadline.2,7 Bristol Myers Squibb, the company responsible for developing the product, submitted additional information to the agency following the initial BLA submission, which resulted in more review time. Once again, the target action date was pushed in November, this time due to incomplete manufacturing facility inspections resulting from ongoing travel restrictions due to COVID-19. The FDA provided no new action date.8

For patients with multiple myeloma, the B-cell maturation antigen (BCMA)-targeting CAR T-cell therapy idecel is currently under review for approval in patients who have received at least 3 prior therapiesincluding an immunomodulatory drug (IMiD), a proteasome inhibitor (PI), and an anti-CD38 antibodybased on results of the phase 2 KarMMa trial (NCT03361748).9

Updated trial results were presented at the American Society of Clinical Oncology 2020 Virtual Scientific Program, and showed that both the primary and key secondary end points of ORR and CR rate were 75% and 33%, respectively. The median duration of response was 10.7 months, and the median progression-free survival was 8.8 months in all patients receiving ide-cel. Corresponding medians were 19.0 and 20.2 months among those achieving a CR or stringent CR. The median OS was 19.4 months in all treated patients.

The 128 patients treated received 1 of 3 target dose levels: 150, 300, or 450 106 CAR-positive T cells. The investigators noted that the highest efficacy outcomes were seen in patients in the 450 106 CAR-positive T-cell group, with an ORR of 82% and a 39% CR rate.

The incidence of CRS was 84% across the treatment cohort and increased with higher target doses. Overall, less than 6% of patients have grade 3 or higher CRS and only 1 patient in the highest target dose cohort had a grade 5 event. Neurological toxicity was low across target doses, with no grade 4 or 5 events reported.

At baseline, the majority of patients (51%) had high tumor burden, 39% had extramedullary disease, and 35% had high-risk cytogenetics including deletion 17p or translocations in t(4;14) or t(14;16).

In May, the FDA issued a refusal letter regarding the BLA for ide-cel because the Chemistry, Manufacturing, and Control (CMC) module required more information before they could complete the review.10 In September, the resubmitted application received a priority review and the agency assigned a PDUFA action date of March 27, 2021.11

If approved, ide-cel would be the first CAR T-cell therapy available for the treatment of patients with multiple myeloma.

References:

1. FDA approves brexucabtagene autoleucel for relapsed or refractory mantle cell lymphoma. FDA. Updated July 27, 2020. Accessed November 18, 2020. https://bit. ly/3pEDQV5

2. US Food and Drug Administration (FDA) accepts for priority review Bristol-Myers Squibbs biologics license application (BLA) for lisocabtagene maraleucel (liso-cel) for adult patients with relapsed or refractory large B-cell lymphoma. Press release. Bristol Myers Squibb. February 13, 2020. Accessed November 18, 2020. https:// bit.ly/37ruQbs

3. US Food and Drug Administration (FDA) accepts for priority review Bristol Myers Squibb and bluebird bio application for anti-BCMA CAR T cell therapy idecabtagene vicleucel (ide-cel, bb2121). Press release. Bristol Myers Squibb. September 22, 2020. Accessed November 18, 2020. https://bit.ly/3kDhakH

4. Wang M, Munoz J, Goy A, et al. KTE-X19 CAR T-cell therapy in relapsed or refractory mantle-cell lymphoma. N Engl J Med. 2020;382(14):1331-1342. doi:10.1056/ NEJMoa1914347

5. Abramson JS, Palomba ML, Gordon LI, et al. Lisocabtagene maraleucel for patients with relapsed or refractory large B-cell lymphomas (TRANSCEND NHL 001): a multicentre seamless design study. Lancet. 2020;396(10254):839-852. doi:10.1016/ S0140-6736(20)31366-0

6. Bachier CR, Palomba ML, Abramson JA, et al. Outpatient treatment with lisocabtagene maraleucel (liso-cel) in 3 ongoing clinical studies in relapsed/refractory (R/R) large B cell non-Hodgkin lymphoma (NHL), including second-line transplant noneligible (TNE) patients: Transcend NHL 001, Outreach, and PILOT. Paper presented at: 2020 Transplantation & Cellular Therapy Meetings; February 19-23, 2020; Orlando, FL. Abstract 29. Accessed November 18, 2020. bit.ly/37I7DC9

7. Bristol Myers Squibb provides update on biologics license application (BLA) for lisocabtagene maraleucel (liso-cel). Press release. Bristol Myers Squibb. May 6, 2020. Accessed November 18, 2020.https://bit.ly/2YFWAs8

8. Bristol Myers Squibb provides regulatory update on lisocabtagene maraleucel (liso-cel). News release. Business Wire. November 16, 2020. Accessed November 18, 2020. https://bwnews.pr/3pKQMZI

9. Bristol Myers Squibb and bluebird bio announce submission of biologics license application (BLA) for anti-BCMA CAR T cell therapy idecabtagene vicleucel (ide-cel, bb2121) to FDA. Press release. Bristol Myers Squibb. March 31, 2020. Accessed November 18, 2020. https://bit.ly/2JwKbxO

10. Bristol Myers Squibb and bluebird bio provide regulatory update on idecabtagene vicleucel (ide-cel, bb2121) for the treatment of patients with multiple myeloma. News release. Business Wire. May 13, 2020.Accessed November 18, 2020. https:// bwnews.pr/3cpgJa1

11. US Food and Drug Administration (FDA) accepts for priority review Bristol Myers Squibb and bluebird bio application for anti-BCMA CAR T cell therapy idecabtagene vicleucel (ide-cel, bb2121). Press release. Bristol Myers Squibb. September 22, 2020. Accessed November 18, 2020. https://bit.ly/3kDhakH

Originally posted here:
CAR T-Cell Therapies Are Set to Expand Into More Hematologic Malignancy Indications - Targeted Oncology

Bone Marrow Stem Cells Comments Off on CAR T-Cell Therapies Are Set to Expand Into More Hematologic Malignancy Indications – Targeted Oncology | December 26th, 2020

Marco Kaltofen was 11 when he noticed his first white hairs. As his hair grew whiter, his middle-school friends started calling him the professor. By his mid-30s, it was completely white, as it had been for three of his grandparents. His parents went white in their 40s, so I had no chance of avoiding this, Kaltofen says.

Now 61, he is a civil engineer who lives in Boston. He wears his white hair in a ponytail. White hair is part of my identity, and I am completely at peace with it, he says.

Then there is Joe Rees, 75, a retired customs attache who lives in Washington. He is balding, but the hair that remains on the sides and in the back is the same dark brown it always has been. He jokingly attributes this to clean living and a pure heart, although, like Kaltofen, it probably is genetic. His mothers black hair didnt start to go gray until she was in her 70s, and was 50/50 when she died at 88, he says.

Still, Id rather be gray than bald, he says. That way, I wouldnt have to worry about wearing a hat all the time.

To be sure, Rees and Kaltofen are exceptions, since most people start graying in their 50s and 60s. Nevertheless, their experiences are among the many mysteries of gray, white or silver-looking hair that scientists are exploring to learn more about aging. They want to know why some people turn gray early and others late or not at all and what this might signal about their health. They also want to understand the factors that hasten graying, and even whether gray hair is reversible which could be a boon to those allergic to hair dye, or who hate spending money to keep the gray away.

Most important, studying gray hair could point to new approaches in promoting healthier aging, says Candace Kerr, health scientist administrator in the National Institute on Agings Division of Aging Biology.

While graying is one of the markers of aging aging is the ultimate risk factor for why hair goes gray it highlights the need for better understanding of the mechanisms that drive aging and age-related diseases, she says. To be able to target these pathways will be critically important for our aging population to live longer and happier lives.

Hair that looks gray, white or silver actually is colorless. Hair color comes from melanin, a pigment produced by cells in the hair follicles. Over time, these cells suffer damage and become depleted, losing their ability to make melanin. This results in new hair without pigment meaning, no color.

People use gray, white and silver interchangeably to describe hair that is turning or has turned. Its appearance whether it looks, gray, white or silver depends on how much natural color, or pigment, remains, experts say. Hair that has lost all its color typically appears white.

Studies have identified a number of factors that also may speed up gray hair, including smoking, diet, stress and genetics.

Our hair color depends on a set of specialized stem cells called melanocyte stem cells, and every time a new hair grows, these melanocyte stem cells have to divide in two and make a new melanocyte, [or] pigment cells, explains Melissa Harris, assistant professor of biology at the University of Alabama at Birmingham. These pigment cells stay in the base of your hair and their job is to produce pigment. These melanocytes reach out skinny arms, called dendritic processes, that shuttle the pigment to the hair shaft as it grows. So if all your melanocyte stem cells disappear, so do your melanocytes and so does your hair pigment. Thus gray hair.

