Potential Friedreich's Ataxia Drug Based on Gottesfeld Lab Work Poised to Start Clinical Trials
A compound developed by Repligen Corporation based on work from The Scripps Research Institute laboratory of Professor Joel Gottesfeld is poised to move into human clinical trials as a potential therapy for the devastating disease Friedreich's ataxia. Repligen has announced that it had filed an Investigational New Drug Application with the U.S. Food and Drug Administration for a Phase 1 study to evaluate RG2833, a selective histone deacetylase 3 (HDAC-3) inhibitor.
"I'm as pleased as can be," said Gottesfeld. "It's wonderful news that our lab's work could be launching human trials for 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 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 wheelchair and many die as young adults.
For more information on the Gottesfeld lab's work, see News&Views articles "Mechanism 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 Repligen's website.
Martin Lotz Selected for OARSI Award
Professor Martin Lotz has been selected to receive the Osteoarthritis Research Society International (OARSI) Basic Science Award for his "outstanding work on chondrocyte differentiation, apoptosis, and osteoarthritis." His studies on human joint aging, in vitro and in animal models, led to the identification of cell death as an important event in the pathogenesis of aging-related and posttraumatic osteoarthritis. He also found that inhibitors of cell death were effective in reducing cartilage damage in vitro and in vivo; some of these are currently being developed as treatments to address inflammation after joint injury and to prevent the development of osteoarthritis.
The award will be presented during the opening ceremony at the OARSI World Congress in Brussels, Belgium, on September 23.
Tiansheng Mei and Dong-Hui Wang Win China Scholarship Council Awards
Two Kellogg School of Science and Technology graduate students—Tiansheng Mei and Dong-Hui Wang, both of Associate Professor Jin-Quan Yu's lab—have received prestigious scholarships in organic chemistry from the China Scholarship Council. The Chinese Government Award for Outstanding Self-Financed Students Abroad is given to only 10 organic chemistry students selected from those in 29 countries. Both Mei and Wang are interested in developing catalytic carbon-hydrogen activation/carbon-carbon bond forming reactions and employing these methods in the total synthesis of natural products and drug candidates.
Keary Engle Receives ACS Green Chemistry Institute Award
Keary Engle, a graduate student in the Kellogg School and member of Associate Professor Jin-Quan Yu's lab, has received the Joseph Breen Memorial Fellowship from the American Chemical Society (ACS) Green Chemistry Institute. In the Yu lab, Keary investigates new methods to functionalize unactivated carbon–hydrogen bonds using transition metal catalysts, with a special focus on developing reactions that are operationally simple and environmentally benign. The fellowship sponsors a young international green chemistry scholar to participate in an international green chemistry technical meeting, conference, or training program.
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