Grief is a personal matter.Each of us has our own mechanisms to copethere is no format set in stone, there are no boundaries. For me, the week leading up to my son Arjan Vir's birthday has always been the most difficult to deal with.

I am overwhelmed by a well of emotions: On the one hand, there are all those happy memories, so much excitement building up to planning those wonderful birthday parties themes to be decided, lists to be made, cards to be distributed, menus, games and oh, the return gifts one mustn't forget and then this sudden feeling of hollowness, the sinking depths of which words cannot describe.

Beyond words

I lost my 26-year-old son Arjan Vir to Leukaemia in 2012. Arjan was one of those hugely social people with an enviable optimism about him he loved to have people around him and had the enormous ability to attract people, make friends and share his life with them. His friendships were deeply honest and truly meaningful, there was nothing hollow about them. Those around Arjan loved his happy-go-lucky nature and his laidback attitude towards life.

Losing Arjan did not just leave us his family and friends with an irrevocable sense of vacuum, it was felt by the many lives he had touched in some way or the other. Photo:Simi Singh

My son never lost his ability to make friends despite the battle he was fighting with cancer. Arjan had a battalion of friends in the hospital: ward boys, nurses, lab technicians and resident doctors could be seen about his room whenever they had spare time; some asking for advice on which phone to buy, to have the odd computer issue sorted, if nothing else, just to watch him play computer games.

Losing Arjan did not just leave us his family and friends with an irrevocable sense of vacuum, it was felt by the many lives he had touched in some way or the other.

An intensely sensitive child, Arjan worried more about others than himself he was an avid reader, wrote beautiful poetry and had an imagination that went beyond words.

His passion for computer games had pre-determined his career options, he had decided to study computer graphics and 3D computer animation. Even at the hospital, as he underwent treacherous rounds of chemotherapy, cycle after cycle, his imagination worked overtime planning some game or the other based on his treatment.

Knowing BMT

A Leukaemia patient, Arjan needed a bone marrow transplant (BMT). In a layperson's terms, BMT means that the unhealthy bone marrow is killed under highly sanitised conditions by giving the patient very high doses of chemotherapy and radiation and replaced by a healthy bone marrow. That sounds perfectly simple, but bone marrow transplant remains a complicated and dangerous procedure.

What consequences does that come with?

For the uninitiated, bone marrow is the soft tissue where all our vital blood components RBCs, WBCs, platelets, plasma and stem cells are formed. Killing one's bone marrow essentially means there is no immunity left to take care of our body.

Where does the healthy bone marrow come from if we are to attempt to rid the body of cancer?

There are two broad types of BMT: Autologous where the unhealthy bone marrow from our body is removed, worked upon or mutated and replaced, and the allogenic transplant in which another person's healthy bone marrow replaces our own.

With the second type of transplant come incredible complications and the daunting task of finding the donor bone marrow that must replace ours: one needs to find another person whose DNA is identical to ours. The first and most obvious choice, of course, would be a sibling.

However, the chances of finding the identical DNA HLA typing that matches your siblings' is only 1:4, and if such a match isn't possible, where do we go?

In Arjan's case, our younger son's HLA typing did not match, and the chances of finding an unrelated donor match were one in a million.

This was the worst possible news we could get, worse than the news of Arjan being diagnosed with Leukaemia.

How does one find an identical HLA typing match in this whole world where do you start, whom do you turn to?

[Photo: Weill Cornell Medecine]

Discovering stem cell registry

In 2012, there were no substantial HLA typing registries in India unlike in developed countries, which maintain nationwide registries that are linked to the worldwide bone marrow registry.

The doctors guided us to approach All India Institute of Medical Sciences (AIIMS) while AIIMS did not have a significant registry of its own, it had a membership with the World Marrow Donors Association (WMDA), and hence could do a worldwide search to find an HLA match for Arjan.

However, institutes likeAIIMS have become desensitised to the urgency that such cases demand and we got no response from them.

At the time, Datri in Chennai was the sole functioning stem cell registry it had about 12,000 donors in its data bank, but we did not get a quick response from them either.

Our son's doctors here told us that we were sitting on a "time bomb" we needed to act swiftly, we could lose no time and that's when we decided to take Arjan to the US for his further treatment and then, hopefully, a BMT.

Arjan was distressed to discover the situation in India; when he heard about the lack of registries, his first thought was that once he had recovered, he would set up a meaningful registry at home. His biggest concern was: What do the poor do, where do they go?

And so, five years on, the Arjan Vir Foundation was set up in the memory of our very dear son. Our aim is to run a widespread registry that addresses all blood disorders.

We hope to provide assistance at all stages of treatment, recovery, after care, and the rehabilitation and resettlement of patients.

Registering as a donor is easy: any individual over age 18 can become a donor and be a part of the registry till the age of 60, provided they are healthy.

All that one needs is a simple mouth swab test and the consent to donate stem cells when the need arises. The swabs are sent to a highly-specialised laboratory in the US for HLA typing and the results shared with the worldwide registry maintained by WMDA.

Upon finding a match for a patient, the registry contacts the concerned donor.

The process is not complicated, it is exactly like platelet donation, only a few hours longer: avolunteer must undergo a complete medical check-up prior to donating stem cells and is put on stem cell boosting therapy for about four days before the procedure. No incision is involved and the donor does not require hospitalisation.

It just takes one day of your life and busy schedule to save a life.

***

Today, as I sat down to write this article, I also planned another kind of a celebration for Arjan's birthday on September 6: this year, we are holding a camp to bring about awareness about stem cells and register donors at a university in Noida.

Once again there is excitement, albeit of a different kind one held together with a sense of pathos.

Also read: Memories of my mother that Alzheimer's can't wipe clean

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My son died of cancer: Why I'm celebrating his birthday with stem cell awareness - DailyO

Bone Marrow Stem Cells Comments Off on My son died of cancer: Why I’m celebrating his birthday with stem cell awareness – DailyO | September 7th, 2017

MIAMI, Sept. 6, 2017 /PRNewswire/ --Longeveron LLC, a regenerative medicine company developing cellular therapies, announced today that it treated its first patient in the Company's Phase 2b clinical trial evaluating the safety and efficacy of Longeveron human Allogeneic Mesenchymal Stem Cells (LMSCs) in patients with Aging Frailty Syndrome. This trial is being conducted pursuant to an Investigational New Drug Application (IND) in conformance with U.S. Food & Drug Administration (FDA) regulations. Aging Frailty is a common geriatric medical condition that is serious and life-threatening, and for which there are currently no U.S. Food and Drug Administration-approved therapeutics available.

The clinical trial is designed to enroll 120 subjects from approximately 10 medical centers around the U.S. The primary objective of the study is to evaluate the effect that LMSCs have on functional mobility and exercise tolerance in elderly Aging Frailty subjects. Three different LMSC dose groups will be compared to placebo over 12 months in a randomized, double-blinded, parallel arm design.Specifically, the trial will evaluate changes to the following:

"Frailty Syndrome is a very common and difficult situation to manage from a clinician's and caregiver's standpoint," stated Marco Pahor, M.D., Director of the Institute on Aging at the University of Florida. "The goal of intervention is to stop or slow the progression towards dependence and adverse health outcomes common to the syndrome, and to restore the patient to a state of healthy aging and functional independence. Longeveron's regenerative medicine trial is an important step towards the development of an effective therapeutic."

Allogeneic mesenchymal stem cells (MSCs) were previously tested in a Phase I/2 proof-of-concept study conducted by investigators at the University of Miami'sMiller School of Medicine. In that study, MSCs were shown to be safe and well-tolerated in frail, elderly subjects in a Phase 1 open label single ascending dose trial (publication link here) with a similar safety profile observed in the randomized, placebo-controlled Phase 2 study (publication link here) Subjects treated with a dose of 100 million MSCs showed significant improvements in six minute walking distance, and significant decreases in systemic inflammation, both relative to baseline.

"As individuals age, stem cell production and proliferation decreases, systemic inflammation increases, and a person's ability to repair and regenerate worn out or damaged tissue diminishes," remarked Suzanne Liv Page, Longeveron Chief Operating Officer. "In frail individuals this is particularly problematic. Our hypothesis is that exogenously infused allogeneic mesenchymal stem cells that are derived from the bone marrow of a healthy young donor, and culture expanded in our lab, will have potent regenerative and restorative effects."

Participants in this study must be between the ages of 70 and 85, be diagnosed as mildly to moderately frail due primarily to aging, and be able to walk between 200 and 400 meters over six minutes. Detailed information about the trial, subject eligibility and participating centers can be found by clicking here or by visiting the website http://www.clinicaltrials.gov and entering trial ID: NCT03169231.

About LMSCs

LMSCs is an allogeneic product, which means it is produced from stem cells derived from human donor bone marrow, and not from the patient's own stem cells, (referred to as autologous). LMSCs are manufactured at Longeveron's Cell Processing Facility in Miami, Fl. using a proprietary ex vivo culture expansion process.

About Longeveron

Longeveron is a regenerative medicine therapy company founded in 2014. Longeveron's goal is to provide the first of its kind biological solution for aging-related diseases, and is dedicated to developing safe cell-based therapeutics to revolutionize the aging process and improve quality of life. The company's research focus areas include Alzheimer's disease, Aging Frailty and the Metabolic Syndrome. Longeveron produces LMSCs in its own state-of-the-art cGMP cell processing facility. http://www.longeveron.com

Contact:Suzanne Liv Pagerel="nofollow">spage@longeveron.com305.909.0850

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Every mom anticipates her childs first day at kindergarten.

For Jessica Pequeno, that kind of milestone is something this mom is only now ready to imagine.

The last time the Napa Valley Register wrote about the Pequeno family, it was October 2015. Their then 22month-old toddler Xavier was about to begin the fight of his life against an immune deficiency disorder so rare it had no name.

