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6-year-old thalassemia patient from Punjab meets his stem cell … – Times of India

By daniellenierenberg

BENGLURU: Fateh Singh, a six-year-old thalassemia major patient from Amritsar, underwent a bone marrow transplant last May which gave him a new lease of life. A year later, the boy met his saviour, Naval Chaudhary, whose stem cells were used for the procedure. The child was diagnosed with the condition when he was one-and-a-half years old.

On Thursday, the donor and recipient met for the first time. Naval, 28, a professional living in Bengaluru, had registered with DATRI, an unrelated blood stem cell donors registry in 2015. He said: "I was very happy to hear I was a potential match for a patient. But then I was told the donation process had to be done through bone marrow harvesting. Initially, I was a tad hesitant but then I researched the procedure and was counselled by Dr Sunil Bhat, paediatric haemato-oncologist from Mazumdar Shaw Cancer Centre."

"I realized that saving a life is more important than the type of procedure I had to go through. So I decided to go ahead," he added.

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Athlone mother’s desperate search for bone marrow donor for son (3) – Eyewitness News

By LizaAVILA

Athlone mother's desperate search for bone marrow donor for son (3)

Raqeeb Palm was diagnosed with Aplastic Anaemia in October after his mother noticed unusual bruises on his body.

Three-year-old Raqeeb Palm was diagnosed with Aplastic Anaemia in October after his mother noticed unusual bruises on his body. Picture: Monique Mortlock/EWN.

CAPE TOWN A mother from Heideveld in Athlone is desperately trying to find a bone marrow donor for her three-year-old son.

Raqeeb Palm was diagnosed with Aplastic Anaemia in October after his mother noticed unusual bruises on his body.

The boy had to undergo various blood tests and two bone marrow biopsies over a two-month period, before being diagnosed with the rare disease which damages bone marrow and stem cells.

Zaida Palm says her outgoing child can no longer play outside or do many of the activities three-year-olds enjoy due to his severely weakened immune system.

Hes got practically no immune system. So going out, malls, play areas, doing fun things is on a stop. Because any germ, he gets admitted [to the hospital] for a cold, he needs to go to the hospital.

Palm says they have been unable to find a bone marrow donor in South Africa.

A transplant is her son's only chance of survival.

Her medical aid won't cover an investigation for international donors, which is why she's turned to online crowd-funding.

The hundred thousand on the Backabuddy [website] is just the start to the campaign.

Palm has also urged people to become bone marrow donors.

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renovacareinc.com – The Christian Institute

By Sykes24Tracey

The new technique heals burns much faster and more effectively than traditional skin grafting.

Burn victims may no longer be forced to undergo painful skin grafts, thanks to a revolutionary piece of technology that uses adult stem cells.

Instead of taking skin from one part of the body and transplanting it onto the burned area, a stem-cell spraying device simply covers the affected area with the victims own stem cells.

By taking adult stem cells from a healthy section of skin, placing them in a solution, and spraying the solution onto the wound, the patients own skin grows back and heals naturally.

The procedure has been in development for some time, and is not yet commercially available, but its capability was publicised in the press earlier this month.

The technology was featured in the Journal of the International Society for Burn Injuries, and showed incredible before and after images of the horrific injuries, and the victims almost full recoveries.

Patients who have benefitted from early treatments say their new skin is virtually indistinguishable from the rest of their body.

Commenting on the journals research, Thomas Bold, CEO of RenovaCare a company developing this technology said, the skin that regrows looks, feels and functions like the original skin.

By using adult stem cells, the healing process of the victims was also vastly accelerated.

While a skin graft treatment can take weeks or even months, and leave scarring, these patients were able to grow healthy skin in as little as four days.

In one case, a man who had suffered electrical burns to over a third of his body after touching a live wire had 24 million adult stem cells harvested and then sprayed back onto his body.

The process itself lasted only 90 minutes, and within four days, he had regrown a thin layer of skin over his arms and chest, where the burns were least severe.

After 20 days, all of the areas treated by the stem cell grafting process were described as completely healed.

RenovaCare is applying for a licence to use the technology in routine practice in Europe.

In January, it was revealed that a new technique allowed adult stem cells to be used in the treatment of heart problems.

The technique involves implanting synthetic cardiac stem cells which repair heart muscle. It has been praised as both an ethical and less risky alternative to other treatments.

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From hopeless to a miracle: How he got his life back after a crash left him paralyzed – fox6now.com

By Dr. Matthew Watson


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From hopeless to a miracle: How he got his life back after a crash left him paralyzed
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"We came to know he would be a good candidate for this regenerative treatment that we offer, meaning the stem cell injection into the spinal cord. ... "He was only the second to receive the stem cells -- at least that dose he received," added Dr. Kurpad.

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Regenerating medical research payouts? – OCRegister

By daniellenierenberg

In 2004 California ballot measure Proposition 71 was passed, granting $3 billion ($6 billion including interest) in state funds to support politically controversial embryonic stem cell research in California at a time when the federal government was restricting this research. A public agency was established, the California Institute for Regenerative Medicine, to dole out this money across California universities, medical research institutions and biotech companies. During the election campaign, California voters were assured of breakthroughs and cures for conditions like Parkinsons and spinal cord paralysis through celebrity endorsements featuring actors, Nobel prize winners and other notables. Prop. 71 money is dwindling and there is talk about putting a $5 billion renewal initiative on the ballot. So its reasonable to ask what California taxpayers got out of this deal over the past 13 years. Sadly, CIRM hasnt generated a single approved medical treatment. Through September 2016, CIRM has funded only three stem cell research projects that have reached Phase 3 clinical trials (the final step before FDA marketing approval). One of these trials was terminated and the other two are still recruiting patients and are not expected to report out for several years. During the same time, despite embryonic stem cell research restrictions, the federal National Institutes of Health has funded 50 stem cell research projects in Phase 3 trials. The NIH cost per Phase 3 research trial has been five times lower than the state program. Nearly half of the state funding has gone to research infrastructure rather than to actual research.

