Archive for the ‘Bone Marrow Stem Cells’ Category

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

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

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

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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 ... and more »

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

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

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

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An 'incredibly strong and brave' three-year-old is on the road to recovery after having a stem cell transplant to cure her rare form of cancer.

Hazel Richardson, who lives in the village of Albury near Bishop's Stortford, was diagnosed with Juvenile Myelomonocytic leukaemia (JMML) in 2015.

She had her second stem cell transplant at Great Ormond Street Hospital in September 2016 after a donor in Germany was found.

Hazel's aunt, Jemma MacFadyen said: "Hazel is such a little character, so strong and brave and cheeky. She turned three in April, but she thinks she is four.

"She was incredibly strong and brave, I think it was much harder for her parents. She was very strong.

"She was diagnosed with an incredibly rare form of leukaemia in November 2015, while her mum Alice was in Addenbrooke's Hospital having a baby.

"There did not appear to be anything that wrong with Hazel, but her mum knew that something was not right.

"She was a bit floppy and kept getting these temperatures and she also had spots on her face, which we now know is quite characteristic of JMML, but at the time did not seem like anything."

READ MORE: Cheshunt boy with cerebral palsy takes first steps after potentially life-changing 75,000 operation

Hazel had her first stem cell transplant in April of last year, but unfortunately it did not take and her disease returned.

Mrs MacFadyen explained: "The only treatment for JMML is a bone marrow transplant, or stem cell transplant as it is known now.

"Hazel had her first transplant at Great Ormond Street Hospital in April last year.

"How it works is they gave her a very strong dose of chemotherapy, then they attach a drip with the transplant.

"It did not work and quickly she began relapsing even before she left Great Ormond Street."

Fortunately the blood cancer charity Anthony Nolan managed to find Hazel another donor, one with an even higher match percentage.

Mrs Facfadyen said: "JMML is very, very rare, Addenbrooke's said they have only had six or seven patients who have had the disease.

"Anthony Nolan, who have the register for donors, found another match for Hazel. He was German and was a nine out of ten match, which was better than the first one.

"So far this one has helped. We are hopeful that this has been more successful than the first one.

"They say if it comes back it comes back quickly and very hard. So every day is a day away from where we were.

"All donors make their donations in their home countries then an Anthony Nolan courier brings the stem cells over. It is all very secretive.

"When two years elapses after the transplant you can meet with the donor, if they want to, and I think this is what the family is planning on doing."

Hazel has recently started to go to Albury Acorns pre-school, and the Furnuex Pelham Church of England School is planning to donate some of the money raised from the Felham Fayre on June 25 to Anthony Nolan.

In the future Hazel's family hopes to raise money for the charity themselves according to Mrs Macfadyen.

She said: "We definitely want to do some fundraising for Anthony Nolan, something big.

"We want to be sure and we want to now as a family that we can handle it because we have just come out of a difficult time."

NEXT STORY: Cheshunt seven-year-old comes through life-changing 75,000 operation

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'Incredibly strong and brave' Albury girl, 3, recovering after stem cell transplant to cure cancer - Hertfordshire Mercury

May 17, 2017 06:00 ET | Source: CytoDyn Inc.

VANCOUVER, Washington, May 17, 2017 (GLOBE NEWSWIRE) -- CytoDyn Inc. (OTC.QB:CYDY), a biotechnology company focused on the development of new therapies for combating human immunodeficiency virus (HIV) infection, announces the treatment of the first patient in its Phase 2 clinical trial for Graft versus Host Disease (GvHD), its leading immunologic indication for PRO 140.

GvHD is a potentially life-threatening complication in patients requiring a bone marrow transplant because their immune systems have been depleted during aggressive cancer therapy for certain types of leukemia. These patients have a 40-60% one-year survival rate, with relapsed GvHD as the leading causes of death.

The multicenter, 60-patient Phase 2 trial will evaluate the safety and efficacy of PRO 140 with an equal number of patients receiving PRO 140 and placebo. The trial is supported by study data using a xeno-GvHD animal model where human bone marrow stem cells were administered to immunocompromised mice, which leads to severe GvHD culminating in death. PRO 140 at a comparable dose to that being employed in CytoDyns Phase 2 trial completely eliminated any signs of GvHD in these mice. Effects of stem cell engraftment was apparent in blood, spleen and bone marrow of the mice without signs of GvHD. This preclinical study is being submitted to the U.S. Food and Drug Administration (FDA) in support of CytoDyns Orphan Drug Designation application and publication of this data is forthcoming.

We selected the transplantation indication called GvHD as our first expansion of PRO 140 into a non-HIV clinical indication because it targets the CCR5 receptor, which is known to be an important mediator of GvHD, especially in the organ damage that is the usual cause of death, said Denis R. Burger, Ph.D., CytoDyns Chief Science Officer. We plan to explore additional opportunities to expand the clinical applications of PRO 140 to those indications where CCR5 plays an important role, namely certain autoimmune diseases and cancer.

If CytoDynreceives positive results from this Phase 2 study, the Companyexpects to file for Breakthrough Designation with the FDA to expedite the commercialization of PRO 140 for this clinical indication. As previously reported, PRO 140 is considered safe and well tolerated with negligible toxicities or side effects. The Company believes these attributes make it promising for the treatment of GvHD.

About Graft versus Host Disease GvHD occurs after a bone marrow or stem cell transplant in which an individual receives bone marrow tissue or cells from a donor, known as allogeneic transplant.The transplanted cells regard the recipient's body as foreign and attack the recipient's body. GvHD does not occur when an individual receives his or her own cells during a transplant. Before a transplant, tissue and cells from possible donors are tested to determine how closely they match the person having the transplant with GvHD is less likely to occur, or symptoms to be milder, when the match is close. The chance of GvHD can be between 30% and 40% when the donor and recipient are related and 60% to 80% when the donor and recipient are not related. There are two types of GvHD: acute and chronic. Symptoms in both acute and chronic GvHD range from mild to severe. Acute GvHD usually occurs within the firstthree months after a transplant. According to the U.S. Department of Health and Human Services, nearly 5,000 allogeneic transplants were performed in the U.S. in 2016.

