Archive for the ‘Bone Marrow Stem Cells’ Category

Updated October 04, 2019 14:43:48

The hardest thing Daniel Roberts has ever had to do was watch his young niece Remy battle aplastic anaemia.

Remy was just a toddler when diagnosed with the condition, in which the body stops producing enough new blood cells.

The once-energetic and vivacious girl became lethargic, covered in deep bruises.

Her only hope was a bone marrow transfusion, but finding a match was easier said than done.

"I wanted to be able to save my brother's little girl, but no-one in the family was a match, which was quite unbelievable," Mr Roberts said.

Remy's condition deteriorated she required blood transfusions every seven to 10 days but thankfully an unrelated donor was found.

Now six, she is back at school and getting stronger every day.

"Remy, she was right on the cusp of not being here with us, so to see her with us today is absolutely amazing," he said

Despite not being able to help his niece, Mr Roberts stayed on the bone marrow donor list only one in 1,500 donors are asked to donate in any given year.

Just two years after a random donor saved his niece's life, Mr Roberts got the call.

"I'm six-foot-three and pretty bulletproof. I'm tough as nails and never get a tear in my eye or anything like that, but I couldn't stop actually crying," he said.

"I just couldn't believe I got to pay it back so quickly."

His donation helped save a five-year-old boy who was also fighting aplastic anaemia.

"The amount of tests and all that you go through is nothing compared to what that little fella was probably going through," Mr Roberts said.

"But that said, I'd do it again in a heartbeat.

"My niece was dying. She got a random match from somebody, and now she's fantastic. I just can't believe you can pay someone back so quickly."

Every 40 minutes, someone in Australia is diagnosed with a blood cancer.

And for most, a blood stem cell or bone marrow transplant from a stranger is their only hope.

But if you are Indigenous or ethnically diverse, the chances of finding a match are much harder, because donors need to have the same genetic background as the recipient.

Less than 1 per cent of people on the bone marrow donor registry are from Aboriginal or Middle Eastern background, less than 3 per cent are Asian, and less than 5 per cent are Pacific islander.

The head of the registry, Lisa Smith, said despite Australia's multicultural make-up, eight of every 10 people on the registry were of north Caucasian background.

"The registry itself is largely unknown and really lacking diversity that reflects Australia's multiculturalism," she said.

"It genuinely is one-in-a-million odds to get a match, so when we ring a donor, they could very well be the only person on the planet that's able to help that patient."

The Australian Bone Marrow Donor Registry is trying to find 5,000 new donors this year.

"We need donors that are young and ethnically diverse, because our current donor pool, if characterised as a person, is a middle-aged white female," Ms Smith said.

"From a transplant clinician's perspective, they're looking for donors that are young between 18 and 30, are male and are reflecting the ethnicity of their patient."

Pamela Bou Sejean knows the importance of a strong donor list better than most.

She was diagnosed with Hodgkin lymphoma in 2010, and after chemotherapy and radiation failed, she was told her "last chance" was a stem cell transplant.

Stem cells from the umbilical cord blood of a Spanish donor gave Ms Bou Sejean a second chance at life.

"To know someone's out there that's actually saved my life, 'thank you' doesn't seem enough," she said.

She went into remission in 2012 and founded the charity Ur The Cure, which aims to demystify stem-cell donations and promote greater diversity on the donor list.

"Often it's people's last chance for a cure, and you need a stem cell match with someone who shares an ethnical or cultural heritage."

There is a 30 per cent chance that a match can generally be found within a person's immediate family. After that, it gets harder.

"It's kind of like winning TattsLotto, but it's not winning money it's winning the chance to live."

For Warrnambool mother Julia Thompson, storing her umbilical cord for stem cells was a no-brainer.

Following her own cancer battle, Ms Thompson gave birth to her son Hugh in 2017.

She is now in remission, having used her stem cells as part of her treatment.

It was that treatment that inspired her to store Hugh's umbilical cord and associated stem cells for future use.

"It's difficult that it's only available in selected hospitals in Australia, but it's really an amazing insurance policy for him," she said.

"We knew that because of my history with cancer, the likelihood of Hugh having siblings was very slim.

"Given my history and the fact that stem cell blood saved my life, I knew how important that was. It's really something to consider for the future of your child and family."

Topics:leukaemia,bones-and-muscles,people,medical-research,health,science-and-technology,human-interest,warrnambool-3280,geelong-3220,melbourne-3000

First posted October 02, 2019 06:54:09

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Bone marrow donor registry pleas for more diversity to help save people with cancer - ABC News

PHOENIX, Oct. 1, 2019 /PRNewswire/ --R3 Stem Cell, a national leader in stem cell therapy marketing and education, today announced that they are launching a new Master Class centered around the topic of stem cells and regenerative procedures. To commemorate their launch, they will be giving away one out of the eight episodes of their Master Class series for free and the rest will be available for only $49. Purchase the R3 Stem Cell Master Class series and receive Stem Cell Therapy with an R3 Center for $500 off. Register for the Master Class at https://stemcellmasterclass.org/.

The Master Class offers those considering regenerative procedures with stem cells vital information such as discovering how the process works, understanding the options that are given to them, learning the research behind it and gaining realistic expectations.

"Experiencing chronic pain is tiring and can lead to anxiety, depression, obesity, and addiction of medication," said David Greene, MD, MBA, Founder and CEO of R3 Stem Cell. "For those who are considering a stem cell procedure, our Master Class will provide them with the knowledge needed in order to make the decision. Questions regarding common misconceptions in the ever-growing field will be uncovered, giving the patient a realistic understanding of what a stem cell is, where they come from and what they can do."

The Master Class series explains what to expect with a regenerative procedure investment, what the different ways are to optimize stem cell therapy outcomes, what to look for in a stem cell clinic, and a patient's real-life experience with a stem cell procedure. The series is divided into eight different episodes, offering the patient a four-hour long learning opportunity with information on discovering a procedure that is right for them.

"It is important for patients to fully understand what type of stem cell therapies are being offered to them and know the full effect it can have on their everyday lives," continues Dr. Greene. "Regenerative therapies may give patients the opportunity to return to pain-free personal everyday activities. Our purpose is to make people educated consumers in a time when so much misinformation is being disseminated."

About R3 Stem Cell

R3 Stem Cell offers regenerative stem cell therapies to those who suffer from chronic pain. Their sole purpose is to repair, regenerate, and restore damaged tissue from the body. Giving hope and options for patients of relieving chronic pain and avoiding surgery. Stem cell procedures are done using bone marrow, adipose, amniotic, PRP or umbilical cord tissue containing platelets, cytokines, growth factors and exosomes that work to start a healing process within the body and repair damaged tissue. R3 Stem Cell has over 35 nationwide Centers and there is most likely a clinic near you. To learn more about R3 Stem Cell, visit their website at http://www.r3stemcell.comor call (844) GET-STEM.

Media Contact

Jennifer RodriguezFirecracker PR(888)317-4687 ext. 703223962@email4pr.com

SOURCE R3 Stem Cell

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R3 Stem Cell Launches Master Class Series on Stem Cell Therapy and Regenerative Medicine - PRNewswire

Anthony DeAngelis reads the birthday card his daughter Alyssa gave him after she donated two-thirds of her liver so he could undergo a transplant. Jerry Carino, @njhoopshaven

Such encounters are rare, especially across an ocean. 'She's like my daughter,' Lael McGrath said.

After four months of battling acute myeloid leukemia, Lael McGraths lifeline arrived in a cooler, the kind youd take to a football tailgate or picnic in the park.

Only it didnt contain ham-and-cheese subs from the local deli. These were stem cells from Germany, a lifesaving gift from an anonymous donor.

Last week McGrath, a grandmother of six who lives in Toms River, got to thank that donor face to face. Wiebke Rudolph visited New Jersey and the two celebrated their connection at Robert Wood Johnson University Hospital in New Brunswick.

For me its very emotional, McGrath said. Shes like my daughter. Thats how I feel. Its a life relationship that will always be there.

Paying it forward:Neptune family holds blood drive in honor of 2-year-old with cancer

Neptune: Daughter donates two-thirds of her liver, giving dad 2nd chance after alcoholism

WATCH: In the video atop this story, Neptune's Anthony DeAngelis reads a birthday card from his daughter, who donated two-thirds of her liver to him.

Bone marrow donor Wiebke Rudolph from Germany (left) hugs transplant recipient Lael McGrath of Toms River (right) at Robert Wood Johnson University Hospital New Brunswick.(Photo: Daniel DellaPiazza)

Fitness always has been a priority for McGrath, who turned 68 on Monday. In August of 2016 she didnt think much of it when her breathing became a bit labored. The flu, she figured.

Because I was such a fit person, my body didnt have any sickly warnings, she said.

The diagnosis was acute myeloid leukemia (AML), which occurs mostly in older adults the average age at diagnosis is 68. According to the American Cancer Society, there are about 21,000 new cases and 11,000 deaths from AML each year in the U.S. For treatment, McGrath was referred to the Blood and Marrow Transplant Program at Rutgers Cancer Institute of New Jersey and Robert Wood Johnson University Hospital New Brunswick. She would need a stem cell transplant.

Every four minutes a new person in the U.S. is being diagnosed with blood cancer and will need to find a donor, said Dr. Vimal Patel, McGraths hematologist/oncologist at Rutgers Cancer Institute of New Jersey and assistant professor of medicine at Rutgers Robert Wood Johnson Medical School.It can be hard (to find a match). We first look at siblings; each sibling has a 1-in-4 chance of being a match.

Do you enjoy tales of remarkable people in Monmouth and Ocean counties? Maybe you'd like to read about the Miss NJ USA contestant who learned to dealwith the suicide of her mother and grandmother and help others too. Or how a woman with Lyme disease wouldn't give up her dream of becoming a doctor. You can read more of them by becoming a digital subscriber to APP.com and downloading our app today.

(from left) Bone marrow transplant coordinator Mary Kate McGrath, transplant recipient Leal McGrath, hematologist/oncologist Dr. Vimal Patel, and bone marrow donor Wiebke Rudolph at a special celebration with members of the Blood and Marrow Transplant team at Rutgers Cancer Institute of New Jersey and Robert Wood Johnson University Hospital, New Brunswick.(Photo: Daniel DellaPiazza)

None of McGraths siblings came up as a match. So McGraths care team turned to Be The Match, the worlds largest bone-marrow donor registry. They hit the jackpot with Rudolph, although her identity remainedanonymous in keeping with widely accepted transplant protocol.

I just did it, and I didnt think much about it, Rudolph said of her decision to enroll in the registry. I didnt think I would ever know the name (of the recipient) or meet them. I just thought, You get some real-life karma if you do that.

The transplant went well. McGrath returned home two weeks later, on Jan. 1,2017. Then came three months of recovery at home. Now, two-and-a-half years later, McGrath says she is in remission, running a couple of days per week and doing power yoga.

Shes amazing, said daughter Torrey DiMeo, who also lives in Toms River. There are 20-year-olds in her (yoga) class and she flies past them. She is still standing on her head at 67.

