Parkinson's stem cell effort holds fundraiser

Attendees at a fundraiser for Parkinson's patients enjoy food and the setting sun at the home of Jeffrey Strauss, owner of the Pamplemousse Grill. The proceeds help Summit4StemCell.org.

A bold experiment to relieve Parkinson's disease symptoms for many years faces a Nov. 4 deadline to raise a total of $2.5 million. That money will allow the group running the project to get matching funds from California's stem cell agency.

Thanks to the owner/chef of Pamplemousse Grill and a number of donors, the group just took a giant step toward that goal.

The group, Summit4StemCell.org, has been holding events for years to raise money to research the therapy, which will use skin cells from eight patients to form new brain cells. The cells will be implanted in the patients' brains to replace the cells destroyed in Parkinson's that make the neurotransmitter dopamine.

Supporters and some patients have climbed atop Mt. Kilimanjaro in Africa and to Base Camp at Mt. Everest, as well as holding local fundraisers. That money has advanced the research so it's feasible to try it in the patients. But more money is needed to pay for the treatment and related expenses.

So last week, supporters gathered at the home of Jeffrey Strauss, owner of Pamplemousse Grille in Solana Beach.

Proceeds from the $500-a-plate dinner brought in nearly $1 million for the project, said Sherrie Gould, an organizer and project sparkplug. Gould, a nurse practitioner at Scripps Clinic, interfaces between the clinical side, led by neurologist Melissa Houser of Scripps Clinic, and the research side, led by Jeanne Loring, a stem cell scientist at The Scripps Research Institute, and Andres Bratt-Leal of the Parkinsons Association of San Diego. The association is the nonprofit under which Summit4StemCell is held.

"Jeffrey is very close to a couple of our patients, and he literally opened up his home to about 130 people, completely provided food, drink, open bar, wine, wait staff, linens..." Gould said.

Counting in-kind donations, the total raised so far is about $1.5 million, meaning that Summit4StemCell needs to raise another $1 million by September, for a total of $2.5 million, Gould said. With that amount, the group can apply for $2.5 million in matching funds from the California Institute for Regenerative Medicine.

"Funding our mission has been at a grass-roots level, philanthropy, and a few minor grants," the project said in a fundraising email. "With over 900 people donating to the project we have been able to successfully biopsy the patients skin and create dopamine-producing neurons from eight of Dr. Houser's PD patients. We are now comparing cell lines with other researchers' lines, characterizing/purifying the cells, and most exciting: testing the neurons from our patients in an animal model of PD."

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Parkinson's stem cell effort holds fundraiser

The possible alternatives to bone marrow transplant

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AP Photo/Agapito Sanchez, Baylor College of Medicine

MONTREAL Finding a donor for a stem cell transplant is perhaps one of the most difficult things for a cancer patient.

This is because stem cells are one of the few things that patients cannot rely on their immediate family to donate, according to to Doctor Silvy Lachance, Director of the stem cell transplant program at Hpital Maisonneuve-Rosemont.

Of course, we first look within the family, she said.

But there is only 25 per cent chance of identifying a donor. If we dont find a donor within the family, we try the international donor registry.

According to the National Cancer Institute, bone marrow and peripheral blood stem cell transplantations are most commonly used to treat leukemia, lymphoma, neuroblastoma (a cancer that affects mostly infants and children) and multiple myeloma.

While they wait for a compatible donor, patients will be assigned a conditioning regiment, which may include radiation.

This conditioning regiment will be followed by the infusion of stem cells that are compatible with the recipient, said Lachance.

Yet, for most ethnic minorities or anyone of mixed-birth, the chances of finding an anonymous donor remain very difficult.

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The possible alternatives to bone marrow transplant

Can stem cell treatment reverse heart damage?

In medical school, Gerald Karpman was taught that when it comes to matters of the heart, what's done is done.

"If you survived the heart attack, you survived at the level that you were going to be," he recalls. "Whatever damage was done was permanent."

That thinking has prevailed until very recently, when studies involving a handful of patients showed an infusion of stem cells might help rebuild healthy hearts in heart attack survivors.