About CytoDyn CytoDyn is a biotechnology company focused on the clinical development and potential commercialization of humanized monoclonal antibodies for the treatment and prevention of HIV infection. The Company has one of the leading monoclonal antibodies under development for HIV infection, PRO 140, which has completed Phase 2 clinical trials with demonstrated antiviral activity in man and is currently in Phase 3. PRO 140 blocks the HIV co-receptor CCR5 on T cells, which prevents viral entry. Clinical trial results thus far indicate that PRO 140 does not negatively affect the normal immune functions that are mediated by CCR5. Results from seven Phase 1 and Phase 2 human clinical trials have shown that PRO 140 can significantly reduce viral burden in people infected with HIV. A recent Phase 2b clinical trial demonstrated that PRO 140 can prevent viral escape in patients during several months of interruption from conventional drug therapy. CytoDyn intends to continue to develop PRO 140 as a therapeutic anti-viral agent in persons infected with HIV and to pursue non-HIV indications where CCR5 and its ligand CCL5 may be involved. For more information on the Company, please visit http://www.cytodyn.com.

About PRO 140 PRO 140 belongs to a new class of HIV/AIDS therapeutics viral-entry inhibitors that are intended to protect healthy cells from viral infection. PRO 140 is a humanized IgG4 monoclonal antibody directed against CCR5, a molecular portal that HIV uses to enter T-cells. PRO 140 blocks the predominant HIV (R5) subtype entry into T-cells by masking this required co-receptor, CCR5. Importantly, PRO 140 does not appear to interfere with the normal function of CCR5 in mediating immune responses. PRO 140 does not have agonist activity toward CCR5 but does have antagonist activity to CCL5, which is a central mediator in inflammatory diseases. PRO 140 has been the subject of seven clinical trials, each demonstrating efficacy by significantly reducing or controlling HIV viral load in human test subjects. PRO 140 has been designated a fast track product by the FDA. The PRO 140 antibody appears to be a powerful antiviral agent leading to potentially fewer side effects and less frequent dosing requirements as compared to daily drug therapies currently in use.

Forward-Looking Statements This press release includes forward-looking statements and forward-looking information within the meaning of United States securities laws, including statements regarding CytoDyns current and proposed trials and studies and their results, costs and completion. These statements and information represent CytoDyns intentions, plans, expectations, and beliefs and are subject to risks, uncertainties and other factors, many beyond CytoDyns control. These factors could cause actual results to differ materially from such forward-looking statements or information. The words believe, estimate, expect, intend, attempt, anticipate, foresee, plan, and similar expressions and variations thereof identify certain of such forward-looking statements or forward-looking information, which speak only as of the date on which they are made.

CytoDyn disclaims any intention or obligation to publicly update or revise any forward-looking statements or forward-looking information, whether as a result of new information, future events or otherwise, except as required by applicable law. Readers are cautioned not to place undue reliance on these forward-looking statements or forward-looking information. While it is impossible to identify or predict all such matters, these differences may result from, among other things, the inherent uncertainty of the timing and success of and expense associated with research, development, regulatory approval, and commercialization of CytoDyns products and product candidates, including the risks that clinical trials will not commence or proceed as planned; products appearing promising in early trials will not demonstrate efficacy or safety in larger-scale trials; future clinical trial data on CytoDyns products and product candidates will be unfavorable; funding for additional clinical trials may not be available; CytoDyns products may not receive marketing approval from regulators or, if approved, may fail to gain sufficient market acceptance to justify development and commercialization costs; competing products currently on the market or in development may reduce the commercial potential of CytoDyns products; CytoDyn, its collaborators or others may identify side effects after the product is on the market; or efficacy or safety concerns regarding marketed products, whether or not scientifically justified, may lead to product recalls, withdrawals of marketing approval, reformulation of the product, additional preclinical testing or clinical trials, changes in labeling of the product, the need for additional marketing applications, or other adverse events.

CytoDyn is also subject to additional risks and uncertainties, including risks associated with the actions of its corporate, academic, and other collaborators and government regulatory agencies; risks from market forces and trends; potential product liability; intellectual property litigation; environmental and other risks; and risks that current and pending patent protection for its products may be invalid, unenforceable, or challenged or fail to provide adequate market exclusivity. There are also substantial risks arising out of CytoDyns need to raise additional capital to develop its products and satisfy its financial obligations; the highly regulated nature of its business, including government cost-containment initiatives and restrictions on third-party payments for its products; the highly competitive nature of its industry; and other factors set forth in CytoDyns Annual Report on Form 10-K for the fiscal year ended May 31, 2016 and other reports filed with the U.S. Securities and Exchange Commission.

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CytoDyn Treats First Patient with PRO 140 in Phase 2 Trial for Graft versus Host Disease - GlobeNewswire (press release)

Patients dealing with blood and immune disorders, especially those in the most advanced stages, often have no choice but to undergo bone marrow transplants. Ironically, even if the treatment can be life-saving, it would only work when the bone marrow cells of the recipients are completely eliminated using drugs and radiation. And this could cause serious negative side effects such as organ damage, cataracts, infertility, new cancers, and even death.

Thanks to the work of engineers at the University of California San Diego (UCSD), that kind of bone marrow transplant may soon be rendered obsolete. Rather than using a live bone marrow from a compatible donor or from the patients themselves, a synthetic bone implant will instead be used and such will not require the use of drugs that can cause all those harmful side effects.

Bone marrow is the flexible tissue inside the bones that is responsible for producing red blood cells from stem cells. If, for some reason, the bone marrow fails to do its job, the result can either be severe anemia or an impaired immune system. Whichever of these conditions arise, the most effective treatment is typically a bone marrow transplant.

To reduce the undesirable side effects caused by traditional bone marrow transplants, the UCSD team of bioengineers led by Shyni Varghese have developed a synthetic bone implant with a practical marrow that can produce its own blood cells. The implant is divided into two sections, both of which are engineered from a hydrogel matrix. The exterior layer containing calcium phosphate minerals functions as a bone, while the interior layer contains donor stem cells for bone marrow growth. The exterior layer works together with the hosts cells to assist in bone building, merging the implant with the natural structure of the body.