Bone marrow donor Wiebke Rudolph from Germany (right) stands with transplant recipient Lael McGrath of Toms River (left) at Robert Wood Johnson University Hospital in New Brunswick.(Photo: Daniel DellaPiazza)

Upon learning each others identity, McGrath and Rudolph began a long-distance correspondence that blossomed into a friendship. Last week they met for the first time and visited Robert Wood Johnson together. It was a big moment for all involved.

Its pretty rare in terms of having an unrelated donor meet up with the actual patient themselves, Patel said. To see it happen is remarkable truly heartwarming.

DiMeo was similarly moved.

The emotions were just a whirlwind, she said. Words cant thank someone enough. I really didnt know what to say besides thank you.

McGrath already had begun paying it forward. In 2017 she threw a block party where representatives from Be The Matchregistered dozens of people. One of them could end up rescuing someone halfway around the world, just like Rudolph did.

This is the reason why I do what I do, Patel said. Its amazing to see the pieces of the puzzle that needed to come together, amazing to see the donor and her generosity the gift she gave, the gift of life.

For more information on Be The Match, visit http://www.bethematch.org.

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Jerry Carino is news columnist for the Asbury Park Press, focusing on the Jersey Shores interesting people, inspiring stories and pressing issues. Contact him atjcarino@gannettnj.com.

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Toms River grandmother with leukemia meets the stem-cell donor who saved her life - Asbury Park Press

A gene therapy approach using a so-called antibody-drug conjugate (ADC) conditioning regimen led to safe and sustained production of factor VIII (FVIII) in platelets, and prevented joint bleeding in a mouse model of hemophilia A, according to new research.

The study, Nongenotoxic antibody-drug conjugate conditioning enables safe and effective platelet gene therapy of hemophilia A mice, appeared in the journal Blood Advances.

Prior work in mice showed that stem cell-based gene therapy specifically targeting platelets leads to the production of FVIII the clotting protein missing or defective in people with hemophilia A and induces immune tolerance (no development of exacerbated immune response).

However, the procedure required a conditioning regimen with chemotherapy or total body irradiation (TBI) that may be toxic to genetic material (or genotoxic), so patients may not be agreeable to using the protocol.

Safer approaches may come from ADCs, which use antibodies against cell surface proteins for more specific targeting of cell populations.

A team of researchers from the Blood Research Institute, in Milwaukee, tested ADC-based conditioning with stem cell-based F8 gene therapy that targets platelets in a mouse model of hemophilia A. This ADC consists of saporin a plant-derived toxin that halts protein production bound to antibodies specific for the CD45.2 and CD117 cell surface proteins.

The scientists found that, three weeks after hematopoietic stem cell transplant (HSCT), peripheral blood counts were higher with ADC conditioning than with TBI. This suggested a lower risk of cytopenia (reduced number of mature blood cells), the investigators said.

Then they observed that the new conditioning regimen led to effective engraftment, the process through which transplanted stem cells establish themselves in the bone marrow and start producing new blood cells. At 20 weeks after transplant, all mice receiving the ADC regimen had more than 15% of donor-derived white blood cells.

Sixteen weeks after HSCT, the number of copies of therapeutic genes was similar in the mice receiving ADC and the controls on TBI. Two-thirds of the mice receiving the viral-delivered gene therapy under ADC conditioning showed sustained increases in FVIII levels in platelets.

The ADC regimen also led to increasing reconstitution of platelets and white blood cells, meaning a higher percentage of cells derived from donors.

Subsequent experiments showed that ADC also targeting the CD8 protein led to significantly higher white blood cell reconstitution, frequency of regulatory T-cells (which dampen excessive immune responses), and FVIII levels in platelets than the regimen specific for CD117 and CD45.2 only.

These effects were sustained long-term, as found when transplanting bone marrow cells from animals that had undergone HSCT to new mice under the same conditioning regimen.

Blood clotting was normalized with this gene therapy approach, and hemoglobin levels were higher than in mice with no FVIII. Joint bleeding and limping were effectively prevented in a knee joint injury model. Also, no animal with sustained FVIII expression in platelets developed anti-FVIII inhibitors.

The central finding of this report is that platelet-directed HSC-based FVIII gene therapy is safe and effective for eliminating [hemophilia A] using nongenotoxic hematopoietic-targeted ADC conditioning, the researchers wrote.

This safe and effective treatment strategy could be especially meaningful for [hemophilia A] patients who are especially wary of standard preconditioning, they added.

Jos is a science news writer with a PhD in Neuroscience from Universidade of Porto, in Portugal. He has also studied Biochemistry at Universidade do Porto and was a postdoctoral associate at Weill Cornell Medicine, in New York, and at The University of Western Ontario in London, Ontario, Canada. His work has ranged from the association of central cardiovascular and pain control to the neurobiological basis of hypertension, and the molecular pathways driving Alzheimers disease.

Total Posts: 121

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

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Hemophilia A Study Finds Benefits With New Gene Therapy Approach - Hemophilia News Today

SAN DIEGO, Sept. 30, 2019 /PRNewswire/ -- Results from a study sponsored by Stemedica Cell Technologies, Inc., a global biotechnology company that uses allogeneic stem cells for ischemic conditions, form the basis for a peer-reviewed paper published inStrokeentitled "Phase I/II Study of Safety and Preliminary Efficacy of Intravenous Allogeneic Mesenchymal Stem Cells in Chronic Stroke." Co-authors include Michael L. Levy, MD, PhD, John R. Crawford, MD, Nabil Dib, MD, Lev Verkh, PhD, Nikolai Tankovich, MD, PhD and Steven C. Cramer, MD.

As indicated in theStrokepublication, "Stroke is perennially among the leading causes of human disability and the leading neurological cause of lost disability-adjusted life years. The mean survival after stroke is 6-7 years, with more than 85% of patients living past the first-year post-stroke, many with years of enduring disability. Many restorative therapies are under study to improve outcomes after stroke." However, restorative therapies often have a short time window for improvement usually measured in days-months.

"Based on Stemedica's preclinical data that supported the safety and efficacy of its MSCs as a restorative therapy to improve outcomes after stroke, the company was granted approval by the FDA to conduct a Phase I/IIa dose escalation trial that examined the effects of a single IV infusion of Stemedica's cGMP manufactured allogeneic ischemia-tolerant MSCs," said Dr. Lev Verkh, Chief Regulatory and Clinical Development Officer of Stemedica.

Study:The target population included patients with chronic ischemic stroke and substantial functional deficits; a group for whom treatment options remained limited. The primary outcome of the study was safety, based on serial measures of behavior, CT scans, and laboratory testing. Four secondary endpoints were scored serially to derive estimates of behavioral changes relatively to the baseline over a period of 12 months: NIH Stroke Scale (NIHSS) for neurological assessment, Barthel Index (BI) for ability to perform daily tasks, Mini-Mental Status Exam (MMSE) for mental status, and Geriatric Depression Scale (GDS) for degree of depression. The study was conducted at three centers: University of California, San Diego (UCSD); Mercy Gilbert Medical Center, Gilbert, Arizona; and University of California, Irvine (UCI).

Entry criteria included ischemic stroke >6 months prior to administration, substantial functional deficits (subject confined to a wheelchair, had home-nursingcare, orneeded assistance withactivities ofdailyliving), no substantial improvement in neurologic orfunctional deficits for the2 months prior to enrollment in thestudypermedical history, and NIHSS score=6-20.

Enrollees received a single intravenous dose of allogeneic mesenchymal bone marrow cells. Phase I used a dose escalation design (3 tiers, n=5 each). Phase IIa (n=21) was an expanded safety cohort. The primary endpoint was safety over 1-year. Secondary endpoints examined behavior, with a pre-specified focus at 6-months.

Subject status at enrollment prior to treatment:At baseline, subjects (n=36) averaged 4.24.6 years post-stroke, age 61.110.8 years, NIHSS score 8 [6.5-10], and Barthel Index 6529.

Safety:Study testing disclosed no safety concerns. No subject showed a positive reaction to intradermal testing. In Phase I, each dose (0.5, 1.0, and 1.5 million cells/kg body weight) was found safe, as a result Phase IIa subjects received 1.5 million cells/kg. Two subjects were lost to follow-up, one was withdrawn, and two died (unrelated to study treatment). There were 15 serious adverse events, none possibly or probably related to study treatment. Two mild adverse events were possibly related to study treatment, a urinary tract infection and IV site irritation. Treatment was determined to be safe based on serial exams, EKGs, laboratory tests, and pan-CT scans.

Behavioral Effects:Improvements across all subjects post-transfusion and for all four secondary endpoints were achieved. Improvements in each index were: Barthel Index (6.811.4 points, p=0.002); in NIHSS (-1.251.7 points, p<0.001); Mini Mental Status Exam (1.82.8 points, p<0.001); and Geriatric Depression Scale (-1.63.8 points, p=0.015). At baseline 11.4% (4/35 subjects) had Barthel Index=95-100 (favorable outcome); at 6-months, 27.3% (9/33); by 12-months, 35.5% (11/31).

Conclusions:The current study is the largest trial of intravenous MSCs in patients with chronic stroke and the first to evaluate allogeneic MSC therapy in this population. It is also the first study to evaluate MSCs grown under hypoxic conditions favorable to cell proliferation, gene expression, cytokine production and migration. While patients with stroke in the chronic stage generally show significant functional decline, enrollees in the current study showed 12 months of continued functional improvements across all secondary endpoints.

Intravenous transfusion of allogeneic ischemia tolerant MSCs in patients with chronic stroke and substantial functional deficits was safe and suggested behavioral gains. These data support proceeding to a randomized, placebo-controlled study of this therapy in this population.

Dr. Nikolai Tankovich, President and Chief Medical Officer added, "Stemedica is encouraged by the results of the study which demonstrated safety and preliminary efficacy of its cell therapy product for the treatment of chronic ischemic stroke patients. It is a significant milestone for Stemedica to bring this new cellular medication to patients with debilitating conditions caused by a stroke. Stemedica plans to move forward to a Phase-IIb discussion with the FDA."

Michael Levy, MD, PhD, FACS, FAANS, Professor of Neurosurgery at UCSD and the Principal Investigator of this study commented: "Based on my clinical trial work in Stemedica's Ischemic Stroke trial, my experience to date with Stemedica's allogeneic ischemic tolerant mesenchymal stem cell product suggests that the product is first and foremost safe and secondarilyhas the potential to produce unparalleled medical benefits."

About Stemedica Cell Technologies, Inc.Stemedica Cell Technologies, Inc. is a global biopharmaceutical company that manufactures best-in-class allogeneic adult stem cells. The company is a government licensed manufacturer of cGMP, clinical-grade stem cells currently used in US-based clinical trials for ischemic stroke, and Alzheimer's Disease. Stemedica's cell are also used on a worldwide basis by research institutions and hospitals for pre-clinical and clinical (human) trials. Stemedica is currently developing additional clinical trials for other medical indications using adult, allogeneic stems cell under the auspices of the FDA and other international regulatory institutions. The company is headquartered in San Diego, California and can be found online atwww.stemedica.com.

Forward Looking StatementsThis press release may contain forward-looking statements. Forward-looking statements are based on management's current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance and you are cautioned not to place undue reliance on these forward-looking statements. These statements reflect the views of Stemedica as of the date of this press release with respect to future events and, except as required by law, it undertakes no obligation to update or revise publicly any forward looking statements, whether as a result of new information, future events or otherwise after the date of this press release.