Because stem cells directly influence hair color, studying gray hair can provide insights about why stem cells age and ultimately fail, offering important clues about the workings of other stem cells in the body for example, those found in muscles, bones and organs. In turn, these ultimately could point to whether gray hair could be a marker for disease, or the opposite, a longer life. Previous studies have not shown a relationship between life span and gray hair, including whether late onset of gray hair predicts longevity. Some research, however, indicates that gray or white hair can be a sign of early heart disease, regardless of age.

In some people, gray hair could potentially serve as indication of their health for instance when caused by stress, or a signal for those who may be developing cardiovascular disease, Kerr says. We still need to learn more about whether and, if so, how late onset of gray hair can signal better health and longevity in some people under certain circumstances, as well as whether early graying means stem cells might be aging.

There are many different stem cells in our body which may or may not age by different means, she says. How stem cells mark aging overall and how they could interact to promote aging is an important question.

This is why scientists who study gray hair regard it as a valuable research tool.

As gray hair researchers, we often have to defend why we study a cosmetic characteristic, rather than a life-threatening disease, Harris says. But what is very cool about gray hair from a scientific point of view is that we can see it with our own eyes, meaning we dont have to take invasive biopsies, and it doesnt kill you. We have asked a lot of important and interesting questions about stem cells by studying gray hair in mice. And, we are constantly on the lookout for gray-haired mice so we can use our scientific skills to find out what makes them gray.

A 2018 mouse study by Team Hair-Us (Harris nickname for her lab colleagues) found a connection between MITF (microphthalmia), a transcription factor (a protein involved in gene expression) important in managing pigment production, and the innate immune system, suggesting that some peoples hair may turn gray in response to serious illness or chronic stress. They discovered a relationship between genes involved in hair color and those that trigger an immune response to a viral infection, suggesting this interaction could increase the chances of developing gray hair.

MITF, in a sense, shields melanocyte stem cells from our own immune system, she says. Normally our immune system protects our bodies from infection. But for melanocyte stem cells, too much immune response is bad for their health, and this leads to their loss and to gray hair. Why melanocyte stem cells are so sensitive to our own natural means for protection, we still dont know.

Im very curious to see whether we see an uptick in individuals with gray hair due to coronavirus infection, she says. Unfortunately, we probably wont know because gray hair is rarely documented clinically, unless it is very extreme.

Scientists still dont know why some people turn gray early, late, or not at all, although they suspect genes, nutrients and possibly the immune system play a role in depleting melanocyte stem cells.

There is still much to learn about what regulates these stem cells and what may contribute to their loss, says Ya-Chieh Hsu, associate professor of stem cell and regenerative biology at Harvard University and principal faculty member of the Harvard Stem Cell Institute.

Among other things, Hsu studies the effect of stress on graying. Most of us are familiar with those before-and-after photographs of U.S. presidents most recently Barack Obama showing a striking increase in gray hair during their terms, even in relatively young presidents. Its known as the Marie Antoinette Syndrome, after the 18th-century French queen whose hair allegedly turned white overnight before she went to the guillotine and her death at age 38 during the French Revolution.

With the aging process, we gradually lose melanocyte stem cells one-by-one over a very long period of time, Hsu says. What we found in our research was that the stress can accelerate that process.

Hsu and her colleagues found that stress stimulates the same nerves that trigger the fight-or-flight response, which in turn causes permanent damage to the pigment-producing cells in hair follicles. The fight or flight response is thought to be a good thing in stressful situations because it can drive us and other organisms to respond to danger rapidly, Hsu says. This activation causes a spike in the neurotransmitter norepinephrine. Norepinephrine raises our heartbeat and allows us to react quickly to danger without having to think about it.

But norepinephrine also tells melanocyte stem cells to pump up their activity and proliferate, and too much norepinephrine, in this case triggered by stress, causes the melanocyte stem cells to burst into so much activity it leads to rapid depletion of the stem cell reservoir, she says. If all the stem cells are depleted, no more pigment-producing cells can be produced anymore, and the hair turns gray.

Other stress hormones, ACTH (adrenocorticotropic hormone) for example, can cause melanocyte stem cells to migrate away from the hair follicle before they can produce the melanocytes needed for hair and skin color, according to research. Such hormones are known to increase in the body after stress, and may have the potential to promote the loss of these cells, regardless of age, says study author Mayumi Ito, associate professor in the departments of cell biology and dermatology at the New York University Grossman School of Medicine.

Hsu believes the connection between stress and hair color could reveal additional information about how stress affects other biological processes. How stress affects our tissues is still poorly understood, and one of the powerful aspects about the melanocyte is that it provides a visible and highly trackable system to study stress, she says.

Ito also found that certain cell signaling proteins called endothelins (substances known to constrict blood vessels and raise blood pressure) bind to melanocyte stem cells and, in doing so, keep them healthy. Interrupting the process causes cell loss and early graying in mice. They are studying whether the same happens in human hair follicles, hoping to find ways to preserve or regenerate the key stem cells that give hair its color.

All of this raises the intriguing possibility that scientists could discover ways to prevent or reverse gray hair.

Team Hair-Us recently published a paper describing a topical drug combination that increased melanocyte stem cells in gray mice, ridding them of their gray and restoring their original fur color perhaps for good. Because the treatment originally developed to regrow hair replenished pigment-producing stem cells, the effects could be long-lasting, Harris says.

We didnt keep the mice forever so we dont know, says Harris, who plans more studies. This has made us very interested in whether gray hair really is permanent, and if we can do something about it. We really want to know and so does everyone else we talk to is whether and when we can bring this to humans.

Excerpt from:
Getting to the root of why hair goes gray - messenger-inquirer

Skin Stem Cells Comments Off on Getting to the root of why hair goes gray – messenger-inquirer | December 26th, 2020

Chafing occurs when the skin rubs against something, such as clothing, for a prolonged period. The friction can cause a painful rash that typically resolves when the chafing stops.

A person can experience chafing on any part of the body that encounters friction. The resulting rash is often minor, but there are some possible complications.

In this article, we look at what causes chafing rash and how to treat it. We also look at how a person can prevent chafing from occurring.

A chafing rash occurs anywhere on the body where the skin is rubbing against clothing or other skin.

Mild chafing rashes tend not to be too painful, but more serious rashes could bleed, crust, or blister. Chafing can occur anywhere where there is friction, but some areas of the body are more prone to chafing than others.

Areas where chafing is most likely to happen are generally moist or generate friction. They include the:

The direct cause of chafing is the skin is rubbing against something for a prolonged period. This could happen, for instance, when someone is walking and their inner thighs are rubbing together.

Chafing is more likely to occur in particular areas of the body, and it tends to happen during physical activity, such as running.

Others causes of chafing include:

Additionally, an infant or toddler wearing a diaper may experience chafing due to the added moisture and movement in the area.

Learn more about the types of diaper rash here.

Chafing causes a burning or stinging rash to occur where the skin is rubbing against something. The rash will not spread to other areas.

Possible complications of chafing rashes include:

Bacteria could enter through the broken or damaged skin around a chafing rash and cause an infection. For that reason, a person should make sure that they take care of the rash and keep it clean. The Centers for Disease Control and Prevention (CDC) list several symptoms of a skin infection, which include:

Chafing rashes will typically clear up within a few days if a person stops the activity causing the chafing or takes action to prevent further chafing.

Excessive moisture and skin friction may lead to skin breakdown. Patting the skin dry to remove excess moisture may prevent complications of chafing rash.

A person may also wish to consider applying shea butter lotion to the rash. A 2012 study found that shea butter has anti-inflammatory properties, which could make it a good option for people who want to soothe a chafing rash with cream.

For pain, a person can try applying aloe vera to the skin, as it may soothe sore skin and help prevent infections.

People have also traditionally used a range of other plant oils, including olive oil and coconut oil, to treat skin-related issues. Further research is necessary to confirm the benefits of each of these oils.

Unless the skin becomes infected, doctors do not have a standard way of treating chafing rash. A person should avoid the activity that caused the chafing. If they cannot avoid the activity, they should take steps to prevent further chafing from occurring.

If a person needs treatment, a doctor may prescribe or recommend a corticosteroid cream or ointment to help reduce the inflammation resulting from the rash.

It is not always possible to prevent chafing from occurring, but a person can take certain steps to reduce the risk.

According to the American Academy of Dermatology Association, these steps include:

Other steps a person can take to help prevent chafing include:

Additionally, the findings of an older study suggest that a person can apply petroleum jelly to the skin to minimize friction and prevent chafing rash.

If a person develops a rash that spreads, it is likely not a chafing rash. A person should consider seeing their doctor if the rash does not go away within a few days of home treatment.

