Stem Cell Therapy Shows 2-year Benefit for Progressive MS Patients in Phase 1 Trial – Multiple Sclerosis News Today

Lesser or stable disability over two years was evident in most progressive multiple sclerosis (MS) patients given a stem cell treatment in a small Phase 1 clinical trial, supporting a larger study now underway, researchers report.

These results suggest that a treatment using mesenchymal stem cell-derived neural progenitors (MSC-NPs) can safely and effectively ease inflammation in progressive MS.

But for a subset of patients, particularly those with more advanced disease and greater disability, this treatment did not sufficiently counter a continued inflammatory response in the brain.

The study, Mesenchymal stem cell-derived neural progenitors in progressive MS: Two-year follow-up of a phase I study, was published in the journal Neurology: Neuroimmunology and Neuroinflammation.

MSC-NPs are seen as a possible way of treatingpeople with progressive MS, who have few effective disease-modifying treatments available. They are essentially stem cells collected from a patients bone marrow that are expanded and matured to produce factors involved in modulating the immune response and innervous tissue growth and survival.

An open-label Phase 1 trial (NCT01933802) investigated this stem cell treatment in 20 adults with stable primary(four PPMS patients) or secondary progressive MS(16 SPMS patients) and significant disability.

All received a total of three injections of MSC-NPs, given directly into the spinal canal three months apart. They were then evaluated at three and six months, and again at two years, after the final treatment to determine its long-term safety and tolerability, and for signs of potential effectiveness.

An initial analysisat six months post-treatment found lesser disability in most trial participants (15 of the 20) andbetter muscle strength in 14 of them. Greater exercise capacity was also seen in four of the 10 patients able to walk at the studys start, and two nonambulatory patients gained an ability to walk using assistive devices.

Researchers now reported clinical findings at two years after treatment. All 20 completed two-year follow-up assessments, buttwo who were severely disabled could not do a final in-person visit. They were examined via telemedicine and did not provide biomarker samples.

Disability was evaluated using the Expanded Disability Status Scale (EDSS), in which a higher score indicates more severe disability. The two who moved to telemedicine had EDSS scores of 8.0.

At six months, eight participants had an EDSS reduction of at least 0.5 points, including four with disability reductions of two or more points. At the two-year follow-up, seven of these eight people continued to show improvements in their EDSS scores, including two who showed a sustained 2.0 or more point reduction.

The eighth patient, whose disability had initially improved by one point, showed a worsening in disability at two years.

Of the 10 patients without initial improvements in EDSS scores, six had no evidence of disease progression throughout the study and follow-up. Two others worsened at each follow-up, and two showed worsening disease between the six-month and two-year examinations.

Of the 10 nonambulatory patients at the trials start, four showed improvements in walking speed greater than 20% at three months post-treatment. At two years, three had maintained these walking speed gains, while one fell just below the 20% improvement mark.

One of the two people unable to walk at the beginning of the study completed the walking test at both the three-month and two-year exams. One other patient, with an initial normal walking speed, maintained that speed throughout the trial and follow-up periods.

These results indicate that multiple MSC-NP treatments led to disability reduction for most progressive patients with long-standing disease. But those who sustained these gains at two years after treatment had lower EDSS and ambulatory status at baseline or the studys start, the researchers wrote.

A subset of patients with initial improvement failed to maintain shown benefits, while others showed no disease progression throughout the follow-up.

Cerebrospinal fluid (CSF) levels of CCL2, a pro-inflammatory factor, were lower following treatment, while levels of the anti-inflammatory TGF beta 2 rose post-treatment, consistent with previous studies of similar treatments.

Interestingly, no difference here was observed between patients whose disability improved in response to the treatment (responders) and those who failed to improve (non-responders).

However, some inflammatory factors were seen to rise after treatment in non-responders, but not among those who responded to treatment. This suggests that a continued inflammatory response may hinder clinical response to MSC-NP use.

Neurofilament light chain (NfL) levels in the CSF, a marker of nerve cell degeneration and damage, can be elevated in MS patients. Among a small number of trial patients with high NfL levels prior to treatment, these levels rose further in nonresponders after treatment while they declined among responders.