Media ContactStemedica Cell Technologies, Inc.Dave McGuiganEVP, Marketing & Business Developmentdmcguigan@stemedica.com+1 858-658-0910 x7203

SOURCE Stemedica Cell Technologies, Inc.

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Positive Study Results of Phase IIa Clinical Trial Using Intravenous Administration of Mesenchymal Stem Cells for Ischemic Stroke Published in...

CAMBRIDGE, Mass.--(BUSINESS WIRE)--ElevateBio, a Cambridge-based biotechnology holding company, today announced it has established and launched HighPassBio, a company dedicated to advancing novel targeted T cell immunotherapies. The companys lead product is an engineered T cell receptor (TCR) T cell therapy for HA-1 expressing tumors, which is designed to treat and potentially prevent relapse of leukemia following hematopoietic stem cell transplant (HSCT). The product and approach were developed by researchers at Fred Hutchinson Cancer Research Center.

At ElevateBio we are committed to building therapeutics companies with the worlds leading innovators in cell and gene therapy to advance novel treatments that have strong potential to dramatically improve patient lives, said David Hallal, Chairman and CEO of ElevateBio. We look forward to leveraging our centralized industry-leading cell and gene therapy process development and manufacturing capabilities while working closely with Dr. Marie Bleakley and her team, to accelerate their impressive work through clinical development with the goal of serving patients who have no other treatment options. Additionally, we will explore this approach as a potential treatment for other diseases that are treated by stem cell transplants.

HighPassBios scientific founder is Marie Bleakley, M.D., Ph.D., M.ClinEpi, pediatric oncologist and stem cell transplant physician at Fred Hutchinson Cancer Research Center and chair of the scientific advisory board (SAB) of HighPassBio. As the chair of HighPassBios SAB, Dr. Bleakley will continue working with ElevateBio to advance and accelerate this innovative program through clinical development toward patients.

A Phase 1 clinical trial has treated initial patients and is recruiting adult and pediatric patients who have relapsed with leukemia or related conditions following blood and marrow transplantation (also known as stem cell transplantation). More details on http://www.clinicaltrials.gov under the study ID number NCT03326921.

About TCR-Engineered T Cell Therapy

A key role of the immune system is to detect tumor antigens, engage T cells and eradicate the tumor. However, the immune response to tumor antigens varies and is often insufficient to prevent tumor growth and relapse. An approach known as adoptive T cell therapy, using T cell receptors, or TCRs, can overcome some of the obstacles to establishing an effective immune response to fight off the target tumor. TCRs are molecules found on surface of T cells that can recognize tumor antigens that are degraded to small protein fragments inside tumor cells. Unlike CAR T cells that recognize only surface antigens, TCRs can recognize small protein fragments derived from intracellular and surface antigens offering a more diverse way to attack tumors. These small protein fragments show up on the tumor cell surface with another protein called major histocompatibility complex (MHC), get recognized by the TCRs and consequently signal the bodys immune system to respond to fight off and kill the tumor cells.

Tumor-specific TCRs can be identified and then engineered into T cells that recognize and attack various types of cancers, representing a novel approach to treating and potentially preventing disease.

Adoptive T cell therapy can be applied to tackling relapse of leukemia post hematopoietic stem cell transplant (HSCT) by targeting the antigens expressed only by the patient and not by the stem cell transplant donor, known as minor histocompatibility antigens, or HA1. HA1 is expressed predominantly or exclusively on hematopoietic cells and leukemic cells. There is evidence that T cells specific for HA1 can induce a potent and selective antileukemic effect. HA1 TCR T cell therapy is a new investigational immunotherapy for the management of post transplantation leukemia relapse.

About Leukemia post HSCT Treatment and the Risk of Relapse

Leukemia, a cancer of the blood or bone marrow characterized by an abnormal proliferation of blood cells, is the tenth most common type of cancer in the U.S. with an estimated 60,140 new cases and 24,400 deaths in 2016. Leukemia arises from uncontrolled proliferation of a specific type of hematopoietic (blood) cell that is critical for a functional immune system. As a result, when patients are given very high doses of chemotherapy to eradicate leukemic cells most normal cells are killed as well, necessitating a transplant of hematopoietic stem cells from a donor to reconstitute the patients bone marrow and circulating hematopoietic cells. In some cases, the transplanted T cells from the donor can also recognize and eliminate the hematopoietic cells, including leukemia, from the recipient, thus preventing relapse.

While the majority of HSCT recipients are cured, 25-50 percent of HSCT recipients relapse, leukemia relapse remains the major cause of allogeneic HSCT failure, and the prognosis for patients with post-HCT relapse is poor. Relapse occurs following allogeneic HSCT in approximately one-third of patients with acute leukemia who undergo the procedure, and most patients subsequently die of their disease.

About HighPassBio

HighPassBio, an ElevateBio portfolio company, is working to advance a novel approach to treating hematological malignancies by leveraging T cell receptor (TCR)-engineered T cells, known as TCR T cells. The companys lead program is designed to treat or potentially prevent relapse of leukemia in patients who have undergone hematopoietic stem cell transplant (HSCT). The technology was born out of Fred Hutchinson Cancer Research Center by world renowned expert, Dr. Marie Bleakley.

About ElevateBio

ElevateBio, LLC, is a Cambridge-based cell and gene therapy holding company, established to create and operate a broad portfolio of cell and gene therapy companies with leading academic researchers, medical centers and entrepreneurs. ElevateBio builds single- and multi-product companies by providing scientific founders with fully integrated bench-to-bedside capabilities including world-class scientists, manufacturing facilities, drug developers and commercial expertise. ElevateBio BaseCamp, a company-owned Cell and Gene Therapy Center of Innovation, will serve as the R&D, process development and manufacturing hub across the entire ElevateBio portfolio while also supporting selected strategic partners.

ElevateBios lead investors are the UBS Oncology Impact Fund (OIF) managed by MPM Capital, as well as F2 Ventures. Investors also include EcoR1 Capital, Redmile Group, and Samsara BioCapital.

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ElevateBio Launches HighPassBio to Advance Novel Targeted T Cell Immunotherapies with Technology from Fred Hutchinson Cancer Research Center -...

Press release

Gosselies, Belgium, 2 October 2019, 7am CEST BONE THERAPEUTICS (Euronext Brussels and Paris: BOTHE), the leading biotech company focused on the development of innovative cell and biological therapies to address high unmet medical needs in orthopaedics and bone diseases, announces that the Company will today present at the 27th Annual Meeting of the European Orthopaedic Research Society (EORS), in Maastricht, The Netherlands.

The Annual EORS Meeting is Europe's Summit for orthopaedic research and is attended by scientists, clinicians and entrepreneurs in the field. In the oral presentation, Bone Therapeutics will highlight additional preclinical in vitro and in vivo results demonstrating the potent osteogenic properties of its allogeneic bone-forming cell therapy platform, ALLOB, to promote bone-formation and improve fracture healing in relevant models.

ALLOB is the Companys allogeneic product that consists of human bone-forming cells derived from cultured bone marrow mesenchymal stem cells of healthy adult donors, and is manufactured through a proprietary, scalable production process. ALLOB successfully completed two Phase II studies in two indications and the Company is on track to submit a Clinical Trial Application with the regulatory authorities before the end of the year to allow the start of the next clinical development phase in patients with delayed-union fractures.

Presentation Details:

Title: Injectable cryopreserved allogenic bone-forming cells derived from bone marrow MSC (ALLOB) display potent osteogenic and bone repair propertiesAuthors: Sylvain Normand, Delphine De Troy, Coline Muller, Pierre-Yves Laruelle, Anna Tury, Sandra PietriSession: Cellular Regenerative Medicine (FP16)Date: Wednesday, 2 October 2019Time: 5:30pm CESTLocation: Room 08, Maastricht Exhibition & Congress Centre (MECC), Maastricht, The Netherlands

About Bone Therapeutics

Bone Therapeutics is a leading biotech company focused on the development of innovative products to address high unmet needs in orthopaedics and bone diseases. Based in Gosselies, Belgium, the Company has a broad, diversified portfolio of bone cell therapy and an innovative biological product in later-stage clinical development across a number of disease areas, which target markets with large unmet medical needs and limited innovation.

Bone Therapeutics core technology is based on its allogeneic cell therapy platform (ALLOB) which uses a unique, proprietary approach to bone regeneration, which turns undifferentiated stem cells from healthy donors into bone-forming cells. These cells can be administered via a minimally invasive procedure, avoiding the need for invasive surgery, and are produced via a proprietary, cutting-edge manufacturing process.

The Companys ALLOB product pipeline includes a cell therapy product candidate that is expected to enter PhaseII/III clinical development for the treatment of delayed-union fractures and a PhaseII asset in patients undergoing a spinal fusion procedure. In addition, the Company is also developing an off-the-shelf protein solution, JTA-004, which is expected to enter PhaseIII development for the treatment of pain in knee osteoarthritis.

Bone Therapeutics cell therapy products are manufactured to the highest GMP (Good Manufacturing Practices) standards and are protected by a broad IP (Intellectual Property) portfolio covering ten patent families as well as knowhow. Further information is available at http://www.bonetherapeutics.com.

Contacts

Bone Therapeutics SAThomas Lienard, Chief Executive OfficerJean-Luc Vandebroek, Chief Financial OfficerTel: +32 (0) 71 12 10 00investorrelations@bonetherapeutics.com

International Media Enquiries:Consilium Strategic CommunicationsMarieke VermeerschTel: +44 (0) 20 3709 5701bonetherapeutics@consilium-comms.com

For French Media and Investor Enquiries:NewCap Investor Relations & Financial CommunicationsPierre Laurent, Louis-Victor Delouvrier and Arthur RouillTel: + 33 (0)1 44 71 94 94bone@newcap.eu

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

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Bone Therapeutics to present preclinical data on the osteogenic properties of ALLOB in bone repair at 27th Annual Meeting of the European Orthopaedic...

Global Cell Therapy Technologies Market

was valued US$ 12 billion in 2018 and is expected to reach US$ 35 billion by 2026, at CAGR of 12.14 %during forecast period.

The objective of the report is to present comprehensive assessment projections with a suitable set of assumptions and methodology. The report helps in understanding Global Cell Therapy Technologies Market dynamics, structure by identifying and analyzing the market segments and projecting the global market size. Further, the report also focuses on the competitive analysis of key players by product, price, financial position, growth strategies, and regional presence. To understand the market dynamics and by region, the report has covered the PEST analysis by region and key economies across the globe, which are supposed to have an impact on market in forecast period. PORTERs analysis, and SVOR analysis of the market as well as detailed SWOT analysis of key players has been done to analyze their strategies. The report will to address all questions of shareholders to prioritize the efforts and investment in the near future to the emerging segment in the Global Cell Therapy Technologies Market.

