Betibeglogene autotemcel (beti-cel), a one-time gene therapy, enabled durable transfusion independence in most patients with transfusion-dependent -thalassemia (TDT) who were treated across 4 clinical studies.

Of 60 patients enrolled overall, 17 of 22 (77%) treated in the 2 phase 1/2 studies were able to stop packed red blood cell transfusions. In the 2 phase 3 studies, which used a refined manufacturing process resulting in improved beti-cel characteristics, 89% (n = 31/35) of patients with at least 6 months of follow-up achieved transfusion independence for more than 6 months,1 reported Suradej Hongeng, MD, during the virtual 2021 Transplantation & Cellular Therapy Meetings.

The median follow-up after beti-cel infusion in the 4 studies has been 24.8 months (range, 1.1-71.8).

With up to 6 years of follow-up, 1-time beti-cel gene therapy enabled durable transfusion independence in the majority of patients, said Hongeng, from Ramathibodi Hospital of Mahidol University, in Bangkok, Thailand.

Patients who achieved transfusion independence experienced a 38% median reduction in liver iron concentration (LIC) from baseline to month 48. The median reduction in LIC was 59% in patients with a baseline LIC more than 15 mg/g dw. A total of 21 of 37 (57%) patients who achieved transfusion independence have stopped iron chelation for 6 months or longer, with a median duration of 18.5 months from stopping iron chelation to last follow-up.

Erythropoiesis as determined by soluble transferrin receptor level was also improved in transfusion-independent patients. Bone marrow biopsies showed improvement in the myeloid:erythroid ratio.

Beti-cel adds functional copies of a modified form of the -globin (A-T87Q-globin) gene into a patients own hematopoietic stem cells (HSCs) through transduction of autologous CD34+ cells using a BB305 lentiviral vector. Following single-agent busulfan myeloablative conditioning, beti-cel is infused, after which the transduced HSCs engraft and reconstitute red blood cells containing functional adult hemoglobin derived from the gene therapy.

Of the 60 patients treated, 43 were genotype non-/ and 17 were / . The median age at consent was 20 years in the phase 1/2 trials and 15 years in the phase 3 trials. Median LIC at baseline was 7.1 and 5.5 mg Fe/g dw, respectively, and median cardiac T2 was 34 and 37 msec, respectively. The vector copy number was 0.8 in the phase 1/2 trial and 3.0 in the phase 3 study. Additionally, 32t and 78t CD34+ cells were transduced, respectively.

The phase 1/2 studies showed promising results but lower achievement of transfusion independence in patients with the / genotype, leading to a refinement in the manufacturing process, which resulted in a higher number of transduced cells and a higher number of vector copy number, said Hongeng.

The median time to neutrophil engraftment was 22.5 days and the median time to platelet engraftment was 44 days. Lymphocyte subsets were generally within the normal range after beti-cel infusion, which is different from allogeneic stem cell [transplantation], which is probably around 6 months to a year to get complete recovery of immune reconstitution, he said. The median duration of hospitalization was 42 days.

All patients were alive at the last follow-up (March 3, 2020). Eleven of 60 (18%) of patients experienced at least 1 adverse event (AE) considered related or possibly related to beti-cel, the most common being abdominal pain (8%) and thrombocytopenia (5%). Serious AEs were those expected after myeloablative conditioning: veno-occlusive liver disease (8%), neutropenia (5%), pyrexia (5%), thrombocytopenia (5%), and appendicitis, febrile neutropenia, major depression, and stomatitis (3% each).

Of the 7 patients experiencing veno-occlusive liver disease, 3 were of grade 4 and 2 were of grade 3. Two other patients had grade 2 veno-occlusive disease. There were no cases of insertional oncogenesis.

Persistent vector-positive hematopoietic cells and durable HbaT87Q levels supported stable total hemoglobin over time. In phase 3 trials, the median peripheral blood vector copy number was 1.2 c/dg at month 12 and 2.0 c/dg at month 24, and the median total hemoglobin was 11.5 g/dL at month 12 and 12.9 g/dL at month 24.

The weighted average of hemoglobin during transfusion independence in the phase 1/2 trials was 10.4 g/dL, and patients were transfusion-independent for a median of 51.2 months. In the phase 3 studies, the weighted average of hemoglobin during transfusion independence was 11.9 g/dL, and patients were transfusion-independent for a medium 17.7 months.

Hongeng S, Thompson AA, Kwiatkowski JL, et al. Efficacy and safety of betibeglogene autotemcel (beti-cel; LentiGlobin for -thalassemia) gene therapy in 60 patients with transfusion-dependent -thalassemia (TDT) followed for up to 6 years post-infusion. Presented at: 2021 Transplantation & Cellular Therapy Meetings; February 8-12, 2021; virtual. Abstract 1.

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Beti-Cel Gene Therapy Frees Patients With Beta-Thalassemia From Red Blood Cell Transfusions - OncLive

Cardiac Stem Cells Comments Off on Beti-Cel Gene Therapy Frees Patients With Beta-Thalassemia From Red Blood Cell Transfusions – OncLive | February 17th, 2021

New smart stem cells show a promising power to heal.

Researchers have reprogrammed human fat cells into adaptive smart stem cells that can lie dormant in the body until they are needed to heal various tissues. They demonstrated the cells' effectiveness at healing damaged tissue in a mouse study.

To create the smart stem cells, the team from UNSW Sydney exposed human fat cells to a compound mixture. After about three and a half weeks, the cells lost their original identity and began acting like stem cells, or iMS (induced multipotent stem cells).

"The stem cells acted like chameleons. They followed local cues to blend into the tissue that required healing."

"The stem cells we've developed can adapt to their surroundings and repair a range of damaged tissues," said UNSW hematologist John Pimanda, and co-author of the study, which they published in Science Advances.

"To my knowledge, no one has made an adaptive human multipotent stem cell before. This is uncharted territory."

