Scientists report partial reversal of age-related degeneration in aged mice

Source: Dana-Farber Cancer Institute
Date: November 29, 2010

Summary:

Scientists at Dana-Farber Cancer Institute say they have for the first time partially reversed age-related degeneration in mice, resulting in new growth of the brain and testes, improved fertility, and the return of a lost cognitive function. In a report posted online by the journal Nature in advance of print publication, researchers led by Ronald A. DePinho, MD, said they achieved the milestone in aging science by engineering mice with a controllable telomerase gene. The telomerase enzyme maintains the protective caps called telomeres that shield the ends of chromosomes.

As humans age, low levels of telomerase are associated with progressive erosion of telomeres, which may then contribute to tissue degeneration and functional decline in the elderly. By creating mice with a telomerase switch, the researchers were able to generate prematurely aged mice. The switch allowed the scientists to find out whether reactivating telomerase in the animals would restore telomeres and mitigate the signs and symptoms of aging. The work showed a dramatic reversal of many aspects of aging, including reversal of brain disease and infertility.

Researchers said these results may provide new avenues for regenerative medicine, because they suggest that quiescent adult stem cells in severely aged tissues remain viable and can be reactivated to repair tissue damage. In addition, researchers said, these results may provide new avenues for regenerative medicine, because they suggest that quiescent adult stem cells in severely aged tissues remain viable and can be reactivated to repair tissue damage.

Coverage Summary: Advanced Cell Technology Receives FDA Clearance For Rare Disease

Below is a summary of media coverage about the announcement by Advanced Cell Technology Inc. that it received FDA approval for the first clinical trial Using embryonic stem cells to treat Stargardt disease, a form Macular Degeneration:

Associated Press, November 25, 2010 at 9:06 PM: "Test OK'd for stem-cell treatment of rare disease":

NEW YORK — For only the second time, the U.S. government has approved a test in people of a treatment using embryonic stem cells — this time for a rare disease that causes serious vision loss. Advanced Cell Technology (ACT), a biotechnology company based in Santa Monica., Calif., said the research should begin early next year, following the green light from the U.S. Food and Drug Administration.

...ACT's experiment will focus on Stargardt disease, which affects only about 30,000 Americans. But the company hopes the same approach will work for similar and more common eye disorders like age-related macular degeneration, which affects millions. Stargardt is an inherited disorder that attacks central vision used for tasks like reading and recognizing faces. Some patients go totally blind, even losing peripheral vision, while others are severely impaired and can only perceive light or see their hands moving in front of their faces.

...The disease typically starts in adolescence. The key problem is that impaired scavenger cells fail to remove toxic byproducts from the eye, allowing them to build up and kill other cells. There is no proven treatment. In the new study, 12 patients will be treated with healthy scavenger cells, created in a laboratory from human embryonic stem cells. This early phase of the research is primarily to test the safety of various doses, injecting only one eye of each patient.

Los Angeles Times, November 22, 2010, 2:02 p.m.: "FDA clears second clinical trial involving human embryonic stem cells":

Another therapy derived from human embryonic stem cells is headed for clinical trials. Advanced Cell Technology Inc. said Monday that the Food and Drug Administration has cleared the way for its Phase I/II trial of retinal cells for patients with Stargardt’s macular dystrophy, a childhood version of macular degeneration. Up to 12 patients will be enrolled at several sites across the country, including the Casey Eye Institute in Portland, Ore., the University of Massachusetts Memorial Medical Center in Worcester, and UMDNJ – New Jersey Medical School in Newark.

...Advanced Cell Technology hopes to treat these patients by giving them new retinal pigment epithelium cells to replace the ones that are lost to the disease. The therapy has restored vision in rats and mice, according to published studies. The Phase I/II trial is designed to test the safety and tolerability of the RPE cells in humans.