According to the team, they have tested their engineered implant in mice, and the tests proved successful. Specifically, they implanted the synthetic bone under the skin of mice, some of which had functional bone marrow, and some of which had defective bone marrow due to radiation.

Within a four-week observation period, the implant developed bone-like structures that didnt only have blood vessels, but also marrow that actually produced red blood cells. And after six months, the synthetic implants and the bloodstream of the mice showed a mix of blood cells from both the donor and the host. This shows that the implants can function as natural bones, with the blood cells produced by the synthetic implant naturally circulating within the hosts bloodstream without being rejected.

As promising as those results are, however, there is no guarantee that the technique will be as effective in humans. Further study will be required before it can be accepted and approved by the FDA.

Theres also the matter of the treatment only being effective on patients with non-malignant bone marrow disorders. The implant cannot do anything to stop or prevent cancerous mutation from spreading, which means when it comes to cancer patients, undergoing radiation therapy will still be required to kill off their cancer cells, before a bone marrow transplant can work.

Nevertheless, this is still considered a step forward and an exciting development, particularly for individuals suffering from blood disorders. Not only will the treatment ease their pain and distress because theyll be free of their disease; it will also keep them from suffering negative side effects.

The research was recently published in the journal PNAS.

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New Discovery Could Soon Replace The Painful Bone Marrow ... - Wall Street Pit

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Engineered bone marrow could make transplants safer Science Daily Bone marrow transplants are used to treat patients with bone marrow disease. Before a transplant, a patient is first given doses of radiation, sometimes in combination with drugs, to kill off any existing stem cells in the patient's bone marrow. This ... Engineered Bone Marrow Improves Transplant SafetyR & D Magazine Engineered bone marrow may ease transplantsThe San Diego Union-Tribune all 4 news articles »

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Engineered bone marrow could make transplants safer - Science Daily

Mom, we did it.

With those words, Jonathan Pitre hugged his mother, Tina Boileau, and shared his joy and relief at learning the news late Tuesday afternoon that his stem cell transplant has worked.

Blood tests revealed that all of the new white cells in his bloodstream are from his mothers donated stem cells, and contain her two telltale X chromosomes. It means his mothers donated stem cells have taken root in his bone marrow and have started to produce new blood cells.

This is the best news ever, the best Mothers Day gift, said an elated Boileau, who has remained at her sons side throughout his marathon treatment for epidermolysis bullosa, a rare and painful disease that causes his skin to blister and tear easily.

Jonathan Pitre rests in bed, his pillow with his Boston terrier, Gibson, on it close by. Tina Boileau / -

Oftentimes, doctors find a mix of white blood cells, from the donor and patient, soon after a stem cell transplant. But in Pitres case, all of the new white blood cells, 100 per cent, were donor cells.

Jon is full of me, said Boileau. He doesnt have any T-cells that are his.

Pitre, who turns 17 next month, was allowed out of his room for the first time Tuesday since his April 13 transplant when he was infused with stem cells drawn from his mothers hip bone. His infection-fighting white blood cells are now numerous enough his count hit 1.0 on Tuesday that he was allowed to emerge from medical isolation.

We celebrated our good news by going for a walk in the hallway, Boileau said.

Pitre has been in Minnesota since mid-February to undergo his second attempt at the experimental treatment pioneered by doctors at the University of Minnesota Masonic Childrens Hospital.His first transplant ended in disappointment on Thanksgiving Day last year, but the family opted to undergo a second transplant, despite its risks and hardships.

This time, wonderfully, it worked.

I just got official results: Jon is 100 per cent donor! Boileau said in a text message late Tuesday afternoon.

Offered to children and adolescents with severe EB as part of a clinical trial, the stem cell transplant is physically demanding and comes with a host of life-threatening side effects. One of those potential side effects is graft-versus-host-disease (GVHD), a complication in which the new white blood cells turn on the patients tissues and attack them as foreign.

BACKGROUND:Butterfly child dreams of the Northern Lights

Pitre has suffered infections, fevers and profound exhaustion ever since his transplant while battling to get his pain levels under control. Doctors will now be on guard for signs of GVHD.

A Grade 11 student from Russell, Pitre suffers from a rare form of EB that complicates how he moves, eats, bathes and sleeps. Many of those with severe EB die from an aggressive form of skin cancer in their 20s.

The stem cell transplant holds the potential to dramatically improve Pitres life and produce tougher skin that blisters less and heals more readily.

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It worked! Jonathan Pitre's transplant takes root - Ottawa Citizen

A new study has determined bone marrow stem cell transplants improved the motor skills and nervous system of mice with amyotrophic lateral sclerosis (ALS) by repairing damage to the blood-spinal cord barrier.

ALS is a progressive neurodegenerative disease that affects neuronal cells in the brain and the spinal cord, which send signals to control muscles throughout the body. The progressive degeneration of motor neuron cells leads to death. It is estimated more than 6,000 Americans are diagnosed with the ALS yearly.

The University of South Floridas Center of Excellence for Aging and Brain Repair study findings were published in the journal Scientific Reports, determining results of their experiment are an early step in pursuing stem cells for potential repair of the blood-spinal cord barrier, which has been identified as key in the development of ALS.

Previous studies in development of various therapeutic approaches for ALS typically used pre-symptomatic mice, Svitlana Garbuzova-Davis, leader of the research project and University of South Florida health professor, said. This is the first study advancing barrier repair that treats symptomatic mice, which more closely mirrors conditions for human patients.

Using stem cells harvested from human bone marrow, researchers transplanted cells into mice modeling ALS and already showing disease symptoms. The transplanted stem cells differentiated and attached to vascular walls of many capillaries, beginning the process of blood-spinal cord barrier repair delaying progression of the disease and improving motor function in the mice, as well as increased motor neuron cell survival the study reported.

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Stem cell transplants beneficial to mice with ALS - Life Science Daily

Researchers at the University of South Florida show in a new study that bone marrow stem cell transplants helped improve motor functions and nervous system conditions in mice with the disease Amyotrophic Lateral Sclerosis (ALS) by repairing damage to the blood-spinal cord barrier.

