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 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

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

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

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

Bone Marrow Stem Cells Comments Off on Keep it Flowing: Combating COVID-19 Blood Shortages in Cancer Treatment – Curetoday.com | 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

Apamistamab (Iomab-B) conditioning treatment with targeted radioimmunotherapy to the bone marrow resulted in high rates of successful allogeneic hematopoietic stem cell transplants in patients with active, relapsed, or refractory acute myeloid leukemia (AML), according to interim results from the phase 3 SIERRA trial, which were presented virtually at the 2021 Transplant and Cellular Therapies Meetings.1

In these patients with relapsed or refractory AML, we observed high rates of allogeneic stem cell transplant with curative intent [in] 88% of patients on the Iomab-B arm, 18% of patients who were randomized to the conventional care arm achieved complete remission and received standard of care allo-transplant, and an overall rate of 79% of allo-transplant in all enrolled patients, Boglarka Gyurkocza, MD, said in a virtual presentation.

Investigators sought to prove with this study that targeted radiation to the marrow with apamistamab, a radioactive iodine (131I)labeled anti-CD45 antibody, could enable the successful engraftment of patients despite active disease in the marrow. Safety and robust efficacy had previously been demonstrated with the agent in 271 patients treated in 9 different phase 1 and 2 clinical trials.

The SIERRA trial is looking to enroll 150 patients, and the trial is already over 75% enrolled. Recently, an independent data monitoring committee recommended that the trial continue to the planned full enrollment based on a positive pre-planned ad-hoc analysis.2

In the study, patients with active, relapsed, refractory AML are randomized 1:1 to receive either apamistamab conditioning therapy and allogeneic HCT or conventional care. In the control arm, patients who do not achieve a complete remission (CR) by day 42 are allowed to cross over to receive Iomab-B, and those who do have a CR undergo HCT or receive standard-of-care therapy of the physicians choice.

Durable CR (dCR) rate is the primary end point of the study, characterized as complete response at 6 months after initial CR, and the secondary end point is overall survival (OS) rate at 1 year.

Patients are eligible for enrollment if they have marrow blast count 5% or the presence of peripheral blasts, age 55 years, a Karnofsky score 70, and related/unrelated donor matching at human leukocyte antigen (HLA)-A, HLA-B, HLA-C, and DRB-1. Active, relapsed, or refractory AML was defined for the sake of the trial as primary induction failure after 2 cycles of therapy including chemotherapy or 2 cycles of venetoclax (Venclexta) with a hypomethylating agent or low-dose cytarabine, first early relapse after first CR of less than 6 months, relapse refractory to salvage chemotherapy regimen, or second or subsequent relapse. Secondary or treatment-related AML was also allowed.

In the SIERRA trial, patient-specific dosimetry was used to generate an individualized therapeutic dose to target marrow and spare non-hematopoietic organs. Patients in the investigational arm received a dosimetric dose of apamistamab ( 20 mCi) approximately 19 days prior to HCT followed by a therapeutic dose of apamistamab, which is individually calculated for each patient based on an upper limit of 24 Gy to the liver. After, patients remain on radiation isolation for several days before receiving fludarabine conditioning therapy (30 mg/m2/day for 3 days) and finally low-dose total body irradiation (200 cGy) prior to HCT.

Among the first 75% of enrolled patients (n = 113), patients in the apamistamab arm (n = 56) had a median age of 63 years (range, 55-77), 35% had intermediate risk and 61% had adverse risk, the median

percent of marrow blasts at baseline was 29% (range, 4%-95%), and had received a median of 3 prior treatment regimens (range, 1-7). At randomization, 56% were in primary induction failure, 16% were in first early relapse, 15% had relapsed or refractory disease, and 13% were in their second or later relapse.

In the conventional care arm, the median age was 65 years (range, 55-77), 32% had intermediate risk and 63% had adverse risk, median marrow blasts was 20% (range, 5%-97%), and had received a median of 3 prior regimens (range, 1-6). At randomization, 49% were in primary induction failure, 21% were in first early relapse, 21% had relapsed or refractory disease, and 8.8% were in their second or later relapse. Patients who crossed over to receive apamistamab (n = 30) had similar baseline characteristics.

Forty-nine patients in the apamistamab-randomized arm were able to go on and undergo allogeneic HCT compared with 10 patients in the conventional care arm. In the investigational arm, a median of 646 mCi (range, 3541027) of apamistamab was infused at a dose of 14.7 Gy (range, 4.6-32) to the marrow. The median infused CD34-positive cell count was 5.6 x 106/Kg (range, 1.8-208). Forty-five patients received peripheral blood stem cells (PBSCs), 3 received marrow grafts, 17 had related donors, and 31 had unrelated.

Individualized therapy of Iomab-B provided myeloablative doses of radiation to the marrow, Gyurkocza, a medical oncologist at Memorial Sloan Kettering Cancer Center, commented.

These patients had a median of 30 days (range, 23-60) to HCT after randomization and 14 days (range, 9-22) to neutrophil engraftment, with no graft failure reported. Patients also had 18 days (range, 4-39) until platelet engraftment.

We also observed 100% neutrophil and platelet engraftment in patients who received Iomab-B conditioning, despite a heavy leukemia burden, Gyurkocza said.

In patients in the conventional arm who went on to HCT, conditioning regimens for HCT consisted of fludarabine/melphalan in 2, fludarabine/melphalan/total body irradiation in 1, busulfan/fludarabine in 1, cyclophosphamide/fludarabine/total body irradiation in 2, and 4 had no data on conditioning regimens available. Eight of these patients had PBSCs, 2 had marrow, 3 had related donors, 6 had unrelated, and 1 was unreported.

Median days to HCT was 67 (range, 52-104) with 17 days (range, 13-83) to neutrophil engraftment and 22 days (range, 8-35) to platelet engraftment. There was 1 graft failure.

Among the patients who crossed over to receive apamistamab before HCT, the median infused dose was 592 mCi (range, 313-1013) with 15.5 Gy (range, 6.3-42) to the marrow. The median infused CD34-positive cell count was 5.1 x 106/Kg (range, 1.8-16.1). Twenty-eight patients had PBSCs, 2 had marrow, 10 had related donors, and 20 had unrelated.

Patients had a median of 62 days (range, 36-100) to HCT, 14 days (range, 10-37) to neutrophil engraftment, and 19 days (range, 1-38) to platelet engraftment. No graft failure was reported in this group.

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Apamistamab Conditioning Treatment Induces High Rates of HCT Success in AML - OncLive

Bone Marrow Stem Cells Comments Off on Apamistamab Conditioning Treatment Induces High Rates of HCT Success in AML – OncLive | February 11th, 2021

REDWOOD CITY, Calif.--(BUSINESS WIRE)--Jasper Therapeutics, Inc., a biotechnology company focused on hematopoietic cell transplant therapies, today announced positive preliminary findings from its ongoing multicenter Phase 1 clinical trial of JSP191, a first-in-class anti-CD117 (stem cell factor receptor) monoclonal antibody, as a conditioning agent in older patients with myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML) undergoing hematopoietic (blood) cell transplantation.