A person should also talk to their doctor if they suspect that their rash has become infected. A doctor can examine the rash and determine whether additional treatment is necessary.

A chafing rash is often a mild rash that occurs when skin rubs against clothing or other areas of skin. Many cases are mild and will go away when a person stops the activity causing the chafing.

A possible complication of chafing rash is a skin infection. A person should speak with their doctor if the rash is severe or they think that it has become infected.

At-home treatments for chafing rash include aloe vera and other soothing ointments. A person can take preventive steps to reduce the risk of a rash forming, such as wearing moisture-wicking clothing and applying petroleum jelly to areas prone to chafing.

Read more:
What causes chafing rash? Remedies, treatment, and prevention - Medical News Today

Skin Stem Cells Comments Off on What causes chafing rash? Remedies, treatment, and prevention – Medical News Today | December 24th, 2020

Rashes can appear differently on different skin tones. For example, a heat rash on darker skin may look like a series of gray or white spots, while many medical sources describe them as red.

It is crucial for medical professionals to understand how health issues can present on the full range of skin tones. A lack of this understanding can lead to misdiagnoses.

It is generally advisable to contact a doctor about any rash that lasts longer than a week. The Skin of Color Society offer a database of dermatologists in the United States who have experience treating conditions in people of color.

In this article, we explore what rashes can look like on skin of color. We also describe the symptoms and treatments of specific issues.

The appearance of a rash varies with skin tone. On skin of color, a rash may be purple, gray, or white while medical texts often simply describe these rashes as red. Some redness may appear, but typically not very much.

This is due to melanin, a molecule that gives the skin and hair their color. Generally, the more melanin a person has in their skin, the darker their skin tone. It affects how the skin reacts to sunlight, damage, and health conditions that cause rashes.

Some doctors are unaware of how skin conditions present on darker skin. A 2018 study of four major medical textbooks found that darker skin tones were underrepresented in the imagery.

The studys authors report that while, at the time, 20.4% of the U.S. population was Black, only 4.5% of the photographs in the textbooks showed people with dark skin. They suggest that this is likely an example of racial bias in the healthcare system.

Below, we explore how various health issues can appear in skin of color.

Heat rash, sweat rash, and prickly heat are all common names for miliaria, a skin condition that occurs when the skins sweat ducts become blocked.

The symptoms of heat rash include:

On darker skin, the blisters may be gray or white.

This rash often occurs due to heat and humidity, but intense exercise, nonbreathable clothing, and medical dressings can also cause it. The condition typically improves within 12 days.

Cooling down, wearing more breathable clothing, and changing or, if recommended, removing dressings can help.

Eczema causes patches of dry, itchy skin, and the affected area may also be flaky or scaly. If the skin is very dry, it may crack or bleed.

In darker skin tones, patches of eczema may be red, pink, magenta, or darker than surrounding skin.

Eczema is also more likely to form in specific ways in people of color. For example, there is a higher chance that the rash may be papular, characterized by a series of small bumps like goosebumps.

There are several types of eczema, including:

This occurs when the skin comes into contact with an irritant, such as poison ivy, nickel, or fragrance. The skin may itch, sting, burn, or blister.

People with certain jobs have a higher risk of contact dermatitis. These jobs may involve regular contact with chemicals, food, or water. People with a higher risk may include:

Avoiding contact with the trigger, such as a specific chemical, is the best approach. A dermatologist can also describe products that can help and perform skin allergy testing to identify the trigger.

Atopic dermatitis is eczema with no clear cause. The condition often begins in childhood and may not last into adulthood. In the U.S., it appears to be more common among African Americans, Asian Americans, and Pacific Islanders than other groups.

There is no cure for atopic dermatitis, but there are ways of alleviating and preventing the rash from forming.

This typically involves using a non-irritating moisturizer regularly, after bathing or showering. It is also important to avoid fragranced products, food allergens, and irritating fabrics, such as wool. In addition, a person may need a topical corticosteroid, which a doctor can recommend or prescribe.

Learn more about eczema on black skin and its treatments.

Psoriasis is a long-term inflammatory condition that causes thickened patches of skin. On skin of color, these patches may be red or violet and have a top layer of silver or gray scales.

In the U.S., psoriasis is more common in white people than Black people. However, in Black people, it may be more likely to cover larger areas of the body.

Psoriasis can affect the nails as well as the skin, and a significant portion of people with psoriasis also have arthritis.

There is no cure, but it is possible to manage the symptoms. Among the range of options are:

Learn more about psoriasis on black skin and its treatments.

Lichen planus causes a series of bumps to form on the skin. Each bump is shiny, raised, and has a flat top. On skin of color, these bumps may be gray-brown or purple.

The most commonly affected areas are the back, neck, lower legs, ankles, and the insides of the wrists. In 20% of cases, lichen planus causes no symptoms beyond the bumps, but in others, it causes intense itching.

Lichen planus can last for months or years, and there is no known cure. Treatments that can relieve the symptoms include:

Learn more about the causes and treatments of lichen planus.

Vitiligo is a condition that causes patches of skin to lose its color, or become depigmented. If the condition affects areas with hair, the hair may turn white, too.

The condition occurs when the immune system attacks melanocytes, the cells that produce melanin. Scientists are not sure why this happens, but they believe that it could stem from an autoimmune response.

Areas commonly affected by vitiligo include:

Vitiligo can affect anyone, but it is more noticeable in people with darker skin. People with vitiligo are more likely to have a family history of autoimmune disorders.

The depigmentation often stops or slows over time. Treatments focus on reducing the spread of depigmentation and restoring color to the skin. This may include the use of medicated creams or light therapy.

Shingles is caused by the same virus as chickenpox. Anyone who has had chickenpox can develop shingles later on. One symptom that most people with shingles develop is a rash of small blisters.

The symptoms of shingles are:

Some people also experience a fever, headaches, muscle aches, stomach pain, or nausea and vomiting.

On highly pigmented skin, a shingles rash may be red, the same color as the skin, or slightly darker. The scabs may be grey.

While the rash often heals within 24 weeks, it is important for anyone with shingles symptoms to see a doctor for treatment within 23 days. This is because shingles can cause long-term complications.

The doctor can prescribe antiviral medication, which can reduce the risk of complications and shorten the healing time.

Ringworm is a fungal infection of the skin. It often causes a round or ring-shaped rash with a raised border, though the rash may be shaped differently on certain areas, such as the feet or hands.

On darker skin, the ringworm rash is often gray or brown.

Ringworm is contagious, so it is important to seek treatment right away and avoid touching the rash, even if it is itchy.

Other names for this infection include:

Treatment may involve antifungal cream or, if the rash covers a large area, oral medication.

The best approach to treatment at home depends on the cause of the rash. But there are some general strategies for preventing further irritation, such as:

It is also important to protect the skin from sun damage with a product that contains SPF 30 or higher. UV light can also worsen skin irritation for people with certain conditions.

In some cases, a rash signals a medical emergency. Seek urgent medical attention if a rash:

Anyone who may have shingles or ringworm should see a doctor promptly to prevent transmission. For shingles, a person should wait no more than 23 days.

Otherwise, it is generally a good idea to contact a doctor if any rash lasts longer than a week.

The Skin of Color Society provide a database of dermatologists in the U.S. who have experience treating conditions in people of color.

In skin of color, a rash may be red, brown, purple, or gray.

Some doctors may misdiagnose skin issues in people of color due to a lack of awareness. Skin of color is often underrepresented in medical texts.

A dermatologist who understands how rashes present in the full range of skin tones is best prepared to provide the right treatment.

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As for your lips, we dont grow hair there because our lips are made up of a different type of cell, as theyre considered to be an extension of the gastrointestinal (GI) tract, says Mariwalla.

If having hair is so crucial to the function of our bodies, why have we been removing it for hundreds of years? Well, we have no one to blame but ourselves. We have, through communication with one another, established a globalized practice of removing hair to make women especially look very smooth and have baby-like skin; and for men to retain their body hair, says Jablonski. We tend to think, Oh, these signals are very ancient. These practices are very ancient. They're not. This is a pretty recent obsession. Jablonski estimates the practice of body hair removal started about only 500 years ago.

While modern societal standards of what femininity and masculinity are still very much linked to hairiness or lack of hairiness, weve begun to see a shift in the acceptance and normalization of body hair, thanks in part to social media, which has even helped us celebrate body hair for the first time. (Remember #freeyourpits?)

It's really wonderful when people examine those social norms and say, hold on, who started this? This is a bunch of nonsense, says Jablonski. And they realize, Hey, I can be a beautiful person inside and out without following these practices. It is tremendously liberating.

That sense of celebration seems to be more prevalent than ever before, as were living through a pandemic, which has put physical interactions between people on pause. Because of this, many of the performances we put on for others, like body hair removal, have become one-woman shows, with one-woman audiences.

