Category Archives: Stem Cell Treatments

Cancer cells pose an uncanny ability to make new cells and dodge drugs, "somewhat like would-be robbers hacking the bank's alarm code," one doctor explains. But researchers at Cincinnati Children's Hospital Medical Center have figured out how cancer cells rewire themselves and, in turn, how to possibly overcome drug resistance.

This drug resistance may explain why some acute myeloid leukemia (AML) and other cancer patients suffer fatal relapses despite many improvements in leukemia outcomes over the years, according to a Children's news release.

"Overcoming resistance to therapy remains a holy grail of leukemia treatment," says Yi Zheng, Ph.D., director, experimental hematology and cancer biology at Cincinnati Children's. Zheng and his colleagues have now discovered a way to boost the effectiveness ofmTOR inhibitors, which prohibit unwanted cell proliferation.

"While the latest study is based on mouse models, building upon the findingspublished Dec. 21, 2020, in PNASeventually could improve outcomes for people with AML, and possibly other forms of cancer," a release says.

What Happens When Treatments Target mTOR?

"Using a novel mouse model, we have learned that deleting the mTOR gene prompts blood stem cells to multiply rapidly to open other pathways to continue producing new blood cells," says Zheng, the study's senior author. "We also found that leukemia cells use a similar response to continue multiplying despite mTOR-inhibiting treatments."

He says attacking mTOR essentially sets off alarms among hemopoietic stem cells (HSCs), which act like blood cell factories deep in bone marrow. Then the cells themselves produce a flood of new, re-wired blood cells. These re-wired stem cells, treated with mTOR inhibitors, can begin multiplying, rendering mTOR inhibitor drugs useless.

The co-authors say mTOR treatment resistance can be counteracted by inhibiting activity of the MNK, CDK9 or c-Myc genes. So-called BET inhibitors can act against c-Myc activity. Other inhibitors that are in clinical trials can act against CDK9.

Next Steps

Scientists at Cincinnati Childrens have already launched some of the research needed to prepare the combination therapies for in vivo test leading to human clinical trials, the news release says. That process will take time, but since mTOR inhibitors have been widely tested in clinical trials, investigators have a head start on exploring combination therapies.

Longer term, the findings may extend beyond AML, Zheng says, because mTOR has been a recognized target in most human cancers, including solid tumors like brain tumors.

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New Combination Therapy Tested By Children's May Offer Hope For Leukemia Patients - WVXU

NEW YORK, Dec. 17, 2020 /PRNewswire/ -- Physio Logic, a leading provider of integrated health services in New York City and surrounding areas, continues to demonstrate its commitment to excellence in the field of Regenerative Medicine and Stem Cell Therapy by entering into a collaboration with Regenexx, a worldwide network of specially trained physicians providing the world's most advanced, research-driven, regenerative medicine and stem cell therapy treatments. The partnership brings cutting-edge regenerative treatments to New York City residents suffering from sports injuries or degenerative diseases.

The Regenerative Medicine division of Physio Logic is led by Dr. Tanuj Palvia, MD, a specialist in regenerative medicine and interventional orthopedics focused on the treatment of musculoskeletal injuries and degenerative orthopedic conditions.

"Stem Cell Therapy is one of the most innovative treatments available today but, being so new, patients need to know they're receiving the best possible care. As a physician, I hold myself and my practice to the highest standards and, being aligned with Regenexx adds that extra assurance patients need to know they're in good hands. Whether it's a nagging sports injury or slow degeneration, you're going to get the highest quality of integrated care right here at Physio Logic," said Dr. Palvia.

Interventional Orthobiologics is a specialty that focuses on using your body's natural healing agents to treat orthopedic injuries with the goal of reducing pain and improving joint function. The variety of orthobiologics available to Regenexx physicians, such as bone marrow stem cells and platelet-rich plasma (PRP), allow them to create a treatment plan to best support your recovery. It can be used in the treatment of conditions such as arthritis and injury to ligaments, tendons, cartilage, or bone.

"Being selected to represent the Regenexx brand in New York City speaks to the quality of our facility, our providers, and the care we give our patients," said Dr. Rudy Gehrman, CEO & Founder of the Brooklyn based clinic. "Physio Logic is raising the standard of healthcare in New York and our partnership with Regenexx is an extension of the quality, integrative care we provide to every patient that walks through our door."

Regenexx physicians are required to have thousands of hours of experience performing precise, injection-based treatments using image guidance for a range of body parts and injuries. Their strict acceptance criteria means that Regenexx only chooses the most qualified physicians to join their network. Physio Logic's Interventional Pain Specialist, Dr. Tanuj Palvia, MD, is ranked among them.

To learn more about Physio Logic and Regenerative Medicine, go to https://physiologicnyc.com/regenerative-medicine/

About Physio LogicPhysio Logic brings together an expert team of open-minded medical doctors, physical therapists, chiropractors, acupuncturists, massage therapists, nutritionists, health coaches, biohackers, and Pilates instructors. Our unique collaborative approach, coupled with our ability to assess patients holistically, is used to create a custom care plan tailored to patients' needs. For more information on Physio Logic, visit https://physiologicnyc.com or call (718) 260-1000.