On March 7, Karpman joined that perilous club. A dermatologist in Camarillo, California, and a former marathon runner, the 66-year-old had a rigorous routine: eight to 10 miles of walking each day and a meticulous, meatless diet.

But that morning, sitting at his home computer, a pain kicked in.

"Within about 30 seconds, I was in extreme discomfort," recalls Karpman, who says it was worse than the kidney stones he once suffered. "I couldn't sit still. I mean even driving the car (to the hospital), I couldn't put a seat belt on; I'm just moving around, just trying to think of something else."

Karpman made it to Los Robles Hospital and Medical Center in Thousand Oaks, where doctors used stents to reopen an artery in his heart and save his life.

As he lay recovering, he took in some grim news: Nearly 20 percent of his heart muscle was dead, starved of oxygen. Dead heart tissue leaves a scar, interrupting the coordinated muscle action that makes the heart such an efficient pump.

A standard measure of the heart's pumping ability is the ejection fraction, the percentage of blood in the left ventricle that is pumped out with each heartbeat. A healthy ejection fraction is between 55 and 70, according to the American Heart Association. Karpman's was 30.

Damage as severe as what Karpman suffered carries a high risk of developing heart failure.

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Can stem cell treatment reverse heart damage?

Heart attack damage may be reversible

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For more, watch "Sanjay Gupta | M.D." on Saturday at 4:30 p.m. and Sunday at 7:30 a.m. ET.

(CNN) -- In medical school, Gerald Karpman was taught that when it comes to matters of the heart, what's done is done.

"If you survived the heart attack, you survived at the level that you were going to be," he recalls. "Whatever damage was done was permanent."

That thinking has prevailed until very recently, when studies involving a handful of patients showed an infusion of stem cells might help rebuild healthy hearts in heart attack survivors.

On March 7, Karpman joined that perilous club. A dermatologist in Camarillo, California, and a former marathon runner, the 66-year-old had a rigorous routine: eight to 10 miles of walking each day and a meticulous, meatless diet.

But that morning, sitting at his home computer, a pain kicked in.

"Within about 30 seconds, I was in extreme discomfort," recalls Karpman, who says it was worse than the kidney stones he once suffered. "I couldn't sit still. I mean even driving the car (to the hospital), I couldn't put a seat belt on; I'm just moving around, just trying to think of something else."

Karpman made it to Los Robles Hospital and Medical Center in Thousand Oaks, where doctors used stents to reopen an artery in his heart and save his life.

As he lay recovering, he took in some grim news: Nearly 20% of his heart muscle was dead, starved of oxygen. Dead heart tissue leaves a scar, interrupting the coordinated muscle action that makes the heart such an efficient pump.

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Heart attack damage may be reversible

How safe is stem cell therapy for children affected with autism spectrum disorder – Video


How safe is stem cell therapy for children affected with autism spectrum disorder
In conversation with Dr Alok Sharma (MS, MCh.) Professor of Neurosurgery Head of Department, LTMG Hospital LTM Medical College, Sion, Mumbai. Explains, How safe is stem cell therapy for...

By: Neurogen Brain and Spine Institute

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How safe is stem cell therapy for children affected with autism spectrum disorder - Video

Saved from amputation – how a stem cell gel rebuilt my shattered leg

Clive Randell, 57, injured his leg in a motorcycle accident in 2011 Thanks to a new stem cell procedure, he can now ride his bike again Stem cells taken from the pelvis are blended with gel to 'glue' the bone

By David Gerrie

Published: 16:01 EST, 12 July 2014 | Updated: 02:33 EST, 13 July 2014

A pioneering stem cell procedure to repair fractured bones could provide a lifeline for accident victims facing the amputation of a limb.

The development involves harvesting stem cells master cells that are able to transform into any kind of body tissue from the patients pelvis, blending them with a specially created gel and injecting the solution into the damaged bone.

One patient already benefiting is lifelong motorcycle enthusiast Clive Randell who suffered horrific injuries to his left leg when his Harley-Davidson was rammed by a car in 2011.