In a study recently published in the journal Scientific Reports, researchers in USF's Center of Excellence for Aging and Brain Repair say the results of their experiment are an early step in pursuing stem cells for potential repair of the blood-spinal cord barrier, which has been identified as key in the development of ALS. USF Health Professor Svitlana Garbuzova-Davis, PhD, led the project.

Previous studies in development of various therapeutic approaches for ALS typically used pre-symptomatic mice.

"This is the first study advancing barrier repair that treats symptomatic mice, which more closely mirrors conditions for human patients," Dr. Garbuzova-Davis said.

Using stem cells harvested from human bone marrow, researchers transplanted cells into mice modeling ALS and already showing disease symptoms. The transplanted stem cells differentiated and attached to vascular walls of many capillaries, beginning the process of blood-spinal cord barrier repair.

The stem cell treatment delayed the progression of the disease and led to improved motor function in the mice, as well as increased motor neuron cell survival, the study reported.

ALS is a progressive neurodegenerative disease that affects neuronal cells in the brain and the spinal cord, which send signals to control muscles throughout the body. The progressive degeneration of motor neuron cells leads to death from ALS. More than 6,000 Americans each year are diagnosed with the disease.

Because stem cells have the ability to develop into many different cell types in the body, researchers at USF's Center of Excellence for Aging and Brain Repair, Department of Neurosurgery & Brain Repair have focused on using stem cells to restore function lost through neurodegenerative disorders or injuries.

Damage to the barrier between the blood circulatory system and the central nervous system has been recently recognized as a factor in ALS development, leading researchers to work on targeting the barrier for repair as a potential strategy for ALS therapy.

In this study, the ALS mice were given intravenous treatments of one of three different doses of the bone marrow stem cells. Four weeks after treatment, the scientists determined improved motor function and enhanced motor neuron survival. The mice receiving the higher doses of stem cells fared better in the study, the researcher noted.

The transplanted stem cells had differentiated into endothelial cells -- which form the inner lining of a blood vessel, providing a barrier between blood and spinal cord tissue -- and attached to capillaries in the spinal cord. Furthermore, the researchers observed reductions in activated glial cells, which contribute to inflammatory processes in ALS.

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Stem cell transplants may advance ALS treatment by repair of blood-spinal cord barrier - Science Daily

This month is the annual ExplorAsian festival, which celebrates Asian heritage in Metro Vancouver. It features a large number of events from lectures to arts and entertainment.

One of the events held Saturday, May 13, at Metrotown in Burnaby is a little bit different. Held in partnership with Canadian Blood Services, its an outreach to the Asian community and those from multi-ethnic or biracial backgrounds to consider becoming a stem cell donor. Matching blood types is relatively easy matching stem cell and bone marrow donors to patients in need is quite hard, especially for those from diverse backgrounds. In fact the more diverse we become in B.C., the more critical our need for diverse donors.

I talked to the organizers of the Thanks Mom Give Life 2017 campaign this week.

You can find out more information about stem cell and blood donation at Canadian Blood Services.

For more information on craft beer, you can find The Growler here.

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Lotusland 17: BC's diverse population needs diverse stem cell donors - Delta-Optimist

On Tuesday, Carson Tahoe Cancer Center opened a new blood and bone marrow transplant care clinic with support from the Huntsman Cancer Institute (HCI) at the University of Utah. Under the collaboration, a Bone Marrow Transplant (BMT) physician and nurse from HCI will travel to Carson City once a month to treat patients both before and after they receive a transplant.

Blood and marrow transplants are performed in patients with cancers of the blood and lymphatic systems, including leukemia, lymphoma and multiple myeloma. The transplants replace bone marrow that has been damaged or destroyed with a supply of healthy blood stem cells, which in turn travel to the bone marrow and promote growth of new marrow.

Currently, patients in the Northern Nevada area who need a transplant must travel outside the area for treatment. Through this model, patients will still receive their transplant at HCI in Salt Lake City. But they will now be able to receive care at the Carson Tahoe clinic for planning and follow-up appointments, which typically occur every month for a year following transplant.

"This clinic is going to enable patients to receive more of their pre-and post-BMT care closer to home," said Daniel Couriel, MD, Professor of Medicine at the University of Utah and Director of HCI's BMT program. "We hope to maximize the time patients can spend in their own homes with their loved ones as they recover."

The BMT clinic will be open the third Monday of every month. Patients can access the clinic by referral.

"Because of the added bench strength we receive from HCI, we are better equipped to provide outstanding bone marrow transplant care, close to home," said Ed Epperson, CEO of CTH.

CTH formally affiliated with University of Utah Health in 2013 and with Huntsman Cancer Institute in 2015 in an effort to improve accessibility to specialty care for Northern Nevada residents. The relationship between the health care systems provides resources that allow Carson Tahoe Health to meet the ever-changing health care needs of the community.

"Both organizations share a commitment to providing the highest quality cancer care to patients, no matter where they live," said John Sweetenham, MD, Executive Medical Director at Huntsman Cancer Institute and Professor of Medicine at the University of Utah. "BMT treatment is a very unique type of care, and we look forward to working with Carson Tahoe to bring this service to the community."

To find out more about the clinic, residents can call Carson Tahoe Cancer Center at 775-445-7500.

More here:
Carson Tahoe Health opens blood and bone marrow transplant care clinic - Nevada Appeal

An umbilical cord after birth yields about three to five ounces of cell-rich cord blood. That's not a lot, but enough of it can help treat more than 80 or so diseases. A North Texas oncologist says education's key to boosting limited supply.

The KERA Interview with Dr. Sharif

Dr. Suhail Sharif is a surgical oncologist with Texas Health Fort Worth.

Interview Highlights:

Whats special about cord blood: Cord blood has immature blood cells, and you can use these stem cells to, basically, harvest into these patients that have problems with their own blood; for example, because of leukemia or lymphoma or other types of diseases that affect their own blood lines. These can grow into the red blood cells if [they're] deficient or the white blood cells if [they're] deficient or even platelets, for that matter.