About RegenexxRegenexx is a nationwide network of physicians who practice Interventional Orthopedics, a new specialty that focuses on using the most advanced regenerative protocols available as an alternative to many orthopedic surgeries. Regenexx has published roughly half of the research worldwide on the use of orthobiologics for treating orthopedic injuries, and our patented treatment lab-processing and treatment protocols allow us to achieve unmatched results. Our procedures use your body's natural healing agents including blood platelets and bone marrow concentrate to repair damaged bone, muscle, cartilage, tendons and ligaments. For more information on Regenexx, visit https://regenexx.com.

Media contact:Alan Sott[emailprotected](718) 260-1000

SOURCE Physio Logic

http://www.physiologicnyc.com

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Physio Logic Brings Cutting-edge Regenerative Treatments for Sport Injuries and Arthritis to New York City - PRNewswire

Although hypomethylating agents (HMAs) are a cornerstone of treatment for the majority of myelodysplastic syndrome (MDS) cases, there are patients who are resistant to such treatment, representing a need for other treatment options, reports the study in Therapeutic Advances in Hematology.

Both azacitidine (AZA) and decitabine (DEC) are HMAs approved in the United States for all patients with MDS, and most patients who respond to the treatments will do so within the first 6 cycles. With this threshold, its recommended to complete 6 cycles of AZA before considering a patient as HMA refractory.

Since the prognosis of patients failing HMA therapy is dismal except for the small minority of patients eligible for allo-SCT [allogeneic stem cell transplant], there is an urgent need for both prevention of HMA failure by (1) optimization of frontline therapies (eg, adding synergistic agents to HMA therapy) and/or (2) improved salvage therapies for HMA-refractory MDS patients, explained the researchers.

For these patients who are resistant to HMA treatment, there are no formal recommendations, but several options, spanning from novel HMAs to chemotherapy, have been or are currently being studied in the setting.

Outside of AZA and DEC, which have short half-lives potentially limiting their biologic activity, guadecitabine has emerged as a more effective, easier-to-administer option that has also demonstrated tolerability.

Oral ASTX727, a combination of cedazuridine and DEC, has preliminary data from 50 patients with MDS or chronic myelomonocytic leukemia. The data show an overall response rate (ORR) of 62%, and the most common grade >3 adverse events were hematologic (eg, neutropenia, 48%; thrombocytopenia, 38%; anemia, 22%; leukopenia, 20%), febrile neutropenia (38%), and pneumonia (20%). There is currently an ongoing phase 3 trial comparing the oral option with intravenous DEC.

An oral option of AZA, CC-486, is also being tested in clinical trials.

The review, while highlighting promising treatment options for these patients, also offers rationale for not using certain treatment options. For example, although lenalidomide has proven effective in the first-line setting for patients with del(5q), the treatment has a limited benefit in these patients who are refractory to HMAs, leading researchers to recommend against lenalidomide being used outside of the frontline setting for the subgroup of patients.

Chemotherapy, frequently used for medically fit patients as a bridge to allo-SCT, was tested in an international multicenter retrospective analysis of over 300 patients who were refractory to HMA. The study, which used 3 intensive induction chemotherapy regimens, boasted an ORR of 41% and median overall survival of 10.8 months. Forty percent of patients were bridged to allo-SCT, the only potentially curative options for patients with MDS.

Other research has indicated that medically fit should not exclusively qualify patients for chemotherapy treatment, with some data suggesting that the decision should also take into account molecular and cytogenetic features.

Ongoing research is also being targeted at combining HMAs with intensive chemotherapy, other forms of epigenetic therapy, venetoclax, and immune checkpoint inhibitors.

Reference

Bewersdorf J, Carraway H, Prebet T. Emerging treatment options for patients with high-risk myelodysplastic syndrome. Ther Adv Hematol. Published online November 11, 2020. doi:10.1177/2040620720955006

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For Patients With HMA-Resistant MDS, What Are Their Options? - AJMC.com Managed Markets Network

Offering more hope to patients with COVID-19, cancer experts throughout Baptist Health South Florida are launching new clinical trials. The trials, developed as a result of promising initial emergency and experimental COVID-19 therapies at Miami Cancer Institute on the Baptist Hospital campus in Miami and at the Eugene M. & Christine E. Lynn Cancer Institute at Boca Raton Regional Hospital, span the range of care from the most critically ill to those with mild symptoms.

Guenther Koehne, M.D., Ph.D., director and chief of Stem Cell Transplantation, Hematologic Oncology and Benign Hematology at Miami Cancer Institute.

Its exciting to lead these next-generation clinical trials, said Guenther Koehne, M.D., Ph.D., principal investigator of two of the studies, and director and chief of Stem Cell Transplantation, Hematologic Oncology and Benign Hematology at Miami Cancer Institute. We have learned much about COVID-19 since the pandemic began last winter and our hope is that these trials will lead to tremendous treatment options for patients.