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Global Cell Therapy Technologies Market: OverviewCell therapy is a transplantation of live human cells to replace or repair damaged tissue and/or cells. With the help of new technologies, limitless imagination, and innovative products, many different types of cells may be used as part of a therapy or treatment for different types of diseases and conditions. Celltherapy technologies plays key role in the practice of medicine such as old fashioned bone marrow transplants is replaced by Hematopoietic stem cell transplantation, capacity of cells in drug discovery. Cell therapy overlap with different therapies like, gene therapy, tissue engineering, cancer vaccines, regenerative medicine, and drug delivery. Establishment of cell banking facilities and production, storage, and characterization of cells are increasing volumetric capabilities of the cell therapy market globally. Initiation of constructive guidelines for cell therapy manufacturing and proven effectiveness of products, these are primary growth stimulants of the market.

Global Cell Therapy Technologies Market: Drivers and RestraintsThe growth of cell therapy technologies market is highly driven by, increasing demand for clinical trials on oncology-oriented cell-based therapy, demand for advanced cell therapy instruments is increasing, owing to its affordability and sustainability, government and private organization , investing more funds in cell-based research therapy for life-style diseases such as diabetes, decrease in prices of stem cell therapies are leading to increased tendency of buyers towards cell therapy, existing companies are collaborating with research institute in order to best fit into regulatory model for cell therapies.Moreover, Healthcare practitioners uses stem cells obtained from bone marrow or blood for treatment of patients with cancer, blood disorders, and immune-related disorders and Development in cell banking facilities and resultant expansion of production, storage, and characterization of cells, these factors will drive the market of cell therapy technologies during forecast period.

On the other hand, the high cost of cell-based research and some ethical issue & legally controversial, are expected to hamper market growth of Cell Therapy Technologies during the forecast period

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AJune 2016, there were around 351 companies across the U.S. that were engaged in advertising unauthorized stem cell treatments at their clinics. Such clinics boosted the revenue in this market.in August 2017, the U.S. FDA announced increased enforcement of regulations and oversight of clinics involved in practicing unapproved stem cell therapies. This might hamper the revenue generation during the forecast period; nevertheless, it will allow safe and effective use of stem cell therapies.

Global Cell Therapy Technologies Market: Segmentation AnalysisOn the basis of product, the consumables segment had largest market share in 2018 and is expected to drive the cell therapy instruments market during forecast period at XX % CAGR owing to the huge demand for consumables in cell-based experiments and cancer research and increasing number of new product launches and consumables are essential for every step of cell processing. This is further expected to drive their adoption in the market. These factors will boost the market of Cell Therapy Technologies Market in upcoming years.

On the basis of process, the cell processing had largest market share in 2018 and is expected to grow at the highest CAGR during the forecast period owing to in cell processing stage,a use of cell therapy instruments and media at highest rate, mainly in culture media processing. This is a major factor will drive the market share during forecast period.

Global Cell Therapy Technologies Market: Regional AnalysisNorth America to held largest market share of the cell therapy technologies in 2018 and expected to grow at highest CAGR during forecast period owing to increasing R&D programs in the pharmaceutical and biotechnology industries. North America followed by Europe, Asia Pacific and Rest of the world (Row).

Browse Full Report with Facts and Figures of Cell Therapy Technologies Market Report at: https://www.maximizemarketresearch.com/market-report/global-cell-therapy-technologies-market/31531/

Scope of Global Cell Therapy Technologies Market

Global Cell Therapy Technologies Market, by Product

Consumables Equipment Systems & SoftwareGlobal Cell Therapy Technologies Market, by Cell Type

Human Cells Animal CellsGlobal Cell Therapy Technologies Market, by Process Stages

Cell Processing Cell Preservation, Distribution, and Handling Process Monitoring and Quality ControlGlobal Cell Therapy Technologies Market, by End Users

Life Science Research Companies Research InstitutesGlobal Cell Therapy Technologies Market, by Region

North America Europe Asia Pacific Middle East & Africa South AmericaKey players operating in the Global Cell Therapy Technologies Market

Beckman Coulter, Inc. Becton Dickinson and Company

MAJOR TOC OF THE REPORT

Chapter One: Cell Therapy Technologies Market Overview

Chapter Two: Manufacturers Profiles

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

Chapter Four: Global Cell Therapy Technologies Market Size by Regions

Chapter Five: North America Cell Therapy Technologies Revenue by Countries

Chapter Six: Europe Cell Therapy Technologies Revenue by Countries

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

Chapter Eight: South America Cell Therapy Technologies Revenue by Countries

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

Chapter Ten: Global Cell Therapy Technologies Market Segment by Type

Chapter Eleven: Global Cell Therapy Technologies Market Segment by Application

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

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Global Cell Therapy Technologies Market Industry Analysis and Forecast (2018-2026) - Markets Gazette

By: Portia Wofford

September is National Sickle Cell Awareness Month. Sickle Cell hits close to home for me. My nephew has the trait. My goddaughter has the disease and is frequently in crisis. Several other family members and friends are also fighting this illness.

One of the most common issues they all deal with is treatment by healthcare professionals particularly nurses when they seek treatment for this disease. Its shameful, but often their symptoms often cause them to be mistaken for:

These are all terms thrown at people with Sickle Cell Disease (SCD). The treatment or mistreatment of Sickle Cell patients contradicts our title as the most trusted profession.

So, nurses, what can we do? How do we advocate, treat, and care for this population of patients?

SCD is a group of red blood cell disorders. Unlike healthy red blood cells (RBC) which are round, patients with SCD have RBC that take on a C or sickle shape. Sickle cells die early which causes a constant shortage of red blood cells.

SDC commonly affects those whose ancestors came from:

The only cure for SCD is bone marrow or stem cell transplant.

When sickle cells travel through small blood vessels, they get stuck and clog the blood flow causing excruciating pain and other serious problems including:

SCD warriors and their caregivers report being stigmatized when they seek care. With patients showing signs as early as birth, nurses attitudes can contribute to negative stigmatization and may affect patients' response to sickle cell cues, potentially causing patients not to seek care and negatively impacting patient outcomes.

Cleverly Changing founder Elle Cole's daughter has SCD. She gives a brief description of an ER visit after a physicians assistant at their primary care office suggested her daughter go to the nearest hospital via ambulance.

Elle recalls, In the ER, the nurse was upset and asked why we were there and which clinic sent us. She stated my daughter didnt need any oxygen, the hematologist was busy (but would come in about an hour), and she needed to get a mucus sample. My daughter was scared and started to cry. Then, the nurse told four nurses to join her, and they proceeded to hold my daughter down and extract the mucus from her nostrils. I was completely terrified! My husband was at work. I felt alone and scared with my daughter.

One mom, Shaynise Robinson, drives three to four hours to seek care for her daughter, because of the lack of understanding from nurses and other healthcare professionals at their local hospital.

In an article posted on Pubmed, researchers found that sickle cell patients in one hospital waited for 60% longer to get pain medication although other patients reported less severe pain. They were also triaged into a less serious category. 63% of nurses surveyed said many patients with sickle cell are addicted to opioids, according to another study. But according to Dr. Alexis Thompson, president of the American Society of Hematology, rates of addiction among SCD patients are no higher than the general populations.

Elle Cole, who has a blog that brings awareness to SCD, wants nurses to be patient. Asking questions that indicate that you are genuinely concerned about your patients wellbeing can help open communication with your patient.

Shauna Chin, RN, says, In my experience, in addition to the pain, many patients with SCD exhibit symptoms of depression. The nurse needs to distinguish between solemness due to pain and solemness due to despondence. Many symptoms of depression go undiagnosed and can be remedied by encouraging health providers to engage in dialogue with the patient. She expresses that nurses can advocate best for patients with SCD by identifying early non-verbal signs and symptoms of pain and anticipate their patient's needs.

Ayana Spearman, RN, BSN, believes more education is needed during nursing school. Noting in the last five years, few co-workers were adequately informed about the disease. SCD was just 'glanced over' and not taught about in-depth, in nursing school, she says. Spearman believes that SCD patients need to have a holistic care approach.

Shaynise Robinson encourages nurses to go through diversity training. Give patients the benefit of the doubt. Understand that they are looking for pain relief and equal treatment, she says. Proper bedside manner is a concern for her as well.

Sickle Cell Disease doesn't just affect the body, but the holistic health of those with the disease. Education and awareness are critical for nurses to provide proper care. Shaynise Robinson leaves us with this: The same compassion thats shown to cancer patients should be shown to sickle cell patients.

To learn more about sickle cell visit:

Sickle Cell Disease Association of America

American Sickle Cell Anemia Association

Sickle Cell Society

Portia Wofford is a staff development and quality improvement nurse, content strategist, healthcare writer, entrepreneur, and nano-influencer. Chosen as a brand ambassador or collaborative partner for various organizations, Wofford strives to empower nurses by offering nurses resources for developmentwhile helping healthcare organizations and entrepreneurs create engaging content. Follow her on Instagram and Twitter for her latest.

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The Shocking Culture of Nurses and the Treatment of Patients with Sickle Cell - Nurse.org

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Vor Biopharma, an oncology company pioneering engineered hematopoietic stem cells (eHSCs) for the treatment of cancer, today announced senior appointments to its leadership team. Sadik Kassim, PhD, a cell and gene therapy bioprocessing and translational research expert, joins Vor from Kite Pharma as Chief Technology Officer. Tirtha Chakraborty, PhD, a hematological and gene engineering research specialist with experience at Sana Biotechnology and CRISPR Therapeutics, joins as Vice President of Research. These new positions follow Vors recent move into an integrated headquarters in Cambridge, Mass., the appointment of Robert Ang, MBBS, MBA, as President and Chief Executive Officer and a $42 million Series A financing directed at developing Vors platform technology and advancing its pipeline of eHSC-based candidates.

Vor is bringing a fundamentally novel approach to hematopoietic stem cells to empower targeted cancer therapies, and we are rapidly building an industry-leading team to realize the value in this scientific foundation, said Dr. Ang. Dr. Kassim brings his substantial experience with the complex methods and processes that are required for manufacturing genetically-manipulated cell therapies, and Dr. Chakraborty provides deep expertise in hematology and genetic engineering. Their complementary knowledge will aid Vors expansion, platform development and the move towards our first Investigational New Drug filing for VOR33.

I am impressed that compelling in vivo data already supports the potential of Vors cellular engineering platform to protect healthy cells from antigen-directed therapies via antigen removal, said Dr. Kassim. This is especially noteworthy when therapeutic effectiveness is so often highly limited by co-location of target antigens on healthy immune cells, creating a huge opportunity for Vor to significantly broaden the applicability of these and future therapies.

Its exciting to join the Vor team during this period of accelerated expansion, said Dr. Chakraborty. As a geneticist and cell biologist, I look forward to developing this new approach to treat a range of devastating cancers, beginning with VOR33 in acute myeloid leukemia.

Dr. Kassim is a former Executive Director at Kite Pharma where he led the development of manufacturing processes for autologous CAR- and TCR-based gene-modified cell therapies. Prior to Kite, he served as Chief Scientific Officer at Mustang Bio, where he was the first employee and oversaw the foundational build-out of the companys preclinical and manufacturing activities. Prior to Mustang, Dr. Kassim was Head of Early Analytical Development for Novartis Cell and Gene Therapies Unit, where he contributed to the BLA and MAA filings for Kymriah. Earlier in his career, Dr. Kassim was a research biologist at the National Cancer Institute, where he was involved in early research and CMC work that led to the development of several first-in-human TCR and CAR-T products, including Kites Yescarta. Dr. Kassim has also conducted preclinical immunology research at Janssen and was a research fellow in the University of Pennsylvania Gene Therapy Program, where he led the initial discovery and preclinical studies for an AAV8 gene therapy for familial hypercholesterolemia, a program that is now in the clinic. Dr. Kassim earned his BS in Cell and Molecular Biology from Tulane University and received his PhD in Microbiology and Immunology from Louisiana State University.

