Brave Ava Stark has gone back to nursery for the first time since undergoing a life-saving bone marrow transplant.

The four-year-old was all smiles when she arrived at Noahs Ark Nursery in Lochgelly, Fife, yesterday morning.

She said she was looking forward to laughing at the nursery teacher and happily ran around the toy-filled garden before starting at 8am.

But her return was cut short after another child was ill and she had to go home due to her lowered immune system.

Mum Marie said that the half an hour she spent at the nursery was a great start and theyre now looking forward to her returning for longer.

The 34-year-old said: Its absolutely amazing that shes managed to get back to nursery for the first time. We honestly didnt think this day would ever happen.

My mum has been teaching her at home and I think shes going to miss her wee side-kick. It really is a big day.

She may only have been able to stay for half hour but thats a great start and were aiming for more next week. She was too excited to sleep last night.

If it wasnt for all those amazing people who heard about Ava and registered to become donors, then we may never have got here.

We just cant thank everyone who supported us enough.

Nursery manager Karen Robertson added: Its absolutely fantastic, we couldnt have wished for a better outcome.

We always said that when she took ill that we couldnt wait until she was well enough to come back and Im just delighted to see her.

Ava underwent her stem cell transplant in December after a Daily Record appeal which saw more than 83,000 people across the UK sign up to try help her.

She was first diagnosed with inherited bone marrow failure in April 2016 and relied on blood and platelet transfusions to keep her alive.

A matching donor was initially found but pulled out weeks before the procedure went ahead prompting her brave mum to launch the worldwide appeal for help.

A second match was then found but they pulled out just 24 hours before the youngster was due to go to hospital leaving her entire family devastated.

The campaign continued and two more matching donors were eventually found meaning she could undergo the operation in December.

She has recently celebrated her 100 day post-transplant milestone and will become the face of a donor recruitment drive by the Anthony Nolan charity.

She was also named one of the Daily Records Little Heroes at an award ceremony in May.

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Bone marrow transplant tot Ava Stark goes back to nursery for first time since live-saving op - Scottish Daily Record

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Gary Campbell's partner, Leanne Crawford, with helpers Lauryn MacKenna (9) and Kye Crawford (12) at the home baking stall at Sunday's fun day.PICTURE IAIN FERGUSON, THE WRITE IMAGE

Two fundraising events have been held in Lochaber at the weekend to help send a local man halfway round the world for ground-breaking treatment.

Gary Campbell, from Caol, who is just 29, was diagnosed with progressive MS in April.

But family, friends and supporters are pulling out all the stops to raise 45,000 in order to send him to Mexico for stem-cell treatment.

Hematopoietic stem cell transplantation, HSCT, involves the intravenous infusion of stem cells collected from bone marrow or peripheral blood.

On Saturday, youngsters and their parents took park in a teddy bear toddle to raise cash for Mr Campbells cause, while yesterday, a large crowd attended a fun afternoon at An Aird in Fort William.

Leanne Crawford, Garys partner, said: The fun day went really well and there was a good turnout.

There were lots of fund-raising activities including a charity shinty match, bouncy castle, beat the goalie, a nail bar, home baking and raffles with loads of prizes.

Gary used to play shinty and two of his old teams from Caol and Banavie took part. Some people had obviously not played in a wee while and were falling about, but it was great fun.

Unfortunately, Gary wasnt well enough to come along which was a pity as he would have really enjoyed it.

He had a fall in the garden on Saturday night and I couldnt get him up. Fortunately his mum was there and between us we managed to get him back into the wheelchair.

His right leg was shaking constantly it was really stressful for him.

Ms Crawford said she has known Mr Campbell for nine years, and for the past five has noticed different symptoms.

He wasnt very good on his feet and sometimes his legs would give way. It was as though he was drunk when he hadnt been drinking.

Gary actually thought he had a brain tumour and, in a way, it was a relief when he was diagnosed with MS.

Ms Crawford said she is hoping by the time enough cash is raised, HSCT might be available closer to home.

I believe hospitals in Galway and Sheffield are looking into the treatment, but if he has to go to Mexico, Ill get him on the plane even though he is petrified of flying.

I dont know how much has been raised yet from these events, but we collected 663 from a recent baking sale and have 2,500 already on the just giving page. We also have 1,500 in a bank account collected from fund-raising events.

Gary is over the moon with the support he is receiving.

Another Lochaber resident with MS, Frances OConnell, received HSCT in Mexico last year.

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Fun weekend activities will help send Lochaber man for ground-breaking MS treatment - Press and Journal

Bone Marrow Stem Cells Comments Off on Fun weekend activities will help send Lochaber man for ground-breaking MS treatment – Press and Journal | August 7th, 2017

Tom Parham remembers the time his Woodrow Wilson baseball team was playing Class AAA power Huntington East.

It was 1980, and the Flying Eagles were hosting the Pony Express at Harry Lewin Field. Not known to be a cavernous venue, the field lent itself to an offensive barrage and Huntington East was the last team standing.

A few weeks later, Parham led Woodrow to the state championship game and a rematch with the Express. Woodrow fell short again, but this time it was by the more purist-friendly score of 2-1.

It was then that Parham knew the Eagles needed a new field.

In stepped Doug Epling, Beckley businessman and community leader. He would later be known for refurbishing the old East Bank High School field for WVU Tech to use, as well as the construction of Linda K. Epling Stadium in Beckley, the home of the West Virginia Miners.

The latter, of course, bears the name of Epling's wife. The Tech field is named for Epling himself.The field he helped build on the Woodrow Wilson campus doesn't have an official name.

That will change Saturday.A ceremony will be held at 2 p.m. in the school cafeteria to officially rename the field for Thomas Parham.

The effort to honor the longtime coach was started by Sheila Brown.

"Words cannot describe how it feels," Parham said. "When Mrs. Brown started talking about it, I always told her, nah (modestly). I just thought, 'Let it go.' And finally she told me in April, 'Well, I'm going to the board. I'm going to ask them.' So she did and they told her what to do (at the next meeting)."

The Raleigh County School Board laid out a plan for Brown, and at the next meeting former coaches, colleagues and friends voiced their support.

Legendary boys basketball coach Dave Barksdale. State championship-winning football coach Pete Culicerto. Fellow New Hope Baptist Church member C.W. Claytor. Even Epling himself. They all showed up to see that Parham got the respect they feel he deserves.

"It was just touching to hear former coaches Coach Barksdale, Coach Culicerto, and I even heard from one of my coaching buddies from out of town, Ron Rose," Parham said. "He told Pete what (he wanted) to say. It was just touching, and a humbling experience."

Parham is being recognized for a career that spanned nearly three decades. He was hired as a biology teacher by Ross Hutchens before the start of the 1974-75 academic year.

"He said, 'I need a good biology teacher. I can get a coach anywhere,'" Parham said, laughing.

His first season as head baseball coach was 1975, and he remained there until his retirement in 2000. Along the way, his teams rolled up over 200 wins and appeared in the state tournament five times. Two of those trips resulted in runner-up finishes the 1980 meeting with Huntington East, and in 1983 against Martinsburg.

And the list of star players Parham coached seems endless Chuck Tate, Andy "Bam Bam" Wakefield, Larry Maiolo, Mason Basham, Larry Hickman, Joe Joe Maiolo, Larry Pat Farley, Phil Culicerto, Tim Epling, Phil Lane, Ronnie Fama, John O'Dell.

There were many others, and many of themare members of the Woodrow Wilson Baseball Hall of Fame.

"I was fortunate I came across some good ball players," Parham said. "You don't like to toot your own horn, but like a fella said, we put Woodrow Wilson baseball on the map."

Another was Ronnie Scott, who went on to work for NASA in Florida before returning to Beckley in 2010. Sadly, he passed away in May at age 59.

"He wanted to see baseball dominant again like it was when he played," Parham said.

When Parham retired from baseball in 2000 he stayed on as a biology teacher for one more year it was the emphatic end of an era at the school. Not only did Parham retire, but Culicerto retired after the 1999 football season, and Barksdale left the bench just months before Parham to take a coaching job in Aiken, S.C.

"Indeed it was," said Parham, now 74. "I enjoyed working with Coach Culicerto (as an assistant football coach). He was a great football coach, and he was a baseball supporter. He had seven sons play baseball for me. Three of them played in the state tournament."

After his retirement, Parham's was a familiar face in the stands at Woodrow baseball games. But in 2009, his ability to be a spectator slowed down when it was discovered that he had cancer.

Parham was diagnosed with multiple myeloma, a blood cancer that develops in the plasma cells located in bone marrow. The cancer did eventually go into remission, but Parham was still getting checkups when something told him he needed to go to Johns Hopkins in Baltimore.

It was there that he was introduced to autologous stem cell transplant. It's a procedure that involves collecting the patient's stem cells and following it up with high doses of chemotherapy or a combination of chemo and radiation. The process kills cancer cells while also killing blood-producing cells left in the bone marrow.

The collected stem cells are later transplanted back into the patient, allowing the marrow to produce new blood cells.

"I met a very interesting and a caring doctor up there, Dr. (Ivan) Borrello. He told me (about the transplant)," Parham said. "As a matter of fact, they have been doing this since 1980. He asked, 'What do you think about a stem cell transplant?' And I said yeah. Anything to get rid of this cancer.

"At that time my cancer was in remission, so he couldn't do anything. He said we would have to wait until it comes back. He hoped it didn't come back, but if it does ..."

It did, and in February he had the procedure performed.

"It came back in 2016, and when you're over 70 they don't usually do these things," Parham said. "But he felt like I was in good shape, which I think well, I know I am. I went through it, successful, no problems whatsoever.

"After teaching biology, I thought I knew some things. Now I know I know some things."

Just like baseball.