On yer bike: Clive Randell, 57, pictured with his 'saviour' Professor Anan Shetty at Kents Canterbury Christ Church University, can now ride his bike again after undergoing the new stem cell procedure

He suffered multiple open fractures, leaving bone protruding through the skin, and extensive skin loss. Doctors repeatedly told him his leg would have to be amputated.

Today, though, Clive, 57, is back on his feet and, astonishingly, also his bike thanks to the ground-breaking stem-cell treatment.

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Saved from amputation - how a stem cell gel rebuilt my shattered leg

California stem cell agency to review dealings with StemCells Inc.

Californias stem cell agency announced Wednesday it was taking steps to deal with a risk of conflict of interest two days after its former president took a position with a company that the state agency had been funding.

Newark, Calif.-based StemCells Inc. announced that Alan Trounson would be joining its board of directors a week after Trounson left the top spot at the California Institute for Regenerative Medicine, according to California Stem Cell Report, a blog that follows developments related to the state agency.

The movehas an appearance of a serious conflict of interest, said John M. Simpson, director of the stem cell project for Santa Monica-based nonprofit Consumer Watchdog, in an interview.

The thing smacks as if this is StemCells Inc. giving a payback to Trounson after the agency awarded $19 million to StemCells Inc. Thats just the way it looks, Simpson said. And I think they have to explain quite clearly why that is not the case.

In a statement, the state stem cell agency said it had not known of the move and learned of Trounson's new position from a press release.

We take even the appearance of conflicts of interest very seriously, the agencys current president, C. Randal Mills, said in a statement, adding that they would be taking actions to address mounting concerns. We understand that the appointment of CIRMs former president to the board of directors of a CIRM loan recipient creates a risk of a conflict of interest.

Under current law, it is illegal for Trounson to communicate with the stem cell agencys board members or employees on behalf of his company to further its interests. The restriction lasts one year; Simpson argued that such a restriction should last for two years.

The agency has also banned its employees and its governing board from communicating with Trounson about any matter regarding StemCells Inc., and will be reviewing all of its work relating to the company, which the state agency says is receiving funding for Alzheimers research.

Simpson praised the agencys actions, but pointed out that conflict of interest has been a recurrent issue between the state agency and the organizations it funds. The stem cell agency was created by Proposition 71 in 2004 to distribute $3 billion of taxpayer funds to companies and institutions performing stem cell research.

With some 90% of the agency's grants having gone to institutions with representatives on its board, Times columnist Michael Hiltzik wrote in a 2012 story, the agency has long been vulnerable to charges of conflicts of interest.

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California stem cell agency to review dealings with StemCells Inc.

Conflicts of interest at stem-cell agency yet again

Here we go again. In the decade since California voters established a one-of-a-kind state stem-cell research agency with $3 billion in bond funding, the agency has been in the news over and over because of conflicts of interests.

Members of the board governing the California Institute for Regenerative Medicine often are employed by institutions seeking board grants. While these members cant vote directly to give grants to their employers, the incentive for mutually beneficial voting is obvious. Thats why ethical complaints began almost as soon as the first grants were made.

Last year, finally, the institute added some strong new safeguards. Its key members then made the rounds at California newspapers to argue that it was time for the medias focus to shift to all the promising work that agency grants had yielded. Their argument seemed reasonable.

But this week, the U-T reported that recently resigned institute President Alan Trounson is joining the board of StemCells Inc. which got $19.4 million in grants from the agency.

Trounson hasnt broken any law, and agency officials are properly critical of his decision. But this still makes the stem-cell institute look shabby and the timing could hardly be worse. The original $3 billion in funding will be used up within three years.

Whether the institute seeks additional money from voters or the Legislature, its unsavory history will be hard to overcome.

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Conflicts of interest at stem-cell agency yet again

Patient-specific stem cells and personalized gene therapy

PUBLIC RELEASE DATE:

10-Jul-2014

Contact: Lucky Tran lt2549@cumc.columbia.edu 212-305-3689 Columbia University Medical Center

NEW YORK, NY (July 10, 2014) Columbia University Medical Center (CUMC) researchers have created a way to develop personalized gene therapies for patients with retinitis pigmentosa (RP), a leading cause of vision loss. The approach, the first of its kind, takes advantage of induced pluripotent stem (iPS) cell technology to transform skin cells into retinal cells, which are then used as a patient-specific model for disease study and preclinical testing.

