Proud auntie Paola Mayfield says her niece, who has Down syndrome, is in remission from the leukemia that she battled for two years.

Last year I went to Colombia when I was pregnant, I needed to see my sister and my niece, Mayfield wrote alongside a photo of niece standing on a hospital bed with her arm attached to some medical devices. My niece has Down Syndrome and had been battling leukemia for about 2 years. But TODAY we received the greatest news! She is finally cancer free!

Paola said that the journey has been extremely difficult. With tears in my eyes I feel full of joy and happiness because I know how hard it has been the past few years for my sister.

And she wanted others who are struggling and fighting for their lives to know that there is always hope: For those who battle everyday to have another day of life, stay strong and dont lose faith. Thank you [hearts].

Leukemia is the most common type of childhood cancer. The most frequent type of childhood leukemia is acute lymphoblastic leukemia (ALL). Three out every four cases of childhood leukemia are diagnosed as acute, meaning that the leukemia can progress quickly, and if not treated, would probably be fatal within a few months.

Acute lymphoblastic leukemia is a rare cancer thatoccurs when the bone marrow makes too much of a type of white blood cell calledlymphocytes, according to the National Cancer Institute. Signs of childhood ALL include fever and bruising. The disease can be detected using tests that examine the blood and bone marrow. Over time, there has been a lot of improvement in treatments for childhood leukemia.

There are several different approaches to treating the disease, and the treatment plan will depend on the type of ALL. Chemotherapy, radiation, chemotherapy with a stem cell transplant, and targeted therapy are all considered standard treatment, according to the American Cancer Society.

The next most common type of childhood leukemia is called acute myeloid leukemia, which occurs when the bone marrow makes a large number of abnormal blood cells called myeloblasts. As these cells build up, they prevent the growth ofhealthy white blood cells, red blood cells, and platelets.

Learn more about SurvivorNet's rigorous medical review process.

Proud auntie Paola Mayfield says her niece, who has Down syndrome, is in remission from the leukemia that she battled for two years.

Last year I went to Colombia when I was pregnant, I needed to see my sister and my niece, Mayfield wrote alongside a photo of niece standing on a hospital bed with her arm attached to some medical devices. My niece has Down Syndrome and had been battling leukemia for about 2 years. But TODAY we received the greatest news! She is finally cancer free!

Paola said that the journey has been extremely difficult. With tears in my eyes I feel full of joy and happiness because I know how hard it has been the past few years for my sister.

And she wanted others who are struggling and fighting for their lives to know that there is always hope: For those who battle everyday to have another day of life, stay strong and dont lose faith. Thank you [hearts].

Leukemia is the most common type of childhood cancer. The most frequent type of childhood leukemia is acute lymphoblastic leukemia (ALL). Three out every four cases of childhood leukemia are diagnosed as acute, meaning that the leukemia can progress quickly, and if not treated, would probably be fatal within a few months.

Acute lymphoblastic leukemia is a rare cancer thatoccurs when the bone marrow makes too much of a type of white blood cell calledlymphocytes, according to the National Cancer Institute. Signs of childhood ALL include fever and bruising. The disease can be detected using tests that examine the blood and bone marrow. Over time, there has been a lot of improvement in treatments for childhood leukemia.

There are several different approaches to treating the disease, and the treatment plan will depend on the type of ALL. Chemotherapy, radiation, chemotherapy with a stem cell transplant, and targeted therapy are all considered standard treatment, according to the American Cancer Society.

The next most common type of childhood leukemia is called acute myeloid leukemia, which occurs when the bone marrow makes a large number of abnormal blood cells called myeloblasts. As these cells build up, they prevent the growth ofhealthy white blood cells, red blood cells, and platelets.

Learn more about SurvivorNet's rigorous medical review process.

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Tears And Joy This Toddler with Down Syndrome Who Was Battling Leukemia Is Finally Cancer Free - SurvivorNet

Bone Marrow Stem Cells Comments Off on Tears And Joy This Toddler with Down Syndrome Who Was Battling Leukemia Is Finally Cancer Free – SurvivorNet | November 8th, 2019

MONTREAL, Nov. 06, 2019 (GLOBE NEWSWIRE) -- ExCellThera Inc., an advanced biotechnology company delivering molecules and bioengineering solutions to expand stem and immune cells for therapeutic use, announced today the publication of full data from the first clinical trial using ECT-001 (single UM171-expanded cord blood) in patients with haematological malignancies. The data were published in the peer-reviewed medical journal, The Lancet Haematology.

The clinical trial findings indicate that ECT-001 cell therapy is feasible, safe (as suggested by the low transplant-related mortality, low incidence of severe acute graft-vs-host disease (GVHD), and absence of moderate to severe chronic GVHD) and allows for the use of small cords without compromising engraftment. In addition, ECT-001 has shown potential to overcome the disadvantages of unexpanded cord blood transplants while maintaining their benefits of low risk of chronic GVHD and relapse. The Lancet Haematology paper provides the first detailed analysis of the study results presented at the 60th American Society of Hematology Annual Meeting (ASH 2018) in December 2018 and supports the recent advancement of the ECT-001 clinical program.

Were pleased that these important results from the first clinical trial using ECT-001 in haematological malignancies are now fully available to the broader bone marrow transplant community, said Dr. Guy Sauvageau, CEO and founder of ExCellThera, and co-senior author of the paper. These results indicate that ECT-001 transplants combine the advantages of conventional grafts using bone marrow (low treatment-related mortality), peripheral blood (fast engraftment) and cord blood (greater accessibility, low relapse and chronic GvHD) in a single, low cost, easy to produce 7-day culture product, which could lead to a paradigm shift in bone marrow transplantation.

The FDA granted ECT-001 Orphan Drug Designation for the prevention of graft-versus-host disease in 2018 and Regenerative Medicine Advanced Therapy Designation in the treatment of hematologic malignancies in 2019. ECT-001 is currently being used for the treatment of blood disorders in other ongoing and approved clinical trials in the United States and Canada. ExCellThera also plans to initiate a European clinical trial as well as a pivotal, multi-centre clinical trial in the coming months.

About ExCellThera Inc.

ExCellThera is an advanced clinical stage biotechnology company delivering molecules and bioengineering solutions to expand stem and immune cells for use in novel one-time curative therapies for patients with hematologic malignancies, autoimmune and other diseases. ExCellTheras lead solution combines a proprietary small molecule, UM171, and an optimized culture system. In pursuit of better treatments for patients, the company is building out its portfolio of products, as well as supporting best-in-class clinical trials. excellthera.com

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Arizona has been called "the wild, wild west" of regenerative medicine.

The Valley is one of the most popular places in the country for stem cell clinics. The new and controversial therapy is being marketed and practiced all over Phoenix and Scottsdale.

The less invasive procedure promises to heal pain, nearly anywhere in their body. It is advertised as effective, safe, and ethical, but outside experts and industry insiders say consumers need to do their research to avoid being exploited, and potentially spending thousands in cash on a worthless injection.

"IT HAS GREAT POTENTIAL"

The world of regenerative medicine is still being explored and developed.

"It actually gives you really good results," explained Dr. Matthew Hernandez, a naturopathic physician with Ethos.

"There's a lot of hope and promise, generally around the prospects for stem cells," said ASU Professor Emma Frow.

"Were still in the developmental stage. Stem cell therapy has been around for less than ten years. Thats new in medicine," said Dr. Steven Sorr, a naturopathic physician who runs Source of Health in Scottsdale.

"It encourages your own body to heal itself," said Janet McConnell, a 63-year-old bodybuilder who "had cartilage damage several years ago."

Instead of a surgery that would have derailed her competition training for months, she opted for injections.

"Three years ago, instead of the surgery, I had a PRP treatment," said McConnell. "It was very effective."

Years later, she returned to Dr. Hernandez for another round.

For most, Stem Cell and Platelet Rich Plasma (PRP) therapy is a mystery. "It's kind of controversial and experimental," said Matthew Riddle, Director of Sales for Celling Biosciences.

The treatments concentrate platelets or stem cells, usually from the patient's own blood. Experts say it is important to always ask the doctor or provider where the "growth factors" are coming from, because in order to ensure they are alive they should be coming from the patient's own blood, fat, or bone marrow. Otherwise, patients can receive "dead" stem cells, which are not nearly as effective.

