University creates new scoring system for transplant recipients

University researchers have developed a new testing system that can improve care for patients who need bone marrow and stem cell transplants.

Graft-versus-host disease is a life-threatening condition that can occur in response to transplants. GVHD causes immune cells from the transplant to attack the bodys healthy tissue. In patients with diseases such as leukemia, which compromises the bodys immune system, bone marrow or stem cell transplants are necessary.

John Levine, professor of pediatrics and the study's lead author, said in these types of cases, GVHD is a real danger.

Following transplantation surgeries, our major concern is the development of GVHD in our patients, Levine said. However, it is difficult to predict the severity of GVHD at the onset of the symptoms as it varies from patient to patient.

Prior to the research, there was no method for determining the severity of a GVHD case and whether or not it needed treatment. The treatment involves high doses of medication that reduce immune activity, so doctors must be extremely cautious when treating GVHD. Levine and his co-investigators assessed nearly 800 patients and created a scoring system that uses three proteins to assess the severity of each case of the disease.

We found out that it was not one protein but a combination of three recently validated biomarkers TNFR1, ST2, and Reg3, Levine said. We then formulated an equation which computes the concentration of the biomarkers into three Ann Arbor scores. The scores are positively correlated with the amount of risk the diagnosed patient is in, so a score 1 indicates a patient with minimal risk while a patient diagnosed with a score of 3 will subjected to intensive primary therapy.

The Ann Arbor scoring system will help ensure patients at lower risk are subjected to less aggressive treatments than patients at higher risk. Patients will then gain individualized treatments based on their needs.

More than half of the patients undergoing bone marrow transplantation develop GVHD. Though the degree of severity differs in patients, the disease is highly lethal if not treated immediately.

The research began in the late 1990s when investigators analyzed blood samples from 500 GVHD patients. The results were verified when another 300 patient blood samples from across the United States were analyzed.

The next step, according to Levine, is the launch of a clinical trial. The U.S. Food and Drug Administration has approved this step.

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University creates new scoring system for transplant recipients

Health researchers digging into own pockets to bridge gap in funding

Later this month, Torontos University Health Network is planning to enroll a dozen patients with arthritis-wracked knees in a clinical trial of a stem-cell treatment that researchers hope could one day make artificial joint replacements obsolete.

The trial, a Canadian first, wont be cheap, despite the small number of recruits. To cover the estimated $500,000 cost, the researchers turned to an unconventional source of funding: 10 orthopedic surgeons at UHNs Toronto Western site who donated a total of $1.25-million of their own money beginning five years ago to kick-start the networks research into a cure for arthritis.

The orthopedic surgeons decision to raid their own bank accounts to help pay for research is a rare but not unheard-of move in Canada, one that was driven in part by how much harder it has become to score publicly funded medical research grants in this country.

We felt very strongly that in order to go to anybody and say, Would you please give me support for this idea? we had to have our own commitment beyond just the time and effort we would all have to put in, said Nizar Mahomed, director of the UNH arthritis program and one of the surgeons who donated $125,000 over five years. We needed to make a commitment of actual dollars and put skin in the game.

The doctors gift prompted a philanthropic avalanche from grateful patients toward a campaign that has now raised $38-million to combat osteoarthritis, a disease that affects 4.6 million Canadians but does not traditionally have the fundraising pull of cancer or heart disease. The upcoming stem-cell experiment is the first human trial to be funded by the campaign.

In 2011, the neurosurgery team of 14 doctors at UHN, a network of four downtown Toronto hospitals, followed the lead of the orthopedic surgeons and made a collective $1-million donation to brain research. Last year, a group of eight orthopedic surgeons in Thunder Bay announced a $2-million personal donation over 10 years to a research project aimed at reducing diabetic limb amputations in Ontarios remote northern communities. And it is not uncommon for doctors and other health-care workers to give to the charitable foundations that support their hospitals.

What all these physicians have in common is the challenging research-funding climate in which they are operating.

I think its pretty clear that the accessibility to funds has never been more competitive, said Jim Woodgett, director of research at the Lunenfeld-Tanenbaum Research Institute at Torontos Mount Sinai Hospital.

To give one telling example, the success rate for applications to the Canadian Institutes of Health Researchs open operating grant program the federal agencys largest pot of money, which accounts for a little over half of all the funding it doles out fell to 18 per cent for 2014-2015, down from 33 per cent less than a decade earlier.

