Stem Cell Clinic

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

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

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

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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Are Hiccups a Sign of the New Coronavirus? - Healthline

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