Stargardt Macular Dystrophy

 

Juvenile macular degeneration was first reported in 1901 by German ophthalmologist, Karl Stargardt, from whom the disease gets its name. There are several forms of early onset macular degeneration, some of which are inherited and correctly called macular dystrophies.

Stargardt’s macular dystrophy is an inherited juvenile macular degeneration that causes progressive vision loss usually to the point of legal blindness. The progression usually starts between the ages of six and twelve years old and plateaus shortly after rapid reduction in visual acuity.

At present there is no cure for Stargardt disease and there is very little that can be done to slow its progression.

 

Human and Social Costs

Causes blindness, generally among youths 10 to 20 years in age, and affects less than 200,000 people in the US.

 

Recent Advancements

  • In July 2011, two patients became the first people to be treated in FDA-approved clinical trials for a therapy derived from embryonic stem cells for dry age-related macular degeneration and Stargardt’s macular dystrophy. Advanced Cell Technology of Santa Monica, Calif., developed the treatment, which in rats and mice has prevented further vision loss without adverse side effects. (Los Angeles Times, July 14, 2011)
  • A Massachusetts based bio-firm announced US FDA approval for human trials of their retinal pigment epithelial cells to treat Stargardt’s Macular Dystrophy.  The recently approved trials will only involve 12 patients, and are looking primarily to establish that using the ACT cells is safe. There is hope, however, that the vision of those treated could be improved or restored. (Advanced Cell Technology (ACT), Nov. 22, 2010)
  • The Foundation for Fighting Blindness and Oxford Biomedical’s gene therapy treatment, StarGen, has been given orphan drug status by the European Medicines Agency (EMEA, similar to the FDA). Clincial tials are planned for late 2010.