Stem Cell Clinic

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

Marius Wernig receives New York Stem Cell Foundation's Robertson Stem Cell Prize

By Uncategorized

PUBLIC RELEASE DATE:

14-Oct-2014

Contact: David McKeon DMcKeon@nyscf.org 212-365-7440 New York Stem Cell Foundation @nyscf

NEW YORK, NY (October 14, 2014) The New York Stem Cell Foundation (NYSCF) announced today that Marius Wernig, PhD, Associate Professor in the Institute for Stem Cell Biology and Regenerative Medicine and the Department of Pathology at Stanford University School of Medicine, is the 2014 recipient of the NYSCF Robertson Stem Cell Prize, which has been awarded since 2011 for extraordinary achievements in translational stem cell research by a young scientist.

Dr. Wernig and his team discovered that human skin cells can be converted directly into functional neurons, termed induced neuronal (iN) cells, in a period of four to five weeks with the addition of just four proteins.

"Dr. Wernig's groundbreaking research has the potential to accelerate all research on devastating neurodegenerative diseases," said Susan L. Solomon, CEO and Co-founder of NYSCF. "His work can impact and accelerate research on multiple sclerosis, Alzheimer's disease, and autism among many other conditions."

At Stanford, Dr. Wernig focuses on using induced pluripotent stem (iPS) cells and iN cells for disease modeling and as potential cellular therapy. This new technique transformed the field of cellular reprogramming by eliminating the need to first create iPS cells, making it easier to generate patient or disease-specific neurons. These cell types hold tremendous therapeutic and translational relevance for patients around the world. Potential applications range from replacing damaged brain tissue to repairing the myelinating nerves lost in multiple sclerosis to identifying novel drugs and treatments for a range of neurological diseases.

In addition to his recent scientific achievements, Dr. Wernig was part of the inaugural class of NYSCF Robertson Stem Cell Investigators in 2010, and is the first NYSCF Robertson Investigator to receive the NYSCF Robertson Stem Cell Prize.

"I am delighted that Dr. Wernig is being recognized with this year's NYSCF Robertson Prize for his important research that has opened entirely new avenues for studying brain diseases. The NYSCF Robertson Prize was created to acknowledge the most important work being down by young stem cell scientists and I am thrilled to see a NYSCF Robertson Investigator go on to receive NYSCF Robertson Prize," said Julian Robertson, whose foundation underwrites the $200,000 prize. The terms of the prize require that the $200,000 stipend be used, at the recipients' discretion, to further support their research.

The NYSCF Robertson Stem Cell Prize will be presented to Dr. Wernig at a ceremony in New York City by Susan L. Solomon on October 14th.

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Marius Wernig receives New York Stem Cell Foundation's Robertson Stem Cell Prize

Stem cell treatment may harbor blindness cure, says study

By Stem Cell Treatment

A new study has revealed that stem cell treatment may be helpful in treating blindness.According to the study, a pioneering treatment for progressive blindness has been proved safe three years after patients were injected with stem cells derived from human embryos.

The researchers said that more than half of the patients with macular degeneration where the eye's light-sensitive cells are progressively destroyed experienced a significant improvement in their eyesight, but none showed any adverse effects due directly to the transplant of the embryonic cells.

Doctors injected the stem cells into the eyes of 18 patients nine with Stargardt's macular dystrophy and nine with dry, age-related macular degeneration with the ultimate aim of repairing damaged photoreceptors in the retina at the back of the eye.

It was found that about half of the patients had an improvement in visual acuity of three lines or more, which corresponds to a doubling of the visual angle, and is generally accepted as clinically significant.Follow-up testing found that 10 out of the 18 patients experienced substantial improvements in how well they could see.

The study was published in the journal The Lancet.