Specialists at both centers are accomplished in many of the techniques and technologies that are doing double-duty as oncology and COVID-19 treatments and pivoted at the start of the pandemic to lead numerous COVID-19 clinical trials.

Trial to Save the Most Critical Patients

Miami Cancer Institute is leading a phase I/IIa clinical trial using mesenchymal stem cells for critically ill patients with COVID-19 induced respiratory failure. Mesenchymal stem cells are derived from umbilical cord lining tissue and aid in healing by regenerating damaged lung tissue. The trial is for hospitalized patients who are receiving oxygen therapy or who are on ventilation support and are not showing improvement.

Early in the pandemic, Miami Cancer Institute treated several patients with mesenchymal stem cells through single-use emergency approval from the U.S. Food and Drug Administration (FDA). The patients, who were among the most ill COVID-19 patients, recovered.

In our early experience with these umbilical cord lining stem cells, we had very promising results, Dr. Koehne said. We are very hopeful that the clinical trial will give us evidence that this treatment can save the lives of those who experience respiratory failure due to COVID-19.

Low-dose Radiation may Reverse Pneumonia

Both Lynn Cancer Institute and Miami Cancer Institute are participating in the PREVENT trial, Low-Dose Radiotherapy for Patients with SARS-COV2 (COVID-19) Pneumonia. With a single, low-dose of thoracic radiation, researchers hope that inflammation in the lungs is reduced and that patients with pneumonia associated with COVID-19 may not need to be placed on a ventilator.

Minesh Mehta, M.D., deputy director and chief of Radiation Oncology at Miami Cancer Institute.

We have seen this treatment option benefit many cancer patients, and the hope is that it also helps those affected by the virus, said Minesh Mehta, M.D., co-principal investigator, deputy director and chief of Radiation Oncology at Miami Cancer Institute.

Patients eligible for the study include hospitalized men and women ages 50 and up who are diagnosed with COVID-19 and pulmonary pneumonia but who are not on ventilators.

This trial gives us the opportunity to administer potentially effective treatment before the need for ICU placement or mechanical ventilation, said Michael Kasper, M.D., co-principal investigator and director of Radiation Oncology at Lynn Cancer Institute.

Radiation therapy has shown a reduction in inflammation in a number of conditions, including viral pneumonia, autoimmune disorders and degenerative joint disorders. At much higher doses, it is also used to treat cancer.

Trial to Shorten Recovery and Reduce Symptoms For Those With Mild Disease

Michael Kasper, M.D., director of Radiation Oncology at Lynn Cancer Institute.

Miami Cancer Institute is also enrolling patients in a phase 2 clinical trial known as BLAZE-4, which continues previous work using a monoclonal antibody, bamlanivimab, to treat patients with milder cases of COVID-19. The Institute participated in the phase 1 study, BLAZE-1, which resulted in Emergency Use Authorization (EUA) status by the FDA.

Bamlanivimab is for patients who are COVID-19 positive but are not hospitalized and have mild symptoms. It must be administered within 72 hours of a positive test result.

The BLAZE-1 study showed a lower subsequent hospitalization rate among those who received the drug versus those who received a placebo, and may reduce the viral load, leading to better outcomes. The BLAZE-4 trial will evaluate the efficacy and safety of bamlanivimab both on its own and in combination with another monoclonal antibody. The drugs work by prohibiting the spread of the virus to additional cells in the body.

We have reached a new level of sophistication trying to treat the virus before it makes you really sick, Dr. Koehne said. Despite the prospect of having COVID-19 vaccines, which are intended to prevent us from getting sick, we need to stay focused on the treatment of those individuals who are symptomatic from the virus.

Tags: COVID-19, Lynn Cancer Institute, Miami Cancer Institute

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In COVID-19 Clinical Trials, Experts from Baptist Health's Cancer Institutes Treat Patients With Mild or Severe Symptoms - Baptist Health South...

In the run-up to Christmas (between 13th and 24th December) we bring you a different charity that needs your help at this time of year, as part of our dedicated 12 Charities of Christmascampaign

Anthony Nolan saves the lives of people with blood cancer and blood disorders.

For someone with blood cancer, a stem cell transplant could be their last chance of survival. Your support could help it give that person, their family and their friends a second chance of life.

Anthony Nolan is the charity that makes lifesaving connections between people with blood cancer and incredible strangers ready to donate their stem cells. It is saving lives right now. Three lives a day, in fact.

By growing the stem cell register, carrying out ground-breaking research and providing the best post-transplant care, it is giving families a future. But it cant do it without you. Without you, there is no cure.

Anthony Nolans vision: To save and improve the lives of everyone who needs a stem cell transplant.

This is a fundamental vision and, if youve known the charity a while, youll notice it hasnt changed. Thats because its cause is as relevant now as it was 45 years ago when Shirley Nolan first started the charity to find a matching stem cell donor for her son, Anthony.