Next, they injected the experimental iMS cells into healthy mice to see how the cells would respond. The cells remained dormant for some time, but they activated when the mouse was injured. Because of the cells' regenerative ability to act as "smart stem cells," they transformed themselves into whatever tissue was needed to heal the injured mouse --- like bone tissue, heart, or skin.

"The stem cells acted like chameleons," said Avani Yeola, lead author on the study at UNSW Medicine & Health. "They followed local cues to blend into the tissue that required healing."

All cells in a human body contain the same DNA. To differentiate between tissues, like a skin cell versus a bone cell, the cells only use a small portion of their total DNA. The rest of the DNA is shut down naturally by local modifications.

"The idea behind our approach was to reverse these modifications," said Pimanda. "We wanted the cells to have the option of using that part of the DNA if there was a signal from outside the cell."

Tissue-specific stem cells, like those that are restricted to becoming parts of the liver or lung, are limiting. But smart stem cells that can respond to their environment and become any tissue, like multipotent stem cells, will have many uses.

In the future, doctors could take a patient's fat cells, incubate them with the compound, and inject them into the patient to heal heart damage or trauma injuries.

But applications like this could be a long way off. The team needs to do much more research to prove this is safe in humans for different kinds of trauma before it becomes a real therapy.

We'd love to hear from you! If you have a comment about this article or if you have a tip for a future Freethink story, please email us at [emailprotected]

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Smart Stem Cells Made From Fat Have the Power to Heal - Freethink

Skin Stem Cells Comments Off on Smart Stem Cells Made From Fat Have the Power to Heal – Freethink | February 14th, 2021

Black patients in need of bone marrow or blood stem cell treatments have a decreased chance of matching with a donor. The Seattle branch hopes to change that.

Seattles Be The Match Collection Center opened up less than a year ago and is celebrating its 100th blood cell donation with an important message: More bone marrow donors of color are needed.

The nonprofit donation center is a part of the National Marrow Donor Program and increases the capacity to collect blood cells in the Pacific Northwest. Seattles Clinical Manager Hannah Erskine said this month is an important time to focus on the donation gap.

In the midst of Black History Month, its important to note that we frankly dont have enough Black and African American donors on the registry, said Erskin.

Only 4% of approximately 22 million donors on the registry are African American, lowering the chances that a Black patient can find a bone marrow donor who is a genetic match.

According to Be The Match data, the likelihood of finding a matched adult donor is only around 23% for an African American or Black patient, versus a 77% match rate for a white patient.

These matched bone marrow or blood stem cell transplants can help cure blood cancers like leukemia and lymphoma, as well as other blood conditions, such as sickle cell disease. Be The Match has coordinated more than 100,000 transplants.

Erskine said registering is a simple mouth swab that will be mailed to potential donors. They will be contacted if they are a match with a patient.

Being a matching blood stem cell donor can potentially save a life. The first step in changing the trend is to join the registry at http://www.bethematch.org.

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Be The Match encourages people of color to join bone marrow registry - KING5.com

Bone Marrow Stem Cells Comments Off on Be The Match encourages people of color to join bone marrow registry – KING5.com | February 14th, 2021

Leukemia is a type of cancer that affects the blood and bone marrow, where blood cells are formed. All types of leukemia cause rapid, uncontrolled growth of abnormal bone marrow and blood cells.

The main differences between the types include how fast the disease progresses and the types of cells it affects.

There are four main types of leukemia, which we describe in detail below:

Lymphocytic leukemia affects the lymphocytes, a type of white blood cell. Myeloid leukemia can affect the white blood cells, red blood cells, and platelets.

According to the National Cancer Institute, roughly 1.5% of people in the United States will receive a leukemia diagnosis at some point.

In this article, explore the four main types, their symptoms, the treatment options available, and the outlook.

The full name of this type of cancer is acute lymphocytic leukemia, and acute means that it grows quickly. Lymphocytic means that it forms in underdeveloped white blood cells called lymphocytes.

The disease starts in the bone marrow, which produces stem cells that develop into red and white blood cells and platelets.

In a healthy person, the bone marrow does not release these cells until they are fully developed. In someone with ALL, the bone marrow releases large quantities of underdeveloped white blood cells.

There are several subtypes of ALL, and the subtype may influence the best course of treatment and the prognosis.

One subtype is B-cell ALL. This begins in the B lymphocytes, and it is the most common form of ALL in children.

Another subtype is T-cell ALL. It can cause the thymus, a small organ at the front of the windpipe, to become enlarged, which can lead to breathing difficulties.

Overall, because ALL progresses quickly, swift medical intervention is key.

As research from 2020 acknowledges, healthcare providers still do not know what causes ALL. It may occur due to genetic factors or exposure to:

Although genetic factors may play a role, ALL is not a familial disease.

Learn more about ALL here.

ALL is the most common form of leukemia in children.

The risk of developing it is highest in children under 5 years old. The prevalence slowly rises again in adults over 50.

ALL symptoms can be nonspecific difficult to distinguish from those of other illnesses.

They may include:

In a person with AML, the bone marrow makes abnormal versions of platelets, red blood cells, and white blood cells called myeloblasts.

The full name of this disease is acute myeloid leukemia, and acute refers to the fact that it is fast-growing.

It forms in one of the following types of bone marrow cell:

Doctors classify AML by subtype, depending on:

AML can be difficult to treat and requires prompt medical attention.

Learn more about AML here.

The most common risk factor is myelodysplastic syndrome, a form of blood cancer that keeps the body from producing enough healthy blood cells.

Other factors that increase the risk of developing AML include:

Most people who develop AML are over 45. It is one of the most common types of leukemia in adults, though it is still rare, compared with other cancers.

It is also the second most common form of leukemia in children.

Symptoms of AML can vary and may include:

CLL is the most common form of leukemia among adults in the U.S. and other Western countries.

There are two types. One progresses slowly, and it causes the body to have high levels of characteristic lymphocytes, but only slightly low levels of healthy red blood cells, platelets, and neutrophils.

The other type progresses more quickly and causes a significant reduction in levels of all healthy blood cells.