So then you realize, in your heart of hearts, this is a waste of time, says Jablonski. Why should I take this time to do this thing that is socially acceptable and allows me to cleave towards a social norm? I'm doing just fine. People feel a lot of freedom now.

Of course, if you do choose to remove your body hair and thats fine too! depending on the location of the hair, there are a number of methods that will get the job done, says Mariwalla.

Shaving

For starters, theres shaving, the act of removing hair with a razor. When you shave the hair on your body (typically on the legs, underarms, and face), youre removing hair from above the top layer of skin, says Mariwalla. She recommends shaving with a cream or gel to keep the skin hydrated. The process of shaving is almost like exfoliating that top layer of skin, she says. So, it's like a two for one.

Tweezing

Tweezing, on the other hand, pulls the hair directly from the follicle. When you think about a hair follicle, it's like the little house that your hair lives in, explains Mariwalla. Any time you pull a hair from a follicle, it's always going to grow back. But its going to take longer than when you only remove it from above the skin.

Waxing and Sugaring

Waxing and sugaring use the same mechanism as tweezing but with warm substances (i.e. wax and sugar) that sit on top of the skin and around the hair to coax it out of the follicle. Its a process that is trying to warm the skin, so that the hair follicle opens up a little bit and the wax solidifies around the hair, says Mariwalla. When you remove the wax you basically rip the hair out of the follicle as you're doing it.

Depilatory Cream

There are also depilatory creams. Instead of grabbing onto the hair and pulling it, you're putting on a chemical that's dissolving the hair at the root, explains Mariwalla. So then you basically then just wipe it away.

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Covid-19 sucked most of the oxygen out of science this year. But we still had brilliant wins.

The pandemic couldnt bring rockets or humans down: multiple missions blasted off to the red planet in the summer of Mars. Two astronauts launched to the International Space Stationand made it safely backin a game-changer for commercial space travel. NASA released dozens of findings on how space travel changes our bodies, paving the way to keep us healthy in orbitor one day, on Mars and beyond.

Back on Earth, scientists scoured mud ponds and fished out a teeny-tiny CRISPR enzyme that packs a massive punch for genome editing. AI and neuroscience became even more entwinedsometimes literally. Biological neurons got hooked up to two silicon-based artificial neurons, across multiple countries, into a fully-functional biohybrid neural network. Others tapped dopaminethe main messenger for the brains reward systemto unite electricity and chemical computing into a semi-living computer. While still largely a curiosity, these studies take brain-inspired computers to another level by seamlessly incorporating living neurons into AI hardware. Now imagine similar circuits inside the brainNeuralink sure is.

More abstractly, biological and artificial brains further fed into each other in our understandingand craftingof intelligence. This year, scientists found mini-computers in the input tree-like branches of neurons. Like entire neural networks, these cables were capable of performing complex logical calculations, suggesting our brain cells are far brainier than we previously thoughtsomething AI can learn from. On the flip side, a hotshot algorithm inspired by the brain called reinforcement learning pushed neuroscientists to re-examine how we respond to feedback as we learn. AI also helped build the most dynamic brain atlas to date, a living map that can continuously incorporate new data and capture individual differences.

As we leave 2020 behind, two main themes percolate in my mind, not just for what theyve accomplished, but as indicators of what lies ahead. These are the trends Ill be keeping my eyes on in the coming year.

Why we age is extremely complex. So are methods that try to prevent age-related diseases, or slow the aging process itself. This nth-dimensional complexity almost dictates that longevity research needs to self-segregate into lanes.

Take probing the biological mechanisms that drive aging. For example, our cells energy factory spews out bullet-like molecules that damage the cell. The genome becomes unstable. Cells turn zombie-like. Working stem cells vanish. Tissue regeneration suffers. Scientists often spend entire careers understanding one facet of a single hallmark of aging, or hunting for age-related genes. The lucky ones come up with ways to combat that one foefor example, senolytics, a family of drugs that wipe out zombie cells to protect against age-related diseases.

But aging hallmarks dont rear their heads in isolation. They work together. An increasing trend is to unveil the how of their interactions workcrosstalk, in science-speakwith hopes of multiple birds with one stone.

This year, longevity researchers crossed lanes.

One study, for example, took a stem cell playbook to rejuvenate eyesight in aged mice with vision loss. They focused on a prominent aging hallmark: epigenetics. Our DNA is dotted with thousands of chemical marks. As we age, these marks accumulate. Using gene therapy, the team introduced three superstar genes into the eyes of aged mice to revert those marks and reprogram cells to a younger state. Youve probably heard of those genes: theyre three of the four factors used to revert adult skin cells into a stem-cell-like state, or iPSCs (induced pluripotent stem cells). Resetting the epigenetic clock was so powerful it improved visual acuity in old mice, and the team has now licensed the tech to Life Biosciences in Boston to further develop for humans.

Another study combined three main puzzle pieces in agingzombie cells, inflammation, and malfunctioning mitochondriainto a full picture, with the surprise ending that senolytics has multiple anti-aging powers in cells. Talk about killing two birds with one stone. Finally, one team (which I was a part of) combined two promising approaches for brain rejuvenationexercise and young bloodto begin pushing the limits of reigniting faltering memory and cognition due to aging.

Longevity research has long been fragmented, but its starting to coalesce into a multidisciplinary field. These crossovers are just the start of a rising trajectory to combat the multi-headed Hydra thats aging. More will come.

If youre looking for a sign that AI is leaving the digital realm of Atari games and heading into the real world, this year was it.

In biotech, theres no doubt of AIs promise in drug discovery or medical diagnoses. In late 2019, a team used deep learning and generative modelssimilar to AlphaGo, the DeepMind algorithm that trounced humans at Go and wiped the Atari libraryto conjure over 30,000 new drug molecules, a feat chemists could only dream of. This year, the viral hurricane thats Covid-19 further unleashed AI-based drug discovery, such as screening existing drugs for candidates that may work against the virus, or newlydesigned chemicals to fight off SARS-CoV-2 infectionthe virus that causes Covid-19.

For now, we dont yet have an AI-designed drug on the market, an ultimate test for the technologys promise. However, although AI wasnt able to make a splash in our current pandemic battle, the scene is set for tackling the next oneand drug discovery as a whole.

In contrast, AI-based medical diagnosis had a resounding win. This year, the FDA approved a software that uses AI to provide real-time guidance for ultrasound imaging for the heart, essentially allowing those without specialized training to perform the test. The approval brings a total of 29 FDA-approved AI-based medical technologies to date. Even as the debate on trust, ethics, and responsibility for AI doctors cranked up in temperature, the Pandoras box has been opened.

Medicine aside, deep learning further honed its craft in a variety of fields. The neuroscience-AI marriage is one for the ages with no signs of fracture. Outside the brain, AI also gave synthetic biology a leg up by parsing the interactions between genes and genetic networksa mind-bending, enormously complex problem previously only achieved through trial and error. With help from AI, synthetic biologists can predict how changes to one gene in a cell could affect others, and in turn, the cells biochemistry and behavior. Bottom line: it makes designing new biological circuits, such as getting yeast to pump out green fuels or artificially hoppy beer, much easier.

But the coup de grce against AI as an overhyped technology is DeepMinds decimation of a 50-year-long challenge in biology. With a performance that shocked experts, DeepMinds AlphaFold was able to predict a proteins 3D structure from its amino acid sequencethe individual components of a proteinmatching the current gold standard. As the workhorses of our bodies, proteins dictate life. AlphaFold, in a sense, solved a huge chunk of the biology of life, with implications for both drug discovery and synthetic biology.

One more scientific brilliance this year is the use of light in neuroscience and tissue engineering. One study, for example, used lasers to directly print a human ear-like structure under the skin of mice, without a single surgical cut. Another used light to incept smell in mice, artificially programming an entirely new, never-seen-in-nature perception of a scent directly into their brains. Yet another study combined lasers with virtual reality to dissect how our brains process space and navigation, mentally transporting a mouse to a virtual location linked to a reward. To cap it off, scientists found a new way to use light to control the brain through the skull without surgerythough as of now, youll still need gene therapy. Given the implications of unauthorized mind control, thats probably less of a bug and more of a feature.

Were nearing the frustratingly slow, but sure, dying gasp of Covid-19. The pandemic defined 2020, but science kept hustling along. I cant wait to share what might come in the next year with youmay it be revolutionary, potentially terrifying, utterly bizarre* or oddly heart-warming.

* For example, Why wild giant pandas frequently roll in horse manure. Yes thats the actual title of a study. Yes, its a great read. And yes, its hilarious but has a point.

