Stem cell trial aims to cure macular degeneration
pharmafile | June 17, 2011 | News story | Research and Development | AMD, Advanced Cell Technology, Stargardt’s Macular Dystrophy, Stem cells, stem cell
A groundbreaking trial to explore the use of stem cells to treat macular degeneration has been launched.
US company Advanced Cell Technology is now enrolling patients in two phase I/II clinical trials for Stargardt’s Macular Dystrophy (SMD) and Dry Age-Related Macular Degeneration (Dry AMD) using eye cells derived from human embryonic stem cells (hESCs).
The trials are open-label studies to determine the safety and tolerability of the stem cells following sub-retinal transplantation into patients with SMD and Dry AMD. Each study will enrol 12 patients and last a year.
Once the safety profile of the treatment is established, further trials would then look at the effectiveness of the procedure.
“The enrolment of the first patients in our two clinical trials marks an important step forward for the field of regenerative medicine,” said Gary Rabin, interim chairman and chief executive of ACT. “We are very pleased with the progress that has been made toward bringing this ground-breaking technology to the patients who need it most.
“If these therapies work as we hope they will, particularly with small volumes of cells, then we should be in an excellent position to take advantage of our patented techniques for manufacturing large numbers of doses of RPE cells that can be conveniently stored and shipped to clinicians following the basic manufacturing and distribution systems already familiar to pharmaceutical and biotech companies.”
Principal investigator Steven Schwartz, retina division chief at the Jules Stein Eye Institute at UCLA, said: “These trials mark a significant step toward addressing what is one of the largest unmet medical needs of our time – treatments for otherwise untreatable and common forms of legal blindness, Dry AMD, SMD and other forms of atrophic macular degeneration.”
Dry AMD is the most common form of macular degeneration, and the leading cause of blindness in the developed world, with cases expected to double over the next 20 years as the population ages.
Numerous other research projects involving stem cell treatments are focusing on treating different eyes diseases and forms of blindness.
Last year AstraZeneca and University College London announced a stem cell research partnership aimed at repairing eyes damaged by diabetic retinopathy.
Andrew McConaghie
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