Dr. Chakraborty joins Vor from Sana Biotechnology, where he served as the Vice President of Cell Therapy Research. Prior to Sana, Dr. Chakraborty was the Head of Hematology at CRISPR Therapeutics, where his teams work on hemoglobin disorders paved the way for the first clinical trial for the CRISPR industry. Before that, at Moderna Therapeutics, Dr. Chakraborty led synthetic mRNA platform technology research. He was trained as an RNA biologist and an immunologist during his postdoctoral research at Harvard Medical School. Dr. Chakraborty received his PhD from the Tata Institute of Fundamental Research in Mumbai, India.

About VOR33Vors lead engineered hematopoietic stem cell (eHSC) product candidate, VOR33, is in development for acute myeloid leukemia (AML). VOR33 is designed to produce healthy cells that lack the receptor CD33, thus enabling the targeting of AML cells through the CD33 antigen, while avoiding toxicity to the bone marrow. Currently, targeted therapies for AML and other liquid tumors can be limited by on-target toxicity. By rendering healthy cells invisible to CD33-targeted therapies, VOR33 aims to significantly improve the therapeutic window, utility and effectiveness of these AML therapies, with the potential to broaden clinical benefit to different patient populations.

About Vor BiopharmaVor Biopharma aims to transform the lives of cancer patients by pioneering engineered hematopoietic stem cell (eHSC) therapies. Vors eHSCs are designed to generate healthy, fully functional cells with specific advantageous modifications, protecting healthy cells from the toxic effects of antigen-targeted therapies, while leaving tumor cells vulnerable.

Vors platform could potentially be used to change the treatment paradigm of both hematopoietic stem cell transplants and antigen-targeted therapies, such as antibody drug conjugates, bispecific antibodies and CAR-T cell treatments. A proof-of-concept study for Vors lead program has been published in Proceedings of the National Academy of Sciences.

Vor is based in Cambridge, Mass. and has a broad intellectual property base, including in-licenses from Columbia University, where foundational work was conducted by inventor and Vor Scientific Board Chair Siddhartha Mukherjee, MD, DPhil. Vor was founded by Dr. Mukherjee and PureTech Health and is supported by leading investors including 5AM Ventures and RA Capital Management, Johnson & Johnson Innovation JJDC, Inc. (JJDC), Novartis Institutes for BioMedical Research and Osage University Partners.

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Vor Biopharma Hires Senior Cell and Gene Therapy Leaders as Chief Technology Officer and Vice President of Research - Business Wire

SALT LAKE CITY When 7-year-old Ian Dahl was diagnosed with cancer in 1994, his parents thought he was going to die.

They were given three options for treatment, none of them certain to work, recalls Hilary Saunders, a nurse practitioner at Intermountain Healthcares Primary Childrens Hospital oncology unit. For acute myeloid leukemia, the type of cancer Dahl had, she said, no one was sure what would happen.

At that time, the success rate for treating AML was 20% or less, and that was generous, in my opinion, Saunders said.

On Nov. 11 of that year, the young boy was given a bone marrow transplant using cells harvested from his dad, the closest available match at the time and Dahls only chance for survival.

Not a day goes by when I dont think, None of this was really supposed to happen for me. ... My life, my kids, I really appreciate all the little things a lot more, Dahl, 32, of Sandy, said Thursday during a celebration of 25 years of bone marrow transplants at Primary Childrens Hospital.

He was the hospitals 17th bone marrow transplant, and the fourth who survived.

Its the start of a new you, Saunders said about a patient who gets new bone marrow, where the blood cells are produced. The old marrow is deteriorated by the cancer and new marrow makes life possible. Its a whole new immune system so your body can fight. It gives you a new outlook on life, she said.

It gives you a chance to be healthy, Dr. Michael Boyers, medical director of the pediatric blood and marrow transplant program at Primary Childrens Hospital, said Thursday. He said bone marrow transplant is a tough treatment.

In 25 years, doctors at Primary Childrens have performed more than 700 bone marrow transplants, including for cancer patients, but also certain blood-related disorders. The patients came to the hospital from 13 states and four countries.

Doctors have done 43 of the procedures this year, Boyers said.

Ten years ago, he said, bone marrow transplants were proven to cut the death rate in half for cancer patients.

It has evolved a lot, Boyers said.

Dahl doesnt remember much about his lengthy stays in the hospital as a kid, but he will never forget the bad taste of the cyclosporine he was given to combat side effects of chemotherapy, the friendly hospital staff and all the activities he was able to do in the unit, as well as what it was like to be treated like he was special.

Ian is all around, a very special patient, Saunders said, because he was one of the first to undergo the procedure, but also because Dahls family has kept in touch with the doctors and nurses who worked to save his life.

Its been fun to see him grow up. I love seeing that Ian has children, which is another thing about him that is beyond amazing, she said, adding that cancer treatments often preclude bearing children. He has always held a special place in my heart.

Dahl inherited an allergy to cats after the transplant, as his dad was allergic but Dahl wasnt prior to the procedure. He sometimes has issues with eczema, akin to his dad. But looking at him, no one would know he survived cancer as a child, using bone marrow from a family member.

Dahls father has since joined the official Bone Marrow Registry, operated by the nonprofit National Marrow Donor Program, and has had the opportunity to donate his bone marrow multiple times. Dahl said the experience has enriched the lives of everyone in his family.

It was a huge miracle for our family, he said.

Its important that everybody register, Saunders said. Its a beautiful thing to save the life of an adult or child.

The younger the better when it comes to bone marrow donors, Boyers said, as there is less pain resulting from the procedure and a quicker recovery. However, donors of all ages are needed, as well as people from various ethnic backgrounds, specifically Hispanic, Asian or Native American, he said.

Sometimes people are on the registry for years waiting to be called, Boyers said. About one in 430 registrants end up donating to a patient, as they must meet age and health guidelines and be willing to donate to any patient in need. Donors between the ages of 18 and 44 are always needed.

At the time of Dahls procedure, the national registry was quite small. Finding a match was more difficult. His dad met five of six qualifications, whereas his mother and sister each met three of six not enough to be viable.

Siblings, Boyers said, have a one in four chance of being a good match for transplantation, so the larger the registry, the better the odds are of finding a better match. Patients who share the same ancestry are most likely to match.

Be the Match, the worlds largest and most diverse registry, has nearly 35 million registrants. Healthy bone marrow, as it has for Dahl, can be the the cure for someone facing life-threatening disease.

All it takes to register as a bone marrow donor is a cotton swab collection of DNA from the mouth. A person can donate stem cells and/or marrow. For more information, visit bethematch.org.

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Bone marrow patient returns to celebrate 25 years of success at Primary Children's Hospital - KSL.com

T cells in the bone marrow of patients with acute myeloid leukemia (AML) over-express PD-1 leading to anti-tumor activity,1but checkpoint inhibitors have been shown to overcome this in mouse models.2 Cytarabine is known to suppress the expression of PD-1 allowing cytotoxic T lymphocytes (CTLs) to attack AML cells more efficiently, while idarubicin causes the release of antigens which prime CTLs to further promote anti-tumor activity. The combination of both idarubicin and cytarabine has resulted in remission rates of 80%, but despite this high initial response, only 3050% of patients with AML are disease-free long-term2. Alterations of dosing and treatment schedules of this standard induction method have had a limited effect on this outcome.

Professor Farhad Ravandi, The University of Texas MD Anderson Cancer Center, Houston, TX, US, and colleagues conducted a phase II trial to assess nivolumab in combination with idarubicin and cytarabine as a frontline treatment for patients with newly diagnosed AML. They hypothesised that the addition of a further anti-PD-1 agent may improve remission duration by enhancing the anti-tumor activity of CTLs2. Professor Ravandi previously presented this data at the 59th American Society of Hematology (ASH) Annual Meeting and Exposition in 2017 in Atlanta (our interview with him can be found here).

In this single-arm phase II part of the phase I/II study (NCT02464657), 44 patients aged 1860 years (>60 years if eligible for intensive chemotherapy) with newly diagnosed AML (n=42) or high-risk myelodysplastic syndrome (n=2) who had an Eastern Cooperative Oncology Group Performance (ECOG) status of 02 were eligible for inclusion induction treatment.

Induction treatment included a 1.5g/m2, 24-hour infusion of cytarabine daily on Days 14 (three days only for patients >60 years), alongside 12mg/m2 daily on days 13 of idarubicin. Nivolumab was then given on Day 24 at a dose of 3mg/kg which was repeated every two weeks for a year in responders. Initially, a run-in phase was performed with patients with relapsed AML (n=3) who received 1mg/kg nivolumab with idarubicin and cytarabine and no toxicity was observed.

Responders were given consolidation cycles of attenuated doses of idarubicin and cytarabine (up to five) or allogeneic hematopoietic stem cell transplantation (allo-HSCT). The primary endpoint was event-free survival (EFS), with relapse-free survival (RFS) and overall survival (OS) as secondary outcomes. The trial would have stopped if the median EFS was less than seven months or if there was significant toxicity associated with nivolumab use (>10%) at one year.

Grade 12

n (%)

Grade 3

n (%)

Grade 4

n (%)

Nausea

1 (2)

1 (2)

0

Diarrhea

3 (7)

7 (16)

0

Mucositis or stomatitis

1 (2)

0

0

Muscle weakness

0

1 (2)

0

Syncope

0

1 (2)

0

Elevated transaminases

3 (5)

1 (2)

0

Elevated bilirubin

0

1 (2)

0

Febrile Neutropenia

1 (2)

13 (30)

1 (2)

Rash

1 (2)

2 (5)

0

Pneumonitis

1 (2)

0

0

Colitis

1 (2)

1 (2)

1 (2)

Pancreatitis

1 (2)

1 (2)

0

Cholecystitis

0

1 (2)

0

Small bowel obstruction

0

1 (2)

0

Thrombosis or embolism

1 (2)

0

0

Despite a small sample size, short follow-up and a lack of comparator population, the study demonstrates that the use of nivolumab alongside idarubicin and cytarabine as an intensified induction therapy in patients with AML (including those over 60 years old) is safe and feasible. Patients undergoing subsequent allo-HSCT showed promising responses and no increase in complications such as severe GvHD. Whether this combination produces similar outcomes compared to standard induction therapy with or without allo-HSCT needs to be confirmed in larger, randomized trials.

Link:
Nivolumab as an addition to frontline therapy of AML in younger patients - AML Global Portal

Stem cells are found in all human beings, from the initial stages of human growth to the end of life. All stem cells are beneficial for medical research; however, each of the different kinds of stem cells has both limitations and promise. Embryonic stem cells that can be obtained from a very initial stage in human development have the prospect to develop all of the cell types in the human body. Adult stem cells are found in definite tissues in fully developed humans. Stem cells are basic cells of all multicellular animals having the ability to differentiate into a wide range of adult cells. Totipotency and self-renewal are characteristics of stem cells. However, totipotency is seen in very early embryonic stem cells. The adult stem cells owes multipotency and difference flexibility which can be exploited for next generation therapeutic options. Recently, scientists have also recognized stem cells in the placenta and umbilical cord blood that can give rise to several types of blood cells. Research for stem cells is being undertaken with the expectation of achieving major medical inventions. Scientists are attempting to develop therapies that replace or rebuild spoiled cells with the tissues generated from stem cells and offer hope to people suffering from diabetes, cancer, spinal-cord injuries, cardiovascular disease, and many other disorders.

