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A few days after my stem cell transplant this year, a young cleaner entered my hospital room to disinfect and swab. Broad faced and friendly, she saw me lying in bed reading a book.

Do you like reading, she asked? Well, I have the book for you. It is called Fifty Shades of Grey. Its porno!

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That last part was whispered behind a cupped hand, as she grinned and then giggled. For good measure, she also recommended the teen vampire series Twilight.

Once shed left I laughed out loud in a way I hadnt done for days, weeks in fact. When you have cancer, these moments are golden.

Over the last year I have spent months in hospitals, being infused with chemotherapy that laid me low and then undergoing a risky transplant of stem cells from a heroic unknown donor. During this long period of remission and recovery, I have valued every opportunity to smile, to breathe and to feel hope. Much of this sense of being fully alive has come from the kindness of others.

The transplant had made me feel very sick and there was a point at which I was terrified of dying. I asked the hospital staff for a spiritual adviser and the next day a Buddhist monk came to visit me. I didnt expect this, but his calm face and compassionate manner brought me peace. He read me poems for meditation, encouraged deep breathing, and assured me that all emotions in illness are human expressions of identity and not to be judged or feared. His gentleness was echoed two days later, when a nurse with the loveliest face I had ever seen knelt down next to my bed, held my hand, and reassured me I would be okay.

Day by day, my son, his girlfriend, and my husband encouraged and supported me, too, even when I could barely hold up my head or speak without tears. My 21-year-old son sat with me through many painful procedures, setting his phone to play Bachs Brandenburg Concertos, squeezing my hand, looking into my face, loving me and giving me strength I didnt think I had.

I was diagnosed with acute myeloid leukemia in February 2019; before that fateful month I was a modern German historian teaching university students on the Weimar Republic, Nazism and the Holocaust. There were days when I had wept and raged with my students over the historical accounts of Nazi inhumanity, barbarity and chilling callousness inflicted upon innocent civilians, especially the Jews. I have often questioned whether human nature is fundamentally selfish, violent and nasty. Right now, in this world of hateful populism and climate devastation, I ask these questions even more. But since I became sick, the kindness, indeed the goodness, of other people has been a constant companion to me. I have been overwhelmed by the extraordinary outpouring of support and concern from so many. Compassion, care, affection, hope all have been expressed to me by family, friends, students and colleagues. Blood drives were organized in my name, and students asked me if they could be tested as a possible bone-marrow donor. My sister (who hates medical procedures) underwent several tests to see if she could be a sibling transplant. One colleague even offered me the umbilical blood he had saved from his three children. (Ultimately the hospital found a donor from an international registry.)

Friends and family kept in touch or visited despite the long drives to the two hospitals where I received treatment. Two of my girlfriends texted me every day, sending love, inspiration and photos of flowers. From other well-wishers I received quilts and artwork and shawls, books and lotion and lip balm. I read notes and e-mails that told me I was not alone, that love surrounded me and would lift me up. Prayers were said for me in Protestant, Catholic, Unitarian, Muslim and Jewish places of worship. Students sent me good luck charms, including a chemo bear (it worked! I went into remission). Money was donated in a go-fund-me campaign to help with the costs of travel and accommodation to cancer centres. Strangers (friends of friends) offered their homes at the times when we couldnt find accommodation. Delicious meals were dropped off at my home or brought to the hospitals: lentil soups, macaroni and cheese, banana bread and smoothies, all preventing me from having to imbibe those horrible meal-replacement drinks or the cafeteria food. Cancer patients came to see me and shared their experiences and wisdom. A quietly stoic man in his 40s with Stage 4 colorectal cancer expressed hope in the advances in cancer treatment; another inspirational friend with breast cancer revealed she had undergone over 100 chemo treatments and still managed to propel her bike in the annual Ride to Conquer Cancer. Other leukemia patients in my wards became friends and sources of enormous support. My sister-in-law, a liver transplant survivor, understood my physical and emotional pain and talked me through several hard times. On the stranger than fiction level, old boyfriends and ex-friends reappeared, expressing their love and sending me cards or messages that brought tears to my eyes. At the same time high-school and university pals from my ancient past got in touch and told me to hang in there!

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I got through the worst days because of the superb doctors and nurses, the donor who gave her or his stem cells, and our excellent health-care system. But I also made it this far because I did not feel alone. I was constantly reminded that I am loved and that I have so much to live for. In the arduous world of my cancer treatment, the face of compassion has appeared so many times and in such beautiful ways that I now place much more faith in the goodness of human nature because I have seen that many of us will care for each other, especially in hard times.

I may not decide to read Fifty Shades of Grey, but I love that this young woman wanted to suggest something to make me forget the cancer and feel better. And, really, because of her and the support that surrounded me, I did.

Carolyn Kay lives in Peterborough, Ont.

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Im grateful for the kindness of strangers in my cancer recovery - The Globe and Mail

Bone Marrow Stem Cells Comments Off on Im grateful for the kindness of strangers in my cancer recovery – The Globe and Mail | November 15th, 2019

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A 25-year-old Redding, Connecticut woman meets the Arizona man who was battling deadly Acute Lymphoblastic Leukemia (ALL) until she saved his life by donating her bone marrow.

Jennie Bunce joined Gift of Life Marrow Registry through a sorority swab drive at North Carolinas High Point University in 2016. "I can remember being like 13 or 14 years old during some school bucket list project. On there was save a life and I got to cross it off so thats pretty cool."

Her life-saving match-- 33-year-old father of six from Mesa, Mark Roser. Roser learned he had ALL after breaking a hip and feeling increasingly weak in 2018.

He needed a bone marrow transplant to survive. He says, "When they discovered it, 94% of my blood cells basically contaminated, so I was really at the final deadline."

Gift of Life Marrow Registry matched the Jennie to Mark with months.

The pair met for the first time at Boca Oyster Bar in Bridgeport in October. Mark says, " I feel great. Im much more positive between work and family. My priorities have completely changed. Time with the kids, time with my wife, just being there for them instead of working so much... I treasure every moment with them now."

According to the gift of Life marrow registry website: "Blood cancer is an umbrella term for cancers that affect the blood, bone marrow and lymphatic system. In most blood cancers, normal blood cell development is interrupted by uncontrolled growth of abnormal blood cells. The abnormal blood cells can prevent blood from fighting off infection or preventing uncontrolled bleeding.

Unfortunately, blood cancer can strike any one of us at any time. Approximately every three minutes, a child or adult in the United States is diagnosed with a type of blood cancer. Thats 360 people a day, 130,000 people a year.

There are three main types of blood cancers: Leukemia, cancer that is found in your blood and bone marrow; Lymphoma, blood cancer that affects the lymphatic system; and Myeloma, blood cancer that specifically targets your plasma cells.

For many, there is hope of a cure through a bone marrow or peripheral blood stem cell transplant. Today, transplantation, of healthy stem cells donated by related and unrelated volunteers, offers hope to many patients suffering from these sometimes deadly diseases.

Advances in transplantation have made this procedure a reality for thousands who are alive today because a stranger gave them the Gift of Life!."

check out: https://www.giftoflife.org to learn more and even register for a swab kit and become a donor yourself.

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Redding woman donates bone marrow, saves life of a father - FOX61 Hartford

Bone Marrow Stem Cells Comments Off on Redding woman donates bone marrow, saves life of a father – FOX61 Hartford | November 15th, 2019

SHANGHAI and SUZHOU, China, Nov. 15, 2019 /PRNewswire/ --Gracell Biotechnologies Co., Ltd. ("Gracell"), a clinical-stage immune cell therapy company, today announced five presentations to be delivered at the upcoming American Society of Hematology (ASH) Annual Meeting in Orlando, Florida, held from December 7-10.

The presentations centre on Gracell's breakthrough FasTCARtechnology, and other two platform technology in four product categories used in the treatment of hematological malignancies, each with well-defined objectives, including:

The four product candidates are currently being studied in ongoing phase I clinical trials conducted by Gracell, Hebei Yanda Lu Daopei Hospital, and Xinqiao Hospital of AMU, and six other hospitals nationwide in China.

"These clinical studies demonstrated Gracell's product development strategy and strong capabilities to bring multiple novel therapies through clinical investigations," said Dr. William CAO, CEO of Gracell. "These invaluable data provides guidance for and enhance our confidence in pipeline selection."

Oral presentations:

A Feasibility and Safety Study of a New CD19-Directed Fast CAR-T Therapy for Refractory and Relapsed B cell Acute Lymphoblastic LeukemiaAbstract #825Session Name: 612. Acute Lymphoblastic Leukemia: Clinical Studies: Therapeutics StrategiesPresenter: Peihua Lu, M.D., Hebei Yanda Lu Daopei HospitalLocation: Orange County Convention Center, Tangerine 1 (WF1), Level 2Time: 5:00 pm, Monday, December 9, 2019https://ash.confex.com/ash/2019/webprogram/Paper121751.html

Anti-CD19/CD22 Dual CAR-T Therapy for Refractory and Relapsed B-Cell Acute Lymphoblastic LeukemiaAbstract #284Session Name: 612. Acute Lymphoblastic Leukemia: Clinical Studies: Novel TherapiesPresenter: Peihua Lu, M.D., Hebei Yanda Lu Daopei HospitalLocation: Orange County Convention Center, W224, Level 2Time: 4:15pm, Saturday, December 7, 2019https://ash.confex.com/ash/2019/webprogram/Paper126429.html

Poster presentations:

CD19-Directed Fast CART Therapy for Relapsed/Refractory Acute Lymphoblastic Leukemia: From Bench to BedsideAbstract #1340Session Name: 614. Acute Lymphoblastic Leukemia: Therapy, excluding Transplantation: Poster IPresenter: Cheng Zhang, M.D., Xinqiao Hospital of AMULocation: Orange County Convention Center, Hall B, Level 25:30-7:30 pm, Saturday, December 7, 2019https://ash.confex.com/ash/2019/webprogram/Paper128006.html

A Bcma and CD19 Bispecific CAR-T for Relapsed and Refractory Multiple MyelomaAbstract # 3147Session Name: 653. Myeloma: Therapy, excluding Transplantation: Poster IIPresenter: Hua Zhang, PhD., Gracell Biotechnology Ltd., Shanghai, China, Shanghai, ChinaLocation: Orange County Convention Center, Hall B, Level 26:00 PM-8:00 pm, Sunday, December 8, 2019https://ash.confex.com/ash/2019/webprogram/Paper131056.html

Role of Donor-Derived CD19.CAR-T Cells in Treating Patients That Relapsed after Allogeneic Hematopoietic Stem Cell TransplantationAbstract #4561Session Name: 723. Clinical Allogeneic and Autologous Transplantation: Late Complications and Approaches to Disease Recurrence: Poster IIIPresenter: Cheng Zhang, M.D., Xinqiao Hospital of AMULocation: Orange County Convention Center, Hall B, Level 26:00-8:00 pm, Monday, December 9, 2019https://ash.confex.com/ash/2019/webprogram/Paper128262.html

About FasT CAR-19

FasT CAR-19, or GC007F, is an investigational CD19-targeted CAR-T cell therapy for adolescent and adult patients with refractory or relapsed B-ALL, as well as aggressive non-Hodgkin lymphoma. Thanks to Gracell's patented FasTCAR technology, the bioprocessing time for GC007F has been significantly reduced from two weeks to 24 hours with substantially lower cost. The improved CAR-T cell fitness resulted in superior proliferation capabilities, potency, and extensive bone marrow migration making GC007F a potential best-in-class therapy for refractory or relapsed B-ALL.

About Dual CAR-19-22

Dual CAR-19-22, or GC022, is an investigational CAR-T cell therapy redirected to target CD19 and CD22, in treating patients with CD19+, or/and CD22+ relapsed/refractory B-ALL. A low toxicity with dose-dependent high CR rate including patients who previously treated with CD19 CAR-T cells were observed.

About Dual CAR-BCMA-19

Dual CAR-BCMA-19, or GC012, is an investigational CAR-T cell therapy redirected to target BCMA and CD19, in treating patients with BCMA+, or/and CD19+ relapsed/refractory multiple myeloma. Previous research shows CD19 could express on the myeloma progenitor cells, while BCMA is a well validated target for MM.