Data from the first six patients who received a single dose of JSP191 prior to transplantation showed successful engraftment in all six patients. Complete donor myeloid chimerism (equal or greater than 95%) was observed in five of six evaluable patients at 28 days, and all three evaluable patients had total donor chimerism equal or greater than 95% observed at day 90. In addition, at 28 days, three of five evaluable patients showed complete eradication of measurable residual disease (MRD) as measured by next-generation sequencing. Two of the five evaluable patients showed substantial reductions in MRD. No treatment-related serious adverse events were reported.

The findings were presented by lead investigator Lori Muffly, M.D., M.S., Assistant Professor of Medicine (Blood and Bone Marrow Transplantation) at Stanford Medicine, as a late-breaking abstract at the 2021 Transplantation & Cellular Therapy (TCT) Meetings of the American Society for Transplantation and Cellular Therapy (ASTCT) and the Center for International Blood & Marrow Transplant Research (CIBMTR).

These early clinical results are the first to demonstrate that JSP191 administered in combination with a standard non-myeloablative regimen of low-dose radiation and fludarabine is well tolerated and can clear measurable residual disease in older adults with MDS or AML undergoing hematopoietic cell transplantation a patient population with historically few options, said Kevin N. Heller, M.D., Executive Vice President, Research and Development, of Jasper Therapeutics. These patients could be cured by hematopoietic cell transplantation, but the standard-of-care myeloablative conditioning regimens used today are highly toxic and associated with high rates of morbidity and mortality particularly in older adults. Traditional lower intensity transplant conditioning regimens are better tolerated in older adults, but are associated with higher rates of relapse in MDS/AML patients with measurable residual disease. JSP191, a well-tolerated biologic conditioning agent that targets and depletes both normal hematopoietic stem cells and those that initiate MDS and AML, has the potential to be a curative option for these patients.

The open-label, multicenter Phase 1 study (JSP-CP-003) is evaluating the safety, tolerability and efficacy of adding JSP191 to the standard conditioning regimen of low-dose radiation and fludarabine among patients age 65 to 74 years with MDS or AML undergoing hematopoietic cell transplantation. Patients were ineligible for full myeloablative conditioning. The primary outcome measure of the study is the safety and tolerability of JSP191 as a conditioning regimen up to one year following a donor cell transplant.

We designed JSP191 to be given as outpatient conditioning and to have both the efficacy and safety profile required for use in newborn patients and older patients for successful outcomes, said Wendy Pang, M.D., Ph.D. Executive Director, Research and Translational Medicine, of Jasper Therapeutics. We are enthusiastic about the reduction of measurable residual disease seen in these patients, especially given that it is associated with improved relapse-free survival. We are excited to continue our research in MDS/AML, with plans for an expanded study. We are evaluating JSP191, the only antibody of its kind, in two ongoing clinical studies and are encouraged by the positive clinical data seen to date.

About MDS and AML

Myelodysplastic syndromes (MDS) are a group of disorders in which immature blood-forming cells in the bone marrow become abnormal and do not make new blood cells or make defective blood cells, leading to low numbers of normal blood cells, especially red blood cells.1 In about one in three patients, MDS can progress to acute myeloid leukemia (AML), a rapidly progressing cancer of the bone marrow cells.1 Both are diseases of the elderly with high mortality. Each year, about 5,000 patients with MDS and 8,000 people with AML in the G7 countries receive hematopoietic cell transplants. These transplants are curative but are underused due to the toxicity of the current high-intensity conditioning regimen, which includes the chemotherapy agents busulfan and fludarabine.

About JSP191

JSP191 (formerly AMG 191) is a first-in-class humanized monoclonal antibody in clinical development as a conditioning agent that clears hematopoietic stem cells from bone marrow. JSP191 binds to human CD117, a receptor for stem cell factor (SCF) that is expressed on the surface of hematopoietic stem and progenitor cells. The interaction of SCF and CD117 is required for stem cells to survive. JSP191 blocks SCF from binding to CD117 and disrupts critical survival signals, causing the stem cells to undergo cell death and creating an empty space in the bone marrow for donor or gene-corrected transplanted stem cells to engraft.

Preclinical studies have shown that JSP191 as a single agent safely depletes normal and diseased hematopoietic stem cells, including in animal models of SCID, myelodysplastic syndromes (MDS) and sickle cell disease (SCD). Treatment with JSP191 creates the space needed for transplanted normal donor or gene-corrected hematopoietic stem cells to successfully engraft in the host bone marrow. To date, JSP191 has been evaluated in more than 90 healthy volunteers and patients.

JSP191 is currently being evaluated in two separate clinical studies in hematopoietic cell transplantation. A Phase 1/2 dose-escalation and expansion trial is evaluating JSP191 as a sole conditioning agent to achieve donor stem cell engraftment in patients undergoing hematopoietic cell transplantation for severe combined immunodeficiency (SCID), which is potentially curable only by this type of treatment. Data presented at the 62nd American Society of Hematology (ASH) Annual Meeting showed that a single dose of JSP191 administered prior to stem cell transplantation in a 6-month-old infant was effective in establishing sustained donor chimerism followed by development of B, T and NK immune cells. No treatment-related adverse events were reported. A Phase 1 clinical study is evaluating JSP191 in combination with another low-intensity conditioning regimen in patients with MDS or AML undergoing hematopoietic cell transplantation. For more information about the design of these two ongoing clinical trials, visit http://www.clinicaltrials.gov (NCT02963064 and NCT04429191).

Additional studies are planned to advance JSP191 as a conditioning agent for patients with other rare and ultra-rare monogenic disorders and autoimmune diseases.

About Jasper Therapeutics

Jasper Therapeutics is a biotechnology company focused on the development of novel curative therapies based on the biology of the hematopoietic stem cell. The companys lead compound, JSP191, is in clinical development as a conditioning antibody that clears hematopoietic stem cells from bone marrow in patients undergoing a hematopoietic cell transplant. This first-in-class conditioning antibody is designed to enable safer and more effective curative hematopoietic cell transplants and gene therapies. For more information, please visit us at jaspertherapeutics.com.

1 https://www.cancer.org/cancer/myelodysplastic-syndrome/about/what-is-mds.html

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Jasper Therapeutics Announces Positive Data from Phase 1 Clinical Trial of JSP191 as Targeted Stem Cell Conditioning Agent in Patients with...

Bone Marrow Stem Cells Comments Off on Jasper Therapeutics Announces Positive Data from Phase 1 Clinical Trial of JSP191 as Targeted Stem Cell Conditioning Agent in Patients with… | February 11th, 2021

BOSTON--(BUSINESS WIRE)--Gamida Cell Ltd. (Nasdaq: GMDA), an advanced cell therapy company committed to cures for blood cancers and serious hematologic diseases, today announced the results of a Phase 3 clinical study of omidubicel presented in an oral session at the Transplantation & Cellular Therapy Meetings of the American Society of Transplantation and Cellular Therapy (ASTCT) and Center for International Blood & Marrow Transplant Research (CIBMTR), or the TCT Meetings. Omidubicel is an advanced cell therapy under development as a potential life-saving allogeneic hematopoietic stem cell transplant solution for patients with hematologic malignancies.

This clinical data set was from the international, multi-center, randomized Phase 3 study of omidubicel that was designed to evaluate the safety and efficacy of omidubicel in patients with high-risk hematologic malignancies undergoing a bone marrow transplant compared to a comparator group of patients who received a standard umbilical cord blood transplant. This is the first presentation of these data in a peer-reviewed conference. The full presentation is available on the Gamida Cell website.