We observed that most subjects who received repeated [MSC-NP] injections exhibited either a reversal in disability or lack of disease progression that was sustained for 2 years after treatment, the researchers wrote.

The impact of any efficacy conclusion, however, are severely limited by the very small number of patients in the study and the lack of blinding and placebo controls, they added.

An ongoing and placebo-controlled Phase 2 clinical trial (NCT03355365), which opened last year, is now investigating the safety and efficacy of repeat MSC-NP injections in progressive MS patients. The study is expected to have enrolled50 adults with progressive MS (40 SPMS and 10 PPMS), being given a total of six injections of either MSC-NPs or a placebo every other month for a first year.

In its second year, those in the MSC-NP group cross to the placebo group and those previously on a placebo move to treatment again for six total injections given every two months. This single-site trial at the Tisch MS Research Center of New Yorkwill run for three years, and is expected to finish in late 2023.

Aisha Abdullah received a B.S. in biology from the University of Houston and a Ph.D. in neuroscience from Weill Cornell Medical College, where she studied the role of microRNA in embryonic and early postnatal brain development. Since finishing graduate school, she has worked as a science communicator making science accessible to broad audiences.

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Dr Clarence Moore Discusses the Importance of Stem Cell Therapy, Heart Health in Beta Thalassemia – AJMC.com Managed Markets Network

Clarence Moore, PharmD, BCPS, BCOP, assistant professor at Shenandoah University in Ashburn, Virginia, discusses how stem cell therapy and heart health can help patients manage beta thalassemia.

Transcript

How has the emergence of stem cell therapy changed the lives of people with beta thalassemia?

Stem cell therapy is offering a cure. So, with us really looking at stem cell therapy, with the more and more work [being done], we'll be able to see that these individuals will no longer have to experience this lifelong disease, and they could potentially be cured.

What is the importance of heart health for patients, especially those with beta thalassemia major?

Heart health is very important. When you look at one of the largest comorbidities that is associated with [beta thalassemia], it is heart health. You can see some cardiomyopathy develop. So, maintaining heart health is very important in decreasing the morbidity and mortality associated with the disease state.

What is the importance conducting blood tests to determine beta thalassemia types in young children?

I think that it's very important. Knowing upfront what disease or what type of beta thalassemia that these individuals may have is going to be vital for us to manage it. And if we're able to potentially manage that at the earlier stage, we could potentially have better outcomes in the future.

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

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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The Adrenomyeloneuropathy Treatment Market to grow on an emphatic note from 2019 to 2029 – PharmiWeb.com

Adrenomyeloneuropathy is a rare genetic neuro-degenerative disease. Adrenomyeloneuropathy is the adult onset of adrenoleukodystrophy caused by the mutation in ABCD1 gene occurs usually in young boys. Adrenomyeloneuropathy disease affect the nerve cells in the spine and brain and the adrenal glands. Adrenomyeloneuropathy symptoms includes stiffness, weakness and pain in the legs. Adrenomyeloneuropathy is also known as progressive spastic paraparesis. Damage to the nerves of the legs which causes unsteadiness and fall, also the bladder, bowel and sexual organs are affected by the adrenomyeloneuropathy. Rare diseases affect vast numbers of people, with current data representing 30 million sufferers in the EU alone and 30 million affected in the US. There is no cure to Adrenomyeloneuropathy. However some treatment might stop the progression of Adrenomyeloneuropathy such as stem cell transplants. Blood testing, MRI test, vision screening and Skin biopsy and fibroblast cell culture are done for the diagnosis for the adrenomyeloneuropathy. Continued advances in the treatment of adrenomyeloneuropathy will further propel the adrenomyeloneuropathy treatment market.

Growing cases of rare disease and development of new and advanced treatment for rare disease is expected to boost the adrenomyeloneuropathy treatment market. Growing preference for healthy lifestyle and favorable government regulation spur the Adrenomyeloneuropathy treatment market in the forecast period. Development of new technology and devices for the diagnosis of genetic disorders will propel the adrenomyeloneuropathy treatment market. Rising focus on the research and development of new therapeutic and drug treatment and growing government funding for the orphan drug is expected to drive the adrenomyeloneuropathy treatment market.

