VANCOUVER, Washington, Nov. 11, 2019 (GLOBE NEWSWIRE) -- CytoDyn Inc. (OTC.QB: CYDY), (CytoDyn or the Company"), a late-stage biotechnology company developing leronlimab (PRO 140), a CCR5 antagonist with the potential for multiple therapeutic indications, announced today encouraging initial results from the first patient in a Phase 1b/2 clinical trial with metastatic triple-negative breast cancer (mTNBC). Circulating tumor cells (CTC) in the patients blood decreased significantly after leronlimab therapy at both two-week and five-week time points. Furthermore, a reduction in CCR5 expression on presumed metastatic tumor cells was evident.

We are excited to be involved with CytoDyn in evaluating the efficacy of leronlimab in mTNBC," stated IncellDx CEO, Bruce Patterson, M.D. These results at both two-week and five-week time intervals post-leronlimab therapy indicate initial efficacy against this most aggressive tumor type. Moreover, the reduction of CCR5 expression on EMT cells may prove to be significant, as high CCR5 expression is believed to be crucial for metastases.

The treatment of mTNBC with leronlimab in this Phase 1b/2 trial is in addition to metastatic breast cancer (MBC) patients treated with leronlimab under an emergency use IND. Results from both of the ongoing trials in MBC will dictate the Companys regulatory pathway, including the potential to seek Breakthrough Therapy designation and accelerated approval with the U.S. FDA for the use of leronlimab in MBC. Leronlimab has been granted Fast Track designation for mTNBC by the FDA based on a greater than 98% reduction of metastatic tumor volume in a murine xenograft model.

Today marks yet another significant milestone in our Companys history, advancing CytoDyns clinical development in oncology. Although these are early results in our first patient, we are encouraged by the reduction in both CTC and tumor size. Our safety record with leronlimab, and preclinical results in multiple oncology trials in various cancer indications, solidifies our vision to explore oncology indications. We are optimistic about the opportunity to provide a potential new therapeutic option for the women that are diagnosed with invasive breast cancer each year in the United States. We wish to thank the women who have agreed to participate in our trials and will endeavor to provide each of them with clinical benefit," stated CytoDyn President and CEO, Nader Pourhassan, Ph.D.

About Leronlimab (PRO 140)The U.S. Food and Drug Administration (FDA) has granted a "Fast Track" designation to CytoDyn for two potential indications of leronlimab for deadly diseases. The first as a combination therapy with highly active antiretroviral therapy (HAART) for HIV-infected patients, and the second is for metastatic triple-negative breast cancer. Leronlimab is an investigational humanized IgG4 mAb that blocks CCR5, a cellular receptor that is important in HIV infection, tumor metastases, and other diseases, including non-alcoholic steatohepatitis (NASH). Leronlimab has successfully completed nine clinical trials in over 800 people, including meeting its primary endpoints in a pivotal Phase 3 trial (leronlimab in combination with standard anti-retroviral therapies in HIV-infected treatment-experienced patients).

In the setting of HIV/AIDS, leronlimab is a viral-entry inhibitor; it masks CCR5, thus protecting healthy T cells from viral infection by blocking the predominant HIV (R5) subtype from entering those cells. Leronlimab has been the subject of nine clinical trials, each of which demonstrated that leronlimab can significantly reduce or control HIV viral load in humans. The leronlimab antibody appears to be a powerful antiviral agent leading to potentially fewer side effects and less frequent dosing requirements compared with daily drug therapies currently in use.

In the setting of cancer, research has shown that CCR5 plays a vital role in tumor invasion and metastasis. Increased CCR5 expression is an indicator of disease status in several cancers. Published studies have shown that blocking CCR5 can reduce tumor metastases in laboratory and animal models of aggressive breast and prostate cancer. Leronlimab reduced human breast cancer metastasis by more than 98% in a murine xenograft model. CytoDyn is, therefore, conducting a Phase 2 human clinical trial in metastatic triple-negative breast cancer and was granted Fast Track designation in May 2019. CytoDyn is conducting additional research with leronlimab in the setting of oncology and NASH with plans to conduct further clinical studies when appropriate.

The CCR5 receptor appears to play a central role in modulating immune cell trafficking to sites of inflammation. It may be important in the development of acute graft-versus-host disease (GvHD) and other inflammatory conditions. Clinical studies by others further support the concept that blocking CCR5 using a chemical inhibitor can reduce the clinical impact of acute GvHD without significantly affecting the engraftment of transplanted bone marrow stem cells. CytoDyn is currently conducting a Phase 2 clinical study with leronlimab to support further the concept that the CCR5 receptor on engrafted cells is critical for the development of acute GvHD. Blocking the CCR5 receptor from recognizing specific immune signaling molecules is a viable approach to mitigating acute GvHD. The FDA has granted "orphan drug" designation to leronlimab for the prevention of GvHD.

