Stem cell transplants hold hope for treating blindness

31 January 2012
By Rosemary Paxman
Appeared in BioNews 642

A clinical trial testing the safety of using human embryonic stem cell (hESC) in the treatment of progressive eye conditions has been performed in the USA.

The small scale phase I/II trial looked at the safety of transplanting hESCs into two people with different types of eye disease: one person with Stargardt's macular dystrophy and one person with dry age-related macular degeneration (AMD). The researchers found no signs of rejection or abnormal cell growth indicating that hESCs may one day be safely used for therapeutic use in humans.

Researchers at Advanced Cell Technology, a biotech company which develops stem cell-based technologies, along with the Jules Stein Eye Institute at the University of California, Los Angeles, cultivated the hESCs using mouse skin cells into cell types found inside the eye. The cells were then purified and subsequently injected into specific locations within the eye.

'The cells seem to have transplanted into both patients without abnormal proliferation, tumour formation, graft rejection or any untoward pathological reactions or safety signals', the researchers said. However, they added that follow-up studies were needed to further establish the safety of the procedure.

After the injections of stem cells, the patients also demonstrated a functional improvement in their vision. The patient with Stargardt's macular degeneration could previously only determine hand motions, but two weeks after the injections they could reportedly count fingers using their treated eye. The patient with AMD also reported an improvement in vision, although the researchers could not see that the cells had survived the procedure.

Professor Daniel Brison, co-director of the North West Embryonic Stem Cell Centre, Manchester, described the results as a 'very exciting moment for embryonic stem cell therapies'.

'Although the study is limited to safety considerations, very small in scope, and at a very early stage, this is nonetheless a ground breaking moment for embryonic stem cell therapies', he said.

Professor Chris Mason, chair of Regenerative Medicine Bioprocessing, University College London described the findings as 'only the start of gathering the necessary safety data before it is possible to test if the therapy will have an impact on patients' vision'.

'Overall the process of testing for safety and efficacy is likely to take a minimum of 5-10 years before the potential therapy could enter routine clinical practice', he said.

The study was published in the Lancet.

Stem cell treatment bears fruit

Published: January 28, 2012

Before treatment, the 51-year-old graphic artist was legally blind, unable to read a single letter on a standard eye chart. She has suffered from Stargardt’s disease, the most common form of macular degeneration in young patients, since she was a teenager, and it was getting progressively worse.

A second patient, aged 78, suffered from dry macular degeneration — the leading cause of blindness in the elderly — and could not even see well enough to go shopping.

But after being treated with stem cells from a donated human embryo, both women have improved dramatically, researchers revealed.

Stem cells are master cells that can differentiate into any of the 200 kinds of cells in the human body. Their results are the first-ever report of the medical use of stem cells taken from human embryos, making them crucial barometers of whether the controversial technique will ever find widespread therapeutic uses.

In a paper published online in The Lancet on Monday, physicians at the University of California, Los Angeles, and scientists at biotechnology company Advanced Cell Technology report that the first two patients in the clinical trial suffered no adverse health effects from the treatment and seem to have benefited from it.

A week after having cells derived from a days-old embryo injected into her eye, the graphic artist could count fingers, and after one month she could read the top five letters on the eye chart. She can see more color and contrast, has started using her computer, and for the first time in years can read her watch and thread a needle. The macular degeneration patient recently went to the mall for the first time in years.

Objections and risks

Using human embryonic stem cells for research or treatment has incited controversy for ethical and medical reasons. Some opponents argue that because removing stem cells from days-old human embryos almost always destroys the embryo, the technique amounts to murder.

ACT is the only company currently testing human embryonic stem cells in study patients. Last November, stem-cell pioneer Geron announced that it was halting what had been the first-ever clinical trial of the cells-testing them in patients with spinal cord injuries-and leaving the field.

When Robert Lanza, chief scientific officer of ACT, approached ophthalmic surgeon Steven Schwartz of UCLA about leading the clinical trial, Schwartz asked for ethical advice from two of his patients: elderly nuns. They gave him the go-ahead, he said last year.

Even scientists who support stem cell research argue that they could be dangerous to use therapeutically. The very property that makes them so valuable in research — stem cells can morph into any of the kinds of cells in the human body — also makes them risky. They can form teratomas, a type of tumor that arises when stem cells differentiate into a profusion of cell types. reuters

Published in The Express Tribune, January 28^th, 2012.

New stem cell therapy could be used to halt Huntington’s advance

Last Updated: Friday, January 20, 2012,16:26

 117  0  

 

Tags: Stem cell,  Huntington`s disease,  Brain disorder

 

Washington: A new technique has been developed by researchers which uses stem cells to deliver therapy that specifically targets the genetic abnormality found in Huntington’s disease.

