Martina Wiedau-Pazos, M.D., Ph.D. University of California, Los Angeles

Martina Wiedau-Pazos, MD, PhD is an Associate Professor of Neurology who directs the UCLA ALS Clinic and Research Center. Dr. Wiedau-Pazos has been studying disease mechanisms in Amyotrophic Lateral Sclerosis for more than 20 years. She participated in one of the first projects to study disease mechanisms of mutations in superoxide dismutase linked to familial ALS. Her group is interested in using disease models of ALS, such as transgenic animals and stem cell-derived motor neurons to study disease mechanisms and therapeutic approaches to ALS.

Why Dr. Wiedau–Pazos collaborates on the molecular-tweezers project:

I became interested in the molecular tweezer, CLR01, as a potential treatment candidate for ALS, because protein aggregation is an evolving disease mechanism for ALS, and CLR01 has great potential not only to treat disease-related protein aggregation, but also help us understand which aspect of protein malfunction is toxic to neurons.

ALS is one of the most devastating neurological diseases and a cure is urgently needed. There is a great need to work together with related fields in neuroscience. The molecular-tweezers project provides a crucial opportunity to bundle expertise and make a potential therapeutic approach work for many different neurodegenerative disorders. Seeing Dr. Gal Bitan’s results with the collaborators in the Alzheimer’s and Parkinson’s disease fields gave me hope that we could advance the search for therapeutic approaches in ALS using this compound.

We are currently testing the drug in cell culture and in a transgenic SOD-1 mouse model of ALS. This will hopefully pave the way toward clinical studies in ALS, which give new hope and opportunity to ALS patients in our clinic.