Its aims:

1. Together, we can provide outstanding services to patients and the clinical community

2. Together, we can drive lifesaving research and turn it into action for patients

3. Together, we can harness the collective power of our supporters to champion and enable our lifesaving work

4. Together, we can be a stronger voice for patients to ensure they receive the best possible care

5. Together, we can

Anthony Nolans 2019-2022 Organisational Strategy

In April 2019, it launched our new strategy, Together, we can save lives. This strategy sets out what it wants to achieve in these three years and beyond, as it leads the way into a future of new treatments and new ways of working. For more information or to donate, click here.

This feature is brought to you as part of our 12 Charities of Christmas campaign and has been sponsored by:

William Wallace has been self-employed for 54 years, starting with Braefoot Garage in Methil, with his junior John Stewart, who he is still in contact with today.

Aberhill Metals was created in 1986 and has grown, with his sons Derek and William Jnr joining him, from leaving school. He has always had the loyal and supportive help of his wife Margo, of 58 years, who has endured the pressures of running a business, and his extended family of 5, grandchildren of 16 and great grandchildren of 5

The company is well known throughout North East Fife for Skip Hire.

During the last year with the Covid lockdown, it kept working supplying skips. Even after a major fire at its premises, Aberhill didnt stop. Still up and running with the support of all staff.

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SPONSORED: 12 Charities of Christmas - Anthony Nolan - The Courier

14 December 2020

The recent close-call US presidentialelection grabbed headlines, but no less closely-fought was California's Proposition 14, also on the ballot in October, which will have a huge impact on the future of stem cell research in the state.

The Stem Cell Research Institute Bond Initiative (Prop 14),whichwas on theballotinthe initiated state statutewill allow the state to issue billions of dollars in bonds for its stem cellresearch programme. The vote could not have been closer, with 51 percent of ballots for and 49 percent against.

This initiative will enable financiers to lend US $5.5 billion to a stem cell agency, the California Institute for Regenerative Medicine (CIRM) whichthe state's taxpayers will repay with interest over the next three decades. The sizeable bond fund will be allocated to research, human clinical trials and programmes and also for start-up costs for facilities in the stem cell field. About US $1.5 billion of the money will be spent researching neurodegenerative conditions, including Alzheimer's, Parkinson's and dementia. Some of the funds will be allocated to the shared labs programme:state-funded facilities dedicated to conducting studies on human embryonic stem cells. A working groupfocused on improving access to medical treatments will be established, as well astraining and fellowship programmes for undergraduate and postgraduate students.

Stem cell research is in its infancy, with some treatments that have worked in animals now progressing to humantrials. The research is promising, as the ability to generate specialised cells could eventually be used torepair or replace damaged tissues and organs. However, there are ethical controversies surrounding stem cell research, especially regarding embryonic stem cells derived from early human embryos.

In 2001, the conservative Bush administration prohibited federal funding for stem cell research owing to the ethical concerns. This promptedreal estate developer and investor Robert Kleinto initiate the original Proposition 71 which aimed to establish a state constitutional right to conduct stem cell research in California in 2004.It was believed that Prop 71 would propel California as a world leader in stem cell research and 59 percent of Californians voted in favour.

The proposition also established CIRM the only state-funded stem cell research agency in the USA in the California Constitution. Since its inception, it has granted research funds to various institutions and companies including Stanford University and the University of California. So far, it has funded 68 clinical trials (phases 1-3) for a wide range of diseases and conditions, includingcancer, diabetes and many rare disorders.

In 2019, CIRMstarted to run out of funds, and by2020 hadsuspended applications for new projects except US $5 million in emergency funding for COVID-19 research. The Californians for Stem Cell Research, Treatments and Cures political action committee (PAC) responded with a campaign to boost funding via Prop 14. SupportersincludedUS House speaker Nancy Pelosi, California governorGavinNewsom, LA mayor EricGarcetti, the California Democratic Party and several organisations such as ALS Association and the Michael J Fox Foundation for Parkinson's research and Huntington's Disease Society of America.

There is also resistance to Prop 14:dissenters feel thattheinitiative has notdelivered its promises after 16 years. Furthermore the original rationale no longer applies asfederal funding is no longer blocked. Former President Barack Obama then removed the restrictions on embryonic stem cell research (a position unchanged by the Trump administration) which means California scientists can apply for (highly competitive) federal research grants.

In the current climate, Prop 14 will exacerbate California's already colossal budget deficits. The coronavirus pandemic has worsened housing crisesand unemployment rates, highly visible in cities like San Francisco and Los Angeles.

As Marcy Darnovsky, executive director of the Center for Genetics and Society, said:'It does nothing to address CIRM's built-in conflicts of interest, or its lack of legislative oversight despite it being an agency supported wholly by public funds. The new proposition makes some things worse; for example, it outsources critically important decisions about ethical standards to an unaccountable national committee... In the meantime, that campaign's shameless over-promising and hype set the stage for the hundreds of under-regulated commercial stem cell clinics now offering unapproved 'treatments' that have caused tumours and blindness...'

It is not surprising that Californians were so torn. Perhaps it's wiser to allocate the pot to more pressing matters like job creation, housing and other urgent needs?