There also appears to have been blatant conflicts of interest in CIRM research awards. Around 80 percent of CIRM grants have gone to institutions represented on its board of directors. One out of seven CIRM research dollars has gone to Stanford University. One awardee, StemCells Inc., was co-founded by Irving Weissman, Stanfords stem cell program director. StemCells received at least $40 million from CIRM before going belly up. The CIRM board initially turned down a $20 million funding proposal to StemCells, until Bob Klein, the Northern California real estate investor who drafted Prop. 71 and was the first chairman of CIRMs governing board, was reported to have pressured the board to reverse that decision. CIRMs President Alan Trounson abruptly resigned in October 2013, joined the board of StemCells one week later, and then received $435,000 in cash and stocks from them before the company folded last year.

Does it make sense for California taxpayers to fund biotechnology research? Perhaps. A good case can be made that public investments in basic biotechnology infrastructure can have enormous benefits to Californias economy and job growth while generating significant improvements in human health. But public funding should have broader scope and flexibility to go after all promising new technological advances, not just current scientific fads or political controversies. Public funds should be awarded with rigorous oversight and accountability. There should be a sharp line between basic research, which requires public funding and is unlikely to yield short-term tangible cures, despite what celebrity actors say, and getting new medicines to market. Promising new treatments are already well-funded through private venture capital funds and biotech companies, who are much better at picking winners and losers than California taxpayers.

By not providing adequate oversight over potential conflicts of interest and not holding CIRM funding recipients to the same rigorous standards as NIH grant recipients, CIRMs 13 year record of zero new medicines for $6 billion in taxpayer funds is not an experiment that the voters should regenerate at the ballot box.

Joel W. Hay is a professor of Health Economics and Policy at the University of Southern California.

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Cancer-cardiac connection illuminates promising new drug for heart … – Medical Xpress

By NEVAGiles23

May 17, 2017 Images of heart muscle cells derived from induced pluripotent stem cells. Credit: Q. Duan et al., Science Translational Medicine (2017)

A team of researchers at the Gladstone Institutes uncovered a new strategy to treat heart failure, a leading contributor to mortality and healthcare costs in the United States. Despite widespread use of currently-approved drugs, approximately 40% of patients with heart failure die within 5 years of their initial diagnosis.

"The current standard of care is clearly not sufficient, which highlights the urgent need for new therapeutic approaches," said Saptarsi Haldar, MD, an associate investigator at Gladstone and senior author of a new study featured on the cover of the scientific journal Science Translational Medicine. "In our previous work, we found that a drug-like small molecule called JQ1 can prevent the development of heart failure in mouse models when administered at the very onset of the disease. However, as the majority of patients requiring treatment already have longstanding cardiac dysfunction, we needed to determine if our strategy could also treat established heart failure."

As part of an emerging treatment strategy, drugs derived from JQ1 are currently under study in early-phase human cancer trials. These drugs act by inhibiting a protein called BRD4, a member of a family of proteins called BET bromodomains, which directly influences heart failure. With this study, the scientists found that JQ1 can effectively treat severe, pre-established heart failure in both small animal and human cell models by blocking inflammation and fibrosis (scarring of the heart tissue).

"It has long been known that inflammation and fibrosis are key conspirators in the development of heart failure, but targeting these processes with drugs has remained a significant challenge," added Haldar, who is also a practicing cardiologist and an associate professor in the Department of Medicine at the University of California, San Francisco. "By inhibiting the function of the protein BRD4, an approach that simultaneously blocks both of these processes, we are using a new and different strategy altogether to tackle the problem."

Currently available drugs used for heart failure work at the surface of heart cells. In contrast, Haldar's approach goes to the root of the problem and blocks destructive processes in the cell's command center, or nucleus.

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"We treated mouse models of heart failure with JQ1, similarly to how patients would be treated in a clinic," said Qiming Duan, MD, PhD, postdoctoral scholar in Haldar's lab and co-first author of the study. "We showed that this approach effectively treats pre-established heart failure that occurs both after a massive heart attack or in response to persistent high blood pressure (mechanical overload), suggesting it could be used to treat a wide array of patients."

Using Gladstone's unique expertise, the scientists then used induced pluripotent stem cells (iPSCs), generated from adult human skin cells, to create a type of beating heart cell known as cardiomyocytes.

"After testing the drug in mice, we wanted to check whether JQ1 would have the same effect in humans," explained co-first author Sarah McMahon, a UCSF graduate student in Haldar's lab. "We tested the drug on human cardiomyocytes, as they are cells that not only beat, but can also trigger the processes of inflammation and fibrosis, which in turn make heart failure progressively worse. Similar to our animal studies, we found that JQ1 was also effective in human heart cells, reaffirming the clinical relevance of our results."

The study also showed that, in contrast to several cancer drugs that have been documented to cause cardiac toxicity, BRD4 inhibitors may be a class of anti-cancer therapeutics that has protective effects in the human heart.

"Our study demonstrates a new therapeutic approach to successfully target inflammation and fibrosis, representing a major advance in the field," concluded Haldar. "We also believe our current work has important near-term translational impact in human heart failure. Given that drugs derived from JQ1 are already being tested in cancer clinical trials, their safety and efficacy in humans are already being defined. This key information could accelerate the development of a new heart failure drug and make it available to patients more quickly."

Explore further: Heart failure is as 'malignant' as some common cancers

More information: Q. Duan el al., "BET bromodomain inhibition suppresses innate inflammatory and profibrotic transcriptional networks in heart failure," Science Translational Medicine (2017). stm.sciencemag.org/lookup/doi/10.1126/scitranslmed.aah5084

A new analysis finds that, despite advances in care, men and women with a diagnosis of heart failure continue to have worse survival rates than patients with certain common cancers.

Patching a damaged heart with a patient's own muscle stem cells improves symptoms of heart failure, according to a Phase I clinical trial reported in Journal of the American Heart Association, the Open Access Journal of the ...

Researchers have completed a randomized clinical trial in patients with heart failure with preserved ejection fraction (HFpEF), which currently has no effective treatment for reducing morbidity and mortality.

A new analysis describes different classifications of patients who are hospitalized with acute heart failure based on various characteristics, which may help guide early decisions regarding triage and treatment.