In someone with CLL, the lymphocytes often look fully formed but are less able to fight infection than healthy white blood cells. The lymphocytes tend to build up very slowly, so a person might have CLL for a long time before experiencing symptoms.

Learn more about CLL here.

Genetic factors are the most likely cause. Others might include:

CLL is rare in children. It typically develops in adults aged 70 or over. However, it can affect people as young as 30.

CLL typically causes no early symptoms. When symptoms are present, they may include:

Also, 5090% of people with CLL have swollen lymph nodes.

CML is a slow-growing type of leukemia that develops in the bone marrow.

The full name of CML is chronic myeloid leukemia. As the American Cancer Society explain, a genetic change takes place in the early forms of the myeloid cells, and this eventually results in CML cells.

These leukemia cells then grow, divide, and enter the blood.

CML occurs due to a rearrangement of genetic material between the chromosomes 9 and 22.

This rearrangement fuses a part of the ABL1 gene from chromosome 9 with the BCR gene from chromosome 22, called the Philadelphia chromosome. The result of this fusion is called BCR-ABL1.

BCR-ABL1 produces a protein that promotes cell division and stops apoptosis, the process of cell death, which typically removes unneeded or damaged cells.

The cells keep dividing and do not self-destruct, resulting in an overproduction of abnormal cells and a lack of healthy blood cells.

This occurs during the persons lifetime and is not inherited.

CML typically affects adults. People aged 65 and older make up almost half of those who receive a CML diagnosis.

The symptoms of CML are unclear, but they may include:

The symptoms may vary, depending on the type of leukemia. Overall, a person should get in touch with a doctor if they experience:

Learn more about the symptoms of leukemia here.

Treatment for ALL typically involves three basic phases: induction, consolidation, and maintenance. We describe these in detail below.

Treatment for AML involves the first two phases. The induction phase may include treatment with the chemotherapy drugs cytarabine (Cytosar-U) and daunorubicin (Cerubidine) or idarubicin (Idamycin). The doctor may also recommend targeted drugs.

The goal of this phase is to kill the leukemia cells, causing the cancer to go into remission, using chemotherapy.

The doctor may recommend:

People having chemotherapy may need to see their doctors frequently and spend time in the hospital, due to the risk of serious infections and complications.

This phase of the treatment lasts for about 1 month.

Even if the treatment so far has led to remission, cancer cells may be hiding in the body, so more treatment is necessary.

The consolidation phase may involve taking high doses of chemotherapy. A doctor may also recommend targeted drugs or stem cell transplants.

This phase, consisting of ongoing chemotherapy treatments, usually lasts for 2 years.

Since CLL tends to progress slowly, and its treatment can have unpleasant side effects, some people with this condition go through a phase of watchful waiting before starting the treatment.

For a person with CML, the focus is often on providing the right treatment for the phase of the illness. To do this, a doctor considers how quickly the leukemia cells are building up and the extent of the symptoms. Stem cell transplants can be effective, but further treatment is necessary.

Overall, the initial treatment tends to include monoclonal antibodies, targeted drugs, and chemotherapy.

If the only concern is an enlarged spleen or swollen lymph nodes, the person may receive radiation or surgery.

If there are high numbers of CLL cells, the doctor may suggest leukapheresis, a treatment that lowers the persons blood count. This is only effective for a short time, but it allows the chemotherapy to start working.

For people with high-risk disease, doctors may recommend stem cell transplants.

A persons prognosis depends on the type of leukemia.

Learn more about survival rates for people with leukemia here.

About 8090% of adults with ALL experience complete remission for a while during treatment. And with treatment, most children recover from the disease.

Relapses are common in adults, so the overall cure rate is 40%. However, factors specific to each person play a role.

The older a person is when they receive an AML diagnosis, the more difficult it is to treat.

More than 25% of adults who achieve remission live for 3 years or more after treatment for AML.

A person may live for a long time with CLL.

Treatments can help keep the symptoms under control and prevent the disease from spreading. However, there is no cure.

Stem cell transplants can cure CML. However, this treatment is very invasive and is not suitable for most people with CML.

The United Kingdoms National Health Service estimate that 70% of males and 75% of females live for at least 5 years after receiving a CML diagnosis.

The earlier a person receives the diagnosis, the better their outlook.

Leukemia is a type of cancer that affects the blood and bone marrow. It can affect people of all ages.

There are four main types of leukemia. They differ based on how quickly they progress and the types of cells they affect.

Treatments for all types of leukemia continue to improve, helping people live longer and more fully with this condition.

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Types of leukemia: Prevalence, treatment options, and prognosis - Medical News Today

Bone Marrow Stem Cells Comments Off on Types of leukemia: Prevalence, treatment options, and prognosis – Medical News Today | February 14th, 2021

One-year-old Max Gardner was diagnosed with aplastic anaemia, in October 2020, a serious condition in which the bone marrow and stem cells do not produce enough blood cells.

After Max developed significant bruises and a rash over his body, parents, Connor Gardner and Rachel Nicholson, from Hebburn, were referred to South Tyneside District Hospital, where their brave little boy underwent tests.

Doctors initially believed that Max had an immune disorder but after he was admitted to the Royal Victoria Infirmary (RVI) further tests helped to diagnose him with aplastic anaemia.

The family was told that the condition could be fatal if not treated properly.

Doctors said Max needed to have a bone marrow transplant, which has the potential to cure him.

Dad Connor, 29, and mum Rachel, 27, were both tested to see if they would be a bone marrow match and the pair were overjoyed when Rachel was found to be a 9/10 match.

Max started chemotherapy on January 7 at the RVI and mum Rachel donated stem cells on January 13 at Newcastles Freeman Hospital.

The following day, January 14, Max underwent the transplant at the RVI.

The family is now waiting for the results of a Chimerism Test which will tell them for definite whether the stem cells have worked but signs are already looking positive.

Delighted dad, Connor, said: "His neutrophils [a type of white blood cell that protect us from infections] have been more than 0.50 for three days in a row, which means that he is essentially engrafted, which means that his body is accepting the transplant.

