Stem cell treatment may harbor blindness cure, says study

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

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?

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?

Cell Discovery Challenges Dogma on How Fetus Develops; Holds Insights for Liver Cancer and Regeneration

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Newswise (New York, NY Oct. 14, 2014) 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.

The research teams newfound, liver-based stem cell type has the ability to become part of newly formed blood vessels. Thus, a detailed understanding of it may have a decisive impact on understanding liver cancer progression, said Dr. Gouon-Evans. If similar bi-potential progenitor cells are found in liver cancers, they may be ideal targets for drugs that eradicate not only their descendant liver cancer cells but also the formation of blood vessels that feed tumors.

The new study also has implications for the field of liver regeneration. Many labs seek to understand how the liver repairs itself when damaged, and many clinical trials to determine whether injecting healthy liver cells into damaged livers can repair them.

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Cell Discovery Challenges Dogma on How Fetus Develops; Holds Insights for Liver Cancer and Regeneration

Stem cells successfully treat blindness

Published: 1:48PM Wednesday October 15, 2014 Source: AP

An experimental treatment for blindness that uses embryonic stem cells appears to be safe, and it improved vision in more than half of the patients who got it.

Researchers followed 18 patients for up to three years after treatment.

The studies are the first to show safety of an embryonic stem cell treatment in humans for such a long period.

"It's a wonderful first step but it doesn't prove that (stem cells) work," said Chris Mason, chair of regenerative medicine at University College London, who was not part of the research.

He said it was encouraging the studies proved the treatment is safe and dispelled fears about stem cells promoting tumour growth.

Embryonic stem cells, which are recovered from embryos, can become any cell in the body.

They are considered controversial by some because they involve destroying an embryo and some critics say adult stem cells, which are derived from tissue samples, should be used instead.

Scientists have long thought about transforming them into specific types of cells to help treat various diseases.

In the new research, scientists turned stem cells into retinal cells to treat people with macular degeneration or Stargardt's macular dystrophy, the leading causes of blindness in adults and children.

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Stem cells successfully treat blindness

Stem Cells Seem Safe in Treating Eye Disease

A treatment based on embryonic stem cells clears a key safety hurdle, and might help restore vision.

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.

Its all taken longer than they thought. But today, a Massachusetts biotech firm 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 today 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 Stargardts macular dystrophy.

The idea behind Advanced Cells treatment is to replace retinal pigment epithelium cells, known as RPE cells, a type of caretaker tissue without which a persons 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 Stargardts patients. But if the vision results seen so far continue, Lanza says this would be a therapy.

Some eye specialists said its too soon to say whether the vision improvements were real. The patients werent 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. Its 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 Cells Seem Safe in Treating Eye Disease

Stem Cell Therapy For ALS Gets FDA's Fast Track Designation

By C. Rajan, contributing writer

The U.S. FDA has just granted BrainStorm Cell Therapeutics novel stem cell therapy, NurOwn, Fast Track status for the treatment of amyotrophic lateral sclerosis (ALS), the company announced via press release.

"We are pleased that the FDA has granted Fast Track status for NurOwn as this will allow us greater and more frequent dialogue with the Agency as we continue the development of this ground-breaking cell therapy for the treatment of ALS," said Tony Fiorino, MD, PhD, CEO of BrainStorm. "We expect Fast Track designation, which recognizes the potential of NurOwn as to address an unmet medical need in ALS, to help speed and improve our development program."

Israeli biotech company BrainStorm is developing novel adult stem cell technologies for neurodegenerative diseases, such as ALS. The company licensed the exclusive rights to the NurOwn technology from Ramot, the technology transfer company of Tel Aviv University.

NurOwn is a personalized stem cell product made from autologous mesenchymal stem cells. These adult stem cells are obtained from the patients bone marrow and are induced to secrete neurotrophic factors, which are growth factors that can stimulate the survival and maintenance of neurons that degenerate in neurologic disorders.

NurOwn is currently being studied in randomized, double-blind, placebo-controlled phase 2 clinical trials in ALS patients in both Israel and the U.S. Reuters reports that the last patient visit has been completed in the phase 2a clinical trial in Jerusalem. The company expects to release final results of the study by the end of this year. The U.S. arm of the Phase 2 study is being conducted at three sites in the U.S., and is expected to be wrapped up in early 2015.

The FDA's Fast Track program aims to speed up the development of new drugs and biologics in order to get them to patients suffering from serious, unmet medical needs. The Fast Track designation will allow BrainStorm Cell to submit an NDA on a rolling basis and will grant the company more communication and support from FDA during the development process.

ALS, also known as Lou Gehrig's disease, is a rapidly progressive neurological disease that results in death within 2 to 5 years of diagnosis in most cases, and less than 20 percent of patients live more than 5 years after onset of symptoms. The relatively rare condition affects about 2 persons in every 100,000, with approximately 5,600 new cases diagnosed every year in the U.S, according to the ALS Association.

There is no cure for the disease to date, although the only approved ALS drug, Riluzole, has demonstrated its ability to extend survival by at least a few months.

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Stem Cell Therapy For ALS Gets FDA's Fast Track Designation