The stem cell therapy market is segmented on the basis of type, therapeutic applications, cell source, and geography. On the basis of type, the stem cell therapy market is categorized into allogeneic stem cell therapy and autologous stem cell therapy. Allogeneic stem cell therapy includes transferring the stem cells from a healthy person (the donor) to the patients body through high-intensity radiation or chemotherapy. Allogeneic stem cell therapy is used to treat patients who do not respond fully to treatment, who have high risk of relapse, and relapse after prior successful treatment. Autologous stem cell therapy is a type of therapy that uses the person's own stem cells. These type of cells are collected earlier and returned in future. The use of stem cells is done to replace damaged cells by high doses of chemotherapy, and to treat the person's underlying disease. On the basis of therapeutic applications, the stem cell therapy market is segmented into cardiovascular diseases, wounds and injuries, musculoskeletal disorders, gastrointestinal diseases, surgeries, neurodegenerative disorders, and others. On the basis of cell source, stem cells therapy is segmented into bone marrow-derived mesenchyme stem cells, adipose tissue-derived mesenchyme stem cells, and cord blood or embryonic stem cells

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By geography, the market for stem cell therapy is segmented into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa. North America leads the stem cell therapy market owing to rising awareness among people, early treatment adoption, and new product innovations. Europe is the second leading market for stem cell therapy due to development and expansion of more efficient and advanced technologies. The Asia Pacific stem cell therapy market is also anticipated to grow at an increasing rate owing to increasing healthcare spending, adoption of western lifestyles, and growth in research and development. Asia Pacific is the fastest growing region for stem cell therapy as several players have invested in the development of new stem cell technologies. These factors are expected to drive the growth of the stem cell therapy market globally during the forecast period.

The major player in the stem cell therapy market are Regenexx, Takara Bio Company, Genea Biocells, PromoCell GmbH, CellGenix GmbH, Cellular Engineering Technologies, BIOTIME, INC., Astellas Pharma US, Inc., AlloSource, RTI Surgical, Inc., NuVasive, Inc., JCR Pharmaceuticals Co., Ltd., Holostem Terapie Avanzate S.r.l., PHARMICELL Co., Ltd, ANTEROGEN.CO., LTD., The Future of Biotechnology, and Osiris Therapeutics, Inc. Rising demand for advanced stem cell therapies will increase the competition between players in the stem cell therapy market.

The report offers a comprehensive evaluation of the market. It does so via in-depth qualitative insights, historical data, and verifiable projections about market size. The projections featured in the report have been derived using proven research methodologies and assumptions. By doing so, the research report serves as a repository of analysis and information for every facet of the market, including but not limited to: Regional markets, technology, types, and applications.

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The study is a source of reliable data on:Market segments and sub-segmentsMarket trends and dynamicsSupply and demandMarket sizeCurrent trends/opportunities/challengesCompetitive landscapeTechnological breakthroughsValue chain and stakeholder analysis

The regional analysis covers:North America (U.S. and Canada)Latin America (Mexico, Brazil, Peru, Chile, and others)Western Europe (Germany, U.K., France, Spain, Italy, Nordic countries, Belgium, Netherlands, and Luxembourg)Eastern Europe (Poland and Russia)Asia Pacific (China, India, Japan, ASEAN, Australia, and New Zealand)Middle East and Africa (GCC, Southern Africa, and North Africa)

The report has been compiled through extensive primary research (through interviews, surveys, and observations of seasoned analysts) and secondary research (which entails reputable paid sources, trade journals, and industry body databases). The report also features a complete qualitative and quantitative assessment by analyzing data gathered from industry analysts and market participants across key points in the industrys value chain.

A separate analysis of prevailing trends in the parent market, macro- and micro-economic indicators, and regulations and mandates is included under the purview of the study. By doing so, the report projects the attractiveness of each major segment over the forecast period.

Highlights of the report:A complete backdrop analysis, which includes an assessment of the parent marketImportant changes in market dynamicsMarket segmentation up to the second or third levelHistorical, current, and projected size of the market from the standpoint of both value and volumeReporting and evaluation of recent industry developmentsMarket shares and strategies of key playersEmerging niche segments and regional marketsAn objective assessment of the trajectory of the marketRecommendations to companies for strengthening their foothold in the market

Note:Although care has been taken to maintain the highest levels of accuracy in TMRs reports, recent market/vendor-specific changes may take time to reflect in the analysis.

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Stem Cell Therapy Market Foraying into Emerging Economies 2017-2025 - Techdadz

GENES are the building blocks of life but like all things, they can sometimes go wrong, resulting in a range of conditions and diseases.

Repairing or replacing these genes with good ones, however, could solve or at the very least treat the problem, and this is what the emerging science of gene therapy is all about.

It was first suggested in the early-1970s that using good DNA (genes are short sections of DNA) to replace defective DNA could treat inherited diseases, and since then scientists have been trying to work out how to do it, both for inherited conditions and many others.

The British Society for Gene and Cell Therapy (bsgct.org) says the first approved human gene therapy took place in 1990, on four-year-old Ashanti DeSilva who had ADA-SCID an inherited disease that prevents normal development of the immune system. The therapy made a huge difference, meaning the little girl no longer needed to be kept in isolation and could go to school.

When the human genome was mapped nearly 20 years ago, the notion that it could potentially unlock therapies capable of fixing genes responsible for some of the worlds most devastating diseases was an idea of the future, says gene therapy expert Professor Bobby Gaspar, speaking on behalf of Jeans for Genes Day, the annual campaign for Genetic Disorders UK (geneticdisordersuk.org).

We are at the forefront of a new era of treatment for genetic diseases using gene and cell therapies. Some of these are one-time, potentially curative investigational therapies that could provide life-changing benefits to patients and their families.

Gaspar says there are currently more than 10 cell and gene therapy products approved in the European Union, ranging from products that treat cancer to rare immune deficiencies. A number of these are approved in the UK and available on its National Health Service in specialised centres.

And with nearly 3,000 clinical gene therapy trials underway worldwide, the number of available treatments is expected to grow significantly over the next few years.

Here, Gaspar a professor of paediatrics and immunology at the UCL Great Ormond Street Institute of Child Health and chief scientific officer at Orchard Therapeutics, a gene therapy company that seeks to permanently correct rare, often-fatal diseases outlines five of the ways gene therapy can cure, stop, or slow a disease...

A variety of efforts are underway to use gene therapy to treat cancer. Some types of gene therapy aim to boost the bodys immune cells to attack cancer cells, while others are designed to attack the cancer cells directly.

One way the body protects itself from cancer is through T-cells, a main component of the immune system. But some cancers are good at avoiding these protection mechanisms, says Gaspar.

Chimeric antigen receptor, or CAR T-cell therapy, is a new form of immunotherapy that uses specially altered T-cells to more specifically target cancer cells.

Some of the patients T-cells are collected from their blood, then genetically modified to produce special CAR proteins on the surface.

When these CAR T-cells are reinfused into the patient, the new receptors help the T-cells identify and attack cancer cells specifically and kill them.

There are more than 250 genetic mutations that can lead to a type of blindness called inherited retinal diseases, or IRD. People with a defect in the RPE65 gene start losing their vision in childhood.

As the disease progresses, patients experience gradual loss of peripheral and central vision, which can eventually lead to blindness.

Gene therapy for some IRD patients became available in 2017, delivering a normal copy of the RPE65 gene directly to the retinal cells at the back of the eye using a naturally-occurring virus as a delivery vehicle.

For children with the genetic disorder spinal muscular atrophy, or SMA, a rare muscular dystrophy, motor nerve cells in the spinal cord are damaged, causing patients to lose muscle strength and the ability to walk, eat or even breathe, says Gaspar.

SMA is caused by a mutation in a gene called SMN which is critical to the function of the nerves that control muscle movement. Without this gene, those nerve cells cant properly function and eventually die, leading to debilitating and often fatal muscle weakness.

Researchers recently developed the first US-approved gene therapy to treat children less than two years of age with SMA.

The therapy is designed to target the cause of SMA by replacing the missing or nonworking gene with a new, working copy of a human SMN gene, helping motor neuron cells work properly.

Researchers believe targeted gene therapy and gene editing may have widespread application for a range of infectious diseases that arent amenable to standard clinical management, including HIV.

Although HIV isnt a hereditary disease, the virus does live and replicate in DNA, Gaspar explains.

Another early but encouraging approach uses a gene editing technology combined with a new long-acting, antiretroviral treatment to suppress HIV replication and eliminate HIV from cells and organs of infected animals.

Gene editing is an approach that precisely and efficiently modifies the DNA within a cell. In this approach, gene editing can knock out a receptor called CCR5 on immune cells used by HIV to enter and invade cells. Without CCR5, HIV may no longer invade and cause disease.

One approach being investigated for a number of rare, often-fatal diseases uses gene-modified blood stem cells with a goal of permanently correcting the underlying cause of disease.

Blood stem cells are taken from the patient, and corrected outside the body by introducing a working copy of the gene into the cells. The gene-corrected cells are then put back into the patient to potentially cure the disease.

Gene-modified blood stem cells have the capacity to self-renew and, once taken up in the bone marrow, can potentially provide a lifelong supply of corrected cells. Because of their ability to become many different types of cells in the body, this approach has the potential to provide a lasting treatment for many different severe and often life-limiting inherited disorders, many of which have no approved treatment options available, says Gaspar.

For instance, ADA-SCID, sometimes referred to as bubble baby syndrome, is a disease where babies lack almost all immune protection, leading to frequent and devastating infections. Left untreated, babies rarely live past two years of age. Standard treatment options are not always effective or can carry significant risks. In 2016, the European Medicines Agency approved Strimvelis, a blood stem cell gene therapy for the treatment of ADA-SCID. Strimvelis was the first approved ex vivo gene therapy product in Europe.

Jeans for Genes Day helped fund some of the earliest work using this type of gene therapy at Great Ormond Street Hospital in 2002, when Rhys Evans, a little boy with SCID, became one of the first children worldwide to be treated by gene therapy.

Jeans for Genes Day aims to raise money for children with life-altering genetic disorders by asking people to donate money for wearing jeans to work, school or wherever they like, on any day between September 16-20. Visit jeansforgenesday.org.

