In what could be a significant step forward for human embryonic stem cell science Massachusetts based Advanced Cell Technology (ACT)  has announced that the FDA have granted orphan drug status to MA09-hRPE ¨C a human embryonic stem cell (hESC) derived treatment for a rare type of blindness known as Stargardt's Macular Dystrophy. Orphan drug status, from the US Orphan Drug Act,  is aimed at accelerating therapies for diseases afflicting fewer than 200,000 people in America. The special status will give ACT access to grant funding for clinical trials and up to seven years of market exclusivity.

ACT's therapy uses hESCs to recreate retinal pigment epithelium cells, which support the photoreceptors needed for vision and has shown promise in rat and mouse models of macular degenerative disease. Abstracts of supporting peer-reviewed journal articles can be found here and here.

In the excitement of this latest announcement, it is easy to forget that it has been just 12 short years since the discovery of human embryonic stem cells. Whilst there is much to learn about the basic biology of these cells, several companies are pushing hard to begin the first clinical trials. Until the ACT announcement, the only other company to obtain FDA approval is Californian based biotech Geron. Geron's hESC derived therapy targeted toward treating acute spinal cord injury was initially cleared by the FDA in January 2009 but at time of writing is in a clinical hold whilst new data is reviewed.

Read more at Nature or ACT's media release.

To find out more about stem cells see our fact sheets and find out more about stem cell therapies in our Patient Information Handbook.