The results of this global Phase 3 study of omidubicel in patients with hematologic malignancies show that omidubicel resulted in faster hematopoietic recovery, fewer bacterial and viral infections and fewer days in hospital, all of which are meaningful results and represent potentially important advancements in care when considering the patient experience following transplant, said Mitchell Horwitz, M.D., principal investigator and professor of medicine at the Duke Cancer Institute. The comparator, a transplant with umbilical cord blood, has been historically shown to result in low incidence of graft versus host disease (GvHD) in relation to other graft sources, and in this study, omidubicel demonstrated a GvHD profile similar to the comparator. Moreover, previous studies have shown that engraftment with omidubicel is durable, with some patients in the Phase 1/2 study receiving their transplant more than 10 years ago. The data presented at this meeting indicate that omidubicel has the potential to be considered a new standard of care for patients who are in need of stem cell transplantation but do not have access to a matched donor.

Details of Phase 3 Efficacy and Safety Results Shared at the TCT Meetings

Patient demographics including racial and ethnic diversity and baseline characteristics were well-balanced across the two study groups. The studys intent-to-treat analysis included 125 patients aged 1365 years with a median age of 41. Diseases included acute lymphoblastic leukemia, acute myelogenous leukemia, chronic myelogenous leukemia, myelodysplastic syndrome or lymphoma. Patients were enrolled at more than 30 clinical centers in the United States, Europe, Asia, and Latin America.

Gamida Cell previously reported in May 2020 that the study achieved its primary endpoint, showing that omidubicel demonstrated a statistically significant reduction in time to neutrophil engraftment, a measure of how quickly the stem cells a patient receives in a transplant are established and begin to make healthy new cells, and a key milestone in a patients recovery from a bone marrow transplant. The median time to neutrophil engraftment was 12 days for patients randomized to omidubicel compared to 22 days for the comparator group (p<0.001).

All three secondary endpoints demonstrated a statistically significant improvement among patients who were randomized to omidubicel in relation to patients randomized to the comparator group (intent-to-treat). Platelet engraftment was significantly accelerated with omidubicel, with 55 percent of patients randomized to omidubicel achieving platelet engraftment at day 42, compared to 35 percent for the comparator (p = 0.028). The rate of infection was significantly reduced for patients randomized to omidubicel, with the cumulative incidence of first grade 2 or grade 3 bacterial or invasive fungal infection for patients randomized to omidubicel of 37 percent, compared to 57 percent for the comparator (p = 0.027). Hospitalization in the first 100 days after transplant was also reduced in patients randomized to omidubicel, with a median number of days alive and out of hospital for patients randomized to omidubicel of 60.5 days, compared to 48.0 days for the comparator (p = 0.005). The details of these data were first reported in December 2020.

Previously unpublished data from the study relating to exploratory endpoints also support the clinical benefit demonstrated by the studys primary and secondary endpoints. There was no statistically significant difference between the two patient groups related to grade 3/4 acute GvHD (14 percent for omidubicel, 21 percent for the comparator) or all grades chronic GvHD at one year (35 percent for omidubicel, 29 percent for the comparator). Non-relapse mortality was shown to be 11 percent for patients randomized to omidubicel and 24 percent for patients randomized to the comparator (p=0.09).

These clinical data results will form the basis of a Biologics License Application (BLA) that Gamida Cell expects to submit to the U.S. Food and Drug Administration (FDA) in the second half of 2021.

We believe that omidubicel has the potential to transform the field of hematopoietic bone marrow transplant by expanding access to this potentially curative cell therapy treatment for thousands of patients who are in need of a transplant but lack access to a matched related donor, said Julian Adams, Ph.D., chief executive officer of Gamida Cell. Sharing the results of the Phase 3 study of omidubicel with the transplant community is a major moment for Gamida Cell, and we are forever grateful to the patients who participated in this study, their caregivers, and the work of the investigators and their teams.

About Omidubicel

Omidubicel is an advanced cell therapy under development as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant solution for patients with hematologic malignancies (blood cancers). In both Phase 1/2 and Phase 3 clinical studies (NCT01816230, NCT02730299), omidubicel demonstrated rapid and durable time to engraftment and was generally well tolerated.1,2 Omidubicel is also being evaluated in a Phase 1/2 clinical study in patients with severe aplastic anemia (NCT03173937). The aplastic anemia investigational new drug application is currently filed with the FDA under the brand name CordIn, which is the same investigational development candidate as omidubicel. For more information on clinical trials of omidubicel, please visit http://www.clinicaltrials.gov.

Omidubicel is an investigational therapy, and its safety and efficacy have not been established by the FDA or any other health authority.

About Gamida Cell

Gamida Cell is an advanced cell therapy company committed to cures for patients with blood cancers and serious blood diseases. We harness our cell expansion platform to create therapies with the potential to redefine standards of care in areas of serious medical need. For additional information, please visit http://www.gamida-cell.com or follow Gamida Cell on LinkedIn or Twitter at @GamidaCellTx.

Cautionary Note Regarding Forward Looking Statements

This press release contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995, including with respect to timing of anticipated regulatory submissions, which statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to the progress and expansion of Gamida Cells manufacturing capabilities and other commercialization efforts and clinical, scientific, regulatory and technical developments. In light of these risks and uncertainties, and other risks and uncertainties that are described in the Risk Factors section and other sections of Gamida Cells Annual Report on Form 20-F, filed with the Securities and Exchange Commission (SEC) on February 26, 2020, its Report on Form 6-K filed with the SEC on August 12, 2020, and other filings that Gamida Cell makes with the SEC from time to time (which are available at http://www.sec.gov), the events and circumstances discussed in such forward-looking statements may not occur, and Gamida Cells actual results could differ materially and adversely from those anticipated or implied thereby. Any forward-looking statements speak only as of the date of this press release and are based on information available to Gamida Cell as of the date of this release.

1 Horwitz M.E., Wease S., Blackwell B., Valcarcel D. et al. Phase I/II study of stem-cell transplantation using a single cord blood unit expanded ex vivo with nicotinamide. J Clin Oncol. 2019 Feb 10;37(5):367-374.

2 Gamida Cell press release, Gamida Cell Announces Positive Topline Data from Phase 3 Clinical Study of Omidubicel in Patients with High-Risk Hematologic Malignancies, issued May 12, 2020. Last accessed August 31, 2020.

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Gamida Cell Presents Efficacy and Safety Results of Phase 3 Study of Omidubicel in Patients with Hematologic Malignancies at the 2021 TCT Meetings of...

Bone Marrow Stem Cells Comments Off on Gamida Cell Presents Efficacy and Safety Results of Phase 3 Study of Omidubicel in Patients with Hematologic Malignancies at the 2021 TCT Meetings of… | February 11th, 2021

Gene therapy companies have been under pressure lately, but Orchard Therapeutics got a lift yesterday from promising early data with its mucopolysaccharidosis type I candidateOTL-203.

The company is seeking to supersede the current standard of care, enzyme-replacement therapy or bone marrow transplant. But other gene therapy contenders are not too far behind, notablyRegenxbio, which in December started a proof-of-concept study of its rival project, RGX-111.