Reuters, November 22, 2010 5:20pm EST: "Second U.S. company gets stem cell go-ahead":

The U.S. Food and Drug Administration has approved the second human trial of human embryonic stem cells -- this one testing cells in people with a progressive form of blindness, the company said on Monday. Massachusetts-based Advanced Cell Technology said it would start testing its stem cell-based treatment on 12 patients with Stargardt's macular dystrophy. It is the second trial of human embryonic stem cells to be approved by the FDA this year. Last month Geron Corp enrolled the first patient in its study using the cells in people whose spinal cords have been crushed.

Time, November 22, 2010: "FDA Approves Second Trial of Stem-Cell Therapy":

For only the second time, the Food and Drug Administration approved a company's request to test an embryonic stem cell-based therapy on human patients. Advanced Cell Technology (ACT), based in Marlborough, Mass., will begin testing its retinal cell treatment this year in a dozen patients with Stargardt's macular dystrophy, an inherited degenerative eye disease that leads to blindness in children. ...ACT's trial will involve injecting retinal pigment epithelium (RPE) cells, which nurture the retina, into volunteers with the most advanced forms of Stargardt's, in an attempt to replace dying and no longer functioning photoreceptor cells. In animals, the infusion of healthy cells improved vision and rescued the function of some diseased cells.

New Scientist, 22 November 2010, 16:36 GMT: "Stem cell trial for blindness gets green light":

Twelve people left almost blind by a hereditary condition that strikes in childhood are to receive the world's first eye therapy derived from human embryonic stem cells (hESCs). The treatment is for Stargardt's macular dystrophy, which affects 1 in 8000 people in the US. Their sight deteriorates from around age 6 when retinal pigment epithelial cells (RPEs) start to die off rapidly, possibly due to a defective gene. Without RPEs to support and nourish them, adjacent photoreceptor cells which capture light signals, die too and blindness is the result. People in the trial will be those whose vision has deteriorated to the point where they can see the movement of their own hand, but little else. They will receive injections into their eyes of between 50,000 and 200,000 RPEs.

"The goal is to halt the rate of photoreceptor loss," says Robert Lanza, chief scientist at Advanced Cell Technology (ACT) of Worcester, Massachusetts, the company that has been developing the treatment since first turning hESCs into RPEs in 2004. By implanting new RPEs, which do not contain the defective gene, the team hopes to prevent further deterioration or perhaps even reverse it.

Boston Globe, November 22, 2010: "ACT wins FDA’s approval to test stem cell therapy":

Advanced Cell Technology, a stem cell company with a research laboratory in Marlborough, has received federal approval to test a human embryonic stem cell treatment on patients with a rare disease that causes blindness. ...ACT filed an investigational new drug application with the Food and Drug Administration a year ago to use retinal cells derived from human embryonic stem cells to treat patients with Stargardt’s, a rare genetic disease that affects about 30,000 people in the United States. The disease causes progressive vision loss, starting in patients between 10 and 20 years old, and results in blindness.

Rare disease reveals new path for creating stem cells

Source: University of Pennsylvania School of Medicine
Date: November 22, 2010

Summary:

PHILADELPHIA - Researchers at the University of Pennsylvania School of Medicine and Harvard Medical School and School of Dental Medicine have found that the mutation that causes a rare genetic disorder of bone formation (fibrodysplasia ossificans progressiva, or FOP) can reset an internal program to change cell fate, driving it back into an adult stem-cell stage. Immediate application for these findings is the field of tissue engineering and personalized medicine. It is conceivable that a transplant patient may one day have some of their own endothelial cells extracted, reprogrammed, and then grown into the desired tissue type for implantation. Host rejection would not be an issue.

New study into bladder regeneration heralds organ replacement treatment

Source: Children's Memorial Research Center
Date: November 22, 2010

Summary:

Researchers at Children's Memorial Research Center have developed a medical model for regenerating bladders using stem cells harvested from a patient’s own bone marrow. The research, published in STEM CELLS, is especially relevant for pediatric patients suffering from abnormally developed bladders, but also represents another step towards new organ replacement therapies.

URGENT ACTION ALERT: Contact Congress! Help Pass Federal Embryonic Stem Cell Research Funding!