About CytoDynCytoDyn is a biotechnology company developing innovative treatments for multiple therapeutic indications based on leronlimab, a novel humanized monoclonal antibody targeting the CCR5 receptor. CCR5 appears to play a crucial role in the ability of HIV to enter and infect healthy T-cells. The CCR5 receptor also appears to be implicated in tumor metastasis and immune-mediated illnesses, such as GvHD and NASH. CytoDyn has completed a Phase 3 pivotal trial with leronlimab in combination with standard anti-retroviral therapies in HIV-infected treatment-experienced patients. CytoDyn plans to seek FDA approval for leronlimab in combination therapy and plans to complete the filing of a Biologics License Application (BLA) in 2019 for that indication. CytoDyn is also conducting a Phase 3 investigative trial with leronlimab as a once-weekly monotherapy for HIV-infected patients. CytoDyn plans to initiate a registration-directed study of leronlimab monotherapy indication, which, if successful, could support a label extension. Clinical results to date from multiple trials have shown that leronlimab can significantly reduce viral burden in people infected with HIV with no reported drug-related serious adverse events (SAEs).Moreover, results from a Phase 2b clinical trial demonstrated that leronlimab monotherapy can prevent viral escape in HIV-infected patients. Some patients on leronlimab monotherapy have viral suppression for more than four years. CytoDyn is also conducting a Phase 2 trial to evaluate leronlimab for the prevention of GvHD and has received clearance to initiate a clinical trial with leronlimab in metastatic triple-negative breast cancer. More information is at http://www.cytodyn.com.

Forward-Looking StatementsThis press release contains certain forward-looking statements that involve risks, uncertainties, and assumptions that are difficult to predict. Words and expressions reflecting optimism, satisfaction or disappointment with current prospects, as well as words such as "believes," "hopes," "intends," "estimates," "expects," "projects," "plans," "anticipates" and variations thereof, or the use of future tense, identify forward-looking statements but, their absence does not mean that a statement is not forward-looking. The Company's forward-looking statements are not guarantees of performance, and actual results could vary materially from those contained in or expressed by such statements due to risks and uncertainties including: (i)the sufficiency of the Companys cash position, (ii)the Companys ability to raise additional capital to fund its operations, (iii) the Companys ability to meet its debt obligations, if any, (iv)the Companys ability to enter into partnership or licensing arrangements with third parties, (v)the Companys ability to identify patients to enroll in its clinical trials in a timely fashion, (vi)the Companys ability to achieve approval of a marketable product, (vii)the design, implementation and conduct of the Companys clinical trials, (viii)the results of the Companys clinical trials, including the possibility of unfavorable clinical trial results, (ix)the market for, and marketability of, any product that is approved, (x)the existence or development of vaccines, drugs, or other treatments that are viewed by medical professionals or patients as superior to the Companys products, (xi)regulatory initiatives, compliance with governmental regulations and the regulatory approval process, (xii)general economic and business conditions, (xiii)changes in foreign, political, and social conditions, and (xiv)various other matters, many of which are beyond the Companys control. The Company urges investors to consider specifically the various risk factors identified in its most recent Form10-K, and any risk factors or cautionary statements included in any subsequent Form10-Q or Form8-K, filed with the Securities and Exchange Commission. Except as required by law, the Company does not undertake any responsibility to update any forward-looking statements to take into account events or circumstances that occur after the date of this press release.

CONTACTSInvestors: Nader Pourhassan, Ph.D.President & CEOnpourhassan@cytodyn.com

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First Patient in CytoDyn's Triple-Negative Metastatic Breast Cancer Trial Shows Significant Reduction in Circulating Tumor Cells (CTC) and Reduced...

Bone Marrow Stem Cells Comments Off on First Patient in CytoDyn’s Triple-Negative Metastatic Breast Cancer Trial Shows Significant Reduction in Circulating Tumor Cells (CTC) and Reduced… | November 11th, 2019

Another seminar is again advertised in The Union. I first thought it might help my wife with neuropathy until I did some extensive research.

First off, Medicare does not cover stem cell injections. Bone marrow stem cell injections range from $2,000 to $5,000 or more. Read Consumer Research report at: https://www.consumerreports.org/medical-treatments-procedures/trouble-with-stem-cell-therapy.

Stem cell treatments are widely accepted only for two broad medical indications: to help treat a handful of blood disorders including leukemia and some forms of anemia and in some cases to help burn victims. Ask questions. Any doctor who offers stem cell therapy should be able to explain where the cells will come from, what will be done to them before theyre injected into your body, and how, exactly, they will resolve your illness or injury. He or she should also be able to offer you proof of safety and efficacy, even for experimental treatments. Dont rely on patient testimonials.

Stem cells survive much longer than ordinary cells, increasing the chance that they might accumulate genetic mutations. It might take only a few mutations for one cell to lose control over its self-renewal and growth and become the source of cancer. Please do your own research.