Huntington’s is a hereditary brain disorder that causes progressive uncontrolled movements, dementia and death. This new approach might block Huntington’s from advancing.

Researchers think that the best chance to halt the disease’s progression will be to reduce or eliminate the mutant huntingtin (htt) protein found in the neurons of those with the disease.

“For the first time, we have been able to successfully deliver inhibitory RNA sequences from stem cells directly into neurons, significantly decreasing the synthesis of the abnormal huntingtin protein,” Jan A Nolta, principal investigator of the study and director of the UC Davis stem cell program and the UC Davis Institute for Regenerative Cures, was quoted as saying.
Huntington’s disease can be managed with medications, but currently there are no treatments for the physical, mental and behavioral decline of its victims.

“Our team has made a breakthrough that gives families affected by this disease hope that genetic therapy may one day become a reality,” Nolta said.

RNA interference (RNAi) technology has been shown to be highly effective at reducing htt protein levels and reversing disease symptoms in mouse models.

“Our challenge with RNA interference technology is to figure out how to deliver it into the human brain in a sustained, safe and effective manner,” said Nolta.

“We’re exploring how to use human stem cells to create RNAi production factories within the brain,” she said.

The findings of this study are available in the article ‘Examination of mesenchymal stem cell-mediated RNAi transfer to Huntington’s disease affected neuronal cells for reduction of huntingtin’ that has been published online in the journal Molecular and Cellular Neuroscience.

ANI

First Published: Friday, January 20, 2012, 16:26

Novel stem cell treatment may hold promise for type 1 diabetes

By Serena Gordon, HealthDay

Updated 21h 8m ago

• Comments

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A new type of stem cell treatment for people with type 1 diabetes appears to help re-educate rogue immune system cells, which allows cells in the pancreas to start producing insulin again.

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  Paul Sancya, AP
  The average daily dose of insulin dropped almost 39 percent after 12 weeks for the group with some beta cell function and 25 percent in those with no beta cell function.

Enlarge

Paul Sancya, AP

The average daily dose of insulin dropped almost 39 percent after 12 weeks for the group with some beta cell function and 25 percent in those with no beta cell function.

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The treatment, which combines a patient's immune system cells with stem cells from a donor's cord blood, even worked in people with long-standing diabetes who were believed to have no insulin-producing ability.

Although the treatment didn't wean anyone off insulin completely, average blood sugar levels dropped significantly, which would reduce the risk of long-term complications.

"Our study brings a new hope for people with type 1 diabetes. If we can control the autoimmunity, we may reverse the diabetes. We showed that the islets 1/8cells3/8 can start to work again," said Dr. Yong Zhao, an assistant professor in the section of endocrinology, diabetes and metabolism at the University of Illinois at Chicago.

This treatment could potentially be useful in other autoimmune diseases, such as lupus and rheumatoid arthritis.

"It's quite remarkable that this approach, based on the re-education of immune cells, might work so well. The concept is very intriguing, and the treatment seems to be so simple and so safe," said Dr. Luca Inverardi, deputy director of translational research at the Diabetes Research Institute, University of Miami School of Medicine.

But he's also "reasonably cautious," he said. "The follow-up is long, up to 40 weeks, but it's not long enough to declare victory against diabetes yet," said Inverardi.

Also, he noted that the study involved only 15 Chinese people, and that type 1 diabetes is a bit different in that population. He said he'd like to see larger studies with a more diverse population, followed for a longer time.

Results of the study were published online Jan. 9 in the journal BMC Medicine.

Type 1 diabetes, an autoimmune disease, occurs when the body's immune system cells mistakenly attack the insulin-producing (beta) cells in the pancreas. Because their beta cells don't produce enough or any insulin, people with type 1 diabetes have to replace the lost insulin through injections to survive.

Stopping that autoimmune attack appears to be crucial to any treatment that hopes to cure or reverse type 1 diabetes.

Zhao's team developed a completely new approach. They take blood from a patient and separate out the immune system cells (lymphocytes). They briefly expose those cells to stem cells from umbilical cord blood from an unrelated infant and return the lymphocytes alone to the patient's body. The researchers have dubbed this "Stem Cell Educator Therapy," because while exposed to the stem cells, the lymphocytes seem to relearn how they should behave.

The study participants, who were 15 to 41 years old, had had type 1 diabetes for an average of nine years. Six had some residual beta cell function and six did not. Both groups were given stem cell educator therapy. The other three people served as the control group.

The researchers measured C-peptide, a protein fragment that's a byproduct of insulin production, and found that the educator therapy group had improved levels of C-peptide at 12 weeks. These levels continued to improve until 24 weeks, and remained stable through the follow-up at 40 weeks. There were no changes in C-peptide in the control group.

The average daily dose of insulin dropped almost 39 percent after 12 weeks for the group with some beta cell function and 25 percent in those with no beta cell function, suggesting that the group with no beta cell function now produced insulin.