Cord blood cells vs. bone marrow cells: Cord blood stem cells are actually stored in a blood bank that you can use on patients that need it. But bone marrow, you actually have to go through a process of harvesting the bone marrow. Its a very painful procedure for whoever is donating the bone marrow. And then they have to go through an extensive and rigorous testing, not only for infectious causes, but also to see if they match with the patient. And then they have to harvest, and they basically have to transplant it. Now, that whole process can take a few months. If you just have cord blood stem cells, these have already been stored and are readily available. And if you have a match with the donor and the recipient, you can use them right away.

Cord blood is limited in supply: If you think about the blood that is in the placenta and the cord, its in the range of three to five ounces. Thats about like half a cup. Thats the reason why you have to gather it from a lot of patients. At this point, there are, I believe, close to 175,000 matched cord blood available."

But its not enough: If you think about what percentage of deliveries actually translate into donating cord blood, its very miniscule. Thats why were educating the parents about the benefits of cord blood so they can donate to a public blood bank so that we can use it in treating patients with deadly cancers and so forth in our community.

For more information:

See the article here:
Cord Blood: A Small Amount Does A Lot Of Good | KERA News - KERA News

Science Daily

New intervertebral discs from stem cells Science Daily The study on the sick German shepherds was organized as follows: With the permission of the dog owners, neurologist Frank Steffen and his team removed stem cells from the marrow of the pelvic bone of the affected animals. After the cleaning and ...

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New intervertebral discs from stem cells - Science Daily

A new biomimetic bone tissue may help improve bone marrow transplants.

Engineers at the University of California San Diego have developed a bone-like implant that eliminates the need for donor cells to wipe out the hosts pre-existing cells, by allowing donor cells the space to live and grow.

Weve made an accessory bone that can separately accommodate donor cells. This way, we can keep the host cells and bypass irradiation, bioengineering professor Shyni Varghese, from the UC San Diego Jacobs School of Engineering, said in a statement.

The implants are made of a porous hydrogel matrix that contains calcium phosphate minerals in the outer matrix and donor stem cells that produce blood cells in the inner matrix.

The researchers successfully tested the bone tissues in mice and the donor cells survived for at least six months, while supplying the mice with new blood cells.

The structures matured into bone tissues of the mice that have a working blood vessel network and a bone marrow inside that supplies new blood cells. After a month the implanted marrow contained a mixture of host and donor blood cells, which remained circulating in the bloodstream even after 24 hours.

In the future, our work could contribute to improved therapies for bone marrow disease, Yu-Ru (Vernon) Shih, a research scientist in Vargheses lab and the studys first author said in a statement. That would have useful applications for cell transplantations in the clinic.

The researchers also took stem cells from the implanted marrow and transplanted them into another group of mice with their marrow stem cells eradicated by radiation and drugs. The transplanted cells diffused into the bloodstream of the mice in the second group.

Were working on making this a platform to generate more bone marrow stem cells, Varghese said.

According to Varghese, the implants could only be used in patients with non-malignant bone marrow diseases, where there arent any cancerous cells that need to be eliminated.

The researchers said this discovery indicates that implanted marrow is functional and donor cells can form and survive for long periods of time in the presence of host cells. They also said that the host and donor cells can travel between the implanted marrow and the hosts circulating blood through the blood vessel network formed in the implanted bone tissue.

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Engineered Bone Marrow Improves Transplant Safety - R & D Magazine

12 May 2017 Bone marrow: be someones match A bone-marrow donation is not a scary procedure and doesnt involve drilling into your bones. We found out more.

Unfortunately many people still believe that donating bone marrow means doctors will need to drill into your bones. The reality is the donation process requires no surgery, no general anaesthetic and no drilling.

South African patients who have leukaemia or other blood disorders need a life-saving stem cell transplant and rely on the South African Bone Marrow Registry (SABMR) to find a match.

The challenges faced in growing the SABMR lies in the sentence itself, says Alana James, CEO of the Sunflower Fund. Its called The South African Bone Marrow registry, so when people see the word bone marrow they go Oh no I cant do that, it will hurt!

Understanding bone marrow

Bone marrow is found inside your bones its a soft, fatty tissue that helps with the production of red blood cells (to carry oxygen), white blood cells (to find infection) and platelets (to prevent bleeding). Some of the cells in the bone marrow can be pushed into the blood stream, where it can be collected and then used to help someone in need of a donation.

Real-life hero

Carey Symons, a blood stem cell donor and long-time support of The Sunflower Fund, was on the registry for 10 years before she was called to help a leukaemia patient.

The stats of a perfect match are 1 in 100 000 so you can only imagine my joy in being that 1 in 100 000 and that I was able to contribute to giving someone a second chance.

She travelled from Durban to Mediclinic Constantiaberg in Cape Town where she had a series of painless Neupogen injections that helped stimulate the production and release of blood stem cells.

Three days of injections later she was able to begin the donation process.

1. Two needles (similar to those used when you donate blood) were inserted in each arm. 2. Blood was drawn from one arm and circulated through a cell-separator machine. 3. Her stem cells were collected and the remaining blood was returned through the other arm.

Typically, the donation process takes between four and six hours.

I realised that the day I signed as a donor, I was only hoping to make a difference. I will never know whose life I made a difference to, and part of that mystery excites me. Its a blessing to give without knowing and without being thanked.

Harsh reality

There are just under 74 000 donors on the registry, but at least 400 000 are needed.

We definitely still have a mountain to climb and are committedly doing so. Registering as a donor on the SABMR is a simple process and can be very rewarding, says James. You could be someones perfect match.

There is a 1 in 100 000 chance of being a match. (Image: iStock)

Be someones 1 in 100 000

Signing up to be a donor is simple- if you do meet the criteria, you will receive a reference number and form to fill out. Next, youll go to your nearest Donor Recruitment Clinic where theyll take two test tubes of blood.

Your blood is analysed and put onto the national database. Unfortunately the tissue typing test is expensive (it costs R2 000) but you can make a donation to help free up their funds.

Youll receive your donor card in six to eight weeks, and if youre ever a match for a patient, you will be called.

Read more:

CT teen desperately needs bone marrow transplant

URGENT need for more bone marrow donors in SA

Needed: Black bone marrow donors

A leukaemia patient who had recently undergone a bone marrow transplant received a massive surprise when Taylor Swift visited him n hospital. When she saw that he had a keyboard in his room she asked him to play for her. As he plays Adele's "Someone like you", Taylor joins in, singing along!