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Five benefits of gene therapies - Echo Live

JavaScript is disabled on your browser. Please enable JavaScript to use all the features on this page.Abstract

Oxidative stress on transplanted bone marrow-derived mesenchymal stem cells (BMSCs) during acute inflammation is a critical issue in cell therapies. N-acetyl-L cysteine (NAC) promotes the production of a cellular antioxidant molecule, glutathione (GSH). The aim of this study was to investigate the effects of pre-treatment with NAC on the apoptosis resistance and bone regeneration capability of BMSCs. Rat femur-derived BMSCs were treated in growth medium with or without 5mM NAC for 6h, followed by exposure to 100MH2O2 for 24h to induce oxidative stress. Pre-treatment with NAC significantly increased intracellular GSH levels by up to two fold and prevented H2O2-induced intracellular redox imbalance, apoptosis and senescence. When critical-sized rat femur defects were filled with a collagen sponge containing fluorescent-labeled autologous BMSCs with or without NAC treatment, the number of apoptotic and surviving cells in the transplanted site after 3 days was significantly lower and higher in the NAC pre-treated group, respectively. By the 5th week, significantly enhanced new bone formation was observed in the NAC pre-treated group. These data suggest that pre-treatment of BMSCs with NAC before local transplantation enhances bone regeneration via reinforced resistance to oxidative stress-induced apoptosis at the transplanted site.

Acute inflammation

Apoptosis

Cell conditioning

Glutathione

Local transplantation

Senescence

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2018 Elsevier Ltd. All rights reserved.

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Preconditioning of bone marrow-derived mesenchymal stem ...

JavaScript is disabled on your browser. Please enable JavaScript to use all the features on this page.Abstract

Bone has well documented natural healing capacity that normally is sufficient to repair fractures and other common injuries. However, the properties of bone change throughout life, and aging is accompanied by increased incidence of bone diseases and compromised fracture healing capacity, which necessitate effective therapies capable of enhancing bone regeneration. The therapeutic potential of adult mesenchymal stem cells (MSCs) for bone repair has been long proposed and examined. Actions of MSCs may include direct differentiation to become bone cells, attraction and recruitment of other cells, or creation of a regenerative environment via production of trophic growth factors. With systemic aging, MSCs also undergo functional decline, which has been well investigated in a number of recent studies. In this review, we first describe the changes in MSCs during aging and discuss how these alterations can affect bone regeneration. We next review current research findings on bone tissue engineering, which is considered a promising and viable therapeutic solution for structural and functional restoration of bone. In particular, the importance of MSCs and bioscaffolds is highlighted. Finally, potential approaches for the prevention of MSC aging and the rejuvenation of aged MSC are discussed.

MSC

Aging

Stem cell niche

Bone healing

Rejuvenation

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2018 Published by Elsevier Ltd.

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Bone marrow mesenchymal stem cells: Aging and tissue ...

What are Bone Marrow and Stem Cells?

Bone marrow is a sponge-like tissue found inside bones. Within bone marrow, stem cells grow and develop into the three main types of blood cells:

Stem cells also grow many other cell types of the immune system.

At IU Health, we offer many types of bone marrow transplant, including:

For this type of transplant, we use your own stem cells. We collect the stem cells and then place them back into your body.

We use this method to treat blood-related cancers like multiple myeloma, non-Hodgkin lymphomas and Hodgkin disease, as well as certain germ-cell cancers.

CAR T-cell therapy is an emerging form of cancer immunotherapy. This therapy involves supercharging a patients T cells, a subtype of white blood cell, to recognize and attack cancer cells.

IU Health is the first healthcare system in Indiana to offer CAR T-cell therapy to treat non-Hodgkin lymphoma and Acute Lymphoblastic Leukemia (ALL).

For this type of transplant, the stem cells of another person are used. The donor can be a relative or a nonrelative whose blood cells are a close match.

The stem cells can come from peripheral (circulating) blood, bone marrow or umbilical cord blood (the blood in the cord connecting a fetus to a placenta).

This method is used to treat blood-related cancers like leukemias and some lymphomas or multiple myeloma. It is also used to treat bone marrow failure disorders like myelodysplastic syndrome (MDS) and aplastic anemia.

If you have an acute leukemia or lymphoma, IU Health Medical Center conducts haploidentical (half-matched) stem cell transplantation. This procedure also greatly expands the potential donor pool, making more patients eligible for the transplant.

Read more from the original source:
Bone Marrow & Blood Stem Cell Transplant | IU Health

Join Be The Match Registry

The first step to being someone's cure is to join Be The Match Registry. If you are between the ages of 18-44, committed to donating to any patient in need, and meet the health guidelines, there are two ways to join.

Join in-person at a donor registry drive in your community.Be The One to Save a Life

Find a donor registry drive

Or join online today:

Join online

If you are between the ages of 18 and 44 patients especially need you. Research shows that cells from younger donors lead to more successful transplants. Doctors request donors in the 18-44 age group 86% of the time.

At donor registry drives, we focus on adding registry members most likely to donate. If you are between the ages of 45 and 60 and want to join the registry, you're welcome to join online with a $100 tax-deductible payment to cover the cost to join.

There are many other ways you can be the cure for patients with blood cancers.

Check outFAQs about donationor call us at 1 (800) MARROW2 for more information about bone marrow donation.

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Learn How to Donate Bone Marrow | Be The Match

Stromal vascular fraction was dramatically better than bone marrow concentrate in its ability to differentiate into cartilage.Two other important features were also well documented in this study. SVF created significantly more colony forming units than BMC, another significant predictor of healing response. Perhaps most importantly, SVF was dramatically better than BMC in its ability to differentiate into cartilage.

Second, a study by Han Chao et al has also demonstrated that fat derived stem cells also have a higher proliferation potential for neural tissue and are a better source for not only cartilage regeneration but also for nervous system regeneration.

The studies gave a very comprehensive look at comparing BMC and SVF in the ability to repair cartilage damage in a same procedure protocol. Every significant measurement comparing bone marrow to adipose tissue for stem cell harvesting demonstrated that adipose derived stem cells provided better cell content and superior ability to differentiate into cartilage than bone marrow. Our extensive clinical experience with the procedure for Colorado patients suffering from pain in the knees, other joints, soft tissue, and a wide range of back problems clearly demonstrates the same.

Using the most effective combination of autologous stem cell sources is one of several criteria to identify a legitimate stem cell clinic. Other important characteristics we recommend paying attention to when choosing a stem cell clinic, include the presence of a physician who owns and operates the clinic, X-ray guided injections administered by a trained injection specialist, and a clinic that takes time to discuss your questions. A review of your imaging and clinical data is needed in order to determine if stem cell therapy is right for you.

*Individual patient results may vary. Contact us today to find out if stem cell therapy may be able to help you.

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Stem Cells from Fat vs. Bone Marrow Best Sources for ...

Inside of our bones is where we find this soft, sponge-like material called bone marrow. This bone marrow is filled with blood-forming stem cells that can either divide and form more blood-forming stem cells, or they can transform into three types of blood cells: white blood cells, red blood cells, or platelets.

This method of stem cell therapy is most commonly used for patients suffering from some types of cancer.

How it Works

There are two types of bone marrow transplants; autologous and allogeneic. An autologous bone marrow transplant is when the stem cells are taken from your own body, while an allogeneic process will use the stem cells from a healthy donor.

The procedure starts with an anesthesia being administered to the patient before a doctor begins harvesting the bone marrow from the hip bone, or sometimes, the sternum. The bone marrow is then moved through a process that removes blood and bone from the marrow. The stem cells are then isolate and will be released into your bloodstream, like a blood transfusion.

Who Can Benefit

The conditions most commonly treated with a bone marrow transplant include:

If you are suffering from any of the above diseases, it doesnt mean you are automatically a candidate for a bone marrow transplant. You need to meet with a physician first to be sure this is the most appropriate treatment for your needs. Here at Stem Cell International, our expert physicians would love to talk with you.

What You Can Expect

If you decide this therapy may be right for you, each one of our patients will meet with a physician to discuss your medical history and desired outcomes of the entire process. This is also important for you and the physician to become more comfortable with each other and be absolutely sure this is the best route for your needs.

Did You Know

If you decide a bone marrow transplant is the best route for your needs, you can expect to see and feel improvements anywhere from 2 to 8 weeks. Although, complete recovery of immune function could take several months.

If youre interested in being treated with a bone marrow transplant at Stem Cell International, one of our stem cell experts would be happy to help you decide. Get in touch today!

Read more here:
BONE MARROW - Stem Cell International

Overview

If you are planning to donate stem cells, you have agreed to allow doctors to draw bone marrow stem cells from either your blood or bone marrow for transplantation.

There are two broad types of stem cells: embryonic and bone marrow stem cells. Embryonic stem cells are studied in therapeutic cloning and other types of research. Bone marrow stem cells are formed and mature in the bone marrow and are then released into the bloodstream. This type of stem cell is used in the treatment of cancers.

In the past, surgery to draw bone marrow stem cells directly from the bone was the only way to collect stem cells. Today, however, it's more common to collect stem cells from the blood. This is called peripheral blood stem cell donation.

Stem cells can also be collected from umbilical cord blood at birth. However, only a small amount of blood can be retrieved from the umbilical cord, so this type of transplant is generally reserved for children and small adults.

Every year, thousands of people in the U.S. are diagnosed with life-threatening diseases, such as leukemia or lymphoma, for which a stem cell transplant is the best or the only treatment. Donated blood stem cells are needed for these transplants.

You might be considering donating blood or bone marrow because someone in your family needs a stem cell transplant and doctors think you might be a match for that person. Or perhaps you want to help someone else maybe even someone you don't know who's waiting for a stem cell transplant.

Bone marrow stem cells are collected from the posterior section of the pelvic bone under general anesthesia. The most serious risk associated with donating bone marrow involves the use and effects of anesthesia during surgery. After the surgery, you might feel tired or weak and have trouble walking for a few days. The area where the bone marrow was taken out might feel sore for a few days. You can take a pain reliever for the discomfort. You'll likely be able to get back to your normal routine within a couple of days, but it may take a couple of weeks before you feel fully recovered.

The risks of this type of stem cell donation are minimal. Before the donation, you'll get injections of a medicine that increases the number of stem cells in your blood. This medicine can cause side effects, such as bone pain, muscle aches, headache, fatigue, nausea and vomiting. These usually disappear within a couple of days after you stop the injections. You can take a pain reliever for the discomfort. If that doesn't help, your doctor can prescribe another pain medicine for you.

For the donation, you'll have a thin, plastic tube (catheter) placed in a vein in your arm. If the veins in your arms are too small or have thin walls, you may need to have a catheter put in a larger vein in your neck, chest or groin. This rarely causes side effects, but complications that can occur include air trapped between your lungs and your chest wall (pneumothorax), bleeding, and infection. During the donation, you might feel lightheaded or have chills, numbness or tingling around your mouth, and cramping in your hands. These will go away after the donation.

If you want to donate stem cells, you can talk to your doctor or contact the National Marrow Donor Program, a federally funded nonprofit organization that keeps a database of volunteers who are willing to donate.

If you decide to donate, the process and possible risks of donating will be explained to you. You will then be asked to sign a consent form. You can choose to sign or not. You won't be pressured to sign the form.

After you agree to be a donor, you'll have a test called human leukocyte antigen (HLA) typing. HLAs are proteins found in most cells in your body. This test helps match donors and recipients. A close match increases the chances that the transplant will be a success.

If you sign up with a donor registry, you may or may not be matched with someone who needs a blood stem cell transplant. However, if HLA typing shows that you're a match, you'll undergo additional tests to make sure you don't have any genetic or infectious diseases that can be passed to the transplant recipient. Your doctor will also ask about your health and your family history to make sure that donation will be safe for you.