Email: gfauber@register-herald.com and follow on Twitter @GaryFauber

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Woodrow Wilson baseball field to be renamed for Tom Parham - Beckley Register-Herald

Bone Marrow Stem Cells Comments Off on Woodrow Wilson baseball field to be renamed for Tom Parham – Beckley Register-Herald | August 6th, 2017

Nalini Ambady, the first Indian-American woman to teach psychology at three major universities in the U.S., died in 2013 due to leukaemia when she was just 54.

For the medical fraternity in Kerala, her native place, it turned the spotlight on the lack of awareness of stem cell transplant, which could have saved her life.

Four years down the lane, doctors say the situation has changed only marginally, as many patients who require the treatment have not been able to do it because of high expenses, lack of matching donors, and lack of facilities at hospitals.

Doctors note that stem cell transplant is being proposed as an effective treatment for cancers such as leukaemia and lymphoma, and primary immune deficiency disorders. Stem cells do not develop normally in such patients and it affects the blood cells that they make. By a transplant, the patient gets new stem cells that can make new and healthy blood cells. Earlier, stem cells were collected from the bone-marrow. Now, it is being collected from blood cells.

Neeraj Sidharthan, bone marrow transplant physician at Amrita Institute of Medical Sciences, Kochi, told The Hindu that in Prof. Ambadys case, though matching donors were found, they had all dropped out.

Lack of awareness is still a major issue though there are some positive signs. In some cases, because of lack of infrastructure, cancer cases are not being diagnosed early and treatment is delayed too, he said.

Ajith Kumar V.T., professor, department of paediatrics, Government Medical College, Manjeri, said donors could not be found often from the same families because of the nuclear family system.

There are not many places where you can match the human leukocyte antigen (HLA) typing with donors. Another problem is the lack of stem cell registries in the State from where matching unrelated donors could be found.

Even if doctors suggest a stem cell transplant, many families dont opt for it because of the high cost involved. If the donor is from the same family, the cost is relatively low. But for unrelated donors, it is very high, Dr. Sidharthan said. The solution, Dr. Ajith Kumar said, was government intervention to set up HLA registries and bone marrow transplant centres. nestCare Foundation, a not-for-profit organisation based in the U.S., had recently approached us expressing interest to set up these facilities in the State. Talks are on, he said.

A.S. Jayanth

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Trust those cells to help cure cancer - The Hindu

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In 2002, Ian Thompson, a specialist in facial reconstruction at Kings College, London, received an urgent phone call. A patient in his late 20s had been struck by an out-of-control car mounting the pavement. The impact had sent him catapulting over the bonnet of the car, smashing his face and shattering the fragile orbital floor the tiny bone, no more than 1mm thick, which holds the eyeball in place in the skull.

Without the orbital floor, your eye moves backwards into the skull, almost as a defensive mechanism, Thompson explains. But this results in blurred vision and lack of focus. This patient had also lost the ability to perceive colour. His job involved rewiring aircraft and as he could no longer detect a red wire from a blue one, hed barely been able to work in three years.

The accident had happened three years earlier. Since then, surgeons had desperately tried to reconstruct the bony floor and push the eye back into position, first using material implants and then bone from the patients own rib. Both attempts had failed. Each time, infection set in after a few months, causing extreme pain. And now the doctors were out of ideas.

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Thompsons answer was to build the worlds first glass implant, moulded as a plate which slotted in under the patients eye into the collapsed orbital floor. The idea of using glass a naturally brittle material to repair something so delicate may seem counterintuitive.

But this was no ordinary glass.

If you placed a piece of window glass in the human body, it would be sealed off by scar tissue, basically wobble around in the body for a while and then get pushed out, says Julian Jones, an expert in bioglass at Imperial College London. When you put bioglass in the body, it starts to dissolve and releases ions which kind of talk to the immune system and tell the cells what to do. This means the body doesnt recognise it as foreign, and so it bonds to bone and soft tissue, creating a good feel and stimulating the production of new bone.

Bioglass actually works even better than the patients own bone Ian Thompson

For Thompson, the results were immediate. Almost instantaneously, the patient regained full vision, colour and depth perception. Fifteen years on, he remains in full health.

Thompson has gone on to use bioglass plates to successfully treat more than 100 patients involved in car or motorcycle accidents. Bioglass actually works even better than the patients own bone, Thompson says. This is because weve found that it slowly leaches sodium ions as it dissolves, killing off bacteria in the local environment. So, quite by chance, you have this mild antibiotic effect which eliminates infections.

Cutting edge

Bioglass was invented by US scientist Larry Hench in 1969. Hench was inspired by a chance conversation on a bus with an army colonel who recently had returned from the Vietnam War. The colonel told Hench that while modern medical technology could save lives on the battlefield, it could not save limbs. Hench decided to shelve his research into intercontinental ballistic missiles and instead work on designing a bionic material which would not be rejected by the human body.

Hench ultimately took his research to London, and it has been in Britain where some of the most revolutionary bioglass innovations are being made in fields from orthopaedic surgery to dentistry.

Over the last 10 years, surgeons have used bioglass in a powdered form, which looks and feels like a gritty putty, to repair bone defects arising from small fractures. Since 2010, this same bioglass putty has hit the high street as the key component in Sensodynes Repair and Protect toothpaste, the biggest global use of any bioactive material. During the brushing process, the bioglass dissolves and releases calcium phosphate ions which bond to tooth mineral. Over time, they slowly stimulate regrowth.

But many scientists feel that the current applications of bioglass are barely scratching the surface of what could be possible. New clinical products are being developed which could revolutionise bone and joint surgery like never before.

Sitting in his office in Imperial Colleges Department of Materials, Jones is holding a small, cube-shaped object hes dubbed bouncy bioglass. Its similar to the current bioglass but with a slight twist: subtle alterations in the chemical composition mean its no longer brittle. Instead it bounces,like a kids power ball as Jones describes it, and its incredibly flexible.

The point of this is that it can be inserted into a badly broken leg and can support both the patients weight and allow them to walk on it without crutches, without requiring any additional metal pins or implants for support. At the same time, the bouncy bioglass also will stimulate and guide bone regrowth while slowly, naturally assimilating into the body.

To regenerate large pieces of bone, for example in a really big fracture, its very important to be able to put weight on your leg, Jones says. And its really important that the bio-implant in your leg is able to transmit the force from your weight to the bone cells, like a signal. Our body makes its own bone in the architecture that its in, because the cells feel the mechanical environment. So to grow back a big piece of bone you need to be able to transmit the right signals to them. The reason why astronauts in space lose bone mass is because without gravity, the cells arent receiving the same information as they do on Earth.

Further alterations to the chemical makeup of bioglass produce a different form which is much softer and has an almost rubbery feel. It feels almost like a piece of squid at a seafood restaurant. This bioglass is designed for possibly the holy grail of orthopaedic surgery: cartilage repair.

Right now, surgeons attempt to repair damaged cartilage in arthritic hips or damaged knee joints with a fiddly procedure called microfracture. This involves smoothing over the damaged area to expose the bone underneath, then pricking it to release stem cells from the bone marrow which stimulate repair. But this results in scar cartilage and within a few years, as many athletes have found, the original problem returns.

As a solution, Jones is looking to produce bioglass which can be 3D-printed and then slotted into any hole in the cartilage. For the cells to accept it, the material must retain all the natural properties of cartilage. To test its effectiveness, Jones uses a simulator that has human knee joints from cadavers donated for medical research.

We simulate the walking action, bending, all the things a knee would do, and make sure that the bioglass actually preserves the rest of the joint and behaves as it should do, he says. If that works then well proceed to animal and then clinical trials.

This same bioglass could find an additional use in aiding people with chronic back pain due to herniated discs. At the moment surgeons treat this by replacing the dysfunctional disc with a bone graft which fuses the vertebrae in the back together. But while this takes away the pain, it results in a considerable loss in mobility. Instead, a bioglass implant could be printed and simply inserted to replace the faulty disc.

It seems the obvious thing to do, Jones says. So far nobody has been able to replicate the mechanical properties of cartilage synthetically. But with bioglass, we think we can do it.

Weve just got to prove that we can. If all goes well and we pass all the necessary safety tests, it could reach the clinic in 10 years.

Using man-made materials which can fuse to the body may seem far-fetched but it is appearing to be a more and more likely component of future medicine. Already, millions of people brush their teeth with it. And that may just be the start.

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The best way to fix broken bones might be with glass - BBC News

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BINGHAMTON (WBNG) -- Thursday, a cancer survivor met her bone marrow donor for the first time, just days before her wedding.

"They told me that without a transplant I really only had about six months to a year," said Vivian Nolan, a bone marrow transplant recipient.

In 2008, Vivian Nolan was diagnosed with a rare form of cancer called multiple myeloma. Later on, she was diagnosed with leukemia.

Doctors tried a bone marrow transplant with her own stem cells. When that didn't work, they said she needed a donor.

"The only cure or chance of holding it off at all is a bone marrow transplant," Nolan said.

Lucky for Nolan, doctors found a match.

A stranger volunteered to save her life. Scott Durbin is Nolan's donor. He lives in Kentucky, over 850 miles away.

Thursday, Durbin and Nolan met for the first time.

Nolan is getting married on Saturday.Durbin and his family flew in to support her in her next phase of life, a life that she wouldn't have without him.

"This is the man who gave me my life back. So I'm really happy," Nolan said.

For Durbin, the decision to help someone in need was second nature.

"I signed up. 7 months later I got that phone call saying they was gonna fly me to Atlanta," bone marrow donor Scott Durbinsaid.

Nolan was still in shock that someone would do something so kind for a person he had never met.

"I just couldn't believe that there was someone out there that I never knew that would go through that for me," she said.