Image Credit: Greyson Joralemon on Unsplash

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I am seeing tons of hair loss, Mona Gohara says.

Patients come to Gohara, a dermatologist and professor at the Yale School of Medicine, for all kinds of reasons from skin cancer screenings to cosmetic procedures. But this year more than ever, theyre worried about their hair.

Its not a coincidence. Stress like, say, that brought on by living through a deadly pandemic is known to cause hair loss. Ordinarily, 90 percent of the hairs on our head are in the growing cycle; 10 percent are in the shedding cycle, Gohara explained. But when were subject to some type of physiologic or emotional stress, that cycle shifts to where the shed outweighs the grow. The result: people notice a massive, massive shed.

And those stray hairs are part of a bigger trend. At this point, millions of Americans have spent nine months living through a public health nightmare and an unprecedented economic crisis at the same time. They have also had to cope with all this while avoiding gatherings, limiting physical contact, and, when possible, staying inside their homes. Put together, the isolation and anxiety of life in 2020 have brought with them numerous side effects. For one, they might be doing weird things to our bodies.

If youve noticed your menstrual cycle is more irregular this year, for example, youre not alone: More patients are reporting irregular periods since the pandemic began, Mary Jane Minkin, an OB-GYN who teaches at the Yale School of Medicine, told Vox. The likely culprit, as with hair loss, is the anxiety of living in such a difficult and uncertain time. When stressors come into play, Minkin said, we end up with screwy periods.

If youve spotted more gray hairs on Zoom calls, there may be a pandemic-related explanation for that too. And according to some, life in lockdown may even be changing peoples body odor.

Those are just some of the smaller effects. Some experts are also concerned because isolation has documented effects on health, increasing the risk of cardiovascular disease and even death. Humans are considered a social species, Julianne Holt-Lunstad, a professor of psychology and neuroscience at Brigham Young University who studies the impact of social relationships on health, told Vox. When we lack proximity to others, and particularly trusted others, this creates a heightened state of alert or stress which, over time, can have harmful effects on our bodies.

To be clear, none of this is an argument for getting rid of pandemic-related restrictions after all, the effects of Covid-19 on the body can be far more severe than the effects of isolation.

But the rise of pandemic periods, weird smells, and other bodily indignities are a reminder that Americans are going through something right now that most of us have never experienced before. And that takes its toll in a lot of ways some of them stranger than others.

Lets talk about periods first. Trend pieces about menstrual changes either irregularity or worsened symptoms like cramps began popping up in the spring. A couple of weeks into the stay-at-home order in Washington State, where I live, I woke up in the middle of the night with the worst cramps Ive ever had, Colleen Stinchcombe wrote at Self in May.

And while its likely too soon for any published research on the impact of the pandemic on menstruation, Minkin isnt the only one to see increased reports of irregularity among her patients. Its common for us to see patients with changes in their menstrual cycle, but anecdotally, it seems like its been happening more over the last six months, Beth Schwartz, an OB-GYN at Thomas Jefferson University Hospital in Philadelphia, told the Washington Post in August.

These changes arent necessarily surprising, Minkin told Vox. Most people think that the ovaries and the uterus regulate periods, she said. But actually, the boss is sitting in our brain.

Specifically, its the hypothalamus and the pituitary gland that control the ovaries, regulating their hormone production, which in turn regulates the menstrual cycle. Its our nice, regular hormonal activity from the hypothalamus and the pituitary which stimulate the ovaries to do their thing appropriately and get us nice, regular periods, Minkin said.

And when were under stress, that can disrupt the functioning of the hypothalamus and pituitary, leading to irregularity. Researchers have noticed a similar effect among young people who go away to college and often experience irregular periods as they adjust to a new environment and a new set of worries and pressures.

The good news, Minkin said, is having an irregular period generally isnt dangerous. When her patients report irregularity, shell typically test their thyroid function and levels of certain hormones to rule out conditions like polycystic ovary syndrome, but as long as everythings normal, no treatment is needed. If the irregularity is especially bothersome, people can take hormonal contraception to regulate their periods, Minkin said. Once we get through things and peoples lives get back toward normal, most folks are probably going to regulate themselves just fine.

But irregular periods arent the only strange symptom people are reporting after months of reduced contact with others. Another is body odor some say theyve started to smell worse, or just different, since the pandemic began.

I am a man who prides himself on smelling fresh and fancy free at all times, Joseph Lamour wrote at Mic in July. But during the pandemic summer, he became so limburger-esque that my own odor woke me up in the middle of the night.

As with periods, theres not yet published research on changes in body odor during the pandemic. But anecdotal reports of the issue have gotten back to Julie Horvath, head of the Genomics and Microbiology Research Lab at the North Carolina Museum of Natural Sciences and a professor at North Carolina Central University. An expert in primate genetics who expanded into studying microbes and odor (I never thought I would be the armpit researcher, she says), Horvath explains that a big factor in the way we smell is our skin microbiome, or the mix of bacteria, fungi, and viruses that live on our skin.

When youre in your home, youre now coming into contact more with maybe your pets and your family, who you didnt see as many hours a day, she told Vox. This means were exchanging microbes with a different group of people (and animals) than usual, which could affect our smell. Spending more time indoors can also affect the microbiome, as can wearing different types of clothes synthetic fabrics can host different kinds of microbes than cotton, for example. And a lot of people have changed their style (if you can call it that anymore) during the pandemic. When I talk to people, maybe theyre wearing a nice shirt, but now they have jeans or sweatpants on, Horvath said.

Stress can also affect the microbiome, Horvath said. A specific set of glands, the apocrine glands, release sweat when youre nervous. That sweat contains different compounds from sweat that comes from the ecrine glands, which get to work when youre too hot. And if your apocrine glands found in the armpit and a few other places on the body are highly active, then they are providing a different food source to some of those microbes there, and maybe youre promoting the growth of some that smell different than what they wouldve before, Horvath said.

But the microbiome doesnt just affect the way we smell. Beneficial bacteria on our skin create a protective barrier, Horvath explained. If you have these good, beneficial microorganisms that are on your skin, eating oil or sweat and living there happily, theyre taking up residence, she said. Then, if something lands on your skin that can make you sick a staph bacterium, for example then it cant take hold very quickly, because your beneficial organisms are going to outcompete it for resources.

Washing your hands with soap and water just washes away the lop layer of microbes, potentially allowing some of the good ones to stick around. But hand sanitizer kills the microorganisms on your skin, the good along with the bad, Horvath said. Thus, using too much hand sanitizer during the pandemic could leave us more vulnerable to staph, acne, or other infections down the road.

Airborne spread of the coronavirus in close contact is the main danger in the pandemic, but we still need to wash our hands, too. Horvath recommends using soap and water when possible to help maintain a healthy microbiome. Beyond that, habits like eating a healthy diet could be good for your microbial balance, though they may or may not help you smell better. Spending time outside if you can is also a good idea, Horvath said. Certain organisms that are outside in the soil are actually beneficial for your overall body.

Beyond weird smells and irregular periods, the isolation of this year has brought with it other physical changes for many. In addition to hair loss, a proliferation of gray hair is a common complaint one that can also likely be pinned on stress, as Deanna Pai reports at Medium. While the mechanism by which stress causes graying isnt fully understood, one recent study in mice found that stress led to the death of stem cells that produce melanocytes, the cells in hair follicles that produce pigment.

Gray hair isnt reversible (except with dye), Pai points out, but managing stress as much as anyone can during a pandemic can help slow the process.

Stress could also be making our skin look worse, Gohara, the dermatologist, said. It causes an increase in the hormone cortisol, which wreaks cosmetic havoc on your skin and can lead to anything from dryness to puffy eyes, she explained. Everything just looks worse with a surge in cortisol.

An increase in stress can also lead to more acne, something also exacerbated by the friction of wearing a mask (hence the 2020 neologism maskne, or breakouts on the lower part of the face linked to mask-wearing). Luckily, unlike gray hair, much of this is reversible you can combat maskne by washing masks in the same gentle cleanser you use for your face, Gohara said, and using a product with salicylic acid or benzoyl peroxide. For hair loss, meanwhile, she sometimes prescribes supplements, but also reassures patients that when it comes to shedding, eventually the cycle is going to re-equilibrate itself and your hair will be back on track.

While things like hair loss are typically harmless, if annoying, the way we live in 2020 could be causing more serious issues too.

Researchers have long known that isolation the condition of having little or no contact with other people and loneliness the subjective feeling of being alone, regardless of how much contact with people one has can be harmful, Holt-Lunstad, the psychologist, said. For example, in one 2015 analysis, she and her coauthors found that isolation was associated with a 29 percent increased likelihood of mortality, while loneliness was associated with a 26 percent increase.

