Last month saw the publication of a German study using lab mice with SMA. The drug is a potential treatment, not a cure, but the researchers seem to think it could be better than CarniVAL, which the babies are currently taking on an investigational basis. The drug is called Saha, and it's actually already on the market for the treatment of cancer, which makes it much easier for us to get our hands on if it's eventually found to be effective in increasing strength in our SMA kids.
Also biotech companies Repligen, deCODE Chemistry, and Invitrogen (and others?) continue to study quinazoline. This drug looks very promising and has "prolonged survival significantly" in SMA lab mice. Quinazoline is a new drug, and so is not currently on the market for any other indications.
California Stem Cell and UC Irvine are planning their human trials for a motor neuron replacement therapy, derived from stem cells, on SMA type 1 patients to occur sometime this year. The Families of SMA have invested in this project over the years, and I can't wait for more information to become available on this trial. For SMA families, if you have heard of Dr. Hans Keirstead, this is the trial in which he is involved.
Geron, a San Francisco based firm, is planning to begin human trials on victims of spinal cord injury in 3Q 2010. According to their product pipeline, Geron is focusing on researching the use of human embryonic stem cells (hESC) in not only spinal cord injuries but also heart disease, cancer, and even diabetes.
This month, Maryland-based Neuralstem
What do you think about any of this? Or if you know of something important that I've missed, share that, too.