After the transplant, Nolan wanted to meet the man who now is a part of her.

Today, she was able to introduce her family to its newest member.

"Now I've got this whole new life and he's got this whole big new family."

For Durbin, it's a choice he'd make over and over.

"I would do it again to give you a second chance," Durbin said.

Nolan remains forever grateful for that second chance.

Since her bone marrow transplant, Nolan's leukemia is virtually gone. She says she feels great, and can't wait for her new lease on life.

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Cancer survivor meets bone marrow donor days before wedding - WBNG-TV

Bone Marrow Stem Cells Comments Off on Cancer survivor meets bone marrow donor days before wedding – WBNG-TV | August 5th, 2017

The excitement at Jerusalem-based Gamida Cell, a maker of cell and immune therapy technologies, is palpable.

The biotechnology company has started enrolling patients for a last-stage clinical trial for a drug it believes will help increase the success of bone marrow transplants in blood cancer patients, and help them better withstand the ordeal of the lifesaving procedure.

The patients are being enrolled in the US, Spain, The Netherlands and Singapore. Should the results of the trial, as hoped, be positive, that would lead to the launch of a commercially available product in 2020, Gamida Cells CEO Yael Margolin said in an interview with The Times of Israel.

We are at an exciting transition point, and moving from being a research and development firm, based in Israel, to an international commercial firm, said Margolin who has headed the company for the past 12 years in her sun-drenched office at the biotech firms headquarters in Jerusalem. We need to prepare to commercialize the product. We are now looking at various sites in Israel for a new manufacturing facility and looking to employ some 100 people. These workers will be added to the 40 already employed in Jerusalem.

Gamida Cells CEO, Dr. Yael Margolin (Courtesy)

Preliminary clinical data has already revealed that the risk of their leading product for blood cancers, NiCord, not meeting its targets in the Phase 3 trial, is low, added Margolin.

The drug has already received a breakthrough therapy designation by the US Food and Drug Administration (FDA). The designation is given to a drug that is meant to treat a serious or life-threatening condition, and where preliminary clinical evidence indicates that it may demonstrate a substantial improvement on at least one clinically significant target (endpoint) over other available therapies. The designation also entitles the company to get more and closer FDA guidance to help bring the treatment faster to patients.

The combination of the low clinical risk based on the previous trial results and the lower regulatory risk, because the drug is being developed in close collaboration with the FDA, has spurred the company into a flurry of activity that is aimed at scaling up its production facilities to get ready for the day NiCord hits the markets.

The company said last month it raised $40 million from investors including Novartis, which is already a major shareholder in the firm. The funds will support the ongoing Phase 3 stage for NiCord. The company also announced, on July 20, that it received a $3.5-million grant from the Israeli government that will support the further development of NiCord and other drugs that the company is developing, including therapies for sickle cell disease and for blood and solid cancers. Gamida has also appointed a new chief medical officer, Ronit Simantov, who will be based in the US.

The first market for our drug will be the US, Margolin said.

The Gamida Cell lab in Jerusalem where umbilical cord blood is stored in tanks, July 16, 2017. (Shoshanna Solomon/Times of Israel)

NiCord, which would be the first drug developed by Gamida to hit the market if the trial goes well is believed to increase the chances of a successful bone marrow transplantation process for patients who do not have a rapidly available, fully matched, bone marrow donor.

Today some high-risk blood cancers cannot be cured unless patients undergo a bone marrow graft. For that purpose, a perfect 100-percent match needs to be found, a process that in the US takes an average of three to four months, if the patient is lucky. Sometimes, no match is found.

There are 70,000 patients a year globally with blood cancers who need a bone marrow transplant, Margolin said. It is a rare condition. But for that transplant, you need a donor with full tissue matching. As many as 50% dont get to the transplant phase, because they havent found a matching donor in time.

Umbilical cord blood collected from newborn babies contains stem cells, which can be used to treat diseases. Today cord-blood banks around the world store the cord blood. It great advantage is that because it is so young, there is no need for a full tissue matching.

The big advantage with umbilical blood is that you dont need full tissue matching; a partial match is enough, Margolin continued. Most patients generally find at least one unit of cord blood that partially matches them.

Stem cells in a bag in Gamida Cells Jerusalem lab, July 16, 2017 (Shoshanna Solomon/Times of Israel)

The problem is that the quantity of cells in each unit is not huge, and it is the number of stem cells in the cord blood that is critical to the success of transplantation.

Our idea is to leverage the advantages of the cord blood and overcome the limitations of the cell number by applying our own platform technology, called NAM Technology, added Margolin. This technology allows us to take one unit of umbilical cord blood and expand the number of stem cells within it and enhance their performance.

Gamida Cell selects the stem cells from the unit and puts them in a culture together with a molecule called Nicotinamide (NAM) a form of Vitamin B3 and adds other ingredients. This culture, to which the firm holds intellectual property rights, increases the number of stem cells, and enhances their functionality, Margolin said.

The cells are then harvested from the culture, frozen in a small blood-bag in a final formulation that is ready for infusion, and then shipped to hospitals via couriers. Doctors thaw the product by the bedside of the patients and infuse the fluid into them.

From start to finish, our process takes three weeks, Margolin said. The average search for a bone marrow match takes three to four months.

The clinical trial underway is enrolling patients aged 16 years and older.

An earlier trial of the drug showed that patients transplanted with NiCord showed a more rapid engraftment the amount of time needed for the development of a minimal amount of white blood cells, or neutrophils, in the blood. That minimum amount indicates the patient is now less vulnerable to infections and bleeding following the transplant, and is an indication of success.

In the pilot phase clinical trials, the median time to neutrophil engraftment with NiCord was 11 days, compared to three to four weeks in patients who received standard umbilical cord blood. The results in a study conducted at Duke University also showed a lower rate of infection 22% vs 54%; and a lower duration of hospitalization compared to standard umbilical cord engraftment, Margolin said.

Now the company is enrolling patients for its larger, Phase 3 multi-national, randomized controlled registration study. And in February it said it had already transplanted its first patient, as part of the trial.

We hope to publish positive topline data from the Phase3 study in the first half of 2019 and launch the product on the market in 2020, she said.

Metal barrel with a frozen bag of umbilical cord stem cells ready for delivery from Gamida Cells Jerusalem lab, July 16, 2017. (Shoshanna Solomon/Times of Israel)

A metal barrel within which was a frozen bag of umbilical cord stem cells was waiting to be picked by a courier in the lobby of the Gamida Cell offices, ready to be thawed and injected into a patient somewhere around the world.

We have a sophisticated infrastructure that coordinates everything between the cord bank blood and our manufacturing site and the hospital where the patient is to be treated, Margolin continued. This infrastructure is 100% robust, but we plan to scale this up toward commercialization.

The $40 million in funds the company raised last month is expected to last until late 2019. After that, she added, all options are on the table: an IPO, or teaming up with a strategic partner, are both possibilities for the future.

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Cell therapy firm in flurry of activity as hope nears for bone marrow patients - The Times of Israel

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Marie Grim of Langley is looking for 100 people between the ages of 17 and 35 who are willing to take a few moments to have their cheeks swabbed.

Theres no pain, no fuss, she said.

And you could save someones life, anywhere in the world.

You could match with someone in Japan.

The campaign to sign up more potential stem cell donors, people who are willing to allow DNA samples to taken using cotton swabs, was inspired by the experience of her sister-in-law.

Cloverdale resident Tania Grim, a mother of four was diagnosed with leukemia in January.

She had to wait several months before a compatible donor was found whose stem cells will be used to replace bone marrow and abnormal white blood cells eradicated by a combination of chemotherapy and radiation.

We have been on quite the journey, Marie said.

I have sat with her at appointments and heard others get news of their donor while she had not.

Now that Tania has her donor, Marie would like to improve the odds for other families.

She already has a location and tentative date to collect the swabs September 8 at Immanuel Christian Reformed Church in Langley if she can round up enough donors.

Tania, who is preparing for her stem cell procedure in September, urged prospective stem cell contributors to sign up.

I am so grateful that the word is being spread about the huge need for donors, Tania said.

It is a very simple thing to do that can save a life.

If you are the right age to be a donor, you can contact Marie at 604-530-1326 or by email at mariegrim@hotmail.com.

Interested donors can also contact Canadian Blood Services directly at https://blood.ca/en.

More than 390,000 Canadians have joined the OneMatch Stem Cell and Marrow Network registry maintained by Canadian Blood Services, volunteering to be stem cell donors for any patient in need of a transplant, anywhere in the world.

Right now, the agency says about 70 per cent of eligible donors on the registry are Caucasian, which means the odds of finding match for other ethnicities, such as Canadians with indigenous, Asian or African heritage, are not good.

The Canadian registry connects to an international network established by the World Marrow Donor Association (WMDA) that has access to over 28 million donors in over 53 countries.

Not everyone who registers will be matched to a patient and asked to donate, but each registrant provides hope for those waiting, a message posted to the agency website states.

A person could be a match within a few months of registering, a year later or even seven years later.

If a volunteer donor is found to be a match, they face a relatively minor surgical procedure and can expect to make a quick recovery.

The agency says over 80 diseases and disorders can be treated with a stem cell transplant.

There are hundreds of patients in Canada waiting for a match, but only half of them find a match.

Patients are more likely to find a matching donor from within their own ethnic group.

The odds of family members matching is extremely slight, the agency said, which is why it does not support donor drives targeting relatives.

RELATED STORY: Surrey teen rallies stem cell donors to help with desperate need for South Asians

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Drive for stem cell donors in Langley - Surrey Now-Leader

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Credit: University of California, San Francisco

A virus hiding quietly in the gut may trigger the onset of a severe complication known as graft-versus-host disease (GvHD) in patients who receive bone marrow transplants, according to a new study led by scientists at UC San Francisco and Saint-Louis Hospital in Paris, France.