CANSAs purpose is to lead the fight against cancer in South Africa. Its mission is to be the preferred non-profit organisation that enables research, educates the public and provides support to all people affected by cancer. Questions are answered by CANSAs Head of Health Professor Michael Herbst and Head of Advocacy Magdalene Seguin. For more information, visit cansa.org.za.

The information provided does not constitute a diagnosis of your condition. You should consult a medical practitioner or other appropriate health care professional for a physical exmanication, diagnosis and formal advice. Health24 and the expert accept no responsibility or liability for any damage or personal harm you may suffer resulting from making use of this content.

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Bone marrow: be someone's match - Health24

Science World Report

Artificial tissues can provide bone marrow The Hindu Bone marrow transplants are used to treat patients with bone marrow disease. Before a transplant, a patient is first given doses of radiation, sometimes in combination with drugs, to kill off any existing stem cells in the patient's bone marrow. This ... Newly Discovered Synthetic Bone Implant Could Provide Healthy ...Science World Report all 4 news articles »

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Artificial tissues can provide bone marrow - The Hindu

South African patients suffering from Leukaemia and other blood disorders who need a life-saving stem cell transplant, rely on the South African Bone Marrow Registry (SAMBR) to find a donor who is a genetic match.

But, the untrue and frightening belief that donating blood stem cells, or bone marrow, involves drilling through bones is a common misconception and is one of the challenges faced in growing the SAMBR.

These misconceptions, often stopping people from registering to become donors and giving someone the hope of life.

The Sunflower Fund educates, raises funds and recruits potential blood stem cell donors to this registry and pays for the tissue typing test cost for each person who joins.

The fund also prioritises educating people on the truths of becoming a donor and works hard to debunk any myths that exist. Sometimes it is just the words themselves that act as barriers. The challenges faced in growing the SABMR lies in the sentence itself, Alana James, CEO of the Sunflower Fund said. Its called The South African Bone Marrow registry, so when people see the word bone marrow they go Oh no I cant do that, it will hurt!.

Blood stem cell donor and long-time supporter of the Sunflower Fund Carey Symons shares her own story at events and conferences, spreading the message that the process of potentially saving a life is not as scary as we might think, encouraging others to do the same.

The stats of a perfect match are 1 in 100 000 so you can only imagine my joy in being that 1 in 100 000 and that I was able to contribute to giving someone a second chance, Symons said, who was called ten years after joining the registry to donate her stem cells to a patient suffering from leukaemia.

The Durban mother travelled to Constantiaberg hospital in Cape Town, to begin a series of painless Neupogen injections which stimulate the production and release of blood stem cells.

After three days of injections, she was ready to begin the donation process: Two needles, similar to the ones used when donating blood, were inserted; one in each arm. Blood was drawn from one arm, circulated through a cell-separator machine where her stem cells were collected and the remaining blood was returned through the other arm.

After 4-6 hours, the life-saving stem cells were harvested and for Symons, the process was over.

She said she often thought about the person she donated her stem cells to and sometimes wonders who they were. I realised that the day I signed as a donor, I was only hoping to make a difference. I will never know whose life I made a difference to, and part of that mystery excites me. Its a blessing to give without knowing and without being thanked.

There are just under 74 000 donors on the registry, but at least 400 000 are need. We definitely still have a mountain to climb and are committedly doing so. Registering as a donor on the SABMR is a simple process and can be very rewarding, James explained. You could be someones perfect match.

Find out more about becoming a blood stem cell a donor by contacting The Sunflower Fund on toll-free number: 0800 12 10 82 or visit http://www.sunflowerfund.org.za.

About The Sunflower Fund:

The Sunflower Fund, a South African Non-Profit Company (NPC), is dedicated to creating awareness, educating the public and handling the registration process for people to join the South African Bone Marrow Registry (SABMR).

The Sunflower Fund pays for the test cost of people joining the SABMR. This is fundamental to saving the lives of thousands of South Africans each year. The chance of finding a matching donor is 1 in 100,000 and as ethnic origin plays a significant role in the search for a donor, South Africas rainbow nation is at a distinct disadvantage, requiring a large pool of prospective donors.

Should you wish to become a donor, support one of the fundraising projects or make a financial contribution, please contact The Sunflower Fund on toll-free number:

0800 12 10 82. Visit http://www.sunflowerfund.org.za to learn more or look out for the DONATE button to make a cash donation via the website.

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The simple truth of saving lives - Independent Online

Be The Match BioTherapies LLC, a recently created subsidiary of the Minneapolis-based National Marrow Donor Program/Be The Match, this month emerged as a biotechnology venture investor with its participation in a $50 million financing round for a Massachusetts stem cell company. And, its leaders say, it likely wont be the last time the 17-month-old nonprofit spinoff will take part in venture funding to support the commercialization of biotech related to NMDP/Be The Matchs mission of advancing cell therapies for leukemia patients and others needing bone marrow/stem cell transplants. NMDP/Be The Match moved its 995 employees into a newly constructed headquarters building in the North Loop in December 2015. It runs a network of more than 486 organizations that support marrow transplant worldwide, including 178 transplant centers in the United States and more than 45 international donor centers and cooperative registries. Wholly-owned subsidiary Be The Match BioTherapies was among the Series B investors for Magenta Therapeutics of Cambridge, Massachusetts, a biotech company developing therapies to improve and expand the use of curative stem cell transplantation for more patients. Other participants in the oversubscribed round included new lead investor GV (formerly Google Ventures) and existing investors such as Atlas Venture, Third Rock Ventures, Partners Innovation Fund and Access Industries. A major feature of the Magenta deal was also Be The Match BioTherapies new involvement as a strategic partner for the company, under which the two sides will explore opportunities to work together across all of Magentas research efforts, from discovery through clinical development. Magentas lead drug candidate is MGTA-456, which it claims is capable of expanding the number of cord blood stem cells available for transplantation, thus achieving superior clinical outcomes compared to standard transplant procedures. John Wagner M.D., executive medical director of the Bone Marrow Transplantation Program at the University of Minnesota is leading the research. The strategic agreement allows Magenta to leverage Be The Match BioTherapies capabilities, including its cell therapy delivery platform, industry relationships, clinical trial design and management and patient outcomes data generated from the parent organization. According to NMDPs 2015 annual report, Be The Match BioTherapies was established on Dec. 4, 2015, and authorized to do business as a nonprofit limited liability company. The report said it was anticipated the subsidiary would conduct certain business in the field of cellular therapy consistent with the nonprofit mission of its parent corporation, National Marrow Donor Program, but outside the scope of NMDPs customary core business. Led by NMDP Chief Financial Officer Amy Ronneberg, Be The Match BioTherapies says it is making the parent organizations capabilities available to commercial entities developing new allogeneic and autologous cellular therapies. For example, it says it is collaborating with an unnamed biotech company to design a donor identification and cell harvest strategy for white blood cells from donors with specific human leukocyte antigen types. When asked if the subsidiarys venture investment into Magenta Therapies was a sign that it is staking out ground as a stem cell industry investment player, company spokeswoman Melissa Neill told TCB its indeed a scenario that could play out again. We are continually looking for ways to advance science and research in new cellular therapies, she said in an email. In the future, this might mean investments in or additional partnerships with companies whose goals align with our goal of developing and delivering cellular therapies to positively impact patients lives.