Gary Pesselt

Grass Valley

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Gary Pesselt: Vitality Healthcare is it worth the cost? - The Union of Grass Valley

Bone Marrow Stem Cells Comments Off on Gary Pesselt: Vitality Healthcare is it worth the cost? – The Union of Grass Valley | November 10th, 2019

Whoever saves a single life is considered by the Talmud to have saved the whole world.

In August 2013, Jeffrey Altadonna, who was on a Birthright trip, was tested at a bone marrow testing drive at the Jerusalem Gate Hotel.

It was perfectly ordinary summer day when the 29-year-old accountant from Sherman Oaks, California received the fateful phone call.

A 77-year-old woman from Los Angeles was the perfect stranger that he was deemed to save.

Diane Gebel, a widow from Cyprus, California was diagnosed with Acute Myeloid Leukemia (AML). Her husband had passed away right before she was diagnosed with cancer.

For an entire year, the donor and recipient need to remain anonymous to each other, but last week, the time came for the two to finally meet.

The two were honored at the Los Angeles One Huge Night Gala event hosted by Gift of Life.

In a statement, Birthright Israel explained that the gala also celebrated the successful 15-year partnership of Birthright Israel and Gift of Life.

This partnership has so far resulted in 83,000 Birthright donors joining the registry, with 1,900 matches made between patients and Birthright donors, and 241 life-saving transplants to date.

Prior to the meeting, Altadonna recalled his Birthright trip explaining that it was really great to see that part of the world, to go to where its our given right to visit.

It had a profound effect on my friendships and cultural Jewish identity to see that Jewish people are one people, and we have each others backs, he said. It left me with the feeling that I had backing in anything that I wanted to do in my life. It really felt like a family.

He recalled that after being swabbed at the drive, he didnt really think too much of it because everyone did it.

I got the donation call 15 months ago, he explained, adding that he immediately decided to donate. I find it very bizarre, that everyone is telling me Its such a great thing that you are doing. For me, it wasnt an option to say yes or no, its just, Okay, lets do this, Im a match. Im surprised that more people dont donate.

After doing preliminary tests, he took the plunge and donated.

I had to do it early in the morning, it lasted 6-8 hours a marathon blood donation and it was finished,Altadonna continued. It didnt seem all that hard to me.

He made it clear that this opportunity to help only came about because of the Birthright Israel and Gift of Life collaboration.

It wasnt a mission of mine. I wouldnt have gone out of my way to get swabbed, so it only happened as a result of their collaboration, he said.

In an emotional meeting, the two finally met. Of the meeting, Gebel stressed that she is here because of my selfless and generous donor.

For me, it was easy, I just had an infusion, but for him it was hard, she said. It takes a very special person to do that, to actually give the gift of life.

Gebel said she had been waiting to meet him.

I didnt know he was such a good looking guy, Gebel joked. Im here because of him. I was not ready to die. I had too much to live for.

She stressed that she has changed because of my new life.

I take risks, I live my life fully, she explained. My motto has always been that I want to go through life with a Cosmopolitan in one hand and a travel book in the other. Because of my donor, I can do that, so I thank him from the bottom of my heart.

Altadonna called on others to also take the plunge by getting tested and donating.

I stand here for my recipients valor, for her victory. I ask you to do the same: sit and swab today, so someone can swim and live tomorrow, he said.

Birthrights International CEO Gidi Mark said he was proud of our participants who register as donors and the powerful impact of such a simple choice when they get the call that they are the perfect match for a perfect stranger.

It is a great honor that Birthright Israel is able to assist in this mission, he added.

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Perfect match: How Birthright alumni saved the lives of 100s of strangers - The Jerusalem Post

Bone Marrow Stem Cells Comments Off on Perfect match: How Birthright alumni saved the lives of 100s of strangers – The Jerusalem Post | November 10th, 2019

When Nicky Turkoz (aboveleft) was diagnosed with leukaemia, her only hope was an anonymous stem-cell donation. That donor was Annette Hamson (above right) and the two, once strangers, now share an unbreakable bond

I was wrestling the Christmas decorations down from the loft when I got the call. I can remember hearing the landline ring and telling my daughter, Meltem, who was steadying the ladder, to leave it. They can call back if its urgent, I said. My mobile rang and we left that too, but the landline went again. I better get it, Mum, said Meltem. Someone clearly wants to talk to you. She answered it as I was hauling the last of the decorations down. Its the surgery, she said, passing the phone to me.

That morning Id had a blood test. Id been feeling strange for months and had finally made an appointment to see the GP. I told her I was feeling lethargic and depressed, which was very unlike me, but Id put it down to ageing and a spot of empty-nest syndrome, as my youngest daughter Zeynep had just left home to work abroad. Odd things, such as a recurring gum infection, were making me wonder if something else was going on. My GP seemed unconcerned but sent me for blood tests anyway. A few hours later she was telling me to pack an overnight bag and get to hospital. Your results show youre very anaemic, she said. Theyll need to do some more tests. You might be there a while.