About Donor CAR-19

Donor CAR-19, or GC007G, is an investigational CD19 targeted CAR-T cell therapy manufactured in use of donor's lymphocytes. The objective of this study is to further investigate and better understand the safety and efficacy of donor derived CAR-T cells in treatment of relapsed and refractory B-ALL patients.

About B-ALL

B-ALL is a sub-type of acute lymphoblastic leukemia, although rare, is one of the most common forms of cancer in children between the ages of two and five and adults over the age of 50[1]. In 2015, ALL affected around 876,000 people globally and resulted in 110,000 deaths worldwide[2]. It is also the most common cause of cancer and death from cancer among children. ALL is typically treated initially with chemotherapy aimed at bringing about remission. This is then followed by further chemotherapy carried out over several years.

About MM

Myeloma begins when a plasma cell becomes abnormal. The abnormal cell divides to make copies of itself. These abnormal plasma cells are called myeloma cells. In time, myeloma cells collect in the bone marrow. They may damage the solid part of the bone. When myeloma cells collect in several of your bones, the disease is called "multiple myeloma." This disease may also harm other tissues and organs, such as the kidneys. Myeloma cells make antibodies called M proteins and other proteins. These proteins can collect in the blood, urine, and organs[3].

About Gracell

Gracell Biotechnologies Co., Ltd. ("Gracell") is a clinical-stage biopharma company, committed to developing highly reliable and affordable cell gene therapies for cancer. Gracell is dedicated to resolving the remaining challenges in CAR-T, such as high production costs, lengthy manufacturing process, lack of off-the-shelf products, and inefficacy against solid tumors. Led by a group of world-class scientists, Gracell is advancing FasTCAR, TruUCAR (off-the-shelf CAR), Dual CAR and Enhanced CAR-T cell therapies for leukemia, lymphoma, myeloma, and solid tumors.

CONTACT:

Linc HE Associate Director of Business Developmentsunwei.he@gracellbio.com

Dr. William Cao Founder, Chairman and CEOwilliam.cao@gracellbio.com

View original content to download multimedia:http://www.prnewswire.com/news-releases/gracell-announces-five-presentations-at-the-annual-meeting-of-american-society-of-hematology-ash-300958982.html

SOURCE Gracell

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Gracell Announces Five Presentations at the Annual Meeting of American Society of Hematology (ASH) - BioSpace

Bone Marrow Stem Cells Comments Off on Gracell Announces Five Presentations at the Annual Meeting of American Society of Hematology (ASH) – BioSpace | November 15th, 2019

A 6-year-old cocker spaniel in California that was recently diagnosed with cancer is slated to receive stem cells from her mother living 4,000 miles away in the United Kingdom.

Coco the cocker spaniel gave birth to a litter of puppies six years ago. One of those puppies, Millie, was adopted by Serena and Andrew Lodge, who now live in San Francisco. They may live across the world from each other, but the mother and daughter will soon be reunited for the rare treatment, reported South West News Service, or SWNS, a British news agency.

CHEAPER MEDICATION FOR DOGS WITH SEPARATION ANXIETY NOW APPROVED, FDA SAYS

Coco, left, and daughter, Millie. (SWNS)

The transplant will occur at the North Carolina State Veterinary Hospital in Raleigh. The facility isreportedly one of only a few animal hospitals in the world to offer the treatment, which involves taking healthy stem cells from Cocos bone marrow and injecting them intoMillies.

"Serena and Andrew started chemo on Millie three months ago but they've been told the only chance they'll have of curing her is if they find a positive donor so she can have a transplant, said Cocos owner, Robert Alcock, 52. He arrived with Coco in North Carolina on Wednesday.

Millie while in an animal hospital. (SWNS)

"They contacted us, and we sent some blood samples for testing, along with samples from one of Coco's other pups, he added. They both came back positive but because Coco is Millie's mother the vet said she would be a better match."

"Coco will go into hospital on Sunday for the procedure and then the cells will be donated on Monday, he continued, noting the Lodges have paid for everything.

Robert Alcock and his cocker spaniel, Coco. (SWNS)

BLACK LAB GIVES BIRTH TO 13 PUPPIES, SHOCKS OWNERS: 'THEY WERE JUST FLYING OUT'

Coco is expected to make a full recovery following the procedure. However, there is only a 50 percent chance Millie will be cured even if the treatment is successful, according to SWNS.

Stem cell therapy for pets is costly, typically setting an owner back between $2,000 and $3,000, according to Pet WebMD.

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Cocker spaniel with cancer to receive stem cells from mother living 4,000 miles away - Fox News

Bone Marrow Stem Cells Comments Off on Cocker spaniel with cancer to receive stem cells from mother living 4,000 miles away – Fox News | November 14th, 2019

Bone marrow is soft, spongy tissue within some bones, including those in the hips and thighs. People with certain blood-related conditions benefit from a transplant that replaces damaged cells with healthy cells, possibly from a donor.

Bone marrow transplants can be lifesaving for people with conditions such as lymphoma or leukemia, or when intensive cancer treatment has damaged blood cells.

This type of transplant can be an intensive procedure, and recovery can take a long time.

Here, we provide an overview of bone marrow transplants, including their uses, risks, and recovery.

Bone marrow contains stem cells. In healthy people, stem cells in bone marrow help create:

If a medical condition such as one that damages the blood or immune system prevents the body from creating healthy blood cells, a person may need a bone marrow transplant.

A person with any of the following conditions may be a candidate for a bone marrow transplant:

There are three types of bone marrow transplant, based on where the healthy bone marrow cells come from.

In many cases, the donor is a close family member, such as a sibling or parent. The medical name for this is an allogenic transplant.

Transplants are more likely to be effective if the donated stem cells have a similar genetic makeup to the person's own stem cells.

If a close family member is not available, the doctor will search a registry of donors to find the closest match. While an exact match is best, advances in transplant procedures are making it possible to use donors who are not an exact match.

In a procedure called an autologous transplant, the doctor will take healthy blood stem cells from the person being treated and replace these cells later, after removing any damaged cells in the sample.

In an umbilical cord transplant, also called a cord transplant, doctors use immature stem cells from the umbilical cord following a baby's birth. Unlike cells from an adult donor, the cells from an umbilical cord do not need to be as close a genetic match.

Before a bone marrow transplant, the doctor will run tests to determine the best type of procedure. They will then locate an appropriate donor, if necessary.

If they can use the person's own cells, they will collect the cells in advance and store them safely in a freezer until the transplant.

The person will then undergo other treatment, which may involve chemotherapy, radiation, or a combination of the two.

These procedures typically destroy bone marrow cells as well as cancer cells. Chemotherapy and radiation also suppress the immune system, helping to prevent it from rejecting a bone marrow transplant.

While preparing for the transplant, the person may need to stay in the hospital for 12 weeks. During this time, a healthcare professional will insert a small tube into one of the person's larger veins.

Through the tube, the person will receive medication that destroys any abnormal stem cells and weakens the immune system to prevent it from rejecting the healthy transplanted cells.

Before entering the hospital, it is a good idea to arrange:

A bone marrow transplant is not surgery. It is similar to a blood transfusion.

If a donor is involved, they will provide the stem cells well in advance of the procedure. If the transplant involves the person's own cells, the healthcare facility will keep the cells in storage.

The transplant typically takes place in several sessions over several days. Staggering the introduction of cells in this way gives them the best chance of integrating with the body.

The healthcare team may also use the tube to introduce liquids such as blood, nutrients, and medications to help fight infection or encourage the growth of bone marrow. The combination depends on the body's response to treatment.

The procedure will temporarily compromise the person's immune system, making them very susceptible to infection. Most hospitals have a dedicated, isolated space for people undergoing bone marrow transplants to help reduce their risk of infection.

After the last session, the doctor will continue to check the blood each day to determine how well the transplant has worked. They will test whether new cells are beginning to grow in bone marrow.

If a person's white blood cell count starts to rise, it indicates that the body is starting to create its own blood, indicating that the transplant has been successful.

The amount of time that it takes for the body to recover depends on:

Many other factors can affect recovery, including:

Some people are able to leave the hospital soon after the transplant, while others need to stay for several weeks or months.

The medical team will continue to monitor the person's recovery for up to 1 year. Some people find that effects of the transplant remain for life.

A bone marrow transplant is a major medical procedure. There is a high risk of complications during and after it.

The likelihood of developing complications depends on various factors, including:

Below are some of the more common complications that people who receive bone marrow transplants experience:

Some people die as a result of complications from bone marrow transplants.

A person who receives a bone marrow transplant may also experience reactions that can follow any medical procedure, including:

The body's response to a bone marrow transplant varies greatly from person to person. Factors such as age, overall health, and the reason for the transplant can all affect a person's long term outlook.

If a person receives a bone marrow transplant to treat cancer, their outlook depends, in part, on how far the cancer has spread. Cancer that has spread far from its origin, for example, responds less well to treatment.

According to the National Marrow Donor Program, the 1-year survival rate among people who have received transplants from unrelated donors increased from 42% to 60% over about the past 5 years.

A bone marrow transplant is a major medical procedure that requires preparation. This involves determining the best type of transplant, finding a donor, if necessary, and preparing for a lengthy hospital stay.

The time that it takes for the body to recover from a transplant varies, depending on factors such as a person's age and overall health and the reason for the transplant.

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Bone marrow transplant: What it is, uses, risks, and recovery - Medical News Today

Bone Marrow Stem Cells Comments Off on Bone marrow transplant: What it is, uses, risks, and recovery – Medical News Today | November 14th, 2019

ONE man and his dog are travelling to North Carolina this week, to provide a puppy with a pioneering stem cell transplant that could save her life.

Robert Alcock and his cocker spaniel, Coco, are making the journey so Coco, 7, can donate her stem cells to one of her own puppies, Millie, 6, who has cancer.

The experimental procedure is not yet available in the UK and can only be performed at one US hospital, the NC State Veterinary Hospital.

It involves using stem cells from the bone marrow of one dog and injecting them into the other.

Even if the operation is a success, there is only a 50 percent chance that Millie will be cured.

Millie was taken to the USA when her owners, Serena and Andrew Lodge, emigrated for work. After moving across the pond, Millie contracted cancer.

Millie the dog last week and (inset) before she became ill

Mr Alcock, who lives in Darwen, said the only way to help her is the transplant.

The 52-year-old catering manager said: Serena and Andrew started chemo on Millie three months ago but theyve been told the only chance theyll have of curing her is if they find a positive donor so she can have a transplant.

They contacted us, and we sent some blood samples for testing, along with samples from one of Cocos other pups.

They both came back positive but because Coco is Millies mother the vet said she would be a better match.

Mr and Mrs Lodge then asked Mr Alcock if he would fly to the USA with Coco so she could help save Millies life.

On Wednesday, Mr Alcock made the journey to North Carolina, to the only animal hospital in the States that can perform that kind of transplant on dogs.

Mr Alcock added: The Lodges have paid for everything, and I didnt like to ask how much the operation is costing but I think it will be in the thousands.

We will be in America for about a week.

Coco will go into hospital on Sunday for the procedure and then the cells will be donated on Monday.

Coco is expected to make a full recovery from the operation, but there is only a 50 per cent chance that Millie could be cured once the transplant has been completed.

Robert and Coco

Mr Alcock added: If it was a human then the chances of survival would be really good.

But this is a pioneering procedure, they havent done very many of these transplants before, so well have to wait and see what happens.

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Cocker Spaniel Coco goes to US to try and save her puppy's life - Lancaster and Morecambe Citizen

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NEW YORK, Nov. 14, 2019 (GLOBE NEWSWIRE) -- BrainStorm Cell Therapeutics, Inc. (NASDAQ:BCLI), a leading developer of adult stem cell therapies for neurodegenerative diseases, announced today that the Company has received a $495,330 grant from the National Multiple Sclerosis Society, through its Fast Forward program, to advance BrainStorms Phase 2 open-label, multicenter clinical trial of repeated intrathecal administration of NurOwn (autologous MSC-NTF cells) in participants with progressive Multiple Sclerosis (NCT03799718).

Chaim Lebovits, President and CEO of BrainStorm stated, We are very pleased to receive this generous grant from the National MS Society. Currently, we are conducting our Phase 2 study in three leading US medical centers: The Keck School of Medicine of USC, The Stanford School of Medicine, and Cleveland Clinic. This research funding will help advance our investigational therapy NurOwn as a potential unmet need for patients with progressive MS. MS continues to devastate the lives of patients and their families and we thank the National MS Society for helping us advance our innovative research program.

