Over the past two years, I have written extensively about the human clinical trials underway for treating eye diseases using both stem cells and gene therapy. There are currently eighteen clinical trials using stem cells and sixteen trials using gene therapy to treat about a dozen different eye diseases. At latest count, at least as I have been able to determine, nearly 80 patients have had gene therapy treatments and more than two-dozen have had stem cell treatments. The initial results of many of these trials will probably be discussed at the upcoming American Academy of Ophthalmology Meeting to be held in Chicago in mid-November of this year.
Gene Therapy Trials
The most advanced of these studies are those underway at Children’s Hospital of Philadelphia, where doctors have administered gene therapy treatments to more than 16 patients suffering from Leber’s Congenital Amaurosis (LCA), some having received the therapy in their second eye after successful treatment of their first eye.
Leber’s is a rare form of inherited blindness caused by retinal degenerative disease that strikes in infancy and causes sever loss of vision. It is an autosomal recessive disorder that affects both the rods and cones. In the first completed trial, the light sensitivity of all 12 partially blind patients improved. Four of the children gained enough vision to play sports and stop using learning aids at school.
Other clinical trials are aimed at the wet form of age-related macular degeneration (which, if not halted leads to blindness), choroideremia (a rare inherited disorder that causes progressive loss of vision due to degeneration of the choroid and retina), Stargardt’s disease (an inherited juvenile macular degeneration that causes progressive vision loss), and Usher’s Syndrome (a relatively rare genetic disorder that is a leading cause of deaf blindness). In addition to Children's Hospital, Oxford BioMedica has several clinical studies underway
Stem Cell Trials
Advanced Cell Technology is at the forefront of the use of stem cells to treat ocular diseases. The company is sponsoring three clinical trials to treat Stargardt’s disease (one trial each in the United States and one in the United Kingdom) and one clinical trial to treat the dry form of age-related macular degeneration in the U.S. To date, four patients have received retinal pigment epithelial (RPE) cells derived from human embryonic stem cells for the dry AMD, and seven patients have been treated with RPE cells in both the Stargardt’s trials. Early results indicate improved vision in all of the treated patients and, detailed results are expected to be presented in November at the AAO Meeting in Chicago.
Other clinical trials underway include those aimed at treating corneal surface defects, corneal epithelial cell deficiencies, and treatment of optic nerve atrophy. In addition to the eleven patients being treated in the ACT trials noted above, an additional fourteen patients, at least, have received stem cell treatments.
To read more about what I have written on both stem cells (more than 20 articles) and gene therapy (16 articles), see the links referenced below.
To obtain copies of my current tables listing all of the clinical trials underway, including the number of patients treated to date in each trial, please send me an email request.
References:
A List of Writeups on Stem Cells Used in Ophthalmology
A List of Writeups on Gene Therapy Used in Ophthalmology