"We are very adamant to use the patient's own cells," said Riddle, who uses a centrifuge to separate out the blood, saline and growth factors that will be re-injected. "When we inject that into an area, we are telling your body to go heal that spot," said Dr. Hernandez.

"Stem cell treatment is really about trying to take the stem cells out of your body and...inject them back into another part of your body, in order to try and heal whatever part of the body is suffering," said Professor Frow.

"IT'S THE NEW WAVE"

According to researchers, Scottsdale and Phoenix are two of the seven "hot spot" cities in the country.

Arizona State University professors Emma Frow and Dave Brafman spent years studying the industry , and mapping out dozens of clinics in the Valley. They believe there are many more, as some intentionally practice under the radar. "I don't believe right now that there is enough evidence to suggest that they work," said Professor Frow.

"They are unregulated, unproven and for-profit," added Professor Brafman.

The profits are plentiful. "There's cash involved, so this isn't covered by insurance," said Dr. Hernandez.

"PREYING ON PEOPLE'S PAIN"

The thousands in cash is one of many reasons the burgeoning industry is ripe for exploitation.

"The other piece too, it is it is new and upcoming," said Dr. Hernandez.

Many potential patients do not know the first thing about the procedure they are being sold, and doctors say many fall for sales tactics that are practiced at traveling seminars.

"They are preying on people's pain," said Dr. Sorr. "I think its really unethical and it upsets me."

Dr. Sorr believes the seminars are "a scam" that specifically targets an elderly clientele.

"They wine you and dine you. They go through a little dinner presentation and it is not the doctor, it's a marketing agency," he said.

The doctor told ABC15 he has had clients who have been duped, even after he told them they were not ideal candidates for stem cell or PRP therapy.

"It really broke my heart that he spent thousands upon thousands of dollars for something that was worthless.

"I don't agree with how they are done," said Dr. Hernandez. "They inject people and they get money. That's not practicing medicine, that is selling."

Both naturopathic physicians told ABC15 that some patients do not need the treatment, or will get subpar results from the injections. They say it is well known in the industry that some practices will continue to sell in order to reap the thousands in cash.

"ALL OF IT FALLS ON THE PATIENT"

Right now, there is little regulation or oversight of the industry in Arizona.

"Really all of it the falls on the patient, with very little recourse if things go wrong," said Dr. Emma Frow.

During the course of our investigation, ABC15 discovered the Arizona Medical Board and County Health Department do not take complaints or oversee the people performing injections. The federal government has also been slow to implement widespread regulation.

"The FDA has their hands tied," said Dr. Sorr. "There are too many people out there that are doing this that havent had the proper training, they dont have the right experience, the right tools and all that."

There are some larger regulations in Arizona, governing who can handle a needle and perform injections.

Unlike other industries though, including massage therapy, there is no board that checks on licensing or investigates complaints involving botched procedures or alleged fraud.

"The state medical boards, need to become a little bit more involved in sort of identifying, or responding to claims," said Professor Brafman.

"I don't think it would hurt to have it, for sure. At the end of the day it's about protecting the public," said Dr. Hernandez.

For thousands of Arizonans, like Janet McConnell, regenerative medicine has helped heal chronic pain. Before spending thousands thousands though, do your research. "Always get a second opinion," said Dr. Sorr.

"I think this is really a case of buyer beware, or consumer beware," said Professor Frow.

If you are planning on undergoing a stem cell or PRP treatment, click here for questions experts say you should always ask ahead of time.

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Arizona the "wild west" of stem cell therapy; experts say promising therapy ripe for exploitation - ABC15 Arizona

Bone Marrow Stem Cells Comments Off on Arizona the "wild west" of stem cell therapy; experts say promising therapy ripe for exploitation – ABC15 Arizona | November 6th, 2019

A potentially life shattering diagnosis turned into motivation to help others. A Peoria woman is hosting a Be the Match event at the Greater Peoria YMCA on Wednesday.

While many might say Why me? when diagnosed with a rare bone marrow cancer, Marsha Krone continued to fight on, choosing to make the most of the life she has been given.

A mother of three, grandmother of two, lover of sewing, and teacher for over 30 years, Marsha Krone had a lot to smile about. Until an existing blood disorder threatened to steal her joy.

I went to the Mayo Clinic and they did a bone marrow biopsy and they told me that yes, you have progressed to Myelofibrosis. And so what that means is my bone marrow is essentially turning hard. said Marsha Krone, diagnosed in 2016

Krone needs a stem cell transplant. Her sisters were not a match.Despite millions of donors being listed on the registry she is still waiting for the perfect stranger. However of the ten markers needed to match, two are extremely rare, making it difficult, but not impossible to find one.

Her hope comes from faith.

I cant write my story because he writes my story and I just can choose how Im going to live it. So I choose joy. said Krone

As an ambassador for Be the Match she has put on numerous events, including the one coming up at the Greater Peoria YMCA where shes been a member for many years.

We talked about how could we help in this way and she said well lets hold a match event and try to get as many people engaged as we possibly can. Shes not doing this for herself, shes doing this for everyone out there that needs to find a match said President and CEO of the Greater Peoria YMCA, Andy Thornton

The process is simple. You swab your cheek for DNA, provide your contact information, and join the registry where you could match anyone in the world for stem cells and bone marrow.

If someone else was matched from a drive we promoted, that would be really exciting for me to know that I was the go between to helping someones life be saved. said Krone.

The event takes place Wednesday 11/6 from 3 7 P.M. at The Greater Peoria YMCA

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Using her diagnosis to help others, Peoria woman to host 'Be the Match' event - week.com

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Shahid Akhter, editor, ETHealthworld spoke to Dr Na'eem Sadiq, Medical Director, PLEXUS NEURO and STEM CELL RESEARCH CENTER, Bengaluru to know more about stem cell therapy and the challenges associated with it.

STEM CELL : TRENDSStem cell is a word which evokes lot of responses both positive and negative. Few people know that the origin was in the 1800s. The very first bone marrow transplant happened in the year 1968 and then subsequently stem cells have been used for various diseases and much more in blood cancer. They have also been used in chronic neurological disorders, autoimmune disorders and sports injuries.Globally, its all over the world such as in the US, Canada, Germany, China, Ukraine and of course in India as well. In India there are lots of centers and states who have been practicing stem cell technology for quite some time.

STEM CELLS : MARKETThe market is growing since stems cells promise hope for those who have lost hope, where there is no viable treatment and proper cure available for lots of diseases. Stem cells is emerging as a champion for all these people. It was much more available internationally and in the last decade India has taken up.

PLEXUS NEURO AND STEM CELL RESEARCH CENTER- JOURNEYI have been practicing in the field of neuroscience for the last 30 years. Neurosciences is a field where you see patients suffering from chronic diseases. I have been in this field right from early 90s and have been seeing trends changing, but when it comes to neurodegenerative disorders such as Parkinsons, ALS, Multiple Sclerosis, billions of dollars have been spent, new treatment modalities have been found, but nothing has been found to be successful.

We have very strict and rigid eligibility criteria. Once the patient approaches us, we subject the patient to a thorough clinical examination, which lasts anywhere between 2- 3 hours. Once we find that the patient is clinically treatable, or that the patient can be helped, then we subject the patient to other investigations.

The other major difference we have at Plexus is that we do not do only stem cells. Stem cell therapy is a part of our complete regenerative rehabilitation. The program starts after we do the transplant. The patient undergoes rigorous rehabilitation, which includes the entire gamete of practices such as physical therapy, occupational therapy, hand splinting, cognitive rehabilitation therapy, cognitive behavior therapy, speech therapy etc.

We customize and provide a tailor made program as per the patient's needs, with a goal once the patient joins the program and almost all the patients who are in the program get more than what we had aimed at achieving. At the end of the program we evaluate the goals and find that every single patient achieves them. We train the patients as to what they need to do once they finish the program and insist on regular follow up.

We have a team of learned scientists who are all qualified from the UK and our research is ongoing. We are working exclusively in the field of neurosciences to get the best quality of cells and to make it very affordable. Research is on and our data is huge, we will be publishing the results very soon.

