Year 2

The goal of this project is to produce a clinical grade of human neural progenitor cells that are modified to release the powerful growth factor GDNF that protects dying motor neurons in the spinal cord. In year 2 of this project, we have significantly advanced all aspects of the study and overcome a major hurdle related to the production of the clinical grade human neural progenitor cells (our product that is called CNS10-NPC-GDNF). The challenge was to scale up our laboratory methods (where we produce only a few vials of the cells for lab use) to a clinical grade set of over 1000 vials. Thanks to a major collaborative effort with the City of Hope, many weeks of trouble shooting, and the tenacity of our own scientists, and the CIRM funding, we are happy to report that we now have a clinical grade lot of cells (1,200 vials) for use in all of our animal testing studies and the clinical trial itself. In addition we have now completed all of our dose ranging studies and demonstrated that transplantation CNS10-NPC-GDNF in the lumbar spinal cord of an ALS rat model has a neuroprotective effect on motor neurons at all doses investigated. During this year we have completed more pilot studies in the pig using a novel delivery device (developed by Cedars-Sinai) that will now be used to deliver the cells to the spinal cord of the patients in the trial and is currently moving though the regulatory pathway. Our ALS clinic has expanded rapidly over the past year and implemented more extensive patient testing using the new CIRM funded ATLAS chair to assess overall body strength. Given the size of our clinic we are now confident of recruiting enough patients within southern California to alter the trial from multi sites to a single site within California – Cedars-Sinai. This will allow a more focused approach and development of this novel treatment locally – with subsequent expansion to more sites. We have recruited more members of the clinical team to allow for this. Finally we have continued to present our results at meetings around the world and publish our data in the spirit of communicating this important work to both the scientific community and public.