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First Stem Cell Drug Fails 2 Late-Stage Clinical Trials

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What might become the first drug derived from human stem cells failed in two late-stage clinical trials, dealing a setback to the drug’s developer and to the stem cell field (…)

Prochymal is a preparation of mesenchymal stem cells, which are obtained from the bone marrow of healthy young adults.
Because the cells are derived from adults, they sidestep the ethical issues stemming from the destruction of human embryos needed to make embryonic stem cells. Unlike most other types of adult stem cells, mesenchymal cells grow well in culture, so thousands of doses can be produced from a single donation.

Stem cells, particularly in the form of bone marrow transplants, are already used in medicine. Osiris is hoping that Prochymal will become the first stem cell product approved by the Food and Drug Administration and sold as a mass-produced pharmaceutical product (…)

from http://www.nytimes.com/2009/09/09/health/research/09drug.html

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Yale Endometrial Stem Cell Expert Joins Medistem’s Scientific Advisory Board

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Medistem Inc. announced the appointment of Dr. Hugh S. Taylor to its Scientific Advisory Board. Dr. Taylor is the first scientist to identify the bone marrow origin of endometrial tissue, and performed independent experiments demonstrating that endometrial stem cells are capable of treating diabetes and Parkinson’s Disease in animal models.

Dr. Taylor is Professor of Obstetrics, Gynecology, and Reproductive Sciences; Section Chief, Reproductive Endocrinology and Infertility; Director, Yale Center for Endometrium and Endometriosis and Director of the Yale Center for Reproductive Biology.

“Dr. Taylor has literally defined the field of endometrial stem cells. His fundamental discovery of the bone marrow origin of endometrial cells in humans was one of the major forces behind the now growing field of endometrial stem cell research,” said Thomas Ichim, CEO of Medistem. “We strongly believe that Dr. Taylor’s deep scientific understanding of biological mechanisms occurring in the endometrium will serve as a foundation for our clinical development programs.”

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Stem Cells Against False Joints

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The Thin Red Line (1854 battle)
Image via Wikipedia

Russian scientists started clinical trials of treating false joints by means of demineralized bone matrix with introduced mesenchymal stem cells of a patient.

False joints often occur as a complication during fractures of long bones, when splinters do not adhere, and cartilage layer forms between them. This layer is called false joint, and in this case, additional surgery is required to help a bone to heal.

Modern surgeons fight this problem with bone transplants, but bone recovery takes about one year. Possible solution is transplantation of mesenchymal stem cells of bone marrow, which can turn into various cells, including osteocytes. However, before differentiating cells require a substrate to attach to, and false joint has neither appropriate surface, nor nutrients for stem cells. In this case, the majority of stem cells dies or migrates away the desired place.

Russian scientists suggest putting stem cells on demineralized bone transplants, made of human long bones. Cell were extracted from patient’s bone marrow, cultivated in nutrient medium, and then inhabited bone matrix.

from Russia IC

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Stem-cell treatment offers hope to Colorado boys

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http://video.google.com/videoplay?docid=-1882394447517464643

Dominic King didn’t have much to say.
Worn out from horseback riding, the 10-year-old sat quietly on a recent summer afternoon, smiling, amused by his best friend, Harrison Spiers.
Harrison, for his part, was hosting a one-man yak-fest, and his topic was one any 10-year-old boy, or former 10-year-old boy, should relate to: baseball.

Neither boy, though, volunteered comment about what will surely be the most memorable event of their activity-packed summer: the trip to Germany they’re about to take. Asked about it, the boys shrug it off. No big deal. We’ve flown in airplanes before.
No big deal, except that this trip isn’t about a relaxing summer getaway to the land of bratwurst and the Brandenburg Gate.

This is a trip about hope.
Dominic and Harrison, friends, classmates since preschool, have cerebral palsy.

And after years of watching physical therapy and speech therapy, of watching their sons — who are as smart as anybody else’s 10-year-olds — struggle inside their own bodies, the boys’ parents have found, maybe, real promise.
That promise is a stem-cell treatment, one that isn’t available in the United States. So both boys and their families will head to the XCell Center in Köln, Germany, late in July.

There, technicians will collect stem cells from the boys’ bone marrow. According to the clinic, the marrow is then checked for quality and quantity of stem cells. Then, several days later, the cells are re-injected, through the spine.
“It’s not invasive, but they will have to be under anesthesia because you don’t want them to move around,” said Dominic’s mom, Christina King.

According to XCell’s website, “these re-injected stem cells have the potential to transform into multiple types of cells and are capable of regenerating damaged tissue.”
XCell statistics indicate that 70 percent of patients who undergo the treatment for cerebral palsy experience some improvement, including decreased spasticity, better coordination and motor function, improved stability or better speech.

