Category Archives: Stem Cell Human Trials

New research published in The Lancet provides the first evidence of the medium-term to long-term safety and tolerability of transplanting human embryonic stem cells (hESCs) in humans. hESC transplants used to treat severe vision loss in 18 patients with different forms of macular degeneration appeared safe up to 3 years post-transplant, and the technology restored some sight in more than half of the patients. "Embryonic stem cells have the potential to become any cell type in the body, but transplantation has been complicated by problems including the risk of teratoma formation and immune rejection," explains lead author Professor Robert Lanza, Chief Scientific officer at Advanced Cell Technology in the USA. "As a result, immunoprivileged sites (that do not produce a strong immune response) such as the eye have become the first parts of the human body to benefit from this technology." In the two phase 1/2 studies, hESCs were differentiated into retinal pigment epithelium cells and transplanted into nine patients with Stargardt's macular dystrophy and nine patients with dry atrophic age-related macular degeneration, the leading causes of juvenile and adult blindness in the developed world, respectively. No effective treatments exist for either condition, and eventually the light-receiving (photoreceptor) cells of … Continue reading →

No effective treatments exist for either (AMD) or Stargardt's macular dystrophy, both of which can result in complete blindness caused by the loss of light-receiving photoreceptor cells in the retina. The new treatment uses stem cells to recreate a type of cell in the retina that supports those photoreceptors. Stem cells derived from embryos that are only a few days old have the ability to develop into any kind of tissue in the body. By bathing the stem cells in a specially formulated cocktail of chemicals the scientists were able to stimulate them into turning into fully mature retinal pigment epithelium cells. They were then transplanted directly into the eyes of patients suffering from blindness. Tests showed substantial improvement in 10 of 18 treated eyes. Eight patients were able to read more than 15 additional letters on a sight chart in their first year after treatment. "Embryonic stem cells have the potential to become any cell type in the body, but transplantation has been complicated by problems including the risk of tumour formation and immune rejection, said Professor Robert Lanza, chief scientific officer at the US company Advanced Cell Technology, which funded the research. "As a result, sites that do … Continue reading →

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 16 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 … Continue reading →

PUBLIC RELEASE DATE: 14-Oct-2014 Contact: Robert Lanza rlanza@advancedcell.com 508-756-1212 x315 The Lancet @TheLancet New research published in The Lancet provides the first evidence of the medium-term to long-term safety and tolerability of transplanting human embryonic stem cells (hESCs) in humans. hESC transplants used to treat severe vision loss in 18 patients with different forms of macular degeneration appeared safe up to 3 years post-transplant, and the technology restored some sight in more than half of the patients. "Embryonic stem cells have the potential to become any cell type in the body, but transplantation has been complicated by problems including the risk of teratoma formation[1] and immune rejection"[2], explains lead author Professor Robert Lanza, Chief Scientific officer at Advanced Cell Technology in the USA. "As a result, immunoprivileged sites (that do not produce a strong immune response) such as the eye have become the first parts of the human body to benefit from this technology."[2] In the two phase 1/2 studies, hESCs were differentiated into retinal pigment epithelium cells [3] and transplanted into nine patients with Stargardt's macular dystrophy and nine patients with dry atrophic age-related macular degeneration, the leading causes of juvenile and adult blindness in the developed world, respectively. … Continue reading →

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 … Continue reading →

Scientists may have discovered a method of treating type 1 diabetes that could mean the end of insulin injections. They say the treatment, having successfully cured mice of the disorder for six months and counting, is moving on to primate trials before testing is done on humans. Using an injection of stem cells -- which are able to develop into a range of different kind of cells -- researchers say the new method could regulate a diabetics blood sugar without any other form of treatment. The research was found by stem cell scientist Douglas Melton and a team of Harvard researchers and published in the journal Cell Thursday. You never know for sure that something like this is going to work until you've tested it numerous ways," Melton said in a press release. Weve given these cells three separate challenges with glucose in mice and they've responded appropriately. That was really exciting. Researchers grew billions of insulin-secreting, or beta, cells using stem cells from humans. They then injected the cells into diabetic mice, and observed their blood sugar levels, which remained at healthy levels after six months. Melton said he was driven to find a cure for diabetes ever since … Continue reading →