(HealthDay)Patients with rheumatoid arthritis (RA) have increased risk of heart failure, according to a study published in the March 14 issue of the Journal of the American College of Cardiology.

In the largest German survey on heart failure to date, investigators found that the overall awareness of heart failure has not increased over the past decade and is not at a satisfactory level.

Shortness of breath is the No.1 complaint of people suffering from heart failure. Now a University of Guelph researcher has discovered its surprising cause - and an effective treatment - in a groundbreaking new study.

A team of researchers at the Gladstone Institutes uncovered a new strategy to treat heart failure, a leading contributor to mortality and healthcare costs in the United States. Despite widespread use of currently-approved ...

Although the absolute difference in U.S. county-level cardiovascular disease mortality rates have declined substantially over the past 35 years for both ischemic heart disease and cerebrovascular disease, large differences ...

Waist-to-hip ratio may be a stronger indicator of some cardiovascular illnesses than the commonly-used measure BMI, according to a new UCL-led study.

New research has found that genetic differences in antibody genes alter individuals' susceptibility to rheumatic heart disease, a forgotten inflammatory heart condition known as 'RHD' that is rife in developing countries.

People who use commonly prescribed non-steroidal anti-inflammatory drugs (NSAIDs) to treat pain and inflammation could be raising their risk of having a heart attack, as early as in the first week of use and especially within ...

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Creative Medical Technology Holdings to Expand into 10 Billion Dollar per Year Lower Back Pain Market with … – PR Newswire (press release)

By NEVAGiles23

"Creative Medical Technology Holdings will develop this patent through the same process that we are using for our clinical-stage Caverstem procedure for erectile dysfunction," stated Timothy Warbington, President and Chief Executive Officer of the Company. "We plan to identify and engage key opinion leaders who will lead clinical trials, which will serve as the basis for accelerated commercialization."

The Company is currently running a clinical trial using autologous non-manipulated bone marrow stem cells for patients suffering from erectile dysfunction that are non-responsive to standard approaches such as Viagra.Once the trial is completed, the results will serve as the basis for marketing of disposables utilized in administration of stem cells.

"Although numerous companies are injecting stem cells directly into the disc, direct injection may only cause temporary benefit because the root cause of the pathology, in our opinion, is the reduced blood supply," stated Dr. Amit Patel, Director of Thoracic Surgery at University of Miami and co-founder of Creative Medical Technology Holdings. "By recreating in the microenvironment of the lower back the same thing that we do in atherosclerotic heart patients, we believe we have a novel way to treat this terrible condition that wreaks havoc on our health care system."

Several studies have shown that administration of stem cells possesses a therapeutic effect in cardiac conditions associated with poor circulation by stimulation of new blood vessel production, a process termed "angiogenesis".The current patent covers stimulation of angiogenesis in the lower back using mesenchymal stem cells.These cells can be used from the same patient, which is considered an "autologous therapy" as well as using stem cells in a universal donor manner, which is termed "allogeneic".

"The acquisition of this patent not only positions the company to expand into the disc degenerative space, but also provides a powerful platform for collaboration with other companies that are administering regenerative cells directly into the nucleus pulposus of the disc," commented Thomas Ichim, Ph.D., Chief Scientific Officer of the Company and inventor of the technology. "Stem cells are like seeds, they need to be planted into fertile soil. We feel that in certain patients it is essential to treat the lumbar ischemia, which is present in some patients suffering from disc degenerative disease, which will then allow the stem cells administered directly in the disc to perform their regenerative effects."

About US

Creative Medical Technology Holdings, Inc. is a clinical-stage biotechnology company with two focus areas; 1) personalized stem cell procedures for sexual dysfunction and infertility, and 2) universal, off-the-shelf amniotic fluid-based stem cells that possess superior healing potential without negative medical or ethical issues. Through our own research and collaborations with leading academic institutions, we have developed proprietary protocols, built an extensive intellectual property portfolio, developed complete treatment offerings for erectile dysfunction and are performing ground-breaking research with our amniotic fluid-based stem cell.

For additional information visit http://www.CREATIVEMEDICALTECHNOLOGY.com

Forward-Looking StatementsThis release may contain "forward-looking statements." Forward-looking statements are identified by certain words or phrases such as "may", "aim", "will likely result", "believe", "expect", "anticipate", "estimate", "intend", "plan", "contemplate", "seek to", "future", "objective", "goal", "project", "should", "will pursue" and similar expressions or variations of such expressions. These forward-looking statements reflect the Company's current expectations about its future plans and performance. These forward-looking statements rely on a number of assumptions and estimates which could be inaccurate and which are subject to risks and uncertainties. Actual results could vary materially from those anticipated or expressed in any forward-looking statement made by the Company. Please refer to the Company's most recent Forms 10-Q and 10-K and subsequent filings with the SEC for a further discussion of these risks and uncertainties. The Company disclaims any obligation or intent to update the forward-looking statements in order to reflect events or circumstances after the date of this release.

To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/creative-medical-technology-holdings-to-expand-into-10-billion-dollar-per-year-lower-back-pain-market-with-acquisition-of-issued-us-stem-cell-patent-300459902.html

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Canadian Doctors Like Cameron Clokie Are The Innovators Behind The New Era of Regenerative Medicine – French Tribune

By LizaAVILA

Heavy increases in obesity have led to an epidemic of various heart diseases, including cardiac arrests and even strokes. These dangers have compelled doctors and research specialists to seek out new ways of managing these problems. One method that has been getting a lot of attention is regenerative medicine.

This treatment method, while occasionally controversial, shows an incredible potential that could solve many serious health problems. Specialists like Dr. Cameron Clokie, a health expert with decades of experience, are currently trying to find ways to make this treatment method more accepted by those who oppose it.

The Potential for Serious Health Benefits is Huge

Regenerative medicine is the use of stem cells and other regeneration items to promote more efficient healing. Dr. Cameron Clokie has preached about the effectiveness of this treatment method for years. And it seems like the rest of the world is finally catching up with him and others like him. For example, a recent study found that stem cells could help manage cardiac and nervous system diseases.

