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Thursday, November 19, 2009
ACT seeks FDA OK for stem cell study to treat blindness
By Rodney H. Brown
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Worcester-based biotech Advanced Cell Technology Inc. has filed an investigational new drug application to use human embryonic stem cells to treat an untreatable disease that is the most common cause of juvenile blindness.
ACT has filed the IND with the U.S. Food and Drug Administration to begin a consolidated Phase 1 and 2 clinical trial to treat patients with Stargardt’s Macular Dystrophy in a multicenter study. According to ACT chief scientific officer Robert Lanza, Stargardt’s is one of a number of similar conditions, such as age-related macular degeneration (AMD), that cause vision impairment or blindness because of the loss of certain cells in the retina.
“There are literally dozens of types of different macular degenerative or retinal degenerative diseases,” Lanza said. “For all of these diseases, one of the main causes of the blindness is the loss of the retinal pigment epithelium cells. If you lose those, you lose your vision.”
What ACT has found is that by using retinal cells derived from human embryonic stem cells, they can restore vision in animal models suffering from Stargardt’s. According to Lanza, in a Royal College of Surgeons study using rats, implantation of stem cell-derived RPE cells resulted in a 100 percent improvement in visual performance over untreated controls, with no adverse effects.
Since Stargardt’s has been untreatable, any possible treatment could be given Orphan Drug status by the FDA, which gives financial and regulatory incentives to companies to attempt to treat such diseases. That could speed up the approval process for the trials to begin.
“We’re hoping to get the green light early next year, perhaps in March or so,” Lanza said.
If the trials do begin by then, it might be the first trials of a stem cell-based therapy in the world, coming ahead of the trials planned by Geron Corp. of California, which in January received FDA approval to conduct what was then assumed to be the world’s first human clinical trial of that type. Since then, the FDA has placed a “clinical hold” on the trials to treat spinal cord injuries after some laboratory animals developed cysts. Geron is now anticipating it could start human trials by the third quarter of 2010.
The ACT study will happen at three clinical sites, including the Casey Eye Institute in Portland, Ore., under Dr. Peter Francis; the UMDNJ – New Jersey Medical School in Newark, N.J., under Dr. Marco Zarbin; and Worcester-based University of Massachusetts Memorial Medical Center, under Dr. Shalesh Kaushal.
While waiting on that approval, ACT will be moving forward with other IND applications using the same stem cell therapy, Lanza said. “We will be filing another IND in the next few months, specifically for AMD.”
Lanza said that the company has enough financial resources to take both trials through the next couple of years. Just last summer, ACT was laying off staff and came perilously close to the edge — a victim of the expectations of stem cell therapies versus the realities of developing real treatments, Lanza said.
“Obviously we had a very rough financial patch, and I think that was very typical of some stem cell companies,” he said. “The point here is it takes time and a lot of money and lot of investment to get to where we are here. We are in excellent shape now, our coffers are full.”
Helping pull ACT away from that edge was a deal last December with Korean biotech CHA Biotech Co. Ltd. that resulted in a joint operation based also in Worcester called Stem Cell & Regenerative Medicine International. The joint venture is using ACT’s hemangioblast cell technology to develop human blood cells, for potential use in critical shortage situations.
In addition, earlier this month ACT filed documents with the U.S. Securities and Exchange Commission showing that the company had a commitment for $10 million in a Series B stock placement deal with Optimus Capital Partners LLC. ACT also earlier this month raised approximately $1.7 million in a notes sale.
Last month, Lanza signed a new two-year employment agreement with ACT, according to federal documents, which adds an award of common stock equal to the greater of 20 million shares or 3 percent of any newly authorized employee stock pool to Lnaza’s base salary of $375,000 per year.
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