Today, there is good news about Xavier and his family.

The now 4-year-old completed a grueling, yet successful stem cell transplant, just started his second year of preschool and is making progress with his health, said his mother. Come this time next year, shell be enrolling him in kindergarten.

Every day is different. We still deal with so many unknowns, she said. But, Hes doing so much better than anybody would have ever expected.

Just getting to this point was a long road.

Two years ago, the Pequenos told their story in hopes of finding a bone marrow transplant match for their son. Doctors originally told them there was no match within the family. Fortunately, after a second set of tests, the Pequenos middle son, Higinio Pequeno IV, was identified as a partial match.

That news was awesome, said Jessica Pequeno.

The family prepared for the transplant to take place in June 2015, but a stubborn infection put those plans on hold. By December, his health care team at the University of California at San Francisco wouldnt even give us odds as his percentage of survival because they didnt know, she said.

They finally had a name for his disease IKBa gain of function mutation with ectodermal dysplasia but there were too many unknowns.

Pequeno said she realized the stem cell transplant was a kind of a now-or-never situation.

We just kept saying, We just have to keep doing this. Giving up wasnt an option.

On Dec. 1, 2015, Xavier was admitted to the hospital for the transplant. The process began with eight days of chemotherapy followed by the stem cell transplant.

Putting a line in his femoral artery, blood was collected from Higinio, then 10. Then a machine separated the stem cells from the blood. Higinios stem cells were then given to Xavier. The stem cells were put into a vein, much like a blood transfusion. The stem cells are then meant to travel to the bone marrow, engraft, and hopefully begin making new, normal blood cells.

On the day of the transplant, the whole Pequeno family, including her husband Higinio, son Higinio and daughter Maya and Jessica Pequenos mom were there. Seeing those potentially life-saving cells go into her son was very emotional, said Jessica Pequeno.

We all cried, she said. It was really scary, but you cant stop. You have to keep going.

During the procedure, Xavier was awake, she said. But the side-effects of the chemotherapy were starting to set in. His hair was falling out, and he had stopped eating and drinking because his mouth sores were so bad and painful, she said. He was on morphine for the pain.

The waiting began. Would the stem cell transplant be a success?

The family was told that Xavier would likely spend many months in the hospital. We planned to be separated as a family for at least six months, said Pequeno. We just expected it to be really hard.

She spent her nights in the room with her son, sleeping on a blow-up twin mattress. The rest of her family went back to Napa. Because Jessica was unable to work and her husband couldnt work because he needed to have knee surgery, the family had moved in with Jessicas mother.

Meanwhile, doctors continued to check Xaviers blood to see if his body was responding to the stem cell transplant.

Every day Id ask, Where are we at? his mother said.

And then, one day in early January, the doctors came to see Xavier, and they said, We have good news.

The transplant was starting to work and the new cells were starting to grow, she said.

I cried, said Pequeno. It happened so much faster than what they had expected.

By the end of January, Xavier was well enough to go home to Napa.

It was scary to come home and super exciting, she said.

Back at home, a new routine was created. Xavier was still taking 25 different medications, some multiple times per day. He had a gastrostomy or G tube for feeding the nutritional liquid he eats and a central line a thin, flexible tube used to give medicines, fluids, nutrients, or blood products over a long period of time.

Honestly I dont remember a lot of it. It becomes a big blur, said Pequeno.

The family continued to visit UCSF at least once a week for blood counts and other checks. There were more ups and downs. Infections and illnesses caused him to be hospitalized for days at a time in February, May and June. His central line got infected. He got shingles.

His immune system was still really weak, said Pequeno.

But he kept bouncing back.

Just two weeks ago, doctors finally removed his central line.

It was a huge step, she said.

Challenges remain. Before the stem cell transplant, Xavier had about 5 to 10 percent of a normal immune system. Now he has about 60 to 70 percent, doctors said.

Were starting to learn hes really prone to sinus and respiratory infections, and viruses, said Pequeno. His body just doesnt fight like everyone elses.

Other habits are harder to change.

Before Xavier went to preschool, Pequeno and her family were able to carefully control his exposure to germs.

When he was able to go to preschool, I wasnt in control of those environments anymore. Its really hard. It gets easier, but it takes a while to learn how to kind of let go, she said.

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Today, Xavier takes only six medications a day and can receive infusions of antibodies at home. Visits to UCSF have been cut back to once every four to five weeks.

Developmentally, Xavier is doing well, she said.

He has some hearing loss, which we continue to monitor. Its hard to say if its a side effect of chemo or other drugs. Right now it doesnt affect his speech. He also has skin, hair and teeth health issues to manage.

Xavier doesnt complain at lot, said Pequeno.

Hes always handled everything so well. When he suffered, He would get quiet. Even now when hes not feeling good, instead of crying like many small children would, Xavier is quiet.

Financially, its hard because Im still not able to work, she said.

Xaviers medical care is provided by Partnership HealthPlan/Medi-Cal and California Childrens Services. Her husband went back to work. Pequeno is taking classes at Napa Valley College while her son is in preschool.

I want to be a nurse but I want to go into pediatrics I want to teach parents how to advocate for their kids.

One of the most significant changes for Pequeno was becoming more confident in working with health care providers regarding her sons care.

Nobody could hand me a book when this started (that said) these are the things you need to know and questions to ask. No one told me I was the captain of his team. Her confidence grew. You have to get comfortable in that role.

The past several years have left a lasting imprint on the whole family, she said. Signs of post-traumatic stress have been seen in all family members. Learning coping skills and how to manage stress is important.

Especially for their son Higinio, said his mother. Its not easy for young boy to come to terms with what his brother went through and his own unique contribution.

I dont think any 10-year-old is capable of understanding the weight that carries, she said.

The struggles havent ended, said Pequeno.

Weve just learned to manage them and adjust and deal with the financial part. We juggle. You learn how to change your priorities.

Its easy to say her son looks healthy, said Pequeno, but thats also frustrating because it takes so much work to get him to continue to look like that.

It definitely takes a toll and lot of work and sacrifice to keep him where hes at, she said.

And Xaviers condition isnt going away, she noted. This is something we will manage for the rest of his life one way or the other.

People say, Oh youre so strong. But I think that as a mom, you just do it, said Pequeno. You pull the strength from somewhere. Because you dont give up on your kids.

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A new therapy changes the balance of osteoblasts (pictured here) and fat cells in the bone marrow, leading to stronger bones.

Science Picture Co/Science Source

By Emma YasinskiSep. 5, 2017 , 2:59 PM

A drug that can reverse diabetes and obesity in mice may have an unexpected benefit: strengthening bones. Experiments with a compound called TNP (2,4,6-trinitrophenol, which is also known as picric acid), which researchers often use to study obesity and diabetes, show that in mice the therapy can promote the formation of new bone. Thats in contrast to many diabetes drugs currently in wide use that leave patients bones weaker. If TNP has similar effects in humans, it may even be able to stimulate bone growth after fractures or prevent bone loss due to aging or disuse.

As more and more patients successfully manage diabetes with drugs that increase their insulin sensitivity, doctors and researchers have observed a serious problem: Thedrugs seem to decrease the activity of cells that produce bone, leaving patients prone to fractures and osteoporosis.

There are millions and millions of people that have osteoporosis [with or without diabetes], and it's not something we can cure, says Sean Morrison, a stem cell researcher at University of Texas Southwestern in Dallas. We need new agents that promote bone formation.

Morrison and his colleagues have shown that a high-fat diet causes mice to develop bones that contain more fat and less bone. The diet increased the levels of leptina hormone produced by fat cells that usually signals satiety in the brainin the bone marrow, which promoted the development of fat cells instead of bone cells. That suggests that nutrition has a direct effect on the balance of bone and fat in the bone marrow.

After reading Morrisons work, Siddaraju Boregowda, a stem cell researcher at the Scripps Research Institute in Jupiter, Florida, was reminded of genetically altered mice that dont gain body fat or develop diabetes, even when fed high-fat diets. He and his boss, stem cell researcher Donald Phinney, wondered whetherthose mice were also protected from the fattening of the bone marrow that accompanies a high-fat diet.

They contacted Anutosh Chakraborty, a molecular biologist who was studying such mice down the hall at Scripps at the time. The animals lack the gene for an enzyme called inositol hexakisphosphate kinase 1 (IP6K1), which is known to play a role in fat accumulation and insulin sensitivity. The scientists suspected that the lost enzyme might affect the animals' mesenchymal stem cells (MSCs)stem cells found in the bone marrow that are capable of developing into both thebone cells and fat cells that make up our skeletons. If too many fat cells develop, they take the place of bone cells, weakening the bone.

The researchers fed genetically altered and normal mice a high-fat diet for 8weeks. Not only did the genetically altered mice develop fewer fat cells than their normal counterparts, but their production of bone cells was higher than that of the normal mice, the team reported last month in Stem Cells.

The scientists then set out to see whetherthey could use a drug to achieve the same effect in normal mice. For 8weeks, they fed normal mice a high-fat diet and gave them daily injections of either TNP, a well-known IP6K1 inhibitor, or a placebo. When they analyzed the animals bones and marrow, they found that mice that had received TNP had significantly more bone cells, fewer fat cells, and greater overall bone area. The IP6K1 inhibitor apparently protected the mice from the detrimental effects of the high-fat diet.

The study provided thesurprising result that one new therapy currently being explored to lower insulin resistance promotes, rather than decreases, the formation of bone in mice, says DarwinProckop,a stem cell researcher at Texas A&M College of Medicine in Temple, who was not involved in the work.

The researchers still need to figure out how to deliver TNPs effects only to MSCs, instead of the entire body, given that it sometimes blocks other enzymes along with IP6K1. Inhibition of IP6K1 is a promising target for patients with both diabetes and obesity, Boregowda says. He says he and his colleagues are now enthusiastic about testing their findings in a wide range of bone-related diseases and disorders. It might even help heal broken bones, he speculates.