PLEXUS : FUTURE PLANSWe have a complete state of the art rehabilitation center where we have some of the best therapists in the world working with us. In fact a few months back we launched one of its kind, Sensory Gym at Plexus and now we have started virtual and augmented reality.

In the last 4-5 years we have received more than 75 national and international awards and we stand as one of the leading regenerative rehabilitation centers not only in India, but in Asia.Our endeavor here is to make the treatment the best possible, to make the cells much more advanced, affordable to also provide the treatment in the shortest possible time.

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Cost is a major challenge in stem cells therapy: Dr Na'eem Sadiq - ETHealthworld.com

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Three researchers at theEli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLAhave received awards totaling more than $18 million from the California Institute for Regenerative Medicine, the states stem cell agency.

The recipients are Dr. Sophie Deng, professor of ophthalmology at the UCLA Stein Eye Institute;Yvonne Chen, a UCLA associate professor of microbiology, immunology and molecular genetics; and Dr. Caroline Kuo, a UCLA assistant clinical professor of pediatrics. The awards were announced at a CIRM meeting today.

Dengs four-year, $10.3 million award will fund a clinical trial for a blinding eye condition called limbal stem cell deficiency. Limbal stem cells are specialized stem cells in eye tissue that help maintain the health of the cornea. Because of genetic defects or injuries caused by infections, burns, surgeries or other factors, some people do not have enough limbal stem cells, which results in pain, corneal scarring and blindness.

The approach she is testing involves extracting a small number of limbal stem cells from a persons eye, multiplying them in a lab, and then transplanting them back into the eye, where they could regenerate the cornea and restore vision. The research will be conducted in collaboration with theUCLAUCI Alpha Stem Cell Clinic, a partnership between UCLA and UC Irvine.

The grants awarded to Chen and Kuo are for projects that are heading toward the FDAs investigational new drug application process, which is required by the agency before a phase 1 clinical trial the stage of testing that focuses on a treatments safety.

Chens two-year, $3.2 million award will fund efforts to create a more effectiveCAR T cell therapyfor multiple myeloma, a blood cancer that affects white blood cells. The research will evaluate a specialized form of CAR T therapy that simultaneously targets two markers, BCMA and CS1, commonly found on multiple myeloma cells. CAR T therapies that target BCMA alone have been effective in clinical trials, but the presence of BCMA on multiple myeloma cells is not uniform.

Previous research has shown that the marker CS1 is present in around 90% of multiple myeloma cells. A CAR T therapy that targets both markers could potentially help more patients and reduce the likelihood of a cancer relapse.

Kuos 2 1/2-year, $4.9 million award, will support the development of a stem cell gene therapy for a deadly immunodeficiency called X-linked hyper IgM syndrome, or XHIM.

The syndrome, which is caused by a mutation in the CD40LG gene, results in invasive infections of the liver, gastrointestinal tract and lungs. Currently, the only potential cure is a bone marrow transplant from a matched donor, which carries life-threatening risks and is often less effective for XHIM patients than patients with other forms of immune deficiency. Even with current treatments, only 30% of people with the syndrome live to age 30.

Kuo will evaluate a stem cell gene therapy that corrects the genetic mutation that causes XHIM. After removing blood-forming stem cells from a person with the syndrome, the therapy would use a genetic engineering technique called CRISPR to insert a correct copy of the affected gene into the DNA of the stem cells. The corrected blood-forming stem cells would be infused back into the patient, where they could regenerate a healthy immune system.

She will collaborate with Dr. Donald Kohn, a UCLA distinguished professor of microbiology, immunology and molecular genetics who has successfully treated two other immune deficiencies bubble baby disease and X-linked chronic granulomatous disease with a similar therapy.

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Three UCLA scientists receive grants totaling more than $18 million - UCLA Newsroom

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NEW YORK, Nov. 05, 2019 (GLOBE NEWSWIRE) -- BrainStorm Cell Therapeutics, Inc. (NASDAQ:BCLI), a leading developer of adult stem cell therapies for neurodegenerative diseases, today announced that the Company will hold a conference call to update shareholders on financial results for the third quarter ended September 30, 2019, and provide a corporate update, at 8:00 a.m., Eastern Standard Time, on Thursday, November 14, 2019.

BrainStorms President & CEO, Chaim Lebovits, will present a corporate update, after which, participant questions will be answered. Joining Mr. Lebovits to answer investment community questions will be Ralph Kern, MD, MHSc, Chief Operating Officer and Chief Medical Officer, and Preetam Shah, PhD, Chief Financial Officer.

Participants are encouraged to submit their questions prior to the call by sending them to: q@brainstorm-cell.com; Questions should be submitted by 5:00 p.m., Eastern Standard Time, Tuesday, November 12.

The investment community may participate in the conference call by dialing the following numbers:

Those interested in listening to the conference call live via the internet may do so by visiting the Investors & Media page of BrainStorms website at http://www.ir.brainstorm-cell.com and clicking on the conference call link.

A webcast replay of the conference call will be available for 30 days on the Investors & Media page of BrainStorms website:

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

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 to Announce Third Quarter Financial Results and Provide a Comprehensive Corporate Update - Yahoo Finance

Bone Marrow Stem Cells Comments Off on BrainStorm Cell Therapeutics to Announce Third Quarter Financial Results and Provide a Comprehensive Corporate Update – Yahoo Finance | November 5th, 2019

According to results of a study published in Blood, children and young adults with relapsed/refractory B-cell acute lymphoblastic leukemia (ALL) who had minimal residual disease (MRD) prior to treatment and received high-dose preconditioning chemotherapy were most likely to respond to a second-generation CD-19 chimeric antigen receptor-T cell (CAR-T) therapy.1

Although it has been estimated that 90% or more of pediatric patients witha diagnosis of ALL will respond to multi-agent chemotherapy, the prognosis forthose with relapsed/refractory disease remains poor. One CD19-directed CAR-Ttherapy, tisagenlecleucel, is approved by the US Food and Drug Administration inpatients up to age 25 years with B-cell precursor ALL who either have refractorydisease or have experienced a second or later relapse.2

This open label, nonrandomized, phase 1 study (Clinical Trial Identifier: NCT01860937), evaluated the toxicity, feasibility, and response of 19-28z CAR-T therapy, a second-generation CD19-directed CAR-T therapy involving T cells expressing a chimeric receptor composed of an anti-CD19 antibody binding site and intracellular domains from the T-cell coactivating receptors, CD28 and the CD3-zeta chain3 in children and young adults up to 25 years of age with very high-risk ALL.1 Inclusion criteria included at least 2 relapses, early bone marrow relapse following complete response (CR), intermediate/late CR with poor response to re-induction therapy, or those with refractory disease, or ineligibility for allogeneic hematopoietic stem cell transplantation (allo-HSCT) or additional chemotherapy.1

The age range of the 25 patients treated with 19-28z CAR-T therapy onstudy was 1 to 22.5 years, with a median age of 13.5 years. Preconditioningchemotherapy involved high-dose cyclophosphamide (15 patients) and low-dosecyclophosphamide (8 patients), with 3 patients in each subgroup also receivingfludarabine.1

Regarding the feasibility of this approach, the prespecified CAR-Tcell dose was achieved for all patients for whom the 19-28z CAR-T therapyprocedure was undertaken.1

With respect to treatment toxicity,approximately one-third of patients experienced a grade 3/4 adverse event,including cytokine release syndrome (CRS) and neurotoxicity in 16% and 28% ofpatients, respectively. With the exception of 1 patient with grade 4 CRS andneurotoxicity who died following refractory Stenotrophomonas septic shock,these adverse events were reversible.1

Of the 24 patients includedin the response analysis, 75% achieved either a CR or a CR with incompletecount recovery (CRi). In the subsets of patients receiving preconditioningchemotherapy with either high- or low-dose cyclophosphamide, the CR/CRi rateswere 94% and 38%, respectively. Furthermore, treatment response was influencedby disease burden as evidenced by the considerably higher CR/CRi rate inpatients with baseline minimal residual disease (ie, less than 5% bone marrowblasts; 93%) compared with morphological evidence of disease at baseline (5% orhigher bone marrow blasts; 50%).1

The CR/CRi rate for thesubset of patients with pretreatment MRD treated with high-dose cytarabine was100%.1

Consolidation allo-HSCT was performed in 83% (15) of the patientsresponding to CAR-T therapy, with a median time from CAR-T infusion toallo-HSCT of 57 days. At a median follow-up of 28.6 months for respondingpatients, over half of these patients (8) were alive and had no evidence ofdisease.1

In their concluding remarks, the study authorscommented that thisanalysis has allowed us to determine the toxicity profile, confirm feasibility,evaluate response of this approach, and provide a direct comparison of the sameCD19-specific CAR T cell product that was previously published[3] inadult patients for the same indication.

