Year 4

During the final year of this proposal we filed with the FDA for an investigational new drug (IND) to transplant stem cells releasing the powerful growth factor GDNF into the spinal cord of 18 patients with ALS using a new stereological delivery surgical device. Following a 6 month interaction with the FDA involving multiple letters and more data generation (especially with regards to the device) we were finally approved to move forward with the patient trial by FDA in late October 2016. Upon approval we started to make final preparations for the trial and applied for a CLIN2 award from CIRM to fund the clinical portion of the program. This grant was awarded in early 2017 and will start on June 1st. Our current and original disease team CIRM grant closed on May 1st 2017 following completion of all preparations for the first patient including final training of the neurosurgeons in animal models and final approvals from local IRB committees. We thank CIRM for all their support during the course of this disease team and look forward to reporting back on the trial during the course of our new CLIN2 award.