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Vision Quest: Stem Cells Treat Blinding Disease

By Stem Cell Medical Center

Powerful stem cells injected into the eyes of 18 patients with diseases causing progressive blindness have proven safe and dramatically improved the vision of some of the patients, scientists report.

Three years of follow up show that vision improved measurably in seven of the patients, the team at Advanced Cell Technology report in the Lancet medical journal. In some cases, the improvement was dramatic.

For instance, we treated a 75-year-old horse rancher who lives in Kansas, said Dr. Robert Lanza, chief medical officer for the Massachusetts-based company. The rancher had poor vision 20/400 in one eye.

Once month after treatment his vision had improved 10 lines (20/40) and he can even ride his horses again. Other patients report similarly dramatic improvements in their lives, Lanza added. For instance, they can use their computers or read their watch. Little things like that which we all take for granted have made a huge difference in the quality of their life.

Not all the patients improved and one even got worse. But overall, Lanzas team reported, the patients vision improved by three lines on a standard vision chart.

"They can use their computers or read their watch. Little things like that which we all take for granted have made a huge difference in the quality of their life.

The researchers treated only one eye in each patient. There was no improvement in vision in the untreated eyes.

The patients had either Stargardts disease, a common type of macular degeneration, or dry macular degeneration, which is the leading cause of blindness in the developed world. There are no treatments for either condition, and patients gradually lose vision over the years until they are, often, blind.

Lanzas team used human embryonic stem cells, made using human embryos. They are powerful cells, each one capable of giving rise to all the cells and tissues in the body. The ACT team took one cell from embryos at the eight-cell stage to make batches of these cells.

They reprogrammed them to make immature retinal cells, which they injected into the eyes of the patients. The hope is that the immature cells would take up the places of the degenerated cells and restore vision.

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Vision Quest: Stem Cells Treat Blinding Disease

Discovery of Repair Process After Heart Attack Suggests Potential for New Treatment Strategy

By Stem Cell Medical Center

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Newswise In a study that could point the way toward a new strategy for treating patients after a heart attack, UCLA stem cell researchers led by associate professor of medicine (cardiology) and Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research member Dr. Arjun Deb have discovered that some scar-forming cells in the heart, known as fibroblasts, have the ability to become endothelial cells (the cells that form blood vessels). The UCLA team also found that a drug could enhance this phenomenon and improve the repair process after a heart attack.

The findings are reported in the October 15, 2014 edition of the journal Nature.

It is well known that increasing the number of blood vessels in the injured heart following a heart attack improves its ability to heal, said Dr. Deb, the studys senior author. We know that scar tissue in the heart is associated with a poorer prognosis. Reversing or preventing scar tissue from forming has been one of the major challenges of cardiovascular medicine.

Heart disease remains a leading cause of death in the United States. Each year in this country, approximately 720,000 people experience a heart attack (roughly one every 30 seconds) and about 600,000 people die of heart disease.

A heart attack most commonly occurs when there is a sudden blockage of the flow of blood through a vessel in the heart. The portion of the heart muscle that fails to receive adequate blood dies and is replaced by non-functional scar tissue (a process known as fibrosis) reducing the ability of the heart to adequately pump blood. Once scar forms it is thought to persist throughout the lifetime of the individual. Scar-forming cells (fibroblasts) and blood vessel-forming cells (endothelial cells) exist in close proximity in the injured heart.

Several years ago, Dr. Deb and his colleagues were investigating the relationship between the fibroblasts and endothelial cells. Performing experiments in mice in which scar-forming cells in the heart were genetically labeled, the researchers unexpectedly discovered that many of the fibroblasts in the hearts injured region changed into endothelial cells and contributed directly to blood vessel formation, a phenomenon the research team coined mesenchymal-endothelial transition, or MEndoT.

The researchers identified a molecular mechanism regulating MEndoT and administering a small molecule to augment MEndoT led to less scarring and better healing of the heart. The researchers plan to test similar small molecules in other models to determine whether the strategy could potentially be translated for human benefit.

Our findings suggest the possibility of coaxing scar-forming cells in the heart to change their identity into blood vessel-forming cells, which could potentially be a useful approach for better heart repair, said Dr. Deb. There are remarkable similarities in the process of scarring in different organs after injury. Our hope is that this approach can be used to treat scar tissue in other organs as well.

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Discovery of Repair Process After Heart Attack Suggests Potential for New Treatment Strategy

Stem cells from human embryos prove safe, improve vision, study says

By Stem Cell Clinic

Published October 15, 2014

For the first time, researchers have created functioning human lung cells from stem cells.

