In June, Dr. Klassen was invited to attend a special meeting held at the National Institutes of Health (NIH), in Bethesda, MD. The meeting was specifically organized to facilitate the progress into clinical trials of stem cell therapeutics for retinal degenerations. The meeting was attended by key members of the NIH and FDA. A number of stem cell translational teams were represented, including those working on RPE cell transplantation in AMD as well as our efforts with retinal progenitors for RP.
Programs currently in trials include those of ACT and Stem Cells, Inc. Both involve types of stem cells, although neither is working with RP patients. Another trail, at UC Davis, is using bone marrow progenitors in RP and is currently enrolling as well. In the near term pipeline are at least 3 more teams aiming to begin trials in AMD using stem cell-derived RPE cells, plus our effort using retinal progenitors in RP. All are targeting 2014 or thereabouts for initial enrollment. It is important for RP patients to realize that the majority of current projects are using stem cell-derived RPE cells and directed towards forms of macular degeneration, be it AMD or Stargardt’s Disease.
No doubt the era of stem cells for the retina is finally underway. The next several years will be of great interest as the results of these various clinical studies become known. There are a lot of high hopes going in, from investigators and patients alike, but it is important to remember that this still represents the initial phase of clinical testing.