The authors went on to highlight the findingof a reversible toxicity profile in the patients within their study as well asthe impact of preconditioning chemotherapy dose intensity and minimal pretreatmentdisease burden on response.

They further noted that within this cohort,the long-term persistence of response is encouraging, and in our primarilytransplant-naive patient population, the ability to proceed to allo-HSCT hasdemonstrated a favorable overall survival, manageable toxicity, and limitedincidence of relapse.

References

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19-28z CAR-T Therapy in Children and Young Adults With Relapsed/Refractory ALL: Promising Early Results - Cancer Therapy Advisor

Bone Marrow Stem Cells Comments Off on 19-28z CAR-T Therapy in Children and Young Adults With Relapsed/Refractory ALL: Promising Early Results – Cancer Therapy Advisor | November 5th, 2019

Stem cells from the patients body when isolated and administered at an appropriate time and at the right place, with the right dose, is expected to help the patient in various ways

Stem Cells In The Body

All humans are born and develop from a small tiny structure called an egg. The cells in the egg have a tremendous potential to develop, multiply and form different cells that are functional in the body. These cells are called mother cells or in scientific terms, they are called stem cells. And all human beings have these stem cells preserved in the body. It is these cells that help us in every day wear and tear and also for tissue repair.

The Body Can Heal Itself

Most of the cells in our body have a definite lifespan that need to be replaced by new cells. The stem cell reserves in the body make up for this and it is done without our knowledge! In fact, any cut or injury, external or internal is healed by the bodys innate mechanism. Our intelligent body recognises the signal of injury and recruits the required stem cells. These stem cells transform themselves into the cells that are required for the repair of the injury and it is always many types of cells in various permutations and combinations.

Where Stem Cells Reside

Bone marrow can be considered as the manufacturing unit of stem cells as it is continuously making blood cells and keeps our circulatory system working perfect all the time. Circulating blood is another source of stem cells, because it works as a courier, carrying cells and other essential enzymes, hormones from one organ to the other in the body. The body converts all the extra material into fat which gets accumulated around the belly. This fatty tissue works like a fixed deposit of stem cells.

Stem cells either from the donor (allogenic) or from the patient (autologous) are being used for more than 50 years and especially for treatment. Blood cancers and other blood-related diseases can be cured using a perfect matched donor stem cells obtained from bone marrow. Patients suffering from organ cancers like breast cancer etc. are given autologous stem cells as a supportive treatment along with chemotherapy and/or radiation.

Protocols for these treatments are standardised globally and considered as standard-of-care. In recent years, umbilical cord blood derived stem cells are being used as an alternative to bone marrow, especially in the paediatric age group. People fall victim to numerous degenerative diseases which occur, as the repairing stem cell system from the body fails slowly with age. Stem cells from the patients body when isolated and administered at an appropriate time and at the right place, with the right dose, is expected to help the patient in various ways. It may also replace, rejuvenate or restore the damaged tissues.

Our body carnes its own repairing kit in the form of stem cells and the body tries its level best to make use of these stem cells to ward off diseases. However, it is possible that with age, the bodys power to recruit and make use of the stem cells diminishes slowly. This is when dreadful degenerative diseases like diabetes, arthritis, Parkinsons disease and heart problems, set in. Heres what the clinical applications of regenerative medicine have found novel mechanisms of:

It is increasingly observed that this kind of autologous therapy takes care of the root cause of disease and offers benefits to patients to whom there is no further solution in other modalities of treatment.

Since each tissue and organ of our body is made up of cells that are derived from the egg cell, any disease which is due to derangement or degeneration of cells can be cured using autologous cellular therapy. And though the list can be endless, here are some examples where there have been very promising results:

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How Stem Cells Can Heal The Body - Version Weekly

Bone Marrow Stem Cells Comments Off on How Stem Cells Can Heal The Body – Version Weekly | November 4th, 2019

This level of disease stabilization has not been observed to this date in approved or investigational ALS therapies.

-Mr Chaim Lebovits, CEO, Brainstorm Cell Therapeutics

In May of this year, I published an article on Brainstorm Cell Therapeutics (BCLI). This small company is developing a Mesenchymal stem cell product called NurOwn, which is in late phase 3 trials targeting Amyotrophic lateral sclerosis (ALS), or Lou Gehrig's disease. My article was bearish, deploring not only the company's cash position, but also phase 2 trial data. The article can be read here.

That article received a lot of critical comments from the ALS community. That made me realize that a fair overview of the issues could be best addressed by going through the comments, as well as my own coverage, and by asking BCLI management, specifically its CEO, Chaim Lebovits, to clarify some of these issues. So that's what I did. I emailed a set of 11 questions to Mr Lebovits, and he was kind enough to respond to them in great detail. The entire interview, sans any edits, is available to Total Pharma Tracker members.

Mr Lebovits has been with BCLI for well over 12 years, joining in 2007 as president and also becoming the CEO in 2015. He has helped develop NurOwn through its preclinical stage to its current stage, and is therefore just the right person to talk to if we want to understand NurOwn and BCLI.

I began by asking him to locate NurOwn in the ALS therapy space and where it stands with respect to competitors. What's its mechanism of action, and how does that MOA distinguish it from competition?

Mr Lebovits said that there are "currently 4 products active in phase 3 ALS clinical trials (Brainstorm (NurOwn, autologous MSC-NTF cells secreting neurotrophic factors), Orion (levosimendan, muscle troponin calcium sensitizer), Orphazyme (arimochlomol, heat shock protein enhancer), and Biogen (SOD1, antisense oligonucleotide)." Top line data from these ALS phase 3 trials is expected in 2020 (Q4 2020 for Brainstorm) and Orion, 2021 (Orphazyme), and 2022 (Biogen). He discussed a number of earlier stage compounds, as well as various stem cell therapies. He said that what distinguishes NurOwn among ALS therapies is that it "confers both neuroprotection and immunomodulation by delivering neuronal survival factors and immune regulatory molecules, including microRNA directly to the CNS compartment at or near the site of disease, and therefore directly addresses two important ALS disease mechanisms."

Among stem cell therapies, Mr Lebovits said that NurOwn distinguishes itself by being autologous and because it can produce high levels of neurotrophic factors. Moreover, unlike most stem cell competitors, it's delivered directly into the spinal fluid through bimonthly lumber punctures, unlike others which need an invasive surgical procedure "that carries considerable morbidity."

This feature it shares with a competing product from Corestem. However, it's differentiated from Corestem because "NurOwn is more convenient than the Corestem product as a single bone marrow cell harvest due to validated cryopreservation, whereas the Corestem product requires repeat bone-marrow aspiration for each treatment."

My next question was a technical question about pharmacoresistance. I wanted to know how NurOwn is managing to cross the blood-spinal cord barrier despite the strong pharmacoresistance (body's resistance to drugs) seen in ALS, specifically for disease-modifying neurotrophic factors. What was it about NurOwn's delivery mechanism that the company thinks is overcoming this natural resistance. So I asked: "Talking about MOA, pharmacoresistance is a disease driving mechanism in ALS. Can you discuss NurOwns delivery mechanism vis-a-vis the inability of neurotrophic factors to effectively cross the blood-brain barrier, or, specifically, the blood-spinal cord barrier (BSCB)? Please correlate that discussion regarding the observed increase in CSF NTFs post treatment as seen in the phase 2 trial."