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California's Proposition 14: short in the arm for stem cell research - BioNews

As the second wave of the pandemic engulfs us and the world works at warp speed to develop vaccines and therapies to respond, the importance of regenerative medicine has never been higher. Since 2017, Goldman Sachs has touted the sector as one of the most compelling areas for venture capital investment. With billions of dollars of global government spending being poured into the search for vaccines and therapies to respond to the novel coronavirus, and with the FDA having now granted approval to the first vaccines based on CRISPR mRNA gene-editing technologies, business models based on regenerative medicines are commanding record values. Despite the flood of cash into regenerative medicine, legal and ethical considerations will continue to cause much controversy.

Regenerative medicine ultimately accelerates the human bodys healing process. It is an area of biomedical sciences that involves medical treatments to repair or replace damaged cells, tissues, or organs. Instead of merely focusing on the symptoms, regenerative medicine uses cellular therapies, tissue engineering, medical devices, and artificial organs to improve peoples health. For example, stem cell therapies, tissue grafts, and organ transplants are all part of regenerative medicine.

Today, cellular and acellular regenerative medicines are often used in clinical procedures such as cell, immunomodulation, and tissue engineering therapies. They have the potential to effectively treat many chronic diseases, including Alzheimers, Parkinsons and cardiovascular disorders, osteoporosis, and spinal cord injuries.

A small number of unscrupulous actors, according to the FDA, however, have seized on the clinical promise of regenerative medicine to offer patients unproven treatments. The FDA and other regulators are challenged to provide assurances of safety for these therapies without stifling development, as well as to approve treatments based on manipulation of stem cells derived from human and animal embryos given the ethical issues involved.

In the future, stem cell research will play an increasingly outsized role in regenerative medicine techniques. In November 2020, voters in California narrowly passed Proposition 14, a referendum to approve $5.5 billion in new government funding for stem cell research. Other governments around the world are doing the same.

Today, the growing prevalence of chronic medical ailments and genetic disorders across the globe is a primary factor driving the regenerative medicine industrys growth, according to the Regenerative Medicine Market: Global Industry Trends, Share, Size, Growth, Opportunity and Forecast 2020-2025. The increasing aging population, prone to various musculoskeletal, oncological, dermatological, and cardiological disorders, is a key growth driver. Widespread adoption of organ transplantation is another contributing factor to this growth in market share. The current pandemic that began in January 2020, however, has changed the paradigm for regenerative medicine.

Market applications are burgeoning. Regenerative medicine can prevent and cure disease through effective vaccines and efficacious therapies. It can minimize the risk of organ rejection post-transplant and speed recovery. Technological advancements in cell-based therapies, such as the development of 3-D bioprinting techniques and the adoption of artificial intelligence in the production of regenerative medicines, are also stimulating growth. These advancements also facilitate dermatological grafting procedures to treat burns, bone defects, and skin wounds. Other factors, including extensive research and development activities in medical sciences and improving healthcare infrastructure, are also predicted to drive the market even further.

According to the Alliance for Regenerative Medicine, there are approaching approximately 1,000 companies focusing on this evolving area worldwide. These new companies are focusing on gene therapy, cell therapy and tissue engineering therapeutic developers. More than half of these companies are in North America, followed by almost a quarter in Europe and Israel and approximately 20% in Asia. More than 50% of these companies are focusing on cell therapy and gene therapy.

From 2014 to 2019, the global regenerative medicine market experienced a nearly 16% CAGR. Companies involved in gene and cell therapies as well as other regenerative medicine areas raised $4.8 billion during the first half of 2019, including $2.6 billion in the second quarter. Meanwhile, companies in Europe and Israel saw an acceleration of fundraising, with $1.3 billion amassed in just the first half of 2019, representing a 17% increase over the same period in 2018. Project Warp Speed has attracted billions of dollars of U.S. government spending, and similar efforts are ongoing in China, Russia, the European Union and among other major powers. Consequently, regenerative medicine has never before benefited from such a combination of public and private investment.

Whenever the viability and quality of human life are at stake, ethical and legal considerations always arise.

The modern ethical controversy surrounding regenerative medicine began in 1998 when research scientists at the University of Wisconsin succeeded in deriving and growing stem cells from early-stage human embryos. Ethicists and right-to-life activists protested that scientists were taking away human life (embryos) to conduct scientific experiments. Left unchecked, so the argument went, doctors could usurp nature and play God by developing the power to create and terminate life. A society where human life could be fundamentally perverted by medicine conjured up comparisons to Nazi Germany and Frankenstein. In 2001, then-U.S. President George W. Bush cut off federal funding for any research involving newly created embryonic stem cell lines, but agreed to continue funding research on 60 existing stem cell lines, where the life and death decision ha[d] already been made. The State of California responded in 2004 and again in 2020 with voter-approved programs directing billions of funding into stem cell research, making the region the global hub of regenerative medicine.

The use of human-derived embryonic stem cells, or animal-derived stem cells, continues to cause much controversy among ethicists and society at large. Some fear the risks of enrolling humans in experimental stem cell studies. Others fear the use of organs from human-animal chimeras in transplantation.

