Potential Friedreich’s Ataxia Drug Based on Gottesfeld Lab Work Starts Clinical Trials

A compound developed by Repligen Corporation based on work from The Scripps Research Institute laboratory of Professor Joel Gottesfeld has moved into human clinical trials as a potential therapy for the debilitating disease Friedreich’s ataxia. This study is being conducted in Turin, Italy, and is the first clinical trial of a drug that targets the core genetic defect in Friedreich’s ataxia.

Friedreich’s ataxia, which afflicts about one of every 20,000 to 50,000 people in the United States, is caused by an inadequate production of the protein frataxin, which leads to degeneration of nerve tissue and an array of associated complications including heart disease and scoliosis. In most cases, sufferers are ultimately confined to a wheel chair and many die as young adults.

For more information on the Gottesfeld lab’s work, see News &Views articles “Team Implicates Wayward DNA Repair Enzyme in Friedreich’s Ataxia”, “Mechanisms for Friedreich’s Ataxia Uncovered”, and “Scripps Research Team Reverses Friedreich’s Ataxia Defect in Cell Culture”.

For more information on the Phase 1 study, see press releases from Repligen or The Friedreich’s Ataxia Research Alliance.

Send comments to:

Opening Doors to Scripps Research Graduate Studies
Hosting more than 125 students from 29 states and 9 foreign countries, the Kellogg School of Science and Technology recently held its annual recruitment weekends on Scripps California and Florida campuses. Designed to acquaint students with the institute's PhD program, the visitors participated in faculty presentations, campus tours, laboratory visits, discussions with postdocs, and personal interviews with professors. (Photos by Jeremy Pyle, Mika Ono)


SMART Teams Prepare for National Poster Competition
Working with mentors from Scripps California, teams of local high school students in the Students Modeling a Research Topic (SMART) program recently practiced their poster presentation skills. Above, Canyon Crest High School students Nachi Baru (left), a junior, and Rachel Hvasta, (center), a freshman, explain their project, ”Tomato Bushy Stunt Virus,” to Joe Nagano (second from right) and Sandip Chatterjee, graduate students in the Cravatt lab. (Photo by Cindy Brauer.)