Mr Lebovits explained this with great clarity - for his entire response, take a look at the complete interview. Broadly, what he said was that NurOwn, being delivered through lumber puncture directly into the spinal fluid, has an advantage. Moreover, the cells secrete neuronal survival factors as well as molecules that regulate the immune system, so that they are able to survive and overcome the pharmacoresistance. Systemically administered NTFs are unable to do that.

As he said, "In the phase 2 trial, CSF biomarkers obtained just prior to treatment and two weeks afterwards demonstrated that MSC-NTF cell secreted neurotrophic factors were significantly increased post treatment and correlated with the reduction in inflammatory biomarkers, consistent with the proposed mechanism of action."

My third and fourth questions related to aspects of the phase 2 study. One, comparison of safety and efficacy data with competitors, and two, the relevance of the reported Caspace-3 reduction of 60% in responders versus 30% in non-responders.

Mr Lebovits said that although the phase 2 study was not powered for efficacy, it exhibited a "level of disease stabilization (that) has not been observed to this date in approved or investigational ALS therapies." About the ongoing phase 3 study, he said the following:

Those who read my original article will recall I was particularly puzzled by the increased occurrence of serious adverse events in active-treatment groups than in placebo groups. 8/36 or 22.2% patients in the treatment arm had an SAE compared to only one out of 12 placebo patients, or 8.3%. Most SAEs were related to the progression of the underlying ALS, most commonly dysphagia. No SAEs were related to study treatment. So I asked Mr Lebovits how this data could be interpreted in the most positive way.

According to him, this decline was not an effect of treatment itself and simply indicated the need for repeat dosing in this patient group. His exact response was as follows:

The MSC-NTF treated group had a slightly more rapid rate of decline compared to the placebo group in the three-month run-in period and most ALS disease progression in the treated group was seen toward the end of the clinical trial, long after a single transplantation. In fact, the bulbar subscale, that includes assessment of swallowing, was the subscale most improved after MSC-NTF treatment in rapid progressors, suggesting that the late decline in motor function was not an adverse effect of treatment per se. Hence the need for repeated dosing.

Last week, the DSMB recommended continuation of the phase 3 trial without any modification. This was major good news, so we asked him about this. Mr Lebovits said that this was a second interim safety review, and there was no significant safety concerns. Therefore, the DSMB recommended no modification in protocol, and no other interim analysis is planned. Phase 3 data will be available by mid-2020 according to this interviewer's reading of the press release.

Now we moved on to another critical aspect of our analysis - funds, or rather, the lack of it. Since this is an important issue, here's the exact exchange we had.

Dr. Ashok Dutta: How does the company plan to fund its operations through the next couple years until the lead development candidate is approved and commercialized? Given the weak financial position, does Brainstorm see the possibility for ATM operations, or thinks about selling rights in regions like China, Japan or Europe to increase the financial condition?

CEO Chaim Lebovits: As you are aware we do receive proceeds from the hospital exemption pathway and also receive grant funding from CIRM and IIA. These avenues have allowed to fund and continue with our trials over the years with non-dilutive financing. From a business standpoint as our ALS phase 3 trial is now fully enrolled, the management team continues to hold high level conversation with some of the leading global pharmaceutical and biotechnology companies. We are actively engaged in strategic partnering and collaboration discussions and although we cannot disclose the details of our conversations due to NDAs we signed with them... we are exploring several opportunities with key interested parties to advance the opportunities for NurOwn development and commercialization. As you have rightly pointed out, we have a $20mm ATM facility in place with Raymond James. We may activate the ATM as required and raise up to $20mm by selling our stock at the market only if the prices are attractive to us. So far as of end of Q319, we have not activated the ATM. If the need arises and the prices are attractive to us, we may employ this tool to raise capital.

This is reassuring that the company intends to focus on non-dilutive financing. The ATM facility, coupled with the grants, should ideally see them through the approval phase. We still wonder how they will manage marketing and sales. Perhaps those commercialization NDAs they have signed will help.

Next, we discussed market potential and a question about a recent patent grant. The CEO's detailed responses can be found in the complete interview material.

The strong involvement of the ALS community impressed us previously, so we now asked the CEO about the recent roundtable convention they had with ALS advocacy groups. Since this will be important for the ALS community as a whole, here's Mr Lebovits' entire response on the question:

Finally, we asked him what we ask everyone: Give us three simple and straightforward reasons why investors would be interested. Here's what he said:

Thanks to the ALS community for inspiring us to conduct this interview, and to Mr Chaim Lebovits, CEO of Brainstorm Cell Therapeutics, for answering our questions.

Thanks for reading. At the Total Pharma Tracker, we interview management of important small biotech doing disruptive work in healthcare. Our members are given exclusive access to these interviews, which helps them with additional primary resource in doing DD on their investments. Sometimes, extracts from these interviews may be published for everyone; but TPT members always get the exclusive view.

Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

Additional disclosure: General Disclaimer - This is to confirm that Avisol Capital Partners has neither requested, nor been offered, any monetary compensation for conducting this interview, by any party other than Seeking Alpha.

Also to be noted, this was an emailed questionnaire, and certain editorial material is present in this version, which may or may not reflect BCLI or its CEO's position on the issues discussed.

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Interview With Chaim Lebovits, CEO Of Brainstorm Cell Therapeutics - Seeking Alpha

Bone Marrow Stem Cells Comments Off on Interview With Chaim Lebovits, CEO Of Brainstorm Cell Therapeutics – Seeking Alpha | November 4th, 2019

In September end, a good news greeted the biomedical world when a team led by Takanori Takebe at Cincinnati Children's Hospital Medical Center succeeded at growing a connected set of three organs: the liver, pancreas and biliary ducts, in the lab, from human stem cells. The findings were published in journal Nature.

While human organoids already provide a sophisticated tool for research, the connected set of three organs, for the first time, allow scientists to study how human tissues work in concert. This was dubbed as a significant step forward.

In October, another news came. At the annual meeting of the Society for Neuroscience, researchers said that brain cell clusters prepared in the lab- a type of organoid- show abnormal behaviour as compared to the normal brain cells.

They said that the cells in these clumps had ambiguous identities and made more stress molecules than cells taken directly from human brains. However, these abnormalities were found to be alleviated a little bit when the implanted into a more hospitable environment - a mouses brain.

What are organoids?

With the available technology, scientists can grow a group of cells in laboratories into three-dimensional, miniature structures that mimic the cell arrangement of a fully-grown organ.

This is done using stem cells.

Stem cells are special human cells that have the ability to develop into many different cell types, from muscle cells to brain cells.

The embryonic stem cells that are derived from unused embryos (These are created from an in vitro fertilization procedure and used for scientific research) are pluripotent, meaning, they can turn into any type of cell.

On the other hand are adult stem cells. They are derived from fully developed tissues, like the brain, skin, and bone marrow. These cells often have capability of turning into only certain types of cells. For example, a stem cell derived from the liver will only generate more liver cells.

However, the adult stem cells can be manipualted in the laboratory to act like embryonic stem cells. These are called induced pluripotent stem cells. (The technique was developed in 2006). However, scientists are yet to find adult pluripotent stem cells that can develop every kind of cell and tissue.

When scientists create right environment in the laboratory for them, these stem cells follow their own genetic instructions to develop into tiny structures that resemble miniature organs composed of many cell types.

Using these, researchers have been able to produce organoids that resemble the brain, kidney, lung, intestine, stomach, and liver etc.

For example, in the three-organ research mentioned above, Dr Takebe started with stem cells from human skin cells and then guiding and prodding those stem cells to form two very early-stage "spheroids" of cells loosely termed the foregut and the midgut (In human embryos, these form late in the first month of gestation. Over time, they merge and morph into the organs that constitute the digestive tract).

The spheroids were first placed next to each other in a lab dish suspended in a gel used to support organoid growth, then placed on top of a thin membrane that covered a carefully mixed batch of growth medium.

From this point on, the cells knew what to do, and 70 days later, the mini organoids began processing bile acids as if they were digesting and filtering food.

Why are organoids important?

The technique to develop organoids was named by The Scientist as one of the biggest scientific advancements of 2013.

Organoids are an excellent tools to study biological processes like uptake of nutrients, drug transport, secretion of hormones and enzymes etc. This way, diseases related to malabsorption of nutrients, and metabolism-related diseases like obesity, diabetes, insulin resistance can be studied at the cellular-level.