Currently, progressive MS treatment options are limited and NurOwn is a promising new autologous cellular treatment modality that has the potential to directly address MS disease pathways, said Ralph Kern MD MHSc, COO and CMO of BrainStorm. He added, This funding from the National MS Society will help us explore key neuroinflammation and neural repair biomarkers in progressive MS to confirm NurOwns unique mechanism of action and guide the design of future clinical trials to address this important unmet patient need.

Leveraging resources in this Phase 2 clinical study of a cell-based therapy for progressive MS exemplifies our work to accelerate research to improve clinical care for people living with MS. said Mark Allegretta, PhD, Vice President of Research at the National MS Society. Were pleased to work with BrainStorm to test a broad panel of biomarkers of neuroinflammation and repair as correlates of the effect of treatment with NurOwn.

About Multiple SclerosisMultiple sclerosis is an unpredictable, often disabling disease of the central nervous system. There is currently no cure for MS. Symptoms vary from person to person and range from numbness and tingling, to mobility challenges, blindness and paralysis. An estimated 1 million people live with MS in the United States. Most people are diagnosed between the ages of 20 and 50 and it affects women three times more than men.

About The National Multiple Sclerosis Society:The National MS Society, founded in 1946, funds cutting-edge research, drives change through advocacy, and provides programs and services to help people affected by MS live their best lives. Connect to learn more and get involved: nationalMSsociety.org, Facebook, Twitter, Instagram, YouTube or 1-800-344-4867.

About NurOwnNurOwn (autologous MSC-NTF) cells represent a promising investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors. Autologous MSC-NTF cells can effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. BrainStorm has fully enrolled a Phase 3 pivotal trial of autologous MSC-NTF cells for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm also recently received U.S. FDA acceptance to initiate a Phase 2 open-label multicenter trial in progressive MS and enrollment began in March 2019.

About BrainStorm Cell Therapeutics Inc.BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwn technology platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug status designation from the U.S. Food and Drug Administration (U.S. FDA) and the European Medicines Agency (EMA) in ALS. BrainStorm has fully enrolled a Phase 3 pivotal trial in ALS (NCT03280056), investigating repeat-administration of autologous MSC-NTF cells at six U.S. sites supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). The pivotal study is intended to support a filing for U.S. FDA approval of autologous MSC-NTF cells in ALS. BrainStorm also recently received U.S. FDA clearance to initiate a Phase 2 open-label multicenter trial in progressive Multiple Sclerosis. The Phase 2 study of autologous MSC-NTF cells in patients with progressive MS (NCT03799718) started enrollment in March 2019. For more information, visit the company's website at http://www.brainstorm-cell.com.

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Safe-Harbor Statement

Statements in this announcement other than historical data and information, including statements regarding future clinical trial enrollment and data, constitute "forward-looking statements" and involve risks and uncertainties that could causeBrainStorm Cell Therapeutics Inc.'sactual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may", "should", "would", "could", "will", "expect", "likely", "believe", "plan", "estimate", "predict", "potential", and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, BrainStorms need to raise additional capital, BrainStorms ability to continue as a going concern, regulatory approval of BrainStorms NurOwn treatment candidate, the success of BrainStorms product development programs and research, regulatory and personnel issues, development of a global market for our services, the ability to secure and maintain research institutions to conduct our clinical trials, the ability to generate significant revenue, the ability of BrainStorms NurOwn treatment candidate to achieve broad acceptance as a treatment option for ALS or other neurodegenerative diseases, BrainStorms ability to manufacture and commercialize the NurOwn treatment candidate, obtaining patents that provide meaningful protection, competition and market developments, BrainStorms ability to protect our intellectual property from infringement by third parties, heath reform legislation, demand for our services, currency exchange rates and product liability claims and litigation,; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available athttp://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.

CONTACTS

Corporate:Uri YablonkaChief Business OfficerBrainStorm Cell Therapeutics Inc.Phone: 646-666-3188uri@brainstorm-cell.com

Media:Sean LeousWestwicke/ICR PRPhone: +1.646.677.1839sean.leous@icrinc.com

Link:
BrainStorm Cell Therapeutics Announces Research Grant Award From the National Multiple Sclerosis Society - Yahoo Finance

Bone Marrow Stem Cells Comments Off on BrainStorm Cell Therapeutics Announces Research Grant Award From the National Multiple Sclerosis Society – Yahoo Finance | November 14th, 2019

Dongmei Yan,1,* Botao Tang,2,* Lixin Yan,3 Lei Zhang,1 Meijuan Miao,1 Xi Chen,4 Guangyi Sui,5 Qi Zhang,1 Daoyuan Liu,1 Hui Wang1

1Department of Blood Transfusion, The Second Affiliated Hospital of Harbin Medical University, Harbin, Peoples Republic of China; 2Department of Cardiology, Heilongjiang Red Cross Hospital, Harbin, Peoples Republic of China; 3Department of Laboratory Medicine, The Second Affiliated Hospital of Harbin Medical University, Harbin, Peoples Republic of China; 4Department of Hematology, The Second Affiliated Hospital of Harbin Medical University, Harbin, Peoples Republic of China; 5Ethics Committee, The Tumor Hospital Affiliated to Harbin Medical University, Harbin, Peoples Republic of China

*These authors contributed equally to this work

Correspondence: Hui WangDepartment of Blood Transfusion, The Second Affiliated Hospital, Harbin Medical University, Xuefu Road No. 246, Nangang District, Harbin, Heilongjiang Province, Peoples Republic of ChinaTel +86-451-86605134Email wanghui@hrbmu.edu.cn

Purpose: Sodium selenite (Na2SeO3) has been known to restore the antioxidant capacity of bone marrow mesenchymal stem cells (BMSCs), reduce the production of reactive oxygen species (ROS) in the cells, and promote cell proliferation and inhibit cell apoptosis. However, it is still not clear whether selenium can mediate the differentiation and inhibit the induced hemagglutination of BMSCs. In this study, we attempted to explore the effect of Na2SeO3 on these aspects of BMSCs.Methods: We evaluated the fate of the MSCs isolated from the bone marrow of mice by studying their differentiation and proliferation after treatment with Na2SeO3. We also simultaneously evaluated the coagulation reaction induced by Na2SeO3-treated BMSCs in vitro.Results: While the mice-derived BMSCs expressed CD44, CD73, CD90, and CD105, they did not express CD45. The morphology of the derived cells was homogeneously elongated. These results showed that the isolated cells are indeed BMSCs. We found that 0.1 M and 1 M of Na2SeO3 promoted the proliferation and apoptosis of BMSCs, respectively. This showed that Na2SeO3 can be toxic and exert certain side effects on the BMSCs. The results of the osteogenic and adipogenic assay showed that 0.1 M Na2SeO3 could significantly promote the osteogenic and adipogenic differentiation of BMSCs by upregulating the lipid factors (LPL and PPRAG) and osteogenic factors, RUNX2, COL1, and BGP, in a concentration-dependent manner. Coagulation experiments in animals (mice and rats) revealed that Na2SeO3 can reduce the coagulation time of BMSCs in a concentration-dependent manner, which is related to the high expression of hematopoietic factors (SDF-1, GM-CSF, IL-7, IL-8, IL-11, and SCF).Conclusion: Na2SeO3 promotes the proliferation and differentiation as well as reduces the coagulation time of BMSCs, and this effect might enhance the therapeutic effect of BMSCs.

Keywords: sodium selenite, BMSCs, proliferation, differentiation, coagulation factors, clotting time

This work is published and licensed by Dove Medical Press Limited. The full terms of this license are available at https://www.dovepress.com/terms.php and incorporate the Creative Commons Attribution - Non Commercial (unported, v3.0) License.By accessing the work you hereby accept the Terms. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. For permission for commercial use of this work, please see paragraphs 4.2 and 5 of our Terms.

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Sodium Selenite Improves The Therapeutic Effect Of BMSCs Via Promoting | OTT - Dove Medical Press

Bone Marrow Stem Cells Comments Off on Sodium Selenite Improves The Therapeutic Effect Of BMSCs Via Promoting | OTT – Dove Medical Press | November 13th, 2019

On Wednesday, Nov. 6, the Be the Match Club at Fairfield University hosted a donor registration drive to encourage students to sign up to be a bone marrow donor.

Be the Match is an organization that seeks to help people who are suffering from blood cancer or blood diseases and are in need of a transplant. Be the Match, operated by the National Marrow Donor Program, provides patients with a way to find a transplant match, which could be their last chance for a cure. There is a large number of diseases that could be treated with a transplant, including Hodgkin and non-Hodgkin lymphoma, different types of leukemia and severe aplastic anemia.

The Be the Match Club at Fairfield University started in the fall of 2018 when a group of studentswas inspired by a fellow Stag who had to withdraw from school when he was re-diagnosed with cancer. The clubs goal is to spread awareness about the organization and increase the database of donors, so there is a greater chance for a patient to find their match.

Were trying to get as many people as possible to sign up, Brian Gozzo 20, Vice President of the Be the Match Club, says. Currently were at 20 million. We hope to one day have basically the entire planet, ideally, on it.

A donor-patient match is found by having the donor swab the inside of their cheek to gather DNA that is tested against the patient. If the two have a similar human leukocyte antigen, they are a close match for a transplant. After signing up for the registry at Fairfield University, a cheek-swabbing kit will be sent to your house, and then will be sent back for the stem cells to be tested.

Its super simple, Gozzo says. All it takes is a cheek swab, and, like, five minutes of your time, and youre put on [the registry] till youre 61. If youre matched with someone right then, youll probably receive a phone call, and then that will take another couple weeks until you actually have to donate.

If a match is found, the actual donation process could go one of two ways. One way to donate is to give peripheral blood stem cells, which is a non-surgical procedure that extracts blood through a needle, puts it through a machine that separates the blood cells and then returns the remaining blood into the body. The other option for donation is to give bone marrow through a surgical procedure that removes liquid marrow from the pelvic bone.

Gozzo understands that this can sound scary, but the chances of getting a phone call is pretty slim. He says that only one in about four hundred people on the registry will ever have to donate.

One thing we want people to know is dont be scared that were gonna call you and say the next day you have to be here, across the country and donate, Gozzo said. Its a very lengthy process, theres a lot of people involved and its very safe.

For Gozzo, the most important thing is to spread awareness and increase the number of people on the registry and the chances of a life-saving donation. He says, You could just sit on the registry until you turn 61 and never once receive a phone call, but just know that, like, you still were there and youve still done your part.

Gozzo, a resident assistant, was motivated to form the Be the Match Club at Fairfield University with a few other RAs last year when the student had to withdraw. They reached out to Be the Match for help.

When the student had to withdraw, a couple of the RAs wanted to know what we could do to help. Could we find a match for this kid? Gozzo said. Course, thats very, very difficult.

However, last years drive was not the first appearance Be the Match has made at Fairfield University. Senior Julia Giampietro and her roommate brought a Be the Match drive themselves to Fairfield in their sophomore year. She reached out to the Connecticut Be the Match region leader, who helped them set up a drive that brought over 60 students to join the national bone marrow donor registry. Giampietro was influenced to raise awareness for this organization for a personal reason.

I wanted to bring [Be the Match] to Fairfield in honor of my cousin Christopher who passed away from AML Leukemia in October of my freshman year at Fairfield, Giampietro said via email. He went through a bone marrow transplant, was in remission and relapsed a year later. He received a second bone marrow transplant but the cancer took over his body He was and still is the biggest inspiration in my life and no matter what would always say its all good which is the motto my family and I live by now.

Giampietro continued work with Be the Match throughout her junior and senior years. She was also inspired by the Fairfield student who had to withdraw last year, so she worked with students in younger grades to put on another drive in the fall of 2018, which is when the Be the Match Club was born. Giampietro and her roommate decided to put together an event for the student at the Seagrape Cafe, where they raised almost $2,000 for his family from donations at the door and from other Fairfield Students.

That was probably the biggest accomplishment of our 3 years involved with Be the Match and was a great way to close the year and our time in the club, Giampietro said. It was also amazing to see how much support we got and the feeling of being able to make a small difference for a local peer and family.

Although her time with Be the Match at Fairfield University is over, Giampietro has high hopes for the club and the organization in the years to come.

My biggest hope really is to have students become more aware and educated about [Be the Match], Giampietro said. It is so important for people our age to be educated on this amazing cause because we are the ones who can save peoples lives.