She never used the word cancer. Looking back, I think she knew then but was sugar- coating it for me. We left the decorations.

Three hours later, after more prodding and poking, a consultant told me I had acute myeloid leukaemia, a blood cancer. It seems unbelievable now but I can remember thinking, Well, thank goodness theyve found something wrong with me, I knew I wasnt feeling great. I had no idea, of course, what was coming down the track.

It was a Wednesday evening and I was told they would start my chemotherapy on the Friday, but Id need to stay in hospital in the meantime. So while I was being installed on a ward, my poor daughter was handed a leaflet about myeloid leukaemia and drove home to our undecorated tree.

The first round of chemo lasted 10 days, which meant I had to miss Meltems 23rd birthday on 21 December. Zeynep left Gran Canaria, where she had just started working as a holiday rep, and flew home to be with us. It has always been just the three of us, ever since they were tiny. Im a very can-do sort of person and have always just got on with life whatever it has thrown at us. I couldnt bear it when my girls suddenly had to look after me. It all just seemed so unfair. I suppose everyone feels like that though.

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'I've become best friends with the woman who saved my life' - Telegraph.co.uk

Bone Marrow Stem Cells Comments Off on ‘I’ve become best friends with the woman who saved my life’ – Telegraph.co.uk | November 9th, 2019

Scientists at Harvard-affiliated Massachusetts General Hospital (MGH) have identified a previously unknown biological pathway that promotes chronic inflammation and may help explain why sedentary people have an increased risk for heart disease and strokes.

In a study to be published in the November issue ofNature Medicine, MGH scientists and colleagues at several other institutions found that regular exercise blocks this pathway. This discovery could aid the development of new therapies to prevent cardiovascular disease.

Regular exercise protects the cardiovascular system by reducing risk factors such as cholesterol and blood pressure. But we believe there are certain risk factors for cardiovascular disease that are not fully understood, said Matthias Nahrendorf of the Center for Systems Biology at MGH. In particular, Nahrendorf and his team wanted to better understand the role of chronic inflammation, which contributes to the formation of artery-clogging blockages called plaques.

Nahrendorf and colleagues examined how physical activity affects the activity of bone marrow, specifically hematopoietic stem and progenitor cells (HSPCs). HSPCs can turn into any type of blood cell, including white blood cells called leukocytes, which promote inflammation. The body needs leukocytes to defend against infection and remove foreign bodies.

When these [white blood] cells become overzealous, they start inflammation in places where they shouldnt, including the walls of arteries.

Matthias Nahrendorf

But when these cells become overzealous, they start inflammation in places where they shouldnt, including the walls of arteries, said Nahrendorf.

Nahrendorf and his colleagues studied a group of laboratory mice that were housed in cages with treadmills. Some of the mice ran as much as six miles a night on the spinning wheels. Mice in a second group were housed in cages without treadmills. After six weeks, the running mice had significantly reduced HSPC activity and lower levels of inflammatory leukocytes than the mice that simply sat around their cages all day.

Nahrendorf explains that exercising caused the mice to produce less leptin, a hormone made by fat tissue that helps control appetite, but also signaled HSPCs to become more active and increase production of leukocytes. In two large studies, the team detected high levels of leptin and leukocytes in sedentary humans who have cardiovascular disease linked to chronic inflammation.

This study identifies a new molecular connection between exercise and inflammation that takes place in the bone marrow and highlights a previously unappreciated role of leptin in exercise-mediated cardiovascular protection, said Michelle Olive, program officer at the National Heart, Lung, and Blood Institute Division of Cardiovascular Sciences. This work adds a new piece to the puzzle of how sedentary lifestyles affect cardiovascular health and underscores the importance of following physical-activity guidelines.

Reassuringly, the study found that lowering leukocyte levels by exercising didnt make the running mice vulnerable to infection. This study underscores the importance of regular physical activity, but further focus on how exercise dampens inflammation could lead to novel strategies for preventing heart attacks and strokes. We hope this research will give rise to new therapeutics that approach cardiovascular disease from a completely new angle, said Nahrendorf.

The primary authors of theNature Medicinepaper are Nahrendorf, who is also a professor of radiology at Harvard Medical School; Vanessa Frodermann, a former postdoctoral fellow at MGH who is now a senior scientist at Novo Nordisk; David Rohde, a research fellow in the Department of Radiology at MGH; and Filip K. Swirski, an investigator in the Department of Radiology at MGH.

The work was funded bygrantsHL142494 andHL139598from the National Heart, Lung, and Blood Institute (NHLBI), part of the National Institutes of Health.