To all readers of Ben's Stem Cell News:

A critical vote on federal stem cell research funding legislation--H.R. 873: The Stem Cell Research Enhancement Act of 2009--may be taking place in the House of Representatives soon! If you support stem cell research, please click on the title of this post, take a moment to complete the Genetics Policy Institute's FORM-LETTER, send it to your senators and representatives, post the letter on social networking sites, and forward it around to everyone you can! For the complete text of H.R. 873, see the "Federal Stem Cell Legislation" section under the LINKS column on Ben's Stem Cell News.

Help make a difference!

THANKS FOR YOUR SUPPORT!

Ben Kaplan
Publisher
Ben's Stem Cell News

Advanced Cell Technology Receives FDA Clearance For the First Clinical Trial Using Embryonic Stem Cells to Treat Macular Degeneration

Source: Advanced Cell Technology, Inc.
Date: November 22, 2010

Summary:

MARLBOROUGH, MA – Advanced Cell Technology, Inc. announced today that the US Food and Drug Administration (FDA) has cleared the Company’s Investigational New Drug (IND) application to immediately initiate a Phase I/II multicenter clinical trial using retinal cells derived from human embryonic stem cells (hESCs) to treat patients with Stargardt’s Macular Dystrophy (SMD), one of the most common forms of juvenile macular degeneration in the world. The decision removes the clinical hold that the FDA had placed on the trial.

Study of genetic disease reveals new path to cell reprogramming

Source: Harvard University
Date: November 21, 2010

Summary:

As debilitating as disease can be, sometimes it acts as a teacher. Researchers at Harvard Medical School and the Harvard School of Dental Medicine have found that by mimicking a rare genetic disorder in a dish they can rewind the internal clock of a mature cell and drive it back into an adult stem-cell stage. This new “stem cell” can then branch out into a variety of differentiated cell types, both in culture and in animal models. These findings appear online in Nature Medicine on Nov. 21.

Reprogrammed amniotic fluid cells can generate all types of body cells

Source: Max-Planck-Gesellschaft
Date: November 19, 2010

Summary:

High hopes rest on stem cells: one day, they may be used to treat many diseases. To date, embryos are the main source of these cells, but this raises ethical problems. Scientists at the Max Planck Institute for Molecular Genetics in Berlin have now managed to convert amniotic fluid cells into pluripotent stem cells. These amniotic fluid-derived iPS cells are hardly distinguishable from embryonic stem cells - however, they "remember" where they came from. (PLoS One, October 29, 2010)

StemCells plans chronic spinal cord injury trial

Source: Reuters
Posted: November 15, 2010 1:19 p.m. EST

Summary:

Reuters reports StemCells Inc. filed for Swiss regulatory approval to begin a clinical trial of its nerve stem cells in patients with spinal cord injuries:

LOS ANGELES - StemCells Inc. has filed for Swiss regulatory approval for the first clinical trial of its nerve stem cells in patients with spinal cord injuries as much as a year old, the company said. It expects to enroll about a dozen patients whose injuries are between three and 12 months old.

Umbilical Cord Cells May Treat Arthritis, Studies Suggest

Source: BioMed Central
Date: November 15, 2010

Summary:

Umbilical cord stem cells may be useful in the treatment of rheumatoid arthritis (RA). Animal and in vitro experiments, described in BioMed Central's open access journal Arthritis Research and Therapy, have shown that mesenchymal stem cells (MSCs) taken from umbilical cord blood can suppress inflammation and attenuate collagen-induced arthritis. The researchers took immune cells from RA patients and showed that the umbilical MSCs were able to suppress the cells' proliferation, invasive behavior and inflammatory responses. Systemic infusion of the umbilical MSCs into mice was shown to significantly reduce the severity of collagen-induced arthritis.

Bioengineers Provide Adult Stem Cells with Friendly Environment: Simultaneous Chemical, Electrical and Mechanical Cues

Source: University of California - San Diego
Date: November 16, 2010

Summary:

Bioengineers from the University of California, San Diego have achieved the “Triple Crown” of stem cell culture – they created an artificial environment for stem cells that simultaneously provides the chemical, mechanical and electrical cues necessary for stem cell growth and differentiation. Building better microenvironments for nurturing stem cells is critical for realizing the promises of stem-cell-based regenerative medicine, including cartilage for joint repair, cardiac cells for damaged hearts, and healthy skeletal myoblasts for muscular dystrophy patients. The advance could also lead to better model systems for fundamental stem cell research. This work appears in a paper published online in Advanced Functional Materials on November 13.