Recently, scientists at the at Memorial Sloan Kettering created a tumor organoid to develop a more accurate rectal cancer model.

In the case of the human brain, organoids opens a window to understand some of the most complicated and hidden aspects of our own biology. They can be used to study neuropsychiatric or neurodevelopmental diseases like schizophrenia or autism spectrum disorder, which are uniquely human diseases that affect the whole human genome.

Organoids also provide a window into how cells interact with each other and their environment. They can be used to create cellular models of human disease, which can be studied in the laboratory to better understand the causes of disease and identify possible treatments. The effects of different drugs and be tested.

Scientists have even used gene editing techniques (CRISPR-Cas9) on the stem cells to to introduce targeted mutations in genes corresponding to two different kidney diseases. When these modified pluripotent cells grew into human kidney organoids, they exhibited the diseases.

Using such organoids relieves the scientific community from experimenting on human and animal subjects. Also, certain treatments that would be unethical to administer on the latter, can be tested on the organoids.

With organoids, researchers can produce a limitless supply of tissue from each patient. This will also be extremely useful for the study of rare diseases, where the number of patients on which to conduct research and test treatments is limited.

Organoids are also being used to develop personalised and precision medicine.

For example, it was found that repairing the CFTR protein could give relief to a patient suffering from non-cystic fibrosis, an inherited disease caused due to a gene mutation. Using the Intestinal organoids grown from a patients stem cells, the doctors could quantify the patients response to the CFTR modulating therapy.

Organoids can have significant therapeutic applications. For example, pluripotent stem cells derived from a diabetes patient could be transformed into insulin-producing beta-like cells.

Organoids also offer an incredible opportunity to study developmental biology. Using them, for example, we can learn more about how organs are formed in embryonic stages and associated disorders.

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Growing Human Organs In A Lab: As Scientists Develop Pathbreaking Three-Organ System, Heres All You Need To Know - Swarajya

Bone Marrow Stem Cells Comments Off on Growing Human Organs In A Lab: As Scientists Develop Pathbreaking Three-Organ System, Heres All You Need To Know – Swarajya | November 4th, 2019

SAN FRANCISCO--(BUSINESS WIRE)--

Imago BioSciences, Inc., a clinical-stage biotechnology company developing innovative treatments for malignant and life-threatening diseases of the bone marrow, today announced the appointment of James D. Watson as Chief Business Officer.

James has a long history of success in biotech financing, business development, and commercial planning. That experience will guide the strategic direction and growth of Imago, said Hugh Young Rienhoff, Jr., M.D., Chief Executive Officer of Imago BioSciences. His experience in corporate development and financings will help ensure the advancement of bomedemstat (IMG-7289) through clinical development in myelofibrosis and other indications.

Mr. Watson most recently served as Chief Business Officer at Sigilon Therapeutics where he closed a $473 million strategic partnership with Eli Lilly for a treatment for Type 1 diabetes. Prior to Sigilon, Mr. Watson was Chief Business Officer for Alvine Pharmaceuticals and led strategy, corporate development, new product planning, and finance during which he closed a transaction giving AbbVie the right to acquire Alvine for $345 million. Previously, Mr. Watson was CEO of a San Francisco-based, boutique investment bank focused on mergers, acquisitions, partnering, and raising capital for life science companies.

I am excited to join Imago BioSciences at this important stage of growth, helping the company realize its full potential. Bomedemstat has great promise for the day-to-day management of myelofibrosis and it offers the additional possibility of altering the course of this disease. Furthermore, its broader myeloproliferative neoplasm platform and expertise in life-threatening diseases of the bone marrow represent an opportunity to address areas of high unmet clinical need and to build a valuable company. said Mr. Watson.

About Bomedemstat (IMG-7289)

Bomedemstat is a small molecule discovered by Imago BioSciences that inhibits lysine-specific demethylase 1 (LSD1 or KDM1A), an enzyme essential for production and normal function of megakaryocytes and for self-renewal of malignant hematopoietic stem or progenitor cells. Megakaryocytes are the primary producer of growth factors and cytokines that drive myelofibrosis pathogenesis.

In non-clinical studies, bomedemstat demonstrated robust in vivo efficacy as a single agent and in combination with other therapeutics across a range of myeloid malignancy models including the myeloproliferative neoplasms encompassing myelofibrosis, essential thrombocythemia and polycythemia vera. The U.S. Food and Drug Administration (FDA) has granted Fast Track designation to bomedemstat for the treatment of myelofibrosis which is currently being studied in an international Phase 2b study. Additional clinical studies in hematologic disorders will begin in 2020.

About Imago BioSciences

Imago BioSciences is a clinical-stage, private therapeutics company focused on malignant and life-threatening diseases of the bone marrow. The initial clinical focus is on myeloproliferative neoplasm (MPN) disorders including myelofibrosis, essential thrombocythemia and polycythemia vera. Investors in Imago include a fund managed by Blackstone Life Sciences, Frazier Healthcare Partners, Omega Funds, Amgen Ventures, MRL Ventures Fund, HighLight Capital, Pharmaron, Greenspring Associates and Xeraya Capital.

View source version on businesswire.com: https://www.businesswire.com/news/home/20191104005265/en/

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Imago BioSciences Further Expands Executive Team with Appointment of James D. Watson as Chief Business Officer - Yahoo Finance

Bone Marrow Stem Cells Comments Off on Imago BioSciences Further Expands Executive Team with Appointment of James D. Watson as Chief Business Officer – Yahoo Finance | November 4th, 2019

The proposal was nothing fancy. Shawn Russell arrived at the pub, placed a pint on the table in front of Sarah Hodgetts and asked her to marry him.

It was out of the blue, with no ring, nothing. And I thought: Yeah, thats a really good idea, we should just get married, says Sarah. Id been in love with him for ages.

Their story began seven years earlier, in 2009, also in a pub in north London. He was sitting in his flat cap, good Yorkshireman that he was, and was reading the sports pages. I had just got off the Tube after work. From my perspective, it was love at first sight, says Sarah.

But dating wasnt easy. Shawn, a boarding school-educated boy from an Army family, who had lost his mother to leukaemia when he was two, worked as a picture editor at The Telegraph. Although Sarah was down the road in Westminster, where she worked as a civil servant (and still does), their hours were long and their schedules largely incompatible. They managed just six months initially.

It was a disaster trying to date, so we ended up with a firework display of an argument and decided there was no way we could, says Sarah over coffee near her office.

Their split didnt last, however; they had far too much in common. Besides his ridiculous sense of humour, Shawn was so into news and politics, and I worked in politics, so we couldnt not communicate. We realised we were incredibly good friends, says Sarah. Plus, she adds with a smile, he was a very attractive, 6ft 4in blue-eyed man who I was completely smitten with.

Over time, they got back together in a non-committal way, sharing weekends when their busy lives permitted. It was during this period, three-and-a-half years ago, that Shawn, then 44, proposed. The following week, he moved in with Sarah, then 41, and her son Eddy, 10, from a previous relationship, in Kings Cross, and, like all newly engaged couples, they started making plans for their future. A pair of recovering workaholics, they were going to transform their lives.

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I lost my fianc to leukaemia, but in my dreams hes just working the night shift - Telegraph.co.uk

Bone Marrow Stem Cells Comments Off on I lost my fianc to leukaemia, but in my dreams hes just working the night shift – Telegraph.co.uk | November 3rd, 2019

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Newswise Three researchers at theEli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLAhave received awards totaling more than $18 million from the California Institute for Regenerative Medicine, the states stem cell agency.

The recipients are Dr. Sophie Deng, professor of ophthalmology at the UCLA Stein Eye Institute;Yvonne Chen, a UCLA associate professor of microbiology, immunology and molecular genetics; and Dr. Caroline Kuo, a UCLA assistant clinical professor of pediatrics. The awards were announced at a CIRM meeting today.

Dengs four-year, $10.3 million award will fund a clinical trial for a blinding eye condition called limbal stem cell deficiency. Limbal stem cells are specialized stem cells in eye tissue that help maintain the health of the cornea. Because of genetic defects or injuries caused by infections, burns, surgeries or other factors, some people do not have enough limbal stem cells, which results in pain, corneal scarring and blindness.

