In the current issue of the journal Lancet, Dr. Steven Schwartz shared the results of the first two patients in the Advanced Cell Technology (ACT) clinical trial, using stem cell generated retinal cells. Both patients tolerated the surgery well without complications and at six months, their vision had improved somewhat. The ACT clinical trial is the first embryonic stem cell trial approved in the U.S.  It is currently taking place at UCLA's Jules Stein Eye Institute and at Wills Eye Hospital in Philadelphia.  Other sites will be added as they qualify.  Each site will include 12 patients with either age-related macular degeneration or Stargardt's (a juvenile form of macular degeneration) who are legally blind. Because this is a safety trial, the participants have end stage eye disease.  However, if the therapy proves successful, it will actually be aimed at patients in the earlier stages of macular degeneration.

The patients - a woman in her 50s with Stargardt's macular dystrophy and a woman in her 70s with dry age-related macular degeneration - underwent outpatient transplantation surgeries last July, said principal investigator Dr. Steven Schwartz, chief of the retinal division at the Jules Stein Institute and the Ahmanson Professor of Ophthalmology at the David Geffen School of Medicine at UCLA. Both patients received the lowest dose of stem cell-derived retinal pigment epithelial (RPE) cells (50,000), which were transplanted into the space under the retina. There will be four groups of patients in this trial, each one receiving a higher dose (100,000, 150,000 and 200,000). The researchers monitored the patients' progress over four months and found no safety concerns, no signs of rejection and no abnormal cell growth.

According to the article, standard vision tests suggested some improvement in the vision of both patients. The woman with Stargardt's disease, for example, went from only being able to discern hand movements to seeing a single finger move, according to the Lancet article. On a visual acuity letter-chart, she went from being unable to read any letters prior to treatment to reading five letters. The patient with macular degeneration also showed some improvement after the therapy. Where once she was only able to make out 21 letters on the chart, her reading level stabilized at 28 letters - after peaking at 33 letters just a couple of weeks after the transplantation.

While Dr. Schwartz stated that, "I am more hopeful now than I was a year ago", he cautions that the vision gain seen here is slight. There is no way to know what the patients' vision will be in another six months.  Still, the results to date are encouraging. Dr. Schwartz is set to perform the third surgery soon.

February 2012