Part of the explanation for the low success rate for the 2014-2015 granting year was an unusual spike in applications as investigators scrambled to submit proposals before an administrative revamp takes full effect at CIHR.

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Health researchers digging into own pockets to bridge gap in funding

Alberta MS patient says researcher was seen as 'some sort of god'

Winnipeg Free Press - ONLINE EDITION

By: Mary Agnes Welch

Posted: 01/15/2015 2:00 AM | Comments: | Last Modified: 01/15/2015 12:48 PM | Updates

CHRIS BOLIN / WINNIPEG FREE PRESS Enlarge Image

Lee Chuckry, who has MS, took part in a stem-cell trial run by Doug Broeska and Regenetek Research. The Airdrie, Alta., man eventually became one of Regenetek's most vocal critics. Photo Store

Before flying to India for experimental stem-cell therapy, Alberta businessman Lee Chuckry quit taking Tysabri, a drug many multiple-sclerosis patients use to shrink brain lesions and reduce attacks.

"It was quite effective for me," said Chuckry from his home in Airdrie, Alta. "I didnt have attacks when I was on it."

Doug Broeska, founder of Winnipeg-based Regenetek Research and the clinical trials principal investigator, told Chuckry that Tysabri would damage the effectiveness of the implanted stem cells.

Tysabri is one of a long list of medications Broeska advised clinical-trial participants to avoid, all mentioned in a blog posted last fall.

"My first attack started just when I was leaving India," said Chuckry. "Id stopped the drug three months before."

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Alberta MS patient says researcher was seen as 'some sort of god'

Alberta MS patient says researcher seen as 'some sort of god'

Winnipeg Free Press - PRINT EDITION

By: Mary Agnes Welch

Posted: 01/15/2015 3:00 AM | Comments:

BEFORE flying to India for experimental stem-cell therapy, Alberta businessman Lee Chuckry quit taking Tysabri, a drug many multiple-sclerosis patients use to shrink brain lesions and reduce attacks.

"It was quite effective for me," said Chuckry from his home in Airdrie, Alta. "I didn't have attacks when I was on it."

Doug Broeska, founder of Winnipeg-based Regenetek Research and the clinical trial's principal investigator, told Chuckry that Tysabri would damage the effectiveness of the implanted stem cells.

Tysabri is one of a long list of medications Broeska advised clinical-trial participants to avoid, all mentioned in a blog posted last fall.

"My first attack started just when I was leaving India," said Chuckry. "I'd stopped the drug three months before."

Chuckry knew Broeska was not a physician, but believed Broeska had a PhD and was a bona fide health researcher. Chuckry felt no better after the $24,000 stem-cell therapy. He became increasingly skeptical of Broeska and Regenetek when he returned home from India in May 2013 -- his MS just as bad, if not worse.

Chuckry spent 10 days trying to get in touch with Broeska to find out whether going back on his MS medication, this time a steroid called prednisone, would interfere with the effectiveness of his newly implanted stem cells. He could not get an answer from Broeska for days, and said there was no real followup care typically seen in a proper clinical trial -- no MRIs, no examination by a physician, no tests, no questionnaires.

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Alberta MS patient says researcher seen as 'some sort of god'

New test helps guide treatment for bone marrow transplant patients with graft vs. host disease

Innovative scoring system uses 'Ann Arbor GVHD score' to better predict how patients will respond, minimize side effects

ANN ARBOR, Mich. - A new test can guide treatment for patients with graft versus host disease (GVHD), an often life-threatening complication of bone marrow and stem cell transplants, according to research from the University of Michigan published in Lancet Haematology this month.

Patients with fatal blood cancers like leukemia often need bone marrow or stem cell transplants to survive. But one of the most common and serious side effects that patients face is graft vs. host disease: when a patient's new immune system from the transplant (the graft) attacks the patient's healthy tissue (the host).

Most GVHD starts out as mild, but in two-thirds it eventually becomes severe. The treatment for severe GVHD is high doses of medications that knock out the immune system. But doctors have to be careful with drugs that further weaken a newly transplanted immune system, because they increase the risk for serious and life-threatening infections. Until now there has been no test to determine which cases of GVHD will become severe, so treatment is often delayed until the GVHD worsens.