Good IDUA

Both projects seek to deliver the -l-iduronidase (IDUA) gene, which is mutated in MPS-I, leading to a deficiency of the IDUA enzyme. This enzyme usually breaks down glycosaminoglycans (GAGs), so in MPS-I patients these build up, causing tissue and organ damage. Symptoms of MSP-I, also known as Hurler syndrome, include cognitive impairment and skeletal deformity; if left untreated, patients rarely survive beyond the age of 10.

And both OTL-203 and RGX-111 are designed as one-time therapies, whereas the current enzyme replacement, Biomarin/Sanofis Aldurazyme, is given intravenously once a week.

However, the gene therapy candidates go about restoring IDUA enzyme activity in different ways. OTL-203 uses hematopoietic stem cells taken from the patient, then genetically modified using a lentiviral vector to express the IDUA gene, before being reinfused.

RGX-111, meanwhile, uses an adeno-associated viral vector to deliver the gene directly to the brain, getting around a central problem with Aldurazyme, which cannot cross the blood-brain barrier.

Getting into the brain should not be a problem for OTL-203 either, Orchards head of medical affairs, Leslie Meltzer, told Evaluate Vantage. She explained that hematopoietic stem cells naturally cross the blood-brain barrier and, once in the CNS, differentiate into a microglial-like cell.

This claim appears to be supported by the latest data, which admittedly come in just a handful of subjects. The eight-patient phase I/II trial, presented at the World Symposium yesterday, found increases in the IDUA enzyme in patients blood and cerebrospinal fluid. There was also a decrease in GAGs in the CSF and urine.

Encouragingly, this activity appears to have translated into a clinical benefit: all eight patients showed stable cognitive scores and stable motor function versus baseline, as well as growth in the normal range for patients age.

Its a progressive disease, so youd expect these things to worsen over time, but the fact they continued to be stable is very promising, Ms Meltzer said.She admitted that the data were early, with only around a year of follow-up on most of the clinical endpoints.

Orchard plans to start a registrational study by the end of this year.Ms Meltzer would not give any details ondesign, saying this would be finalised after feedback from regulators.

Regenxbios proof-of-concept study of RGX-111 is due to complete in November, putting the project about a year behind OTL-203.

One candidate that will go no further is Sangamos SB-318. The company reported disappointing data with the in vivo zinc finger nuclease genome-editing project two years ago, and has since said it would focus on second-generation zinc finger projects.

Still, even two gene therapies might be too many for an ultra-rare disease like MPS-I, which affects just one in 100,000 people. Asked whether this market could support more than one gene therapy, Ms Meltzer said newborn screening recently implemented in countries including the UScould lead to a revision of that estimate.

But, as in other rare disorders that have attracted several gene therapy players, a battle over a limited patient pool could be shaping up.

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World Symposium Orchard leads the crop of Hurler syndrome hopefuls - Vantage

Bone Marrow Stem Cells Comments Off on World Symposium Orchard leads the crop of Hurler syndrome hopefuls – Vantage | February 11th, 2021

Global Hematopoietic Stem Cell Transplantation (HSCT) Report offers market size, share, overview, segmentation by types, application, countries, key manufactures, cost analysis, industrial chain, sourcing strategy, downstream buyers, marketing strategy analysis, distributors/traders, factors affecting market, forecast and other important information for key insight.

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As a result, all the businesses are revising their budgets to formulate new profit trajectory for the forthcoming years. Our thorough analysis of this industry space will enable you to come up with contingency plans and prepare you to manage market qualms.

The research document scrutinizes different segmentations to offer comprehensive insights about the growth opportunities in the market.

Major points summarized in Hematopoietic Stem Cell Transplantation (HSCT) market report:

Elucidating Hematopoietic Stem Cell Transplantation (HSCT) market segmentations:

Regional terrain:

Ask for Discount on Hematopoietic Stem Cell Transplantation (HSCT) Market Report at:https://www.marketstudyreport.com/check-for-discount/3147041?utm_source=algosonline.com&utm_medium=Ram

Product scope:

Application landscape:

Competitive hierarchy:

Important Highlights of the Report:

Industry Size & Forecast: Estimations on the global Hematopoietic Stem Cell Transplantation (HSCT) industry size on the basis of value and volume are provided in this part of the report

Segmental Analysis: The report has examined the high-growth segments including product type, application, and end-users

Future Prospects: Future opportunities are estimated to emerge in the industry

Geography-Wise Analysis: The authors of the report have studied the regions having growth potential to help companies plan their future investments

Study on Competitive Landscape: The industry experts have offered thorough information about the strategic tactics adopted by the industry participants.

Key questions answered in the report:

For More Details On this Report: https://www.marketstudyreport.com/reports/global-hematopoietic-stem-cell-transplantation-hsct-market-2021-by-company-regions-type-and-application-forecast-to-2026

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Original post:
Growth Factors of Hematopoietic Stem Cell Transplantation (HSCT) Market with Emerging Trends and Revenue Estimation By 2026 - AlgosOnline

Bone Marrow Stem Cells Comments Off on Growth Factors of Hematopoietic Stem Cell Transplantation (HSCT) Market with Emerging Trends and Revenue Estimation By 2026 – AlgosOnline | February 11th, 2021

Ibram X. Kendi: What I learned from cancer

This is where I find myself, at the threshold between an old familiar state and an unknown future. Cancer no longer lives in my blood, but it lives on in other ways, dominating my identity, my relationships, my work, and my thoughts. Im done with chemo but I still have my port, which my doctors are waiting to remove until Im further out of the woods. Im left with the question of how to repatriate myself to the kingdom of the well, and whether I ever fully can. No treatment protocols or discharge instructions can guide this part of my trajectory. The way forward is going to have to be my own.

My first foray into this new selfhood is learning how to drive. As I get more comfortable behind the wheel, a hazy idea begins to crystallize into a grand plan. I need to leave the familiar, to trust that I can navigate the world alone. I need to become my own caregiver. It took me a while to say I was a cancer patient. Its time for me to figure out who I am now. By the time I finally pass my drivers test, the next step is obvious: Im going to embark on a solo cross-country road trip.

Over the next few weeks, I pack all of my belongings into boxes, put the boxes into storage, and sublet my apartment. I cant afford to buy my own car but my friend Gideon generously offers the use of his old Subaru. Between the extra income from renting out my apartment and the $4,000 in my savings account, I should be able to make do. I plan to camp and crash on couches as often as possible, staying in only the occasional motel room. I scour Craigslist for secondhand camping gear and buy a portable propane gas stove, a subzero sleeping bag, a foam bedroll, and a tent. I pack all this, along with a crate of books, a first-aid kit, a camera, and a sack of kibble for my scruffy terrier mutt, Oscar, into the car. Before leaving, I go in for a last checkup with my oncologist.

My road trip will take me 15,000 miles across 33 states. It will last 100 days, the maximum amount of time my medical team has agreed to until my next follow-up appointment. As I turn the keys in the ignition and drive away from New York City, I realize that this is a rite of passage that I hope will bridge the distance between no longer and not yet.

Either my GPS is a liar or I am an erratic driver, but I always seem to take nearly twice as long as it predicts to get to where Im going. Take a right turn inrecalculating its robotic voice says condescendingly when I miss yet another exit. My next destination, Columbus, Ohio, will entail my longest drive yet. The GPS predicts that, if I follow its barrage of orders exactly as told, I will arrive in nine hours and 21 minutes. Unlikely.

Since hitting the road, Im on no ones clock but my own.