ReNeuron announces first patient treated in landmark stroke stem cell clinical trial.

Source: ReNeuron Group plc
Date: 16 November 2010

Summary:

Guildford, UK - ReNeuron Group plc today announces that the first patient has been treated with the Company’s ReN001 stem cell therapy for stroke in a ground-breaking UK clinical trial. The PISCES study (Pilot Investigation of Stem Cells in Stroke) is the world’s first fully regulated clinical trial of a neural stem cell therapy for disabled stroke patients. ReNeuron is the first company to have received regulatory approval for any stem cell-based clinical trial in the UK. Stroke is the third largest cause of death and the single largest cause of adult disability in the developed world.

StemCells, Inc. Files to Conduct Neural Stem Cell Trial in Chronic Spinal Cord Injury

Source: StemCells, Inc.
Date: November 15, 2010

Summary:

PALO ALTO, Calif., -- StemCells, Inc. announced today that is has filed an application with Swissmedic, the Swiss regulatory agency for therapeutic products, to conduct a clinical trial in Switzerland of the Company's HuCNS-SC(R) purified human neural stem cells in chronic spinal cord injury patients. If authorized, the study would enroll patients who are three to 12 months post-injury.

How do neural stem cells decide what to be -- and when?

Source: Duke University Medical Center
Date: November 15, 2010

Summary;

SINGAPORE – Researchers at Duke-NUS Graduate Medical School in Singapore have uncovered a novel feedback mechanism that controls the delicate balance of brain stem cells. Zif, a newly discovered protein, controls whether brain stem cells renew themselves as stem cells or differentiate into a dedicated type of neuron (nerve cell). In preclinical studies, the researchers showed that Zif is important for inhibiting overgrowth of neural stem cells in fruit flies (genus Drosophila) by ensuring that a proliferation factor (known as aPKC) maintains appropriate levels in neural stem cells. The study was published in the Nov. 16 issue of Developmental Cell journal.

Stem Cell Patch May Result in Improved Function Following Heart Attack

Source: University of Cincinnati Academic Health Center
Date: November 15, 2010

Summary:

University of Cincinnati researchers have found that applying a stem cell-infused patch together with overexpression of a specific cell instruction molecule promoted cell migration to damaged cardiac tissue following heart attack and resulted in improved function in animal models. The researchers also found that function improved more so than when stem cells were directly injected in heart tissue—a therapy that is being studied elsewhere. These findings are being presented for the first time at the American Heart Association’s Scientific Meeting in Chicago Nov. 15.

Embryonic stem cell culturing grows from art to science

Source: University of Wisconsin-Madison
Date: November 14, 2010

Summary:

Growing human embryonic stem cells in the lab is no small feat. Culturing the finicky, shape-shifting cells is labor intensive and, in some ways, more art than exact science. Now, however, a team of researchers at the University of Wisconsin-Madison reports the development of a fully defined culture system that promises a more uniform and, for cells destined for therapy, safer product. Writing this week (Nov. 14, 2010) in the journal Nature Methods, a team led by Laura Kiessling, a UW-Madison professor of chemistry, unveiled an inexpensive system that takes much of the guess work out of culturing the all-purpose cells.

Modeling Autism in a Lab Dish: Researchers Create Autistic Neuron Model

Source: Salk Institute for Biological Studies
Date: November 11, 2010

LA JOLLA, CA—A collaborative effort between researchers at the Salk Institute for Biological Studies and the University of California, San Diego, successfully used human induced pluripotent stem (iPS) cells derived from patients with Rett syndrome to replicate autism in the lab and study the molecular pathogenesis of the disease.

