SMA Research Update and Stem Cell Therapy

The amount of information I'm seeing out there regarding SMA research has just been exploding over the past year. If you have an interest in SMA research--or stem cell research in general, even--read on. I've tried to highlight the most interesting things that I've noticed lately, but surely for every one I see there are a dozen more exciting studies and papers that I am missing.

January 2010 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 twins 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.

Both of these drugs are in the pre-clinical stages of research, which my personal clinical research expert in the next room here tells me means that the research firms involved still have to apply to the FDA to begin their human trials. If their applications are approved, human trials could start in anywhere from two months from now to five years from now. Still, it's nice to know that someone is doing something, right?

My other interest is around U.S. stem cell trials in humans. As everyone knows by now, it took Barack Obama exactly 43 days in office before he reversed the embryonic stem cell research funding ban last year. Like it or hate it, the ban is in fact lifted, and federal dollars can now be used to support some heavy duty stem cell research.

I like it.

SMA is one of the conditions being seriously considered for stem cell treatment here in the U.S. It's been done in other countries, but documentation of these cases is nearly non-existent and what is out there is entirely anecdotal. It's hard for me to wrap my mind around the thought process behind injecting a child with stem cells, but not documenting or publishing the procedure and not having any before/after metrics to even show if the stem cells worked at all whatsoever. I certainly don't judge the families who have chosen to go this route--if you search my archives here, you'll see that if I'd had $50,000 to spare last December, I would have already been to China and back. Twice. Literally. The parent of a child (or children, heh) with SMA is a desperate parent, to be sure.

Here is a basic primer, sort of a Stem Cell Research 101.

So, even though embryonic stem cell therapy on humans has been performed elsewhere, it's not been done in the U.S. or done under the strict regulations of the good old Food & Drug Administration. I bet someone out there has some harsh words regarding the inefficiencies of the FDA, and while I'd love to hear your arguments, that's not really my agenda. Instead, I want to highlight some of the really amazing progress being made here in the U.S. to advance stem cell trials to human subjects. If we're smart, we'll all buy stock in these companies.

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 announced a human clinical trial at Emory University for ALS patients. The trial involves injecting human embryonic stem cells into the spinal cord, and the first patient has already received treatment. The researchers emphasize that they expect this only to slow the progression of the disease, but that it is not a cure. One notable fact is that it has taken Neuralstem only four months to go from FDA approval to their first injection of stem cells.

What do you think about any of this? Or if you know of something important that I've missed, share that, too.