The study's lead author, John Levine, M.D., of the University of Michigan's Blood and Marrow Transplant Program and his colleagues studied almost 800 patients from the US and Germany to develop and validate a new scoring system. The Ann Arbor GVHD score uses the levels of three proteins in the blood (TNFR1, ST2, and REG3a) to determine whether the patient should be treated right away or not and how intense the treatment should be. Patients with Ann Arbor 1 GVHD usually don't need treatment while patients with Ann Arbor 3 GVHD often don't respond to standard treatment and should be considered for clinical trials.

"We often have to treat all patients with GVHD alike with very high-dose steroids, because the severity of symptoms at the disease's onset don't help us predict how sick the patient will get. But this new scoring system will help identify patients that need a different approach, says Levine, who also is clinical director of the Pediatric Blood and Marrow Transplantation program at C.S. Mott Children's Hospital.

"And it can help us with patients with lower-risk GVHD who we may be over-treating. These scores can help us find a better, more individualized fit for our patients as soon as their disease is diagnosed," says Levine, who is professor of pediatrics at the University of Michigan Medical School.

Around half of patients who get a bone marrow transplant will develop GVHD, which can be lethal if it can't be controlled.

"Our goal is to offer personalized care. Doctors have struggled with individualizing therapy for each patient, but there's been no new therapy for GVHD in more than 40 years. So this new scoring system gives us another tool to better take care of our patients," Levine says.

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New test helps guide treatment for bone marrow transplant patients with graft vs. host disease

Quadriplegic veteran to receive stem cell treatments

LAWTON, Okla._A stem cell surgery procedure, not yet approved by the FDA, could give a local paralyzed veteran the use of his arms again.

Two years ago, retired Senior Airman Ted "TJ" Williams was left as a quadriplegic when his Humvee rolled over in a freak accident while on duty in Montana. He spent several weeks in a coma.

Now, he and his wife have found a surgery that may improve his physical abilities. They're dipping into their funds to pay for the procedure, since it's not covered by insurance, but they've set up a GoFundMe account to raise $7,500 to cover travel expenses out of the country to get the treatment.

Williams is able to move his left wrist and arm more, and has even gained more core control, thanks to therapy. But, he still needs his wife's help for simple tasks like getting dressed and using the restroom.

Williams sits next to his wife in his wheelchair and watches TV. Years ago, he would've been running outside, but one accident changed everything.

"I just remember leaving base and then waking up 2 or 3 weeks later, wondering where am I. I couldn't move anything. It was just shocking seeing my family around my bed. I was just like, Wow. What's going on,'" recalled Williams.

On November 29, 2012, Williams was on duty with his security forces team. He was in the back seat when his Humvee suddenly swerved to miss a herd of deer, rolling several times. He was ejected from the vehicle and was later found 60 feet away.

Williams was rushed to the hospital. When he woke up from the coma, doctors told him he had broken the vertebrae in his neck and lost function from the chest down.

"I was really upset and scared. Me and my wife are young. We haven't had children yet or anything. It scared me not knowing what the future was to hold," said Williams.

He was sent to a VA hospital in San Antonio for in-patient rehab. Once he was finished, he met a physical trainer in who specializes in exercises for those who are suffering from spinal cord and other neurological injuries, which was just what he needed.

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Quadriplegic veteran to receive stem cell treatments

Scientists explain how stem cells and 'bad luck' cause cancer

Why are some types of cancer so much more common than others? Sometimes its due to faulty genes inherited from ones parents and sometimes to behaviors like smoking a pack of cigarettes every day. But in most cases, it comes down to something else stem cells.

This is the intriguing argument made by a pair of researchers from Johns Hopkins University. In a study published Friday in the journal Science, they found a very high correlation between the differences in risk for 31 kinds of cancer and the frequency with which different types of stem cells made copies of themselves.

Just how strong was this link? On a scale that goes from 0 (absolutely no correlation) to 1 (exact correlation), biostatistician Cristian Tomasetti and cancer geneticist Bert Vogelstein calculated that it was at least a 0.8. When it comes to cancer, thats high.

No other environmental or inherited factors are known to be correlated in this way across tumor types, Tomasetti and Vogelstein wrote.

Researchers have long recognized that when cells copy themselves, they sometimes make small errors in the billions of chemical letters that make up their DNA. Many of these mistakes are inconsequential, but others can cause cells to grow out of control. That is the beginning of cancer.

