The California Institute for Regenerative Medicine (CIRM) granted a local interdisciplinary research team from UCSD, Salk, and Life Technologies $11.5 million for research aimed at finding a cure for amyotrophic lateral sclerosis (ALS), or Lou Gehrig’s disease. On June 23rd, CIRM hosted a public research update which I attended. The project is an excellent example of local research organizations and companies working together towards translating research into real cures, funded by California citizens who realize the promise of stem cell technologies.
The ALS team is led by Lawrence Goldstein, Ph.D., and Don Cleveland, Ph.D., from UCSD, and Samuel Pfaff from The Salk Institute. Drs. Goldstein and Cleveland spoke at the event, along with Life Technologies’ Mark Bonyhadi, Ph.D., UCSD’s Martin Marsala, MD, and Lucie Bruijn, Ph.D, from the ALS Association. Goldstein and Cleveland gave a great introduction to the project and explained that ALS is caused by the death of motor neurons, and that the cells’ environment is very important to their survival. Astrocyte cells have a very intimate relationship with motor neurons, and disease progression is predicted to be slowed if healthy precursors are introduced. As Goldstein and Cleveland explained, the ‘neighborhood’ of motor neurons is very important in determining their well being, and healthy astrocytes are the equivalent of neighbors who don’t have parties and mow your lawn for you. The team plans to create and transplant human astrocyte precursors (hAPs) from human embryonic stem cells (hESCs) into human spines. Begining with rodent models, they predict that the first clinical trials could take place in 2014.
Dr. Mark Bonyhadi from Life Technologies then explained how they will help create and analyze the hAPs and develop the processes to prepare them for FDA submission. Life Tech’s long history with high quality cell media and wide-ranging protein and cell analysis tools makes them a perfect partner. Dr. Martin Marsala described the preclinical animal studies that will be performed, and indicated that they have worked with a company called Neuralstem previously to show that spinal cell transplantations can be successful.
Dr. Lucie Bruijn from the ALS Association talked about the fact that there is only one drug available for the disease, and that other stem cell efforts are underway which will help develop the transplantation methods in parallel (Side note: normally drug discovery research is highly secretive, but do publicly funded efforts benefit from removing this veil?). ALS clinical trials are tracked on their website.
The research presented was inspiring, but the clear highlight of the event for me was to hear from ALS patient Dan Desmond. As a veteran, he is unfortunately part of a group that has a higher prevalence to contract the disease, the cause of which is only 10% genetic in nature. From his wheelchair, Dan told us about the progression of the disease, his carpe diem attitude, and he thanked the researchers for their work. Dan received a well-deserved standing ovation at the end of his courageous speech.
Hearing directly from patients is perhaps an unexpected benefit of moving basic research towards a more translational model, where curing disease is viewed as a near-term goal. As described by Dr. Goldstein, CIRM has challenged researchers to rethink assumptions and timelines to come up with solutions to fast-track therapeutics. CIRM is clearly ‘stepping up to the plate’ (pun intended) to make Prop 71’s goals of making California a leader in stem cell therapies come to light. This exciting project pairing our local institutions is a fantastic example of the synergies. Have you thought about who you could work with locally? (Hint: come to our events for ideas…)
Photo by GEHealthcare on Flickr under a Creative Comments License