Be the Match will hold their next donor registration drive in the spring of 2020. To learn more about the organization or become a donor, visit https://bethematch.org/.

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Be the Match and Make a Difference - Fairfield Mirror

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NEW YORK, Nov. 12, 2019 (GLOBE NEWSWIRE) -- BrainStorm Cell Therapeutics, Inc. (NASDAQ:BCLI), a leading developer of adult stem cell therapies for neurodegenerative diseases, today announced that the Companys Chief Operating and Chief Medical Officer Ralph Kern MD MHSc will present at the 7th International Stem Cell Meeting, which is hosted by the Israel Stem Cell Society. The Conference will be held November 12-13, in Tel Aviv, Israel.

Ralph Kern, MD, MHSc, said: I welcome the opportunity to participate in the 7th International Stem Cell Meeting where I will share the advances BrainStorm has made with NurOwn. It is a privilege to participate and to exchange ideas with many of the international scientific leaders in stem cell research.

About NurOwn

NurOwn (autologous MSC-NTF) cells represent a promising investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors. Autologous MSC-NTF cells can effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. BrainStorm has fully enrolled a Phase 3 pivotal trial of autologous MSC-NTF cells for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm also recently received U.S. FDA acceptance to initiate a Phase 2 open-label multicenter trial in progressive MS and enrollment began in March 2019.

About BrainStorm Cell Therapeutics Inc.

BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwn technology platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug status designation from the U.S. Food and Drug Administration (U.S. FDA) and the European Medicines Agency (EMA) in ALS. BrainStorm has fully enrolled a Phase 3 pivotal trial in ALS (NCT03280056), investigating repeat-administration of autologous MSC-NTF cells at six U.S. sites supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). The pivotal study is intended to support a filing for U.S. FDA approval of autologous MSC-NTF cells in ALS. BrainStorm also recently received U.S. FDA clearance to initiate a Phase 2 open-label multicenter trial in progressive Multiple Sclerosis. The Phase 2 study of autologous MSC-NTF cells in patients with progressive MS (NCT03799718) started enrollment in March 2019. For more information, visit the company's website at http://www.brainstorm-cell.com

Safe-Harbor Statement

Statements in this announcement other than historical data and information, including statements regarding future clinical trial enrollment and data, constitute "forward-looking statements" and involve risks and uncertainties that could causeBrainStorm Cell Therapeutics Inc.'sactual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may", "should", "would", "could", "will", "expect", "likely", "believe", "plan", "estimate", "predict", "potential", and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, BrainStorms need to raise additional capital, BrainStorms ability to continue as a going concern, regulatory approval of BrainStorms NurOwn treatment candidate, the success of BrainStorms product development programs and research, regulatory and personnel issues, development of a global market for our services, the ability to secure and maintain research institutions to conduct our clinical trials, the ability to generate significant revenue, the ability of BrainStorms NurOwn treatment candidate to achieve broad acceptance as a treatment option for ALS or other neurodegenerative diseases, BrainStorms ability to manufacture and commercialize the NurOwn treatment candidate, obtaining patents that provide meaningful protection, competition and market developments, BrainStorms ability to protect our intellectual property from infringement by third parties, heath reform legislation, demand for our services, currency exchange rates and product liability claims and litigation,; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available athttp://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.

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BrainStorm Cell Therapeutics Announces Ralph Kern MD MHSc to Present at the 7th International Stem Cell Meeting - Yahoo Finance

Bone Marrow Stem Cells Comments Off on BrainStorm Cell Therapeutics Announces Ralph Kern MD MHSc to Present at the 7th International Stem Cell Meeting – Yahoo Finance | November 13th, 2019

This is the tear-jerking moment a dad shares a hug with the woman who saved his life.

James O'Donnell, from Burnage, feared the worst after being diagnosed with a blood disorder similar to leukaemia in 2016.

Usual treatments were failing and James was undergoing a blood transfusion every week while battling constant infections, the Liverpool Echo reports.

James was running out of options and despaired at the pain his death could cause his eight-year-old son, Harrison.

But in a stunning stroke of fortune, his saviour was only the other side of the M62 - LiverpoolCouncil admin worker Leah McDougall.

The 29-year-old mum, from Bootle, had taken the time to sign up to the register of potential stem cell donors on her lunch break at a pop-up stall, organised by blood cancer charity DKSM, the previous year.

James, who despite his Manc heritage is an avid Liverpool FC fan, told staff at the charity that he would be up for meeting his donor, who could have been anyone from a number of European countries using the register.

James, along with his wife Andrea and young Harrison, got the chance to meet Leah for the first time at a DKSM charity gala in London on Wednesday last week (November 6).

James, who says he finally feels like himself after a long period of illness, told the ECHO: "I was just getting chest infections and water infections all the time.

"I am quite a healthy person, and I was in good shape and I knew I should not be getting ill all the time."

He said after a few weeks of tests his was invited to take a bone marrow biopsy and was told the devastating news on his 40th birthday.

The disease meant James' bone marrow was not producing enough white blood cells, but doctors told him a treatment called anti-thymocite globulin (ATG) had a "75% chance" of success.

However, when that failed, fear and doubt began to creep in.

He said: "We are always saying I would get through this, we were thinking I would get better. But I started to think it's not happening, it's not going to be for me, this.

"I thought, I have been good in life, I need some luck. We were having a really hard time.

"My son was four or five then, and it was hard for him having a dad going from playing football with him to being in hospital."

Eventually doctors revealed the only option was for James to have a bone marrow transplant.

The O'Donnell's went through further disappointment when tests on his three siblings revealed none were a match, so the waiting game to find a suitable donor began.

But on a March day in 2017, he got a call to say: "We have got a perfect match, a 10 out of 10."

The operation was a success and after four weeks doctors told James the new bone marrow cells were taking effect.

He said: "We were so lucky to find a donor only about 25 miles away. Some people never find one and we had one on our doorstep."

The powerful emotion of meeting Leah last week is summed up by James: "It was the second best moment of my life after my son being born.

"What she has done means that I can see my son growing up and that he has a father."

Leah did not hesitate to agree to help a total stranger when she was asked by DKSM.

Describing the moment she met James and his family, she told the ECHO: "We were both speechless. When I walked on stage we were just hugging each other for ages.

"It is weird, we felt like we had known each other for years, I felt like I had known him my whole life.

"It just takes five minutes out of your time to sign up to the register; that's like going to the kitchen to make a drink.

"You just think about the impact it is going to have on someone, it is saving someone's life. I feel lucky to have been able to give something back."

James says his family and Leah are planning to meet up again, possibly at a Liverpool FC game.

He said: "Without her, I wouldn't have a future."

DKSM has urged anyone aged 17-55, and in general good health, to sign up to the register here.

Dr Manos Niklolousis, Haematologist at University Hospital Birmingham NHS Foundation Trust, said:"Blood stem cells can be used to treat a wide range of blood cancers and blood disorders and we urgently need more people to come forward as donors.

"Currently, only 2% of the UK population are registered so matching donors with patients isnt easy within a growing multicultural population.

"Many of those in need are unable to find a sibling match and so rely on the generosity of strangers, and a blood stem cell transplant can be some patients only hope of survival.

"As a doctor who treats people with blood cancer or disorders, it is upsetting to know that some patients could have been saved if only more potential donors were registered and available to donate.

"I look forward to the day when there will be a donor for every patient in need."

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Heartbreaking moment dad meets the woman who saved his life - he feared his son would grow up without a father - Manchester Evening News

Bone Marrow Stem Cells Comments Off on Heartbreaking moment dad meets the woman who saved his life – he feared his son would grow up without a father – Manchester Evening News | November 13th, 2019

This is the poignant moment a dad and his young family broke down as they met the woman who saved his life.

James O'Donnell, 43, was running out of options after being diagnosed with aplastic anemia, a blood disorder similar to leukaemia, in 2016.

Usual treatments were failing and James was undergoing a blood transfusion every week while battling constant infections.

James, from Burnage in Manchester, feared his luck was out and despaired at the pain his death could cause his eight-year-old son Harrison.

But in a stunning stroke of fortune, his saviour was only the other side of the M62; Liverpool Council admin worker Leah McDougall.

The 29-year-old mum, from Bootle , had taken the time to sign up to the register of potential stem cell donors on her lunch break at a pop-up stall, organised by blood cancer charity DKMS, the previous year.

James, who despite his Manc heritage is an avid Liverpool FC fan, told staff at he charity that he would be up for meeting his donor, who could have been anyone from a number of European countries using the register.

James, along with his wife Andrea and young Harrison, got the chance to meet Leah for the first time at a DKMS charity gala in London on Wednesday last week (November 6).

James, who says he finally feels like himself after a long period of illness, told the ECHO: "I was just getting chest infections and water infections all the time.

"I am quite a healthy person, and I was in good shape and I knew I should not be getting ill all the time."

He said after a few weeks of tests his was invited to take a bone marrow biopsy and was told the devastating news on his 40th birthday.

The disease meant James's bone marrow was not producing enough white blood cells, but doctors told him a treatment called anti-thymocite globulin (ATG) had "75% chance" of success.

However when that failed, fear and doubt began to creep in for James.

He said: "We are always saying I would get through this, we were thinking I would get better. But I started to think it's not happening, it's not going to be for me, this.

"I thought, I have been good in life, I need some luck. We were having a really hard time. My son was four or five then, and it was hard for him having a dad going from playing football with him to being in hospital."

Eventually doctors revealed the only option was for James to have a bone marrow transplant.

The O'Donnells went through further disappointment when tests on his three siblings revealed none were a match, so the waiting game to find a suitable donor began.

But on a March day in 2017, he got a call to say: "We have got a perfect match, a 10 out of 10."

The operation was a success and after four weeks doctors told James the new bone marrow cells were taking effect.

He said: "We were so lucky to find a donor only about 25 miles away. Some people never find one and we had one on our doorstep."

The powerful emotion of meeting Leah last week is summed up by James: "It was the second best moment of my life after my son being born.

"What she has done means that I can see my son growing up and that he has a father."

Leah did not hesitate to agree to help a total stranger when she was asked by DKMS.

Describing the moment she met James and his family, she told the ECHO: "We were both speechless. When I walked on stage we were just hugging each other for ages.

"It is weird, we felt like we had known each other for years, I felt like I had known him my whole life.

"It just takes five minutes out of your time to sign up to the register; that's like going to the kitchen to make a drink.

"You just think about the impact it is going to have on someone, it is saving someone's life. I feel lucky to have been able to give something back."

James says his family and Leah are planning to meet up again, possibly at a Liverpool FC game.

He said: "Without her, I wouldn't have a future."

DKMS has urged anyone aged 17-55, and in general good health, to sign up to the register here .

Dr Manos Niklolousis, Haematologist at University Hospital Birmingham NHS Foundation Trust, said:"Blood stem cells can be used to treat a wide range of blood cancers and blood disorders and we urgently need more people to come forward as donors.

"Currently, only 2% of the UK population are registered so matching donors with patients isnt easy within a growing multicultural population.

"Many of those in need are unable to find a sibling match and so rely on the generosity of strangers, and a blood stem cell transplant can be some patients only hope of survival.

"As a doctor who treats people with blood cancer or disorders, it is upsetting to know that some patients could have been saved if only more potential donors were registered and available to donate. I look forward to the day when there will be a donor for every patient in need."

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Tearful dad meets woman who saved his life and gave him future with his son - Liverpool Echo

Bone Marrow Stem Cells Comments Off on Tearful dad meets woman who saved his life and gave him future with his son – Liverpool Echo | November 13th, 2019

CAMBRIDGE, Mass. & MENLO PARK, Calif.--(BUSINESS WIRE)--bluebird bio, Inc. (Nasdaq: BLUE) and Forty Seven, Inc. (Nasdaq:FTSV) announced today that they have entered into a research collaboration to pursue clinical proof-of-concept for Forty Sevens novel antibody-based conditioning regimen, FSI-174 (anti-cKIT antibody) plus magrolimab (anti-CD47 antibody), with bluebirds ex vivo lentiviral vector hematopoietic stem cell (LVV HSC) gene therapy platform. This collaboration will focus on a conditioning approach aimed to deliver reduced toxicity and will initially target diseases that have the potential to be corrected with transplantation of autologous gene-modified blood-forming stem cells. If successful, the new conditioning regimen could allow for more patients to undergo gene therapy.