While these techniques have the potential to cure disease and save lives, they also have the potential to forever alter the nature of life as we know it and fundamental aspects of our society.

In the United States, legal jurisdiction for regulating regenerative medicine on a federal level lies with the FDA and in a patchwork of state laws, R&D funding programs and non-binding, NGO-promulgated statements of policy. The main responsibility of the FDA is to protect the public from dangerous products and ensure its safety, including overseeing medications for humans and animals, vaccines, and more.

During the Trump Administration, the FDA has largely focused on enabling developers to gain product approvals through a less burdensome and costly process. In numerous policy statements, the FDA under President Trump has deferred questions about the efficacy of new regenerative health products to the free markets, so long as they posed no serious safety or toxicity concerns.

The U.S. federal government is now transitioning to an administration led by President-elect Biden. The president-elect has spent many years advocating for increased R&D funding and going for moonshots. With a new mandate from the U.S. electorate to address the coronavirus, more money will be earmarked for regenerative medicines and stem cell research. How this will affect the release of new products into the market remains to be seen.

Regenerative medicine is poised to change the way we live, work and interact like never before. The fourth industrial revolution is upon us. CRISPR gene-editing technologies, facilitated by quantum-computing capabilities at the edge of a computer network powered by 5G telecommunications bandwidths, artificial intelligence and machine learning, have changed the game for regenerative medicine. We can foresee a day when those suffering from paralysis regain movement, when a damaged heart reverses course through regeneration, and when a diagnosis of Alzheimers Disease no longer means neurodegeneration. What a wonderful day that will be.

Changing the traditional healthcare model and moving from cure to prevention will take time.

The rise in chronic disease and the effort to reduce healthcare costs presents a large opportunity for the field of regenerative medicine.

As the continent becomes a bigger player, western companies should explore the potential prospects.

Topics from regenerative medicine to artificial intelligence to cannabis will be discussed.

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Regenerative Medicine: Market Trends and Legal Developments on the Horizon for 2021 - MedTech Intelligence

Joceline Colvert was diagnosed with relapsing remitting Multiple Sclerosis in her early 20s and says she spent the first eight years researching and managing her condition while trying to mention it as little as possible to others and completing her Sound Production degree.

I spent most of my late 20s and early 30s finding ways to manage relapses, the symptoms of which have included whole body numbness, loss of the use of both hands, right eye blindness, vertigo and double vision, she said. Thankfully these symptoms did resolve however left scarring on my nerves. This results in reduced vision in my formerly blind eye and hands that dont function very well with repetitive tasks.

This semi-denial worked for me until about 2010 when I started to become a bit limpy which I did my best to hide. After a couple of memorable falls and fractures I decided to face up to being slightly rickety and got a hiking pole that I used occasionally in public. Since then Ive needed to get used to being visibly disabled, and switch between two hiking poles for very short distances and a wheelchair everywhere else.

Joceline, who lives with her husband and her five beloved cats and dogs, says she is not eligible for Haematopoietic Stem Cell Transplantation (HSCT), on the NHS which is the first treatment I have ever got excited about and believe could work. It could be truly life-changing.

As a result she is trying to raise money to fund the treatment herself.

HSCT is a procedure that aims to reset the faulty immune system which, in my case, is attacking my nervous system from within, Joceline said. Stem cells will be taken from my bone marrow or blood before my immune system is wiped out with chemotherapy. My cells are then reintroduced into my blood, where they grow a new immune system which will hopefully no longer attack my nerves or have any memory of MS.

The aim of HSCT is to completely halt progression, putting MS into remission with no requirement for immunosuppressant drug therapy. The success rate for relapsing remitting MS is 80% - 90% which is absolutely phenomenal compared to the limited available drug treatments, which only aim to slow down disability.

HSCT is available on the NHS, however there is a very strict criteria for which I do not qualify. The expense of the treatment and the increased pressures on the public purse mean the NHS will only treat patients who have been diagnosed for fewer than 15 years.

I have been diagnosed for 18 years.

I had prepared myself for this possibility and, for the last year, have been researching treatment with The National Pirogov Medical Centre Russia (Moscow). Russia has been pioneering in their use of HSCT to treat MS and are world renowned for their expertise and care. Im excited to have a treatment date in March 2021 which fills me with hope for a future free from progression. I need your help to get there.

Joceline, who loves making stop-motion animation puppets and props and playing musical instruments, says the treatment will cost 40,800, and the flights 800.

She has launched a Go Fund Me page at https://gf.me/u/y538k2 which has already seen donations of more than 26,000.

I am incredibly grateful for any help you can give towards enabling me to access this life-changing treatment, she said.

After almost two decades of managing MS flare-ups and their consequences, its hard to put into words just what a future without them would mean to me.

Thank you for reading this and for any help you can put towards this goal.

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Haywards Heath woman's bid to fund stem cell treatment to combat MS - Mid Sussex Times

Global Stem Cells Cryopreservation Equipment Market is projected to grow at a CAGR +22% during the forecast period from 2020 to 2028.