The careful use of stem cells could regenerate damaged heart tissues and help a person avoid heart attacks and other serious problems. Even more promising, stem cells could be used to help repair nerve damage that would otherwise leave a person paralyzed for life.

Stem Cell Research Could Save Lives

Think of the stem cells in your body as building blocks that will take whatever shape is necessary. They can become heart cells and patch a hole in this vital organ. However, they could also become spinal cells and repair severe damage to this crucial part of the body.

The possibilities associated with stem cells could be potentially limitless. As they can be manipulated to take the form of any cell, they could be used to treat a variety of serious health problems. For example, they could become white blood cells and fight serious viral problems. In fact, they could even be used to treat life-threatening diseases like AIDS.

One of the understated benefits of regenerative medicine is the way that it uses actual cells from your body. Think of the problems the medical world has had with artificial hearts. While they can be beneficial to many people, they are often rejected by the fickle body as an intruder. However, creating a working heart with your body's stem cells would eliminate that problem.

Why? Your body would recognize the heart's cells as coming from you and would accept it more readily. As a result, you could get a new (and real) heart to replace a severely damaged one.

Profit Levels Could Also Be High

One thing that has interested many people about regenerative health and stem cell research is the potential for huge profits. Many health experts have tried to stress the ways that regenerative health could help boost the world's economy. For example, a recent study on the financial state of this market found that it had an $18.9 billion global impact.

Even more shocking, it was projected to hit $53 billion by 2021. The major focus of this market would be in bone and joint reconstruction. The United States was expected to potentially make the largest profits in this area, which is something Dr. Cameron Clokie has emphasized in the past.

However, the European market is projected to be even bigger if the currently somewhat stagnant American regenerative market is held back by restrictive regulations or laws. In this way, well-meaning politicians could deny their constituents access to lifesaving treatments and severely impact the market at the same time.

Final Thoughts

Regenerative medicine of the type proposed by Dr. Cameron Clokie and others like him could transform the medical world. While the protests of people who find stem cells wrong are understandable, the major benefits of using them cannot be ignored.

This fact is why it is so important to help specialists like Dr. Cameron Clokie get the help they need to promote regenerative medicine breakthroughs. In this way, it is possible to solve serious health dangers.

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Human blood stem cells grown in the lab for the first time – New Scientist

By Sykes24Tracey

Potential for a new supply line

Burger/Phanie/REX/Shutterstock

By Jessica Hamzelou

The stem cells that produce our blood have been created in the lab for the first time. These could one day be used to treat people who have blood diseases and leukaemia with their own cells, rather than bone marrow transplants from a donor. They could also be used to create blood for transfusions.

This is a very big deal, says Carolina Guibentif at the University of Cambridge, who was not involved in the research. If you can develop [these cells] in the lab in a safe way and in high enough numbers, you wouldnt be dependent on donors.

In a healthy adult, blood stem cells are found in bone marrow, where they replenish the supply of red and white blood cells and platelets. They are sort of master cells, says George Daley at Harvard Medical School.

When these cells dont work properly, they fail to maintain an adequate supply of blood cells. As a result, not enough oxygen reaches the bodys tissues. This can cause serious disease if organs such as the heart are affected. Blood stem cells can also be wiped out by chemotherapy for leukaemia and other cancers.

People with these disorders tend to be treated with bone marrow complete with blood stem cells from a healthy donor. The difficulty is finding a match. There is a one in four chance of achieving this from a healthy sibling, but the odds are slashed to one in a million if a stranger needs to be found, says Daley.

In an attempt to create blood stem cells in the lab, Daley and his colleagues started with human pluripotent stem cells which have the potential to form almost any other type of body cell.

The team then searched for chemicals that might encourage these to become blood stem cells.

After studying the genes involved in blood production, the researchers identified proteins that control these genes and applied them to their stem cells.

They tested many combinations of the proteins, and found five that worked together to encourage their stem cells to become blood stem cells. When they put these into mice, they went on to produce new red and white blood cells and platelets. Its very cool, says Daley. Were very excited about the results.

A separate team has achieved the same feat with stem cells taken from adult mice. Raphael Lis at Weill Cornell Medical College in New York and his colleagues started with cells taken from the walls of the animals lungs, based on the idea that similar cells in an embryo eventually form the bodys first blood stem cells. The team identified a set of four factors that could encourage these lung stem cells to make them.

Both sets of results represent a breakthrough, says Guibentif. This is something people have been trying to achieve for a long time, she says. By working with adult mouse epithelial cells, Lis and his team show that the feat could potentially be achieved with cells taken from an adult person. Daleys team used human stem cells that could in theory be made from skin cells, bolstering the prospect that lab-made human blood could be next.

The lab-made stem cells are not quite ready to be used in people just yet, says Daley. Although all of his mice were healthy throughout the experiments, there is a risk that the cells could mutate and cause cancer. And the cells are not quite as efficient at making blood as those found in the body.

But once Daley and his team have honed their procedure, they might be able to make platelets and red blood cells for hospital use. These cell types dont have a nucleus, so are unable to divide and potentially cause cancer. He hopes this procedure could be used within the next couple of years.

Eventually, Daley hopes his cells could be used to create whole blood suitable for transfusions. Not only would such a supply be more reliable than that from donors, but it would also be free of disease. When new pathogens like Zika pop up, you have to make sure that blood is safe, says Daley. Wed be able to have more quality control.

Journal references: Nature, DOI: 10.1038/nature22326; Nature, DOI: 10.1038/nature22370

Read more: Synthetic bone implant can make blood cells in its marrow; Lab-grown blood given to volunteer for the first time

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Another reason to exercise: Burning bone fat a key to better bone health – Science Daily

By raymumme


Science Daily
Another reason to exercise: Burning bone fat a key to better bone health
Science Daily
It could be that when fat cells are burned during exercise, the marrow uses the released energy to make more bone. Or, because both fat and bone cells come from parent cells known as mesenchymal stem cells, it could be that exercise somehow stimulates ...