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However, stringent regulations for the drug development and high cost of associated with the treatment is expected to hinder the adrenomyeloneuropathy treatment market.

The global adrenomyeloneuropathy treatment market is segmented on basis of disease type, drug type and end user and geography.

Development of novel drugs and undergoing clinical trial for the rare disease is expected to boost adrenomyeloneuropathy treatment market. More than 3,000 drugs are in active development for one of the rare disease. Progress in genomics and biomedical science for the development of rare disease drug is expected to spur the adrenomyeloneuropathy treatment market. Various pharmaceutical companies are focusing on developing drug for the low prevalence disease types and rising funding and collaboration among the key players and government is expected to spur the adrenomyeloneuropathy treatment market.

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The North America market for adrenomyeloneuropathy treatment is expected to retain its dominance, owing to increasing patient pool for rare disease, increasing government funding to accelerate the research and development for rare disease. According to Genetic and Rare Diseases Information Center, more than 25 million Americans are suffering from rare disease in United States.Europe is expected to account for the second largest share in the global adrenomyeloneuropathy treatment market owing to growing clinical trial funding programs for orphan drug development and high prevalence of adrenomyeloneuropathy and high treatment seeking rate. Asia Pacific is expected to show significant growth, owing to increasing diagnosis rate and improvement in healthcare infrastructure. China is expected to show significant growth in the adrenomyeloneuropathy treatment market, due to rising population improving R&D capability, increasing per capita heath spending. Latin America and Middle East & Africa is expected to show growth owing to lack of diagnosis and inadequate healthcare facilities and lack of skilled physicians for Adrenomyeloneuropathy Treatment market.

Examples of some of the key manufacturer present in the global adrenomyeloneuropathy treatment market are Ascend Biopharmaceuticals, Novadip Biosciences, Eureka Therapeutics, Human Longevity, Regeneus, Allogene Therapeutics, BioRestorative Therapies, Immatics Biotechnologies, NewLink Genetics, Cytori Therapeutics, Talaris Therapeutics among others.

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Are Hiccups a Sign of the New Coronavirus? – Healthline

In March 2020, the World Health Organization declared COVID-19, the disease caused by the SARS-CoV-2 virus, a pandemic.

Since then, COVID-19 has affected tens of millions of people around the world, leading to new discoveries about the symptoms that can accompany the disease.

Recently, multiple case studies have suggested that persistent hiccups may be a potentially rare and unusual manifestation of COVID-19.

In this article, well discuss whether hiccups are a sign of the new coronavirus, when to contact your doctor about frequent hiccups, and other important information you should know about COVID-19.

According to the research, it is possible that hiccups are a rare sign of COVID-19.

In one recent 2020 case study, a 64-year-old man was found to have persistent hiccups as the only symptom of COVID-19.

In this situation, the subject of the study visited an outpatient clinic after experiencing a bout of hiccups for 72 hours.

Both blood testing and lung imaging were performed. They revealed evidence of infection in both lungs and low white blood cells. Follow-up testing for COVID-19 revealed a positive diagnosis.

In a different 2020 case study, a 62-year-old man was also found to have experienced hiccups as a symptom of the new coronavirus.

In this case, the subject had been experiencing hiccups for a period of 4 days before presentation to the emergency room.

Upon admission, further testing showed similar findings in their lungs, as well as low white blood cells and platelets. Again, testing for COVID-19 confirmed a positive diagnosis.

It is important to note that the studies mentioned above are only two individual case studies. They only demonstrate a potentially rare side effect of COVID-19.

More research is still needed to determine the link between chronic hiccups and the new coronavirus.

Hiccups are quite common and happen when your diaphragm involuntarily spasms or contracts. Your diaphragm is your muscle directly beneath your lungs that separates your chest from your abdomen.

Hiccups can be caused by everything from eating to swallowing air to stress, and much more.

While they can be somewhat annoying, hiccups are rarely a sign of anything dangerous. Generally, hiccups only last a few minutes although in some cases, they have been known to last for hours.

According to the National Health Service, hiccups that last longer than 48 hours are considered a cause for concern and should be addressed by a doctor.

