Advanced Cell Technology, Inc., or
ACT, a provider of regenerative medicine, has announced that the Data and Safety
Monitoring Board, or 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, or SMD.
In the US 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 patent portfolio including a number of issued broad patents in
key world markets.
The three procedures comprising the first cohort of patients in the US SMD trial
were all conducted at University of California at Los Angeles (UCLA), 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.
ACT is conducting three clinical trials in the US 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 UK 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 US was treated on February 13, 2012.
"DSMB authorization to move to the next higher dosage of cells in our US
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 US 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 US 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."
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