Two weeks earlier, when I first left home, I was so tense that I regularly had to remind myself to breathe. Each minute behind the wheel presented new and overwhelming scenarios: Do I have the right of way? What does a blinking red light mean? Was that an Egyptian hieroglyph on the traffic sign? Lane changes and merging onto the freeway had proved especially stressfulan existential guessing game of will I live or will I not. But with each day, I am feeling more confident, and it has been at least 72 hours since another driver has honked at me in anger or bewilderment.

Originally posted here:
I Survived Cancer, and Then I Needed to Remember How to Live - The Atlantic

Bone Marrow Stem Cells Comments Off on I Survived Cancer, and Then I Needed to Remember How to Live – The Atlantic | February 10th, 2021

Cultures with treated stem cells had a 50% higher stem cell survival rate than untreated cultures. Image by carolem41

Treating equine donor stem cells with a growth factor called TGF-2 may allow them to avoid tripping the immune response in recipients, according to new research.

The work carried out at North Carolina State University could simplify the stem cell treatment process for ligament and tendon injuries in horses, and may also have implications for human stem cell therapies.

Mesenchymal stem cell therapy is a promising avenue for treating musculoskeletal injuries, particularly tendon and ligament injuries, in horses.

Mesenchymal stem cells are adult stem cells found in bone marrow that act as repair directors, producing secretions that recruit healing-related paracrine factors to the site of injury.

Just as blood cells have types, depending upon which antigens are on the blood cells surface, mesenchymal stem cells have differing sets of major histocompatibility complex molecules, or MHCs, on their surfaces.

If the MHCs of donor and recipient arent a match, the donors stem cells cause an immune response. In organ transplants, MHCs are carefully matched to prevent rejection.

These treatments arent like a bone marrow transplant or an organ transplant, says Lauren Schnabel, associate professor of equine orthopedic surgery at the university and corresponding author of the study, reported in the journal Frontiers in Cell and Developmental Biology.

Since the mesenchymal stem cells are being used temporarily to treat localized injury, researchers once thought that they didnt need to be matched that they wouldnt cause an immune response. Unfortunately, that isnt the case.

Schnabel and Alix Berglund, a research scholar at the university and lead author of the paper, wanted to find a way to use mesenchymal stem cell therapy without the time, effort and additional cost of donor/recipient matching.

Since these cells dont have to be in the body as long as an organ does, hiding them from the immune system long enough for them to secrete their paracrine factors could be a way around donor/recipient matching, Berglund says. Downregulating expression of the MHC molecules could be one way to do this.

The researchers cultured stem cells and lymphocytes, or T cells, from eight horses, cross-pairing them in vitro so that the stem cells and lymphocytes had differing MHC haplotypes.

In one group, stem cells had been treated with transforming growth factor beta (TGF-2) prior to being added to the lymphocytes in the culture media; the other group was untreated. TGF-2 is a cell-signaling molecule produced by white blood cells that blocks immune responses.

Cultures with treated stem cells had a 50% higher stem cell survival rate than untreated cultures.

We use mesenchymal stem cells to treat musculoskeletal injuries particularly tendon injuries in horses very effectively, Schnabel says.

And while you can extract the secretions from the stem cells, you get better results with the cells themselves. Stem cells arent just a reservoir of secretions, theyre a communications hub that tells other cells what they should be doing. So finding a way to utilize these cells without stimulating immune response gives us better treatment options.

This is a promising pilot study, Berglund says. Our next steps will be to further explore the immune response in vivo, and to look at human cells in vitro, as this work has excellent potential to help humans with these injuries as well.

The research was supported by the National Institutes of Health and the Morris Animal Foundation. Research specialist Julie Long and statistician James Robertson, both with the university, also contributed to the work.

TGF-b2 Reduces the Cell-Mediated Immunogenicity of Equine MHC-Mismatched Bone Marrow-Derived Mesenchymal Stem Cells Without Altering Immunomodulatory PropertiesAlix K. Berglund, Julie M. Long, James B. Robertson, Lauren V. SchnabelCell Dev. Biol., 04 February 2021 https://doi.org/10.3389/fcell.2021.628382

The study, published under a Creative Commons License, can be read here.

Read more from the original source:
Researchers curb local immune response in horses receiving stem cell injury therapy - Horsetalk

Bone Marrow Stem Cells Comments Off on Researchers curb local immune response in horses receiving stem cell injury therapy – Horsetalk | February 7th, 2021

Cord blood collection process is the easiest and safest for both mother and the baby in normal and C-Section delivery. Cord blood is collected after the baby is born and the umbilical cord is clamped and cut. Read here to know more.

A baby's umbilical cord contains blood-forming stem cells which can rebuild the immune system

As a parent to be, there are multiple things on your mind to consider and plan before the little one arrives. Between scan appointments and packing hospital bags, you certainly are on the lookout for the best for your baby. Every mother is always anxious to give birth to a healthy baby and assure that it has a healthy future. When you want to give it a healthy future, you need to think about life saving benefits. One of the most important decision which is made at birth for the baby and its family is preserving its cord blood from umbilical cord. The umbilical cord, which connects the baby and the mother in womb has life-saving benefits.

After the birth of the baby, the blood left in the umbilical cord has life-saving cells that can potentially treat over 80 medical conditions pertaining to blood disorders. Cord Blood Banking is the procedure of safely collecting blood from umbilical cord and placenta and preserving it in a sterile environment, thereby ensuring access to stem cells for one's lifetime.

The stem cells which is preserved can be used for blood related disorders. In simple words, stem cells can act like the body's own repair kit and help the body heal from life threatening diseases.

With several advancements, community cord-blood banking is a frontier in medical practice that will help secure not just your baby's future but also your extended family's health. Despite its benefits, there remains many common misconceptions about cord blood banking. Dr Anjali Kumar, Director, Obstetrics and Gynecology, C K Birla Hospital, Gurgaon debunks the myths and facts about Cord Blood banking.

Cord blood can secure baby's future and also your extended family's healthPhoto Credit: iStock

Also read:Expert Reveals Common Skin Problems In Newborn Babies And How To Take Care Of Them

Fact: Cord blood collection process is the easiest and safest for both mother and the baby in normal and C-Section delivery. Cord blood is collected after the baby is born and the umbilical cord is clamped and cut. The cord blood that is collected is blood that would normally be discarded after birth, so collection doesn't affect your baby's blood supply during pregnancy or delivery.

Fact: Cord Blood extracted from baby's umbilical cord is rich and can be easily available for the family and the child if preserved at birth. Incase of bone marrow or other source of stem cells, the donor has to be registered and should be a match to the family. Cord blood may be accessed more quickly than stem cells from an adult donor who may have registered for donation years ago. The donor must be located, consented, tested and harvested. The extraction of stem cells through Bone Marrow can be painful as well.

Also read:Newborn Care During Winter Season: Here's A Complete Guide For Parents

Fact: According to The Indian Council of Medical Research (ICMR) guidelines commercial banking of Cord Tissue is not permitted to be collected and preserved.

Fact: A baby's umbilical cord contains blood-forming stem cells which be transplanted and rebuild the immune system and bone marrow by saving a patient life with a certain life-threatening condition such as leukemia, lymphoma or thalassemia. Infusion of these cells can also treat patients with inherited genetic disorders, bone marrow failure or inherited immune deficiency.

