Sandrine Da Cruz, PhD
Ludwig Institute for Cancer Research/ University of California, San Diego
Screening for therapeutic agents that promote muscle innervation or inhibit denervation in ALS
One of the earliest events in ALS is the disruption of the critical connection between motor neurons and muscles, leading to muscle paralysis and ultimately death. Here we propose to identify small molecules that prevent the disconnection or stimulate (re)connection between motor neurons and muscles in ALS. In collaboration with experts in small molecule screening and drug development, we have established an automated co-culture system amenable for testing of potential drugs, including most currently approved drugs and other bio-active molecules known to be safe for use in humans in which motor neurons establish functional connections with muscle cells. This approach, if successful, may yield repurposed drugs for the treatment of ALS and/or lead to the identification of novel drugs that can safeguard nerve attachment to muscle or promote its reattachment.
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Gladstone Institutes, UCSF
Two recently discovered genes that have been associated with both familial and sporadic forms of ALS encode the related proteins TDP43 and FUS cause neuron death in ALS.
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