There are a couple of ways that loneliness can potentially hurt our health. For one, friends and loved ones can influence us to take better care of ourselves having someone who encourages you to get to bed, or eat fruits and vegetables, or quit smoking, is good for our health, Holt-Lunstad said.

But many studies actually control for lifestyle factors like smoking and diet, and still find that loneliness and isolation have a negative effect. One reason, some researchers believe, is that our brains have adapted to expect proximity to others, and particularly trusted others, Holt-Lunstad said. When they arent around, the brain signals other parts of the body to go into a heightened state of alert. That can lead to changes in heart rate and blood pressure that could increase our risk of cardiovascular disease. But it could also lead to systemic inflammation in the body, which in turn has been linked to a host of mental and physical problems, Holt-Lunstad said, from Alzheimers disease to, troublingly, increased susceptibility to viruses.

These impacts are especially concerning because some early research has found high rates of loneliness and isolation during the pandemic. In an August survey, for example, two-thirds of adults reported social isolation, and more than 7 in 10 said the pandemic had made it harder to connect with friends.

Luckily, there are ways to reduce isolation, even during a time of social distancing. In a study this summer sponsored by the neighborhood-focused social network Nextdoor, Holt-Lunstad and her colleagues found that performing small acts of kindness for neighbors, such as bringing them groceries or checking in on them over the phone, was associated with a significant drop in loneliness 1 in 10 participants felt lonely at the beginning of the study, while just 1 in 20 felt the same at the end.

But it cant all be on individuals to fix their isolation during this very lonely time. Instead, Holt-Lunstad has advocated for policymakers to pay more attention to peoples social needs throughout the pandemic and recovery, including increased funding to help students and older people, who may be especially vulnerable to loneliness right now. And while funding for anything remains a fraught subject in Congress, Holt-Lunstad writes at Health Affairs that decisions should be based on scientific evidence of benefits and drawbacks to our well-being, not solely on economic costs and convenience.

Some of the smaller effects of pandemic living may dissipate naturally when this time in our lives is over. When it comes to issues like irregular periods, for example, the biggest takeaway is dont panic, Minkin says. We will get back to normal.

But for other, larger problems, like isolation and its serious effects on the body, the pandemic could be a wake-up call. My hope is now that we have all experienced, in some degree or another, this feeling of isolation and loneliness, that there may be greater awareness and less stigma, Holt-Lunstad said.

Give the gift of understanding

In April, Vox launched a way for readers to support our work with financial contributions and we've been blown away by the response. This year, support from our founding contributors has helped us create projects that millions relied on to understand a year of chaos, and to keep their families safe. Support from our readers helps us rely less on advertising, and keep our resource-intensive work free for everyone who needs it. We want to add 2,020 more founding contributors to our supporter base by the end of the year. Help us reach our goal by making a contribution to Vox today, from as little as $3.

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New York, Dec. 21, 2020 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Organ and Tissue Transplantation and Alternatives" - https://www.reportlinker.com/p096592/?utm_source=GNW g., kidneys, liver, heart-lung, pancreas, intestines) and the tissue transplantation (e.g., bone, skin, cornea, heart valve) markets, along with the pharmaceuticals that accompany each market.

Also included are experimental xenografts and artificial organs; tissue transplants; and cell transplants (e.g., bone marrow, cord blood, peripheral blood, islet cell). The report touches on the use of fetal cells, stem cells and altered cancer cells.

The arrangement of this report offers an overview of the key elements in the transplantation process: tissue typing, procurement and preservation, immunosuppressants for solid organ and tissue transplants, and postoperative monitoring. International markets are discussed, and information is provided on industry structure and the regulatory environment.

Within each section are discussions of commercialization opportunities for each segment of the market. New or emerging devices, techniques and pharmaceuticals are highlighted.

Profiles of leading companies involved with solid organ transplantation, tissue transplantation, and alternative technologies are included. The report provides information on company placement within the market and strategic analyses of the companies available and emerging products.

An appendix featuring various terms and processes used in transplantation is provided at the end of the report.

This report cites autologous products only in relation to their impact on the market for allografts. It does not include blood products, except for peripheral and umbilical cord blood as a source of stem cells.

By geography, the market has been segmented into the North America, Europe, Asia-Pacific, and Rest of the World regions. Detailed analysis of the market in major countries such as the U.S., Germany, the U.K., Italy, France, Spain, Japan, China, India, Brazil, Mexico, GCC countries and South Africa will be covered in the regional segment. For market estimates, data will be provided for 2019 as the base year, with estimates for 2020 and forecast value for 2024.

Report Includes:- 26 data tables and 37 additional tables- An overview of the global organ and tissue transplantation and alternatives market- Estimation of the market size and analyses of market trends, with data from 2018 to 2019, estimates for 2020 and projection of CAGR through 2024- Details about organ and tissue transplantation and alternatives, their pathophysiology and affects, and major advancement and latest trends- A look at the regulatory scenarios and initiatives by government organization- Analysis of current and future market dynamics and identification of key drivers, restraints and opportunities such as increasing incidence of organ donations, improved awareness about organ donations, side effects of organ and tissue transplantation and antibiotic resistance infections- Coverage of emerging procedures and products in development and discussion on prevalence of major chronic diseases which initiates organ damage or donation- Discussion on the role of the organ procurement organization and information on transplantation process and preparation and coverage of issues like black market donors- Impact analysis of COVID-19 on organ and tissue transplantation and alternatives market- Market share analysis of the key companies of the industry and coverage of events like mergers & acquisitions, joint ventures, collaborations or partnerships, and other key market strategies- Company profiles of major players of the industry, including Abiomed Inc., Bayer AG, F. Hoffmann-La Roche & Co., Johnson & Johnson, Novartis AG, Pfizer Inc. and XVIVO Perfusion

Summary:The global organ and tissue transplantation and alternatives market was valued at REDACTED in 2019.The market is expected to grow at a compound annual growth rate (CAGR) of REDACTED to reach approximately REDACTED by 2024.

Growth of the global market is attributed to factors such as the growing prevalence of obesity, diabetes, cancer, and other chronic diseases which leads to organ damage, a strong product regulatory scenario, and strong investment in research and development activities by key market players including Abbott Laboratories, Cryolife Inc., Bristol-Myers Squibb, Novartis Ag, F. Hoffmann-La Roche Ltd., Medtronic, Arthrex Inc., Depuy Synthes (Johnson & Johnson), and Allosource.

Although various factors facilitate the global market for organ and tissue transplantation and alternatives, certain parameters such as challenges in HLA sequencing and gaps in supply and demand can constrain market growth.For instance, although there is an increasing need for organ transplants, the shortage of organs worldwide limits the number of transplant procedures performed, and in turn creates an impact on transplant diagnostics procedures.

An increasing number of candidates on the waiting list for organ transplant procedures worldwide further widens this gap of availability and requirement of organs for transplant purposes.

Successful organ and tissue transplantation began to arrive in the mid-1970s when tissue typing coupled with the use of cyclosporine provided more successful graft and patient survival. Today, patient and graft survival for kidney transplants is higher than 90% for the first year post-transplant, and often the success rate is 80% to 90% for five years post-transplant, with some recipients living more than 20 years after their transplant.

Continuing developments in organ procurement, organ preservation, tissue typing, and immunosuppressant use have bolstered successful transplantation surgical techniques. Evolving posttransplant drug and testing regimens have added to the success rate with close post-transplant monitoring and immunosuppressant dosage review.Read the full report: https://www.reportlinker.com/p096592/?utm_source=GNW

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FDAs decision will enable CytoDyn to respond to ongoing requests for leronlimab until Phase 3 trial data is unblinded

VANCOUVER, Washington, Dec. 22, 2020 (GLOBE NEWSWIRE) -- CytoDyn Inc. (OTC.QB: CYDY), (CytoDyn or the Company"), a late-stage biotechnology company developing Vyrologix (leronlimab-PRO 140), a CCR5 antagonist with the potential for multiple therapeutic indications, announced today a treating physician has received authorization from the U.S. Food and Drug Administration (FDA) to administer leronlimab for a COVID-19 patient under emergency IND (eIND).

Nader Pourhassan, Ph.D., President and Chief Executive Officer of CytoDyn, commented, We are very thankful the FDA is allowing severe-to-critical COVID-19 patients access to Vyrologix (leronlimab) again under eIND while we await the unblinding of data from our recently completed Phase 3 registrational trial. We are receiving daily requests from families seeking our drug for a loved one with COVID-19. In recent months, leronlimab received more than 60 eIND authorizations from the FDA, and during the pendency of our COVID-19 trials, we deferred seeking authorizations for eINDs in order to accelerate the pace of enrollment. Now that enrollment has been completed, we are pleased to be able to assist once again and remain hopeful the upcoming results of our Phase 3 trial will enable leronlimab to be more readily available for severe-to-critical COVID-19 patients.