Using this approach, researchers led by Stephen H. Tsang, MD, PhD, showed that a form of RP caused by mutations to the gene MFRP (membrane frizzled-related protein) disrupts the protein that gives retinal cells their structural integrity. They also showed that the effects of these mutations can be reversed with gene therapy. The approach could potentially be used to create personalized therapies for other forms of RP, as well as other genetic diseases. The paper was published recently in the online edition of Molecular Therapy, the official journal of the American Society for Gene & Cell Therapy.

"The use of patient-specific cell lines for testing the efficacy of gene therapy to precisely correct a patient's genetic deficiency provides yet another tool for advancing the field of personalized medicine," said Dr. Tsang, the Laszlo Z. Bito Associate Professor of Ophthalmology and associate professor of pathology and cell biology.

While RP can begin during infancy, the first symptoms typically emerge in early adulthood, starting with night blindness. As the disease progresses, affected individuals lose peripheral vision. In later stages, RP destroys photoreceptors in the macula, which is responsible for fine central vision. RP is estimated to affect at least 75,000 people in the United States and 1.5 million worldwide.

More than 60 different genes have been linked to RP, making it difficult to develop models to study the disease. Animal models, though useful, have significant limitations because of interspecies differences. Researchers also use human retinal cells from eye banks to study RP. As these cells reflect the end stage of the disease process, however, they reveal little about how the disease develops. There are no human tissue culture models of RP, as it would dangerous to harvest retinal cells from patients. Finally, human embryonic stem cells could be useful in RP research, but they are fraught with ethical, legal, and technical issues.

The use of iPS technology offers a way around these limitations and concerns. Researchers can induce the patient's own skin cells to revert to a more basic, embryonic stem celllike state. Such cells are "pluripotent," meaning that they can be transformed into specialized cells of various types.

In the current study, the CUMC team used iPS technology to transform skin cells taken from two RP patientseach with a different MFRP mutationinto retinal cells, creating patient-specific models for studying the disease and testing potential therapies.

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Patient-specific stem cells and personalized gene therapy

Stem cell researcher targets 'seeds' of breast cancer metastasis

For breast cancer patients, the era of personalized medicine may be just around the corner, thanks to recent advances by USC Stem Cell researcher Min Yu and scientists at Massachusetts General Hospital and Harvard Medical School.

In a July 11 study in Science, Yu and her colleagues report how they isolated breast cancer cells circulating through the blood streams of six patients. Some of these deadly cancer cells are the "seeds" of metastasis, which travel to and establish secondary tumors in vital organs such as the bone, lungs, liver and brain.

Yu and her colleagues managed to expand this small number of cancer cells in the laboratory over a period of more than six months, enabling the identification of new mutations and the evaluation of drug susceptibility.

If perfected, this technique could eventually allow doctors to do the same: use cancer cells isolated from patients' blood to monitor the progression of their diseases, pre-test drugs and personalize treatment plans accordingly.

In the six estrogen receptor-positive breast cancer patients in the study, the scientists found newly acquired mutations in the estrogen receptor gene (ESR1), PIK3CA gene and fibroblast growth factor receptor gene (FGFR2), among others. They then tested either alone or in combination several anticancer drugs that might target tumor cells with these mutations and identified which ones merit further study. In particular, the drug Ganetspib -- also known as STA-9090 -- appeared to be effective in killing tumor cells with the ESR1 mutation.

"Metastasis is the leading cause of cancer-related death," said Yu, assistant professor in the Department of Stem Cell Biology and Regenerative Medicine at the Keck School of Medicine of USC. "By understanding the unique biology of each individual patient's cancer, we can develop targeted drug therapies to slow or even stop their diseases in their tracks."

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The above story is based on materials provided by University of Southern California - Health Sciences. The original article was written by Cristy Lytal. Note: Materials may be edited for content and length.

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Stem cell researcher targets 'seeds' of breast cancer metastasis