Autologous hematopoietic stem cell transplantation (HSCT) and most ex vivo LVV HSC gene therapies require that a patients own stem cells first be depleted from the bone marrow to facilitate the engraftment of the new (or gene-modified) HSCs through a process called conditioning. Conditioning is performed using chemotherapy or radiation, which can place patients at risk for infection and require hospitalization until bone marrow cells have recovered. In addition, conventional conditioning can place patients at risk for secondary malignancy and infertility. As a result, the overall toxicity profile of current conditioning regimens limits the types of patients who are eligible for gene therapy. It is hoped that novel antibody based conditioning regimens could avoid these toxicities.

We are excited about this collaboration, combining our industry-leading LVV HSC gene therapy platform with Forty Sevens novel antibody-based conditioning regimen, said Philip Gregory, chief scientific officer, bluebird bio. We believe that, if successful, this novel conditioning modality could not only increase the number of patients and physicians who may consider gene therapy but also improve the overall risk benefit profile for stem cell-based gene therapy, as well as potentially reduce time and costs associated with hospital visits.

Forty Seven is advancing the pioneering work on CD47 and cKIT from our scientific founder, Irv Weissmans lab. We have shown that antibody blockade of CD47 can synergize with other antibodies targeting cancer to promote tumor engulfment. Based on this experience, coupled with the results of preclinical studies, we are eager to explore this dual-antibody approach for the potential treatment of non-malignant diseases, says Jens Peter Volkmer, M.D., Founder and Vice President of Research and Development at Forty Seven.

Forty Sevens President and Chief Executive Officer, Mark McCamish, M.D., Ph.D., commented, bluebird is a leading gene therapy company and we are excited to collaborate with them. Stem cell transplantation is potentially curative for a variety of blood diseases, including genetic blood disorders like sickle cell disease and beta-thalassemia. If successful, we believe our chemo- and radiation-free, all-antibody approach could expand transplantation beyond genetic blood disorders to a range of indications for which current transplantation approaches are suboptimal. In 2020, we plan to evaluate FSI-174 in healthy volunteers, before initiating a combination study of Forty Sevens novel all-antibody conditioning regimen and bluebirds gene therapy product.

Under the terms of the agreement, bluebird bio will provide its ex vivo LVV HSC gene therapy platform and Forty Seven will contribute its innovative antibody-based conditioning regimen for the collaboration.

About FSI-174 and MagrolimabFSI-174 is a humanized monoclonal antibody targeting cKIT, which is a receptor that is highly expressed on hematopoietic stem cells. Magrolimab is a humanized monoclonal antibody targeting CD47, which is a dont eat me signal to macrophages and is expressed on all cells. Magrolimab is currently being investigated in Phase 2 clinical trials to treat cancer and has established clinical efficacy in four indications, including myelodysplastic syndrome, acute myeloid leukemia, diffuse large B cell lymphoma and follicular lymphoma, with a favorable safety profile in over 350 patients treated, including some patients treated continuously for over two years. When combined, FSI-174 sends a positive signal to macrophages to target blood forming stem cells for removal and magrolimab disengages inhibitory signals that block phagocytosis. Combination of these antibodies has shown efficient removal of blood forming stem cells, allowing for transplantation in pre-clinical models.

About bluebird bio, Inc.bluebird bio is pioneering gene therapy with purpose. From our Cambridge, Mass., headquarters, were developing gene therapies for severe genetic diseases and cancer, with the goal that people facing potentially fatal conditions with limited treatment options can live their lives fully. Beyond our labs, were working to positively disrupt the healthcare system to create access, transparency and education so that gene therapy can become available to all those who can benefit.

bluebird bio is a human company powered by human stories. Were putting our care and expertise to work across a spectrum of disorders by researching cerebral adrenoleukodystrophy, sickle cell disease, transfusion-dependent -thalassemia and multiple myeloma using three gene therapy technologies: gene addition, cell therapy and (megaTAL-enabled) gene editing.

bluebird bio has additional nests in Seattle, Wash.; Durham, N.C.; and Zug, Switzerland. For more information, visit bluebirdbio.com.

Follow bluebird bio on social media: @bluebirdbio, LinkedIn, Instagram and YouTube.

bluebird bio is a trademark of bluebird bio, Inc.

About Forty Seven Inc.Forty Seven, Inc. is a clinical-stage immuno-oncology company that is developing therapies targeting cancer immune evasion pathways based on technology licensed from Stanford University. Forty Sevens lead program, magrolimab, is a monoclonal antibody against the CD47 receptor, a dont eat me signal that cancer cells commandeer to avoid being ingested by macrophages. This antibody is currently being evaluated in multiple clinical studies in patients with myelodysplastic syndrome, acute myeloid leukemia, non-Hodgkins lymphoma, ovarian cancer and colorectal carcinoma.

For more information, please visit http://www.fortyseveninc.com or contact info@fortyseveninc.com.

Follow Forty Seven on social media: @FortySevenInc, LinkedIn

Forward-Looking StatementsThis release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "may," "will," potentially, and similar expressions (as well as other words or expressions referencing future events, conditions, or circumstances) are intended to identify forward-looking statements. These statements include those related to the research and development plans for bluebird bios and Forty Sevens respective platforms and product candidates, the timing and success of Forty Sevens collaboration with bluebird bio, Forty Sevens plans to pursue clinical proof-of-concept for FSI-174 plus magrolimab with the LVV HSC gene therapy platform, the focus on diseases that have the potential to be corrected with transplantation of autologous gene-modified blood-forming stem cells, the tolerability and efficacy of FSI-174 and magrolimab, Forty Sevens plans to continue development of FSI-174 plus magrolimab, as well as related timing for clinical trials of the same.

Any forward-looking statements are based on the companies managements current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risks that the exploratory antibody-based conditioning platform will not be successful or will not be safe or effective in clinical trials, the risks that the collaboration between bluebird bio and Forty Seven will not continue or be successful, and the risk that the parties will not be successful in advancing the collaboration in development, the risk that potential product candidates that bluebird bio and Forty Seven develop may not progress through clinical development or receive required regulatory approvals within expected timelines or at all, the risk that clinical trials may not confirm any safety, potency or other product characteristics described or assumed in this press release and the risk that such product candidates may not be beneficial to patients or successfully commercialized. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the companies actual results to differ from those contained in the forward-looking statements, see the section entitled Risk Factors in each companys most recent Form 10-K as well as discussions of potential risks, uncertainties and other important factors in subsequent filings with the Securities and Exchange Commission at http://www.sec.gov. All information contained in this press release are not guarantees of future performance and speak only as of the date hereof, and each of bluebird bio and Forty Seven disclaims any obligation to update this information to reflect future events or circumstances unless required by law.

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bluebird bio and Forty Seven Announce a Research Collaboration to Study an All Antibody Conditioning Regimen for Use in Combination with Autologous...

Bone Marrow Stem Cells Comments Off on bluebird bio and Forty Seven Announce a Research Collaboration to Study an All Antibody Conditioning Regimen for Use in Combination with Autologous… | November 12th, 2019

NEW YORK, Nov. 12, 2019 (GLOBE NEWSWIRE) -- BrainStorm Cell Therapeutics, Inc. (NASDAQ:BCLI), a leading developer of adult stem cell therapies for neurodegenerative diseases, today announced that the Companys Chief Operating and Chief Medical Officer Ralph Kern MD MHSc will present at the 7th International Stem Cell Meeting, which is hosted by the Israel Stem Cell Society. The Conference will be held November 12-13, in Tel Aviv, Israel.

Ralph Kern, MD, MHSc, said: I welcome the opportunity to participate in the 7th International Stem Cell Meeting where I will share the advances BrainStorm has made with NurOwn. It is a privilege to participate and to exchange ideas with many of the international scientific leaders in stem cell research.

About NurOwn

NurOwn (autologous MSC-NTF) cells represent a promising investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors. Autologous MSC-NTF cells can effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. BrainStorm has fully enrolled a Phase 3 pivotal trial of autologous MSC-NTF cells for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm also recently received U.S. FDA acceptance to initiate a Phase 2 open-label multicenter trial in progressive MS and enrollment began in March 2019.

About BrainStorm Cell Therapeutics Inc.

BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwn technology platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug status designation from the U.S. Food and Drug Administration (U.S. FDA) and the European Medicines Agency (EMA) in ALS. BrainStorm has fully enrolled a Phase 3 pivotal trial in ALS (NCT03280056), investigating repeat-administration of autologous MSC-NTF cells at six U.S. sites supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). The pivotal study is intended to support a filing for U.S. FDA approval of autologous MSC-NTF cells in ALS. BrainStorm also recently received U.S. FDA clearance to initiate a Phase 2 open-label multicenter trial in progressive Multiple Sclerosis. The Phase 2 study of autologous MSC-NTF cells in patients with progressive MS (NCT03799718) started enrollment in March 2019. For more information, visit the company's website at http://www.brainstorm-cell.com

Safe-Harbor Statement

Statements in this announcement other than historical data and information, including statements regarding future clinical trial enrollment and data, constitute "forward-looking statements" and involve risks and uncertainties that could causeBrainStorm Cell Therapeutics Inc.'sactual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may", "should", "would", "could", "will", "expect", "likely", "believe", "plan", "estimate", "predict", "potential", and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, BrainStorms need to raise additional capital, BrainStorms ability to continue as a going concern, regulatory approval of BrainStorms NurOwn treatment candidate, the success of BrainStorms product development programs and research, regulatory and personnel issues, development of a global market for our services, the ability to secure and maintain research institutions to conduct our clinical trials, the ability to generate significant revenue, the ability of BrainStorms NurOwn treatment candidate to achieve broad acceptance as a treatment option for ALS or other neurodegenerative diseases, BrainStorms ability to manufacture and commercialize the NurOwn treatment candidate, obtaining patents that provide meaningful protection, competition and market developments, BrainStorms ability to protect our intellectual property from infringement by third parties, heath reform legislation, demand for our services, currency exchange rates and product liability claims and litigation,; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available athttp://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.

CONTACTS

Corporate:Uri YablonkaChief Business OfficerBrainStorm Cell Therapeutics Inc.Phone: 646-666-3188uri@brainstorm-cell.com

Media:Sean LeousWestwicke/ICR PRPhone: +1.646.677.1839sean.leous@icrinc.com

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BrainStorm Cell Therapeutics Announces Ralph Kern MD MHSc to Present at the 7th International Stem Cell Meeting - GlobeNewswire

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Article by Karen B. Roberts Photos by Evan Krape November 11, 2019

The Merriam-Webster Dictionary defines an inventor as one who creates or introduces something new.

Thomas Edison is one. So is Emily Day.

Edison created the incandescent light bulb and the typewriter, among dozens of other things.

Day, an assistant professor in biomedical engineering at the University of Delaware, is working on technology that may one day replace bone marrow transplants by enabling nanoparticle carrier systems to deliver medication and cargo directly to stem cells without the need to remove them from the body.

The University recognized more than 225 inventors, including Day, on Tuesday, Oct. 29, for their remarkable contributions to UD and to society at large.

The event, held at the Roselle Center for the Arts and coordinated by the UDResearch Office, celebrated researchers with discoveries in engineering, health care, energy, agriculture and many other fields.

You, our inventors, have taken nuggets of ideas, of discoveries that youve made and developed them through hard work, trial and error, failure and success. Youve shown tenacity and drive, patience and persistence, and the results are what were celebrating today, said UD Provost Robin Morgan.

Enriching the environment for entrepreneurship

Since 2008, UD researchers have generated more than 500 inventions.

Working in collaboration with its partners, the University has made a concerted effort to enrich the environment for these types of efforts in Delaware, contributing to the states economic prosperity and positively impacting the greater good.

UD research expenditures for fiscal year 2019 totaled $161 million, a record-setting 10% increase over 2018, to explore pressing topics across the sciences, engineering, humanities and social sciences.

During this same time frame, UD researchers generated 33 patent applications and secured 11 patents, with the support of the Universitys Office of Economic Innovation and Partnerships (OEIP). OEIP has licensed six UD-developed technologies to outside companies and evaluated numerous other potential inventions currently under development.