"That means if you stop the autoimmune reaction, you may see beta cell regeneration, or there might be other precursor cells in the pancreas. If these data are confirmed, this is a very provocative and remarkable finding," Inverardi said.

The average hemoglobin A1C level dropped 1.06 percent for those with residual beta cell function and 1.68 percent for those without beta cell function. A1C levels measure average blood sugar levels over two to three months, and people with type 1 diabetes are advised to maintain A1C levels below 7 percent. A drop of 1 percent in A1C levels can reduce the risk of complications.

This was an initial clinical trial designed to test for safety. Zhao said that in future trials he hopes that with additional treatments people might get off insulin altogether.

But, even if that's not possible, the recovery of some beta cell function would be welcome news. "In the absence of complete remission, there are very sizable advantages to having some beta cell function," Inverardi noted.

Both experts said the treatment appears safe, with no risk of rejection. No significant side effects were reported during the trial, other than some arm soreness where blood was taken and returned.

S. Korea to approve world's 2nd batch of stem cell-based drugs: sources

SEOUL, Jan. 8 (Yonhap) -- Two South Korean biotechnology firms are expected to receive government approval for their stem cell drugs this month, paving the way for the world's second batch of stem cell-based medicines to hit the market, sources said Sunday.

   The Korea Food and Drug Administration (KFDA) will likely approve "Cartistem" produced by Medipost Inc. for treatment of damaged cartilage in mid-January, they said. A stem cell-based anal fistula drug by Anterogen Co. will also receive official permission within this month.

   "We are currently reviewing documents additionally submitted by each company. Permission will be issued sooner or later," a KFDA official said on condition of anonymity.

   Medipost's Cartistem, in particular, is a drug for treating degenerative arthritis and knee cartilage defects.

   The drug uses stem cells from other people, not from patients, so that it can be mass-produced and its quality could be better maintained, experts said.

   If Cartistem and Anterogen's anal fistula treatment medicine get the green light, they could be commercialized within one or two months, according to market watchers.

   In July, South Korea became the world's first country to approve a stem cell-based drug named "Hearticellgram-AMI." The medicine for acute myocardial infarction is produced by FCB-Pharmicell based in Seongnam, south of Seoul.

FBI crackdown on unproven stem cell therapies

• 10:20 05 January 2012 by Peter Aldhous
• For similar stories, visit the Stem Cells Topic Guide

They are the modern equivalent of snake oil merchants: clinics that charge desperately ill people thousands for unproven stem cell "cures". Now the US federal government is cracking down on one of the most notorious – and the defendants include a scientist at a leading research university.

Vincent Dammai, of the Medical University of South Carolina in Charleston, is named in a federal indictment as part of a team that allegedly received more than $1.5 million from people with cancer and neurodegenerative diseases.

The charges follow an investigation by CBS News into Lawrence Stowe of Fort Worth, Texas.

Stowe was filmed claiming that infusions of stem cells, given by his associate Francisco Morales at a clinic in Mexico, could reverse symptoms of amyotrophic lateral sclerosis – a fatal and incurable form of motor neuron disease.

Dammai's role, according to the indictment, was to extract stem cells from umbilical cord blood collected by Jesus Alberto Ramon, a midwife in Del Rio, Texas. Dammai and Ramon allegedly worked with Global Laboratories of Scottsdale, Arizona, which supplied the cells to Morales. Fredda Branyon, who ran Global Laboratories, pleaded guilty to supplying an unapproved treatment in August.

Morales, Dammai and Ramon, all named in the indictment, were arrested in December. Stowe, named in a second indictment, is still at large and"considered a fugitive", according to the FBI.

Warning signal

The case underlines concerns that some mainstream researchers could be abetting clinics offering unproven stem cell therapies. "I'm personally very happy that the FBI and the Food and Drug Administration has stepped in," says Larry Goldstein, a stem cell biologist at the University of California, San Diego. "I hope that it serves as a warning signal."

While Dammai is alleged to have been a knowing participant, other scientists may have been duped into supplying cells that are later used by rogue clinics. To avoid this, biologists should ask for credentials when responding to requests for stem cell samples, argued bioethicists Zubin Master of the University of Alberta in Edmonton, Canada, and David Resnik of the National Institute of Environmental Health Sciences in Research Triangle Park, North Carolina, in the journal EMBO Reports last July (vol 12, p 992). They should also make recipients sign contracts detailing how the cells will be used, the pair added.

Internet searches reveal several hundred clinics offering unproven stem cell treatments, says Douglas Sipp of the RIKEN Center for Developmental Biology in Kobe, Japan. How many, if any, have obtained cells from mainstream biologists remains a mystery. "By nature these clinics are opaque and secretive," says Sipp. "We really don't know what's in the vials."