A donor registry representative may ask you to make a financial contribution to cover the cost of screening and adding you to the registry, but this is usually voluntary. Because cells from younger donors have the best chance of success when transplanted, anyone between the ages of 18 and 44 can join the registry for free. People ages 45 to 60 are asked to pay a fee to join; age 60 is the upper limit for donors.

If you're identified as a match for someone who needs a transplant, the costs related to collecting stem cells for donation will be paid by that person or by his or her health insurance.

Collecting stem cells from bone marrow is a type of surgery and is done in the operating room. You'll be given an anesthetic for the procedure. Needles will be inserted through the skin and into the bone to draw the marrow out of the bone. This process usually takes one to two hours.

After the bone marrow is collected, you'll be taken to the recovery room while the anesthetic wears off. You may then be taken to a hospital room where the nursing staff can monitor you. When you're fully alert and able to eat and drink, you'll likely be released from the hospital.

If blood stem cells are going to be collected directly from your blood, you'll be given injections of a medication to stimulate the production of blood stem cells so that more of them are circulating in your bloodstream. The medication is usually started several days before you're going to donate.

During the donation, blood is usually taken out through a catheter in a vein in your arm. The blood is sent through a machine that takes out the stem cells. The rest of the blood is then returned to you through a vein in your other arm. This process is called apheresis. It takes two to six hours and is done as an outpatient procedure. You'll typically undergo two to four apheresis sessions, depending on how many blood stem cells are needed.

Recovery times vary depending on the individual and type of donation. But most blood stem cell donors are able to return to their usual activities within a few days to a week after donation.

Recovery times vary depending on the individual and type of donation. But most blood stem cell donors are able to return to their usual activities within a few days to a week after donation.

Explore Mayo Clinic studies testing new treatments, interventions and tests as a means to prevent, detect, treat or manage this disease.

Dec. 20, 2018

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Blood and bone marrow stem cell donation - Mayo Clinic

Stem cell therapies have come a long way since the 1970s and 1980s. Today the ethical issues of harvesting stem cells have long been resolved through the discovery of several sources of potent stem cell types. Common sources include in the umbilical cord and placenta (post birth), bone marrow, and the fatty layer that lies just beneath everyones skin (adipose fat tissue). Of these resources, by far the most commonly accessed in the United States are adipose fat and bone marrow stem cells.The National Stem Cell Institute (NSI), a leading stem cell clinic in the U.S., has seen the development of these living resources usher in an exciting new age known as regenerative medicine. Because of their potency and new technologies that allow ease of access, stem cells are changing the very face of medicine. In particular, the harvesting of bone marrow stem cells has developed into a procedure that is minimally invasive, far more comfortable than bone marrow harvesting of the past, and able to be complete in just a few hours.Some Basics About Bone Marrow Stem CellsBone marrow is the living tissue found in the center of our bones. Marrow is a soft, sponge-like tissue. There are two types of bone marrow: red marrow and yellow marrow. In adults, red marrow is found mainly in the central skeleton, such as the pelvis, sternum, cranium, ribs, vertebrae, and scapulae. But it is also found in the ends of long bones such as in the arms and legs.When it comes to bone marrow stem cells, red marrow is what its all about. Red marrow holds an abundance of them. Stem cells are a kind of protocell that has not yet been assigned an exact physical or neurological function. You can think of them as microscopic packets of potential that stay on high alert for signals telling them where they are needed and what type of cell they need to become.Bone marrow stem cells are multipotent, which means they have the ability to become virtually any type of tissue cell, including:

Original post:
Bone Marrow Stem Cells | NSI Stem Cell

Bone Marrow (BM) contains hematopoietic stem/progenitor cells, which have the potential to self-renew, proliferate, and differentiate into multi-lineage blood cells. Multipotent, non-hematopoietic stem cells, such as mesenchymal stem cells, can be isolated from human BM as well. These non-hematopoietic, mesenchymal stem cells are capable of both self-renewal and differentiation into bone, cartilage, muscle, tendons, and fat. BM is drawn into a 60cc syringe containing heparin (80 U/mL of BM) from the posterior iliac crest, 25 mL/site, from a maximum of four sites.CustomizationLet us know how we can customize your product today Custom InquiryDonor CriteriaAge18-65 years oldWeight>= 130 lbsScreened before donationHIV (HIV 1 & 2 Ab)HBV (Surface Antigen HbsAg)HCV (HCVAb)Donation FrequencyMinimum 10 weeks between donationsDonors with any of the following will be excluded from donatingPregnancyHistory of heart, lung, liver, or kidney diseaseHistory of asthmaBlood and bleeding disorders including sickle cell diseaseNeurologic disordersAutoimmune disordersCancerDiabetesOther CriteriaMust be in general good healthMust have accessible hipsComplete Blood Count lab test must meet protocol specsRequired to sign procedure-specific consent form

Originally posted here:
Whole Bone Marrow - AllCells.com

The problem with the embryonic stem cells are the many complications associated with them. Besides the ethical considerations, from a practical point of view, we are still a long way from being able to utilize these cells in a safe and consistent manner.

When using embryonic stem cells, you are inheriting any potential diseases that the baby may have. For instance, the baby may have a gene that increases susceptibility to cancer. In fact, the embryonic cells themselves may act as a tumor since there is no natural check on these cells. Furthermore, these cells are foreign materials to the body, and the body will react and attack these cells in an immune response. This can sometimes cause a serious medical condition called graft versus host disease. In that case, the patient may have to be placed on immunosuppressant drugslike an organ transplant patient. With our present technology, embryonic stem cells are not the answer. For those reasons, the FDA has put significant restrictions on the use of this type of cell in humans.

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Bone Marrow - Boston Stem Cell Center

Hematopoietic stem cell transplantation (HSCT) is the new name for bone marrow transplantation.

The bone marrow is home to hematopoietic stem cells (HSCs), also called pluripotent stem cells because they can give rise to any cell your body requires at any given moment. These specialized cells play an essential role in replenishing our blood supply on a daily basis to maintain blood counts in a healthy host. These cells can be collected either by performing repeated bone marrow aspirations or by mobilizing HSCs into the circulation using special medications called cytokines (like GCSF, also called neupogen), and filtering them out of your blood using a highly specialized process called apheresis. After they are collected from your body, these stem cells can be preserved by storing them in a chemical called DMSO, and placing them in a freezer. Stem cell transplantation refers to a process whereby the patients HSCs are replaced by new cells (either from yourself [autologous] or someone else [allogeneic] that grow into a healthy hematopoietic system.

There are many types of HSCTs depending on the source of stem cells as described below:

Autologous Stem Cell Transplantation:

Autologous stem cell transplants are predicated on a simple concept: if a little chemotherapy has the potential to cure, than a lot could be even better. For lymphoma that has come back after conventional chemotherapy, this disease is not usually sensitive to lower doses of chemotherapy, so there is a need to consider higher doses. The challenge of course, is that higher doses of chemotherapy, while effective at treating the lymphoma, can also destroy all your bodys normal blood cells. Hence, after receiving high dose chemotherapy, there is a need to re-infuse your own normal stem cells, collected before you get the high dose therapy.

The use of your own stem cells, collected and frozen prior to the high dose therapy, is referred to as an autologous stem cell transplant. The most common indications for this kind of stem cell transplant are recurrent non-Hodgkin lymphoma and Hodgkin lymphoma. Typically, the patient undergoes chemotherapy to put their cancer into remission. At some point during their treatment they are assessed for HSCT that includes evaluation of the marrow to ensure healthy stem cells as well as adequate heart, lung and liver function. If they qualify then the stem cells are collected usually by apheresis.

In this process, stem cells that have been stimulated to divide and mobilized by medications (ex: GCSF or Neupogen) are filtered out of the circulation through an IV and stored for future use. Once the stem cells are collected, the patient undergoes further conditioning chemotherapy to destroy all cancer cells in their body. This kind of treatment can be toxic to stem cells and may result in long term inability to produce blood. The previously collected stem cells are infused back into the patient and after 7 to 10 days the blood counts recover and the patient can go home. Since these are the patients own cells there is no danger of graft rejection or graft versus host disease. The immune system may take up to a year to fully recover.

Allogeneic Stem Cell Transplantation:

Unlike autologous stem cell transplants, allogeneic stem cell transplants are predicated on the idea that if your immune system could not detect and destroy your lymphoma before it became obvious, then maybe an immune system from someone else (a sibling or an unrelated but matched person), can identify your lymphoma as foreign, and mount an immune response against it. The problem of course is that while the donor immune system, now transplanted and growing in a new host (that is the patient), can recognize the lymphoma as foreign (graft versus lymphoma effect, or GVL), it can also recognize the normal organs of the host as foreign, and mount a graft versus host (GVHD) response against your skin, lung, liver, and gastrointestinal tract. Drugs to suppress the immune system, called immunosuppressants, are often used to help control GVHD, but can obviously compromise some of the GVL effect as well. It is a double edge sword you want GVL without the GVHD, but unfortunately the two go and-in-hand. Indications for allogeneic stem cell transplant typically include acute myeloid leukemia, aggressive lymphomas, and stem cell disorders. A donor for a patient is defined by HLA typing of blood and tissues.

HLA stands for Human Leukocyte Antigen, and describes a series of proteins that exist on the surface of all cells in your body, and which is defined genetically. The degree of relatedness between individuals can be described by the similarities or differences in these genes that code for the HLA proteins, and are used to determine who might be a suitable donor for any given patient. The more closely related the individuals (say identical twins), the lower the risk of GVHD, but the lower the risk of GVL. The greater the difference in the HLA, the greater the risk of GVHD, but consequently, the greater the GVL benefit. Of course, if the toxicity of the GVHD is so great, producing increased mortality, then the GVL benefit becomes inconsequential. Thus, allogeneic transplanters walk a very fine line in assessing each patients individual risk and benefit with this type of transplant.

An HLA matched donor is needed for the host to allow the donor blood cells to engraft in the marrow, otherwise they will be rejected by the bodys immune system. The best donor, usually meaning the least degree of graft versus host disease (GVHD), is usually a sibling. Each person has about a 25% chance of having an HLA matched sibling donor. HLA matching is different from blood typing and can be done by a simple blood test or obtaining a swab from the inside of a persons mouth. Should no siblings be identified as a match, than a search is initiated to find an unrelated HLA match through the National Marrow Donor Program (NMDP). Once a match is identified, the patient is admitted to the hospital to receive conditioning chemotherapy and / or radiation therapy. At the end of this treatment, stem cells from the donor are infused into the patient and allowed to engraft. Even with an HLA matched donor there is a considerable risk of GVHD where the new grafted donor cells will attack the patients organs.

After the transplant, the patient is given immunosuppressive medications to prevent this condition, and is required to be on these for a considerable period of time.

Cord blood transplants:

Umbilical cord blood is an excellent source of stem cells and can be used as a source of stem cells in cases where an unrelated donor cannot be found. This has saved the lives of many patients. HSCT is a complicated process that requires a commitment from the patient and their families for the best outcome .You will be referred to a specialized center for HSCT where you will receive further details and education about the process.

Read more here:
Bone Marrow Transplantation: Autologous and Allogeneic ...