Fact: It is difficult to find a matching donor of Indian origin of the times hence its important to bank baby's umbilical cord. Incase even if a match is found, it can also be incredibly costly to obtain donor stem cells. The cost to obtain a unit of stem cells can be anywhere between 15-20 lakhs and upwards.

Also read:What Is The Best Time To Give Water To Your Newborn?

(Dr Anjali Kumar, Director, Obstetrics and Gynecology, C K Birla Hospital, Gurgaon)

Disclaimer: The opinions expressed within this article are the personal opinions of the author. NDTV is not responsible for the accuracy, completeness, suitability, or validity of any information on this article. All information is provided on an as-is basis. The information, facts or opinions appearing in the article do not reflect the views of NDTV and NDTV does not assume any responsibility or liability for the same.

Excerpt from:
Cord Blood Banking: Myths And Facts You Should Be Aware Of, As Per Expert - NDTV Doctor

Bone Marrow Stem Cells Comments Off on Cord Blood Banking: Myths And Facts You Should Be Aware Of, As Per Expert – NDTV Doctor | February 7th, 2021

Every year, about 10,000 people in the U.S. need a stem cell transplant but cant find a donor.

The intense medical procedure, which can help those with leukemia, lymphoma, sickle cell anemia and other blood diseases, can save lives but securing a donor can be like finding a needle in a haystack.

Be The Match is a nonprofit, national registry where people can sign up to donate their stem cells. More than 35 million people around the world have volunteered yet only a small percentage of those donors are Americans, and even the registry admits most Americans dont know it exists.

The mother of a 12-year-old boy with leukemia has set out to change that.

Mandy Goldman is a hairdresser who lives with her husband and four children outside Boston. She remembers the devastating day five years ago when doctors told her the chemotherapy they gave her son Mateo Goldman, 9 years old at the time, didnt work.

They told us that our only option of curing Mateo was a bone marrow transplant, she says, a risky procedure that often involves a host of complications. But they had no other choice, she says.

The family got to work on the monumental task finding Mateo Goldman a close enough match.

Linda Matchan first reported the Goldman familys experience for The Boston Globe. In her research, she found very, very few people had any awareness of the need for bone marrow and stem cells donors. The awareness campaign around the subject is severely lacking compared to other campaigns like the importance of donating blood, she says.

For example, there's a little boy right now in North Carolina named Thor Forte, who's 10 and has sickle cell disease. And he has been waiting for literally half his life, five years, for a donor to be available, Matchan says. He's a tough match, but they finally did find somebody. And then when the time came for the procedure, the person backed out. So two years later, the boy is still waiting.

Fortunately, quickly after finding out Mateo Goldman didnt match with anyone in his family, he was paired with a donor on the registry from Germany. Mandy Goldman says Laura Stterlin of Frankfurt was ready to go and donate, ultimately saving her son.

Mateo Goldman wrote Stterlin, whose name he did not know at the time, a thank you note reading: Dear Donor, thank you for giving me the bone marrow. You feel like youre already part of my family, he says.

And unlike usual Make-A-Wish requests, Mateo Goldman asked to meet Stterlin in person halfway across the world. The trip to Germany was planned for summer of 2020 but has since been canceled due to the pandemic.

In 2019 when she was reporting this story, Matchan had a trip planned to Germany. She ended up meeting Stterlin and hearing the story of how she became a donor. Stterlin said she was at a sporting event with her husband when she got hungry and went on the hunt for some grub.

Dear Donor, thank you for giving me the bone marrow. You feel like youre already part of my family.

Germany has a robust public service campaign to get citizens to donate bone marrow, Matchan says. So it came to no surprise to Stterlin when she came across a kiosk to sign up.

Just three months later, she got a call and an email from the registry saying that there is somebody in the United States for whom she could be a match and was asked if she would donate, Matchan says. A couple of days later, she went into the hospital and did the donation.

Stterlins stem cells then crossed the Atlantic Ocean, making their way to America during a snowstorm.

The cells started working in Mateo Goldman right away but not without some difficulties, Mandy Goldman says. He battled total body stiffness from graft-versus-host disease, a complication of the transplant.

But, you know, Matteo's an amazing kid, she says, so through it all, he was smiling and making the best of it, even though he was suffering for a lot of the time.

Two years later, in July of 2020, the cancer came back. But since Mateo Goldmans first transplant, the science had evolved greatly.

So much so that his older brother, Leo Goldman, became a candidate to donate his cells for the second stem cell transplant.

I didn't realize how I could get my brother's cells, Mateo Goldman, now 12 years old, says. Once that sank in, I felt that it would connect me and my brother more.

Right before Christmas last year, the family got extraordinary news: Mateo Goldman had zero cancer in his bone marrow, Mandy Goldman says.

Now the mom of four is on a mission to raise awareness on stem cell donations and share the story of how it saved her sons life.

The amazing feeling Leo got from being able to be the person who saved his brother's life is something he's going to carry with him forever, she says. And even Laura [Stterlin], she gave him three and a half years of his life that we get to spend with him. I just really want to educate people about how empowering it is to do something so incredible for somebody else.

When she started talking to others to raise awareness, she was shocked to discover how fearful people were in committing to be a donor.

If people could see the trauma these patients go through her son had a drain placed in his stomach, total body radiation, chemotherapy that left him head-to-toe in a skin-burning rash she says then maybe they wouldnt be scared to dedicate a small action for someone whose only cure is through a stem cell transplant.

Once people are educated about how much of a difference it makes, she says, then I feel like they would do it.

Click here to learn more about the Be The Match Registry.

Tinku Rayproduced and edited this interview for broadcast.Serena McMahonadapted it for the web.

See original here:
After Bone Marrow Donation Saves 9-Year-Old Boy With Cancer, Boston Mom Fights To Raise Awareness - Here And Now

Bone Marrow Stem Cells Comments Off on After Bone Marrow Donation Saves 9-Year-Old Boy With Cancer, Boston Mom Fights To Raise Awareness – Here And Now | February 6th, 2021

Leukemia is a type of cancer that affects the blood. The two most common types in children are acute lymphoblastic leukemia and acute myelogenous leukemia.

In a person with leukemia, blood cells are released into the bloodstream before they are fully formed, so there are fewer healthy blood cells in the body.

Below, we describe the types of childhood leukemia, the symptoms, and the treatments. We then look at when to contact a doctor, what questions to ask, and where to find support.

Childhood leukemia is the most common form of cancer in children. It affects up to 3,800 children under the age of 15 in the United States each year.

Leukemia occurs when bone marrow releases new blood cells into the bloodstream before they are fully mature.

These immature blood cells do not function as they should, and eventually, the number of immature cells overtakes the number of healthy ones.

Leukemia can affect red and white blood cells and platelets.

The bone marrow produces stem cells. A blood stem cell can become a myeloid stem cell or a lymphoid stem cell.

Lymphoid stem cells become white blood cells. Myeloid stem cells can become:

Leukemia is typically acute or chronic, and chronic types are rare in children. They can include chronic myeloid leukemia or chronic lymphocytic leukemia.

Most childhood leukemias are acute, meaning that they progress quickly and need treatment as soon as possible.

Acute lymphoblastic leukemia (ALL) is the most common type in children, accounting for 75% of childhood leukemia cases.

It affects cells called lymphocytes, a type of white blood cell.

In a person with ALL, the bone marrow releases a large number of underdeveloped white blood cells called blast cells. As the number of these increases, the number of red blood cells and platelets decreases.