Phinney, on the other hand, is aiming even higher. He wonders whetherthe therapy could also be useful for space travel, because bones are especially vulnerable to deterioration in zero gravity. Its a whole new field of science and drug discovery.

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Scanning electron micrograph of blood cells. From left to right: human erythrocyte, thrombocyte (platelet), leukocyte. Credit: public domain

For more than a century, physicians have anecdotally noted that patients with an underactive thyroidoften caused by iodine deficiencytended to also have anemia. But the link between thyroid hormone and red blood cell production has remained elusive. That is, until two postdoctoral researchers in the lab of Whitehead Institute Founding Member Harvey Lodish, Xiaofei Gao and Hsiang-Ying "Sherry" Lee, decided to investigate.

During the development of red blood cells, specialized bone marrow stem cells mature through several stages until they finally turn on the genes for hemoglobin and other red blood cell proteins and become mature red blood cells. In order to simulate this process in the lab, researchers have previously found that culturing blood cell progenitors in serum helps them turn on all of the proper proteins to take the final step and become a red blood cell.

Gao and Lee, now Principal Investigators at Westlake Institute for Advanced Study and Peking University, respectively, wondered if something in the serum was key to flipping the switch to becoming a mature red blood cell. To narrow down which of the molecules in the serum is the trigger, Gao and Lee ran the serum through a standard laboratory filter that many of us use everyday for our tap water: charcoal.

Long known for sucking odors out of the air and flavors from water, charcoal attracts and retains hydrophobic (water repellent) molecules. Gao and Lee noticed that once filtered, the serum no longer supported red blood cell production; they deduced that one of the hydrophobic molecules trapped by charcoal is the key to the final step of red blood cell maturation. Gao and Lee determined that when just the thyroid hormone thyroxin is added back to the serum, the red blood cell progenitors once again start down the path to maturation. Thyroid hormone's role is so important in stimulating red blood cell maturation, they discovered, that if it is added at an earlier stage of development, red blood cells short-circuit their usual developmental processes and begin turning into mature red blood cells.

Gao and Lee then teased apart the mechanism behind thyroid hormone's effect on red blood cell maturation. They pinpointed the specific type of receptor inside maturing red blood cells to which thyroid hormone binds. From there, they identified a protein that is necessary for thyroid hormone stimulation and that acts as a regulator of the final step of red blood cell production.

With this better understanding of the connection between thyroid hormone and red blood cell maturation, scientists may be able to identify new therapies that trigger red blood cells maturation in patients with specific types of anemia, including those with an underactive thyroid.

The study is published in PNAS.

Explore further: Low thyroid hormone before birth alters growth and development of fetal pancreas

More information: Xiaofei Gao et al. Thyroid hormone receptor beta and NCOA4 regulate terminal erythrocyte differentiation, Proceedings of the National Academy of Sciences (2017). DOI: 10.1073/pnas.1711058114

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Kathmandu, September 4

Civil Hospital is launching haploidentical stem cell transplant within a few months.

Its a treatment process for patients with blood-related cancers and certain blood disorders.

Patients who need a stem cell transplant and cant find a donor who matches their tissue type will benefit from the transplant. Haploidentical transplant is a modified form of stem cell transplant in which a healthy first degree relative a parent, or sibling can often serve as a donor.

When no matched donor is available, half-matched related (haploidentical) donors are safely used in stem cell transplantation, informed Dr Bishesh Poudyal, associate professor and chief of Clinical Hematology and Bone Marrow Transplant Unit at the hospital.

The cost of the transplant will be around 12 to 15 lakh rupees. People suffering from blood cancer, aplastic anaemia, sickle cell anaemia and thalassemia will benefit from the transplant.

The hospital has been performing allogeneic and autotransplant stem cell transplant where only siblings can be donors.

Nine patients had undergone autotransplant and one had undergone allogeneic stem cell transplant in the hospital after it started bone marrow transplant in the hospital in 2016.

A version of this article appears in print on September 05, 2017 of The Himalayan Times.

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TEL AVIV, Israel, Sept. 5, 2017 /PRNewswire/ -- Cellect Biotechnology Ltd. (NASDAQ: APOP), a developer of stem cells selection technology, announced today that theU.S. Food and Drug Administration(FDA) has granted orphan drug designation for Cellect's ApoGraft for the prevention of acute and chronic graft versus host disease(GvHD) in transplant patients.

GvHD is a transplant associated disease representing an outcome of two immune systems crashing into each other. In many transplantations from donors, and especially in Bone Marrow Transplantations (BMT), the transplanted immune mature cells (as opposed to stem cells) attack the host (patient receiving the transplant) and create severe morbidity and in many cases even death.

This disease happens as a result of current practices being unable to separate the GvHD causing cells from the much needed stem cells.Cellect's ApoGraft was designed to eliminate immune responses in any transplantation of foreign cells and tissues.

Cellect's AppoGraft technology can be utilized already today to help thousands of development and research centers globally engaged in adult stem cells based therapeutics by providing them with a simplified and cost efficient enriched stem cells for use as a raw material for a wide range of stem cells based therapeutics R&D. Before Cellect's ApoGraft, such procedures were extremely complex, inefficient and required substantial resources in both cost, time and infrastructure requirements. ApoGraft can now be used to significantly advance the use of stem cells across multiple therapeutics indications as well as research and biobanking purposes.

The FDA Orphan Drug Act provides incentives for companies to develop products for rare diseases affecting fewer than 200,000 people inthe United States. Incentives may include tax credits related to clinical trial expenses, an exemption from theFDAuser fee, FDAassistance in clinical trial design and potential market exclusivity for seven years following approval.

About Cellect Biotechnology Ltd.

Cellect Biotechnology (NASDAQ: "APOP", "APOPW") has developed a breakthrough technology for the selection of stem cells from any given tissue that aims to improve a variety of stem cell applications.

The Company's technology is expected to provide pharma companies, medical research centers and hospitals with the tools to rapidly isolate stem cells in quantity and quality that will allow stem cell related treatments and procedures. Cellect's technology is applicable to a wide variety of stem cell related treatments in regenerative medicine and that current clinical trials are aimed at the cancer treatment of bone marrow transplantations.

Forward Looking Statements

This press release contains forward-looking statements about the Company's expectations, beliefs and intentions. Forward-looking statements can be identified by the use of forward-looking words such as "believe", "expect", "intend", "plan", "may", "should", "could", "might", "seek", "target", "will", "project", "forecast", "continue" or "anticipate" or their negatives or variations of these words or other comparable words or by the fact that these statements do not relate strictly to historical matters. For example, forward-looking statements are used in this press release when we discuss the Company's pathway for commercialization of its technology. These forward-looking statements and their implications are based on the current expectations of the management of the Company only, and are subject to a number of factors and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. In addition, historical results or conclusions from scientific research and clinical studies do not guarantee that future results would suggest similar conclusions or that historical results referred to herein would be interpreted similarly in light of additional research or otherwise. The following factors, among others, could cause actual results to differ materially from those described in the forward-looking statements: changes in technology and market requirements; we may encounter delays or obstacles in launching and/or successfully completing our clinical trials; our products may not be approved by regulatory agencies, our technology may not be validated as we progress further and our methods may not be accepted by the scientific community; we may be unable to retain or attract key employees whose knowledge is essential to the development of our products; unforeseen scientific difficulties may develop with our process; our products may wind up being more expensive than we anticipate; results in the laboratory may not translate to equally good results in real clinical settings; results of preclinical studies may not correlate with the results of human clinical trials; our patents may not be sufficient; our products may harm recipients; changes in legislation; inability to timely develop and introduce new technologies, products and applications, which could cause the actual results or performance of the Company to differ materially from those contemplated in such forward-looking statements. Any forward-looking statement in this press release speaks only as of the date of this press release. The Company undertakes no obligation to publicly update or review any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by any applicable securities laws. More detailed information about the risks and uncertainties affecting the Company is contained under the heading "Risk Factors" in Cellect Biotechnology Ltd.'s Annual Report on Form 20-F for the fiscal year ended December 31, 2016 filed with the U.S. Securities and Exchange Commission, or SEC, which is available on the SEC's website, http://www.sec.gov. and in the Company's period filings with the SEC and the Tel-Aviv Stock Exchange.

ContactCellect Biotechnology Ltd. Eyal Leibovitz, Chief Financial Officerwww.cellect.co+972-9-974-1444

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At just four-years-old, Erica Honoways son has gone through more than most people will experience in a lifetime.

In February 2016, the family received devastating news, her son Lincoln had been diagnosed with bone marrow failure. He was just three years old at the time.

Lincoln needed a bone marrow transplant, and doctors were only able to find two matches in the entire world. The first donor fell through, so Lincoln was left with only one option.

It was terrifying. We didnt know what we were dealing with, Honoway said. We didnt know what the chances were they would find a match for him. Even if they did, we didnt know if he would make it through the transplant, so it was the scariest experience of our lives.

After the blood transfusions, chemotherapy, radiation and bone marrow transplant, Lincoln is now a happy and active four year old, all thanks to an unknown hero.

This person has just been our angel, Honoway said. We love her and we dont even know her. We say her We have a feeling its a woman but we dont know anything about this person. We dont know where in the world they live, we dont know if its a man or a woman, we dont know anything. But all we know is that they are our hero.

Honoway added that they must wait a minimum of two years before they can meet the donor.

Lincolns successful transplant was the reason Honoway and her family were supporting the University of Reginas Get Swabbed event on Monday, to encourage students between the ages of 17 and 35 to get their cheeks swabbed and enter a national stem call database.

I heard about Erica and Lincoln and I just thought it was amazing how someone just saved his life, and she doesnt even know who he is or who she is, I just think its amazing, U of R Stem Cell Club president Sylvia Okonofua said. I felt like if I take up this initiative and actually run drives where people [can get] on the stem cell registry, [it can] help save a life someday.