"So it is working, but we still have to wait for the test results."

Doctors say there is no doubt that it has worked with the way the numbers have gone up but they have to officially do it like that to make sure, Connor continued.

"But there is no reason why it shouldnt have [doctors] say.

"He has done really well to get to this stage, he has absolutely sailed through it, everyone is surprised with how well he has done.

This the best outcome we could have hoped for.

But it hasnt been plain sailing for the family, who have also had to face additional challenges during the treatment.

Parents Connor and Rachael initially were not allowed to visit Max at the same time due to Covid rules, however the hospital has now eased the restriction in their case.

The family also became sick with Norovirus in the run-up to the transplant, causing concern over whether it would have to be pushed back.

Thankfully, the transplant went ahead as planned and the family made a good recovery, although Max still needs help with his eating.

Max will now have to remain in hospital for a while longer as he recovers from the transplant.

Connor added: We can feel that we are nearly at the end of it.

"His neutrophils are the highest they have ever been since he became poorly so we feel like we are coming to the end.

The family are sharing Maxs journey to health on Instagram under the name @maxinamillionaajourney and hope his story will encourage people to sign up to the Anthony Nolan register to become a potential donor and help others like Max.

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Bone marrow transplant shows signs of curing brave little boy with one in a million condition - Shields Gazette

Bone Marrow Stem Cells Comments Off on Bone marrow transplant shows signs of curing brave little boy with one in a million condition – Shields Gazette | February 14th, 2021

The global stem cell therapy market is expected to witness a CAGR of 10.6% during the forecast period 2019-2024, and is also anticipated to reach USD 214.5 million by 2024. Growing awareness related to the therapeutic potency of stem cells, development of infrastructure related to stem cell banking and processing, development of advanced genome-based cell analysis techniques, and increasing private-public investment for the development of stem cell therapies are driving the growth of the stem cell therapy market.

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Supportive regulations to drive the growth of the stem cell therapy market

Supporting regulations across developing countries, increasing prevalence of chronic diseases, technological advancement in healthcare, cellular therapies are the major advancements in transforming healthcare and identification of new stem cell lines are also fueling the growth of the stem cell therapy market.

Diseases such as osteoarthritis, multiple sclerosis, heart failure, hearing loss and cerebral palsy are some of the diseases that could be treated using stem cell therapies. For instance, according to the WHO by 2050, it is estimated 900 million people will have disabling hearing loss. Moreover, 60 percent of childhood hearing loss is due to preventable causes.

Allogenic stem cell therapy market to hold the larger share in the market

There are two types of stem cell therapy, allogeneic and autologous. Of both, allogenic segment account for the larger share and is also predicted to grow at the faster rate in the coming years in the market due to its extensive therapeutic applications, increasing commercialization of allogeneic products, easy production scale-up process, and growing number of clinical trials related to allogeneic therapies.

The stem cell therapy market has been segmented by therapeutic application into gastrointestinal diseases, musculoskeletal disorders, surgeries, cardiovascular diseases, and wound and injuries. Musculoskeletal disorders category contributed the largest revenue in the market due to increasing prevalence of musculoskeletal disorders and bone & joint diseases, increasing availability of stem cell-based products for the treatment of musculoskeletal disorders, and growing patient preference for effective & early treatment strategies.

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The global stem cell therapy market has also been segmented by cell source into adipose tissue-derived mesenchymal stem cell, cord blood cells and bone marrow-derived mesenchymal stem cells. Of all the categories, the bone marrow-derived mesenchymal stem cells are increasingly being used for therapeutic applications.

North America offers huge opportunities for stem cell therapy industry players

The North American stem cell therapy market will remain the largest during the forecast period. The region is further predicted to observe the fastest growth during the forecast period in the global market owing to technological upgradation and large capital invested in the research and development activities. Moreover, increasing number of clinical trials to evaluate therapeutic potential of products, increasing prevalence of chronic diseases, the growing patient base for target diseases, growing public awareness related to the therapeutic potency of therapy, and increasing public-private funding & research grants for developing safe and effective stem cell therapy products are also supporting the growth of the North American stem cell therapy market.

Investing in research and development is the key strategy adopted by the market players

Major players in the industry are investing in the development of innovative and new products, which is strengthening their position in the stem cell therapy market. In February 2018, MEDIPOST announced that FDA has approved its stem cell-based Alzheimers disease drug, NEUROSTEM for clinical trials. Similarly, in March 2017, Osiris Therapeutics launched Prestige Lyotechnology, a method for storage of living cells and tissues.

Some of the key players operating in the stem cell therapy industry are Osiris Therapeutics, Inc., RTI Surgical, Inc., MEDIPOST Co., Ltd., Nuvasive, Inc., Pharmicell Co., Ltd., Holostem Terapie Avanzate Srl, JCR Pharmaceuticals Co., Ltd., Anterogen Co., Ltd., and Allosource.

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The global stem cell banking market is growing at a CAGR of 9.1% during the forecast period reaching USD 10.5 billion by 2024, due to the development of novel technologies of storage, preservation and processing. Stem cell banking is the method of accumulating cord blood, extorting and cryogenically freezing its stem cells for forthcoming use. Cord blood stem cells are used for treating blood diseases such as sickle cell disease, leukemia, and thalassemia. The global stem cell banking market is growing at a significant rate due to the development of novel technologies of storage, preservation and processing. The market has witnessed a high demand for placenta stem cells over the last few years, due to the increasing public awareness regarding the therapeutic prospective of stem cells.

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The global protein expression market was evaluated at USD 1,873.1 million in 2018. The protocol for expression of proteins makes use of expression vectors, competent cells, reagents, instrument, and services. The reagents are the estimated to hold the largest share due to large volume used in the bio-experiments. The significant growth in the protein expression industry is primarily due to the increasing funds from government and non-government organization for protein research, the soaring prevalence of chronic diseases, rising life science industry.