Several UD-developed technologies are now featured in the Association of University Technology Managers Better World Project, which highlights successful examples where academic research and technology transfer combine to benefit the broader world. One of these is the UD-patented microbe UD10-22, a unique strain of Bacillus subtilis that helps plants grow stronger, developed by Harsh Bais, associate professor of plant and soil sciences, and Janine Sherrier, a former UD faculty member. UD licensed the technology to BASF, a global chemical company, in 2013. After completing successful trials and regulatory clearances, the technology is now available in the market as a key component of BASFs Velondis and Nodulator Duo product lines in Canada and the United States. Trials are ongoing for the product to be available in four additional product lines and for a range of crops to be sold in several countries in South America, Europe and Asia.

We are building a dynamic and rich ecosystem to support this type of activity, now and in the future, said Charles G. Riordan, UD vice president for research, scholarship and innovation.

Continued growth of UDs Science, Technology and Advanced Research (STAR) Campus through strategic partnerships and infrastructure development is one example that firmly positions the University as an innovation powerhouse for the community, state and region. The Delaware Innovation Space, the business incubator that is a public-private partnership between the state of Delaware, DuPont and UD, is another.

Riordan reported that Delaware Innovation Space, with its 130,000 square feet of lab-based tech space for startups, already is 90% occupied, hosting 13 companies including UD startups W7energy and MCET along with serving an additional dozen companies through its virtual program. The result more than 240 jobs created or retained.

Other resources available on campus to support innovators and entrepreneurs include, but are not limited to, OEIP, competitive funding opportunities, seed funding and training programs at UDs Horn Entrepreneurship,and new and existing core research facilities.

Other UD technologies that have had success in the marketplace during the past year include Avkin, a leading manufacturer of sensor-enabled, high-fidelity, wearable technology for health care simulation education founded byAmy Cowperthwait, director of Healthcare Theatre for the College of Health Sciences.The patented devices are used for training health care workers and caregivers to perform clinical procedures, such asdrawing blood, tracheostomy care or catheter insertion. Designed to be worn by a live actor, Avkin products provide a realistic, patient-centered simulation.

Today, the UD-developed products can be found in select medical and nursing schools and health systems.The company now has five products in the market, and recently launched a new package aimed at equipping todays practitioners with the knowledge and skills necessary to prevent hospital acquired infections and to improve patient outcomes.

Isao Noda, UD affiliated professor in materials science and engineering, said it is particularly important to foster innovation and invention among students. An inventor himself, Noda is named on more than 60grantedU.S. patents.

One of Nodas inventions is abio-basedplasticmade from vegetable oilsknown asNodax, which can be used to make eco-friendlyproductsranging from biodegradable plastic straws topiezoelectricnanofibers forsensors and other electronics. Nodainventedthe material while a research fellow atProcterand Gamble. Today, UDscientistsare part of theexploratorywork onNodax, collaboratingon fundamental research to see just what elsethisnovelmaterialcan do.

In industry, invention is required. If you dont invent, you will be fired. But many graduates get jobs in industry without any of the training on how to invent, so this is amazingly important, said Noda.

Day agreed and said her approach to innovation shifted in recent years, particularly when speaking with students.

In the beginning of my academic career I was more focused on publishing papers, Day said. As my group has become more established, I now tell my students, Hey, before you go present this or publish, its important for you to submit your invention disclosure to protect your ideas.

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From inspiration to innovation | UDaily - UDaily

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Amsterdam, The Netherlands, 12 November 2019 Kiadis Pharma N.V. (Kiadis Pharma or the Company) (Euronext Amsterdam and Brussels: KDS), a clinical-stage biopharmaceutical company, today announced that it has completed a strategic portfolio review and has decided to change its strategy and focus all resources and investments on the companys NK-cell therapy platform and product candidates. The company will discontinue development of ATIR101 and stop its ongoing phase 3 trial.

Kiadis NK-cell program consists of off-the-shelf and haplo donor cell therapy products for the treatment of liquid and solid tumors. Kiadis proprietary off-the-shelf NK-cell platform is based on NK-cells from unique universal donors, expanded and activated ex vivo using our PM21 particle technology. The Kiadis off-the-shelf platform has the potential to make NK-cell therapy products rapidly and economically available for a broad patient population across a potentially wide range of indications.

The companys pipeline includes:

Arthur Lahr, CEO of Kiadis Pharma commented, We believe that our proprietary NK-cell therapy platform has broad potential as stand-alone or adjunctive treatments for patients with both liquid and solid tumors. Our off-the-shelf NK-cell platform is based on NK-cells from unique universal donors, expanded and activated with our PM21 particle technology, to make our NK-cell therapy products rapidly and economically available for patients across a potentially broad range of indications. The proof-of-concept trials for our NK pipeline programs, in which 38 patients have been treated, is very promising and was the basis for our acquisition of Cytosen Therapeutics, Inc. earlier this year. To confirm findings from these trials, we will start two Phase 1/2 clinical trials in 2020. We believe that investing in our NK platform and rapidly advancing development of our off-the-shelf and haplo donor derived NK-cell therapies in solid and liquid tumors will bring value to patients and our investors.

Lahr continued, As part of our strategic portfolio review, we reviewed progress of our phase 3 study, which was designed to show superiority of ATIR101 over the PTCy protocol. We identified that in the phase 3 a higher percentage of patients than expected dropped out of the study before receiving ATIR101. We subsequently collected additional recent external data, which show that outcomes with PTCy have better survival and lower severe GVHD than literature showed when we designed and started the phase 3 study. Based on these data, we no longer believe that the phase 3 ATIR study as currently designed with 250 patients can demonstrate superiority over PTCy and at a minimum would require a much larger trial. In the best interest of patients, we have therefore taken the decision to discontinue the ATIR101 study with immediate effect and are proceeding with close down activities.

RestructuringKiadis is implementing a restructuring program to refocus the organization on its NK-cell therapy platform, which will result in a reduction of approximately half of its workforce, a reduction in external clinical trial costs associated with the phase 3 study, and a reduced company cash burn. The company ended the third quarter of 2019 with approximately 47 million of cash.

About Kiadis K-NK-Cell Therapies Kiadis NK-cell programs consist of off-the-shelf and haplo donor cell therapy products for the treatment of liquid and solid tumors as adjunctive and stand-alone therapies.

Our NK-cell PM21 particle technology enables improved ex vivo expansion and activation of anti-cancer cytotoxic NK-cells supporting multiple high-dose infusions. Kiadis proprietary off-the-shelf NK-cell platform is based on NK-cells from unique universal donors. The Kiadis off-the-shelf K-NK platform can make NK-cell therapy product rapidly and economically available for a broad patient population across a potentially wide range of indications.

Administered as an adjunctive immunotherapeutic on top of HSCT, K-NK002 provides functional, mature and potent NK-cells from a haploidentical family member. In addition, Kiadis is developing K-NK003 for the treatment of relapse/refractory acute myeloid leukemia and has pre-clinical programs evaluating NK-cell therapy for the treatment of solid tumors.

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Kiadis Contacts:

About KiadisFounded in 1997, Kiadis Pharma, is a fully integrated biopharmaceutical company committed to developing innovative cell-based therapies for patients with life-threatening diseases. With headquarters in Amsterdam, the Netherlands, and offices and activities in the US and across Europe, Kiadis Pharma is leveraging the natural strengths of humanity and our collective immune system to source the best cells for life.

Kiadis Pharma is listed on the regulated market of Euronext Amsterdam and Euronext Brussels since July 2, 2015, under the symbol KDS. Learn more at http://www.kiadis.com.

Forward Looking Statements Certain statements, beliefs and opinions in this press release are forward-looking, which reflect Kiadis Pharmas or, as appropriate, Kiadis Pharmas directors current expectations and projections about future events. By their nature, forward-looking statements involve a number of risks, uncertainties and assumptions that could cause actual results or events to differ materially from those expressed or implied by the forward-looking statements. These risks, uncertainties and assumptions could adversely affect the outcome and financial impact of the plans and events described herein. A multitude of factors including, but not limited to, changes in demand, regulation, competition and technology, can cause actual events, performance or results to differ significantly from any anticipated development. Forward-looking statements contained in this press release regarding past trends or activities should not be taken as a representation that such trends or activities will continue in the future. As a result, Kiadis Pharma expressly disclaims any obligation or undertaking to release any update or revisions to any forward-looking statements in this press release as a result of any change in expectations or any change in events, conditions, assumptions or circumstances on which these forward-looking statements are based. Neither Kiadis Pharma nor its advisers or representatives nor any of its subsidiary undertakings or any such persons officers or employees guarantees that the assumptions underlying such forward-looking statements are free from errors nor does either accept any responsibility for the future accuracy of the forward-looking statements contained in this press release or the actual occurrence of the forecasted developments. You should not place undue reliance on forward-looking statements, which speak only as of the date of this press release.

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Kiadis Pharma changes strategy to focus solely on development of Natural Killer (NK) Cell therapeutics and terminates development of ATIR101 - Yahoo...

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What is flow cytometry and how does it work?Flow cytometry(FCM) is a scientific technique used to measure the physical and biochemical characteristics of cells.1The sample is injected into the flow cytometer instrument, where it is typically focused to flow one cell at a time past light sources and detectors. Tens of thousands of cells can be examined in seconds to determine their morphology, granularity, scattering and transmission of light, or fluorescence of biomarkers, depending on the variation of FCM used.

The first conventional fluorescence-based flow cytometer was developed and commercialized in the late 60s/early 70s in Germany.2 Over the last five decades, FCM has developed rapidly in terms of the number of its applications and the quantity and dimensionality of the data it generates.1,3 Dr. Minh Doan, formerly of the Imaging Platform of the Broad Institute (USA) and now head of Bioimaging Analytics at GlaxoSmithKline in the USA, states, There have been significant advances in all three Vs of flow cytometry data: velocity (throughput/speed of data acquisition), volume (data content), and variety (sample types and signal acquisition technology).

Michael Parsons, manager of the Flow Cytometry Core of the Lunenfeld-Tanenbaum Research Institute in Toronto, Canada, agrees. The two biggest trends in flow cytometry are high content data and the merging of technologies from separate disciplines. For example, the last five years or so have seen the emergence of mass cytometry, which merges the disciplines of flow cytometry and mass spectrometry. In its latest iteration, an image cytometry module has been incorporated to generate unprecedented amounts of content (number of measured parameters) from relatively small amounts of patient tissue. Spectral flow cytometry has also established itself as an important emerging technology. Indeed, mass cytometry can now measure up to 50 features on a single cell simultaneously using antibodies tagged with rare earth metals,4 and imaging flow cytometry allows for 1000s of morphological features and multiple fluorescence markers to be analyzed per cell.3Flow cytometry, therefore, has inarguable potential as a clinical tool for disease diagnosis, prognosis, and therapeutic monitoring. However, some challenges remain in translating the full promise of FCM into clinical practice. Here, some of the current clinical applications of FCM will be discussed, as well as some of the compelling new applications being researched.

Similarly, FCM of liquid biopsies could be used to detect circulating tumor cells in the bloodstream.3 These cells are extremely rare, and with its high sensitivity, FCM is perfectly poised to make a significant impact in this area. This approach has potential for the clinical detection of early-stage cancer as well as the detection of circulating metastatic or drug-resistant cancer cells. For example, a study published earlier this year described label-free liquid biopsy with very high throughput (> 1 million cells/second) for drug-susceptibility testing during leukemia treatment.8

Prior to an organ transplant, FCM can be used to crossmatch the patient's serum with donor lymphocytes to detect antibodies that could result in organ rejection.1 Postoperatively, the analysis of various cell markers on the peripheral blood lymphocytes can indicate early transplant rejection, detect bone marrow toxicity arising from immunosuppressive therapies, and help differentiate infections from organ rejection. For blood transfusions, FCM can be used to detect contamination of blood with residual white blood cells, which can have adverse effects such as pulmonary edema.9Groups such as Dr. Roshini Abrahams at Nationwide Childrens Hospital in Ohio, USA, are using FCM to diagnose primary immunodeficiency disorders with the use of immunophenotyping and functional assays.10 These disorders are caused by genetic mutations that result in defects in the immune system, such as X-linked (Brutons) agammaglobulinemia and X-linked hyper-IgM syndrome. Over 300 of these disorders have been identified thus far, and the causative mutations lower immune defense against the attack of infections.

