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Large Study of Stem Cells for Autism Draws Criticism

By Stem Cell Clinic

Experts say a $15 million trial to explore stem cells from cord blood for treating autism is premature.

Cold comfort: Researchers are trying to find out whether stem cells taken from frozen cord blood can improve autism symptoms. Credit:Tbsdy lives via Wikimedia Commons

A team at Duke University in Durham, North Carolina, is set to launch a $40 million clinical trial to explore stem cells from umbilical cord blood as a treatment for autism. But experts caution that the trial is premature.

A $15 million grant from the Marcus Foundation, a philanthropic funding organization based in Atlanta, will bankroll the first two years of the five-year trial, which also plans to test stem cell therapy for stroke and cerebral palsy. The autism arm of the trial aims to enroll 390 children and adults.

Joanne Kurtzberg, the trials lead investigator, has extensive experience studying the effectiveness of cord blood transplants for treating various disorders, such as leukemia and sickle cell anemia. Most recently, she showed that cord blood transplants can improve the odds of survival for babies deprived of oxygen at birth. A randomized trial of the approach for this condition is underway.

To really sort out if [stem] cells can treat these children, we need to do randomized, controlled trials that are well designed and well controlled, and thats what we intend to do, says Kurtzberg, professor of pediatrics and pathology at Duke. We firmly believe we should be moving ahead in the clinic.

Early animal studies have shown that stem cells isolated from umbilical cord blood can stimulate cells in the spinal cord to regrow their myelin layers, and in doing so help restore connections with surrounding cells. Autism is thought to result from impaired connectivity in the brain. Because of this, some groups of children with the disorder may benefit from a stem cell transplant, Kurtzberg says.

But others are skeptical of the approach. Autism is a complex disorder with many possible causes. Also, its unclear how stem cells derived from cord blood can improve connections in the brain. Given these important caveats, its too soon to conduct a test of this scale and investment, some experts say.

Its probably premature to run large trials without evidence that they have a therapeutic effect that [we] understand, cautions Arnold Kriegstein, director of the Broad Center of Regenerative Medicine and Stem Cell Research at the University of California, San Francisco.

Pilot trials In June, Kurtzberg launched the first phase of the trial, with 20 children between 2 and 5 years of age. Her team plans to infuse the children with a single dose of their own cord blood cells, banked at birth and preserved by freezing.

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Large Study of Stem Cells for Autism Draws Criticism

Failed stem cell treatment causes nasal growth on woman's spine

By Stem Cell Treatment

A woman who received stem cell treatment for paralysis needed a growth of nasal tissue removed from her spine eight years later.

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We're still learning about stem cells and what they can and can't do, so it's unsurprising that there will be a few strange accidents. One such accident happened to a woman who underwent stem cell treatment for paralysis.

Eight years ago, the anonymous woman, a US citizen, was treated at Hospital de Egas Moniz in Lisbon, Portugal, according to New Scientist. Doctors took stem cells from her nose and implanted them into her spine, hoping that the olfactory cells would develop into neural cells to help repair spinal nerve damage.

The operation was part of an early stage clinical trial exploring the potential of nasal cells in treating paralysis. Other researchers usually remove and isolate the cells, cultivating them in the lab before transplanting them, but the Lisbon team skipped this step and transplanted the cells directly.

The cells did grow -- but they remained olfactory cells, and the woman's pain worsened. Last year, surgeons removed a three-centimetre growth of nasal tissue, bone and nerve branches from the site; but it wasn't causing the pain by itself. The tissue was also producing mucus, which was pressing on her spine.

"It is sobering," Harvard Medical School stem cell researcher George Daley. "It speaks directly to how primitive our state of knowledge is about how cells integrate and divide and expand."

The Lisbon team published a paper in 2010 detailing the effects of the trial on 20 patients. Of those 20 -- out of an estimated 140 given the treatment to date -- eleven experienced improvement in their condition, one patient's condition worsened, one developed meningitis, and four others had minor adverse reactions.

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Failed stem cell treatment causes nasal growth on woman's spine

News Canada

By Stem Cell Treatment

OTTAWA -- She knows he's tall, slender and generous, but she does not know the name of the man who anonymously donated $128,000 so she can receive "life-saving" stem cell treatment in Chicago.

"We just can't believe it. The future is incredible right now," said Stephanie Headley, 47, who uses an oxygen tank due to damaged lungs.

The single mother of four was diagnosed in 2002 with an aggressive form of systemic scleroderma, an autoimmune disease that hardens the skin and organs.

Doctors didn't expect her to survive this long, but she credits a positive outlook and the support from her parents after her marriage fell apart.

Nonetheless, the disease is progressing and Headley was expecting to die sooner than later from heart failure.

Her family launched a fundraising campaign on youcaring.com, titled Please Save Our Mom, two months ago for a stem cell transplant at Northwestern Memorial Hospital.

Headley was stunned when a man delivered a $128,000 bank draft on July 3.

"He's given me my life," said Headley.

"He's saved my kids enormous pain."

She said the man phoned and wanted to stop by with a donation.

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Wisconsin Scientists Find Genetic Recipe To Turn Stem Cells To Blood

By Stem Cell Medicine

University of Wisconsin-Madison

The ability to reliably and safely make in the laboratory all of the different types of cells in human blood is one key step closer to reality.

Writing today in the journal Nature Communications, a group led by University of Wisconsin-Madison stem cell researcher Igor Slukvin reports the discovery of two genetic programs responsible for taking blank-slate stem cells and turning them into both red and the array of white cells that make up human blood.

[ Watch the Video: What Are Stem Cells? ]

The research is important because it identifies how nature itself makes blood products at the earliest stages of development. The discovery gives scientists the tools to make the cells themselves, investigate how blood cells develop and produce clinically relevant blood products.

This is the first demonstration of the production of different kinds of cells from human pluripotent stem cells using transcription factors, explains Slukvin, referencing the proteins that bind to DNA and control the flow of genetic information, which ultimately determines the developmental fate of undifferentiated stem cells.

During development, blood cells emerge in the aorta, a major blood vessel in the embryo. There, blood cells, including hematopoietic stem cells, are generated by budding from a unique population of what scientists call hemogenic endothelial cells. The new report identifies two distinct groups of transcription factors that can directly convert human stem cells into the hemogenic endothelial cells, which subsequently develop into various types of blood cells.

The factors identified by Slukvins group were capable of making the range of human blood cells, including white blood cells, red blood cells and megakaryocytes, commonly used blood products.

By overexpressing just two transcription factors, we can, in the laboratory dish, reproduce the sequence of events we see in the embryo where blood is made, says Slukvin of the Department of Pathology and Laboratory Medicine in the UW School of Medicine and Public Health and the Wisconsin National Primate Research Center.

The method developed by Slukvins group was shown to produce blood cells in abundance. For every million stem cells, the researchers were able to produce 30 million blood cells.

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Wisconsin Scientists Find Genetic Recipe To Turn Stem Cells To Blood

Can heart damage be fixed?

By Stem Cell Medical Center

STORY HIGHLIGHTS

(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.

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.

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Can heart damage be fixed?

Parkinson's stem cell effort holds fundraiser

By Stem Cell Treatment

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

By Stem Cell Doctors

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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?

By Stem Cell Treatment

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

By Stem Cell Medical Center

STORY HIGHLIGHTS

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.

Read the original here:
Heart attack damage may be reversible

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