Getting students involved and realizing their impact of their involvement through something like this was one of the main goals, U of R student engagement co-ordinator Doug OBrien said. Another goal of having todays Get Swabbed initiative was obviously to support the stem cell database for Canada and through the One Match program.

Approximately 80 students took part in Mondays Get Swabbed event, and organizers are hoping to increase that number for the next event on Sept. 14.

Its a simple way to help save a life.

I hope people realize that they have the opportunity to save someones life, imagine what that would feel like, Honoway said. Youd get to know forever that you saved another humans life. Its pretty special.

2017Global News, a division of Corus Entertainment Inc.

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U of R hosts stem cell swabbing initiative to help save lives through national database - Globalnews.ca

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The US Food and Drug Administration (FDA) intendsto investigate the use of unproven stem cell therapies being offered in the country'sclinics.

Tighter enforcement from the FDA comes as an inspection at StemImmune Inc based in San Diego, California, revealed the use of potentially dangerous treatments administered to vulnerable cancer patients.

Only a small number of stem cell treatments are currentlyFDA approved, including use of bone marrow transplants in cancer patients and cord blood for specific blood-related disorders.However stem cell treatments using only the patient's own cells are not subject to the same level of regulation as drugs if the cells are only 'minimally manipulated'.

FDA commissioner Dr Scott Gottlieb said in a statement:'The FDA will not allow deceitful actors to take advantage of vulnerable patients by purporting to have treatments or cures for serious diseases without any proof that they actually work. I especially wont allow cases such as this one to go unchallenged, where we have good medical reasons to believe these purported treatments can actually harm patients and make their conditions worse.'

Five vials,each containing 100 doses of the live Vaccinia Virus Vaccine, were seized from StemImmune Incby US marshals on25August 2017.

The vaccine, which is usedagainst smallpox, and is not commercially available was combined with stem cells derived from body fat to create an unapprovedtherapy. The concoction was injected directly into tumours of cancer patients at California Stem Cell Treatment Centres in Rancho Mirage and Beverly Hills.

The effects of the vaccine in immunocompromised cancer patients have the possibility to cause severe complications such as inflammation and swelling of the heart and surrounding tissues.

In a separate case, awarning letter was also sent to chief scientific officerKristin Comellaat US Stem Cell Clinic in Sunrise, Florida, after three patients with macular degeneration were blinded following the use of unapproved stem cell injections into their eyes, in a sponsored study (see BioNews 893). The letter lists a number of non-compliance to procedures and 'significant deviations' to current good manufacturing practice and good tissue practice.

'Our actions today should also be a warning to others who may be doing similar harm, we will take action to ensure Americans are not put at unnecessary risk,' Dr Gottlieb commented. 'I also urge health care providers, patients and consumers to report these kinds of activities or any adverse events associated with these unproven treatments to the agency through MedWatch a safety reporting programme.'

Professionals in the field blame the past lack of FDA attention for the widespread problem and are calling for stringent regulation. ProfessorLeigh Turner, fromthe Centre for Bioethics at the University of Minnesota, told CNN: 'This is a space where the FDA could have taken action four or five years ago as far as making this a policy priority.'

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FDA crackdown on unproven stem cell therapies - BioNews

Bone Marrow Stem Cells Comments Off on FDA crackdown on unproven stem cell therapies – BioNews | September 4th, 2017

Irish researcher Prof Daniel Kelly has secured 150,000 in funding to develop a novel implant for treating cartilage damage.

As a recipient of one of the European Research Councils Proof of Concept grants, Prof Daniel Kelly will now spend the next 18 months developing his 3D-printed project entitled Anchor.

Using the 150,000, Kelly will look to develop and commercialise his new medicinal product for cartilage regeneration, employing a postdoctoral researcher to help.

Those active in many sports would be familiar with cartilage damage as a result of injury, of which many cases occur in the knee joint. If left untreated, it can lead to difficulties such as osteoarthritis (OA).

OA can be a debilitating condition, with 80pc of those over the age of 60 experiencing limitations in movement and 25pc saying they cannot perform their major daily activities, according to the World Health Organisation.

Kellys product uses 3D-printed, biodegradable polymer components to make a scaffold, which acts as a template to guide the growth of new tissue by recruiting endogenous bone marrow derived from stem cells.

This, Kelly believes, gives it a competitive edge over similar implants, as standard ones are designed with a finite lifespan, making them unsuitable for younger patients with OA.

Kelly, a principal investigator at AMBER the Trinity College Dublin materials science research centre explained why it could be a major breakthrough for other conditions, such as arthritis.

Our 3D-printed polymer posts will anchor the implant into the bone and will be porous to stimulate the migration of stem cells from the bone marrow into the body of the scaffold, he said.

While various scaffolds like this have been available for some time, they have had limited success, partly because scaffolds need to be anchored securely due to the high forces experienced within the joint. Our 3D-printed posts overcome this problem.

Prior to Anchor, Kelly had worked on this technology in previous projects, such as the ERC-funded StemRepair project to develop a range of porous cartilage-derived scaffolds, and JointPrint.

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Irish researcher bags 150000 to make 3D-printed knee implant - Siliconrepublic.com

Bone Marrow Stem Cells Comments Off on Irish researcher bags 150000 to make 3D-printed knee implant – Siliconrepublic.com | September 4th, 2017

Drugs developed to treat heart and blood vessel problems could be used to treat leukaemia

Drugs developed to treat heart and blood vessel problems could be used in combination with chemotherapy to treat an aggressive form of adult leukaemia.

Researchers at the Francis Crick Institute, Kings College London and Barts Cancer Institute discovered that acute myeloid leukaemia (AML) causes bone marrow to leak blood, preventing chemotherapy from being delivered properly.

Drugs that reversed bone marrow leakiness boosted the effect of chemotherapy in mice and human tissue, providing a possible new combination therapy for AML patients.

To study how AML affects bone marrow, the researchers injected mice with bone marrow from AML patients. Later, they compared their bone marrow with healthy mice using a technique called intravital microscopy that allows you to see biological processes in live animals. They found that pre-loaded fluorescent dyes leaked out of the bone marrow blood vessels in AML mice, but not healthy mice.

Next, the team tried to understand what caused the bone marrow in AML mice to become leaky by studying molecular changes in the cells lining the blood vessels. They found that they were oxygen-starved compared to healthy mice, likely because AML cells use up a lot of oxygen in the surrounding tissue. In response to a reduction in oxygen, there was an increase in nitric oxide (NO) production a molecule that usually alerts the body to areas of low oxygen.

As NO is a muscle relaxant, the team suspected that it might be causing bone marrow leakiness by loosening the tight seams between cells, allowing blood to escape through the gaps. By blocking the production of NO using drugs, the team were able to restore bone marrow blood vessels in AML mice, preventing blood from leaking out. Mice given NO blockers in combination with chemotherapy had much slower leukaemia progression and stayed in remission much longer than mice given chemotherapy alone.

When the vessels are leaky, bone marrow blood flow becomes irregular and leukaemia cells can easily find places to hide and escape chemotherapy drugs, said researcher Dr Diana Passaro. Leaky vessels also prevent oxygen reaching parts of the bone marrow, which contributes to more NO production and leakiness.

By restoring normal blood flow with NO blockers, we ensure that chemotherapy actually reaches the leukaemia cells, so that therapy works properly, she added.

In addition to ensuring that chemotherapy drugs reach their targets, the team also found that NO blockers boosted the number of stem cells in the bone marrow. This may also improve treatment outcomes by helping healthy cells to out-compete cancerous cells.

The team also found that bone marrow biopsies from AML patients had higher NO levels than those from healthy donors, and failure to reduce NO levels was associated with chemotherapy failure.

Our findings suggest that it might be possible to predict how well people with AML will respond to chemotherapy, said Dr Dominique Bonnet, senior author of the paper and Group Leader at the Francis Crick Institute.

Weve uncovered a biological marker for this type of leukaemia as well as a possible drug target. The next step will be clinical trials to see if NO blockers can help AML patients as much as our pre-clinical experiments suggest.

We found that the cancer was damaging the walls of blood vessels responsible for delivering oxygen, nutrients, and chemotherapy. When we used drugs to stop the leaks in mice, we were able to kill the cancer using conventional chemotherapy, said Dr Passaro. As the drugs are already in clinical trials for other conditions, it is hoped that they could be given the green light for AML patients in the future.

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Chemo-boosting drug discovered for leukaemia - Drug Target Review - Drug Target Review

Bone Marrow Stem Cells Comments Off on Chemo-boosting drug discovered for leukaemia – Drug Target Review – Drug Target Review | September 4th, 2017

The bone marrow transplant unit of this hospital was inaugurated in February 2014. The unit was the first of its kind in Cuttack and the government announced that this surgery will be conducted free of cost. Read the full report here.

SCB succeeds in conducting 50 such surgeries free of cost in the past 3 years.

"The successful completion of the surgery is a new milestone for the country and the state." says Rabindra Kumar Jena, head of clinical haematology at SCB. Haematologists revealed that this transplant is conducted when a persons bone marrow becomes weak due to chronic infections, diseases or cancer and does not function properly. In this procedure, new blood stem cells are transplanted which travel to the bone marrow, new blood cells are produced and the growth of a new marrow is promoted.

"A bone marrow transplant replaces your damaged stem cells with the healthy ones. This helps your body make enough white blood cells, platelets or red blood cells to avoid infections, bleeding disorders or anaemia," says haematologist Sudha Sethy.

"The patient is under observation, as the post-operative period is generally critical. She needs to be kept in a sanitised isolation room to avoid infection." Dr Jena informed. He also said that SCB is the only government hospital in the country which provides good quality facilities for bone marrow transplant surgery free of cost. Though the entire process costs two lakhs in a government hospital and over ten lakhs in private hospitals, SCB succeeds in conducting 50 such surgeries free of cost in the past 3 years.