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Exercise found to block chronic inflammation in mice - Harvard Gazette

Bone Marrow Stem Cells Comments Off on Exercise found to block chronic inflammation in mice – Harvard Gazette | November 9th, 2019

Doris A. Taylors so-called replacement ghost heart suggests something otherworldly, but the eerie-looking form is far from an apparition. Its an innovative approach to organ transplantation that has inspired many in the medical community and at least one artist.

The Texas researchers process piggybacks on natures sophisticated design. Together she and a team of researchers strip cells off human and animal cadaver hearts with a soapy solution, leaving ghostly white protein shells that retain the form of the organ. They inject them with a patients blood or bone-marrow stem cells, and the ghost hearts act as scaffolding on which the newly introduced cells can slowly transform into a beating muscle.

What we said was, Wouldnt it be really cool if we could wash the sick cells out and put the healthy cells back in? said Taylor, director of Regenerative Medicine Research and director of the Center for Cell and Organ Biotechnology at the Texas Heart Institute, during a recent talk at the Radcliffe Institute for Advanced Study.

The hope is that one day these regenerated hearts will resolve the most challenging issues transplant patients currently face: the lack of a permanent artificial replacement, concerns about rejection, and the shortage of viable donor hearts.

Taylors efforts are driving what could become a revolution in organ transplants, and they have sparked the creativity of transdisciplinary artist Dario Robleto, whose latest work, on view at the Johnson-Kulukundis Family Gallery in Radcliffes Byerly Hall, recreates in images and sounds the original pulse wave of the heart first captured in visual form by scientists in the 1900s. Robleto and Taylor, longtime friends and Texas residents, explored those connections during Mondays Radcliffe discussion, which was moderated by Jennifer Roberts, Elizabeth Cary Agassiz Professor of the Humanities.

Robletos exhibit, Unknown and Solitary Seas, touches on the overlap between the medical mysteries and workings of the vascular pump, and the metaphor for the heart as the emotional center of the soul. It includes a video installation that features recreated sounds of a beating heart from the 19th century, reconstructed images of how the earliest pulse waves first appeared on the page, and a series of heart waveform sculptures in brass-plated stainless steel.

Roberts said that with his work, Robleto acknowledges the pulse waves promise, their profundity, their scientific value, but he also reclaims some of their ambiguity and asks us to wonder whether we can or should accept that these waveforms have escaped the realms of art, culture, and emotional communication.

Taylor similarly views her work as a blend of the scientific and the human. It transcends complicated, complex science, she says, in that her ghost hearts require a kind of passion, commitment, care, attention, and nurturing similar to whats required by a small child. Its really about building hearts at the emotional, mental, spiritual, and physical level that I think is going to get them to work, she said.

For Robleto, big ideas, like the creation of a new human heart, require multiple perspectives.

The artist called Taylors work one of the most fascinating and definitely one of the most emotional things Ive ever seen. As an object, he added, the ghost heart is stunningly beautiful but it also raises questions about the self, identity, emotion, the notions of form and where memory is truly held, questions he thinks artists can help address. He cited two of the nations earliest heart transplants, after which the patients wives asked their husbands, who had received donor hearts, if they still loved them.

Taylors work, Robleto said, is right at the edge of identity and materiality and so when the day comes when someone says the first ghost heart transplant I think we will have a similar moment where perhaps we will be forced to re-evaluate what we ask from our heart metaphor.

Dario Robletos Unknown and Solitary Seas is on view in Byerly Halls Johnson-Kulukundis Family Gallery through Jan. 18, 2020.

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An artist and a transplant researcher discuss the heart - Harvard Gazette

Bone Marrow Stem Cells Comments Off on An artist and a transplant researcher discuss the heart – Harvard Gazette | November 9th, 2019

NEW YORK--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket), a leading U.S.-based multi-platform clinical-stage gene therapy company, today announces presentations at the upcoming 61st American Society of Hematology (ASH) Annual Meeting being held December 7-10, 2019 in Orlando, Florida. The two poster presentations will highlight clinical data from the Phase 1 study of RP-L102 utilizing Process B for the treatment of Fanconi Anemia (FA), as well as long-term follow-up data from the Phase 1/2 EUROFANCOLEN trial.