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Stem cell treatment may harbor blindness cure, says study

Stem cell discovery challenges dogma on how fetus develops; holds insights for liver cancer and reg

By Stem Cell Treatment

PUBLIC RELEASE DATE:

14-Oct-2014

Contact: Greg Williams newsmedia@mssm.edu 212-241-9200 The Mount Sinai Hospital / Mount Sinai School of Medicine @mountsinainyc

A Mount Sinai-led research team has discovered a new kind of stem cell that can become either a liver cell or a cell that lines liver blood vessels, according to a study published today in the journal Stem Cell Reports. The existence of such a cell type contradicts current theory on how organs arise from cell layers in the embryo, and may hold clues to origins of, and future treatment for, liver cancer.

Thanks to stem cells, humans develop from a single cell into a complex being made up of more than 200 cell types. The original, single human stem cell, the fertilized embryo, has the potential to develop into every kind of human cell. Stem cells multiply (proliferate) and specialize (differentiate) until millions of functional cells result, including liver cells (hepatocytes), blood vessel cells (endothelial cells), muscle cells, bone cells, etc.

In the womb, the human embryo early on becomes three "germ" layers of stem cells the endoderm, mesoderm and ectoderm. The long-held consensus was that the endoderm goes on to form the liver and other gut organs; the mesoderm the heart, muscles and blood cells; and the ectoderm the brain and skin. Researchers have sought to determine the germ layer that yields each organ because these origins hold clues to healthy function and disease mechanisms in adults.

"We found a stem cell that can become either a liver cell, which is thought to originate in the endoderm, or an endothelial cell that helps to from a blood vessel, which was thought to derive from the mesoderm," said Valerie Gouon-Evans, PhD, Assistant Professor in the Department of Developmental and Regenerative Biology and Black Family Stem Cell Institute, Icahn School of Medicine at Mount Sinai, and lead author for the study. "Our results go against traditional germ layer theory, which holds that a stem cell can only go on to become cell types in line with the germ layer that stem cell came from. Endothelial cells may arise from both the endoderm and mesoderm."

Cell Growth Plusses and Minuses

Beyond the womb, many human organs contain pools of partially differentiated stem cells, which are ready to differentiate into specific replacement cells as needed. Among these are stem cells that "know" they are liver cells, but have enough "stemness" to become more than one cell type.

By advancing the understanding of stem cell processes in the liver, the study offers insights into mechanisms that drive liver cancer. The rapid growth seen in cells as the fetal liver develops is similar in some ways to the growth seen in tumors. Among the factors that make both possible is the building of blood vessels that supply nutrients and oxygen.

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Stem cell discovery challenges dogma on how fetus develops; holds insights for liver cancer and reg

Could stem cell jab help elderly blind see again?

By Stem Cell Treatment

Elderly people who received treatment had their vision improved, study says Children who suffer from common form of blindness in young also benefited Some can now do things like read their watch and also work on a computer Expert said even small improvements are 'huge difference to quality of life' Critics say it's wrong to plunder unborn child for spare parts for science

By Fiona Macrae Science Correspondent

Published: 18:28 EST, 14 October 2014 | Updated: 02:30 EST, 15 October 2014

A revolutionary stem cell jab has restored the gift of sight, research suggests.

Men and women with severe age-related macular degeneration, the most common form of blindness in the elderly, are able to see better after having tens of thousands of embryonic stem cells injected into the back of their eye.

Children with Stargardts disease, the main cause of blindness in the young, have also benefited.

Researcher Robert Lanza said that one patient who underwent the trial even 'went to the mall for the first time' (file photo)

Some can now do things most of us take for granted like reading their watch or working on a computer. But one man is able to ride horses again and one of the patients has gone to a shopping mall for the first time.

Researcher Robert Lanza, a world-leading stem cell expert, said that even seemingly small improvements have made a huge difference to quality of life. Others described his work as a major accomplishment.

All of those who took part in the landmark trial had advanced eye disease and were blind in one eye. However, Dr Lanzas goal is to treat people early in the disease process to stop them from ever going blind.

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Could stem cell jab help elderly blind see again?

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