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Be The Match Subsidiary Emerges as a Biotech Venture Player - Twin Cities Business Magazine

A project of the university's transfusion medicine department, the stem cell bank would roll out stem cell therapy to patients of thalassemia and sickle cell anaemia.

A public sector stem cell bank is set to come up at UP's King George's Medical University. A project of the university's transfusion medicine department, the stem cell bank would roll out stem cell therapy to patients of thalassemia and sickle cell anaemia. The proposal is awaiting clearance from state department of medical education.

Stem cells are found in human bone marrow and can be derived from the umbilical cord which contains blood vessels that connect baby in the womb to the mother to ingest nutrition required for development.

Research on the therapeutic use of stem cells is underway in US, Europe, China, South East Asia besides India. In UP, Sanjay Gandhi Post Graduate Institute of Medical Sciences (SGPGIMS) and KGMU are both trying to explore the potential of stem cells to treat various health problems. SGPGI has, so far, restricted itself to use of allogenic (stem cells derived from bone marrow of a person), while KGMU has used stem cells derived from the umbilical cord.

KGMU has sustained access to umbilical cord because of a very developed obstetrics and gynaecology department. The proposal is worth Rs 9 crore including infrastructure cost. Stem cell bank promises to become financially self-sustaining within 2-3 years of inception.

View post:
Stem cell bank to come up at KGMU - BSI bureau (press release)

Sensational 8-Year-Old Violinist Living With Painful Disease

WINSTON-SALEM, NC Its hard to walk through life without hitting a sour note or two. In Winston-Salem, there's a young boy with talent beyond his years and a disease that nearly crippled him. His father gave up his career to take care of his son and to get him healthy.

We only listen to classical music at home, said Lucas Sant, a father of three living in Winston-Salem. He sits with his youngest, Helen, 2, on his lap. His second oldest daughter, Maria-Anita, 7, sits on his right and his only son, Caesar, 8, sits to his left.

Hes telling WFMY News 2s reporter, Hope Ford, about his sons remarkable talent.

When he was just a baby, we bought Baby Einstein, and you know, they have the animals and the music. So, we bought him a little toy piano, Lucas began. And one day, when he was seven months old, we heard this music coming from the room. It sounded like the toy piano, but it was the music from the Baby Einstein.

Lucas turned to his wife, Aline, with a knowing smile and said, We have our work to do with this boy.

Videos uploaded to YouTube, show a baby Caesar, waving his arms along to classical music such as Beethoven, almost as if he were conducting a symphony.

A baby Caesar and his father listening to classical music. (Photo: Sant family)

Violin lessons started the age of two.*

He started playing Vivaldi. He would pick up things very quick, said Lucas. Everybody was very impressed.

GoFundMe

All the Sant children are homeschooled and it would be no surprise to learn Caesar is just as brilliant with a pencil as he is with an instrument. The young boy is ahead in math and other subjects and earned a black belt in karate at 5-years-old.

Lucas sat in his seat, as baby Helen decided she wanted to leave the room to see what her mom was up to. As she ran into the next room, Lucas continued his story.

Immediately, he started to get sick. Before five, he had the first stroke.

Caesar has sickle cell anemia.

You never know anything until you experience, Lucas said in a soft voice.

Sickle cell anemia is a blood disease. Normal red blood cells are round and flexible to carry oxygen throughout the body. Caesars blood cells are sickle-shaped or bent and get stuck, slowing the flood of blood and oxygen.

Lucas explained, Its different. Its my son and I never seen this thing.

Caesar, who up until this point sat quietly next to his father with his violin in his lap said, I feel bad. I dont feel good when Im sick.

The curly haired violinist has three strokes before the age of six. The first two left his arms weak, but he rebounded, performing the National Anthem at the Grasshoppers Game in 2013.

The third one was a different stroke, said his dad.

Caesar lost feeling in his arms and legs after his third stroke, leaving him partially paralyzed for nearly six months.

At first, even his eyes was not moving. But, when he did wake up, all of a sudden your son not walk, not run, not stand up, Lucas said as if he was still trying to make sense of it all.

Doctors told the Sant family, It is very unlikely your son is going to die but do not expect much from him.

Lucas paused for a moment and continued, But the good thing there, you really meet God. What am I supposed to do God? Please tell me.

The only thing that seemed right at the time, was for Lucas to give up his career. The father of three was a neuroscientist at Wake Forest Baptist Medical Center.

Forget about my life. I said, Im going give my life to this boy.

Young Caesar in the hospital. (Photo: Sant family)

The Sant family built a small play gym in the basement of their home. Here, Lucas would help Caesar with physical therapy, as they could not afford to hire someone full time to help him regain strength and movement in his arms and legs.