Stem cells, which hold the promise of new cell-based therapies to treat critical medical conditions, have to be effectively stored until its time to use them. The method is called cryopreservation: it freezes stem cells at temperatures below -150oC, by immersing them in nitrogen vapour.

Stem cells will one day be effective in the treatment of many medical conditions and diseases. But unproven stem cell treatments can be unsafe so get all of the facts if youre considering any treatment. Stem cells have been called everything from cure-alls to miracle treatments.

Types of Stem Cells:-

Stem cells are divided into 2 main forms. They are embryonic stem cells and adult stem cells. Embryonic stem cells. The embryonic stem cells used in research today come from unused embryos.

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Key Players:-

Global Stem Cells Cryopreservation Equipment Market by Product:-

Global Stem Cells Cryopreservation Equipment Market by Applications:-

Due to the pandemic, we have included a special section on the Impact of COVID 19 on the Stem Cells Cryopreservation Equipment Market which would mention How the Covid-19 is affecting the Stem Cells Cryopreservation Equipment Industry, Market Trends and Potential Opportunities in the COVID-19 Landscape, Covid-19 Impact on Key Regions and Proposal for Stem Cells Cryopreservation Equipment Players to Combat Covid-19 Impact.

A new statistical report has recently published by Reports Consultant to its massive repository titled as Global Stem Cells Cryopreservation Equipment market 2020. This informative document takes a closer and analytical look on different aspects of the businesses to understand the business structure clearly. It has been compiled by using primary and secondary research techniques. Furthermore, it makes use of graphical presentation techniques such as ample graphs, charts, tables, and pictures.

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Global Stem Cells Cryopreservation Equipment Market by Region analysis:-

Why This Report important?

Global Stem Cells Cryopreservation Equipment Market Table of Content (TOC):-

Chapter1 Introduction

Chapter2 Research Scope

Chapter3 Global Stem Cells Cryopreservation Equipment Market Segmentation

Chapter4 Research Methodology

Chapter5 Definitions and Assumptions

Chapter6 Executive Summary

Chapter7 Global Stem Cells Cryopreservation Equipment Market Dynamics

Chapter8 Global Stem Cells Cryopreservation Equipment Market Key Players

Chapter9 Conclusion

Chapter10 Appendix

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Global Stem Cells Cryopreservation Equipment Market 2020 forecast to 2028, by types, by applications, by analysis with Worthington Industries, Cesca...

The allogeneic off-the-shelf CD22-directed T-cell product, UCART22, showed early signs of activity and no evidence of unexpected toxicities at 2 dose levels for adult patients with relapsed/refractory CD22-positive B-cell acute lymphoblastic leukemia, according to the results of a study presented during the 2020 ASH Annual Meeting.1

In the phase 1 BALLI-01 (NCT04150497) dose-escalation and dose-expansion study, 2 patients at the 1 x 105 cells/kg dose achieved a complete remission (CR) with incomplete hematologic recovery on day 28. One of these patients attained a minimal residual disease (MRD)positive CR at day 42 followed by subsequent inotuzumab ozogamicin (Besponsa) and then transplant.

One patient at dose level 2, 1 x 106 cells/kg, experienced a significant bone marrow blast reduction at day 28, followed by disease progression.

No patients experienced dose-limiting toxicities (DLTs), immune effector cellassociated neurotoxicity syndrome (ICANS), graft-versus-host disease (GVHD), adverse effects (AE) of special interest (AESI), a UCART22-related AE that was grade 3 or higher, or a serious AE (SAE).

UCART22 showed no unexpected toxicities at the doses of 1 x 105 cells/kg and 1 x 106 cells/kg with fludarabine and cyclophosphamide lymphodepletion, lead study author Nitin Jain, MD, an assistant professor in the Department of Leukemia, The University of Texas MD Anderson Cancer Center, said in a virtual presentation during the meeting. Host immune recovery was observed early, and the addition of alemtuzumab [Lemtrada] to fludarabine and cyclophosphamide lymphodepletion is currently being explored with the goal to achieve deeper and more sustained T-cell depletion and to promote expansion and persistence of UCART22.

Standard treatment for adult patients with B-cell ALL includes multiagent chemotherapy with or without allogeneic stem cell transplant. However, 30% to 60% of patients with newly diagnosed B-cell ALL who achieve a CR will relapse, and the expected 5-year survival rate for those with relapsed/refractory disease is approximately 10%.

Previously, UCART19, when paired with lymphodepletion using fludarabine, cyclophosphamide, and alemtuzumab, was found to show efficacy in this patient population.2

CD22 is an FDA-approved therapeutic target in B-cell ALL. UCART22 is an immediately available, standardized, manufactured agent with the ability to re-dose, and its CAR expression redirects T cells to tumor antigens, Jain explained.