Details for Rockets poster presentations are as follows:Title: Changing the Natural History of Fanconi Anemia Complementation Group-A with Gene Therapy: Early Results of U.S. Phase I Study of Lentiviral-Mediated Ex-Vivo FANCA Gene Insertion in Human Stem and Progenitor CellsSession Title: Gene Therapy and Transfer: Poster IIPresenter: Sandeep Soni, M.D.Session Date: Sunday, December 8, 2019Session Time: 6:00 p.m. 8:00 p.m. ESTLocation: Orange County Convention Center, Hall B

Title: Hematopoietic Engraftment of Fanconi Anemia Patients through 3 Years after Gene TherapySession Title: Gene Therapy and Transfer: Poster IIIPresenter: Paula Ro, Ph.D.Session Date: Monday, December 9, 2019Session Time: 6:00 p.m. 8:00 p.m. ESTLocation: Orange County Convention Center, Hall B

The Sunday poster session will be followed by a breakout session to give investors and analysts the opportunity to ask questions and discuss the data. The breakout session, hosted by Rocket management, will be held on Sunday, December 8th at 8:30 p.m. EST, directly after Dr. Sonis presentation. At the event, Dr. Soni, Clinical Associate Professor of Stem Cell Transplantation and Regenerative Medicine at the Stanford University School of Medicine and principal investigator of the U.S. Phase 1 trial of RP-L102 and Paula Ro, Ph.D., Senior Scientist, Divisin de Terapias Innovadoras en el Sistema Hematopoytico, CIEMAT/CIBERER Unidad Mixta de Terapias Avanzadas CIEMAT/IIS Fundacin Jimnez Daz will be participating in a Q&A panel. For further information, please contact investors@rocketpharma.com.

About Fanconi Anemia

Fanconi Anemia (FA) is a rare pediatric disease characterized by bone marrow failure, malformations and cancer predisposition. The primary cause of death among patients with FA is bone marrow failure, which typically occurs during the first decade of life. Allogeneic hematopoietic stem cell transplantation (HSCT), when available, corrects the hematologic component of FA, but requires myeloablative conditioning. Graft-versus-host disease, a known complication of allogeneic HSCT, is associated with an increased risk of solid tumors, mainly squamous cell carcinomas of the head and neck region. Approximately 60-70% of patients with FA have a FANC-A gene mutation, which encodes for a protein essential for DNA repair. Mutation in the FANC-A gene leads to chromosomal breakage and increased sensitivity to oxidative and environmental stress. Chromosome fragility induced by DNA-alkylating agents such as mitomycin-C (MMC) or diepoxybutane (DEB) is the gold standard test for FA diagnosis. Somatic mosaicism occurs when there is a spontaneous correction of the mutated gene that can lead to stabilization or correction of a FA patients blood counts in the absence of any administered therapy. Somatic mosaicism, often referred to as natures gene therapy provides a strong rationale for the development of FA gene therapy because of the selective growth advantage of gene-corrected hematopoietic stem cells over FA cells1.

1Soulier, J.,et al. (2005) Detection of somatic mosaicism and classification of Fanconi anemia patients by analysis of the FA/BRCA pathway. Blood 105: 1329-1336

About Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket) is an emerging, clinical-stage biotechnology company focused on developing first-in-class gene therapy treatment options for rare, devastating diseases. Rockets multi-platform development approach applies the well-established lentiviral vector (LVV) and adeno-associated viral vector (AAV) gene therapy platforms. Rocket's clinical programs using LVV-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, and Pyruvate Kinase Deficiency (PKD) a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. Rockets first clinical program using AAV-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition. Rockets pre-clinical pipeline program is for Infantile Malignant Osteopetrosis (IMO), a bone marrow-derived disorder. For more information about Rocket, please visit http://www.rocketpharma.com.

Rocket Cautionary Statement Regarding Forward-Looking Statements

Various statements in this release concerning Rocket's future expectations, plans and prospects, including without limitation, Rocket's expectations regarding the safety, effectiveness and timing of product candidates that Rocket may develop, to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Infantile Malignant Osteopetrosis (IMO) and Danon disease, and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket's ability to successfully demonstrate the efficacy and safety of such products and pre-clinical studies and clinical trials, its gene therapy programs, the pre-clinical and clinical results for its product candidates, which may not support further development and marketing approval, the potential advantages of Rocket's product candidates, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, Rocket's and its licensors ability to obtain, maintain and protect its and their respective intellectual property, the timing, cost or other aspects of a potential commercial launch of Rocket's product candidates, Rocket's ability to manage operating expenses, Rocket's ability to obtain additional funding to support its business activities and establish and maintain strategic business alliances and new business initiatives, Rocket's dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rocket's Annual Report on Form 10-K for the year ended December 31, 2018. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

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Rocket Pharmaceuticals to Present Preliminary Phase 1 Data of RP-L102 Process B for Fanconi Anemia at the 61st American Society of Hematology Annual...

Bone Marrow Stem Cells Comments Off on Rocket Pharmaceuticals to Present Preliminary Phase 1 Data of RP-L102 Process B for Fanconi Anemia at the 61st American Society of Hematology Annual… | November 8th, 2019

Global Cell Isolation/Cell Separation Market was valued US$ XX Bn in 2018 and is expected to reach US$ 17.92 Bn by 2026, at a CAGR of around XX % during a forecast period.

The report covers all the trends and technologies playing a major role in the growth of the Cell Isolation/Cell Separation market during the forecast period. It highlights the drivers, restraints, and opportunities expected to influence the market growth during 2019-2026.