The team of haematologists who succeeded in achieving this goal were R.K. Jena, Sudha Sethy, Rajeeb Nayak, Manmohan Biswal, S.B. Rout and C.R. Kar. "Of all the few 50 patients, who have undergone transplants here, 46 people are living a healthy and normal life. There had been three deaths - two due to infection after the transplant and another after 178 days of the transplant due to brain stroke, which was not in anyway related to the disease." said Jena.

The bone marrow transplant unit of this hospital was inaugurated in February 2014. The unit was the first of its kind in Cuttack and the government announced that this surgery will be conducted free of cost. Odisha State Treatment Fund is funding Nayaks surgery. Another haematologist at the department revealed, "The eldest person to have undergone a bone marrow transplant from the entire continents of Asia and Europe is Zabar Kahan, 74, who was a patient here. We have also conducted the transplant on five patients at age of above 65 years, which is the first of its kind in entire India, Asia and Europe."

He added "We are all set to take more complicated cancer patients for bone marrow transplants. Besides, we are also expanding the unit to accommodate more patients suffering from thalassemia, sickle cell disease and cancer."

SCB adds that at least 3000 people of the state need this surgery.

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SCB Medical College And Hospital Completes Their 50th Bone Marrow Transplant - Doctor NDTV

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Bishnupriya Nayak at BMT unit after bone marrow transplantation | Express

BHUBANESWAR: The Haematology Department of SCB Medical College and Hospital (SCBMCH) at Cuttack has notched up a record of sorts and achieved a new milestone in the country by performing 50 bone marrow transplantations in just over three years.

The special Bone Marrow Transplant (BMT) unit started in February 2014 has conducted its 50th procedure on Bishnupriya Nayak (40), a cancer patient from Koelnagar in Rourkela, on Sunday.Head of the department Prof Rabindra Kumar Jena said it is a significant achievement as SCBMCH having all state-of-the-art facilities is the only State-run hospital in the country to complete 50 cases and provide BMT services completely free of cost.

We have a great record of survival rate of patients than other such units elsewhere in the country. Of 50 cases conducted so far, 47 patients are healthy and doing normal activities. Two died due to infection within a month after BMT procedure, another succumbed to brain stroke (not related to BMT or disease) on 178th day, he said.

The BMT unit at SCBMCH has also established a few international and national distinctions. The eldest transplant conducted so far in Asia and Europe region belonged to the unit. Zabar Khan (74), who was suffering from multiple myeloma (a type of blood cancer) is doing fine after the procedure was performed.Similarly, five patients, aged over 65, have been transplanted successfully which is first-of-its-kind in India, Asia and Europe. The first BMT, also known as stem cell transplant, was performed on Sakuntala Sahoo (54) from Kendrapara district on April 23, 2014.

The unit has also mobilised the stem cell adequately in many complicated blood cancer patients who had very low stem cell blood level of 8.7 per micro litre, besides multiple chemotherapy treated cases and successfully performed BMT procedures.

Stating that the priority is being given on adequate stem cell mobilization, collection and engraftment (proper functioning of new bone marrow graft), Prof Jena said the unit is going to start allogenic BMT soon.

We have been doing autologous transplants so far. Our next plan is to start allogenic transplants. We are poised to take complicated cancer patients for BMT. Besides, plans are afoot to expand the unit to a 20-room ward to accommodate huge waiting lists patients, including thalassemia, sickle sell disease and various cancer patients, he added.

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Bone marrow transplant on record run in SCB Medical College and Hospital at Cuttack - The New Indian Express

Bone Marrow Stem Cells Comments Off on Bone marrow transplant on record run in SCB Medical College and Hospital at Cuttack – The New Indian Express | September 3rd, 2017

In BriefResearchers have discovered that acute myeloid leukemia causes leaking from the bone marrow that interferes with chemotherapy delivery. If chemotherapy for AML can be used together with drugs to treat this problem, outcomes may improve dramatically.

Researchers have discovered that the most common form of acute leukemia which strikes adults, acute myeloid leukemia (AML), prevents chemotherapy from being delivered properly by causing bone marrow to leak blood. This means that, by using drugs developed to treat blood vessel and heart problems in concert with chemotherapy, AML might be much more treatable. In this study, these drugs reversed bone marrow leaks in tissue from mice and humans, and also boosted chemotherapy effects. Since these drugs are already in clinical trials for other applications, the team hopes that they may be approved for use in the treatment of AML patients soon.

We found that the cancer was damaging the walls of blood vessels responsible for delivering oxygen, nutrients, and chemotherapy. When we used drugs to stop the leaks in mice, we were able to kill the cancer using conventional chemotherapy, Diana Passaro, Francis Crick Institute researcher and first author of the paper,said in a press release.

The team studied the ways in which AML affects bone marrow by injecting healthy mice with bone marrow from AML patients to create AML mice. They then used intravital microscopy to compare the bone marrow of AML mice with healthy mice and observed pre-loaded fluorescent dyes leaking from the bone marrow blood vessels into the AML mice. Next, they discovered that the cells lining the blood vessels in AML mice were oxygen-starved, which led to increases in nitric oxide (NO), a muscle relaxant. They realized this was probably causing the leaking, and provided NO blockers to the AML mice which slowed leukemia progress and extended remission.

The team not only helped chemotherapy drugs to reach their targets more effectively, but they also found that NO blockers increased stem cells in the bone marrow. This might help healthy cells out-compete cancerous cells, and improve treatment outcomes. Finally, the researchers found that an inability to reduce NO levels and chemotherapy failure were related.

With an average lifetime risk of less than half of one percent among the general population, AML is relatively rare. This is in addition to the fact that AML is a disease that primarily affects older people; the average age of AML patients in the US is 67, and the disease is even rarer before the age of 45. Despite this rarity, however, TheAmerican Cancer Society estimates that there will be around 21,380 new cases of AML in the US in 2017, and about 10,590 deaths, almost all in adults.

This high death rate is linked to the character of this form of cancer, which is particularly aggressive. This is in large part due to chemotherapy resistance and relapse, fewer than 25 percent of patients survive longer than five years after diagnosis. However, if this research leads to a new regimen of combined drug therapy, these numbers may change.

The team is optimistic about their findings and hopes to start clinical trials soon. Weve uncovered a biological marker for this type of leukemia as well as a possible drug target, Francis Crick Institute Group Leader and paper senior author Dominique Bonnet said in the press release. The next step will be clinical trials to see if NO blockers can help AML patients as much as our pre-clinical experiments suggest.

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Chemo-Boosting Drug Discovered for Leukemia - Futurism

Bone Marrow Stem Cells Comments Off on Chemo-Boosting Drug Discovered for Leukemia – Futurism | September 3rd, 2017

A recent study has identified a key protein capable of regulating the process of new blood cells, including immune cells, which can potentially improve bone and stem cell transplants for donors as well as recipients. The researchers at Technical University of Dresden, Germany, led by the University of Pennsylvania, USA, found that a protein known as Del-1 occupies a key role in the process of hematopoiesis. In addition, researchers inferred that the protein regulator may be modulated to act as potential drug targets in patients affected by certain blood cancers types.

The findings were reported this week (August 28 September 1, 2017) in The Journal of Clinical Investigation.

Del-1 Expression in Hematopoetic Malignancy Key to Boost Myelopoesis in Bone Marrow Transplants

Initially, some of the researchers discovered that Del-1 was the soluble protein that acted as a powerful drug target in gum diseases. Further investigating the role of the protein in hematopoetic malignancy, they inferred that it played a more global role by establishing its expression in a variety of cell types in bone marrow, most notable of them being endothelial cells, CAR cells, and osteoblasts.

The scientists observed that hematopoietic stem cells plays an increasingly important role in various stressful conditions such as bone marrow injury, stem cell transplantation, or systemic infection. These cells affect the production of myeloid cells that forms the core of bone marrow transplants.

Modulating Protein Regulator may Prove Promising in Some Chemotherapies

The team found that the presence of Del-1 in recipient bone marrow facilitated the process of engrafting in recipients by greatly influencing myelopoesis and consequently boosting the formation of new blood cells. The results were observed in experiments conducted in mice suffering with systemic infection. Whereas, in donors, limiting the interaction between the protein and hematopoetic stem cells could boost donor cell numbers in the blood stream, inferred scientists.

Furthermore, the research team observed that the protein regulator also boosts the production of immune-related blood cells. Thus, this may prove to benefit patients suffering with febrile neutropenia who are undergoing chemotherapy.

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Regulating Bone Marrow Protein can Improve Stem Cell Transplants - CMFE News (press release) (blog)

Bone Marrow Stem Cells Comments Off on Regulating Bone Marrow Protein can Improve Stem Cell Transplants – CMFE News (press release) (blog) | September 3rd, 2017

Bone marrow contains hematopoetic stem cells, the precursors to every blood cell type. These cells spring into action following bone marrow transplants, bone marrow injury and during systemic infection, creating new blood cells, including immune cells, in a process known as hematopoiesis.

A new study led by University of Pennsylvania and Technical University of Dresden scientists has identified an important regulator of this process, a protein called Del-1. Targeting it, the researchers noted, could be an effective way to improve stem cell transplants for both donors and recipients. There may also be ways to modulate levels of Del-1 in patients with certain blood cancers to enhance immune cell production. The findings are reported this week in The Journal of Clinical Investigation.

Because the hematopoetic stem cell niche is so important for the creation of bone marrow and blood cells and because Del-1 is a soluble protein and is easily manipulated, one can see that it could be a target in many potential applications, said George Hajishengallis, the Thomas W. Evans Centennial Professor in the Department of Microbiology in Penns School of Dental Medicine and a senior author on the work.