HIV is, of course, an example of a secondary (acquired) immunodeficiency disorder. FCM analysis of CD4 and other markers on lymphocytes in the peripheral blood is used to monitor the treatment of HIV patients, and a CD4 count <200 cells/mL together with a positive antibody test for HIV is used as a diagnostic for AIDS.1 Secondary immunodeficiencies can also be caused by e.g., substance abuse, malnutrition, other medical conditions, and certain medical treatments. FCM of a panel of markers can be used to confirm suspected cases.1In pregnancy, when a Rhesus blood group D-negative mother carries a D-positive fetus, fetal-maternal bleeding can sensitize the mother to the D-positive blood cells from the fetus and this can be fatal to subsequent D-positive newborns.11 FCM is used to measure the degree of fetal-maternal hemorrhage to determine the correct dose of prophylactics to be administered shortly after delivery.

In addition to oncology and immunology applications, FCM is also used to diagnose a variety of rare hematologic disorders12 as well as autoimmune/autoinflammatory disorders such as spondylarthritis (arthritis of the spine).13 Another area of research that is likely to give rise to increasing clinical applications in the future is that of platelet activity, which is important in many clinical conditions.1,14

Experts suggest that it may be possible to overcome this data analysis hurdle by applying machine learning approaches coupled with further standardization of FCM workflows.3,15 The most exciting applications of high content data revolve around the use of machine learning, in particular, deep learning, to extract relevant meaning from large data sets. Machine learning, coupled with big data, has the potential for driving diagnosis and treatment options tailored to the patients disease in a timely manner, says Dr. Parsons. In addition, Prof. Sadao Ota of RCAST at the University of Tokyo, Japan, points out, We still need to figure out how to design a workflow that convincingly validates diagnostic results, especially if the diagnosis employs the power of machine learning. Such developments are necessary before the rich information content of advanced FCM technology can be fully applied in the clinic.

In terms of other future advances in the field, Prof. Ota specifically makes mention of the potential of cell sorters combined with FCM.16 There are exciting and unique applications of sorters in fields such as cell therapy and regenerative medicine. Also, creating key applications of imaging cell sorters in pharmaceutical fields may accelerate global drug discovery. Dr. Doan concurs, Disease heterogeneity makes it hard to validate findings. Perhaps the use of flow cytometry with sorting capability can help such validation, where events-of-interest collected by flow cytometry can be validated with other downstream assays. Finally, as Dr. Doan notes, With multiple layers of data(types) incorporated altogether, there are now possibilities to do more with less, i.e., label-free sample measurement, which could lead to more direct, faster, and smarter diagnoses. Rare events (e.g., metastatic cancer cells) may soon be detected better than before.References1.Bakke A.C. Clinical Applications of Flow Cytometry. Laboratory Medicine. 2000; 31(2): 97104. doi: 10.1309/FC96-DDY4-2CRA-71FK.2.Herzenberg L.A., Parks D., Sahaf B., Perez O., Roederer M., Herzenberg L.A. The history and future of the fluorescence activated cell sorter and flow cytometry: a view from Stanford. Clinical Chemistry. 2002;48(10):181918273.Doan M., Vorobjev I., Rees P., Filby A., Wolkenhauer O., Goldfeld A.E., Lieberman J., Barteneva N., Carpenter A.E., Hennig H. Diagnostic potential of imaging flow cytometry. Trends in Biotechnology. 2018;36(7):649652. doi: 10.1016/j.tibtech.2017.12.008.4.Olsen L.R, Leipold M.D., Pedersen C.B., Maecker H.T. The anatomy of single cell mass cytometry data. Cytometry Part A. 2019;95(2):156172. doi: 10.1002/cyto.a.23621.5.Laerum O.D., Farsund T. Clinical application of flow cytometry: a review. Cytometry. 1981;2(1):113. doi: 10.1002/cyto.990020102.6.Li J., Wertheim G., Paessler M., Pillai V. Flow cytometry in pediatric hematopoietic malignancies. Clinics in Laboratory Medicine. 2017;37(4):879893. doi: 10.1016/j.cll.2017.07.009.7.Gupta S., Devidas M., Loh M.L., Raetz E.A., Chen S., Wang C., Brown P., Carroll A.J., Heerema N.A., Gastier-Foster J.M., Dunsmore K.P., Larsen E.C., Maloney K.W., Mattano L.A. Jr., Winter S.S., Winick N.J., Carroll W.L., Hunger S.P., Borowitz M.J., Wood B.L. Flow-cytometric vs. -morphologic assessment of remission in childhood acute lymphoblastic leukemia: a report from the Childrens Oncology Group (COG). Leukemia. 2018;32(6):13701379. doi: 10.1038/s41375-018-0039-7.8.Kobayashi H., Lei C., Wu Y., Huang C-J., Yasumoto A., Jona M., Li W., Wu Y., Yalikun Y., Jiang Y., Guo B., Sun C-W., Tanaka Y., Yamada M., Yatomi Y., Goda K. Intelligent whole-blood imaging flow cytometry for simple, rapid, and cost-effective drug-susceptibility testing of leukemia. Lab on a Chip. 2019;19(16):26882698. doi: 10.1039/c8lc01370e.9.Castegnaro S., Dragone P., Chieregato K., Alghisi A., Rodeghiero F., Astori G. Enumeration of residual white blood cells in leukoreduced blood products: Comparing flow cytometry with a portable microscopic cell counter. Transfusion and Apheresis Science. 2016;54(2):266270. doi: 10.1016/j.transci.2015.10.001.10.Abraham R.S., Aubert G. Flow cytometry, a versatile tool for diagnosis and monitoring of primary immunodeficiencies. Clinical and Vaccine Immunology. 2016;23(4):254271. doi: 10.1128/CVI.00001-16.11.Kim Y.A., Makar R.S. Detection of fetomaternal hemorrhage. American Journal of Hematology. 2012;87(4):417423. doi: 10.1002/ajh.22255.12.Bn M.C., Le Bris Y., Robillard N., Wuillme S., Fouassier M., Eveillard M. Flow cytometry in hematological nonmalignant disorders. International Journal of Laboratory Hematology. 2016;38(1):516. doi: 10.1111/ijlh.12438.13.Duan Z., Gui Y., Li C., Lin J., Gober H.J., Qin J., Li D., Wang L. The immune dysfunction in ankylosing spondylitis patients. Bioscience Trends. 2017;11(1):6976. doi: 10.5582/bst.2016.01171.14.Pasalic L. Assessment of platelet function in whole blood by flow cytometry. Methods in Molecular Biology. 2017;1646:349367. doi: 10.1007/978-1-4939-7196-1_27.15.Doan M., Carpenter A.E. Leveraging machine vision in cell-based diagnostics to do more with less. Nature Materials. 2019;18(5):414418. doi: 10.1038/s41563-019-0339-y.16.Ota S., Horisaki R., Kawamura Y., Ugawa M., Sato I., Hashimoto K., Kamesawa R., Setoyama K., Yamaguchi S., Fujiu K., Waki K., Noji H. Ghost cytometry. Science. 2018;360(6394):12461251. doi: 10.1126/science.aan0096.

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Transplant strategies for AML

Today, the only curative approach to treat patients with AML is the administration of high-dose chemotherapy followed by allogeneic HSCT (allo-SCT). Although autologous HSCT (auto-SCT) may still be an option for certain patients with favorable or intermediate risk AML, its use has been debated due to the fact that AML is a blood and bone marrow malignancy, thus transplantation with the patients own cells runs the risk of giving back some of the patients leukemia cells.1 In contrast, during the process of allo-SCT, cells from a donor are infused. This provides an additional benefit, called the graft-versus-leukemia or tumor (GvL or GvT) effect, whereby the donor immune cells have the potential of recognising and eradicating remaining leukemia cells in the host, thus reducing the risk of relapse.2

Various donors can be used for an allo-SCT, while the best stem cell source remains to be from a human leukocyte antigen (HLA)-matched sibling donor (MSD). However, in approximately 70% of the cases such donors are unavailable, leaving the next best option of an HLA-matched unrelated donor (MUD).3 For patients where MSD or MUD are unavailable, a partially HLA-matched related donor can be used. This is referred to as haploidentical transplant (HD) and is usually a 50% HLA-match. In the past, HD has been associated with a slow immune reconstitution and high mortality from infections. Today, the use of post-transplant management treatments, like post-transplant cyclophosphamide (PTCy), reduces mortality and has made HD a viable option for patients with AML. This was further discussed by Arnon Nagler in his interview4 with the AML Global Portal (AGP) during the 2019 European Society for Blood and Marrow Transplantation (EBMT) meeting (video below). Nevertheless, HD still leads to inferior outcomes when compared to MSD in patients with AML (read AGP article here). Other donor types include cells from umbilical cord (read AGP article here) or from HLA-mismatched unrelated donors (MMUD).2 The impact of donor type on the outcomes of allo-SCT has recently been explored in an article by the AGP here. The authors of the study concluded that the traditional hierarchy of donors (MSD, MUD, and then others) remains true in patients with AML and should be used as a treatment algorithm.

VIDEO INTERVIEW: EBMT 2019 | Haploidentical hematopoietic transplantation: current status and future perspectives

How to choose the right patients for transplantation

Choosing the right patient to receive transplantation following chemotherapy is crucial for maximising outcomes and reducing the risk of relapse and toxicity. As mentioned by Uwe Platzbecker in his AGP interview during the 2019 EBMT meeting (video below), there are two main considerations when choosing the right candidate for allo-SCT:

According to the ELN guidelines, patients are classified as favorable-risk, intermediate-risk, or poor-risk depending on the possibility of disease relapse. Patients with favorable-risk are usually not considered for allo-SCT after achieving their first complete remission (CR1), as the risk of toxicity and serious side effects outweighs the potential benefit from allo-SCT. For these patients, auto-SCT instead of chemotherapy after CR1 could be beneficial (read AGP article here). On the contrary, allo-SCT at CR1 is a common strategy for poor-risk patients with AML. In the case of intermediate-risk patients (the majority of patients with AML), the most suitable treatment option is less clear.6 Due to the high relapse rates seen in AML, allo-SCT has also been considered as a potential treatment strategy in second remission (CR2), although outcome is inferior compared to allo-SCT performed in CR1.7 In a recent study, summarized here by the AGP, it seems that myeloablative conditioning (MAC) and reduced intensity conditioning (RIC) lead to similar outcomes after allo-SCT in CR2, however more prospective trials are needed to tailor them for maximum efficacy and minimum toxicity.7 To date, it is evident that clinical decisions to perform transplantation need to be made on an individual basis. Recently, measurable residual disease (MRD) as a marker for disease severity and relapse risk has emerged as an important factor that can guide treatment decisions in the context of HSCT and has been reviewed in depth here by the AGP.

VIDEO INTERVIEW: EBMT 2019 | Considerations for transplantation in AML

Post-transplant issues & how to tackle them

Regardless of the advances in the transplantation field, allo-SCTs are associated with two main post-transplant issues: disease relapse and graft-versus-host disease (GvHD).

There is still a considerable number of patients that relapse after HSCT. At the moment, the best strategies to decrease the risk of post-transplantation relapse include:

Such agents include the use of FMS-like tyrosine kinase-3 (FTL3) inhibitors that are shown to delay disease relapse and to potentiate the GvL effect in patients with FLT3 mutations after allo-SCT.7 Multiple pre-clinical and clinical trials are currently underway to examine the efficacy of other targeted inhibitors, like sorafenib, lestaurtinib, sunitinib, tandutinib, quizartinib, and midostaurin, amongst others.8 Another drug that has been shown to prevent disease relapse and to potentially increase the GvL effect is azacitidine. This is a hypomethylating agent that is currently used as a safe and effective prophylactic therapy in high-risk patients following allo-SCT (read AGP article here).9

Charlie Craddock provided an extensive presentation on the strategies for GvL effect optimization at the 2019 EBMT meeting (see full article on the AGP here):

VIDEO INTERVIEW: American Society of Clinical Oncology (ASCO) 2019 | Who should get azacitidine after transplant?

GvHD remains a major post-transplant challenge, occurring when transplanted donor cells start attacking host cells and tissues.10 There are two main strategies used today to prevent GvHD:

In a clinical trial, PTCy has shown superior outcomes when compared to ATG in patients undergoing HD transplant, leading to improved overall survival, leukemia-free survival, and GvHD-relapse free survival. The results of this study were discussed by Arnon Nagler in his interview with the AGP at European Hematology Association (EHA) 2019. A comprehensive review on available treatments for GvHD prophylaxis and their efficacies has been published here by our GvHD Hub.

