Friday, November 22, 2013

Forbes takes a look at stem cell therapies for ALS

Forbes has a good story today about a stem cell trial for ALS (Lou Gehrig's disease) being carried out by Neuralstem. We don't fund that company, but we do fund a disease team award to Clive Svendson at Cedars-Sinai that is a sort of son-of-Neuralstem.

The Neuralstem trial is starting with neural progenitor cells--cells that are already well on their way to becoming neurons--and injecting them into the spine of people with ALS. There, the idea is that the cells will repair the damage that is causing the disease. The company is now in the second phase of testing their approach in clinical trials.

The Forbes article makes a point of how complicated the surgery is:
This is a complicated operation (recent video), one requiring special training for the surgeon, as it involves exposing the patient’s spine and using a custom delivery system that has to counteract the subtle movements of the patient, even under anesthesia, to avoid damaging spinal nerves. According to CEO I. Richard Garr, these are the world’s first intraspinal injections, directly into the gray matter, not the spinal cavity or spinal fluid.
Svendson's work starts the same place, but he and his team are modifying the cells so that they produce a protein called GDNF, which can protect neurons. His hope is that the cells combined with the GDNF will be even more effective than the cells alone.

This tends to be the way therapies move forward. One group tries something and has some success. Another group has an idea that can improve on the original success. And so on until you get to the most effective therapies.

Svendson talked about the Neuralstem trial and his own work recently in a Google hangout on ALS. You can watch his description of the work at about 13 minutes into the hangout video:

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1 comment:

  1. Wow its great, stem cell therapies for ALS. your arcticle is very useful. thanks for share it