GvHD affects up to 60 percent of patients who undergo bone marrow stem-cell transplants, and kills about half of those affected. After transplants, to prevent a recipient's immune cells from laying siege to unfamiliar donor cells and rejecting them, clinicians often use drugs to suppress the immune response. GvHD is a mirror image of organ rejection, in which immune cells in the transplant attack its new host, the patient.

Despite the pervasiveness of this disease, there isn't yet a clear way of foretelling patients' risk of developing it before they go into surgery. The new study, published online July 31, 2017, in Nature Medicine, unveils a viral biomarker that could allow clinicians to assess patients' risk of an acute form of the disease known as enteric GvHD, which affects the gastrointenstinal system.

The team used a technique known as metagenomic next-generation sequencing (mNGS) which can rapidly and concurrently sequence genetic material of all organisms present in any biological sample to catalog microbes in patients' digestive tracts, monitoring the evolving bacterial and viral population throughout the transplantation process.

Although mNGS analyses of bacterial populations, called microbiomes, have been much in the news, fewer studies have focused on "viromes," the term for viral populations.

"Viromes can play an important part in health and disease," said Charles Chiu, MD, PhD, an associate professor of laboratory medicine at UCSF and principal investigator of the study. "Our goal was to understand what impact transplantation has on the gut virome."

In the new work, the researchers scanned stool samples taken from 44 patients before they received a transplant and up to six weeks after, and sequenced all the DNA and RNA in the samples in order to assemble a roster of their microbial passengers.

Using this technique, the researchers identified a number of viruses that flared up in the guts of patients who developed the deadly condition. Of particular note were members of the picobirnavirus (PBV) family: the presence of these viruses before transplantation, even in very small populations, was a reliable sign that a patient would likely develop the disease after a transplant.

"I would've expected herpesviruses or adenoviruses to be the more likely cause of infection," said Chiu. "We wouldn't have picked up picobirnaviruses were it not for the metagenomics approach."

PBVs are a very diverse family of viruses more diverse than HIV, said Jrme Le Goff, PhD, associate professor at the University of Paris Diderot and lead author of the new study. "It's very difficult to design a single test to detect all viruses simultaneously," said Le Goff. "So for many years, labs did not have the means to look for PBV." Indeed, each of the 18 patients who tested positive for PBV was carrying a different strain, a diversity that makes it challenging to detect PBVs using a simple lab test.

The team also observed a previously unreported "bloom" of other resident viruses in patients that occurred three to five weeks after they had received transplants. Intriguingly, the onset of GvHD appeared to trigger the late awakening of these covert viruses, laying to rest a longstanding chicken-and-egg debate: which comes first, viral infection or GvHD? The researchers conclude that much of the viral flare they saw is due to reactivation of latent gut infections following transplantation.

Given the potential utility of PBV as a predictive biomarker, Chiu and his team now hope to develop a metagenomics-based test to screen patients before transplantation. "We also saw shifts in the microbiome but those in the virome were more pronounced," said Chiu. "Loss of bacteria colonizing the gut has been thought to predispose patients to GvHD; here we show that shifts in the virome may also play a role in the occurrence of this disease."

Although the new study strongly implicates PBVs in the onset of GvHD, it is too early to tell whether or how these viruses trigger the disease. The team is now enrolling more adult and pediatric patients both in Paris and at UCSF to expand their analyses and uncover the mechanism by which the virus modulates the risk of disease. A systematic understanding of the virus's role could ultimately inform whether using antiviral drugs or tweaking the body's immune response would be the best strategy to temper the disease.

"It would be great to have a tool that can be used to assess GvHD risk in these patients before they undergo a transplant," Chiu said, a step that Le Goff said could lead to new therapies. "We hope that in the next few years we will find a way to prevent virus-associated GvHD," said Le Goff.

Explore further: Researchers develop new strategy to limit side effects of stem cell transplants

More information: Jrme Legoff et al. The eukaryotic gut virome in hematopoietic stem cell transplantation: new clues in enteric graft-versus-host disease, Nature Medicine (2017). DOI: 10.1038/nm.4380

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Gut viruses tied to potentially deadly complication of bone marrow transplant - Medical Xpress

Bone Marrow Stem Cells Comments Off on Gut viruses tied to potentially deadly complication of bone marrow transplant – Medical Xpress | August 1st, 2017

BrainStorm Cell Therapeutics of Petah Tikva is recruiting American patients for a Phase 3 clinical study of its NurOwn stem-cell treatment intended to halt progression of amyotrophic lateral sclerosis (ALS).

The announcement was made in a patient webinar last week.

The NurOwn platform grew out of a technique developed at Tel Aviv University for growing and enhancing stem cells harvested from patients own bone marrow. The enhanced cells, injected via lumbar puncture, secrete elevated levels of nerve-growth factors believed to protect existing motor neurons, promote motor neuron growth and reestablish nerve-muscle interaction.

A 24-week Phase 2 safety study was concluded in 2016 on 48 participants (36 treated, 12 placebo) with possible, probable and definite ALS. This study was done at the University of Massachusetts Medical School, Massachusetts General Hospital and the Mayo Clinic.

The Phase 3 double-blind, placebo-controlled study, to begin enrollment in August, will look at efficacy and safety of repeated doses. The California Institute for Regenerative Medicine has awarded Brainstorm a $16 million grant to support the pivotal trial.

This study will accept 200 randomized study participants between the ages of 18 and 60 (half getting the treatment and half a placebo) at the three previous centers as well as California Pacific Medical Center in San Francisco, UC-Irvine near Los Angeles and another site not announced.

Potential participants must live within about 100 miles of one of the centers for ease of follow-up. They will receive three doses over a 16-week treatment phase and then undergo 28 weeks of follow-up.

BrainStorm President and CEO Chaim Lebovits said he hopes to get approval by the end of the year for a hospital exemption program in Israel an accelerated regulatory pathway that would clear the way for a first batch of 50 patients to receive NurOwn at Tel Aviv Sourasky Medical Center. However, there will be no compassionate treatment using NurOwn in Israel or elsewhere.

The NurOwn platform technology also has potential applications in any neurodegenerative disease, such as multiple sclerosis and Parkinsons.

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JoAnne Viviano The Columbus Dispatch @JoAnneViviano

Dennis Matko was headed for a knee replacement when he discovered a new therapy that would instead inject his own stem cells and plasma into the joint to help prevent degradation.

The 69-year-old Clintonville resident said he had been pretty active in his 50s, leading to problems with the right knee. He eventually had his meniscus removed. He had been through physical therapy, cortisone shots and gel injections, but the pain persisted.

The therapy, he said, was a no-brainer. He was sold because the procedure involved putting his own fluids into his body with no foreign objects and no drugs.

Dr. Joe Ruane, the orthopedic doctor who treated Matko, introduced the therapy at OhioHealth, but there are a number of places using the therapy around the state and country.

It's used to treat people with osteoarthritis, the type of arthritis caused by wear and tear.

Ruane said that the need for total knee replacements in the U.S. is expected to climb by 600 percent in the next 20 years, and there is concern that there might not be enough surgeons to perform the procedures.

We need an alternative, and patients are looking for alternatives, and given the choice between a knee replacement and an injection, many patients would choose an injection, he said.

The treatment involved removing Matkos bone marrow from the back of his pelvic bone, a process done in the office under general anesthesia. The marrow was then processed to form a concentrate of stem cells and other growth factors.

Matko also had blood drawn to create platelet-rich plasma, which acts as a signaling system to get the stem cells to respond.

Ruane injected both components into the knee, delivering more than 100 stimulating and growth factors to the joint.

Ruane said the process inhibits irritating chemicals that contribute to inflammation, decreases the activity of enzymes that break down cartilage, and helps the knee to make some of its own joint fluid again.

And, to a small degree, it does help regrow some of the tissue in the knee that has been destroyed by the arthritis, Ruane said.

The procedures are most effective in young patients with early arthritis, said Dr. Adolph Lombardi of Joint Implant Surgeons in New Albany, where stem-cell and platelet-rich plasma injections are offered as separate therapies. It won't help with bone-on-bone disease, he said.

While other injections might offer short-term pain relief, platelet-rich plasma has been shown to offer a full year of relief, said Lombardi, who works with the Mount Carmel Health System. The idea is that bone-marrow stem cells, when injected into a hip or knee, can differentiate into cartilage cells and help with regeneration.

"All of this is very new but it seems to be extremely promising," Lombardi said. "This is using their own bodies' healing potential to maintain cartilage and relieve pain."

Dr. Michael Baria performs the procedure at Wexner Medical Center at Ohio State University, where the bone-marrow and platelet-rich plasma injections also are offered as separate treatments. He agreed that the hope with the bone-marrow injections is that the stem cells turn into cartilage cells, improving or halting the osteoarthritis disease.

But in his experience, the treatment is helpful for patients with advanced disease.

"The most common patient we see for this is going to be in late-stage arthritis, so kind of at the end of their rope," Baria said. "Platelet-rich plasma is usually not as good for bone-on-bone arthritis. Bone marrow doesnt seem to be limited by bone on bone."

The body has trouble healing arthritis because cartilage doesnt get enough blood supply, Ruane said. Injecting the stem cells boosts the bodys own process.

While platelet-rich plasma has been shown to decrease inflammation, stem-cell use is newer and has yet to be proven effective, Baria noted.

OhioHealth andJoint Implant Surgeons are currently in the midst of controlled randomized trials, hoping to prove the effectiveness of the procedures and obtain approval from the U.S. Food and Drug Administration.

Unless that happens, the procedure will be considered experimental, and insurance doesnt cover costs. Matko paid $2,800 for the injections at OhioHealth.

