Fibrodysplasia ossificans progressiva (FOP) is a devastating disease that has no treatment and very few options on the horizon, but that could change soon. As early as September, a clinical trial testing rapamycin (also known as sirolimus) in FOPpatients could begin.

FOP is a very rare genetic condition, striking about 1 in every 2 million people. The disease involves the growth of a second skeletal in the body as the child ages. As the skeletal growth continues, most patients will die as a result of the chest being unable to move and the person stops breathing.

Sirolimus is an immunosuppressive drug currently approved for treating patients with another rare disease, lymphangioleiomyomatosis(LAM) that largely affects the lungs.

Rapamycin was chosen from studies involving induced pluripotent stem cells(iPS) being grown to mimic FOP cells, and using those cells to test a number of drugs to see if they are effective. The researchers at the Kyoto Universitys Center for iPS Cell Research and Application (CiRA) tested 6,800 substances and found 1 drug that drug inhibited abnormal bone formation rapamycin.

The clinical trial may also trigger a greater appreciation for the value that iPS cells can provide in the drug discovery process.

Shinya Yamanaka, director of the CiRA and co-recipient of the 2012 Nobel Prize in Physiology or Medicine for the creation of iPS cells said, I hope the clinical trial will become the start of wider drug development using iPS cells, and lead to cures for many rare diseases.

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Excerpt from:
Can Sirolimus Help Patients with Fibrodysplasia Ossificans Progressiva? - Rare Disease Report