SMA Today

I've let blogging go for too long now. I have been wanting to sit down and write about many things but I need to prioritize. Let's focus on what is happening in the world of SMA today. There is an article in Esquire magazine talking about Dr. Keirstead's amazing work. Here is an exerpt:

"In 2002, at his lab at UC-Irvine, Hans Keirstead delicately sliced open the spines of eight lab rats with a scalpel, then not so delicately punched into their spinal-cord tissue with the force of two hundred kilodynes. A week later, he reached for a vial inside which was something most of the scientific world believed was impossible: a stem-cell solution so pure that the risk of any newly derived nerve cells morphing into tumors had been all but eliminated. He drew some liquid from the vial and injected his elixir — set to grow into oligodendrocytes, which help ferry movement-generating electric impulses into muscles — into the spines of the recently paralyzed rats. Then an assistant grabbed a camera. The resulting video was short, but its meaning was unmistakable: The rats stood up and wobbly walked. The clip went viral and the public cheered. But many of Keirstead’s colleagues were less sanguine. Was the science right? they wondered. He hadn’t even published a paper on it yet. As early as this spring, we’ll begin to find out.

Pending one final review, next year a handful of paralyzed men and women are set to get Keirstead’s high-purity stem cells injected directly into their spinal cords, above and below the injury site. Conducted by the Geron Corporation — to whom Keirstead turned over his research — it will be the first-ever test of purified stem cells in humans. Again, the public is mesmerized by what could happen, and again, Keirstead’s colleagues are nervous. If the phase-one trial exacerbates a subject’s condition or, far worse, kills one, Keirstead’s test won’t just fail, it could retard progress on stem cells for decades. He is in effect taking the first major step on stem cells for everyone. And he’s doing it with a novel therapy that has a shorter paper trail than most.

Aware of the stakes, the FDA temporarily put the brakes on the trial in August, weeks before it was originally set to begin, so it could rereview the data. But Keirstead is unfazed. He has confidence in his work. The trial application Geron submitted was the longest ever — twenty-two thousand pages — all pointing toward the success and efficacy of Keirstead’s method. He feels certain the trial will go forward. “My guess is that the FDA got new supporting data that is very interesting and they just need time to vet it.”

But Keirstead says he doesn’t have time to wait. And neither do the infants born with spinal muscular atrophy, a genetic mutation that often kills within twelve months of birth. He has already moved on to securing FDA approval for what would be the world’s second clinical trial of human embryonic stem cells to test a different stem-cell-derived nerve cell — the motor neuron — on infants with SMA. This go-round, he wants to increase velocity. No dribbling out a paper here and there and waiting for his colleagues’ comments. “This motor-neuron story, not a single publication out on it yet, but I’m going to the FDA!” trills Keirstead, forty-two. “I did everything at once this time: I did a manufacturing facility. I formulated the clinical plan, gathering all the medical doctors. I did the preclinical efficacy, preclinical safety, lined it all up side by side, moved it all forward.” Sure, he has a handful of papers on his motor-neuron story in review, but those are almost an afterthought. Cures don’t come from pushing paperwork."

To all of you that have helped with Georgia's Journey of Hope we are grateful. Thank you to all of you that joined in The Unite for the Cure campaign. Our family has raised approximately $6000.00 for the research project and as of last night the Uniters had raised over $70 000.00 Congratulations to all of the people that are making this happen. There are still other groups of people around here doing different things and raising money. Whether you donate to FSMAC or Unite for the Cure you are donating to HOPE and that should feel good. So many babies and children are waiting for that cure.

There is also a final push to get the SMA petition signed. If you can think of anybody that hasn't signed it please consider forwarding the information and asking them to sign. We really are close!

Next post will be about what's been happening around here.

Love to you all,

Kristen