Neurons generated from Rett-iPS cells form fewer synapses, the specialized signal transmission points between brain cells. Synapses are shown in red and dendrites, which function as signal receivers, are shown in green. Their findings, published in the November 12, 2010, issue of Cell, revealed disease-specific cellular defects, such as fewer functional connections between Rett neurons, and demonstrated that these symptoms are reversible, raising the hope that, one day, autism maybe turn into a treatable condition.

Geron Announces Two Publications Demonstrating That Its Telomerase Inhibitor Drug Targets Cancer Stem Cells

Source: Geron Corporation
Date: November 11, 2010

Summary:

Geron Corporation today announced today announced the publication of preclinical data demonstrating that the company's telomerase inhibitor drug, imetelstat (GRN163L), currently in Phase 2 clinical trials, targets cancer stem cells from multiple myeloma, pancreatic and breast cancers.

The first publication shows the inhibitory effect of imetelstat on multiple myeloma cancer stem cells in vitro and in animal models of the human disease. The research was co-authored by Dr. William Matsui and colleagues at The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins and scientists from Geron and is published in the journal PLoS ONE.

The second publication demonstrates the inhibitory effect of imetelstat on cancer stem cells from breast and pancreatic tumor cell lines in vitro and in animal models of the human disease. The data, published in the journal Cancer Research.

Stem Cell Transplants in Mice Produce Lifelong Enhancement of Muscle Mass

Source: University of Colorado at Boulder
Date: November 10, 2010

Summary;

A University of Colorado at Boulder-led study shows that specific types of stem cells transplanted into the leg muscles of mice prevented the loss of muscle function and mass that normally occurs with aging, a finding with potential uses in treating humans with chronic, degenerative muscle diseases.

The experiments showed that when young host mice with limb muscle injuries were injected with muscle stem cells from young donor mice, the cells not only repaired the injury within days, they caused the treated muscle to double in mass and sustain itself through the lifetime of the transplanted mice. The study is published in the Nov. 10 issue of Science Translational Medicine.

News stories based on this news release were published in today's Daily Telegraph and Press Association.

Rogue Gene Hijacks Stem Cells to Jumpstart Human Cancer

Source: Queen Mary, University of London
Date: 9 November 2010

Summary:

A gene thought to be responsible for initiating human cancer has been identified by researchers at Barts and The London School of Medicine and Dentistry. The study - published online today (9 November) in the journal Cancer Research - paves the way for developing early cancer diagnostic tests, and finding new treatments that prevent or stop the spread of cancer cells at an early stage.

Researchers have shown that a gene called FOXM1 exploits the inherent self-renewal property of stem cells causing excessive cell proliferation. Using adult human stem cells isolated from mouth tissues the team demonstrated that normal stem cells engineered in the lab to express abnormal levels of FOXM1 gene, triggered excessive cell growth within a 3D tissue culture model system set up to mimic human tissue regeneration in the laboratory.

Scientists turn skin into blood

Source: McMaster University
Date: November 7, 2010

Summary:

In an important breakthrough, scientists at McMaster University have discovered how to make human blood from adult human skin. The discovery, published in the prestigious science journal Nature on Nov. 7, could mean that in the foreseeable future people needing blood for surgery, cancer treatment or treatment of other blood conditions like anemia will be able to have blood created from a patch of their own skin to provide transfusions. Clinical trials could begin as soon as 2012.

Scientists Find Nerve Cell Activity Drains Stem Cell Pool in Developing Brain

Source: Scripps Research Institute
Date: November 3, 2010

Summary:

As babies grow, their brain cells develop from a pool of stem cells—some stem cells continuously divide, replenishing the pool, whereas others morph into mature functioning nerve cells. Now researchers at The Scripps Research Institute have shown that as the newly formed nerve cells start firing electrical signals, this activity slows down stem cell division, emptying out the stem cell pool in favor of nerve cell formation.

The study, published in the November 4 issue of the journal Neuron, shows that brain activity controls the balance between stem cells and mature nerve cells and suggests that abnormal brain activity, as it occurs during seizures, may have long-lasting effects on brain development. The results also have implications for replacing brain cells that are damaged or lost through diseases such as Alzheimer's or Parkinson's disease.