CytoDyns Phase 2b/3 trial to evaluate the efficacy and safety ofleronlimabfor patients with severe-to-critical COVID-19 indications is a two-arm, randomized, double blind, placebo controlled, adaptive design multicenter study. Patients are randomized to receive weekly doses of 700 mg leronlimab, or placebo. Leronlimab and placebo are administered via subcutaneous injection. The study has three phases: Screening Period, Treatment Period, and Follow-Up Period. The primary outcome measured in this study is: all-cause mortality at Day 28. Secondary outcomes measured are: (1) all-cause mortality at Day 14, (2) change in clinical status of subject at Day 14, (3) change in clinical status of subject at Day 28, and (4) change from baseline in Sequential Organ Failure Assessment (SOFA) score at Day 14.

About Coronavirus Disease 2019 CytoDyn completed its Phase 2 clinical trial (CD10) for COVID-19, a double-blinded, randomized clinical trial for mild-to-moderate patients in the U.S. which produced statistically significant results for NEWS2. CytoDyn completed enrollment of 390 patients in its Phase 2b/3 randomized clinical trial for the severe-to-critically ill COVID-19 population and expects to release results in mid-January 2021.

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

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

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

The CCR5 receptor appears to play a central role in modulating immune cell trafficking to sites of inflammation. It may be crucial in the development of acute graft-versus-host disease (GvHD) and other inflammatory conditions. Clinical studies by others further support the concept that blocking CCR5 using a chemical inhibitor can reduce the clinical impact of acute GvHD without significantly affecting the engraftment of transplanted bone marrow stem cells.CytoDyn is currently conducting a Phase 2 clinical study with leronlimab to support further the concept that the CCR5 receptor on engrafted cells is critical for the development of acute GvHD, blocking the CCR5 receptor from recognizing specific immune signaling molecules is a viable approach to mitigating acute GvHD. The FDA has granted orphan drug designation to leronlimab for the prevention of GvHD. Due to the lack of patients during the COVID-19 pandemic, the Company is closing down its Phase 2 trial for acute GvHD.

About CytoDyn CytoDyn is a late-stage biotechnology company developing innovative treatments for multiple therapeutic indications based on leronlimab, a novel humanized monoclonal antibody targeting the CCR5 receptor. CCR5 appears to play a critical role in the ability of HIV to enter and infect healthy T-cells. The CCR5 receptor also appears to be implicated in tumor metastasis and immune-mediated illnesses, such as GvHD and NASH.

CytoDyn has successfully completed a Phase 3 pivotal trial with leronlimab in combination with standard antiretroviral therapies in HIV-infected treatment-experienced patients. The FDA met telephonically with Company key personnel and its clinical research organization and provided written responses to the Companys questions concerning its recent Biologics License Application (BLA) for this HIV combination therapy in order to expedite the resubmission of its BLA filing for this indication.

CytoDyn has completed a Phase 3 investigative trial with leronlimab as a once-weekly monotherapy for HIV-infected patients. CytoDyn plans to initiate a registration-directed study of leronlimab monotherapy indication. If successful, it could support a label extension. Clinical results to date from multiple trials have shown that leronlimab can significantly reduce viral burden in people infected with HIV. No drug-related serious site injection reactions reported in about 800 patients treated with leronlimab and no drug-related SAEs reported in patients treated with 700 mg dose of leronlimab. Moreover, a Phase 2b clinical trial demonstrated that leronlimab monotherapy can prevent viral escape in HIV-infected patients; some patients on leronlimab monotherapy have remained virally suppressed for more than six years.

CytoDyn is also conducting a Phase 1b/2 clinical trial with leronlimab in metastatic triple-negative breast cancer. More information is at http://www.cytodyn.com.

Forward-Looking StatementsThis press release contains certain forward-looking statements that involve risks, uncertainties and assumptions that are difficult to predict. Words and expressions reflecting optimism, satisfaction or disappointment with current prospects, as well as words such as "believes," "hopes," "intends," "estimates," "expects," "projects," "plans," "anticipates" and variations thereof, or the use of future tense, identify forward-looking statements, but their absence does not mean that a statement is not forward-looking. Forward-looking statements specifically include statements about leronlimab, its ability to have positive health outcomes, the possible results of clinical trials, studies or other programs or ability to continue those programs, the ability to obtain regulatory approval for commercial sales, and the market for actual commercial sales. The Company's forward-looking statements are not guarantees of performance, and actual results could vary materially from those contained in or expressed by such statements due to risks and uncertainties including: (i) the sufficiency of the Company's cash position, (ii) the Company's ability to raise additional capital to fund its operations, (iii) the Company's ability to meet its debt obligations, if any, (iv) the Company's ability to enter into partnership or licensing arrangements with third parties, (v) the Company's ability to identify patients to enroll in its clinical trials in a timely fashion, (vi) the Company's ability to achieve approval of a marketable product, (vii) the design, implementation and conduct of the Company's clinical trials, (viii) the results of the Company's clinical trials, including the possibility of unfavorable clinical trial results, (ix) the market for, and marketability of, any product that is approved, (x) the existence or development of vaccines, drugs, or other treatments that are viewed by medical professionals or patients as superior to the Company's products, (xi) regulatory initiatives, compliance with governmental regulations and the regulatory approval process, (xii) general economic and business conditions, (xiii) changes in foreign, political, and social conditions, and (xiv) various other matters, many of which are beyond the Company's control. The Company urges investors to consider specifically the various risk factors identified in its most recent Form 10-K, and any risk factors or cautionary statements included in any subsequent Form 10-Q or Form 8-K, filed with the Securities and Exchange Commission. Except as required by law, the Company does not undertake any responsibility to update any forward-looking statements to take into account events or circumstances that occur after the date of this press release.

CONTACTSInvestors: Michael MulhollandOffice: 360.980.8524, ext. 102mmulholland@cytodyn.com

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Most kids with peanut allergies do not outgrow them. But, with a little help, some might be able to better tolerate accidental exposures.

In January, the Food and Drug Administration approved Palforzia, a new drug designed to help kids who are allergic to peanuts react better, if they are accidentally exposed.

"Because there is no cure, allergic individuals must strictly avoid exposure to prevent severe and potentially life-threatening reactions," Dr. Peter Marks, director of the FDA's Center for Biologics Evaluation and Research said at the time in a news release. "When used in conjunction with peanut avoidance, Palforzia provides an FDA-approved treatment option to help reduce the risk of these allergic reactions."

Palforzia is not designed to be administered during an allergic reaction, instead it works as an allergy exposure therapy: children ages 4 through 17 receive daily doses of peanut powder under clinical supervision, and slowly up-dose it over time.

In clinical trials, the strategy worked well, but not perfectly. When peanut-allergic kids were fed 600 milligrams of peanut protein, 67.2% of Palforzia recipients who'd been using the medication for six months tolerated it, while only 4% of the control group did.

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The 11 most mind-blowing, awe-inspiring health discoveries and innovations of 2020 - Business Insider - Business Insider

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First gene therapy to receivefull EU marketing authorization for eligible MLD patients

One-time treatment with Libmeldy has been shown to preserve motor and cognitive function

Achievement shared with research alliance partners Fondazione Telethon and Ospedale San Raffaele

BOSTON and LONDON and MILAN, Italy, Dec. 21, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, and its research alliance partners Fondazione Telethon and Ospedale San Raffaele, today announced that the European Commission (EC) granted full (standard) market authorization for Libmeldy (autologous CD34+ cells encoding the ARSA gene), a lentiviral vector-based gene therapy approved for the treatment of metachromatic leukodystrophy (MLD), characterized by biallelic mutations in theARSAgene leading to a reduction of the ARSA enzymatic activity in children with i) late infantile or early juvenile forms, without clinical manifestations of the disease, or ii) the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline. Libmeldy is the first therapy approved for eligible patients with early-onset MLD.

MLD is a very rare, fatal genetic disorder caused by mutations in the ARSA gene which lead to neurological damage and developmental regression. In its most severe and common forms, young children rapidly lose the ability to walk, talk and interact with the world around them, and most pass away before adolescence. Libmeldy is designed as a one-time therapy that aims to correct the underlying genetic cause of MLD, offering eligible young patients the potential for long-term positive effects on cognitive development and maintenance of motor function at ages at which untreated patients show severe motor and cognitive impairments.

Todays EC approval of Libmeldy opens up tremendous new possibilities for eligible MLD children faced with this devastating disease where previously no approved treatment options existed, said Bobby Gaspar, M.D., Ph.D., chief executive officer of Orchard. Libmeldy is Orchards first product approval as a company, and I am extremely proud of the entire team who helped achieve this milestone. We are grateful for and humbled by the opportunity to bring this remarkable innovation to young eligible patients in the EU.