There are two subtypes of ALL: B-cell and T-cell.

In most childhood cases of ALL, the cancer develops in the early forms of B-cells. The other type, T-cell ALL, typically affects older children.

Research from 2020 reports that the majority of people diagnosed with ALL are under 18 and typically between 2 and 10 years old.

The American Cancer Society report that children under 5 years old have the highest risk of developing ALL and that this risk slowly declines until a person reaches their mid-20s.

The outlook for ALL depends on the subtype, the persons age, and factors specific to each person.

Myeloid leukemias account for approximately 20% of childhood leukemia cases, and most myeloid leukemias are acute.

Acute myelogenous leukemia (AML) affects white blood cells other than the lymphocytes. It may also affect red blood cells and platelets.

AML can begin in:

Juvenile myelomonocytic leukemia (JMML) accounts for approximately 12% of leukemia cases in children.

This rare type is neither acute nor chronic. JMML begins in the myeloid cells, and it typically affects children younger than 2 years.

Symptoms can include:

The symptoms of leukemia may be nonspecific similar to those of other common childhood illnesses.

A doctor will ask how long the child has been experiencing the symptoms, which can include:

Children may experience specific symptoms depending on the type of blood cell that the leukemia is affecting.

A low number of red blood cells can cause:

A low number of healthy white blood cells can cause infections or a fever with no other sign of an infection.

A low platelet count can cause:

Various factors can increase a childs risk of leukemia, and most are not preventable.

The following genetic conditions can increase the risk of leukemia:

Also, having a sibling with leukemia may increase the risk of developing it.

These can include exposure to:

If a child has symptoms that might indicate leukemia, a doctor may perform or request:

A bone marrow aspiration involves using a syringe to take a liquid sample of bone marrow cells. The doctor may give the child a drug that allows them to sleep through this test.

During the diagnostic process, a person might ask:

The doctor may recommend a variety of treatments for childhood leukemia, and the best option depends on a range of factors specific to each person.

The treatment usually consists of two phases. The first aims to kill the leukemia cells in the childs bone marrow, and the second aims to prevent the cancer from coming back.

The child may need:

Before or during treatment, a person might ask the doctor:

Questions to ask after the treatment might include:

Children who have undergone leukemia treatments require follow-up care, as the treatments often cause late effects.

These can develop in anyone who has received treatment for cancer, and they may not arise for months or years after the treatment has ended.

Treatments that can cause late effects include:

These complications may affect:

The late effects that may come can also depend on the type of treatment and the form of leukemia.

Because many leukemia symptoms can also indicate other issues, it can be hard to know when to contact a doctor.

Overall, it is best to seek medical advice if a child shows symptoms or behaviors that are not normal for them.

If a child has received a leukemia diagnosis, the effects can extend to parents, other family members, caregivers, and friends.

A person can find support and additional resources from:

The following organizations based in the United Kingdom also provide support and guidance:

Childhood leukemia can affect mental health, as well as physical health.

Learn more about mental health resources here.

According to the American Cancer Society, most children with leukemia have no known risk factors. There is no way to prevent leukemia from developing.

Because there are very few lifestyle-related or environmental causes of childhood leukemia, it is very unlikely that a caregiver can do anything to help prevent the disease.

A childs outlook depends on the type of leukemia. It is important to keep in mind that current estimates do not take into account recent advances in technology and medicine.

For example, the most recent 5-year survival rate estimates reflect the experiences of children who received their diagnoses and treatments more than 5 years ago.

The American Cancer Society report that the 5-year survival rate for children with ALL is 90%. The same rate for children with AML is 6570%.

Childhood leukemia is typically acute, which means that it develops quickly. As a result, a person should contact a doctor if they notice any of the symptoms.

The most common type of childhood leukemia is ALL, representing 3 out of 4 leukemia cases in children.

Treatment may include a combination of chemotherapy, targeted drugs, immunotherapy, stem cell transplants, surgery, and radiation.

The prognosis depends on the type of leukemia and the childs age.

This diagnosis can affect mental as well as physical health, and the effects can extend to caregivers, family members, and friends. Many different resources are available for support.

View original post here:
Leukemia in children: Symptoms, causes, treatment, outlook, and more - Medical News Today

Bone Marrow Stem Cells Comments Off on Leukemia in children: Symptoms, causes, treatment, outlook, and more – Medical News Today | February 6th, 2021

Following stem cell transplant or treatment with CAR T-cell therapies, patients with hematologic malignancies and coronavirus disease 2019 (COVID-19) tend to have favorable outcomes, especially if they are diagnosed in complete remission (CR) and further out from their cell infusion, according to Miguel-Angel Perales, MD, underscoring that care should not be delayed despite the ongoing pandemic.

Delayed therapy results in patients with relapse or progression of disease who did not receive the intended cellular therapy; [weve seen this happen] in 34% of cases, Perales, chief of the Adult Bone Marrow Transplant Service at Memorial Sloan Kettering Cancer Center (MSKCC), said during a presentation delivered at the 2021 AACR Virtual Meeting on COVID-19 and Cancer.1 Given that we can avoid the risk of nosocomial transmission, I think this clearly indicates that we should be careful about how we manage these patients and not try to delay their care.

In his talk, Perales highlighted registry data detailing the impact of the pandemic on cellular treatment in patients with cancer, outcomes of patients who were infected with the virus and received hematopoietic cell transplantation, and the impact of virus-related delays in care.

Data reported to the ASH Research Collaborative COVID-19 Registry for Hematology, a global reference tool available to the public, showed that as of January 15, 2021, a total of 813 malignant and non-malignant cases of COVID-19 were reported, with just over 500 cases reported in the United States alone.2

When looking at cellular therapies received prior to a diagnosis with the virus, 10 patients had received CAR T-cell therapies (6 recovered, 4 died), 46 patients had undergone allogeneic stem cell transplantation (34 recovered, 7 died, 5 had unknown outcome), and the majority, or 78 patients, had undergone autologous stem cell transplantation (67 recovered, 7 died, 4 had unknown outcome).

An earlier analysis of data collected from this registry showed that among the first 250 patients for whom data were collected, the overall mortality rate was 28% (95% CI, 23%-34%).3 However, in patients with moderate to severe COVID-19 infection, the mortality rate was even higher, at 42% (95% CI, 34%-50%). This is a condition that has significantly impacted our patients with hematologic malignancies, noted Perales.

Another registry, of the Center for International Blood & Marrow Transplant Research (CIBMTR), requires the inclusion of outcomes of patients who have undergone transplantation or received CAR T cells.4 As of January 15, 2021, data for 1258 patients from 195 centers were reported to the registry and showed that 50.08% of patients had undergone allogeneic transplantation and 44.66% had undergone autologous transplantation. Only a small percentage of patients received cell therapy, according to Perales.

The age of patients at the time of infection ranged from less than 20 years to older than 70 years, with the majority of patients between the ages of 60 years and 69 years. When looking at infections by region, 29.35% of cases were reported in the Midwest, 23.44% were reported in the Northeast, and 22.73% were reported in the South. The majority of cases occurred within the first 2 years of their infusion. A total of 614 casesalmost half of all patientshad their infection resolve, while 58 experienced improvement; 187 patients had died.