Before the treatment, Matko was having trouble with mundane things like going up and down stairs and with other activities, such as taking hikes or walks with his wife or working out. A retired police officer, he now works as a business consultant and spends a lot of time on his feet, so he was looking for better mobility there as well.

Matko said the injections have helped his knee, which is getting progressively better over time. He said hes been able to increase his activity, getting back to the gym and taking hikes and walks. He has minimal pain climbing stairs and hes more comfortable in his work.

Im not saying its all better but its much better, Matko said. Its headed in the right direction.

He realizes the treatment is not a cure.

Im not looking for a miracle, he said. I just want to forestall problems as long as possible.

.

.

jviviano@dispatch.com

@JoAnneViviano

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Patients' plasma, stem cells help knee problems - The Columbus Dispatch

Bone Marrow Stem Cells Comments Off on Patients’ plasma, stem cells help knee problems – The Columbus Dispatch | July 31st, 2017

Regenerative medicine is a revolutionary approach to treating many degenerative conditions and includes a variety of different techniques including stem cell therapy. This field joins nearly all disciplines of science and holds the realistic promise of repairing damaged tissue by harnessing the bodys ability to heal itself.

Adult stem cells are found in every part of the body and their primary role is to heal and maintain the tissue in which they reside. Stem cells are unspecialized cells capable of renewing themselves by cell division. In addition, they have the ability to differentiate into specialized cell types. Adult stem cells can be harvested from a patients own tissue, such as adipose (fat) tissue, muscle, teeth, skin or bone marrow.

One of the most plentiful sources of stem cells in the body is the fat tissue. In fact, approximately 500 times more stem cells can be obtained from fat than bone marrow. Stem cells derived from a patients own fat are referred to as adipose-derived stem cells. The mixed population of cells that can be obtained from fat is called a stromal vascular fraction (SVF). The SVF can easily be isolated from fat tissue in approximately 30-90 minutes in a clinic setting (under local anesthesia) using a mini-lipoaspirate technique. The SVF contains a mixture of cells including adipose-derived stem cells or ADSCs and growth factors and has been depleted of the adipocyte (fat cell) population.

ADSCs are multi-potential and can differentiate into a variety of different types of tissue including but not limited to bone, cartilage, muscle, ligament, tendon and fat. These cells have also been shown to express a variety of different growth factors and signaling molecules (cytokines), which recruit other stem cells to facilitate repair and healing of the affected tissue. ADSCs are very angiogenic in nature and can promote the growth of new blood vessels.

Based on research performed in our FDA registered facilities, stem cell quality and functionality can vary greatly depending on the methods utilized to obtain the cells. It is important to utilize a product that has undergone full characterization to include safety, identity, purity and potency. We have developed a method for harvesting and isolating stem cells from fat for therapeutic use. The use of a cell population that retains the ability to function in vivo will lead to more consistent patient results with long term success.

Adipose stem cells can be obtained from the patient easily, abundantly, and with minimal patient discomfort. Clinical applications for patients can be performed in an office setting safely, legally, and ethically using autologous ADSCs. Current applications include orthopedic conditions (tendon/ligament injuries, osteoarthritis, etc.), degenerative conditions (COPD, diabetes), neurological (MS, Parkinsons, spinal cord injuries, TBI, etc.) and auto-immune (RA, Crohns, colitis, lupus).

Stem cells possess enormous regenerative potential. The potential applications are virtually limitless. Patients can receive cutting edge treatments that are safe, compliant, and effective. Our team has successfully treated over 7000 patients with very few safety concerns reported. One day, stem cell treatments will be the gold standard of care for the treatment of most degenerative diseases. We are extremely encouraged by the positive patient results we are seeing from our physician-based treatments. Our hope is that stem cell therapy will provide relief and an improved quality of life for many patients. The future of medicine is here!

For additional information on our South Miami clinic, visit http://www.stemcellcoe.com.

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When the Japanese researcher Shinya Yamanaka managed to reprogram adult cells into an embryonic-like state to yield induced pluripotent stem cells (iPSCs), this was supposed to herald a revolution in regenerative medicine. But 10 years after their discovery, a therapeutic breakthrough is still outstanding.

The overall stem cell therapy field has failed today to show a very clear cut clinical benefit, told me Georges Rawadi, VP for Business Development at Celyad. The field now needs some significant success to attract attention.

Even though investors prefer placing their bets on the hot T cell therapies these days, some stem cell technologies such as iPSCs are starting to get traction as big industry players are exploring the territory. Last year, Bayer and Versant threw $225M into the pot to launch BlueRock Therapeutics, a regenerative medicine company that plans to develop iPSC-based therapies. A year before, Fujifilm spent $307M to acquire the iPSC company Cellular Dynamics.

Although a big success story is still lagging behind, recent advances in the field argue that stem cells indeed have the potential to translate into effective therapies for currently intractable diseases. Heres an overview of what biotechs stem cells are up to!

Stem cell treatment is not a new concept hematopoietic stem cells (HSCs) were described as early as the 1960s and bone marrow transplants have been used to treat blood cancer for decades.

The reason that we get excited about stem cell therapies comes from our experience with the hematopoietic stem cells. If you want to see what a mature stem cell therapy is like, you only need to look at bone marrow transplantation explained James Peyer, Managing Partner at Apollo Ventures, who has a Ph.D. in stem cell biology.

According to Peyer, the hematopoietic stem cell field is one of the most active areas in the stem cell world right now, mainly fueled by our advances in the gene editing space. Tools like CRISPR and TALEN allow for the genetic modification of a patients own bone marrow stem cells, which can then be expanded and returned to the patient for the correction of a genetic defect.

Last year, regulators gave green light to one of the first therapies of this kind. Strimvelis, developed by GSK, consists of an ex vivo stem cell gene therapy to treat patients with the very rare type of Severe Combined Immunodeficiency (SCID). Using the patients own cells avoids the risk of graft versus host disease (GvHD), which still affects around 30% of people receiving a bone marrow transplant.

Small wonder that the CRISPR companies, CRISPR Therapeutics, Editas, and Intellia are all active in this field, with preclinical programs in a number hematological diseases.

To date, the most prominent stem cells in the clinic are mesenchymal stem cells (MSCs), which are moving through more than 300 registered clinical trials for a wide array of diseases. These cells are able to form a variety of tissues including bone, cartilage, muscle or fat, and can be readily harvested from patients or donors for use in autologous or allogeneic therapies.

While MSCs have deluded the biotech scene with good safety profiles in clinical trials, their actual regenerative potential remains controversial, and there have been a great number of clinical failures, which many blame on a lack of demonstrated mechanisms of action.

As Peyer explained, The problem here is that, as opposed to other adult stem cells, the MSC has been unclearly defined. We know roughly what it does but we dont fully understand the molecular mechanisms driving these cells. On top of being unclearly defined, the regenerative powers of MSCs have been massively over-claimed in the past.

Another reason for the lack of clinical benefit has also been attributed to the use of undifferentiated MSCs, as Rawadi explained to me. The Belgian biotech Celyad, which has been pioneering cell therapy in the cardiovascular space, is using bone-marrow derived autologous MSCs and differentiates them into cardiomyocyte precursors to produce new heart muscle in patients with heart failure.

Although the company missed its primary endpoint in a phase III trial last year, Celyad has staked out a patient subpopulation that showed significant improvement. Its technology still has the confidence of the FDA, which just handed out a Fast Track designation and Celyad is now planning a refined Phase III trial.

One of Celyads major competitors, Australian Mesoblast, is forging ahead using allogeneic MSCs with Phase III programs in heart failure, chronic low back pain (CLBP) due to disc degeneration, as well as a range of inflammatory conditions including GvHD and rheumatoid arthritis.

Although the ability of MSCs to regenerate tissues remains questionable, the Mesoblasts approach hinges on a body of evidence showing that MSCs can suppress inflammation and mobilize endogenous repair mechanisms through indirect effects on immune cells.

Indeed, the first-ever approved stem cell therapy, Prochymal, also depends on this mechanism. Prochymal was developed by US-based Osiris Therapeutics and in 2012 received Canadian approval to treat acute GvHD. But after Sanofi opted to shelve its partnership with Osiris prior to FDA approval, the biotech sold out its off-the-shelf stem cell platform to Mesoblast in a $100M deal.

In Belgium, companies like TiGenix and Promethera are also banking on the immunomodulatory properties of MSCs. The companies are developing treatments for patients with Crohns disease and liver diseases, respectively.

The ultimate hope for stem cell therapies has been to regenerate damaged or diseased tissues as found in diabetes, heart failure or blindness. Holostem Terapie Avanzate, a spin-off from the University of Modena and Reggio Emilia was the first company to move towards this goal.

Building on 20 long years of research, the biotech has developed Holoclar, the first and only autologous stem cell therapy (apart from bone marrow transplants) to enter the European market. Holoclar is based on limbal stem cells, located in a part of the eye called the limbus, which can be used to restore eyesight in patients that have lost sight due to burn injuries.

Meanwhile, UK-based Reneuron is developing off-the-shelf therapies that aim to restore the cognitive function of patients following a stroke. Backed by no other than Neil Woodford, the company recently raised an impressive 100M to advance its lead therapy to the market.

The biotechs fetal-derived neural stem cell line CTX was able to significantly reduce the disability of post-stroke patients in a Phase II trial and ReNeuron is now planning to push its candidate into pivotal trials.

A major question in the space a decade ago was safety. Today, theres been a lot of trials done that show that safety is not an issue. I think safety is kind of off the table but efficacy is still a question mark. And thats what were trying to deliver now, Olav Helleb, CEO of ReNeuron, told me.