With Libmeldy, a patients own hematopoietic stem cells (HSCs) are selected, and functional copies of the ARSA gene are inserted into the genome of the HSCs using a self-inactivating (SIN) lentiviral vector before these genetically modified cells are infused back into the patient. The ability of the gene-corrected HSCs to migrate across the blood-brain barrier into the brain, engraft, and express the functional enzyme has the potential to persistently correct the underlying disease with a single treatment.

The EC approval of Libmeldy comes more than a decade after the first patient was treated in clinical trials performed at our Institute, and ushers in a remarkable and long-awaited shift in the treatment landscape for eligible MLD patients, said Luigi Naldini, M.D, Ph.D., director of the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy. Our team at SR-Tiget has been instrumental in advancing the discovery and early-stage research of this potentially transformative therapy to clinical trials in support of its registration through more than 15 years of studies supported by Fondazione Telethon and Ospedale San Raffaele, and we are extremely proud of this achievement and what it means for patients and the field of HSC gene therapy.

MLD is a heart-breaking disease that causes immeasurable suffering and robs children of the chance of life, said Georgina Morton, chairperson of ArchAngel MLD Trust. As a community, we have been desperate for a treatment for young MLD patients, and we are incredibly excited to now have such a ground-breaking option approved in the EU.

The marketing authorization for Libmeldy is valid in all 27 member states of the EU as well as the UK, Iceland, Liechtenstein and Norway. Orchard is currently undertaking EU launch preparations related to commercial drug manufacturing, treatment site qualification and market access.

Data Supporting the Clinical and Safety Profile of Libmeldy

The marketing authorization for Libmeldy is supported by clinical studies in both pre- and early- symptomatic, early-onset MLD patients performed at the SR-Tiget. Early-onset MLD encompasses the disease variants often referred to as late infantile (LI) and early juvenile (EJ). Clinical efficacy was based on the integrated data analysis from 29 patients with early-onset MLD who were treated with Libmeldy prepared as a fresh (non-cryopreserved) formulation. Results of this analysis indicate that a single-dose intravenous administration of Libmeldy is effective in modifying the disease course of early-onset MLD in most patients.

Clinical safety was evaluated in 35 patients with MLD (the 29 patients from the integrated efficacy analysis as well as six additional patients treated with the cryopreserved formulation of Libmeldy). Safety data indicate that Libmeldy was generally well-tolerated. The most common adverse reaction attributed to treatment with Libmeldy was the occurrence of anti-ARSA antibodies (AAA) reported in five out of 35 patients. Antibody titers in all five patients were generally low and no negative effects were observed in post-treatment ARSA activity in the peripheral blood or bone marrow cellular subpopulations, nor in the ARSA activity within the cerebrospinal fluid. In addition to the risks associated with the gene therapy, treatment with Libmeldy is preceded by other medical interventions, namely bone marrow harvest or peripheral blood mobilization and apheresis, followed by myeloablative conditioning, which carry their own risks. During the clinical studies, the safety profiles of these interventions were consistent with their known safety and tolerability.

For further details, please see the Summary of Product Characteristics (SmPC).

About MLD and Libmeldy

MLD is a rare and life-threatening inherited disease of the bodys metabolic system occurring in approximately one in every 100,000 live births. MLD is caused by a mutation in the arylsulfatase-A (ARSA) gene that results in the accumulation of sulfatides in the brain and other areas of the body, including the liver, gallbladder, kidneys, and/or spleen. Over time, the nervous system is damaged, leading to neurological problems such as motor, behavioral and cognitive regression, severe spasticity and seizures. Patients with MLD gradually lose the ability to move, talk, swallow, eat and see. In its late infantile form, mortality at five years from onset is estimated at 50% and 44% at 10 years for juvenile patients.1

Libmeldy (autologous CD34+ cell enriched population that contains hematopoietic stem and progenitor cells (HSPC) transduced ex vivo using a lentiviral vector encoding the human arylsulfatase-A (ARSA) gene), also known as OTL-200, is approved in the European Union for the treatment of MLD in eligible early-onset patients. In the U.S., OTL-200 is an investigational therapy which has not been approved by the U.S. Food and Drug Administration (FDA) for any use. Libmeldy was acquired from GSK in April 2018 and originated from a pioneering collaboration between GSK and the Hospital San Raffaele and Fondazione Telethon, acting through their joint San Raffaele-Telethon Institute for Gene Therapy in Milan, initiated in 2010.

About Orchard

Orchard Therapeutics is a global gene therapy leader dedicated to transforming the lives of people affected by rare diseases through the development of innovative, potentially curative gene therapies. Our ex vivo autologous gene therapy approach harnesses the power of genetically modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. In 2018, Orchard acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Orchard now has one of the deepest and most advanced gene therapy product candidate pipelines in the industry spanning multiple therapeutic areas where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist.

Orchard has its global headquarters inLondonandU.S.headquarters inBoston. For more information, please visitwww.orchard-tx.com, and follow us on Twitter and LinkedIn.

Availability of Other Information About Orchard

Investors and others should note that Orchard communicates with its investors and the public using the company website (www.orchard-tx.com), the investor relations website (ir.orchard-tx.com), and on social media (Twitter andLinkedIn), including but not limited to investor presentations and investor fact sheets,U.S. Securities and Exchange Commissionfilings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchards investor relations website and may include additional social media channels. The contents of Orchards website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.

About Fondazione Telethon, Ospedale San Raffaele and the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget)

Based in Milan, Italy, the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) is a joint venture between the Ospedale San Raffaele, a clinical-research-university hospital established in 1971 to provide international-level specialized care for the most complex and difficult health conditions, and Fondazione Telethon, an Italian biomedical charity born in 1990 and focused on rare genetic diseases. SR-Tiget was established in 1995 to perform research on gene transfer and cell transplantation and translate its results into clinical applications of gene and cell therapies for different genetic diseases. Over the years, the Institute hasgiven a pioneering contribution to the field with relevant discoveries in vector design, gene transfer strategies, stem cell biology, identity and mechanism of action of innate immune cells. SR-Tiget has also established the resources and framework for translating these advances into novel experimental therapies and has implemented several successful gene therapy clinical trials for inherited immunodeficiencies, blood and storage disorders, which have already treated >115 patients and have led through collaboration with industrial partners to the filing and approval of novel advanced gene therapy medicines.

For more information:

Forward-Looking Statements

This press release contains certain forward-looking statements about Orchards strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements may be identified by words such as anticipates, believes, expects, plans, intends, projects, and future or similar expressions that are intended to identify forward-looking statements. Forward-looking statements include express or implied statements relating to, among other things, Orchards business strategy and goals, including its plans and expectations for the commercialization of Libmeldy, and the therapeutic potential of Libmeldy, including the potential implications of clinical data for eligible patients. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, these risks and uncertainties include, without limitation:: the risk that prior results, such as signals of safety, activity or durability of effect, observed from clinical trials of Libmeldy will not continue or be repeated in our ongoing or planned clinical trials of Libmeldy, will be insufficient to support regulatory submissions or marketing approval in the US or to maintain marketing approval in the EU, or that long-term adverse safety findings may be discovered; the inability or risk of delays in Orchards ability to commercialize Libmeldy, including the risk that we may not secure adequate pricing or reimbursement to support continued development or commercialization of Libmeldy; the risk that the market opportunity for Libmeldy, or any of Orchards product candidates, may be lower than estimated; and the severity of the impact of the COVID-19 pandemic on Orchards business, including on clinical development, its supply chain and commercial programs. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchards quarterly report on Form 10-Q for the quarter endedSeptember 30, 2020, as filed with theU.S. Securities and Exchange Commission(SEC), as well as subsequent filings and reports filed with theSEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

Contacts

InvestorsRenee LeckDirector, Investor Relations+1 862-242-0764Renee.Leck@orchard-tx.com

MediaChristine HarrisonVice President, Corporate Affairs+1 202-415-0137media@orchard-tx.com

1 Mahmood et al. Metachromatic Leukodystrophy: A Case of Triplets with the Late Infantile Variant and a Systematic Review of the Literature.Journal of Child Neurology2010, DOI:http://doi.org/10.1177/0883073809341669

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Orchard Therapeutics Receives EC Approval for Libmeldy for the Treatment of Early-Onset Metachromatic Leukodystrophy (MLD) - GlobeNewswire

Bone Marrow Stem Cells Comments Off on Orchard Therapeutics Receives EC Approval for Libmeldy for the Treatment of Early-Onset Metachromatic Leukodystrophy (MLD) – GlobeNewswire | December 22nd, 2020