FDA clearance for Phase I/II using hESC’s to treat Macular Degeneration, Advanced Cell Technology (ACTC.OB)
The US FDA has lifted the clinical hold and cleared ACT’s Investigational New Drug (IND) application to (immediately) initiate a Phase I/II multicenter study using retinal cells derived from human embryonic stem cells (hESCs) to treat patients with Stargardt’s Macular Dystrophy (SMD).
The Phase I/II trial will be a prospective, open-label study that is designed to determine the safety and tolerability of the RPE cells following sub-retinal transplantation to patients with advanced SMD.
- A total of 12 patients will be enrolled in the study at multiple clinical sites, including the Casey Eye Institute in Portland, Oregon (headed by Dr. Peter Francis of the Oregon Health Sciences University); the University of Massachusetts Memorial Medical Center in Worcester, Massachusetts (headed by Dr. Shalesh Kaushal, Chair of the Department of Ophthalmology); and the UMDNJ – New Jersey Medical School in Newark, New Jersey (headed by Dr. Marco Zarbin, Chair, Institute of Ophthalmology and Visual Science).
Stargardt’s disease causes progressive vision loss, usually starting in children between 10 to 20 years of age. Eventually, blindness results from degeneration in the pigmented layer of the retina, called the retinal pigment epithelium (RPE), and followed by photoreceptor degradation.
- There is currently no treatment for Stargardt’s disease;
“Using stem cells, we can generate a virtually unlimited supply of healthy RPE cells, which are the first cells to die off in SMD and other forms of macular degeneration. We’ve tested these cells in animal models of eye disease. In rats, we’ve seen 100% improvement in visual performance over untreated animals without any adverse effects. Our studies showed that the cells were capable of extensive rescue of photoreceptors in animals that otherwise would have gone blind. Near-normal function was also achieved in a mouse model of Stargardt’s disease. Stargardt’s disease causes progressive vision loss, usually starting in children between 10 to 20 years of age. Eventually see a similar benefit in patients with macular degeneration, said Dr. Robert Lanza, ACT’s Chief Scientific Officer.
Among the most common causes of untreatable blindness in the world are degenerative diseases of the retina.
- As many as 30M people in the US and the EU suffer from macular degeneration, which represents a $25-30B worldwide market that has yet to be effectively addressed;
- Approximately 10% of people ages 66 to 74 will have symptoms of macular degeneration, the vast majority the “dry” form of AMD – which is currently untreatable;
- This prevalence increases to 30% in patients 75 to 85 years of age.
Earlier this year the FDA also granted Orphan Drug designation for ACT’s RPE cells and as a result is eligible to receive a number of benefits, including tax credits, access to grant funding for clinical trials, accelerated FDA approval, and allowance for marketing exclusivity after drug approval for a period of as long as 7 years.
- “Initiating our macular degeneration clinical trial represents a significant milestone in the progress of developing human embryonic stem cell-based therapies aimed at large worldwide markets,” said William M. Caldwell IV, ACT’s Chairman and CEO.