Ornithine Transcarbamylase Deficiency (OTC)

A Quiet Salute.

Devastating genetic diseases are sometimes hardwired into children. One such condition called OTC makes the protein that should fuel growth into a dangerous, toxic substance for these children. OTC robbed the lives of 4 sons of dear friends of mine. Gene therapy is the best hope on the horizon for OTC and many other genetic conditions. Here a child is given a virus that inserts the missing gene. Last month the 18th patient in the OTC gene therapy trial, Arizona teenager Jessee Gelsinger, died within days of getting the virus carrying his missing gene. It was the first known death linked to gene therapy. I’m saddened by the loss. I also salute the pioneers that participate in clinical trials. Their action advances the cause of health for all of us. Thank You.

One good place to look to learn about ongoing clinical trials is ClinicalTrials.gov. This is NIH’s one-stop shopping area for information about clinical trials. Here you can access several databases containing facts about both public and privately supported clinical studies. Some of these studies are open to new participants while others have already completed enrollment.