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Bone marrow transplant facility to be available to public, government employees – The News International

By NEVAGiles23

Islamabad

The Ministry of National Health Services signed a Memorandum of Understanding with the Armed Forces Bone Marrow Transplant Centre here Thursday for provision of bone marrow transplant facility to the general public and federal government employees and their families, along with Armed Forces personnel and their families and defence paid employees.

Under the MOU, the National Institute of Blood and Marrow Transplant shall be established at the Armed Forces Bone Marrow Transplant Centre and will be designated as the National Institute of Blood and Marrow Transplant (NIBMT). This new facility will broaden the scope of the hospital, so that bone marrow/stem cell transplant can be extended to federal government employees and the general public. It will also serve to extend training facilities in the field of Bone Marrow Transplant and Clinical Haematology.

The MOU was signed on behalf of National Health Services by Director General Health Dr. Assad Hafeez whereas Major General Tariq Mehmood Satti Commandant Armed Forces Bone Marrow Transplant Centre, Rawalpindi, signed on behalf of his organization. Commandant of the Armed Forces Institute of Pathology Maj. Gen. Parvez Ahmed was also present on the occasion.

Speaking on the occasion, the Secretary of the Ministry of Health Services Muhammad Ayub Shaikh expressed gratitude to the Commandant of AFIP and AFBMPC for their efforts in making the MOU possible. This noble initiative will benefit a large number of patients, he projected. Major General Parvez Ahmed elaborated the efforts and initiatives taken to make the MOU possible.

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Cancer-cardiac connection illuminates promising new drug for heart failure – Science Daily

By JoanneRUSSELL25

A team of researchers at the Gladstone Institutes uncovered a new strategy to treat heart failure, a leading contributor to mortality and healthcare costs in the United States. Despite widespread use of currently-approved drugs, approximately 40% of patients with heart failure die within 5 years of their initial diagnosis.

"The current standard of care is clearly not sufficient, which highlights the urgent need for new therapeutic approaches," said Saptarsi Haldar, MD, an associate investigator at Gladstone and senior author of a new study featured on the cover of the scientific journal Science Translational Medicine. "In our previous work, we found that a drug-like small molecule called JQ1 can prevent the development of heart failure in mouse models when administered at the very onset of the disease. However, as the majority of patients requiring treatment already have longstanding cardiac dysfunction, we needed to determine if our strategy could also treat established heart failure."

As part of an emerging treatment strategy, drugs derived from JQ1 are currently under study in early-phase human cancer trials. These drugs act by inhibiting a protein called BRD4, a member of a family of proteins called BET bromodomains, which directly influences heart failure. With this study, the scientists found that JQ1 can effectively treat severe, pre-established heart failure in both small animal and human cell models by blocking inflammation and fibrosis (scarring of the heart tissue).

"It has long been known that inflammation and fibrosis are key conspirators in the development of heart failure, but targeting these processes with drugs has remained a significant challenge," added Haldar, who is also a practicing cardiologist and an associate professor in the Department of Medicine at the University of California, San Francisco. "By inhibiting the function of the protein BRD4, an approach that simultaneously blocks both of these processes, we are using a new and different strategy altogether to tackle the problem."

Currently available drugs used for heart failure work at the surface of heart cells. In contrast, Haldar's approach goes to the root of the problem and blocks destructive processes in the cell's command center, or nucleus.

"We treated mouse models of heart failure with JQ1, similarly to how patients would be treated in a clinic," said Qiming Duan, MD, PhD, postdoctoral scholar in Haldar's lab and co-first author of the study. "We showed that this approach effectively treats pre-established heart failure that occurs both after a massive heart attack or in response to persistent high blood pressure (mechanical overload), suggesting it could be used to treat a wide array of patients."

Using Gladstone's unique expertise, the scientists then used induced pluripotent stem cells (iPSCs), generated from adult human skin cells, to create a type of beating heart cell known as cardiomyocytes.

"After testing the drug in mice, we wanted to check whether JQ1 would have the same effect in humans," explained co-first author Sarah McMahon, a UCSF graduate student in Haldar's lab. "We tested the drug on human cardiomyocytes, as they are cells that not only beat, but can also trigger the processes of inflammation and fibrosis, which in turn make heart failure progressively worse. Similar to our animal studies, we found that JQ1 was also effective in human heart cells, reaffirming the clinical relevance of our results."

The study also showed that, in contrast to several cancer drugs that have been documented to cause cardiac toxicity, BRD4 inhibitors may be a class of anti-cancer therapeutics that has protective effects in the human heart.

"Our study demonstrates a new therapeutic approach to successfully target inflammation and fibrosis, representing a major advance in the field," concluded Haldar. "We also believe our current work has important near-term translational impact in human heart failure. Given that drugs derived from JQ1 are already being tested in cancer clinical trials, their safety and efficacy in humans are already being defined. This key information could accelerate the development of a new heart failure drug and make it available to patients more quickly."

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Materials provided by Gladstone Institutes. Note: Content may be edited for style and length.

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Scientists get closer to making personalized blood cells by using patients’ own stem cells – Los Angeles Times

By LizaAVILA

New research has nudged scientists closer to one of regenerative medicines holy grails: the ability to create customized human stem cells capable of forming blood that would be safe for patients.

Advances reported Wednesday in the journal Nature could not only give scientists a window on what goes wrong in such blood cancers as leukemia, lymphoma and myeloma. They could also improve the treatment of those cancers, which affect some 1.2 million Americans.

The stem cells that give rise to our blood are a mysterious wellspring of life. In principle, just one of these primitive cells can create much of a human beings immune system, not to mention the complex slurry of cells that courses through a persons arteries, veins and organs.

While the use of blood-making stem cells in medicine has been common since the 1950s, it remains pretty crude. After patients with blood cancers have undergone powerful radiation and chemotherapy treatments to kill their cancer cells, they often need a bone-marrow transplant to rebuild their white blood cells, which are destroyed by that treatment.

The blood-making stem cells that reside in a donors bone marrow and in umbilical cord blood that is sometimes harvested after a babys birth are called hematopoietic, and they can be life-saving. But even these stem cells can bear the distinctive immune system signatures of the person from whom they were harvested. As a result, they can provoke an attack if the transplant recipients body registers the cells as foreign.