In a subsequent paper, investigators examined risk factors associated with death from COVID-19 in recipients of allogeneic transplantation based on data from the CIBTR registry.5 Results from the multivariate analysis showed that age greater than 50 years (P = .016), male gender (P = .006), and COVID-19 infection in less than 12 months following transplantation (P = .019) were all significantly associated with increased risk of death.

Interestingly, race and ethnicity were not significant in this series, noted Perales. Similarly, when we look at patients [who have undergone] autologous transplant, the only factor that we saw was the diagnosis of lymphoma versus myeloma. Other factors were not significant.

In another analysis, investigators examined outcomes of patients following transplant who were infected with the virus at MSKCC. Of the first 77 patients diagnosed between March 15, 2020 and May 7, 2020, 37 had undergone autologous transplant, 35 had undergone allogeneic transplant, and 5 had received CAR T-cell therapy.6

The disease distribution was as expected, according to Perales. Thirty-eight percent of patients had plasma cell disease, 23% had acute leukemia, 23% had aggressive non-Hodgkin lymphoma (NHL), 5% had Hodgkin lymphoma, 4% had chronic myeloid leukemia, 4% had myelodysplastic syndrome, and 3% had indolent NHL.

When you look at day [of infection] post infusion, you see there was a significant range, said Perales. In fact, the number of patients were diagnosed with COVID-19 several months or even years after their cell therapy. These are the demographics of 77 patients, but this is representative of the patients that we transplant at our center.

Notably, 44% of patients did not have any comorbidities. Investigators also examined the home medications that patients were receiving at the time of their COVID-19 diagnosis. Here, 10 patients were receiving steroids, 18 were receiving immunomodulatory agents, 4 were receiving anticoagulation agents, and 14 were receiving immunosuppressive drugs.

Almost half, or 48%, of patients had mild COVID-19 infection, so they were not admitted to the hospital. Twenty-six percent of patients had moderate infection, and thus, were admitted to the hospital, while 22% had severe infection and were either admitted to the intensive care unit or died.

In that group, the majority of them actually had active malignancy, unlike the other 2 groups where the majority actually were in remission, said Perales. Patients who required high levels of oxygen [were often those who] had active malignancy.

Results from a univariate analysis looking at the predictors of disease severity revealed significant associations between the presence of comorbidities and infiltrates on imaging at the time of diagnosis. Overall, however, we were able to see favorable outcomes with patients after COVID-19 infection, said Perales. Two-thirds of patients actually had a resolution. We did see 14 deaths, which represented 18% of patients. This was 41% of patients who were admitted, but particularly those with an active malignancy.

Among patients who were admitted to the hospital but had a malignancy that was in remission, the mortality rate was 21%. This was due, in part, to the fact that in many cases, patients or their family members decided to forego aggressive medical care.

Additional data revealed that COVID-19 was linked with a drop in lymphocyte populations across the board, added Perales. Notably, lymphopenia with COVID-19 was not found to impair long-term immune reconstitution in patients who had undergone bone marrow transplant.

When looking at survival in patients after infection with COVID-19, overall outcomes were found to be favorable.

Investigators also examined the risk of nosocomial infections in patients who had undergone transplantation or received cellular treatment in light of the pandemic. They looked at a series of 44 cases.

In March 2020, 2 healthcare workers were exposed at MSKCC and 3 patients had documented COVID-19 infection. One patient was receiving treatment in the inpatient setting, but the patient did have frequent visits from family members, according to Perales. So, its unclear when or how the exposure occurred, Perales said. The patient ended up dying.

Two additional patients may have been exposed in the donor room while they were collecting the stem cell from the autologous transplant, added Perales. One patient eventually died from the virus.

Again, its unclear whether these patients were infected in the center or in the community, as COVID-19 was very prevalent at the time, said Perales. Importantly, we have not seen any additional cases of potential or definite COVID-19 nosocomial infection since March 2020 at our center.

When examining the impact of the pandemic on treatment delays, in March 2020, investigators started to prospectively collect data from patients whose transplant or cellular therapy was delayed as a result of the impact of the virus on resources at the hospital, particularly the capability of using intensive care unit beds.1

Results showed that 85 patients delayed treatment; of those patients, 29 have not received their intended cellular treatment. Sixteen were supposed to receive autologous transplant, 12 were supposed to undergo allogeneic transplant, and 1 was supposed to receive CAR T-cell therapy.

Of the 56 patients who eventually proceeded to treatment, 62% received autologous transplant, 67% received allogeneic transplant, and 86% received CAR T-cell therapy. The biggest reason for not proceeding to treatment with autologous transplant and CAR T-cell therapy was because they were deferred due to good disease control. Other reasons included was because of a new comorbidity (12%) or they died from the virus. The most prominent reason for not proceeding to allogeneic transplant during the pandemic was progression of disease (42%).

We conclude that patients who are recipients of allogeneic transplant, and particularly those with acute leukemia, as much as possible should proceed to their indicated therapy and not be delayed, concluded Perales.

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Perales Examines the Impact of COVID-19 on Recipients of Cellular Therapies for Cancer - OncLive

Bone Marrow Stem Cells Comments Off on Perales Examines the Impact of COVID-19 on Recipients of Cellular Therapies for Cancer – OncLive | February 6th, 2021

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A tech entrepreneur, who is determined to live for 180 years, claims his bizarre strategies will soon be as popular as cell phones. Dave Asprey firmly believes one of the keys to living longer is skipping breakfast. The American lifestyle guru has asserted that he can at least live to the year 2153 by using a variety of techniques, including a cold cryotherapy chamber sitting and intermittent-fasting. The 47-year-old millionaire devised the term 'Biohacking' to detail his methods of turning back the biological clock. In an effort to survive for as long, Dave has spent over $1,000,000 on hacks and techniques to improve the overall functioning of his body. He has parts of his bone marrow removed to re-inject his own stem cells back into his body.

Appearing on ITVs This Morning today, he joined host Holly Willoughby for a virtual conversation. Holly quizzed Dave why he wants to live so long, to which he replied that as a curious person, he feels there is a lot in the world that can be fixed and improved that he thinks he has not done yet. Dave confirmed that some of the things he wants to pioneer are expensive. However, there are other ways that are free of cost like fasting.

Dave also speculated that after applying his methods for longer life, people under 40 will be 'happy and highly functional' over a 100-years-old. He stated that he won't be the only one as his wife both is also racing to get to 180 years old. In comparison to others, Dave has set himself up for much less inflammation by controlling what he eats and how he sleeps, besides several other anti-ageing treatments. Explaining the benefits of reintroducing his own stem cells in his body, he said people heal when they are young. With age, the stem cells of the body get exhausted, so he opts for ways which gives him more stem cells.

Dave also follows cryotherapy, which is also known as cold therapy. It involves using low temperatures to treat a variety of tissue lesions. He is also taking cold showers for more than ten years. Dave uses another technique to live a long life known as intermittent fasting. This involves controlling the number of times that one eats meals to create periods of fasting over a certain period. According to Dave, it helps in disease prevention as fasting periods lets his body to 'repair itself' while not digesting food.

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US Man Who Wants to Live for 180 Years Re-injects His Own Stem Cells, Spends Rs 87 Lakh - News18

Bone Marrow Stem Cells Comments Off on US Man Who Wants to Live for 180 Years Re-injects His Own Stem Cells, Spends Rs 87 Lakh – News18 | February 6th, 2021