Medical treatment options for hiccups are generally reserved for people with chronic hiccups that dont resolve on their own. Some of these treatment options may include:

For most people, hiccups will resolve on their own they generally only become a concern if they become chronic or cause other health concerns.

You should talk with a doctor if your hiccups last longer than 48 hours, as this may be a sign of an underlying health condition.

You may also need to talk with a doctor if your hiccups cause you to be unable to eat, breathe, or do anything else you would typically be able to do.

According to the Centers for Disease Control and Prevention (CDC), the most common symptoms of COVID-19 include:

Symptoms of COVID-19 can appear anywhere from 2 to 14 days after exposure to the SARS-CoV-2 virus. Depending on the severity of the disease, the symptoms can range from asymptomatic (no symptoms at all) to severe.

In some situations, COVID-19 can cause uncommon symptoms that are not listed above, such as dizziness or rash.

Even rarer, case studies like those mentioned above have shown how other unusual symptoms can be a sign of the new coronavirus.

If you are experiencing new symptoms and concerned that you may have developed COVID-19, speak with your doctor as soon as possible for testing.

While not everyone needs to be tested for COVID-19, the CDC recommends getting tested if:

There are two types of testing available for COVID-19: viral testing and antibody testing. Viral testing is used to diagnose a current infection, while antibody testing can be used to detect a past infection.

Tests are available nationwide at most local or state health departments, doctors offices, and pharmacies. Some states also currently offer drive-thru testing and 24-hour emergency testing when necessary.

We all play an important role in preventing the spread of the SARS-CoV-2 virus. The best way to reduce your risk of contracting, or spreading, this new coronavirus is to practice personal hygiene and physical distancing.

This means following the CDC guidelines for preventing the spread of COVID-19 and being mindful of your own health and testing status.

Staying informed about current and developing COVID-19 news is also important you can keep up to date with Healthlines live coronavirus updates here.

Below, youll find some CDC recommended guidelines to protect yourself and prevent the spread of COVID-19:

According to the CDC, in December 2020, a vaccine from Pfizer was granted emergency use authorization and approval for a vaccine from Moderna is expected to follow.

It may take months before most people have access to this vaccine, but there are also treatment options available.

The current treatment recommendation for mild cases of COVID-19 is recovery at home. In more severe cases, certain medical treatments may be used, such as:

As the COVID-19 situation continues to develop, so do new treatment options to help combat the disease.

Many of the symptoms of COVID-19 are commonly experienced among people who have developed the disease. However, research has suggested that some people may experience other rare and unusual symptoms.

In two recent case studies, persistent hiccups were the only outward sign of the new coronavirus. While this indicates that hiccups may be a potential symptom of COVID-19, more research is needed on this rare side effect.

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Every Patient Treated With CRISPR Gene Therapy for Blood Diseases Continues to Thrive, More Than a Year On – Good News Network

18 months into the first serious clinical trials of CRISPR gene therapy for sickle cell disease and beta-thalassemiaand all patients are free from symptoms and have not needed blood transfusions.

Sickle cell disease (SCD) can cause a variety of health problems including episodes of severe pain, called vaso-occlusive crises, as well as organ damage and strokes.

Patients with transfusion-dependent thalassemia (TDT) are dependent on blood transfusions from early childhood.

The only available cure for both diseases is a bone marrow transplant from a closely related donor, an option that is not available for the vast majority of patients because of difficulty locating matched donors, the cost, and the risk of complications.

In the studies, the researchers goal is to functionally cure the blood disorders using CRISPR/Cas9 gene-editing by increasing the production of fetal hemoglobin, which produces normal, healthy red blood cells as opposed to the misshapen cells produced by faulty hemoglobin in the bodies of individuals with the disorders.

The clinical trials involve collecting stem cells from the patients. Researchers edit the stem cells using CRISPR-Cas9 and infuse the gene-modified cells into the patients. Patients remain in the hospital for approximately one month following the infusion.

Prior to receiving their modified cells, the seven patients with beta thalassemia required blood transfusions approximately every three to four weeks and the three patients with SCD suffered episodes of severe pain roughly every other month.