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U.S. Protein Expression Market Analysis and Forecast to 2024

The U.S. protein expression market is expected to grow at a CAGR of 11.6% during the forecast period with its market size predicted to reach USD 1.2 billion by 2024. The U.S. protein expression market is primarily driven by the factors such as the increasing prevalence of chronic diseases, increasing investment for recombinant protein expression, advancement in technology for expression systems, increasing geriatric population, and robust growth of the life sciences industry in the country. Prokaryotic expression systems and mammalian cell expression systems are the major contributors to the protein expression industry in the region.

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Global Stem Cell Therapy Market Set to Reach USD 214.5 Million by 2024 - The Courier

Bone Marrow Stem Cells Comments Off on Global Stem Cell Therapy Market Set to Reach USD 214.5 Million by 2024 – The Courier | February 14th, 2021

wA new study led by cancer researchers of Medical University of South Carolina (MUSC) found that a solitary strain of Bacteroides fragilis altogether diminished graft-versus-host disease (GVHD) by ensuring gut integrity.

The findings reported in JCI Insight shows that even though bone marrow transplant can be a lifesaving procedure for patients with blood cancers; however, GVHD is a potentially fatal side effect of transplantation, and it has limited treatment options. This proof-of-concept study demonstrates that better treatment options may be on the horizon for patients with GVHD.

Xue-Zhong Yu, M.D., associate director of Basic Science at Hollings Cancer Center, and lead author Hanief Sofi, Ph.D., realized that protecting the health of the gastrointestinal tract is a good target for reducing severe GVHD.

"If we can figure out how to keep a patient's intestinal tissue healthy before and after bone marrow transplant, then the patient's outcome will be much better. We know that restoring the microbiota diversity in the gut is an effective solution, but that comes with many challenges," said Yu.

Patients with blood cancers, such as leukemia, must undergo radiation and chemotherapy before they can get their new cancer-free immune system through bone marrow transplantation. The balance between the immune system and intestinal microbiota, communities of microorganisms that live in the gut, is especially important for proper intestinal health. Unfortunately, the radiation and chemotherapy radically throw off this balance, and the diversity of the microbiota is reduced 100- or even 1,000-fold. This leads to a condition called "leaky gut."

Clinical studies have shown that patients who recover microbiota diversity faster have better outcomes and less severe GVHD. Reduced microbiota diversity is associated with more severe GVHD.

Other studies have shown that fecal microbial transplantation (FMT) can be effective at reducing GVHD, but the challenge is how to get the right donor. Patients are heavily immune-deficient after bone marrow transplantation, and there is a great risk of bad infection if FMT is used in humans.

The Yu laboratory used two different strains of mice to establish a GVHD model that closely resembles the biology that occurs in humans after bone marrow transplantation. The mice developed acute GVHD. FMT significantly reduced acute GVHD in this model and reduced donor T cell proliferation in the organs, which is what triggers GVHD.

The researchers then used genetic sequencing to see which bacteria strains were most different between the fecal material of GVHD mice that received FMT and those that did not receive FMT.

Mice that had the best outcome, the lowest GVHD, had the highest levels of a bacteria called B. fragilis. Mice given this single bacterial strain had significantly reduced acute and chronic (long-term) GVHD compared to mice that did not get B. fragilis. In fact, B. fragilis alone was as good or even better than FMT.

Administration of B. fragilis increased overall gut microbial diversity, including increasing the amount of other beneficial bacteria strains. Surprisingly, GVHD was reduced in this model not only by live bacteria but also by bacteria that had been killed by short exposure to high heat.

The observation that B. fragilis was the main effective bacteria in the FMT process was not entirely new: B. fragilis also reduces autoimmunity in type 1 diabetes and colitis.

The current study by Yu and colleagues has two important findings. First, a molecule called polysaccharide A on the surface of B. fragilis appears to be critical for the GVHD-reducing functions of this bacteria. When the bacteria were modified to lack polysaccharide A, GVHD was not reduced compared to mice that did not receive any B. fragilis.

Secondly, the administration of B. fragilis did not reduce the graft-versus-leukemia or cancer-killing effect of the bone marrow transplantation, even though it did reduce donor T cell expansion in the gut. This is critical, since GVHD treatment options that reduce the graft-versus-leukemia effect would not be clinically significant.

"If this can be translated into the clinic, it would be a safer, easier and more effective treatment option," said Yu.

Further study in humans is needed to get this potential treatment into the clinic. Hematopoietic stem cells, given via bone marrow transplant, are classic immunotherapies for liquid tumors, but strategies to make the transplantation safer and more beneficial are sorely needed. Hollings Cancer Center researchers continue to search for the most effective therapies to improve patient outcomes and quality of life, he said.

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Ensuring gut integrity may improve results in blood cancer: Study - Hindustan Times

Bone Marrow Stem Cells Comments Off on Ensuring gut integrity may improve results in blood cancer: Study – Hindustan Times | February 14th, 2021

Gorgeous little Arlo McArthur looks the picture of health and happiness.

Loved and adored by his family this little lively three-year-old from Milngavie is spoiled rotten by his three big sisters and his ultimate day out is playing golf with his daddy.

But behind the cheeky grin lies a devastating truth - he's a "ticking-timebomb" and needs a stem cell transplant to save his life.

So today, we've joined with Arlo's mum Nicole, dad Ian and his three doting sisters Carys, Brooke and Holly in asking Glasgow Live readers to step up and help this brave little boy.

They need young men, between 16 and 30 to volunteer to be tested to see if they are a match for the toddler. There's not much to it, a simple swab test carried out at home is enough for the experts to determine if you're a match.

The more people who register to be tested the better chance there is of finding the ideal candidate willing to donate the bone marrow little Arlo desperately needs.

For this family your help could mean the difference between life and death.

They've lived with the knowledge since he was 10 weeks old that a rare genetic condition could rob their precious little boy of his future.

Diagnosed with Wiskott-Aldrich Syndrome, it means Arlo's immune system doesn't function properly and it's difficult for his bone marrow to produce platelets, making him prone to bleeding.

Its estimated there are between 1 and 10 cases per million males worldwide. Arlo was only the third case at Queen Elizabeth University Hospital.