VIDEO INTERVIEW: EHA 2019 | Should we use PTCy or ATG as GvHD prophylaxis in haploidentical stem cell transplantation?

In patients who develop severe GvHD, systemic administration of steroids remains the first choice of treatment. Treating GvHD is considered by many as a double-edged sword, since on one side it is necessary, but on the other hand it may hinder the GvL effect, thus contributing to potential disease relapse. Further research is needed to clarify the role of GvHD treatment on the GvL effect and to establish the best agent to treat GvHD without hindering the benefits of graft transplantation to the host. Some patients do not respond to post allo-SCT corticosteroids and are classified as steroid-refractory GvHD patients. These patients have a high mortality rate after allo-SCT with a 1-year survival between 30-35%. Many novel approaches are being tested for these patients with the Janus kinase 1/2 (JAK1/2) inhibitor, ruxolitinib, and the Brutons tyrosine kinase (BTK) inhibitor, ibrutinib, being recently approved by the Food and Drug Administration (FDA) for steroid-refractory GvHD.11,12

VIDEO INTERVIEW: EHA 2019 | What are the current treatment recommendations for acute GvHD and the promotion of the GvL effect?

Can the new treatments for AML reduce the need for transplantation?

With the recent therapeutic advances in the field of AML, one major question arises: Can these advances in diagnostics and new therapies replace allo-SCT? During the 1st National Cancer Research Institute (NCRI) AML academy meeting, AGP was pleased to film the headline debate on recently licensed drugs versus recent advances in transplantation, which can be accessed here. Although an unresolved issue, it is evident that some of the new treatments lack the toxicity associated with allo-SCT and have demonstrated improved survival rates. Moreover, with new diagnostic tools, the identification of the right subgroups of patients who may benefit from a transplant-free and more targeted approach will be feasible. One such novel approach to AML treatment is the use of CAR-T cells. Their use as monotherapy or in combination with allo-SCT for the treatment of relapsed or refractory AML is currently under consideration and of great interest in the field. More details on the potential of CAR-T cell therapy for AML can be found here in a recently published article by the AGP. However, it is too early to say whether these new treatment approaches can replace allo-SCT as a curative approach to treat AML. This topic was discussed by Gert Ossenkoppele in the interview with the AGP shown below.

VIDEO INTERVIEW: EHA 2019 | What is the clinical value of new drugs in AML?

Conclusions

Despite the curative potential conferred by allo-SCT in patients with AML, there is still a high risk of non-relapse mortality (mostly due to severe GvHD) in addition to the risk of relapse associated with transplantation. This warrants the need for the development of either novel management and prophylactic therapies that can improve post-transplantation outcomes or of transplantation-free approaches for the treatment of AML. With numerous clinical trials underway with novel targeted agents as monotherapy or in combination, the future of AML treatment starts to look more promising.

Originally posted here:
An introduction to the use of transplantation for the treatment of AML - AML Global Portal

Bone Marrow Stem Cells Comments Off on An introduction to the use of transplantation for the treatment of AML – AML Global Portal | November 11th, 2019

Stem Cell Therapy Market is expected to reach 202.77 billion by 2026 from 12.25 billion in 2017 at CAGR of 42.02%.(Detailed analysis of the market CAGR is provided in the report) stands for use of stem cells to treat or prevent disease or condition.

Bone marrow transplant and some therapies derived from umbilical cord blood are mainly used in stem cell therapy. Advancement, in order to establish new sources for stem cells, and to apply stem-cell treatments for neurodegenerative diseases and conditions such as diabetes, heart disease, and other conditions, are increased in recent years. Stem Cell Therapy Market Researchers are making efforts to discover novel methods to create human stem cells. This will increase the demand as well as supply for stem cell production and potential investigation in disease management. Increasing investment & research grants for developing safe and effective stem cell therapy products, the growing patient base for target diseases, concentrated product pipelines, increasing approval of the new clinical trials, rapid technological advancement in genomics, and the rising awareness about the stem cell are expected to drive the growth of the Stem Cell Therapy solutions market during the forecast period.

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However, improper infrastructure, insufficient storage systems, nascent technology in underdeveloped economies, Ethical issues related to an embryonic stem cell, low patient acceptance rate, Difficulty in the preservation of stem cell are expected to restrain the market growth. North America is expected to be the largest growing region by 2026; the reason behind that is extensive funding by Government. However, Emerging countries like India, china, Korea have low growth rate as compared to Developed regions in 2017 but increase in awareness about stem cell therapy will lead the Asia Pacific to generate a significant level of revenue by 2026.

Key Highlights of Stem Cell Therapy Market report

Detailed quantitative analysis of the current and future trends from 2017 to 2026, which helps to identify the prevailing market opportunities.Comprehensive analysis of factors instrumental in changing the market scenario, rising prospective opportunities, market shares, core competencies in terms of market development, growth strategies and identification of key companies that can influence this market on a global and regional scale.Assessment of Market definition along with the identification of key drivers, restraints opportunities and challenges for this market during the forecast period.Complete analysis of micro-markets with respect to individual growth trends, prospects, and contributions to the overall Stem Cell Therapy Solutions market.Stem Cell Therapy market analysis and comprehensive segmentation with respect to the Application, End users, Treatment, and geography to assist in strategic business planning.Stem Cell Therapy market analysis and forecast for five major geographies-North America, Europe, Asia Pacific, Middle East & Africa, Latin America, and their key regions.For company profiles, 2017 has been considered as the base year. In cases, wherein information was unavailable for the base year, the years prior to it have been considered.

Research Methodology:

The market is estimated by triangulation of data points obtained from various sources and feeding them into a simulation model created individually for each market. The data points are obtained from paid and unpaid sources along with paid primary interviews with key opinion leaders (KOLs) in the market. KOLs from both, demand and supply side were considered while conducting interviews to get an unbiased idea of the market. This exercise was done at a country level to get a fair idea of the market in countries considered for this study. Later this country-specific data was accumulated to come up with regional numbers and then arrive at a global market value for the stem cell therapy market.

Key Players in the Stem Cell Therapy Market are:

Chiesi Farmaceutici S.P.A Are:Gamida CellReNeuron Group, plcOsiris Therapeutics, Inc.Stem Cells, Inc.Vericel Corporation.Mesoblast, Ltd.

Key Target Audience:

Stem Cell Associations and OrganizationsGovernment Research Boards and OrganizationsResearch and consulting firmsStem Cell Therapy Market InvestorsHealthcare Service Providers (including Hospitals and Diagnostic Centers)Stem Cell Therapeutic Product Manufacturing OrganizationsResearch LabsClinical research organizations (CROs)Stem Cell Therapy Marketing PlayersPharmaceutical Product Manufacturing Companies

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Scope of the Stem Cell Therapy Market Report:

Stem Cell Therapy market research report categorizes the Stem Cell Therapy market based on Application, End users, Treatment, and geography (region wise). Market size by value is estimated and forecasted with the revenues of leading companies operating in the Stem Cell Therapy market with key developments in companies and market trends.

Stem Cell Therapy Market, By Treatments:

Allogeneic Stem Cell TherapyAutologous Stem Cell Therapy

Stem Cell Therapy Market, By End Users:

HospitalsAmbulatory Surgical Centers

Stem Cell Therapy Market, By Application:

OncologyCentral Nervous System DiseasesEye DiseasesMusculoskeletal DiseasesWound & InjuriesMetabolic DisordersCardiovascular DisordersImmune System Disorders

Stem Cell Therapy Market, By Geography:

North AmericaEuropeAsia PacificMiddle East & AfricaLatin America

Available Customization:

With the given market data, Maximize Market Research offers customization of report and scope of the report as per the requirement

Regional Analysis:

Breakdown of the North America stem cell therapy marketBreakdown of the Europe stem cell therapy marketBreakdown of the Asia Pacific stem cell therapy marketBreakdown of the Middle East & Africa stem cell therapy marketBreakdown of the Latin America stem cell therapy market

Company Information:Detailed analysis and profiles of addition

Browse Full Report with Facts and Figures of Stem Cell Therapy Market Report at: https://www.maximizemarketresearch.com/market-report/stem-cell-therapy-market/522/

MAJOR TOC OF THE REPORT

Chapter One: Stem Cell Therapy Market Overview

Chapter Two: Manufacturers Profiles

Chapter Three: Global Stem Cell Therapy Market Competition, by Players

Chapter Four: Global Stem Cell Therapy Market Size by Regions

Chapter Five: North America Stem Cell Therapy Revenue by Countries

Chapter Six: Europe Stem Cell Therapy Revenue by Countries

Chapter Seven: Asia-Pacific Stem Cell Therapy Revenue by Countries

Chapter Eight: South America Stem Cell Therapy Revenue by Countries

Chapter Nine: Middle East and Africa Revenue Stem Cell Therapy by Countries

Chapter Ten: Global Stem Cell Therapy Market Segment by Type

Chapter Eleven: Global Stem Cell Therapy Market Segment by Application

Chapter Twelve: Global Stem Cell Therapy Market Size Forecast (2019-2026)

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Stem Cell Therapy Market by Treatment,Application,End Users and Geography Forecast To 2026 - Markets Gazette 24

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Myelofibrosis or osteomyelofibrosis is a myeloproliferative disorder which is characterized by proliferation of abnormal clone of hematopoietic stem cells. Myelofibrosis is a rare type of chronic leukemia which affects the blood forming function of the bone marrow tissue. National Institute of Health (NIH) has listed it as a rare disease as the prevalence of myelofibrosis in UK is as low as 0.5 cases per 100,000 population. The cause of myelofibrosis is the genetic mutation in bone marrow stem cells. The disorder is found to occur mainly in the people of age 50 or more and shows no symptoms at an early stage. The common symptoms associated with myelofibrosis include weakness, fatigue, anemia, splenomegaly (spleen enlargement) and gout. However, the disease progresses very slowly and 10% of the patients eventually develop acute myeloid leukemia. Treatment options for myelofibrosis are mainly to prevent the complications associated with low blood count and splenomegaly.

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The global market for myelofibrosis treatment is expected to grow moderately due to low incidence of a disease. However, increasing incidence of genetic disorders, lifestyle up-gradation and rise in smoking population are the factors which can boost the growth of global myelofibrosis treatment market. The high cost of therapy will the growth of global myelofibrosis treatment market.

The global market for myelofibrosis treatment is segmented on basis of treatment type, end user and geography:

As myelofibrosis is considered as non-curable disease treatment options mainly depend on visible symptoms of a disease. Primary stages of the myelofibrosis are treated with supportive therapies such as chemotherapy and radiation therapy. However, there are serious unmet needs in myelofibrosis treatment market due to lack of disease modifying agents. Approval of JAK1/JAK2 inhibitor Ruxolitinib in 2011 is considered as a breakthrough in myelofibrosis treatment. Stem cell transplantation for the treatment of myelofibrosis also holds tremendous potential for market growth but high cost of therapy is foreseen to limits the growth of the segment.

On the basis of treatment type, the global myelofibrosis treatment market has been segmented into blood transfusion, chemotherapy, androgen therapy and stem cell or bone marrow transplantation. Chemotherapy segment is expected to contribute major share due to easy availability of chemotherapeutic agents. Ruxolitinib is the only chemotherapeutic agent approved by the USFDA specifically for the treatment of myelofibrosis, which will drive the global myelofibrosis treatment market over the forecast period.

Geographically, global myelofibrosis treatment market is segmented into five regions viz. North America, Latin America, Europe, Asia Pacific and Middle East & Africa. Northe America is anticipated to lead the global myelofibrosis treatment market due to comparatively high prevalence of the disease in the region.

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Some of the key market players in the global myelofibrosis treatment market are Incyte Corporation, Novartis AG, Celgene Corporation, Mylan Pharmaceuticals Ulc., Bristol-Myers Squibb Company, Eli Lilly and Company, Taro Pharmaceuticals Inc., AllCells LLC, Lonza Group Ltd., ATCC Inc. and others.

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Myelofibrosis Treatment Market To Witness an Outstanding Growth During 2016-2022 - Zebvo

Bone Marrow Stem Cells Comments Off on Myelofibrosis Treatment Market To Witness an Outstanding Growth During 2016-2022 – Zebvo | November 11th, 2019