This response, called graft-versus-host disease, affects as many as 70% of bone-marrow transplant recipients in the months following the treatment, and 40% develop a chronic version of the affliction later. It can overwhelm the benefit of a stem cell transplant. And it kills many patients.

Rather than hunt for a donor whos a perfect match for a patient in need of a transplant a process that can be lengthy, ethically fraught and ultimately unsuccessful doctors would like to use a patients own cells to engineer the hematopoietic stem cells.

The patients mature cells would be reprogrammed to their most primitive form: stem cells capable of becoming virtually any kind of human cell. Then factors in their environment would coax them to become the specific type of stem cells capable of giving rise to blood.

Once reintroduced into the patient, the cells would take up residence without prompting rejection and set up a lifelong factory of healthy new blood cells.

If the risk of deadly rejection episodes could be eliminated, physicians might also feel more confident treating blood diseases that are painful and difficult but not immediately deadly diseases such as sickle cell disease and immunological disorders with stem cell transplants.

The two studies published Wednesday demonstrate that scientists may soon be capable of pulling off the sequence of operations necessary for such treatments to move ahead.

One of two research teams, led by stem-cell pioneer Dr. George Q. Daley of Harvard Medical School and the Dana Farber Cancer Institute in Boston, started their experiment with human pluripotent stem cells primitive cells capable of becoming virtually any type of mature cell in the body. Some of them were embryonic stem cells and others were induced pluripotent stem cells, or iPS cells, which are made by converting mature cells back to a flexible state.

The scientists then programmed those pluripotent stem cells to become endothelial cells, which line the inside of certain blood vessels. Past research had established that those cells are where blood-making stem cells are born.

Here, the process needed a nudge. Using suppositions gleaned from experiments with mice, Daley said his team confected a special sauce of proteins that sit on a cells DNA and program its function. When they incubated the endothelial cells in the sauce, they began producing hematopioetic stem cells in their earliest form.

Daleys team then transferred the resulting blood-making stem cells into the bone marrow of mice to see if they would take. In two out of five mice who got the most promising cell types, they did. Not only did the stem cells establish themselves, they continued to renew themselves while giving rise to a wide range of blood cells.

A second research team, led by researchers from Weill Cornell Medicines Ansary Stem Cell Institute in New York, achieved a similar result using stem cells from the blood-vessel lining of adult mice. After programming those cells to revert to a more primitive form, the scientists also incubated those stem cells in a concoction of specialized proteins.

When the team, led by Raphael Lis and Dr. Shahin Rafii, transferred the resulting stem cells back into the tissue lining the blood vessels of the mice from which they came, that graft also took. For at least 40 weeks after the incubated stem cells were returned to their mouse owners, the stem cells continued to regenerate themselves and give rise to many blood-cell types without provoking immune reactions.

In addition to making a workhorse treatment for blood cancers safer, the new advances may afford scientists a unique window on the mechanisms by which blood diseases take hold and progress, said Lee Greenberger, chief scientific officer for the Leukemia and Lymphoma Society.

From a research point of view you could now actually begin to model diseases, said Greenberger. If you were to take the cell thats defective and make it revert to a stem cell, you could effectively reproduce the disease and watch its progression from the earliest stages.

That, in turn, would make it easier to narrow the search for drugs that could disrupt that disease process early. And it would speed the process of discovering which genes are implicated in causing diseases. With gene-editing techniques such as CRISPR-Cas9, those offending genes could one day be snipped out of hematopoietic stem cells, then be returned to their owners to generate new lines of disease-free blood cells.

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Ontario teen Jonathan Pitre’s second attempt at stem cell transplant is a success – Cantech Letter

By NEVAGiles23

Jonathan Pitre in a 2015 TSN profile.

This time, it worked.

Suffering from a severe form of epidermolysis bullosa (EB), an incurable genetic condition which causes the skin to blister and create painful wounds, Pitre, who turns 17 next month, was given the moniker Butterfly Boy due to his delicate skin.

EB can be fatal, with many people who have severe EB dying from skin cancer in their twenties. Pitre underwent his second stem cell transplant procedure at the University of Minnesota Masonic Childrens Hospital, a pioneer in treating EB though stem cell transplants.

Paediatric hematoligist-oncologist with the University of Minnesota Jakub Tolar calls EB the worst disease youve never heard of, as it affects only one in 20,000 people. Research by Tolar and his colleagues led to the discovery that bone marrow transplantation, a procedure typically used to treat blood cancers in the bone marrow such as leukemia, could benefit those with EB.

This had never been done before, says Tolar, who directs the U of Ms Stem Cell Institute, in a press release. I didnt know it at the time we started this research 10 years ago, but it opened a totally new field in transplantation biology.

Stem cell transplants involve a persons blood-forming stem cells (immature cells that can become various types of specialized cells in the body, in this case, becoming different types of blood cells) from the bone marrow and replacing them with healthy stem cells.

For Pitre, his earlier bone marrow transplant last October proved unsuccessful as doctors learned that his own stem cells had recolonized his bone marrow. This time around, the results look more promising. Pitres mother, Tina Boileau, who was the donor, is now full of joy and relief, according to an Ottawa Citizen report, which states that newly created white blood cells in Pitres system contain a pair of X chromosomes, indicating that they came from Boileaus donated cells.

This is the best news ever, the best Mothers Day gift, said Boileau. Jon is full of me. He doesnt have any T-cells that are his.

Its been over 30 years since bone marrow cells were first used to treat cancer, but recent advances have shown the potential application of stem cell transplantation for a variety of diseases and conditions, from brain and spinal cord injury to neurodegenerative diseases like Alzheimers to HIV/AIDS. Researchers at Cardiff University in Wales, for example, have just announced commencement of stem cell transplants for patients with Huntingtons disease.

The Ontario government has just announced $32 million in new funding to help shorten the long wait times for stem cell transplants in the province, meaning that 150 more patients a year will be able to receive transplant therapy. As reported in the Hamilton Spectator, $10 million of the new funds will be going to the Juravinski Hospital and Cancer Centre in Hamilton for a dedicated unit with 15 inpatient and five outpatient beds.