The longest-running trial of stem cells derived from a human embryo found that the cells caused patients none of the problems scientists feared, such as forming tumors, and reversed partial blindness in about half the eyes receiving transplants, researchers reported on Tuesday.

The results, published in The Lancet, could help re-invigorate the controversial quest to harness stem cells, which have the ability to turn into any of the 200 kinds of human cells, to treat diseases.

In an accompanying commentary, Dr. Anthony Atala of the Wake Forest Institute for Regenerative Medicine called the work "a major accomplishment."

After intense excitement among scientists and the public about the promise of stem cells and ethical debates about destroying human embryos to obtain them, the field stumbled when a high-profile trial for spinal cord injury was halted by Geron Corp in 2011 and the interest of other companies waned.

The small study's main goal was assessing the safety of the transplanted cells. Called retinal pigment epithelial cells, they were created by taking stem cells from a days-old embryo created in a fertility clinic and inducing them to differentiate into the specialized cells.

The study "provides the first evidence, in humans with any disease, of the long-term safety and possible biologic activity" of cells derived from embryos, said co-author Dr. Robert Lanza, chief scientific officer of Advanced Cell Technology, which produced the cells and funded the study.

Nine patients with Stargardt's disease (which causes macular degeneration in childhood) and nine with dry age-related macular degeneration (a leading cause of adult blindness) received implants of the retinal cells in one eye. The other eye served as a control.

Four eyes developed cataracts and two became inflamed, probably due to the patients' age (median: 77) or the use of immune-supressing transplant drugs.

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Stem cells from human embryos prove safe, improve vision, study says

Stem cell, regenerative medicine policies to be discussed at Rice's Baker Institute

By Uncategorized

Dr. Deepak Srivastava, a leading biomedical research policy expert, will discuss "Stem Cells, Regenerative Medicine and Policy Impediments to the New Future" at Rice University's Baker Institute for Public Policy Oct. 21. The event is free and open to the public, but registration is required.

Who: Dr. Deepak Srivastava, the Baker Institute's nonresident scholar for biomedical research policy and the Younger Family Director and senior investigator at the Gladstone Institute of Cardiovascular Disease.

Neal Lane, the Malcolm Gillis University Professor, senior fellow in science and technology policy at Rice's Baker Institute for Public Policy and a professor of physics and astronomy, will give introductory remarks.

Stem cells and regenerative medicine are exciting and emerging fields of biomedical research, according to event organizers. Proposed applications include treating conditions such as blindness, diabetes and heart disease. Regenerative medicine could also help heal failing organ systems and replace damaged tissue. While these fields hold great promise for medicine, external factors limit and, in some cases, stall research, organizers said. Ethical controversies surrounding human embryonic stem cells, policy issues affecting federal and state funding and regulation, and economic pressures all play a role in determining the future of research.

In his presentation, Srivastava will explore the current and future potential of stem cells and regenerative medicine. Following the presentation, he will discuss policy challenges and opportunities with Lane.

The event is sponsored by the Baker Institute's Science and Technology Policy Program and the Health Policy Forum.

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Stem cell, regenerative medicine policies to be discussed at Rice's Baker Institute

Stem Cell Eye Treatment May Restore Vision

By Stem Cell Treatment

Antonio Regalado for MIT Technology Review 2014-10-15 19:15:44 UTC

When stem cells were first culled from human embryos sixteen years ago, scientists imagined they would soon be treating diabetes, heart disease, stroke, and many other diseases with cells manufactured in the lab.

It's all taken longer than they thought. But now, a Massachusetts biotech firm has reported results from the largest, and longest, human test of a treatment based on embryonic stem cells, saying it appears safe and may have partly restored vision to patients going blind from degenerative diseases.

Results of three-year study were described Tuesday in the Lancet by Advanced Cell Technology and collaborating eye specialists at the Jules Stein Eye Institute in Los Angeles who transplanted lab-grown cells into the eyes of nine people with macular degeneration and nine with Stargardt's macular dystrophy.

The idea behind Advanced Cell's treatment is to replace retinal pigment epithelium cells, known as RPE cells, a type of caretaker tissue without which a person's photoreceptors also die, with supplies grown in laboratory. It uses embryonic stem cells as a starting point, coaxing them to generate millions of specialized retina cells. In the study, each patient received a transplant of between 50,000 and 150,000 of those cells into one eye.

The main objective of the study was to prove the cells were safe. Beyond seeing no worrisome side effects, the researchers also noted some improvements in the patients. According to the researchers half of them improved enough to read two to three extra lines on an eye exam chart, results Robert Lanza, chief scientific officer of Advanced Cell, called remarkable.