I think that Del-1 represents a major regulator of the hematopoetic stem cell niche, said Triantafyllos Chavakis, co-senior author on the study and a professor at the Technical University of Dresden. It will be worthwhile to study its expression in the context of hematopoetic malignancy.

For Hajishengallis, the route to studying Del-1 in the bone marrow began in his field of dental medicine. Working with Chavakis, he had identified Del-1 as a potential drug target for gum disease after finding that it prevents inflammatory cells from moving into the gums.

Both scientists and their labs had discovered that Del-1 was also expressed in the bone marrow and began following up to see what its function was there.

In the beginning, I thought it would have a simple function, like regulating the exit of mature leukocytes [white blood cells]from the marrow into the periphery, Hajishengallis said, something analogous to what it was doing in the gingiva. But it turned out it had a much more important and global role than what I had imagined.

The researchers investigations revealed that Del-1 was expressed by at least three cell types in the bone marrow that support hematopoetic stem cells: endothelial cells, CAR cells and osteoblasts. Using mice deficient in Del-1, they found that the protein promotes proliferation and differentiation of hematopoetic stem cells, sending more of these progenitor cells down a path toward becoming myeloid cells, such as macrophages and neutrophils, rather than lymphocytes, such as T cells and B cells.

In bone marrow transplant experiments, the team discovered that the presence of Del-1 in recipient bone marrow is required for the transplanted stem cells to engraft in the recipient and to facilitate the process of myelopoesis, the production of myeloid cells.

When the researchers mimicked a systemic infection in mice, animals deficient in Del-1 were slower to begin making myeloid cells again compared to those with normal Del-1 levels.

We saw roles for Del-1 in both steady state and emergency conditions, Hajishengallis said.

Hajishengallis, Chavakis and their colleagues identified the protein on hematopoetic stem cells with which Del-1 interacts, the 3 integrin, perhaps pointing to a target for therapeutic interventions down the line.

The scientists see potential applications in bone marrow and stem cell transplants, for both donors and recipients. In donors, blocking the interaction between Del-1 and hematopoetic stem cells could enhance the mobilization of those progenitors into the bloodstream. This could be helpful for increasing donor cell numbers for transplantation. Transplant recipients, on the other hand, may need enhanced Del-1 interaction to ensure the transplanted cells engraft and begin making new blood cells more rapidly.

In addition, people undergoing chemotherapy who develop febrile neutropenia, associated with low levels of white blood cells, might benefit from the role of Del-1 in supporting the production of immune-related blood cells such as neutrophils.

Its easy to think of practical applications for these findings, said Hajishengallis. Now we need to find out whether it works in practice, so our studies continue.

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Targeting bone marrow protein could be effective way to improve ... - Gears Of Biz

Bone Marrow Stem Cells Comments Off on Targeting bone marrow protein could be effective way to improve … – Gears Of Biz | September 1st, 2017

Scientific pioneer, superstar surgeon, miracle worker thats how Paolo Macchiarini was known for several years. Dressed in a white lab coat or in surgical scrubs, with his broad, handsome face and easy charm, he certainly looked the part. And fooled almost everyone.

Macchiarini shot to prominence back in 2008, when he created a new airway for Claudia Castillo, a young woman from Barcelona. He did this by chemically stripping away the cells of a windpipe taken from a deceased donor; he then seeded the bare scaffold with stem cells taken from Castillos own bone marrow. Castillo was soon back home, chasing after her kids. According to Macchiarini and his colleagues, her artificial organ was well on the way to looking and functioning liked a natural one. And because it was built from Castillos own cells, she didnt need to be on any risky immunosuppressant drugs.

This was Macchiarinis first big success. Countless news stories declared it a medical breakthrough. A life-saver and a game-changer. We now know that wasnt true. However, the serious complications that Castillo suffered were, for a long time, kept very quiet.

Meanwhile, Macchiarinis career soared. By 2011, he was working in Sweden at one of the worlds most prestigious medical universities, the Karolinska Institute, whose professors annually select the winner of the Nobel prize in physiology or medicine. There he reinvented his technique. Instead of stripping the cells from donor windpipes, Macchiarini had plastic scaffolds made to order. The first person to receive one of these was Andemariam Beyene, an Eritrean doctoral student in geology at the University of Iceland. His recovery put Macchiarini on the front page of the New York Times.

Macchiarini was turning the dream of regenerative medicine into a reality. This is how NBCs Meredith Vieira put it in her documentary about Macchiarini, appropriately called A Leap of Faith: Just imagine a world where any injured or diseased organ or body part you have is simply replaced by a new artificial one, literally manmade in the lab, just for you. This marvelous world was now within reach, thanks to Macchiarini.

Last year, however, the dream soured, exposing an ugly reality.

Macchiarini gave his regenerating windpipes to 17 or more patients worldwide. Most, including Andemariam Beyene, are now dead. Those few patients who are still alive including Castillo have survived in spite of the artificial windpipes they received.

In January 2016, Macchiarini received an extraordinary double dose of bad press. The first was a Vanity Fair article about his affair with Benita Alexander, an award-winning producer for NBC News. She met Macchiarini while producing A Leap of Faith and was soon breaking one of the cardinal rules of journalism: dont fall in love with the subject of your story.

By the time the program aired, in mid-2014, the couple were planning their marriage. It would be a star-studded event. Macchiarini had often boasted to Alexander of his famous friends. Now they were on the wedding guest list: the Obamas, the Clintons, Vladimir Putin, Nicolas Sarkozy and other world leaders. Andrea Bocelli was to sing at the ceremony. None other than Pope Francis would officiate, and his papal palace in Castel Gandolfo would serve as the venue. Thats what Macchiarini told his fiancee.

But as the big day approached, Alexander saw these plans unravel, and finally realised that her lover had lied about almost everything. The pope, the palace, the world leaders, the famous tenor they were all fantasies.

Likewise the whole idea of a wedding: Macchiarini was still married to his wife of 30 years.

Macchiarinis deceit was so outlandish, Vanity Fair sought the opinion of the Harvard professor Ronald Schouten, an expert on psychopaths, who gave this diagnosis-at-a-distance: Macchiarini is the extreme form of a con man. Hes clearly bright and has accomplishments, but he cant contain himself. Theres a void in his personality that he seems to want to fill by conning more and more people.

Which left a big, burning question in the air: if Macchiarini was a pathological liar in matters of love, what about his medical research? Was he conning his patients, his colleagues and the scientific community?

The answer came only a couple of weeks later, when Swedish television began broadcasting a three-part expos of Macchiarini and his work.

Called Experimenten (The Experiments), it argued convincingly that Macchiarinis artificial windpipes were not the life-saving wonders wed all been led to believe. On the contrary, they seemed to do more harm than good something that Macchiarini had for years concealed or downplayed in his scientific articles, press releases and interviews.

Faced with this public relations disaster, the Karolinska Institute immediately promised to investigate the allegations but then, within days, suddenly announced that Macchiarinis contract would not be extended.

Macchiarinis fall was swift, but troubling questions remain about why he was allowed to continue his experiments for so long. Some answers have emerged from the official inquiries into the Karolinska Institute and the Karolinska University hospital. They identified many problems with the way the twin organisations handled him.

Macchiarinis fame had won him well-placed backers. These included Harriet Wallberg, who was the vice-chancellor of the Karolinska Institute in 2010, when Macchiarini was recruited. She pushed through his appointment despite the fact that he had some very negative references and dubious claims on his rsum.

This set a dangerous example. It showed department heads and colleagues that they should give Macchiarini special treatment.

He could do pretty much as he pleased. In the first couple of years at Karolinska, he put plastic airways into three patients. Since this was radically new, Macchiarini and his colleagues should have tested it on animals first. They didnt.

Likewise, they didnt undertake a proper risk assessment of the procedure, nor did Macchiarinis team seek government permits for the plastic windpipes, stem cells, and chemical growth factors they used. They didnt even seek the approval of Stockholms ethical review board, which is based at Karolinska.

Though Macchiarini was in the public eye, he was able to sidestep the usual rules and regulations. Or rather, his celebrity status helped him do so. Karolinskas leadership expected big things from their superstar, things that would bring prestige and funding to the institute.

They also cited a loophole known as compassionate use. Macchiarini, they claimed, wasnt really doing clinical research. No, he was just caring for his patients who were, one and all, facing certain death with no other treatment options available and no time to waste. In such dire circumstances, new treatments can be tried as a last resort.

This argument didnt wash with those who later investigated the case. In their view, Macchiarini was certainly engaged in clinical research. Besides which, compassionate concerns dont override the basic principles of patient safety and informed consent. Macchiarini, meanwhile, said he did not accept the findings of the disciplinary board.

As it turned out, Macchiarinis patients werent all at deaths door at the time he treated them. Andemariam Beyene, for instance, had recurrent cancer of the windpipe but, aside from a cough, was still in good health. But even if his days had been numbered, this didnt necessarily justify what Macchiarini put him through.

Beyenes death two and a half years after the operation, caused by the failure of his artificial airway, was a grueling ordeal. According to Pierre Delaere, a professor of respiratory surgery at KU Leuven, Belgium, Macchiarinis experiments were bound to end badly. As he said in Experimenten: If I had the option of a synthetic trachea or a firing squad, Id choose the last option because it would be the least painful form of execution.

Delaere was one of the earliest and harshest critics of Macchiarinis engineered airways. Reports of their success always seemed like hot air to him. He could see no real evidence that the windpipe scaffolds were becoming living, functioning airways in which case, they were destined to fail. The only question was how long it would take weeks, months or a few years.

Delaeres damning criticisms appeared in major medical journals, including the Lancet, but werent taken seriously by Karolinskas leadership. Nor did they impress the institutes ethics council when Delaere lodged a formal complaint.