Below: TSN Original: The Butterfly Child

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Blasting tiny bubbles at broken pig bones makes them heal on their own – The Verge

By raymumme

Scientists have healed severe bone fractures in pigs by blasting tiny bubbles with ultrasound in the animals bones. The technique encourages the pigs bodies to regenerate themselves, and could one day be used to help humans especially the elderly heal dangerous bone injuries.

Broken bones are common: you wrap an arm or wrist in a cast and the bone eventually heals on its own. But sometimes, people have nonunion fractures, meaning bones fail to produce new bone tissue and dont heal properly. There are about 100,000 cases of this in the United States every year. One solution is bone grafts, or bone transplants using donated marrow, but this procedure is invasive and there is a risk that the body will reject the marrow. Another solution is to use viruses to deliver bone morphogenetic proteins (BMPs) that encourage the bodys own stem cells to create more bone marrow. But using a virus can have negative side effects like inflammation.

In a study published today in Science Translational Medicine, scientists healed a 0.4-inch fracture in pigs in eight weeks without invasive surgery. Going from something invasive to something like this that potentially could be an outpatient procedure has been the holy grail in orthopedics, says Edward Schwarz, director of the University of Rochesters Center for Musculoskeletal Research, who was not involved with the study. He adds that, though these nonunion fractures arent the most common health problem, theyre a serious one. People are shocked when I tell them that the life expectancy with a nonunion fracture is shorter than with pancreatic cancer, he says. Were like horses. If we cant get up and walk again, then were done.

In the study, the researchers first caused a 0.4-inch fracture in the shins of 18 minipigs. Then, they inserted a biodegradable scaffolds into the broken shins, says co-author Gadi Pelled, a professor of surgery at Cedars-Sinai Medical Center. The scaffold helped support bone stem cells in the area. The scientists let the stem cells migrate and populate over the scaffold for two weeks but that wast enough. The stem cells had to be triggered to actually heal the injury. So the scientists injected microbubbles mixed with bone morphogenetic proteins. Immediately after the injection, they applied ultrasound, which stimulated the BMPs to enter into the stem cells and activate them.

The stem cells then turned into bone cells and healed the fracture after eight weeks. This method doesnt have the side effects associated with using viruses, and the fact that it uses the bodys own stem cells means theres no risk of rejection, says co-author Zulma Gazit, also at Cedars-Sinai. This ultrasound and microbubbles combo has already been approved by the Food and Drug Administration and is often used in radiology, so the new technique could be readily approved for use in humans.

Next, says Pelled, the team is studying whether the same technology can also work with tissues like ligaments; they gathering more comprehensive information. Before we move forward into humans, we need to determine that this technology is safe, says Pelled. Theyre hopeful that a clinical trial is on the way.

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Press Release: New Stem Cell Collection Center Opens in Boston – The Scientist

By Sykes24Tracey

Press Release: New Stem Cell Collection Center Opens in Boston
The Scientist
We support biomedical researchers globally by offering human hematopoietic stem cells and blood derived cell products from bone marrow, cord blood, peripheral blood and mobilized peripheral blood. StemExpress guarantees every sample delivers only ...

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NCAA-bound UIC softball pitcher driven to expand bone-marrow donor pool – Chicago Tribune

By daniellenierenberg

On the morning of a game at Youngstown State earlier this season, Illinois-Chicago softball pitcher Karissa Frazier arrived armed for a successful road trip.

Frazier packed enough kits to perform cheek swabs on Youngstown State players who had agreed to add their samples to a worldwide bone-marrow registry. So Frazier hopped on a bus to the ballpark by herself, gave a presentation on the #swab2save campaign in her role as UIC's campus ambassador for Gift of Life, and helped swab the young women she would try to strike out later in the series.

And what a pitch the All-Horizon League hurler made.

One Youngstown State player was inspired enough by Frazier to request 200 swab kits to begin her own drive. Another immediately reached out to Gift of Life the global not-for-profit marrow and blood stem cell donor registry facilitating transplants and became the Ohio campus's representative.

"After Karissa was done swabbing players that day, she came back to our hotel and got ready for the game like normal," UIC coach Lynn Curylo said. "How amazing is that?"

For the UIC softball team, the trip to Eugene, Ore., to play Oregon on Friday in the NCAA tournament, its first appearance in six years, offers an opportunity to provide evidence of progress at the end of Curylo's promising first season. For Frazier, a junior right-hander with a 13-8 record and a 1.53 earned-run average, the journey represents that and more, another chance to spread awareness of a cause as powerful as her fastball.

"This has pushed me in the right direction and opened my eyes to all the things I could do to change people's lives for the better," Frazier said. "I'm hoping to swab all three teams at our NCAA regional. And I'd love to go to the College World Series and swab all the teams there."

Seeing an emotional meeting between a donor and recipient left an indelible impression on Frazier. But a brush with a family friend back home in Temecula, Calif., first lit a fire within the public health major. A friend's decision to become a bone-marrow donor allowed a woman to live an additional six years and see the birth of her first grandchild and the wedding of her daughter.

"I just knew this was something I'd really enjoy doing so one day I could help save someone's life,'' Frazier said.

Back at UIC last August, Frazier interviewed with Gift of Life, which sought college ambassadors to increase potential donors in the 18- to 25-year-old demographic. Frazier's bosses established two goals for her: Swab 500 people overall and 250 males research shows males are three times less likely to sign up than women but twice as likely to be a match. When Frazier left Wednesday for Oregon, she had accumulated more than 700 total swab samples, including nearly 300 from males.

"I used my softball player status to expand getting a broader range of people," Frazier said.

Last fall, Frazier set up a table next to the UIC ticket booth and attended more sporting events than Sparky the mascot. As people passed by, Frazier did her best to demystify the swabbing process.

"I tell people it's easy and if you're willing to take three to five minutes, you could save somebody's life," Frazier said.

Those who say yes start by taking a health survey on their smartphones. Then Frazier gives participants a kit that includes four Q-tips, each to be rubbed on the inside of the corners of a person's mouth. The samples are sealed in the kit, the person's name goes on a label, and the registry grows. It's that simple.