Some days and good and some are bad. Three years after his last stroke, Caesar still winces in pain as he goes through his exercises. But, he finds moments to laugh with his siblings, who cheer him on. And as an 8-year-old, he is a little hard to get under control. For Lucas, the physical therapy takes a toll on his as well.

First, Im not a physical therapist. I have a lot of patience but its very hard for you see your son one way, said Lucas. Sometime, we have to take breaks because it is difficult and it sometimes weighs on my own health.

But, once again, Caesar regained his strength, returning to the Grasshoppers stadium in 2017 to perform the National Anthem once again.

Every month, Caesar and his family travel to Charlotte for blood transfusions to lower the risk of Caesar having another stroke. He'll have to do this for the unforeseeable future and there are risks.*

Frequent blood transfusions can lead to iron overload which is sometimes fatal. Caesar's family is trying for a bone marrow transplant which has a higher percentage of curing his sickle cell disease.

They have a donor- his baby sister, Helen.

As if she knew her name had been mentioned, the young girl, called the boss of the family, walked back into the room, sharing bites of her rice with her siblings and father.

Lucas and his wife wanted another child, but they also wanted to ensure the next child would not have the sickle cell anemia trait. they also wanted to ensure they would have a 100 percent genetic match for Caesar's procedure.

Maria-Anita was also born with sickle cell anemia, but unlike her brother, has yet to experience any complications.

So, Aline got pregnant via in vitro fertilization. Doctors only planted cells that were a genetic match and only healthy cells were selected. Thus, Helen was conceived and at birth, her umbilical cord was collected.

Helen, was born sickle-cell free.

They took the stem cells from the umbilical cord and now they have perfect cells, to do the transplant on him, said Lucas.

The Sant family is trying to raise money for a bone marrow/stem cell transplant. The process is long and costly. According to Johns Hopkins, one hospital that specializes in bone marrow/stem cell transplants, they say the cost can run as high as $500,000.

However, sickle cell anemia can be cured with the procedure.

Offering her big brother another big of food, Helen, Caesars sisterly hero, smiled and ran off.

Lucas continued to explain the familys financial situation.

Its difficult, with me not having a job. But, we have had people help us along the way. But, we are still trying so hard to raise money for the surgery.

A GoFundMe account was started in 2013. To date, $38,000 has been raised. The family also started a website to give updates and sell merchandise to help raise funds as well.

Caesar still walks with a limp and must be careful when sitting down. Lucas looked at his son and said Were so happy because he got back. He got back, but the job is not done. Faith, hope, these things so real. Cause if dont have what you can do? You give up right there.

Caesar piped in again, Sometimes I tell my father, papa, I dont know when Im going to be back, but God is always with me.

Lucas isnt giving up. His hope, to have son healthy by 2018.

And Caesars hope?

I want to be a musician and a conductor.

*This story has been updated to correct information. Lessons for Caesar started at the age of 2 and 300ml of his blood is replaced every month during his blood transfusions.

5 Facts About Sickle Cell Disease (CDC)

More:
Sensational 8-year-old violinist living with painful disease - 13newsnow.com

Iran is expanding investment in stem cell research and its application in various therapies, particularly for hard-to-treat diseases, through the Office of the Vice- Presidency for Science and Technology. As a result of the increase in the number of companies active in the domain of stem cells in the past three years, more than 400 products are processed in the country, indicating a multifold growth compared with the eight-year tenure of previous administration when there were fewer than 50 knowledge-based firms in total.

There are over 40 knowledgebased firms in the field of stem cell and regenerative medicine alone in Iran today, said Amir Ali Hamidiyeh, secretary of the Headquarters for Development of Stem Cell Science and Technology (HDSCST). He made the statement at a press briefing for the second National Festival and International Congress on Stem Cell Sciences and Technologies and Regenerative Medicine to be held July 13- 15 in Tehran, Mehr News Agency reports. According to the conference secretariat, 1,444 people have signed up to attend the event from across the world, including from Iraq, India, Pakistan, Jordan, Russia, Australia, Germany, China, Britain and South Korea. They all are among their countrys respected figures in centers with high academic standing.

The congress is co-sponsored by the Vice-Presidency for Science and Technology and Council on Development of Stem Cell Sciences and Technology. So far, eight stem-cell therapy products for use in hospitals have been produced at the HDSCST laboratories. Manufacturinglicenses have been granted for anadditional number, while others are on thewait list.

Prior to 2014, only 25 knowledgebased companies had applied to operate in this field, of which only one was actively producing quality stem cell products, Hamidiyeh pointed out.

But since then, over 25 workgroups have been formed in cooperation with experts in the specific sciences. Stem cells are cells that have the ability to divide and develop into many different cell types in the body during early life and growth. Stem-cell therapy is the use of stem cells to treat or prevent a disease or condition. Bone marrow transplant is the most widely used stem-cell therapy, but some therapies derived from umbilical cord blood are also in use.

Future of Medicine in Stem

Cells The future of medicine is interrelated with stem cell therapy and the treatment ofrefractory and incurable diseases is in this field of medicine, according to Dr. Ahmad Vosouq Dizaj, the clinical deputy of Royan Institute. Having access to engineering sciences as well as the combination of biology and medicine can play a crucial role in redressing health problems, he said. Stem cells have the ability to replace damaged cells and treat disease. They can also be used to study diseases and provide a resource for testing new medical treatments. The use of stem cells reduces the risk of viral diseases transmission and incidence of Graft Versus Host Disease (GVHD). The ability to perform organ transplants is among the benefits ofumbilical cord blood transfusion.Using stems cells is also one of thebest ways to treat blood diseases sincethe method has a success rate of 70%worldwide.

Storage of stem cells is a valuable investment. So far, 27 cord blood banks have been launched across the country. There are two types: public and private banks for stem cell storage. The former does not charge a fee for storage. But in the latter, the cost of collection and genetictesting is about $645 and the annualcharge for storage is $33, according toISNA.Iran is a leading country in biomedicalresearch. Researchers and physicians have been successfully performing bone marrow transplants during the past fewyears.Irans stem cell research is centeredat the Royan Institute for ReproductiveBiomedicine, Stem Cell Biology andTechnology, located in northern Tehran.

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Growth in Stem Cell Research - Financial Tribune