Support for Macchiarini remained strong, even as his patients began to die. In part, this is because the field of windpipe repair is a niche area. Few people at Karolinska, especially among those in power, knew enough about it to appreciate Delaeres claims. Also, in such a highly competitive environment, people are keen to show allegiance to their superiors and wary of criticising them. The official report into the matter dubbed this the bandwagon effect.

With Macchiarinis exploits endorsed by management and breathlessly reported in the media, it was all too easy to jump on that bandwagon.

And difficult to jump off. In early 2014, four Karolinska doctors defied the reigning culture of silence by complaining about Macchiarini. In their view, he was grossly misrepresenting his results and the health of his patients. An independent investigator agreed. But the vice-chancellor of Karolinska Institute, Anders Hamsten, wasnt bound by this judgement. He officially cleared Macchiarini of scientific misconduct, allowing merely that hed sometimes acted without due care.

For their efforts, the whistleblowers were punished. When Macchiarini accused one of them, Karl-Henrik Grinnemo, of stealing his work in a grant application, Hamsten found him guilty. As Grinnemo recalls, it nearly destroyed his career: I didnt receive any new grants. No one wanted to collaborate with me. We were doing good research, but it didnt matter I thought I was going to lose my lab, my staff everything.

This went on for three years until, just recently, Grinnemo was cleared of all wrongdoing.

The Macchiarini scandal claimed many of his powerful friends. The vice-chancellor, Anders Hamsten, resigned. So did Karolinskas dean of research. Likewise the secretary-general of the Nobel Committee. The university board was dismissed and even Harriet Wallberg, whod moved on to become the chancellor for all Swedish universities, lost her job.

Unfortunately, the scandal is much bigger than Karolinska, which accounts for only three of the patients who have received Macchiarinis regenerating windpipes.

The other patients were treated at hospitals in Barcelona, Florence, London, Moscow, Krasnodar, Chicago and Peoria. None of these institutions have faced the same kind of public scrutiny. None have been forced to hold full and independent inquiries. They should be.

If the sins of Karolinska have been committed elsewhere, it is partly because medical research facilities share a common milieu, which harbours common dangers. One of these is the hype surrounding stem cells.

Stem cell research is a hot field of science and, according to statistics, also a rather scandal-prone one. Articles in this area are retracted 2.4 times more often than the average for biomedicine, and over half of these retractions are due to fraud.

Does the heat of stem cell research the high levels of funding, prestige and media coverage it enjoys somehow encourage fraud? Thats what our experience of medical research leads us to suspect. While there isnt enough data to actually prove this, we do have some key indicators.

We have, for example, a growing list of scientific celebrities who have committed major stem cell fraud. There is South Koreas Hwang Woo-suk who, in 2004, falsely claimed to have created the first human embryonic stem cells by means of cloning. A few years ago, Japans Haruko Obokata pulled a similar con when she announced to the world a new and simple and fake method of turning ordinary body cells into stem cells.

Hwang, Obokata and Macchiarini were all attracted to the hottest regions of stem cell research, where hope for a medical breakthrough was greatest. In Macchiarinis case, the hope was that patients could be treated with stem cells taken from their own bone marrow.

Over the years, this possibility has generated great excitement and a huge amount of research. Yet, for the vast majority of such treatments, there is little solid evidence that they work. (The big exception is blood stem cell transplantation, which has been saving the lives of people with leukemia and other cancers of the blood for decades.)

Its enough to worry officials from the US Food and Drug Administration (FDA). They recently published an article in the New England Journal of Medicine admitting that stem cell research has mostly failed to live up to its therapeutic promise.

An alarmingly wide gap has grown between what we expect from stem cells and what they deliver. Each new scientific discovery brings a flood of stories about how it will revolutionise medicine one day soon. But that day is always postponed.

An unhappy result of this is the rise of pseudo-scientific therapies. Stem cell clinics have sprung up like weeds, offering to treat just about any ailment you can name. In place of clinical data, there are gushing testimonials. There are also plenty of desperate patients who believe because theyve been told countless times that stem cells are the cure, and who cannot wait any longer for mainstream medicine. They and their loved ones fall victim to false hope.

Scientists can also suffer from false hope. To some extent, they believed Macchiarini because he told them what they wanted to hear. You can see this in the speed with which his breakthroughs were accepted. Only four months after Macchiarini operated on Claudia Castillo, his results provisional but very positive were published online by the Lancet. Thereafter it was all over the news.

The popular press also has a lot to answer for. Its love of human interest stories makes it sympathetic to unproven therapies. As studies have shown, the media often casts a positive light on stem cell tourism, suggesting that the treatments are effective and the risks low. It did much the same for Macchiarinis windpipe replacements. A good example is the NBC documentary A Leap of Faith. Its fascinating to rewatch as a lesson on how not to report on medical science.

It is fitting that Macchiarinis career unravelled at the Karolinska Institute. As the home of the Nobel prize in physiology or medicine, one of its ambitions is to create scientific celebrities. Every year, it gives science a show-business makeover, picking out from the mass of medical researchers those individuals deserving of superstardom. The idea is that scientific progress is driven by the genius of a few.

Its a problematic idea with unfortunate side effects. A genius is a revolutionary by definition, a risk-taker and a law-breaker. Wasnt something of this idea behind the special treatment Karolinska gave Macchiarini? Surely, he got away with so much because he was considered an exception to the rules with more than a whiff of the Nobel about him. At any rate, some of his most powerful friends were themselves Nobel judges until, with his fall from grace, they fell too.

If there is a moral to this tale, its that we need to be wary of medical messiahs with their promises of salvation.

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Dr Con Man: the rise and fall of a celebrity scientist who fooled almost everyone - The Guardian

Bone Marrow Stem Cells Comments Off on Dr Con Man: the rise and fall of a celebrity scientist who fooled almost everyone – The Guardian | September 1st, 2017

Wed,17 Dec 2003 05:30:00

Bone marrow transplant is a procedure in which healthy bone marrow is transplanted into a patient whose bone marrow is not functioning properly. Problems in bone marrow are often caused by chemotherapy or radiation treatment for cancer. This procedure can also be done to correct hereditary blood diseases. The healthy bone marrow may be taken from the patient prior to chemotherapy or radiation treatment (autograft), or it may be taken from a donor (allograft).

Wed,17 Dec 2003 05:30:00

Bone marrow is the soft, sponge-like material found inside bones. It contains immature cells called stem cells that produce blood cells. There are three types of blood cells: white blood cells, which fight infection; red blood cells, which carry oxygen to and from organs and tissues; and platelets, which enable the blood to clot.

Wed,17 Dec 2003 05:30:00

Alternatively, hereditary or acquired disorders may cause abnormal blood cell production. In these cases, transplantation of healthy bone marrow may save a patient's life. Transplanted bone marrow will restore production of white blood cells, red blood cells, and platelets.

Wed,17 Dec 2003 05:30:00

Donated bone marrow must match the patient's tissue type. It can be taken from the patient, a living relative (usually a brother or a sister), or from an unrelated donor. Donors are matched through special blood tests called HLA tissue typing.

Bone marrow is taken from the donor in the operating room while one is unconscious and pain-free (under general anaesthesia). Some of the donor's bone marrow is removed from the top of the hip bone. The bone marrow is filtered, treated, and transplanted immediately or frozen and stored for later use. Then, transplant material is transfused into the patient through a vein and is naturally transported back into the bone cavities where it grows to replace the old bone marrow.

Alternatively, blood cell precursors, called stem cells, can be induced to move from the bone marrow to the blood stream using special medications. These stem cells can then be taken from the bloodstream through a procedure called leukapheresis.

The patient is prepared for transplantation by administering high doses of chemotherapy or radiation (conditioning). This serves two purposes. First, it destroys the patient's abnormal blood cells or cancer. Second, it inhibits the patient's immune response against the donor bone marrow (graft rejection).

Following conditioning, the patient is ready for bone marrow infusion. After infusion, it takes 10 to 20 days for the bone marrow to establish itself. During this time, the patient requires support with blood cell transfusions.

Wed,17 Dec 2003 05:30:00

Wed,17 Dec 2003 05:30:00

The major problem with bone marrow transplants (when the marrow comes from a donor, not the patient) is graft-versus-host disease. The transplanted healthy bone marrow cells may attack the patient's cells as though they were foreign organisms. In this case, drugs to suppress the immune system must be taken, but this also decreases the body's ability to fight infections.

Other significant problems with a bone marrow transplant are those of all major organ transplants - finding a donor and the cost. The donor is usually a sibling with compatible tissue. The more siblings the patient has, the more chances there are of finding a compatible donor.

Wed,17 Dec 2003 05:30:00

The patient will require attentive follow-up care for 2 to 3 months after discharge from the hospital. It may take 6 months to a year for the immune system to fully recover from this procedure.

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Bone marrow transplant - Doctor NDTV

Bone Marrow Stem Cells Comments Off on Bone marrow transplant – Doctor NDTV | September 1st, 2017

From left to right: Richard Stone, a doctor at Dana-Farber Cancer Institute in Boston, poses with Peter Karalekas (center), 76, and Matthew Churitch, 22. Churitch donated stem cells to Karalekas two years ago, and he visited Dana-Farber with Karalekas earlier this summer. (Courtesy photo)

BOSTON -- After winding his way through Massachusetts, Connecticut, New Hampshire and Maine for 76 years, Peter Karalekas has a proclamation: He's a Southerner now.

He still lives in Kittery, Maine, just about an hour from the Lowell middle school where he taught for 21 years.

He has no plans to move.

Rather, Karalekas considers himself a Southerner because of his stem cells.

He never exactly felt all that sick.

Karalekas worked tirelessly for decades, first as a teacher and coach at the James S. Daley Middle School in Lowell and then as the owner of a half-dozen T-Bones restaurants across New Hampshire.

Even despite the 12-hour days, seven days a week, in the grind of the restaurant industry, Karalekas felt healthy and rarely fell ill.