Some of the market drivers for the cell isolation/cell separation market are increasing incidences & prevalence of chronic diseases with the aging population, technological advancement in cell isolation, growing demand for bio-pharmaceuticals, personalized medicine, and increasing stem cell research. Cell isolation or separation is a tool used to sort cells into a specific population from a heterogeneous group of cells without contamination. The use of cell isolation techniques helps to open the door of cell-based therapies and thereby improve the quality of treatment and clinical outcome.

REQUEST FOR FREE SAMPLE REPORT: https://www.maximizemarketresearch.com/request-sample/34136

However, the ethical issues regarding the isolation of embryonic stem cells and the high cost of cell separation instruments are expected to restrict the growth of this market during the forecast period.

Based on cell type, the human cell segment is expected to register a major revenue share in the cell isolation/cell separation market globally. Owing to increasing investments by public and private organizations for research on human cells, growing application areas of human stem cells, and the high frequency and growing incidence of diseases such as cancer.

Based on the product, the consumables segment is expected to witness the fastest growth during the forecast period. Because of the increasing investments by companies to develop advanced products and the rising government initiatives for improving cell-based research are driving the growth of this segment.

North America region is expected to grow at a XX % rate of CAGR during the forecast period owing to increasing government support for cancer and stem cell research, the expanding biotechnology and biopharmaceutical industries and the increasing prevalence of chronic and infectious diseases in which cell isolation is required for diagnosis and treatment. Which results in, increase in demand for cell isolation products.

The objective of the report is to present a comprehensive assessment of the market and contains thoughtful insights, facts, historical data, industry-validated market data and projections with a suitable set of assumptions and methodology. The report also helps in understanding Global Cell Isolation/Cell Separation Market dynamics, structure by identifying and analyzing the market segments and project the global market size. Further, the report also focuses on the competitive analysis of key players by product, price, financial position, product portfolio, growth strategies, and regional presence. The report also provides PEST analysis, PORTERs analysis, and SWOT analysis to address the question of shareholders to prioritizing the efforts and investment in the near future to the emerging segment in Global Cell Isolation/Cell Separation Market.

DO INQUIRY BEFORE PURCHASING REPORT HERE: https://www.maximizemarketresearch.com/inquiry-before-buying/34136

Scope of the Global Cell Isolation/Cell Separation Market

Global Cell Isolation/Cell Separation Market, By Product

Consumableso Reagents, Kits, Media, and Serao Beadso Disposables Instrumentso Centrifugeso Flow Cytometerso Magnetic-activated Cell Separator Systemso Filtration SystemsGlobal Cell Isolation/Cell Separation Market, By Cell Type

Human Cellso Differentiated Cellso Stem Cells Animal CellsGlobal Cell Isolation/Cell Separation Market, By Cell Source

Adipose Tissue Bone Marrow Cord Blood/Embryonic Stem CellsGlobal Cell Isolation/Cell Separation Market, By Technique

Centrifugation-based Cell Isolation Surface Marker-based Cell Isolation Filtration-based Cell IsolationGlobal Cell Isolation/Cell Separation Market, By Application

Biomolecule Isolation Cancer Research Stem Cell Research Tissue Regeneration & Regenerative Medicine In Vitro DiagnosticsGlobal Cell Isolation/Cell Separation Market, By End user

Research Laboratories and Institutes Hospitals and Diagnostic Laboratories Biotechnology and Biopharmaceutical Companies Other End UsersGlobal Cell Isolation/Cell Separation Market, By Region

North America Europe Asia Pacific Middle East & Africa South AmericaKey players operating in the Global Cell Isolation/Cell Separation Market

Thermo Fisher Scientific Beckman Coulter Becton, Dickinson and Company GE Healthcare Merck KgaA Miltenyi Biotech pluriSelect STEMCELL Technologies Inc. Terumo BCT Bio-Rad Laboratories Inc.

MAJOR TOC OF THE REPORT

Chapter One: Cell Isolation/Cell Separation Market Overview

Chapter Two: Manufacturers Profiles

Chapter Three: Global Cell Isolation/Cell Separation Market Competition, by Players

Chapter Four: Global Cell Isolation/Cell Separation Market Size by Regions

Chapter Five: North America Cell Isolation/Cell Separation Revenue by Countries

Chapter Six: Europe Cell Isolation/Cell Separation Revenue by Countries

Chapter Seven: Asia-Pacific Cell Isolation/Cell Separation Revenue by Countries

Chapter Eight: South America Cell Isolation/Cell Separation Revenue by Countries

Chapter Nine: Middle East and Africa Revenue Cell Isolation/Cell Separation by Countries

Chapter Ten: Global Cell Isolation/Cell Separation Market Segment by Type

Chapter Eleven: Global Cell Isolation/Cell Separation Market Segment by Application