"We have people saying things no one would make up, like 'Oh I can see the pattern on my furniture, or now I drive to the airport," he says. "Clearly there is something going on here."

Lanza stressed the need for a larger study, which he said the company hoped to launch later this year in Stargardt's patients. But if the vision results seen so far continue, Lanza says "this would be a therapy."

Some eye specialists said it's too soon to say whether the vision improvements were real. The patients weren't examined by independent specialists, they said, and eyesight in patients with low vision is notoriously difficult to measure. That leaves plenty of room for placebo effects or unconscious bias on the part of doctors.

"When someone gets a treatment, they try really hard to read the eye chart," says Stephen Tsang, a doctor at Columbia University who sees patients losing their vision to both diseases. It's common for patients to show quick improvements, he says, although typically not as large as what Advanced Cell is reporting.

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Stem Cell Eye Treatment May Restore Vision

Embryonic Stem Cell Therapy Shows Long-Term Effectiveness, Safety

By Stem Cell Treatment

Posted: Tuesday, October 14, 2014, 7:00 PM

TUESDAY, Oct. 14, 2014 (HealthDay News) -- A new study is the first to show the long-term safety of embryonic stem cell transplants to treat human disease.

The research involved 18 people who received the transplants to treat forms of macular degeneration, a leading cause of vision loss.

The transplants, which restored some sight in more than half of the patients, appeared safe up to three years after the procedure.

The study, funded by a U.S.-based company called Advanced Cell Technology, was published Oct. 14 in The Lancet.

"Embryonic stem cells have the potential to become any cell type in the body, but transplantation has been complicated by problems," lead author Dr. Robert Lanza, chief scientific officer at Advanced Cell Technology, said in a journal news release. Those problems include the rejection of the transplanted cells by the patient's immune system, as well as the danger that the cells might spur certain types of cancers called teratomas.

A teratoma is a type of cancer that occurs when stem cells develop into multiple types of cells and form incompatible tissues that can include teeth and hair.

As Lanza explained, because of these issues, scientists interested in embryonic stem cell therapy have tended to focused on sites in the body that typically do not produce a strong immune response. The eye is one such spot.

In the new study, human embryonic stem cells were first prompted to develop into eye cells called retinal pigment epithelial cells. They were then transplanted into nine people with Stargardt's macular dystrophy, and another nine with dry atrophic age-related macular degeneration.

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Embryonic Stem Cell Therapy Shows Long-Term Effectiveness, Safety

Kingston man hoping for a life-saving stem cell transplant in U.S.

By Stem Cell Doctors

CTV Ottawa Published Tuesday, October 14, 2014 4:36PM EDT Last Updated Tuesday, October 14, 2014 7:25PM EDT

Fourteen months ago, Mike Berry thought he had it all. He and his wife, Christine, has just welcomed their first child into the world.

Now Berry is wondering how much time he has left with his young son, Troy.

Starting about a year and a half ago, the 42-year-old Kingston native began noticing a shortness of breath. Eventually, he was diagnosed with systemic scleroderma.

The rare auto-immune disease is causing his body to produce too much collagen, essentially scarring and eating away at his internal organs. Hardest hit are his lungs which have developed an equally-rare secondary disease. He has now lost almost 60% of his lung capacity and has trouble walking up a flight of stairs.

It is life-threatening. People with symptoms of this severity can have a handful of years to live. In Mike Berry's case the onset has been particularly fast. "It's very aggressive and even surprising the doctors," he says.

There is no cure.

But there is hope. At Northwestern University in Chicago, Dr. Richard Burt has pioneered a stem cell therapy called Hematopoietic Stem Cell Transplant (HSCT). The patient's stem cells are harvested. Then the existing immune system is wiped out. The stem cells are then used to "reset" the immune system, hopefully disease-free, essentially halting the scleroderma in its tracks. Some patients have even experienced partial recovery of their lung function. The success rate is around 70%.

The catch is that it is expensive. The treatment alone costs $150,000 U.S. Transportation, lodgings, food, etc. is over and above that.

Treating scleroderma with HSCT is still considered experimental. It is not available or even approved in Canada. In previous cases, O.H.I.P. has declined to cover the out-of-country medical costs.

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Kingston man hoping for a life-saving stem cell transplant in U.S.

Penn Medicine researcher receives New Innovator Award from National Institutes of Health

By Uncategorized

PUBLIC RELEASE DATE:

13-Oct-2014

Contact: Karen Kreeger karen.kreeger@uphs.upenn.edu 215-349-5658 University of Pennsylvania School of Medicine @PennMedNews

PHILADELPHIA Roberto Bonasio, PhD, an assistant professor of Cell and Developmental Biology, Perelman School of Medicine at the University of Pennsylvania, and a core member of the Penn Epigenetics Program is one of the recipients of a 2014 New Innovator Award from the National Institutes of Health (NIH).