"A lot of people think the process is super scary, but I just explain there's only one in 500 chance of being a match for someone and, if you are a match, then 80 percent of the time you just donate peripheral stem cells via regular blood draw," Frazier said. "And 20 percent of the time, they take bone marrow from your hip. But for the rest of your life, you can say you literally saved someone's life."

Curylo not only encouraged her star pitcher to pursue her passion, even if that meant traveling to Tinley Park on some game days to get swabs from visiting teams, but challenged Frazier to think bigger. It was Curylo's idea to swab every team in the Horizon League, which created the unintended consequence of camaraderie.

"This brought teams in our conference together," said Curylo, the conference coach of the year. "We usually go to games, compete, get on our bus and go home. But after we beat Oakland, we hung out and talked because we were all helping Karissa. She's finding a way to make herself matter off the field as much as she does on it."

She's a college student attacking leukemia and lymphoma as fiercely as she does hitters, a young woman hoping to change the world with the Peace Corps after making it better at UIC.

"What's amazing is Karissa is so completely different as a person than she is as a pitcher," Curylo said. "Pitching, she's poker-faced, no emotion, gets the job done. But away from that, she's one of the sweetest, most giving, best teammates around. She has two sides."

You might say they're a perfect match.

dhaugh@chicagotribune.com

Twitter @DavidHaugh

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‘Future of medicine’: Broken pig bones healed using tiny bubbles & ultrasound – RT

By raymumme

A team of scientists has successfully repaired the broken bones of lab animals without invasive surgery, by using microbubbles and ultrasound to stimulate the growth of stem cells.

In a study published in the journal Science Translational Medicine on Wednesday, Maxim Bez and a team of Cedars Sinai-led scientists were able to facilitate the natural growth of stem cells to create more bone marrow in broken bones that cannot heal on their own, known as nonunion fractures.

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While certain bone injuries only require a few weeks in a cast to heal, more severe injuries can cause large gaps between the edges of a fracture that cannot be healed without invasive surgery or bone grafting.

There are currently two methods for bone grafting, either using autografts that transfer bone marrow from a different part of the patients own body, or allografts that use donated bone marrow from another patient. Artificial transplants are often rejected by the body, making another bone graft necessary.

There are more than 2 million bone grafting procedures performed around the world each year, with roughly 100,000 cases in the US alone.

Nonunion bone fractures can cause great damage to the body, leaving patients crippled or with other severe complications.

In a first of its kind study, Bez and his team developed an alternative that uses microbubbles and ultrasound to facilitate the bodys natural stem-cell growth.

"This study is the first to demonstrate that ultrasound-mediated gene delivery to an animal's own stem cells can effectively be used to treat nonhealing bone fractures," said Gadi Pelled, assistant professor of surgery at Cedars-Sinai and co-author of the study, according to Medical XPress. "It addresses a major orthopedic unmet need and offers new possibilities for clinical translation."

In the study, Bez and his team first created severe bone fractures the tibiae bones of large pigs. Then, they inserted a biodegradable collagen scaffold in the fracture, which supported stem cell growth. Two weeks later, after the stem cells grew around the scaffold, the scientists injected microbubbles containing growth-promoting genes. Finally, they used an ultrasound pulse, which causes the stems cells to become bone cells, healing the fracture.

The technique was able to completely heal nonunion fractures in eight weeks. Bez and his team found their method healed bones to the point that they were just as strong as those treated with bone grafts.

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The technique is minimally invasive and does not have the side effects associated with bone grafts. If the method is found to be safe for humans, it would provide patients with an alternative to replace bone grafting.

Bez and his team say that their method could potentially be used in tissue engineering applications in the future.

"We are just at the beginning of a revolution in orthopedics," said Dan Gazit, co-director of the Skeletal Regeneration and Stem Cell Therapy Program in the Department of Surgery and the Cedars-Sinai Board of Governors Regenerative Medicine Institute and co-author of the study, according to Medical XPress. "We're combining an engineering approach with a biological approach to advance regenerative engineering, which we believe is the future of medicine."

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Babies From Skin Cells? Prospect Is Unsettling to Some Experts … – New York Times

By JoanneRUSSELL25


New York Times
Babies From Skin Cells? Prospect Is Unsettling to Some Experts ...
New York Times
Researchers say that scientists may soon be able to create a baby from human skin cells that have been coaxed to grow into eggs and sperm.

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Encouraging results after Jonathan Pitre’s transplant, mother says – CTV News

By daniellenierenberg

Nick Wells, CTVNews.ca Published Wednesday, May 17, 2017 7:04AM EDT Last Updated Wednesday, May 17, 2017 12:28PM EDT

An Ottawa-area boy who suffers from a rare and painful blistering skin disease is recovering in a Minneapolis hospital, after undergoing a second potentially life-changing transplant.

Jonathan Pitre, known as the "Butterfly Boy" because of his delicate, blistering skin, received a second transfusion of his mother Tina Boileaus stem cells in April.

In a Facebook post Tuesday, Boileau said the donor study tests are showing that her son is officially growing her cells.

Pitre was born with a severe form of epidermolysis bullosa (EB), an incurable genetic collagen disorder. The condition causes a never-ending series of raw and painful blisters -- some of which hes had for years.

His mother told CTV News on Wednesday that the positive turn in Pitres long and painful treatment was exactly what we needed.

Boileau said her son has had infections on top of infections and endured much pain over the past year. The second stem cell transplant has been really hard on his body, she said, but there now seems to be light at the end of the tunnel.

Yesterday was just the greatest day. We were speechless. Jonathan hugged me and we were like, We did it, she said in an interview from the hospital.

Boileau said that even some of the nurses were crying when Pitre received the good news.

Its finally now feeling like its all been worth it.

However, she pointed out that if Pitre is unable to grow his own cells, he could be diagnosed with Graft vs. Host disease a condition where the donor's cells take over the host's organs and bodily functions, leading to complications.

We still have a long road ahead of us, but you know what, this is definitely what weve been waiting for, Boileau said.

The $1.5-million transplant procedure Pitre is undergoing is currently only performed as a University of Minnesota clinical trial.

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