We talk a lot about stem cells, so I thought it would be interesting to hear about one of their clinical applications that is currently being pursued.
(I will emphasize that this is not news of a successful therapy: determining whether or not this method works, and how well it works, is specifically the goal of such a Phase II trial. It is, however, an example of the types of new treatments being designed and investigated.)
As part of a new national Phase II clinical trial for amyotrophic lateral sclerosis, the first two volunteers have now finished receiving stem cell injections to their spinal cords. The operation consists of unroofing the patient’s spinal column (exposing the spinal cord), affixing to the spinal bones a custom-designed delivery device (which is able to move with the patient’s breathing), and then using that device to introduce the stem cells into the spinal cord.
This Phase II trial – a follow-up to a Phase I trial that demonstrated that spinal cord injections of up to 100,000 cells could be delivered safely and tolerated well – is designed to treat up to 15 ambulatory patients in five different dosing cohorts under an accelerated dosing and treatment schedule. The first 12 patients will receive injections only in the cervical region of the spinal cord (where breathing is controlled), progressing from 10 injections of 200,000 cells to a maximum of 20 injections of up to 400,000 cells. The last three patients will receive 20 injections of 400,000 cells in both the cervical and lumbar spinal regions. Based on the current schedule, researchers expect that all of the patients could be treated by the end of June 2014.
Note: amyotrophic lateral sclerosis (a.k.a. Lou Gehrig’s disease) is a “rapidly progressive, invariably fatal neurological disease that attacks the nerve cells responsible for controlling voluntary muscles” (
http://1.usa.gov/GGAtX2). Due to the loss of muscle control, individuals eventually lose the ability to walk, speak, and breathe.
Press release:
http://bit.ly/1amp7AdImage: Spinal cord with the outer bits of vertebrae removed so you can see the human intra net. Justin/Flickr
#lougehrigs #spinalcord #stemcells