The NIH Director's New Innovator Award, totaling $1.5 million over five years for each of the 50 recipients this year, supports highly innovative research and creative, new investigators who exhibit strong potential to make great advances on a critical biomedical or behavioral research problem. The initiative, established in 2007, supports investigators who are within 10 years of their terminal degree or clinical residency, who have not yet received a research project grant (R01), or equivalent NIH grant, to conduct unusually innovative research.

Bonasio studies the molecular mechanisms of epigenetic memory, which are key to a number of biological processes, including embryonic development, cancer, stem cell pluripotency, and brain function. In particular, he will be looking at gene expression controlled by epigenetic pathways that alter the chemical structure of chromosomes and allow for multiple cell identities to arise from a single genome. These pathways are also critical in the brain and their improper functioning can cause mental retardation, cognitive decline, and psychiatric disorders.

Bonasio has chosen ants as a model system. With colleagues Shelley Berger, PhD, who directs the Penn Epigenetics program; postdoctoral mentor Danny Reinberg, PhD, New York University; and Jrgen Liebig, PhD, Arizona State University, Bonasio has established the ant Harpegnathos saltator as a laboratory model to study epigenetics, the process by which a single genome gives rise to a variety of physiological outcomes.

This phenomenon is particularly evident in ants, as they live in caste-based societies in which most of the individuals are sterile females, limited to highly specialized roles such as workers and soldiers. Only one queen and the relatively small contingent of male ants are fertile and able to reproduce. Yet despite such extreme differences in behavior and physical form, all females within the colony appear to be genetically identical.

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Also see the University of Pennsylvania release.

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Penn Medicine researcher receives New Innovator Award from National Institutes of Health

One MS patient's 'starting line' for stem cell therapy

By Uncategorized

By Richard M. Cohen

image courtesy Richard Cohen

I am one of twenty struggling every day with multiple sclerosis to be included in an innovative, phase one stem cell clinical trial at the Tisch MS Research Center of New York. Now theres a mouthful. Please let me explain. Many of us read tidbits about cell therapy and think it simply is space-age medicine that will be launched in the future.

In fact, we are at the starting line now, and the race has begun. A phase one trial tests safety. The group is small, and all are treated with the real thing. No placebos, sugar pills. The trial tests autologous cells, which mean our own. That eliminates rejection and alters risk. No new medical procedure comes risk-free, but the dangers are minimal. The stem cells are pulled from bone marrow harvested from our breast bones. Sounds hideous. It is not.

In this trial, the stem cells are infused directly into the spinal column. Nope. Not painful at all. Then we watch and wait. Results, if there are to be any, can take many months to show themselves. This particular procedure has never been used before. I was the first in the group to be treated, making me the first in the world to have this done. For more than forty years, I have lived with an illness that left no room for hope. Suddenly, that has changed, though change does not necessarily come easily.

The expectation game is dangerous. No one really knows what to expect from this experiment. My doctor makes that point over and over. Yet it is hard to control the fantasies that inevitably pop into my head. The possibility of restoring at least some vision when I have been legally blind for years is enticing, to say the least. I used to run and race or simply hike up country hills. Now I hobble on a cane. I am lucky if I can stay on my feet walking two city blocks. The possibility of restored mobility takes my breath away.

I know better than to go too far down these roads in my mind, but that visual journey is unavoidable. Maybe that is okay. Hope is a funny thing. We need something to hope for. Any doctor will tell you attitude is an important factor in fighting a disease. I have learned the power of remaining positive. We need fuel to keep the engine running. Those flights of fancy, imagining we can be better than we are, to some extent can become self-fulfilling prophecies.

This is an exciting period in the history of medicine. That probably has been said throughout the ages. Science does not stand still. No one can see around the bend. That may be what makes hope possible, the idea that there is something just out of sight that is revolutionary and good, just waiting for us to get there.

Richard M. Cohen writes Journey Man, an independent blog, also carried by The Huffington Post. Cohen is the author of Blindsided, published in 2004, which chronicled his battles with multiple sclerosis and cancer, and Strong at the Broken Places in 2008, both New York Times Best Sellers. Cohens latest book, I Want to Kill the Dog, was published in 2012. Cohen is married to journalist, Meredith Vieira, with whom he has three grown children.

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One MS patient's 'starting line' for stem cell therapy

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