Doctors say they cant take the risk with an older donor as he was lucky to survive a previous transplant which failed when he was a baby.

His back-up is his dad Ian, 31, but he's only a half-match.

Sadly little Arlo's story isn't unique, across the country 2,300 people a year need a stem cell transplant and charity Anthony Nolan coordinates the search and raises money to support their vital work.

Nicole, 37, dreams of seeing her little boy attend his first day at school next August and believes someone out there can help that dream come true.

She pleaded: "Were asking as many people as possible to register and help give Arlo the life he deserves.

"We want to love and enjoy having our little boy around for a long time. He should be able to live out his life of dreams.

"Put yourself in the shoes of a parent whose child is ill, or someone else who is about to lose a loved one. Youve just been told in a room that they wont make it without stem cells. How does it feel?

"Its not just our Arlo, there are plenty of Arlos out there who need your help."

"People don't realise how easy it is to do. It's not this big operation, just a few injections and a day at an out-patient clinic to save someone's life. I wish it was opt-out, like organ donation.

"We dont have much time but I know in my heart the right match is out there."

To find out how you can can join the register and help the fight to save little Arlo and others just like visit Anthony Nolan's website here.

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Arlo's Army needs stem cell donor as mum begs for help to save three-year-old's life - Glasgow Live

Bone Marrow Stem Cells Comments Off on Arlo’s Army needs stem cell donor as mum begs for help to save three-year-old’s life – Glasgow Live | February 14th, 2021

BY Ariana Shah

Despite Canyon Lakes Bernadette Mycroft and her little family not able to catch a break, the five of them wrap their arms around each other and refuse to give in to adversity. Adversity that just keeps piling on.

Bernadette, a popular kindergarten teacher at Tuscany Hills Elementary, is a single mom doing her best to effectively raise her four children, Bryan, Scarlette, James and Julliette. Bryan was diagnosed last year with Myelodysplastic Syndrome, a rare blood cancer. Hes been in and out of ICU for months and has been extremely close to death on multiple occasions.

The goal for months has been for Bryan, a high school senior, to be strong enough to undergo a life-saving stem-cell transplant surgery. That day finally arrived, against all odds, last week. He is currently undergoing chemotherapy in the bone marrow transplant unit at Radys Children Hospital in San Diego.

Bryan has spent most of the last couple of months in Rady Childrens Hospital in San Diego to treat a rare form of blood cancer. After much adversity, a major stem cell transplant of his sisters marrow occurred last week. A Go Fund Me campaign has been set up to help the family.

The battle to get to the transplant is a story of resilience, faith and a family determined to stick together despite blow after blow being dealt them.

Bryan has been battling. Bryan has been fighting for his life for many months in and out of ICU. The treatment Bryan underwent late last year resulted in him losing over 40 pounds. Then, in early December, Bryan and his entire family tested positive for COVID-19.

Bryans immune system was severely compromised, Bernadette said. But he was able to battle through it, despite having no white blood cells. Im so grateful for Dr. John Bradley, who administered Bryan with a COVID-19 antibody infusion that saved him. Bryan is so incredibly strong.

Bryan then had two more emergency surgeries.

The abscess that the chemo caused needed to be drained, Bernadette said. He cant heal from any more invasive surgeries due to his lack of white blood cells. What a nightmare. He just couldnt catch a break. Hes just so incredibly strong.

Everybody in the family recovered from COVID-19 and a weakened Bryan was able to come back home and stay with his family for a couple of weeks at Christmas. New Years Eve, though, found Bryan back in the hospital for more emergency surgery.

Bryans sister, Scarlette, was identified as a match and courageously agreed to give her brother this life-saving bone marrow transplant. On Feb. 1, Scarlette underwent a major operation to donate 8 million stem cells to be transplanted to her brother.

They have both been very courageous, brave and kind, Bernadette said. I could not be more proud of them. Little brother James and sister Juliette, too.

The stem cells were successfully transplanted into Bryan, and Scarlette was able to return home after a three-day stay in the hospital.

Its taken a toll on me to continuously drive back and forth from San Diego to Canyon Lake, Bernadette said. But, of course, Im doing it.

Bryan is currently on medication that prevents graft-versus-host disease. This procedure and aftercare will require him to be hospitalized for approximately four to six more weeks. Bernadette, meanwhile, has been on unpaid leave from Tuscany Hills Elementary for months.

Friends and the Canyon Lake Junior Womens Club are doing their best to take the family under their wing.

To say the Mycroft family is in crisis is an understatement, friend Tiffani Paul said as she set up a Go Fund Me account to help the family. Overcome with stress, worry, medical debt, loss of income and extraordinary expenses, the unbelievably proud and strong Ms. Mycroft has reluctantly allowed us to post this Go Fund Me on her familys behalf.

Those who wish to help the Mycroft family can donate to their Go Fund Me at https://www.gofundme.com/f/help-bernadette-mycroft-help-her-son-fight-cancer.

If there are any other approaches to help the Mycroft family, call or text Sonja of the Canyon Lake Junior Womens Club at 909-230-2702.

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Family's resilience heartwarming - The Friday Flyer

Bone Marrow Stem Cells Comments Off on Family’s resilience heartwarming – The Friday Flyer | February 14th, 2021

When Marie Fuesel was treated for leukemia eight years ago, she needed donated blood products more than 100 times.

Theyd give me my chemotherapy, Id stay in the hospital for a week, then Id go home, get really sick and have to come back in for blood and platelets, says Fuesel, 53, a retired insurance agent who lives in suburban Chicago. I spent over 100 days in the hospital over eight months. The disease and treatments (affect the bone marrow and production of red and white blood cells and platelets), so many transfusions were required to achieve remission.

After eight months of chemotherapy, followed by a year on the targeted drug Sprycel (dasatinib) as part of a clinical trial, Fuesel went into remission. She no longer needs transfusions, but she still appreciates the need for blood donors. I wouldnt be alive if the blood wasnt available when it was needed, she says.