Chuck Bednar for redOrbit.com Your Universe Online Researchers writing in the October 9 edition of the journal Cell report they have for the first time successfully converted human embryonic stem cells into insulin-producing beta cells equivalent in nearly every way to regular, normally-functioning beta cells. The discovery, which was the work of a team led by Douglas Melton of the Harvard University Department of Stem Cell and Regenerative Biology and the Howard Hughes Medical Institute, is being hailed as a breakthrough in the search for an effective way to treat type 1 diabetes a disease which affects an estimated three million Americans each year. According to BBC News online health editor James Gallagher, Melton and his colleagues were able to produce hundreds of millions of the cells in their laboratory. Furthermore, their tests on mice demonstrated that the cells could treat the disease, which is caused when the immune system begins destroying the cells that are responsible for controlling blood glucose levels. Beta cells in the pancreas pump out insulin to bring down blood sugar levels, Gallagher said. But the bodys own immune system can turn against the beta cells, destroying them and leaving people with a potentially fatal disease … Continue reading →

Scientists believe they have made a giant leap forward in the quest to find an effective treatment for Type 1 diabetes. Using human embryonic stem cells as a starting point, experts have for the first time been able to produce human insulin-producing beta cells equivalent in almost every way to normally functioning beta cells in the kind of massive quantities needed for cell transplantation and pharmaceutical purposes. Doug Melton, Xander University professor at Harvard University, who led the work, said he hoped to have human transplantation trials using the cells under way within a few years. The stem cell-derived beta cells are currently undergoing trials in animal models, including non-human primates. Prof Melton, who is also co-director of the Harvard Stem Cell Institute, said the device being tested had so far protected beta cells implanted in mice from immune attack for many months. There have been previous reports of other labs deriving beta cell types from stem cells, he said. No other group has produced mature beta cells as suitable for use in patients. The biggest hurdle has been to get to glucose-sensing, insulin-secreting beta cells, and thats what our group has done. We are now just one pre-clinical step … Continue reading →

Research into a cure for diabetescould result in an end to insulin injections It has beenhailed as the biggest medical breakthrough since antibiotics Harvard researcher Doug Melton promised his children he'd find a cure Treatment involves making insulin-producing cells from stem cells Scientistshope to have human trials under way within a 'few years' By Fiona Macrae for the Daily Mail Published: 17:41 EST, 9 October 2014 | Updated: 04:45 EST, 10 October 2014 2.9k shares 204 View comments Scientists have hailed stem-cell research into a cure for diabetes as potentially the biggest medical breakthrough since antibiotics. It could result in an end to insulin injections, and to the disabling and deadly complications of the disease, such as strokes and heart attacks, blindness and kidney disease. The treatment, which involves making insulin-producing cells from stem cells, was described as a 'phenomenal accomplishment' that will 'leave a dent in the history of diabetes'. Scroll down for video Read this article: Could this stem cell breakthrough offer an end to diabetes? … Continue reading →

The design of the proposed clinical trial, Phase I/IIa Dose Escalation Safety and Efficacy Study of Human Embryonic Stem Cell-Derived Retinal Pigment Epithelium Cells Transplanted Subretinally in Patients with Advanced Dry-Form Age-Related Macular Degeneration with Geographic Atrophy, is based on a pre-IND meeting with the FDA and a series of earlier interactions with the agency. Patients will undergo a single transplantation and the study will explore three different doses of OpRegen. Following transplantation, the patients will be followed over 12 months at specified intervals and then at longer time periods, to evaluate the safety and tolerability of the product. A secondary objective of the clinical trial will be to explore the ability of transplanted OpRegen to engraft, survive, and moderate the disease progression. The filing of this IND is the culmination of 12 years of research and development starting at the Hadassah Human Embryonic Stem Cell Research Center at Hadassah University Medical Center, Jerusalem, Israel, under the direction of Prof. Benjamin Reubinoff, MD, PhD and continuing at Cell Cure Neurosciences Ltd., said Charles S. Irving Ph.D., Cell Cures CEO. We look forward to initiating the clinical trial that will, for the first time, utilize xeno-free grade human embryonic stem cell … Continue reading →