ACT Announces Data & Safety Monitoring Board (DSMB) Approval to Increase RPE Dosage for Stargardt’s Disease Patients in U.S...
Today : Tuesday 24 April 2012
Advanced Cell Technology, Inc. (“ACT”; OTCBB: ACTC), a leader in the field of regenerative medicine, announced today that the Data and Safety Monitoring Board (DSMB), an independent group of medical experts closely monitoring the Company’s three ongoing clinical trials, have recently authorized the Company to move forward with enrollment and treatment of additional patients with Stargardt’s disease (SMD). In the U.S. SMD trial, ACT will screen and enroll patients for the second cohort, who, in keeping with trial protocol, will be injected with 100,000 retinal pigment epithelial (RPE) cells - as compared with the 50,000 cell dose used in the patients of the first cohort. The Company has also been approved to treat the final two patients to round out the initial dosing arm in its European trial. The use of pluripotent stem cells to derive RPE cells, and the use of the resulting RPE cells for treating a wide range of macular degenerative disorders, are covered by a robust patent portfolio owned by ACT, including a number of issued broad patents in key world markets.
“DSMB authorization to move to the next higher dosage of cells in our U.S. clinical trial and complete the treatment of the first cohort of patient in our European trial represents yet another significant advancement for our clinical programs,” commented Gary Rabin, chairman and CEO of ACT. “We are pleased with the pace of progress and the continued finding of safety amongst the participants in both the U.S. and European trials. The results so far have been encouraging, and with our SMD programs having been granted ‘orphan’ medicinal product designation in both the U.S. and Europe, we look forward to eventually reaching a stage at which we can further avail ourselves of all the regulatory and financial benefits this designation brings.”
The three procedures comprising the first cohort of patients in the U.S. SMD trial were all conducted at University of California at Los Angeles (UCLA), by 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. The first procedure in the E.U. trial was conducted at Moorfields Eye Hospital in London, by a team of surgeons led by Professor James Bainbridge, consultant surgeon at Moorfields and Chair of Retinal Studies at University College London.
“We are gratified to be moving to the next stage in both of our SMD trials,” commented Robert Lanza, M.D., ACT’s chief scientific officer. “We remain very encouraged by the preliminary data in the first four SMD patients treated with the lowest dose of RPE cells at UCLA and Moorfields Eye Hospital. We are doubling the number of cells that will be transplanted in the next group of patients in the U.S. trial. We will be anxious to see if the higher dosage of RPE cells will impact visual function and photoreceptor rescue.”
ACT is conducting three clinical trials in the U.S. and Europe using hESC-derived RPE cells to treat forms of macular degeneration. Each trial will enroll a total of 12 patients, with cohorts of three patients each in an ascending dosage format. These trials are prospective, open-label studies, designed to determine the safety and tolerability of hESC-derived RPE cells following sub-retinal transplantation into patients with dry-AMD or Stargardt's macular dystrophy (SMD) at 12 months, the study’s primary endpoint. On January 20, 2012, the first SMD patient enrolled in the Company’s U.K. clinical trial was treated at Moorfields Eye Hospital in London. The final patient of the first cohort in the company’s SMD trial in the U.S. was treated on February 13, 2012.
Further information about patient eligibility for the dry AMD study and the concurrent study on SMD is also available on www.clinicaltrials.gov; ClinicalTrials.gov Identifiers: NCT01345006, NCT01469832 and NCT01344993.