All the individuals with beta thalassemia have been transfusion independent since receiving the treatment, a period ranging between two and 18 months.

Similarly, none of the individuals with SCD have experienced vaso-occlusive crises since CTX001 infusion. All patients showed a substantial and sustained increase in the production of fetal hemoglobin.

15 months on, and the first patient to receive the treatment for SCD, Victoria Gray, has even been on a plane for the first time.

Before receiving CRISPR gene therapy, Gray worried that the altitude change would cause an excruciating pain attack while flying. Now she no longer worries about such things.

She told NPR of her trip to Washington, D.C: It was one of those things I was waiting to get a chance to do It was exciting. I had a window. And I got to look out the window and see the clouds and everything.

MORE: MIT Researchers Believe Theyve Developed a New Treatment for Easing the Passage of Kidney Stones

This December, theNew England Journal of Medicinepublishedthe first peer-reviewed research paperfrom the studyit focuses on Gray and the first TDT patient who was treated with an infusion of billions of edited cells into their body.

There is a great need to find new therapies for beta thalassemia and sickle cell disease, saidHaydar Frangoul, MD,Medical Director of Pediatric Hematology and Oncology at Sarah Cannon Research Institute, HCA Healthcares TriStar Centennial Medical Center. What we have been able to do through this study is a tremendous achievement. By gene editing the patients own stem cells we may have the potential to make this therapy an option for many patients facing these blood diseases.

READ: For the First Time in the US, Surgeons Pump New Life into Dead Donor Heart for Life-Saving Transplant

Because of the precise way CRISPR-Cas9 gene editing works, Dr. Frangoul suggested the technique could potentially cure or ameliorate a variety of diseases that have genetic origins.

As GNN has reported, researchers are already using CRISPR to try and treat cancer, Parkinsons, heart disease, and HIV, as well.

Source: American Society of Hematology

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Every Patient Treated With CRISPR Gene Therapy for Blood Diseases Continues to Thrive, More Than a Year On - Good News Network

Therapeutic Solutions International Files Investigational New Drug Application (IND) with FDA for Treatment of Chronic Traumatic Encephalopathy Using…

OCEANSIDE, Calif., Dec. 17, 2020 /PRNewswire/ -- Therapeutic Solutions International, Inc., (OTC Markets: TSOI), announced today filing of an Investigational New Drug (IND) application seeking permission from the Food and Drug Administration (FDA) to initiate a Phase I/II clinical trial assessing safety and signals of efficacy for treatment of Chronic Traumatic Encephalopathy (CTE) patients with JadiCells.

The JadiCell is a type of umbilical cord derived mesenchymal stem cell which possesses superior therapeutic activity as compared to other stem cell types based on laboratory and clinical studies. The JadiCell was recently reported to possess therapeutic activity against COVID-19 in a recently published clinical trial1. The Company previously obtained an exclusive license for use of JadiCells for CTE and Traumatic Brain Injury (TBI)2,3.

"My team and myself are extremely excited about the potential of using our unique novel cell therapy for treatment of CTE, which is the cause of countless deaths, as well as chronic disabilities" said Dr. Amit Patel, inventor of the JadiCell. "Having worked over the past several years with the multidisciplinary team assembled by Timothy Dixon, I look forward to progress in this area of medicine, for which currently there are limited options."

Chronic Traumatic Encephalopathy (CTE) is caused by repetitive concussive/sub-concussive hits to the head sustained over a period of years and is often found in football players4. The condition is characterized by memory loss, impulsive/erratic behavior, impaired judgment, aggression, depression, and dementia. In many patients with CTE, it is anatomically characterized by brain atrophy, reduced mass of frontal and temporal cortices, and medial temporal lobe. TSOI has previously filed several patents in the area of CTE based on modulating the brain microenvironment to enhance receptivity of regenerative cells such as stem cells.

Suicide is a major cause of death in military veterans, approximately 20 deaths per day5. It is widely known that TBI and CTE are associated with increased propensity for suicide6,7. Furthermore, studies show that cytokines associated with brain inflammation, such as IL-6 and TNF-alpha are found in higher levels in patients with suicidal tendencies8.