Voicing tentative hope

Cerebral palsy is a group of chronic conditions that affect body movement and muscle coordination. It is caused by damage to the brain, usually during fetal development or during or shortly after birth.
The March of Dimes estimates it occurs in two or three of every 1,000 births.

For many of the 800,000 people in the United States who have it, the disorder causes difficulty controlling movement and speech. Some with the condition experience spasticity.
For Dominic and Harrison, the condition has meant life in wheelchairs. It means that Dominic often communicates via a computer and that Harrison doesn’t get to play football and baseball.

Neither boy’s intellect is impaired. As Harrison’s mother, Alicia Spiers, puts it, he understands everything he’s missing.
“But he has a really good attitude. He doesn’t get down too often,” she said.

The boys’ mothers can’t let themselves voice their hopes for the treatment.
Dominic and Harrison, with their 10-year-old trust, have no such hesitation.

“He’s hoping his speech becomes clearer,” Christina King said of Dominic.
And improved motor skills would be nice, she said. “He drives his wheelchair and uses his computer with his head. He’s hoping he can touch the computer screen.”

Harrison wants nothing less than to play football.
“His wish from Santa last Christmas was new stem cells,” Alicia Spiers said.

The boys, who just completed fourth grade at Cottonwood Creek Elementary in the Cherry Creek School District, spend most of their days in regular classrooms — aides help them eat, move around school and get their books out of their backpacks.
They go to birthday parties and have buddies they eat with, their moms said.

But the relationship between the boys is special.
“He has lots of friends,” Harrison’s mom said of her son. “But Dominic gets him.”

A community united

The treatment isn’t approved in the United States, and it definitely isn’t covered by insurance. So the families and their friends and relatives have been pitching in, hoping to raise $12,000 apiece for the treatments.
On June 14, a hair salon offered “Haircuts for Hope,” which raised about $1,600. Before that, runners and walkers tromped around the boys’ elementary school and raised hundreds.

“And some little girls at swim team made bracelets they sold for $1 each and are giving the money to us,” King said.
One of the girls’ moms told King she was learning a lot during the fundraising process, about cerebral palsy, about stem-cell treatment.

Both moms said they’ve learned a lot too — about their neighbors.
“We’re not alone,” King said. “This community is just wonderful.”

from Denver Post

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Face-surgery patients to grow their own bones

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BRITISH face surgeons are to grow new skull, cheek and jaw bones on patients’ backs using their own stem cells.
The surgeons, from Barts and the London NHS Trust, hope to use the technique to help people whose facial bones have been destroyed by cancer or injury.

Four patients are awaiting the treatment, which the surgeons believe could eventually become a less risky alternative to face transplants. Two are cancer victims and two have had accidents.
The team, led by Iain Hutchison, will make the first attempt to grow replacement bone from a patient’s own stem cells in Britain.

The procedure involves constructing a mould in the shape of the bone required and filling it with the patient’s bone marrow. This contains stem cells which can be persuaded to grow into different types of tissue. A genetically modified protein coaxes the stem cells to grow into bone. The mould is then inserted into the patient’s back muscles below the shoulder blade where it establishes a blood supply from the patient.

It is left to grow there for three to six months when it will be transplanted onto the patient’s face.
According to Hutchison, the stem cell procedure also has advantages over existing techniques of removing bones from other parts of the body. (…)

Read full article on Times Online

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Stem cells without genetic defects heralded as breakthrough

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The prospect of treating genetic diseases with corrected stem cells grown from patients’ own bodies has moved closer, after the results of a remarkable experiment.

Scientists have successfully reprogrammed skin tissue from people with a rare form of anaemia to create powerful stem cells, while at the same time rectifying the genetic defect that causes the condition.

The corrected stem cells could be grown into blood precursor cells for therapy. As these would carry a patient’s own DNA, except for the mutation responsible for the illness, they could be transplanted without risk of rejection by the body’s immune system.

Though the research team, from Spain and the United States, has yet to use the cells to treat patients, and several important hurdles still remain, the achievement has been hailed as a significant advance for stem cell research.

It suggests that it should eventually be possible to treat many inherited conditions by making disease-free stem cells from their own bodies. (…)

The cells were infected with a genetically modified virus to correct the gene that causes Fanconi anaemia. These were then reprogrammed into an embryo-like state by modifying further genes, to create versatile master cells known as induced pluripotent stem cells (IPS cells). (…)

Chris Mathew, Professor of Molecular Genetics at King’s College London, said: “This is an important development for families with this rare, inherited blood disorder. The patients have low numbers of blood stem cells in their bone marrow, so there are very few target cells to correct by gene therapy.

“The new research shows that it is possible to reprogramme skin cells from these patients into stem cells in which the genetic defect has been corrected. In future it may become possible to transfer the corrected stem cells back into the patient, but much work remains to be done.”

Read full article on Times Online

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