The approach she is testing involves extracting a small number of limbal stem cells from a persons eye, multiplying them in a lab, and then transplanting them back into the eye, where they could regenerate the cornea and restore vision. The research will be conducted in collaboration with theUCLAUCI Alpha Stem Cell Clinic, a partnership between UCLA and UC Irvine.

The grants awarded to Chen and Kuo are for projects that are heading toward the FDAs investigational new drug application process, which is required by the agency before a phase 1 clinical trial the stage of testing that focuses on a treatments safety.

Chens two-year, $3.2 million award will fund efforts to create a more effectiveCAR T cell therapyfor multiple myeloma, a blood cancer that affects white blood cells. The research will evaluate a specialized form of CAR T therapy that simultaneously targets two markers, BCMA and CS1, commonly found on multiple myeloma cells. CAR T therapies that target BCMA alone have been effective in clinical trials, but the presence of BCMA on multiple myeloma cells is not uniform.

Previous research has shown that the marker CS1 is present in around 90% of multiple myeloma cells. A CAR T therapy that targets both markers could potentially help more patients and reduce the likelihood of a cancer relapse.

Kuos 2 1/2-year, $4.9 million award, will support the development of a stem cell gene therapy for a deadly immunodeficiency called X-linked hyper IgM syndrome, or XHIM.

The syndrome, which is caused by a mutation in the CD40LG gene, results in invasive infections of the liver, gastrointestinal tract and lungs. Currently, the only potential cure is a bone marrow transplant from a matched donor, which carries life-threatening risks and is often less effective for XHIM patients than patients with other forms of immune deficiency. Even with current treatments, only 30% of people with the syndrome live to age 30.

Kuo will evaluate a stem cell gene therapy that corrects the genetic mutation that causes XHIM. After removing blood-forming stem cells from a person with the syndrome, the therapy would use a genetic engineering technique called CRISPR to insert a correct copy of the affected gene into the DNA of the stem cells. The corrected blood-forming stem cells would be infused back into the patient, where they could regenerate a healthy immune system.

She will collaborate with Dr. Donald Kohn, a UCLA distinguished professor of microbiology, immunology and molecular genetics who has successfully treated two other immune deficiencies bubble baby disease and X-linked chronic granulomatous disease with a similar therapy.

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Three UCLA scientists receive grants totaling more than $18 million - Newswise

Bone Marrow Stem Cells Comments Off on Three UCLA scientists receive grants totaling more than $18 million – Newswise | October 31st, 2019

Ezer Mizion, the worlds largest Jewish bone marrow registry, will host its Evening of Heroes for the Teaneck, Bergenfield, and New Milford communities on Saturday, November 9, at Congregation Keter Torah in Teaneck.

The evening begins with a musical Havdalah and mini-concert by the chasidic superstar Shulem Lemmer, the first chasidic singer to sign with Universal Records. Then Ezer Mizion will introduce IDF heroes who defend the State of Israel and have saved lives with their stem cells.

A stem cell recipient will recount the day he received a call letting him know that Ezer Mizion had identified a stem cell match for him a match that saved his life. Bret Stephens, a New York Times Pulitzer Prize-winning columnist, and Nachum Segal will give a fireside chat about innovations from Israel, including the export of more than 60 percent of Ezer Mizions stem cell transplants.

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There will be a swabbing station for people who meet the basic criteria for donations. Israeli wines and shuk foods will be served.

The program aims to bring awareness of the organizations role in saving hundreds of lives around the world every year with its growing bone marrow registry. It has more than 1 million potential stem cell donors, and more than 550,000 of these donors are from the IDF. There is no cost to attend the adults-only evening; RSVPs are requested. For more information, go to eveningofheroes.com; email Ezer Mizions national director of development, Ryan Hyman, at ryan@ezermizionusa.org or call him at (718) 853-8400, ext. 109.

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Ezer Mizion's Evening of Heroes is November 9 in Teaneck - The Jewish Standard

Bone Marrow Stem Cells Comments Off on Ezer Mizion’s Evening of Heroes is November 9 in Teaneck – The Jewish Standard | October 31st, 2019

TEL AVIV, Israel, Oct. 31, 2019 /PRNewswire/ -- BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a clinical-stage biopharmaceutical company focused on oncology, announced today that it will deliver the following poster presentations at the Society for Immunotherapy of Cancer(SITC) 34th Annual Meeting to take place November 6-10, 2019 at the Gaylord National Hotel & Convention Center in Baltimore, Maryland:

About BL-8040

BL-8040 is a short synthetic peptide that functions as a high-affinity best-in-class antagonist for CXCR4, a chemokine receptor over-expressed in many human cancers, where it has been shown to be correlated with poor prognosis, and plays a key role in tumor growth, invasion, angiogenesis, metastasis and therapeutic resistance. CXCR4 is also directly involved in the homing and retention of hematopoietic stem cells (HSCs) and various hematological malignant cells in the bone marrow.

In a number of clinical and pre-clinical studies, BL-8040 has shown a critical role in immune cell trafficking, tumor infiltration by immune effector T cells and reduction in immunosuppressive cells within the tumor niche, turning "cold" tumors, such as pancreatic cancer, into "hot" tumors (i.e., sensitizing them to immune check point inhibitors). BL-8040-mediated inhibition of the CXCR4-CXCL12 (SDF-1) axis has also shown robust mobilization of HSCs for transplantation in hematological malignancies.

BL-8040 was licensed by BioLineRx from Biokine Therapeutics and was previously developed under the name BKT-140.

About BioLineRx

BioLineRx is a clinical-stage biopharmaceutical company focused on multiple oncology indications. The Company'slead program, BL-8040, is a cancer therapy platform currently being evaluated in a Phase 2a study in pancreatic cancer in combination with KEYTRUDA and chemotherapy under a collaboration agreement with MSD. BL-8040 is also being evaluated in a Phase 2b study in consolidation AML and a Phase 3 study in stem cell mobilization for autologous bone-marrow transplantation. In addition, the Company has an ongoing collaboration agreement with Genentech, a member of the Roche Group, evaluating BL-8040 in combination with Genentech's atezolizumab in two Phase 1b/2 solid tumor studies.

BioLineRx is developing a second oncology program, AGI-134, an immunotherapy treatment for multiple solid tumors that is currently being evaluated in a Phase 1/2a study.

For additional information on BioLineRx, please visit the Company's website at http://www.biolinerx.com, where you can review the Company's SEC filings, press releases, announcements and events. BioLineRx industry updates are also regularly updated on Facebook,Twitter, and LinkedIn.

Various statements in this release concerning BioLineRx's future expectations constitute "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. These statements include words such as "may," "expects," "anticipates," "believes," and "intends," and describe opinions about future events. These forward-looking statements involve known and unknown risks and uncertainties that may cause the actual results, performance or achievements of BioLineRx to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. Some of these risks are: changes in relationships with collaborators; the impact of competitive products and technological changes; risks relating to the development of new products; and the ability to implement technological improvements. These and other factors are more fully discussed in the "Risk Factors" section of BioLineRx's most recent annual report on Form 20-F filed with the Securities and Exchange Commission on March 28, 2019. In addition, any forward-looking statements represent BioLineRx's views only as of the date of this release and should not be relied upon as representing its views as of any subsequent date. BioLineRx does not assume any obligation to update any forward-looking statements unless required by law.

Contact:Tim McCarthyLifeSci Advisors, LLC+1-212-915-2564tim@lifesciadvisors.com

or

Tsipi HaitovskyPublic Relations+972-52-598-9892tsipihai5@gmail.com

View original content:http://www.prnewswire.com/news-releases/biolinerx-to-present-two-posters-at-the-society-for-immunotherapy-of-cancer-sitc-2019-300948943.html

SOURCE BioLineRx Ltd.

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BioLineRx to Present Two Posters at the Society for Immunotherapy of Cancer (SITC) 2019 - P&T Community

Bone Marrow Stem Cells Comments Off on BioLineRx to Present Two Posters at the Society for Immunotherapy of Cancer (SITC) 2019 – P&T Community | October 31st, 2019

The National Institutes of Health (NIH) and the Bill & Melinda Gates Foundation will each invest $100 million over the next four years to speed the development of affordable gene therapies for sickle cell disease (SCD) and the human immunodeficiency virus (HIV) on a global scale.