The odds of making a copying mistake are believed to be the same for all cells. But some kinds of cells copy themselves much more often than others. Tomasetti and Vogelstein hypothesized that the more frequently a type of cell made copies of itself, the greater the odds that it would develop cancer.

The pair focused on stem cells because of their outsized influence in the body. Stem cells can grow into many kinds of specialized cells, so if they contain damaged DNA, those mistakes can spread quickly.

The researchers combed through the scientific literature and found studies that described the frequency of stem cell division for 31 different tissue types. Then they used data from the National Cancer Institutes Surveillance, Epidemiology and End Results database to assess the lifetime cancer risk for each of those tissue types. When they plotted the total number of stem cell divisions against the lifetime cancer risk for each tissue, the result was 31 points clustered pretty tightly along a line.

To put this notion in concrete terms, consider the skin. The outermost layer of the skin is the epidermis, and the innermost layer of the epidermis contains a few types of cells. Basal epidermal cells are the ones that copy themselves frequently, with new cells pushing older ones to the skins surface. Melanocytes are charged with making melanin, the pigment that protects the skin from the suns damaging ultraviolet rays.

When sunlight hits bare skin, both basal epidermal cells and melanocytes get the same exposure to UV. But basal cell carcinoma is far more common than melanoma about 2.8 million Americans are diagnosed with basal cell carcinoma each year, compared with roughly 76,000 new cases of melanoma, according to the Skin Cancer Foundation. A major reason for this discrepancy, Tomasetti and Vogelstein wrote, is that epidermal stem cells divide once every 48 days, while melanocytes divide only once every 147 days.

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Scientists explain how stem cells and 'bad luck' cause cancer

Margot Martini's story can save lives with new charity

A charity in memory of inspirational toddler Margot Martini is being set up by her family with the aim of getting more than one million extra people to sign up to the UK stem cell register.

The Team Margot Foundation has the goal to get more than 1.5 million extra potential bone marrow donors to take the total to 2.5 million.

The foundation will also raise money and support the work of five charities - Great Ormond Street Hospital Childrens Charity, Delete Blood Cancer UK, Anthony Nolan, Shooting Stars Chase, and Momentum.

Two-year-old Margot died in October from two rare types of leukaemia following a worldwide search for a bone marrow donor.

Margot's parents, Vicki, originally from Essington, and Yaser, went public in their search for a bone marrow donor to help save their daughter's life.

Speaking exclusively to the Express & Star, Margot's father Yaser outlined plans for 2015 which will see Team Margot in full swing over the next 12 months.

They include, establishing the Team Margot Foundation to support the work of charities close to their heart, holding the first annual international Team Margot Stem Cell and Bone Marrow Awareness Day, supporting bone marrow donor registration events across the country, and a 40-day trek across the ice caps of Greenland, named Expedition Margot.

"We see this as Margot's legacy - and we want that to continue for the sake of others," said Mr Martini.

Margot's mother Vicki, 39, grew up in Essington, Staffordshire, and has marked her first Christmas without her daughter with Yaser, 44, and sons Oscar, seven, and Rufus, six.

Now they want 2015 to be the breakthrough year so no family has to suffer the anguish they did to find a matching bone marrow donor.

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Margot Martini's story can save lives with new charity

Stopping Multiple Sclerosis with Stem Cell Transplants

Washington, DC - infoZine - Three-year outcomes from an ongoing clinical trial suggest that high-dose immunosuppressive therapy followed by transplantation of a person's own blood-forming stem cells may induce sustained remission in some people with relapsing-remitting multiple sclerosis (RRMS). RRMS is the most common form of MS, a progressive autoimmune disease in which the immune system attacks the brain and spinal cord.

Three years after the treatment, called high-dose immunosuppressive therapy and autologous hematopoietic cell transplant or HDIT/HCT, nearly 80 percent of trial participants had survived without experiencing an increase in disability, a relapse of MS symptoms or new brain lesions. Investigators observed few serious early complications or unexpected side effects, although many participants experienced expected side effects of high-dose immunosuppression, including infections and gastrointestinal problems.

Scientists estimate that MS affects more than 2.3 million people worldwide. Symptoms can vary widely and may include disturbances in speech, vision and movement. Most people with MS are diagnosed with RRMS, which is characterized by periods of relapse or flare up of symptoms followed by periods of recovery or remission. Over years, the disease can worsen and shift to a more progressive form.