While neural stem cells and other tissue-specific stem cells are able to regenerate the cells of a particular tissue, Embryonic Stem Cells (ESCs) and their engineered counterparts, iPSCs, are capable of making every cell type in the body, a property known as pluripotency. Pluripotent stem cells can also expand indefinitely in culture and their identification unlocked massive expectations for these cells to transform the regenerative medicine field.

Yet, these cells come with significant challenges associated with the safety of the final preparation. Apart from ethical issues surrounding ESCs, today, a lot of companies have been cautious about using these cells for therapy, because undifferentiated pluripotent cells can drive tumor formation, explained Rawadi. Since ESCs can, in principle, form every cell type, they can lead to the formation of teratomas.

A major reason for the fairly slow progress in the field is based on the difficulties of directing a pluripotent cell to exactly the cell type that is needed for cell therapy. We can readily drive the cells from the undifferentiated state to the differentiated state. However, getting those cells to pause anywhere in the middle of this continuum to yield progenitor cells is incredibly challenging, Peyer explained. Another challenge, he says, is to engraft the cells in the right place to enable them to become fully integrated.

Besides initial hurdles, companies like US-based Asterias or ViaCyte are now running the first Phase I/II trials with ESC-derived cells to treat patients with spinal cord injuries and to restore the beta cells in type I diabetes. So far, the eye has been the the dominant organ for many of the first human clinical trials with pluripotent stem cells, where the cells are assessed in diseases such as age-related macular degeneration (AMD) to restore the loss of the retinal epithelium.

Deriving retinal epithelium from pluripotent cells is relatively easy and in fact, researchers in Japan are now running the very first clinical trial using donor-derived iPSCs to treat patients with AMD. For reasons of safety and standardization, the trial is based on an allogeneic approach. However, since this doesnt offer an exact genetic match, allogeneic therapies raise the prospect of immune rejection, an issue that has been plaguing the use of ESCs.

But the scientists in Japan have contended that iPSC banks could potentially solve this problem. The team in Japan is currently establishing an iPSC bank, consisting of HLA-characterized cell lines from 5-10 different donors, which should match 3050% of Japans population.

Such haplobanks have the benefits of allogeneic cell therapy, namely cost-effectiveness and standardization, but you still have matching immune systems, Peyer agrees.

For now, this remains a vision for the future, but the potential seems enormous. As Julian Howell, CMO of ReNeuron, told me, iPSCs have still got an awful long way to go. For the iPSC program running in Japan, they recently acknowledged that it took about $1.5M and 6 months to treat each patient. Its a great idea but its still got some way to go before it reaches the scale that could get into the clinic.

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Regenerating the Body With Stem Cells Hype or Hope? - Labiotech.eu (blog)

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SINGAPORE The use of stem cell treatment to repair liver cirrhosis, or hardening of the liver, will be tested in a clinical trial here involving 46 patients and costing S$2.6 million.

The four-year study, which was launched yesterday, came amid a growing waiting list in Singapore for a liver transplant, which is currently the only cure for patients with end-stage liver cirrhosis.

Conducted by a multi-centre team from several restructured hospitals here, the study is led by the National University Hospital (NUH).

Liver failure is one of the top 20 causes of death in Singapore, but many patients are not suitable for a transplant due to factors such as age and surgical fitness.

Out of every five patients doctors see with end-stage liver disease, only one qualifies for a liver transplant, said Dr Dan Yock Young, principal investigator of the clinical trial and senior consultant at NUHs division of gastroenterology and hepatology.

(A liver transplant) is curative, but it is a complex procedure, and many patients are not suitable for it. For these patients, treatment is limited, but morbidity and mortality rates are high as high as 50 per cent in one year and this is probably worse than many (of the) other terminal illnesses we talk about today, he said.

Animal studies conducted over the last five years have shown that stem cells can reconstruct the micro-environment of a normal liver.

Like how branches are of critical importance in supporting the leaves and fruits of a tree, the endothelial (stem) cells contribute to supporting a nutritious environment for the hepatocyte (liver) cells, Dr Dan explained.

While similar stem-cell studies have been conducted in other centres in Asia, there has been no definitive evidence of the benefits of the treatment for liver patients.

The study will recruit 46 patients aged between 40 and 70 years old, and who are at the terminal stages of chronic liver disease, over three years. It is funded by the National Medical Research Council.

During the clinical trial, patients will be divided into a therapeutic group and a control group.

All patients will receive an injection to stimulate their bone marrow cells as part of the supportive treatment for their liver cirrhosis. However, only patients in the study group will have the stem cells from the bone marrow extracted and deposited directly into their liver for more targeted repair.

Using ones own stem cells will avoid the problem of cell rejection.

The liver tissue will be examined three months later, and an investigation to compare pre- and post-transplant results will be conducted after a year.

Since invasive surgery is not required for stem-cell therapy, the fatality risk is significantly lowered for the patient. However, other risks such as severe bleeding and infections still remain, given the patients weakened condition.

NUH also noted that the stem-cell therapy does not replace liver transplants, and the latter remains the best available treatment for liver cirrhosis.

It is very painful to turn patients away when we cannot offer them a liver transplant, said Dr Dan, adding that this stem cell therapy will serve as an alternative option.

We hope that this is a stepping stone to trials for stem cell candidates, he added.

MORE WAITING FOR A LIVER

The number of people on the waiting list for a liver transplant has been growing in recent years. In June last year, it was reported that there were 54 people on the list, more than double the 24 patients in 2011.

Chronic Hepatitis B remains the primary cause of non-alcoholic fatty liver disease, which refers to a range of liver conditions affecting people who drink little to no alcohol. However, obesity has become a contributing factor to the illness as well.

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New NUH study to test stem cells as treatment for liver disease - TODAYonline

Bone Marrow Stem Cells Comments Off on New NUH study to test stem cells as treatment for liver disease – TODAYonline | July 12th, 2017

Fear becomes a constant companion when ones child is desperately ill.

Kim Lahti-Scranton knows this all too well as her daughter Jane is fighting for her life at BC Childrens Hospital.

As much as Janes journey is all-consuming, Kim is helping a desperate woman to get young people to donate bone marrow in order to find a better match for her son, Noah, who is in critical need of a bone marrow transplant soon.

The Scranton family is living in Vancouvers Ronald McDonald House and shares a kitchen with 17-year-old Noah Stoltes family.

Noah survived a first round of cancer when he was 10. In April, the family was given the terrifying news that he had relapsed.

His mom thought they were going to find a match within family; theyre 100 per cent Dutch, says Kim, noting family members are usually the best choice, but his three sisters are only a 50 per cent match.

Out of the worldwide pool of donors, there are two people who are a 75 per cent genetic match and are being considered, one of whom lives in Europe. The lower the match, the more chance there is for complications and a less than optimal outcome.

There is a time crunch as Noah needs to have a transplant sometime in September and it can take a couple of months from registration to donation.

One of Kims nephews is an NHL player, who used his connections to help Noahs aunt interest 85 people in becoming bone marrow donors in Edmonton.

Unable to get help from Canadian Blood Services to organize a similar drive in Vancouver because of staffing issues, Kim and Noahs mother, Stacey VanderLee Stolte, went to a Vancouver Canadians game to raise awareness and hopefully encourage people to sign up to be on the registry for stem cell or bone marrow donation.

We were met with a whole range of responses from people who were incredibly receptive, to people who completely ignored us and everything in between, says Kim. We got some interesting comments like no kid would want my bone marrow, Ive done too many drugs, to other people saying no because they thought if they were a match that donating bone marrow would be too painful.

Dr. Lucy Turnham, a clinical associate who oversees the outpatient oncology clinic at BC Childrens Hospital, has performed Janes procedures and says males between the ages of 17 and 35 are the best resource.

Its one of the most wonderful charitable donations you can do, Turnham says, noting many people never receive a call. It has a huge impact on a patient who has no other options.

Turnham explains that donors are educated before they do a swab. If they are told they are a match, they can still say no at any time. They then undergo further testing and maybe counselling at that point, and can still back out an any time.

If they had said yes and the patient begins treatment to get rid of the diseased cells prior to transplant, and then the donor backs out, the patients life is at serious risk, so it is important to know what you are getting into before saying yes, Kim stresses. If the donor says yes to the procedure, they are told beforehand when the treatment would start and told when the last chance to back out would be.

Stem cell donations can be made in two ways: the donor is hooked to a machine with IVs in each arm. Blood is taken from one arm, stem cells are removed, the blood is replaced through the IV in the other arm where the body grows more stem cells.

Or, the donor is anesthetized, a needle is placed into the hip bone from the back and part of the bone marrow is sucked out.

You might be a little bit sore afterwards, but for less than 24 hours, Turnham says, noting women can be donors but not if they have been pregnant. Young bone marrow is more robust and we regenerate marrow and blood all the time.

Becoming a donor is not a speedy process for people living in smaller communities, but would-be donors can receive a swab kit through the mail and return it postage-free.

Marc Plante, a representative with the national office of Canadian Blood Services, says people can go to http://www.blood.ca and proceed to the Stem Cells tab at the top of the page.

If you have an opportunity to save a life and just put up with a couple of days of discomfort, I would do it in a heartbeat, says Kim, who must also face the reality that Jane could one day be in the same dilemma. If most people had the opportunity to save a life, they would do it.

The Scrantons have been living at Ronald McDonald House for several months, while Jane receives treatment.

Shes as good as can be expected; shes very compromised and we need to make sure shes not exposed to anything, says Kim, noting the first, and this, the fourth phase of treatment, are considered to be the toughest. She has nothing to fight off infection.

There is another terrifying aspect to this disease.

As well as living with the fear of potential relapse, some of the chemo drugs being used to treat Jane increase the risk of heart disease and cause secondary cancer, neuro-cognitive issues and behavioural issues.

