Neither patient experienced safety issues during the trial, conducted by biotechnology firm Advanced Cell Technology (ACT) and the University of California, while both patients had measurable improvents in their vision that persisted for more than four months.
Visual acuity of the Stargardt’s patient had improved from hand motions only to 20/800 vision, it was reported in The Lancet. Before treatment, the patient was unable to read any letter on a visual acuity chart, but by two weeks started reading letters, improving to five letters at one to three months in the treated eye.
Dr Robert Lanza, MD and chief scientific officer of ACT, and co-senior author of the paper, said: “This is the first report of hESC-derived cells transplanted into patients, and the safety and engraftment data to date look very encouraging.”
“Although several new drugs are available for the treatment of the wet type of AMD, no proven treatments currently exist for either dry AMD or Stargardt’s disease. Despite the progressive nature of these conditions, the vision of both patients appears to have improved after transplantation of the cells, even at the lowest dosage.
“This is particularly important, since the ultimate goal of this therapy will be to treat patients earlier in the course of the disease where more significant results might potentially be expected.”
ACT has enrolled 12 patients for open-label studies in the US on each of the conditions over the course of 12 months.
Meanwhile, a first SMD patient recently received st-cell treatment at Moorfields Eye Hospital in London as part of recently-approved parallel trials in the UK.
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