Moreover, through its mechanism of action, TRAC becomes disrupted using Transcription activator-like effector nucleases (Talen) technology to eliminate TCR from cell surface and reduce the risk of GVHD. CD52 is also disrupted with the use of Talen to eliminate sensitivity to lymphodepletion with alemtuzumab. Finally, there is a CD20 mimotope for rituximab (Rituxan) as a safety switch, Jain added.

UCART22 has also demonstrated in vivo antitumor activity in immune-compromised mice that were engrafted with CD22-positive Burkitt lymphoma cells in a dose-dependent manner.

In the dose-escalation/dose-expansion BALLI-01 study, investigators are enrolling up to 30 patients in a modified Toxicity Probability Interval design. There are 3 cohorts, which have 2 to 4 patients on each cohort: 1 x 105 cells/kg (dose level 1), 1 x 106 cells/kg (dose level 2), and 5 x 106 cells/kg. The focus of the dose-escalation phase of the trial was to determine the maximum-tolerated dose (MTD) and the recommended phase 2 dose (RP2D) before heading into the dose-expansion portion of the trial.

To be eligible for enrollment, patients must have been between 18 and 70 years old, have acceptable organ function, an ECOG performance status of 0 or 1, at least 90% of B-cell ALL blast CD22 expression, and had previously received at least 1 standard chemotherapy regimen and at least 1 salvage regimen.

End points of the trial included safety and tolerability, MTD/R2PD, investigator-assessed response, immune reconstitution, and UCART22 expansion and persistence.

The lymphodepletion regimens were comprised of fludarabine (at 30 mg/m2 x 4 days) plus cyclophosphamide (1 g/m2 x 3 days); the study has since been amended to include the regimen of fludarabine (at 30 mg/m2 x 3 days), cyclophosphamide (500 g/m2 x 3 days), and alemtuzumab (20 mg/day x 3 days) and is currently enrolling patients.

Following screening, lymphodepletion, and UCART22 infusion, patients underwent an observation period for DLTs with a primary disease evaluation at 28 days; additional efficacy evaluations occurred at 56 days and 84 days. Patients were followed for 2 years and continued to be assessed for long-term follow-up.

As of July 1, 2020, 7 patients were screened, of which 1 patient failed and 6 were therefore enrolled on the study. One patient discontinued therapy before receiving UCART22 due to hypoxia from pneumonitis that was linked with lymphodepletion. Five patients were treated with UCART22 at dose level 1 (n = 3) and dose level 2 (n = 2).

The median age of participants was 24 years (range, 22-52), 3 of the 5 patients were male, and 3 had an ECOG performance status of 0. The median number of prior therapies was 3 (range, 2-6), and there were a median 35% bone marrow blasts (range, 10%-78%) prior to lymphodepletion.

Three patients had complex karyotype and 2 had diploid cytogenetics. One patient each had the following molecular abnormalities: CRLF2, CRLF2 and JAK2, CDKN2A loss, KRAS and PTPN11, and IKZF1. Only 1 patient had undergone haploidentical transplant. Four patients previously received prior CD19- or CD22-directed therapy, including blinatumomab (Blincyto), inotuzumab ozogamicin (Besponsa), and CD19-directed CAR T-cell therapy. At study entry, 3 patients had refractory disease and 2 patients had relapsed disease.

Grade 3 or higher treatment-emergent AEs (TEAEs), which were unrelated to study treatment, included hypokalemia, anemia, increased bilirubin, and acute hypoxic respiratory failure. Also not related to UCART22, 3 patients experienced 4 treatment-emergent SAEs: porta-hepatis hematoma, sepsis, bleeding, and sepsis in the context of disease progression. No treatment discontinuations due to a treatment-related TEAE were reported.

The patient who achieved a CR followed by transplant was a 22-year-old male who had undergone 2 prior treatments for B-cell ALL and received UCART22 at a dose of 1 x 105 cells/kg. He did not experience CRS, ICANS, GVHD, nor a SAE, and all TEAEs were grade 1.

Jain also noted that host T-cell constitution was observed in all patients within the DLT observation period. UCART22 was also not detectable through flow cytometry or molecular analysis, the latter of which was at dose level 1 only.

References:

1. Jain N, Roboz GJ, Konopleva M, et al. Preliminary results of BALLI-O1: a phase I study of UCART22 (allogeneic engineered T cells expressing anti-CD22 chimeric antigen receptor) in adult patients with relapsed/refractory anti-CD22+ B-cell acute lymphoblastic leukemia (NCT04150497). Presented at: 2020 ASH Annual Meeting and Exposition; December 4-8, 2020; Virtual. Abstract 163.

2. Benjamin R, Graham C, Yallop D, et al. Preliminary data on safety, cellular kinetics and anti-leukemic activity of UCART19, an allogeneic anti-CD19 CAR T-cell product, in a pool of adult and pediatric patients with high-risk CD19+ relapsed/refractory b-cell acute lymphoblastic leukemia. Blood. 2018;132(suppl 1):896. doi:10.1182/blood-2018-99-111356.

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Early Signs of Activity and Tolerability Found in Allogeneic Product UCART22 for Patients with Relapsed/Refractory CD22+ B-Cell ALL - Cancer Network