This unprecedented collaboration focuses from the get-go on access, scalability and affordability of advanced gene-based strategies for sickle cell disease and HIV to make sure everybody, everywhere has the opportunity to be cured, not just those in high-income countries, said NIH Director Francis S. Collins, MD, PhD.

Seventy-five percent of babies born with SCD live in sub-Saharan Africa. It is hoped that experimental gene therapies would advance to clinical trials in the United States and relevant African countries within the next seven to 10 years, and that safe, effective, and inexpensive gene therapies be made available globally, including in low-resource settings where the cost and complexity of these therapies make them inaccessible to many.

In recent years, gene-based treatments have been groundbreaking for rare genetic disorders and infectious diseases, Trevor Mundel, MD, PhD, president of the global health program at the Bill & Melinda Gates Foundation said in a news release.

While these treatments are exciting, people in low- and middle-income countries do not have access to these breakthroughs. By working with the NIH and scientists across Africa, we aim to ensure these approaches will improve the lives of those most in need and bring the incredible promise of gene-based treatments to the world of public health, he added.

Hemoglobin is the protein in red blood cells that binds oxygen, allowing oxygen to be transported around the body. Mutations in the HBBgene, which encodes a component of hemoglobin, result in the formation of sickle hemoglobin that causes sickle cell anemia.

Currently, gene therapies for SCD involves altering the patients own hematopoietic stem cells (bone marrow cells that divide and specialize to produce blood cells including red blood cells). Genes are introduced into the cells using a modified, harmless virus (known as a viral vector). The cells are then transplanted back into the patient where they will produce healthy red blood cells. Gene therapy has an advantage over a bone marrow transplant, as it circumvents the complications associated with a bone marrow donation.

The first goal of the collaboration between the NIH and the Gates Foundation is to develop an easy-to-administer gene-based intervention to correct the mutations in the HBBgene or deliver a functional gene that will promote the production of normal levels of hemoglobin without the need to extract cells from patients and modify them in the lab before introducing the cells back. However, this strategy, known as in vivotreatment, requires the advancement of more efficient delivery systems that can deliver the gene therapy specifically to hematopoietic stem cells.

A second goal of the collaboration will be to work together with African partners and bring potential therapies to clinical trials.

Further research is required to understand the burden of SCD in sub-Saharan Africa and to screen newborns at high risk for the disease, a task that the National Heart, Lung and Blood Institute (NHLBI) has started to tackle by building the necessary infrastructure for clinical research.

The NIH and the Gates Foundation will help boost this infrastructure to allow point-of-care screening (for example, when infants receive vaccinations), and to initiate a standard of care. This will occur outside of the official collaboration.

Our excitement around this partnership rests not only in its ability to leverage the expertise in two organizations to reduce childhood mortality rates in low-resource countries, but to bring curative therapies for sickle cell disease and HIV to communities that have been severely burdened by these diseases for generations, said Gary H. Gibbons, MD, director of the NHLBI.

A persons health should not be limited by their geographic location, whether rural America or sub-Saharan Africa; harnessing the power of science is needed to transcend borders to improve health for all, he added.

Matshidiso Rebecca Moeti, the regional director for Africa at the World Health Organization said, We are losing too much of Africas future to sickle cell disease and HIV.

Beating these diseases will take new thinking and long-term commitment. Im very pleased to see the innovative collaboration announced today, which has a chance to help tackle two of Africas greatest public health challenges, Moeti added.

Patricia holds her Ph.D. in Cell Biology from University Nova de Lisboa, and has served as an author on several research projects and fellowships, as well as major grant applications for European Agencies. She also served as a PhD student research assistant in the Laboratory of Doctor David A. Fidock, Department of Microbiology & Immunology, Columbia University, New York.

Total Posts: 94

Margarida graduated with a BS in Health Sciences from the University of Lisbon and a MSc in Biotechnology from Instituto Superior Tcnico (IST-UL). She worked as a molecular biologist research associate at a Cambridge UK-based biotech company that discovers and develops therapeutic, fully human monoclonal antibodies.

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SCD, HIV Gene Therapy Efforts Get $200M from NIH, Gates Foundation - Sickle Cell Anemia News

Bone Marrow Stem Cells Comments Off on SCD, HIV Gene Therapy Efforts Get $200M from NIH, Gates Foundation – Sickle Cell Anemia News | October 31st, 2019

Cell Harvesting MarketReport has newly added to its massive repository. Different industry-specific methods have been used for analyzing the market carefully. The informative data has been inspected through primary and secondary research techniques. The global Cell Harvesting market has been analyzed by focusing on different verticals of the businesses such as market trends, regional outlook, competitive landscape, key players, business approaches, technologies, and standard operating procedures.An exclusiveCell Harvesting Marketresearch report contains a brief on those trends which may enable the companies operating into know to strategize and the current sector to their small enterprise expansion. The investigation report analyses the market size, industry share, growth, key sections, CAGR, and drivers.

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Cell harvesting usually for use in cancer or other treatment. Usually the cells are removed from the patients own bone marrow. Stem cells can be harvested from the blood or bone marrow. Umbilical cords have been saved as a future source of stem cells for the baby.

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Cell Harvesting Market Global Strategies and Insight driven transformation 2019-2024 - The Chicago Sentinel

Bone Marrow Stem Cells Comments Off on Cell Harvesting Market Global Strategies and Insight driven transformation 2019-2024 – The Chicago Sentinel | October 31st, 2019

Zion Market Research published a new 110+ pages industry researchCell Isolation Market by Product (Instruments and Consumables), by Cell Type (Animal and Human), by Cell Source (Adipose Tissue, Embryonic/Cord Blood Stem Cells, and Bone Marrow), by Technique (Surface Marker-Based Cell Isolation, Centrifugation-Based Cell Isolation, and Filtration-Based Cell Isolation), by Application (Cancer Research, Biomolecule Isolation, Tissue Regeneration & Regenerative Medicine, Stem Cell Research, In Vitro Diagnostics, and Others), and By End-User (Hospitals & Diagnostic Laboratories, Research Laboratories & Institutes, Biotechnology & Biopharmaceutical Companies, and Others): Global Industry Perspective, Comprehensive Analysis, and Forecast, 20182025.

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Cell Isolation Market ||Becton, Dickinson, and Company, Thermo Fisher Scientific, Inc., Merck KGaA - Industry News Info

Bone Marrow Stem Cells Comments Off on Cell Isolation Market ||Becton, Dickinson, and Company, Thermo Fisher Scientific, Inc., Merck KGaA – Industry News Info | October 31st, 2019

By Express News Service

KOCHI:Though the impending examinations, the study leave and the hartal called by the merchants association played spoilsport, nearly 525 youngsters came forward to register themselves as stem cell donors on Tuesday at a camp set up by Smilemakers of Cusat and DATRI of St Teresas College. The organisers were expecting around 1,000 registrations.

Next, we will be holding a camp at Lulu Mall on November 14, said Ramiz Rehman of Smilemakers. Not only students but also teachers and people from outside the campus came in to register. However, one big impediment is the lack of awareness about the stem cell donation process, he said.According to him, there is a common notion that bone marrow aspiration needs to be done to extract stem cells. But this is not the case. The stem cells are extracted from the blood. There is no drilling of bones happens, said Ramiz. According to him, stem cell transfusion is the only treatment that can save the lives of those suffering from leukaemia and thalassemia.

The programme has been organised for the benefit of not only the three siblings diagnosed with thalassemia major hailing from Mattancherry, but for the thousands of patients who have registered with DATRI. Since the possibility of obtaining a match is one in 20 lakh, there is a need to have a lot of people registering as fast as possible, he said. Speaking after inaugurating the registry drive, Poornima Jayaram, actor, said: Everyone will come as one to register themselves if they put themselves in the shoes of the recipient.

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Stem cell registry gets good response - The New Indian Express

Bone Marrow Stem Cells Comments Off on Stem cell registry gets good response – The New Indian Express | October 30th, 2019