Peter Karalekas, left, a 76-year-old former Lowellian, smiles during his first meeting with Matthew Churitch, 22, of Nashville, Tennessee, who helped save Karalekas life by donating stem cells. (Courtesy photo)

The two, who do not have children, moved to Kittery 17 years ago.

Everything started to change in 2014.

Karalekas recalls being "short-winded," but he had very few other symptoms when he was diagnosed with myelodysplastic syndrome, a rare type of cancer in which the bone marrow is damaged and cannot produce enough blood cells.

The prognosis was not good.

"They said the only thing that would save me was a stem cell transplant," Karalekas said. "Otherwise, I had a couple of months to live, because my cells were all dropping drastically.

He went onto a registry, hoping for a donor to pop up, but doctors told him it could take from six months to two years to find the right match. Even with a transplant, Karalekas said, his chances of success were "30 to 40 percent."

The call came four weeks later.

Matthew Churitch got his call quickly, too.

He joined the National Marrow Donor Program's Be the Match Registry in 2014, the summer between his freshman and sophomore years at Clemson University. His mother had been on the registry to donate for years. Churitch's decision was simple: When a friend was diagnosed with leukemia, he knew he should sign up, too.

He did the requisite cheek swab, unsure if he would ever even be contacted to donate. By the time he had finished the following semester, he got the call.

A match was found.

Churitch went through several more levels of testing and preparation to donate stem cells to a stranger. He went to Clemson's student health center to have blood drawn.

He returned to his native Nashville, Tennessee, going to a medical center 10 days in a row to receive shots in his stomach that would stimulate his bone marrow and prepare his cells for transplant.

He sat for eight hours, a needle in each arm as his stem cells were filtered out so they could be transferred to Boston.

"Getting the shots isn't fun," he said. "You're pretty sore afterward for a few weeks. But knowing that the person on the other end is in hundreds and hundreds times more pain than any donor would ever go through -- that kind of pushed me through."

Karalekas and Churitch first connected via an anonymous letter, per the transplant registry's rules, updating Churitch on Karalekas's lengthy, isolated recovery. They were able to speak directly after a year.

Churitch dialed Karalekas' number on a lengthy walk to class, took a deep breath and hit the call button. Moments later, both men were crying and laughing.

"That was really awesome, just being able to hear his voice and recognize that there's somebody else on the other end of this," Churitch said. "A lot of people don't get the chance to connect with their recipients or their donors."

Karalekas wanted more. He told his wife early on that he wanted to meet his "angel from heaven," so when Churitch graduated Clemson earlier this year, Karalekas paid to bring the 22-year-old and his mother to New England.

In late June, Karalekas and his wife pulled into a pickup lane at Logan International Airport in Boston.

"I got out of the car, I charged over, and I gave them both a huge hug," Karalekas said.

Karalekas showed Churitch and his mother around for five days.They went on a private tour of Fenway Park; they wandered the historic streets of Portsmouth, New Hampshire; they visited Dana-Farber together to meet the team that treated Karalekas.

Both families quickly bonded. Karalekas recalls his brother George asking Churitch about his portable phone charger, expressing curiosity about how convenient it was. A few weeks later, a brand-new portable charger arrived at George's door, a gift from Churitch.

In January, Karalekas and his wife will vacation in Arizona and will cheer on Churitch's mother -- without Churitch even present -- in the Phoenix Marathon.

Donor and recipient talk every week.

"It's like we're a very, very close-knit family now," Karalekas said. "He's the son we never had."

Churitch is now in his first year at the University of South Carolina School of Medicine Greenville with hopes of becoming a physician. He hopes to use Karalekas's experience as inspiration for any patients facing future hardship, and he hopes that others, especially young people, will see their success and join the registry.

"You never know where that will take you," he said. "You can gain a friend for life, impact somebody and their family in need."

Karalekas said he feels he has a new life: His chances of beating the disease are now 97 percent, he says, up from the 30 percent or 40 percent when he started treatment. Thanks to the transplant from a handsome, athletic college student in Tennessee.

"I said, 'I'm a Southerner now,'" Karalekas said. "My stem cells are 99 percent this gentleman. I'm 99 percent him."

Follow Chris on Twitter @ChrisLisinski.

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For Lowell native, stem cell match becomes a match as friends - Lowell Sun

Bone Marrow Stem Cells Comments Off on For Lowell native, stem cell match becomes a match as friends – Lowell Sun | August 31st, 2017

Tippi MacKenzie, MD, a pediatric and fetal surgeon at UCSF Benioff Childrens Hospital San Francisco, is the principal investigator for a clinical trial that will use in utero stem cell transplants to treat fetuses with an inherited disorder that restricts the bloods ability to carry oxygen to vital organs. Credit: Cindy Chew

UCSF Benioff Children's Hospitals in San Francisco and Oakland will pioneer stem cell transplants for a uniquely challenging patient population: second-trimester fetuses stricken with a potentially fatal disease.

The two hospitals are enrolling 10 pregnant women in the first phase of a clinical trial to treat fetuses with an inherited disorder that restricts the blood's ability to carry oxygen to vital organs. The trial, the first of its kind in the world, is funded by a $12.1 million grant from the California Institute for Regenerative Medicine.

Alpha thalassemia (ATM) affects 5 percent of the world's population, but is significantly more prevalent in China, Southeast Asia, India and the Middle East parts of the globe where many residents of the San Francisco Bay Area claim their origins. In its most extreme form, alpha thalassemia major (ATM), the condition leads to progressive anemia and heart failure before birth. Standard treatment in the United States includes lifelong blood transfusions.

Stem cell transplants from a matched donor in childhood have proven to be curative in some cases, but patients face risks, including graft-versus-host disease and serious side effects from immune-suppression drugs.

The trial is based on the premise that risks could be minimized by harnessing the "tolerance" between the pregnant woman and fetus before birth, said principal investigator Tippi MacKenzie, MD, a pediatric and fetal surgeon at UCSF Benioff Children's Hospital San Francisco.

Hope That Procedure Could Be Adopted Worldwide

"In performing the procedure in utero when the fetus's immune system is underdeveloped, we can avoid the aggressive treatments required for postnatal transplants for children with alpha thalassemia," MacKenzie said. "Eventually, the procedure may become a treatment option in parts of the world where ATM is most common. Due to lack of treatment possibilities in many countries, most pregnancies are either terminated on diagnosis or result in fetal demise," she said.

The trial follows a decades-long odyssey marked by triumphs and tribulations for researchers in the field. Fetal transplants using stem cells from other fetuses to treat blood disorders were carried out in the 1980s, but were only marginally successful due to engraftment failure. Researchers around the world searched for answers by turning to animal studies.

'Eureka Moment' Spurred Sea Change

"The fetus, unlike a fully developed human, can accept foreign cells, because its immune system is not yet primed to fight bacteria and viruses," said MacKenzie. "This undeveloped immune system benefits the fetus throughout the pregnancy, because it prevents it from launching an immune response to its mother's cells that are naturally circulating in its bloodstream."

Further research led to Mackenzie's "eureka moment," when it was discovered that the mother's immune system is actually responsible for rejecting other cells that are transplanted into the fetus. If the mother's cells are transplanted, they can engraft without being rejected. "This led to a sea change in our strategy to use maternal cells for the transplants," she said.

In the trial, bone marrow will be collected from women who are between 18 and 25 weeks pregnant, with a fetal diagnosis of ATM. The bone marrow cells will be processed and hematopoietic cells immature stem cells that can evolve into all types of blood cells will be singled out from the mix. They will then be injected through the woman's abdomen, into the umbilical vein of the fetus, where they can circulate through the bloodstream, developing into healthy mature blood cells.

The procedure is not without risks to the fetus and the pregnant woman. To minimize risks, the researchers restricted the trial to ATM, since the fetus is already undergoing blood transfusions. "An additional procedure for the transplantation is not necessary, since the maternal stem cells are infused at the same time as an in utero blood transfusion," said Elliott Vichinsky, MD, director of hematology/oncology at UCSF Benioff Children's Hospital Oakland, who will head the hematologic management of the fetus and newborn. "This should reduce additional risks to the fetus." Since the underlying disease causes complications, the woman will be monitored throughout her pregnancy and the fetus will continue to receive blood transfusions until birth.

UCSF is a pioneer in thalassemia research and the birthplace of fetal surgery. UCSF Benioff Children's Hospital Oakland is home to the Northern California Comprehensive Thalassemia Center, which was established in 1991 and is now the largest such program nationwide, with a focus on caring for patients and leading research into new treatments.

"We are excited about launching this trial, which combines the expertise of UCSF Benioff Children's Hospitals in San Francisco and Oakland. This study offers families with a usually fatal ATM pregnancy the chance of survival and cure," said Vichinsky, who founded the Northern California Comprehensive Thalassemia Center.

Treatment May Be Tested for Sickle Cell Anemia

Patient recruitment will continue for five years, during which pregnant women and their babies will be followed after birth for 30 days and one year respectively. If successful, the procedure will be carried out for fetuses with beta thalassemia, a more common and less serious variant of the disorder, as well as sickle cell anemia, in collaboration with Children's Hospital of Philadelphia. Other conditions requiring stem cell transplants after birth may be considered, said MacKenzie.

The incidence of ATM is unknown because most fetuses with the disorder die before delivery. The condition occurs when both parents are carriers for thalassemia. In places where women have access to prenatal care, ATM is usually suspected on ultrasound and confirmed by DNA analysis in the second trimester.

Explore further: Immune system drives pregnancy complications after fetal surgery in mice

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In utero stem cell transplants may replace riskier childhood transplants for multiple conditions - Medical Xpress

Bone Marrow Stem Cells Comments Off on In utero stem cell transplants may replace riskier childhood transplants for multiple conditions – Medical Xpress | August 31st, 2017