Back then, blood shortages werent common, but they are now. The stay-at-home orders at the beginning of the COVID-19 pandemic forced the cancellation of numerous blood drives, and safety concerns arising from its spread have prompted some frequent donors to stay away from donation centers.

Thats been a source of worry for oncologists. Patients with cancer use nearly one-quarter of the nations blood supply, according to the American Red Cross, and donated blood is a vital resource in the treatment of hematologic cancers. Patients who receive stem cell transplants often need transfusions of oxygen-carrying red blood cells, infection-fighting white blood cells and platelets to control bleeding. Blood transfusions are common in the supportive care of patients undergoing chemotherapy that suppresses production of all the blood cells that results in anemia, because they relieve symptoms that ensue, such as fatigue and shortness of breath.

Between March and June 2020, 37,000 blood drives were canceled, according to the American Red Cross. The impact of the blood shortage varied across the nation but has hit some cities particularly hard. The New York Blood Center, for example, which supplies New York City

hospitals, reported in December 2020 that it had just three days of supply on hand, down from the five- to seven-day supply it normally has.

Ongoing shortages are forcing cancer centers to change some of their procedures for using donated blood. We all recognize that we are in the midst of a public health crisis and that we all have to do our part, says Dr. Mikkael Sekeres, chief of hematology at the University of Miami Miller School of Medicine and a physician liaison in hematology at Sylvester Comprehensive Cancer Center.

In response to COVID-related blood shortages, several cancer centers adjusted their policies for transfusing blood. Moffitt Cancer Center in Tampa, Florida, for example, developed a blood shortage action plan, according to Dr. Kaaron Benson, director of the blood bank at Moffitt. It basically meant dropping some of the thresholds we would normally use for transfusion, Benson says.

Moffitt has not needed to implement the plan yet, but if it does, Benson says, the change would most likely have the biggest effect on patients with leukemia and lymphoma who are given platelets as a preventive strategy. Provided theyre not bleeding or engaging in activities that increase the risk of bleeding, studies have shown you can allow the platelet threshold to drop from our standard of 10,000 per microliter to 5,000, she says.

The technique was first suggested in a 1991 journal article and has since been widely accepted as an appropriate change to make during blood shortages, Benson says.

In recent years, many oncologists have set lower thresholds for red blood cell transfusions another change that has eased the strain on blood supply. They used to routinely order transfusions for patients with hemoglobin levels below 10 grams per deciliter. That number dropped to between 7 and 8 grams per deciliter after a series of studies showed that infusing red blood cells at the higher threshold did not improve treatment outcomes.

During the pandemic, Moffitt and other cancer centers are also delaying some stem cell transplants and elective surgeries, so that blood used during those procedures can be kept on hand for patients who urgently need it, such as trauma patients or those needing emergent surgery. But those decisions are made on a case-by-case basis, so patients should maintain a frequent dialogue with their oncologists to determine the best plan for managing their symptoms during the pandemic.

Patients with multiple myeloma, for example, can benefit from stem cell transplants, but its usually not urgent, says Dr. Stephanie Lee, a hematologist and professor at the Fred Hutchinson Cancer Research Center in Seattle. We have very good treatments for multiple myeloma, so we can continue to give patients chemotherapy for weeks or months, Lee says.

However, she explains, patients with leukemia who need stem cell transplants may be advised to undergo the procedure as quickly as possible, even during the pandemic, because delaying it could cause the cancer to grow and become resistant to treatment.

And some patients with cancer who are simultaneously fighting other diseases should receive all the blood and platelet transfusions they need to manage their cancer, as well as to address any risks posed by chronic conditions. If you have heart disease, and your hemoglobin drops even further, youre more likely to get angina or suffer a heart attack, Sekeres says. So, for those people with serious comorbidities, we are more aggressive in transfusing blood products.

Growing the Donor Pool

Stephenie Perry, who works as the business operations coordinator for the American Red Cross of Northwest Georgia, knows firsthand the value of donated blood. Perry is a survivor of Hodgkin lymphoma who needed several transfusions during her treatment, which consisted of a round of chemotherapy and two stem cell transplants.

Perry, 31, has been in remission since February 2018, but sometimes her red blood cell count still runs low and she needs another blood transfusion. I feel sluggish, and when I stand up, I get really dizzy, says Perry, who lives in Rome, Georgia. When I get a transfusion, its like someone has just given me a shot of energy.

How can patients adapt when blood shortages mandate less frequent transfusions? Lifestyle changes can make a big difference, Sekeres says. If a patient is becoming progressively anemic, and its someone who usually goes for a 2-mile walk every day, maybe theyll reduce it to 1 mile or cut (exercise) altogether, he says.

Some patients may be eligible for iron infusions, which can relieve symptoms of fatigue and lengthen the period between infusions, says Abbey Fueger, clinical trial nurse navigator for the Leukemia & Lymphoma Society.

In addition, there are other small changes that can lessen the risk of anemia and improve symptoms. Some physicians are trying to limit blood draws for patients and recommending nutritional supplements that might help them feel better and lengthen the time between infusions, she says.

Meanwhile, an effort is underway to expand the pool of potential blood donors. In April, the Food and Drug Administration (FDA) addressed blood shortages brought on by COVID-19 by easing up on some of its restrictions on who can donate. For example, people who are at risk of contracting HIV, and those who have a recent tattoo or piercing or possible exposure to an infected individual no longer have to wait one year to give blood. The new waiting period is three months.

The FDA also dropped the waiting period for donors who have traveled to malaria-endemic countries from one year to three months. And it no longer recommends that blood centers turn away donors who lived in certain European countries during the era when Creutzfeldt-Jakob disease, a rare and fatal degenerative brain disorder, was thought to be spreading.

The hospital community is rallying around the cause, holding blood drives of their own and encouraging family members of patients to donate blood.

During the first few months of the pandemic, Fuesel helped put together five small blood drives in her town of Orland Park, Illinois. They were so successful the American Red Cross and a local news broadcaster asked her to help run the seventh annual Great Chicago Blood Drive. So, she did, and on Jan. 13, that event collected 330 units of blood at the Orland Park location and more than 2,000 units at other drives around the city.