We dont know if its coming or what it will be, but we dont have a choice, we have to save her life now and deal with the consequences later, Kim says. Youre just kind of waiting for the other shoe to drop, for things to take a turn, its not fun. And while Jane will require a lot of follow-up assessment in the years to come, the tough little hero turns six on Aug. 12.

She has everything she needs; I just want people to sign up for the bone marrow registry or donate blood, Kim says, noting she is grateful for the communitys support throughout the ordeal.

If you cant donate but would like to help Jane celebrate, you can take birthday cards to the Salmar Grand Theatre where manager Daila Duford will make sure they get to their destination.

Excerpt from:
Bone marrow transplant last chance - Revelstoke Review - Revelstoke Review

Bone Marrow Stem Cells Comments Off on Bone marrow transplant last chance – Revelstoke Review – Revelstoke Review | July 12th, 2017

Yaser and Vicki Martini, who launched a worldwide campaign to find a bone marrow donor for their daughter Margot, today joined the huge public outpouring after the Nigerian international's blood cancer diagnosis was revealed.

Margot died aged just two after an unsuccessful bone marrow transplant but her story prompted thousands of people to join the stem cell register.

But it also exposed the difficulty in finding donor matches for people from ethnic minorities or mixed heritage.

Bone marrow stem cell transplants are one of the major treatment for leukaemia, as well as chemotherapy, radiation, biological and targeted therapy.

Mr Martini, whose wife Vicki hails from Essington, said: "We are shocked to hear about Carl's diagnosis and our hearts go out to him and his family. Its ironic, because everyone at Wolves have been so supportive of Team Margot and we want to send our best wishes and our support, as they fight his cancer together."

They set up the Team Margot Foundation with a string of friends, family and supporters which is aimed at getting more people to join the stem cell register particularly those from ethnic minorities and mixed backgrounds.

Mr Martini added: As yet, its unclear whether Carl will need to have a bone marrow transplant, but its a certainty that he will need blood and platelet donations to stay clinically well. Anyone reading this can help in two ways by signing up to become a blood donor and also registering as a potential bone marrow donor.

Regarding bone marrow donation, we know first hand from Margots experience that people with a mixed heritage and those from the black, Asian and minority ethnic communities have only a 21 per cent chance of finding a donor with a matching tissue type. For this reason, we urgently need more people from these communities to join the bone marrow registers.

"In that sense, we each have a unique contribution that we can make and Im not exaggerating when I say that you could be the only one who can save a life.

Wolves players, including club legend Steve Bull, have backed Team Margot's campaign.

Margot's uncle Durand Bailey, who lives in Tettenhall and runs Diffusion designer clothing store in Lichfield Street, knows Ikeme. He said: Carl is such a lovely man, a real gentleman. He has done so much to help others. We all wish him and his family well."

Wolves announced the 31-year-old had returned 'abnormal blood tests' as he returned for pre-season and further checks confirmed the diagnosis.

He is to start a lengthy treatment programme.

To sign up as a blood donor visit: blood.co.uk. To find out how you can become a potential stem cell donor, go to teammargot.com

Read the rest here:
Carl Ikeme: Family of toddler Margot Martini support Wolves star after leukaemia diagnosis - expressandstar.com

Bone Marrow Stem Cells Comments Off on Carl Ikeme: Family of toddler Margot Martini support Wolves star after leukaemia diagnosis – expressandstar.com | July 12th, 2017

A few small scars on Ashli Stempels lower back are the only evidence that a drill burrowed into her hipbone last year at Brigham and Womens Hospital in Boston. The surgery was to harvest the stem cells in her bone marrow to save her older brother Andrew Stempels life.

At age 27, Andrew was diagnosed with cancer of the white blood cells called Hodgkins lymphoma. Donating her bone marrow so that Andrews body could manufacture healthy blood cells, was a small price to pay to give him a shot at survival, Ashli says.

Since the transplant last August after years of treatment and testing Andrew has been cancer-free and Ashli now volunteers periodically in their hometown of Greenfield to spread awareness about this life-saving treatment.

Our bodies are a cure for some cancers, says Ashli Stempel on a recent Saturday as she handsout sign-up forms atGreenfields Energy Park for the Be A Matchnational donor registry. If even one person joins the registry that is awesome.

Its a sunnyday and Stempel, 30,wearing a black and white spaghetti-strap dress stands behind a booth smiling and talking to passersby.

Everybody wants to cure cancer, but I think not everybody understands that we, ourselves, can be the cure for some types of cancers, she says. I can say that I killed cancer and I am pretty excited about that.

In the hollow spaces in a bodys bones, stem cells inside the bone marrow tissue work to create red blood cells, which feed oxygen to the organs, and make white blood cells to fight infections. The bone marrow also produces blood platelets to help form clots but when a cancer of the blood like, leukemia or lymphoma strikes, these life-supporting systems are thrown out of whack, leaving the bodys immune system unable to fight diseases, infection or the cancer.

Chemotherapy and radiation also can kill off bone marrow tissue, leaving patients with more damage to their immune systems, says physician assistant Susanne Smith, donor services clinician at Dana-Farber/Brigham and Womens Hospitals Cancer Center in Boston.

When transplanted into a cancer patients bloodstream, stem cells, a precursor to all the immune system cells in the body, colonize the bones and help fight any remaining cancer, says Smith.

In many cases (a transplant) is the only cure for a leukemia or lymphoma diagnosis chemotherapy can only get a patient so far, says Mary Halet, director of community engagement at the Be The Match Registry, the Minneapolis organization that manages the largest bone marrow registry in the world. But first, a patient must find a tissue match, that is, a donor who has a similar protein marker called the human leukocyte antigen, which is found on most cells in the body.

There are up to 14,000 patients every year who could benefit from a bone marrow transplant, but many of these people will not receive a donation, says Halet. In most cases, the patient will not finda tissue match in his or her own family andmust seek help from a stranger, she says. A patients likelihood of finding a matching bone marrow donor ranges from 66 percent to 97 percentdepending on ethnic background. White patients have a 97 percent chance of finding a match, while black patients only find a match 66 percent of the time.The difference reflects the complexity of the tissues makeup and the number of donors.

Thats why Halets organization promotes recruitment events like the one Ashli Stempel held in Greenfield.

Stempel says she was ecstatic when she found out that she was a match for her brother. She was in her late 20s at the time, a bubbly woman working in communications at Smith College in Northampton, who grew up in a close-knit family.

Her brother, who was working as a retail manager in the Boston area, had discovered a bump on his collarbone.

I woke up one morning and there was a non-painful lump, Andrew Stempel says.

He ignored it for as long as he could before seeing a doctor who diagnosed it as a swollen lymph node caused by Hodgkins lymphoma.

Cancer is a very scary word. I think what you learn going through it is that it is not such a scary word, you can survive, says Ashli Stempel.

The Stempel family had seen that firsthand years earlier when Andrew and Ashlis mother, Deborah, recovered from breast cancer.

Still, that didnt lessen the anxiety for Andrew. As soon as the doctor said the word cancer, he says, his life started to unravel with a battery of experimental drugs, chemotherapy and radiation.

In the begining there was a lot of uncertainty, he says.

Even through his cancer went into remission after a year, doctors did not expect it to remain that way without high doses of chemo or radiation. The plan was to do a bone marrow transplant for long-term survival.

Still, using donated bone marrow meant taking the risk that Andrews body would reject it, which could be fatal.

So, doctors first wanted to try using Andrews own tissue. That would require removing some of his bone marrow, treating it and then injecting it back into his bloodstream.

Within months of the procedure, however, Andrews cancer returned, indicating to doctors that his body wasnt strong enough to fight it on its own.

Ashli was tested via a mouth swab and Andrew was relieved to learn that she was a tissue match.

I was just overwhelmed with happiness, he says.

Ashli went through a month-long screening process to ensure that she was healthy enough to be a donor. People who have infectious diseases like HIV or hepatitis cannot be donors, nor can those with immune systems weakened by autoimmune diseases. Doctors also prefer to use bone marrow from young donors under the age of 44, says Halet. The registry wont accept donors over 60.

When we are young, our immune systems are at their healthiest and the older we get the less robust they are, she says.

It took two years from the time Ashli first learned she was a match for her brother for the transplant to take place.

Not long aftershe woke up from the surgery, Ashli saw the bone marrow that had been taken from her, a two-literjug ofmilky, red liquid. It was whisked away to another partof the hospital where it ended up in a drip bag connected to a vein in Andrews arm.

Doctors saw hisred and white blood cell counts go up immediately after the transplant.

My sisters cells were working, he says. It was amazing.

Even though the transplant was a success, Andrew had to stay in the hospital for a month. Chemotherapy had caused sores in his mouth, he lost his ability to taste food along withhis appetiteand he droppednearly 30 pounds.

It was tough, day to day, but progressively got better, he says.

Since he was essentially receiving a new immune system, like a newborn baby he also had to be shielded from germs, says Ashli.

When his wife, Meghan Stempel, came to visit him, she needed to wear a facemask and gloves. Even when he returned home, he had to be careful. Hetook a year off from his job to recover, spending many afternoons resting on the couch watching TV. After spending months working to building hisstrength back up,he says, most of his weakness has subsided.

I feel a thousand times better, he says.

He is now cancer free and is returning to hisjob as a retail managerat Sherwin Williams this week.

Following her operation, Ashli took off a few weeks from her job in communications at Smith College, but was back on her feet within a couple days. Her hips were sore which meant limping around the house for a short time.

I was in pain, of course, she says. But its a quick recovery.

A few weeks ago Ashli decided to signup for the national bone marrow donor registry through Be A Matchto donate for a second time.

Her name will stay in the system for the foreseeable future. A match could come up or it might never.

