ACT Announces Positive Results from Two Clinical Trials Published in The Lancet Using Differentiated Stem Cell-Derived Retinal Pigment Epithelium (RPE) Cells for the Treatment of Macular Degeneration
Download PDF Phase 1/2 Clinical Trials of RPE Cells for the Treatment of Dry Age-Related Macular Degeneration and Stargardt’s Macular Degeneration Show Positive Long Term Safety Results and Signs of Visual Improvement
ACT to Host a Conference Call on Wednesday, October 15, 2014 at 4:30 PM Eastern Time. Interested Parties may access the call live by dialing (888) 264-3177 and using Conference ID 18428590. A Webcast is available at http://ift.tt/1qpE6B2
MARLBOROUGH, Mass.-- Advanced Cell Technology, Inc. ("ACT"; OTCBB: ACTC), a leader in the field of regenerative ophthalmology, announced today that Phase 1/2 clinical data published online in The Lancet demonstrate positive long-term safety results using ACT’s proprietary Retinal Pigment Epithelium (RPE) cells for the treatment of Stargardt’s macular degeneration (SMD) and dry age-related macular degeneration (AMD). The publication features data from 18 U.S.-based patients with at least six months of post-transplant follow-up.
“These study results represent an important milestone and strengthen our leadership position in regenerative ophthalmology,” said Paul K. Wotton, Ph.D., President and Chief Executive Officer. “We would like to thank the patients for their willingness to participate in these studies. Our findings underscore the potential to repair or replace tissues damaged from diseases. We plan to initiate comprehensive Phase 2 clinical trials for the treatment of both AMD and SMD, two disease states where there is currently no effective treatment.”
These two studies provide the first evidence of the mid- to long-term safety, survival, and potential biologic activity of pluripotent stem cell progeny into humans with any disease. In addition to showing no adverse safety issues related to the transplanted tissue, anatomic evidence confirmed successful engraftment of the RPE cells, which included increased pigmentation at the level of the RPE layer after transplantation in 13 of 18 patients.
Robert Lanza, M.D., Chief Scientific Officer of ACT and co-senior author of the paper, commented, “Diseases affecting the eye are attractive first-in-man applications for this type of investigational therapy due to the immune-privileged nature of the eye. Despite the degenerative nature of these diseases, the vision of 10 of 18 patients showed measurable improvement at the six month follow up, after transplantation of the RPE cells. Furthermore, the cells have been well tolerated for a median period of 22 months with two of the patients treated more than three years ago. We are pleased that there have been no serious safety issues attributable to the cells observed in any of the patients.”
Vision was measured using the widely accepted standard for visual acuity testing, the Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity exam.
Steven Schwartz, M.D., Ahmanson Professor of Ophthalmology at the David Geffen School of Medicine at UCLA and retina division chief at UCLA’s Jules Stein Eye Institute, principal investigator and lead author of the publication said “The data published in The Lancet support the potential safety and biological activity of stem cell-derived retinal tissue. Once again, surgical access to the subretinal space has proven safe. However, for the first time in humans, terminally differentiated stem cell progeny seem to survive, and do so without safety signals. Combined with the functional signals observed, these data suggest that this regenerative strategy should move forward. This is a hopeful and exciting time for ophthalmology and regenerative medicine.”
About the Trials
The SMD and dry AMD trials are prospective, open-label studies designed to evaluate the safety and tolerability of human embryonic stem cell (hESC)-derived RPE cells following sub-retinal transplantation into patients at 12 months, the studies’ primary endpoint. Three dose cohorts were treated for each condition in an ascending dosage format (50,000 cells, 100,000 cells, and 150,000 cells). Both the SMD and dry AMD patients had subretinal transplantation of fully-differentiated RPE cells derived from hESCs. In addition to the two clinical trials in the U.S., ACT is carrying out a Phase 1/2 clinical trial of hESC-derived stem cells for the treatment of SMD in the United Kingdom.
About Age-related Macular Degeneration
Age-related macular degeneration is the leading cause of vision loss in people over the age of 50, with late stage AMD affecting about 30 million people worldwide. Dry AMD, accounts for 90 percent of all AMD and occurs when light-sensitive cells in the macula, located in the center of the retina, slowly break down, causing vision loss. As the disease progresses, patients may have difficulty reading and recognizing faces. There is currently no proven medical therapy for dry AMD. Including the earlier stages of disease, the projected number of people worldwide with age-related macular degeneration in 2020 is 196 million, increasing to 288 million in 2040 underscoring the urgent need for new treatments.
About Stargardt’s Disease
Stargardt’s macular degeneration is a form of juvenile macular degeneration that affects vision in children and young adults between the ages of six and 20, with a prevalence of approximately one in 10,000 people. Loss of vision is an inevitable aspect of SMD, with more than half of patients experiencing vision loss in the range of 20/200-20/400. Like dry AMD, there are no treatments currently approved to prevent or slow the vision loss associated with SMD.
About Advanced Cell Technology, Inc.
Advanced Cell Technology, Inc., (ACT) is a clinical stage biotechnology company focused on the development and commercialization of regenerative medicine and cell therapy technology. ACT’s most advanced products are in clinical trials for the treatment of dry age-related macular degeneration, Stargardt’s macular degeneration and myopic macular degeneration. ACT’s preclinical programs involve cell therapies for the treatment of other ocular disorders and for diseases outside the field of ophthalmology, including autoimmune, inflammatory and wound healing-related disorders. ACT’s intellectual property portfolio includes pluripotent stem cell platforms – hESC and induced pluripotent stem cell (iPSC) – and other cell therapy research programs. For more information, visit www.advancedcell.com
Stem cells to treat blindness appear to be safe
LONDON 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, two early studies show.
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 tumor 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.
In each patient, the retinal cells were injected into the eye that had the worst vision. Ten of the 18 patients later reported they could see better with the treated eye than the other one. No safety problems were detected. The studies were paid for by the U.S. company that developed the treatment, Advanced Cell Technology, and were published online Tuesday in the journal, Lancet.
Dr. Robert Lanza, one of the study authors, said it was significant the stem cells survived years after the transplant and weren't wiped out by the patients' own immune systems. For some of the patients, Lanza noted their improved vision changed their lives, referring to a 75-year-old horse rancher who had been blind in one eye before the treatment.
"One month after his treatment, his vision had improved (substantially) and he can even ride his horses again," Lanza said in an email. He said other patients have regained their independence with their newfound vision and said some people are now able to use their computers again, read their watches or travel on their own.
"The next step will be to prove these (stem cell) treatments actually work," Mason said. "Unless there is a sham group where you inject saline into (patients') eyes, we can't know for sure that it was the stem cells that were responsible."
Submitted October 14, 2014 at 06:48PM by eigenman http://ift.tt/1wCfQPT
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