In the study, researchers tested the effectiveness of HDIT/HCT in 25 volunteers with RRMS who had relapsed and experienced worsened neurological disability while taking standard medications. Doctors collected blood-forming stem cells from participants and then gave them high-dose chemotherapy to destroy their immune systems. The doctors returned the stem cells to the participants to rebuild and reset their immune systems.

"Notably, participants did not receive any MS drugs after transplant, yet most remained in remission after three years," said Daniel Rotrosen, M.D., director of NIAID's Division of Allergy, Immunology and Transplantation. "In contrast, other studies have shown that the best alternative MS treatments induce much shorter remissions and require long-term use of immunosuppressive drugs that can cause serious side effects."

The study researchers plan to follow participants for a total of five years, recording all side effects associated with the treatment. Final results from this and similar studies promise to help inform the design of larger trials to further evaluate HDIT/HCT in people with MS.

The trial is funded by the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health, and conducted by the NIAID-funded Immune Tolerance Network (ITN).

The three-year findings are published in the Dec. 29, 2014, online issue of JAMA Neurology.

Related Link Immune Tolerance Network (ITN)

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Stopping Multiple Sclerosis with Stem Cell Transplants

Houston-based company leads nation in medical wave of the future

When Pearland residents Todd and Linsey Hyatts son, Tucker Beau, received a diagnosis of Juvenile Rheumatoid Arthritis when he was just two years old, the future looked agonizing for the previously precocious little boy. His parents refused to lose hope, however, and took a chance on stem cell therapy to improve Tuckers quality of life.

Now at age six, Tucker Beau looks and acts like any other normal boy his age, save for taking rests more often. He has had two separate stem cell infusions in August and November that have turned his slow, painful decline into a fading memory.

Celltex Therapeutics Corp., a Houston-based biotechnology company located in the Galleria area, uses proprietary technology to isolate, multiply and store their clients own stem cells to be used for regenerative therapy. This therapy has been proven effective with many conditions, including vascular (e.g. Raynauds Disease, kidney artery disease), autoimmune (e.g. arthritis, multiple sclerosis, lupus) and degenerative (e.g. Parkinsons, Alzheimers) diseases.

To get stem cells for a client, fat is extracted from the abdomen in a minimally invasive process that takes 15 30 minutes with no recovery time. This fat is then taken to Celltex, where the components of the fat are separated.

Celltex isolates the mesenchymal stem cells (MSCs) and places them in a nutrient-rich environment to grow. The initial extraction contains about 250,000 stem cells. Celltexs methods can produce one billion cells from that original extraction in as little as 5 weeks, making it unnecessary for clients to have a second extraction in most cases. The cells are frozen and banked at Celltexs lab, ready if and when the client needs another infusion, whether that is in three months or 30 years.

Adult [as opposed to embryonic] MSCs have the remarkable potential of migrating to different parts of the body, recognizing sites of injury and inflammation, and are then able to transform into many different types of cells, says Celltex Chairman and CEO David Eller.

Celltex takes great care in providing safe, pure cells to their clients. The Quality Control Dept,, headed up by QC Manager Kathy Gohlke, tests the cells at different stages throughout the process to ensure that no contaminants are present and that the cells are healthy and viable. Should contaminants be found at some point, which has only happened once out of about 500 clients served since 2011, a second fat extraction may be required.

Erik Eller, Head of Operations, explained that Celltex is also prepared for all kinds of negative scenarios so that the banked cells will stay safe. In the event of a long-term power failure, large generators located on site automatically provide electricity for up to two weeks. An even longer-term solution is in the works. The actual vats that the cells are stored in do not require electricity at all.

The labs at Celltex contain two clean rooms; one for manufacturing and another for Quality Control. These rooms are kept at a constant and optimal temperature, are pressure controlled and the air is continuously filtered through hospital-grade HEPA filters to reduce the chances of contamination. Employees who work in the clean rooms must be covered from head to toe in protective gear to keep the stem cells as healthy as possible.

When a client is ready for treatment, Celltex ships that persons harvested stem cells to either Guadalajara or Cancun Mexico, where the client will receive the infusion in a top-of-the-line hospital by a licensed physician.

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Houston-based company leads nation in medical wave of the future