"To our knowledge, this is the first therapeutic intervention addressing CTE using stem cells and we are tremendously excited to finally move into clinical testing for this terrible disease that not only affects athletes but our military and law enforcement as well" said Timothy Dixon, President and CEO of the Company. "Having worked over the last 2 years in developing the preclinical data supporting this IND, we are extremely pleased to file this new drug application today."

About Therapeutic Solutions International, Inc.

Therapeutic Solutions International is focused on immune modulation for the treatment of several specific diseases. The Company's corporate website is http://www.therapeuticsolutionsint.com, and our public forum is https://board.therapeuticsolutionsint.comand Campbell Neurosciences at https://www.campbellneurosciences.com

1https://papers.ssrn.com/sol3/papers.cfm?abstract_id=3696875

2https://www.globenewswire.com/news-release/2019/07/01/1876699/0/en/Therapeutic-Solutions-International-2Obtains-Exclusive-License-for-Patented-Clinical-Stage-Adult-Stem-Cell-for-Treatment-of-Military-and-Sports-Brain-Injuries.html

3https://patents.google.com/patent/US9803176?oq=9803176

4https://www.nytimes.com/interactive/2017/07/25/sports/football/nfl-cte.html

5https://en.wikipedia.org/wiki/United_States_military_veteran_suicide

6https://www.psychcongress.com/news/traumatic-brain-injury-tied-increased-risk-suicide

7https://sbindependent.org/nfl-athletes-with-cte-who-committed-suicide/

8https://www.ncbi.nlm.nih.gov/pubmed/20951793

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

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 Logic Physio 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 Regenexx Regenexx 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

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AlloVir Announces FDA Clearance of Investigational New Drug Application for ALVR106, an Allogeneic, Off-the-Shelf, Multi-Virus Specific T Cell Therapy…

Dec. 17, 2020 12:30 UTC

Proof-of-concept phase 1/2 trial to initiate in 2021 to treat severe respiratory viral infections in patients following hematopoietic stem cell transplantation

ALVR106 designed to target devastating diseases caused by four respiratory viruses: respiratory syncytial virus, influenza, parainfluenza virus, and human metapneumovirus

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- AlloVir (Nasdaq: ALVR), a late clinical-stage cell therapy company, announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for ALVR106, an allogeneic, off-the-shelf virus-specific T cell therapy (VST) designed to target infections and diseases caused by respiratory syncytial virus (RSV), influenza, parainfluenza virus (PIV), and human metapneumovirus (hMPV). The IND enables AlloVir to initiate a Phase 1/2 proof-of-concept clinical study in allogeneic and autologous hematopoietic stem cell transplant (HSCT) patients with respiratory infections caused by RSV, influenza, PIV or hMPV.

Respiratory viruses are a leading cause of morbidity and mortality in HSCT patients, and based on evidence from our preclinical studies, we believe ALVR106 could transform the treatment and prevention of respiratory infections and substantially reduce the associated morbidity and mortality of these infections in the future, said Ercem Atillasoy, M.D., Chief Regulatory and Safety Officer of AlloVir. The clearance of the IND for ALVR106 advances our third program into clinical trials further exploring the power of our proprietary virus-specific T cell therapy platform.

Respiratory tract infections due to RSV, influenza, PIV, and hMPV are a major public health concern and are detected in up to 40 percent of allogeneic HSCT patients. These viral infections can progress from upper respiratory tract infections to more serious lower respiratory tract infections, which are associated with mortality rates of 20-45 percent in HSCT patients.

As previously disclosed, this proof-of-concept clinical trial will initiate in 2021 as the company is assessing the impact of the COVID-19 pandemic on the incidence, diagnosis, and treatment of the respiratory viral infections which ALVR106 targets.

About ALVR106

ALVR106 is an allogeneic, off-the-shelf, multi-virus specific VST investigational therapy designed to target infections and diseases caused by the respiratory syncytial virus (RSV), influenza, parainfluenza virus (PIV), and human metapneumovirus (hMPV). In vitro data demonstrate that ALVR106 has antiviral activity against each of the targeted viruses with minimal or no activity against non-virus-infected cells. This preclinical data supports the potential for antiviral benefit and safety of ALVR106 when administered to patients.