Maybe I will be called on to do it again, who knows?

To learn more about becoming a bone marrow donor or to sign up for the registry, go tobethematch.org.

Potential donors can fill out an online form and the registry will mail a mouth swab kit, which can be returned by mail.

If called, a potential donor will undergo a series of blood tests which will evaluate the suitabililty and safety of the match. Though doctors say risks are low for donors, possible complications include infection and bleeding.

Once a donor is cleared, the transplant procedure could occur within a few weeks or a few months, depending on a recommendation from the patients doctor.

The bone marrow transplant is an outpatient procedure for the donor.Recovery time is only a few days anddonors are typically back to their normal routine in two to seven days.

Donors are told their commitment means being willing to devote up to 30 hours spread over four to six weeks to attend appointments and give the donation.

All medical costs for the donation procedure are covered by the National Marrow Donor Program, which operates the Be The Match Registry, or by the cancer patients medical insurance.

Sometime travel is required. Most travel expenses are covered by Be The Match.

Excerpt from:
Ashli Stempel helped save her brother's life. She hopes to inspire others. - GazetteNET

Bone Marrow Stem Cells Comments Off on Ashli Stempel helped save her brother’s life. She hopes to inspire others. – GazetteNET | July 11th, 2017

All of our blood vessels are lined with a type of cell called endothelial cells. To form vessels, individual endothelial cells begin to create empty holes in themselves, called vacuoles. They then connect with other endothelial cells that have done the same thing and the linked vacuoles form tubes, which ultimately become capillaries. Here, the researchers took endothelial cells and mixed them with either fibrin -- a protein involved in blood clotting -- or a semi-synthetic material called gelatin methacrylate (GelMA), which can be easily 3D-printed. When mixed with fibrin, the endothelial cells formed tubes fairly easily, but that wasn't the case with the GelMA. However, when the researchers added in another type of cell, a stem cell found in bone marrow, the endothelial cells were then able to form tubes in the GelMA.

"We've confirmed that these cells have the capacity to form capillary-like structures, both in a natural material called fibrin and in a semi-synthetic material called gelatin methacrylate, or GelMA," Gisele Calderon, the lead author of the study, said in a statement, "The GelMA finding is particularly interesting because it is something we can readily 3D print for future tissue-engineering applications."

The benefits of this method over others include cells that can be patient-specific, reducing the risk of immune system complications, and growth environments that are well suited for organ and vasculature growth -- they're reproducible, not likely to induce immune responses and help boost cell growth and vessel development. Along with making 3D-printed organs more viable, this method will also allow for the development of tissue that could make for more effective and efficient drug testing. In a statement, Jordan Miller, whose lab the work was done in, said, "Preclinical human testing of new drugs today is done with flat two-dimensional human tissue cultures. But it is well-known that cells often behave differently in three-dimensional tissues than they do in two-dimensional cultures. There's hope that testing drugs in more realistic three-dimensional cultures will lower overall drug development costs."

You can watch a video of the cells beginning to form tubes here and Calderon explaining her work in the video below.

Excerpt from:
Researchers are closer to working capillaries in 3D-printed organs - Engadget

Bone Marrow Stem Cells Comments Off on Researchers are closer to working capillaries in 3D-printed organs – Engadget | July 11th, 2017

SINGAPORE - The use of stem cells to reverse liver cirrhosis - or the hardening of the liver - is being explored in a clinical trial.

Conducted by a multi-centre team led by the National University Hospital (NUH), doctors aim to determine if stem cell therapy can improve liver function.

Previously, liver cirrhosis, caused by various diseases such as chronic hepatitis B and non-alcoholic fatty liver disease, was thought to be irreversible.

A liver transplant provides a definitive cure to end-stage cirrhosis.

However, in Singapore, less than 5 per cent of end-stage liver cirrhosis patients receive a liver transplant.

The number of people on the waiting list for a liver transplant has been increasing over the years, according to statistics from the Ministry of Health.

In 2007, there were nine on the waiting list, compared with 57 last year. There are around 50 waiting for a liver transplant this year.

Also, many patients do not fulfil the eligibility criteria to receive a liver transplant due to other health complications or being above the age limit of 70 years.

The $2.6 million study, which was launched on Tuesday (July 11),is funded by the National Medical Research Counciland 46 patients will be recruited for it. It will run for four years and patients will not need to bear the costs of the stem cell treatment.

Stem cells will be taken from a patient's own bone marrow and will be isolated and injected directly into the patient's liver to initiate the repair.

Similar therapy treatments have been conducted overseas in countries such asEgypt and India, although they have not been fully evaluated for efficacy.

Associate Professor Dan Yock Young, a senior consultant in the division of gastroenterology and hepatology at NUH, said: "We are conducting the study in a systematic and scientific mannerto get definitive evidence of the effects of the treatment."

He also notes that the stem cell therapy is not a substitute for a liver transplant. "This treatment is not intended to pull patients off the waiting list, but provide an option for those who are not eligible for a transplant."

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Clinical trial for stem-cell therapy to reverse liver cirrhosis - The Straits Times

Bone Marrow Stem Cells Comments Off on Clinical trial for stem-cell therapy to reverse liver cirrhosis – The Straits Times | July 11th, 2017

JOEL INESON

Last updated14:01, July 9 2017

IAIN MCGREGOR/Stuff.co.nz

Andrea Cameron-Hill has lived with multiple sclerosis for about 10 years. She wants to receive treatment in Russia that could stop the disease.

Andrea Cameron-Hill thought having to lift her leg to get in the carwhile pregnant was part of carrying twins.

About 10 weeks after they were born she learned she had multiple sclerosis (MS).

"To start with, you wouldn't know I had MS at all. But now it's 10 years on and I'm having to walk with a crutch," she said.

IAIN MCGREGOR/STUFF

MS sufferer Cameron-Hill wants to receive treatment that could stop the disease in its tracks but, despite it being offered here to treat some cancer, must travel to Russia to get it.

"If I have to go down to the floor to load the fire with wood, the difficulty for me now is getting off the floor."

READ MORE: *Multiple sclerosis sufferer Royce Brewer cleared after experimental treatment in Russia *Multiple sclerosis patient to receive 'experimental' treatment in Russia *Friends rallying to help Upper Hutt woman reach Mexico for stem cell treatment *Marlborough woman's search for cure *Plea to help fund stem-cell treatment for Andrea Campbell *Hunt after cure for MS disease

Cameron-Hill has injured her shoulder fromfalls and reliedon her husband, Paul, and sons Lachlanand Oliverto help with household chores.

IAIN MCGREGOR/STUFF

Normal household chores like washing are a challenge because of the debilitating condition.

She wanted to do things like go onto the rugby field while her childrenplayed, but MS meant she had to watch from the car.

"We've got a basketball hoop [at home], which they quite like playing, but they normally play with Grandma because I can't."

"I feel like a spectator in their lives. I hate it. I hate it with a passion."

IAIN MCGREGOR/STUFF

Cameron-Hill's sons, Lachlan and Oliver, pictured, help her complete tasks many do effortlessly.

Cameron-Hill's condition drove herto look at a treatmentused in New Zealand for some forms of cancer, but not available for MS.

She plans to head to Russia for undergohematopoieticstem cell transplantation (HSCT).

HSCT would remove, purifyand concentrate her stem cells.Chemotherapy would wipeher immune system before the stem cells were returned. An extended period of recovery would follow.

IAIN MCGREGOR/STUFF

Cameron-Hill wants to take part in her children's lives, rather than watch from the sidelines.

Cameron-Hill mustraise about $80,000 to get the treatment.So far about $10,000 has been raised through fundraising events and aGivealittlepage.

Leading New Zealand neurologist Dr Deborah Mason said HSCT wasunlikely to be trialledin New Zealand because it wouldnot make drug companies money.

Treatment for MSin New Zealandreliesonimmunosuppressantdrugs.HSCTwaslikened more to a surgical procedure than drug treatment.

"It's incredibly expensive ... We're very keen to participate in [trials and research] and I'd certainly enroll patients, but it's just finding somebody who would fund that, and nobody will because there's no drug involved," Mason said.

Mason saidHSCT might help young patients in the early stages of MS.

She said the treatment was "unproven" and came with risks.

"It's really hard to imagine why I get all these calls about bone marrow transplants ... all bone marrow transplant allows us to [do is] give industrial doses of chemotherapy."

"People talk aboutrebootingthe immune system and all of that. There isn't a lot of proof of that."

Mason knew of about six or seven people who had travelled forHSCT, but did not have a lot of follow-updata.

Studies and clinical trials New Zealanders took part in primarily focussed on medication.

Her "big beef" was with the government not allowing the use of some drugs, "which we absolutely know will benefit the patients".

The Multiple Sclerosis Society of New Zealand recently changed its standpoint on HSCTafter a report indicated the treatment workedfor some with MS in Australia and the UK.

Vice president Neil Woodhams said the group asked the Ministry of Health to start a process to lead to eligible people havingHSCTin New Zealand.

Cameron-Hill's MS had not progressed since 2013and she had not been on medication for it since.

She recently met Christchurch manRoyce Brewer who lived with MS for about 20 years. After undergoing HSCT in Russia in early 2016, hereturned to work as a landscaper at the end of the year.

"I'd just really love to be able to do ... normal stuff that parents do with their kids," Cameron-Hill said.

The Health Research Council of New Zealandhad not funded any research regarding MS andHSCT, a spokeswoman said.

-Stuff

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Christchurch MS patient Andrea Cameron-Hill plans Russia trip for stem cell treatment - The Press

Bone Marrow Stem Cells Comments Off on Christchurch MS patient Andrea Cameron-Hill plans Russia trip for stem cell treatment – The Press | July 9th, 2017