For donors who might be nervous about giving blood during a pandemic, Fuesel has a message: Its safe and important. All the beds are spaced apart, and there are different stations when you walk in for getting your temperature checked and using hand sanitizer, Fuesel says. I know these are hard times, but it doesnt cost anything to give your blood. Its a way to help.

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Keep it Flowing: Combating COVID-19 Blood Shortages in Cancer Treatment - Curetoday.com

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Oslo, Norway, 12 February 2020

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Vistin Pharma ASA: Invitation to Q4 2020 conference call

Global News Feed Comments Off on Vistin Pharma ASA: Invitation to Q4 2020 conference call | February 14th, 2021

NEW YORK and MAINZ, GERMANY, February 12, 2021 — Pfizer Inc. (NYSE: PFE) and BioNTech SE (Nasdaq: BNTX) today announced that the U.S. government has exercised its option for an additional 100 million doses of the Pfizer-BioNTech COVID-19 vaccine. This brings the total number of doses to be supplied by the companies to the U.S. government to 300 million.

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Pfizer and BioNTech to Supply the United States with 100 Million Additional Doses of COVID-19 Vaccine

Global News Feed Comments Off on Pfizer and BioNTech to Supply the United States with 100 Million Additional Doses of COVID-19 Vaccine | February 14th, 2021

CAMBRIDGE, Mass., Feb. 12, 2021 (GLOBE NEWSWIRE) -- Vericel Corporation (NASDAQ:VCEL), a leader in advanced therapies for the sports medicine and severe burn care markets, today announced that its Board of Directors unanimously approved the termination of the Company’s shareholder rights agreement, commonly referred to as a “poison pill”, which was originally scheduled to expire on August 15, 2021. The shareholder rights agreement was amended to accelerate the expiration date to February 11, 2021, effectively terminating the plan as of that date.

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Vericel Announces Termination of Shareholder Rights Agreement

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JERSEY, Channel Islands, Feb. 12, 2021 (GLOBE NEWSWIRE) -- Quotient Limited (NASDAQ: QTNT), a commercial-stage diagnostics company (Quotient or the Company), headquartered in Eysins, Switzerland, today announced the Company will participate in the BTIG Virtual MedTech, Digital Health, Life Science & Diagnostic Tools Conference on Thursday, February 18.

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Quotient Limited Participate in the BTIG Virtual MedTech, Digital Health, Life Science & Diagnostic Tools Conference

Global News Feed Comments Off on Quotient Limited Participate in the BTIG Virtual MedTech, Digital Health, Life Science & Diagnostic Tools Conference | February 14th, 2021

TORONTO, Feb. 12, 2021 (GLOBE NEWSWIRE) -- Cronos Group Inc. (NASDAQ: CRON) (TSX: CRON) (“Cronos Group” or the “Company”) will hold its 2020 Fourth Quarter and Full-year Earnings Conference Call on Friday, February 26, 2021 at 8:30 a.m. EST. Cronos Group’s senior management team will discuss the Company’s financial results and will be available for questions from the investment community after prepared remarks.

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Cronos Group Inc. to Hold 2020 Fourth Quarter and Full-Year Earnings Conference Call on February 26, 2021

Global News Feed Comments Off on Cronos Group Inc. to Hold 2020 Fourth Quarter and Full-Year Earnings Conference Call on February 26, 2021 | February 14th, 2021

Conference Call and Webcast Scheduled for February 18, 2021, 4:30 pm ET Conference Call and Webcast Scheduled for February 18, 2021, 4:30 pm ET

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Oyster Point Pharma to Report Fourth Quarter and Full Year 2020 Financial Results on February 18, 2021

Global News Feed Comments Off on Oyster Point Pharma to Report Fourth Quarter and Full Year 2020 Financial Results on February 18, 2021 | February 14th, 2021

AUSTIN, TX & DURHAM, NC, Feb. 12, 2021 (GLOBE NEWSWIRE) -- Shattuck Labs, Inc. (Shattuck) (NASDAQ: STTK), a clinical-stage biotechnology company pioneering the development of bi-functional fusion proteins as a new class of biologic medicine for the treatment of patients with cancer and autoimmune disease, today announced it will participate in Citi’s 2021 Virtual Immuno-Oncology Day Conference being held virtually February 17-18, 2021.

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Shattuck Labs Announces Participation in Citi’s 2021 Virtual Immuno-Oncology Day

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TORONTO, Feb. 12, 2021 (GLOBE NEWSWIRE) -- Revive Therapeutics Ltd. (“Revive” or the “Company”) (CSE:RVV) (USA: RVVTF), a specialty life sciences company focused on the research and development of therapeutics for medical needs and rare disorders, is pleased to announce that it has closed its previously announced bought deal prospectus offering of 46,000,000 units (“Units”) at a price of $0.50 per Unit for aggregate gross proceeds of $23,000,000 (the “Offering”), which includes the exercise in full of the 15% over-allotment option. The syndicate of underwriters was led by Canaccord Genuity Corp. and Leede Jones Gable Inc. as the co-lead underwriters (together, the "Underwriters"). The Units were offered and sold by way of a short form prospectus filed with the securities commissions in each of the provinces of Canada, other than Québec.

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Revive Therapeutics Announces Closing of $23.0 Million Short Form Prospectus Offering

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Delisting of AS „Grindeks” shares from the Baltic Main list

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Delisting of AS „Grindeks” shares from the Baltic Main list

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Bagsværd, Denmark, 12 February 2021 — This document discloses the data of the transaction(s) made in Novo Nordisk shares by the company’s board members, executives and their associated persons in accordance with Article 19 of Regulation No. 596/2014 on market abuse.

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Trading in Novo Nordisk shares by board members, executives and associated persons on 11-12 February 2021

Global News Feed Comments Off on Trading in Novo Nordisk shares by board members, executives and associated persons on 11-12 February 2021 | February 14th, 2021