About AlloVir

AlloVir is a leading late clinical-stage cell therapy company with a focus on restoring natural immunity against life-threatening viral diseases in patients with weakened immune systems. The companys innovative and proprietary technology platforms leverage off-the-shelf, allogeneic, multi-virus specific T cells targeting devastating viruses for patients with T cell deficiencies who are at risk from the life-threatening consequences of viral diseases. AlloVirs technology and manufacturing process enables the potential for the treatment and prevention of a spectrum of devastating viruses with each single allogeneic cell therapy. The company is advancing multiple mid- and late-stage clinical trials across its product portfolio. For more information visit http://www.allovir.com.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding AlloVirs development and regulatory status of our product candidates and its strategy, business plans and focus. The words may, will, could, would, should, expect, plan, anticipate, intend, believe, estimate, predict, project, potential, continue, target and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on managements current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those related to AlloVirs financial results, the timing for completion of AlloVirs clinical trials of its product candidates, whether and when, if at all, AlloVirs product candidates will receive approval from the U.S. Food and Drug Administration, or FDA, or other foreign regulatory authorities, competition from other biopharmaceutical companies, and other risks identified in AlloVirs SEC filings. AlloVir cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. AlloVir disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent AlloVirs views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date.

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AlloVir Announces FDA Clearance of Investigational New Drug Application for ALVR106, an Allogeneic, Off-the-Shelf, Multi-Virus Specific T Cell Therapy...

ONK Therapeutics Announces Three Exclusive Option License Agreements, Which Extend and Strengthen its Dual-Targeted NK Cell Therapy Pipeline -…

Dec. 17, 2020 08:30 UTC

GALWAY, Ireland, & SAN DIEGO, Calif.--(BUSINESS WIRE)-- ONK Therapeutics Ltd, an innovative natural killer (NK) cell therapy company, today announced that it has secured three new exclusive option license agreements which strengthen its off-the-shelf, dual-targeted natural killer (NK) cell therapy platform and extend its pre-clinical pipeline to four programs across both hematological and solid tumors.

The first option agreement, with Cellerant Therapeutics, gives exclusive rights to a humanized CLEC12A scFv binder. CLEC12A is strongly expressed by blasts in the majority of AML patients. The option to license has enabled ONK to expand its pre-clinical product portfolio, launching a fourth program (ONKT104). This dual-targeted approach combines the CLEC12A CAR with a TNF-related apoptosis-inducing ligand variant (TRAILv) targeting death receptor 4 (DR4).

While expressed on leukemic stem cells, CLEC12A is absent from normal hematopoietic stem cells and we thus expect that our dual-targeted NK cell therapy approach should enable safe targeting, with a reduced risk of prolonged aplasia in AML, said Prof Michael ODwyer MD, ONK Therapeutics co-founder, and CSO.

The second agreement in-licenses a humanized, tumor-specific antibody targeting an aberrantly glycosylated tumor-associated form of MUC1 (TA-MUC1) from Glycotope GmbH. Multiple solid tumor types express the mucin MUC1, including non-small cell lung cancer, breast cancer, and ovarian cancer. This antibody will be integrated into ONKs pre-clinical program ONKT103, for solid tumors.

Non-selective targeting of MUC1 could be problematic since the target is also expressed by healthy tissues, but ODwyer explains how ONKs dual-targeted approach can be used to address this. We have designed a CAR tailored to the glycosylation pattern distinct to tumor-associated MUC1 with specific recognition of the carbohydrate antigens Tn and T on MUC1, the expression of which is restricted to cancer cells. Glycotope has identified the glycosylation pattern as a way to unlock the potential of TA-MUC1 as a solid tumor target. ONK is thus set to bring the natural benefits of NK cells over T cells to bear on TA-MUC1, in a tumor-specific fashion, while also further boosting efficacy and countering resistance through the use of our TRAIL variant targeting DR5, he said.

ONKs unique platform approach combines the expression of a chimeric antigen receptor (CAR) and a high affinity, membrane-bound TRAILv. The incorporation of these two humanized scFvs has the potential to minimize the risk of immunogenicity in the allogeneic setting.