Chapter Twelve: Global Cell Isolation/Cell Separation Market Size Forecast (2019-2026)

Browse Full Report with Facts and Figures of Cell Isolation/Cell Separation Market Report at: https://www.maximizemarketresearch.com/market-report/global-cell-isolation-cell-separation-market/34136/

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Global Cell Isolation/Cell Separation Market Industry Analysis and Forecast (2019-2026) - BoundWatch

Bone Marrow Stem Cells Comments Off on Global Cell Isolation/Cell Separation Market Industry Analysis and Forecast (2019-2026) – BoundWatch | November 8th, 2019

Mr. McManushas over 20 years of leadership experience in the biopharmaceutical industry that includes leading the transformation of several biotech companies through strategic partnerships and execution of value-based strategies.Prior to joining PTx, John was the CEO of Aeolus Pharmaceuticals, where he managed a $100+ million BARDA contract for the advanced development of a radiation medical countermeasure.Before joining Aeolus, John served in strategic and financial roles at Spectrum Pharmaceuticals where he focused on oncology and NeoTherapeutics where he focused on Alzheimer's, Parkinson's, ALS and spinal cord injury.John holds a B.S. in International Finance and Business Economics from the University of Southern California.

"We are very pleased to welcome John to our team to lead our business development activities," said Bob Mulroy, PTx's Chief Executive Officer. "His breadth of experience as a biotech executive and business leader will be tremendous assets for our team as we continue to build and grow the opportunity for Leukine to help patients in need. We also will benefit from John's extensive experience working with the U.S. government to advance our ability to serve as a partner for a wide range of important government programs."

"I am excited to join the PTx team in strengthening and expanding the Leukine franchise and identifying additional products that would benefit from the Company's development and clinical expertise," said Mr. McManus. "Leukine is a critical drug in the treatment of adults and children facing life-threatening diseases and a key medical countermeasure for improving survival after lethal levels of radiation exposure.I am especially excited about working to support the development of Leukine in new indications like melanoma and diseases of the central nervous system like Alzheimer's and Parkinson's and expanding its use as a medical countermeasure through development partnerships with third parties including the U.S. government.I see tremendous potential for Leukine to help patients across a number of difficult-to-treat diseases."

PTx acquired the global rights to develop, manufacture, and commercialize Leukine in 2018.

Leukine is a multi-lineage immune-stimulant that has been demonstrated to promote growth and activation of monocytes, macrophages, neutrophils and dendritic cells.It is the only FDA-approved recombinant human granulocyte-macrophage colony stimulating factor (GM-CSF). It is currently indicated for the treatment of AML in older adults to reduce the incidence of severe and life-threatening infections resulting in death; use in the treatment of allogeneic bone marrow transplants to reduce the incidence of bacteremia and other culture positive infections and shorten the median duration of hospitalization; to prolong the survival of patients who are experiencing bone marrow transplant failure or delay; and to increase survival in adult and pediatric patients from birth to 17 years of age acutely exposed to myelosuppressive doses of radiation (Hematopoietic Syndrome of Acute Radiation Syndrome [H-ARS]).

About Leukine(sargramostim)

Leukine is a yeast-derived recombinant humanized granulocyte-macrophage colony stimulating factor (rhuGM-CSF) and the only FDA approved GM-CSF.GM-CSF is an important leukocyte growth factor known to play a key role in hematopoiesis, effecting the growth and maturation of multiple cell lineages as well as the functional activities of these cells in antigen presentation and cell mediated immunity3.

Important Safety Information for LEUKINE (sargramostim)

Contraindications

Warnings and Precautions

Adverse Reactions

Adverse events occurring in >10% of patients receiving LEUKINE in controlled clinical trials and reported in a higher frequency than placebo are:

Please see full Prescribing Information for LEUKINE at http://www.leukine.com

Indications and Usage

LEUKINE (sargramostim) is a leukocyte growth factor indicated for the following uses:

About Partner Therapeutics, Inc.:

PTx is an integrated commercial-stage biotech company focused on the development and commercialization of therapeutics that improve health outcomes in the treatment of cancer. PTx's development focus spans the entire range of cancer therapy from primary treatments to supportive care. The company believes in delivering great products with the purpose of creating the best possible outcomes for patients and their families.

SOURCE Partner Therapeutics, Inc.

https://www.partnertx.com/

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Partner Therapeutics (PTx) Announces the Appointment of John McManus as Chief Business Officer - PRNewswire

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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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ExCellThera announces publication in The Lancet Haematology highlighting excellent clinical results of ECT-001 in patients with haematological...

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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

Bone Marrow Stem Cells Comments Off on Using her diagnosis to help others, Peoria woman to host ‘Be the Match’ event – week.com | November 5th, 2019

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

Bone Marrow Stem Cells Comments Off on Three UCLA scientists receive grants totaling more than $18 million – UCLA Newsroom | November 5th, 2019

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