ONK is also exploring several innovative strategies to improve the homing of NK cells. This is an important consideration as ex-vivo expansion can lead to changes in chemokine receptor expression. Through this new license agreement with the NIH, ONK plans to enforce the expression of CCR7, which is downregulated on NK cell expansion. This may improve the homing of NK cells to lymph nodes and is expected to be particularly useful for ONKs off-the-shelf CD19 program targeting B cell lymphoma, ONKT101, which is partnered with Avectas.

ONK is making rapid progress since it announced its most recent financing in October. Chris Nowers, ex Kite Pharma Head of Europe, who joined at that time as Chief Executive Officer, said: The recent American Society of Hematology meeting highlighted the NK cell therapy area as offering great hope as the next generation of advanced cell therapies. We believe our best-in-class off-the-shelf, dual-targeted NK cell therapy platform has the potential to improve performance and overcome some of the shortcomings seen with earlier approaches. These new licensing activities strengthen and expand our programs and illustrate our ambition and strategy to become a leader in this exciting field.

The company recently expanded its operations into the USA, moving into JLABS @ San Diego, Johnson & Johnson Innovations flagship facility, at the heart of San Diegos precision medicine and cell therapy cluster. This represents a second facility that complements its main R&D team and operations in Galway, Ireland. The companys recruitment drive across both facilities has been rapid and the company continues to expand its capability in key areas, including NK cell biology, construct design, gene editing, and process development.

-Ends-

ONK Therapeutics http://www.onktherapeutics.com ONK Therapeutics Ltd is an innovative cell therapy company dedicated to developing the next generation of off-the-shelf, dual-targeted NK cell therapies targeting solid and hematological cancers.

The company was founded in 2015, by Prof. ODwyer MD, of NUI Galway, an expert in translational multiple myeloma research, the tumor microenvironment, and exploitation of NK cells as cellular immunotherapy. Its core proprietary platform is based on a dual-targeted NK cell expressing both a chimeric antigen receptor (CAR) targeting a known tumor antigen and a TNF-related apoptosis-inducing ligand variant (TRAILv) targeting the death receptor pathway (i.e. DR4 or DR5). This unique approach has the potential to enhance efficacy by addressing both intrinsic (e.g. CAR engagement of a tumor-specific antigen) and extrinsic (e.g. signaling through the death receptor pathway) apoptotic pathways and to reduce the susceptibility to possible target antigen escape through the engagement of tumor antigen-independent TRAILv.

Its pre-clinical pipeline comprises four programs;

In addition to the unique dual-targeted NK cell therapy platform, the company has a strong research focus on strategies to enhance homing and persistence, and overcome exhaustion, including the exploration of proprietary gene edits, such as the deletion of checkpoint inhibitory receptors in NK cells.

ONK Therapeutics is headquartered in the med-tech hub of Galway, Ireland, with a wholly-owned US subsidiary, ONK Therapeutics, Inc. based at JLabs @ San Diego. Shareholders include Acorn Bioventures, ALSHC (principally Seamus Mulligan), and Enterprise Ireland.

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About Avectas - http://www.avectas.com Avectas is a cell engineering technology business that has developed a unique delivery platform, Solupore to enable the ex vivo manufacture of cell therapy products, which have high in-vivo functionality.

Glycotope http://www.glycotope.com Glycotope is a biotechnology company utilizing a proprietary technology platform to develop highly tumor-specific monoclonal antibodies called GlycoBodies. GlycoBodies bind to targets (GlycoTargets) tumor-specific carbohydrate structure dependent, enabling the development of highly-specific immunotherapies across a broad range of cancer indications. Glycotope has to date discovered in excess of 150 GlycoTargets with GlycoBodies against eight of these targets currently under development.

Each GlycoBody can be developed in an array of modalities with different modes of action providing a unique offering in the (immuno) oncology space. Currently, six clinical and pre-clinical programs based on the GlycoBody technology are under development by Glycotope or its licensing partners.

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ONK Therapeutics Announces Three Exclusive Option License Agreements, Which Extend and Strengthen its Dual-Targeted NK Cell Therapy Pipeline -...