What are we uniting for? Simple! To make sure a very promising Spinal Muscular Atrophy (SMA) stem cell program makes it to and through the FDA approved human clinical trial process as quickly as possible. This research has the potential to cure SMA, ALS/Lou Gehrig’s, and many other conditions, but there is a real possibility that we won’t ever know what its true impact could be if we don’t UNITE as a community to close the $500,000 funding gap that currently exists and that could prevent it from ever getting to the human clinical trial stage.
I’m sure many of you have heard of the heroic efforts of the Gaynor family in New York. A few months ago, after learning of their daughter’s SMA diagnosis, they launched SophiasCure.com and have personally committed to raising $200,000, a monumental amount of money, to fund this specific SMA stem cell program. The Gaynor’s describe themselves as an average middle-class family — Vincent Gaynor is a construction worker — yet what they have accomplished is nowhere near average; it’s simply extraordinary. To date, they have raised over $100,000 from thousands of individuals who donated an average of $25 each — just $25 adding up to a total of over $100,000. We have been so inspired by their personal efforts and, after meeting with Dr. Hans Keirstead, the lead project researcher, are certain of the real promise this research has for curing SMA and many other diseases and conditions. We believe this research could be the END of SMA for good — IF given the funding! And so, there is no way that we can sit back and not help take funding out of the equation. That is why we’re reaching out to you — the SMA community — to ask that we all UNITE together, commit to be part of making this happen, and collectively stand alongside the Gaynor’s to raise the funds necessary to see this program make it to human clinical trials.
Specifically, we’re asking 20 families to commit to raising $5,000 each by the end of November 2009 – for a total of $100,000 – with 100% of the proceeds going directly to fund this SMA stem cell program.
How, you ask? Any way you want! It can be through one big event or through a dozen small ones. You just need to be creative and almost anything can be turned into a fundraiser: weight loss challenges, lemonade stands, yard sales, community walks, going door-to-door, bake sales, casino nights, silent auctions, bowling tournaments, golf tournaments, pancake breakfasts — the list goes on and on. We all need to ask our friends and families to get involved, and we mean, truly involved. We need to inspire our neighbors, ask our local businesses to participate, and we need to be simply relentless. Bottom line, each and every one of us has the tools at our fingertips to come together and make this happen.
Let’s do it together — as a community impacted by and bonded together forever by this horrible disease. If not for our own children, for future SMA generations. If not for SMA, for the countless other diseases and conditions that will be positively impacted by this research. Let’s do it together!
A few quick facts about the SMA stem cell program at UC Irvine:
- The program is headed by Dr. Hans Keirstead, Co-Director at the Sue and Bill Gross Stem Cell Center at UC Irvine
- The SMA program has been in process for five years and is a collaboration between Families of SMA, UC Irvine, California Stem Cell, and Johns Hopkins University
- Dr. Keirstead has already been credited with successfully developing a stem cell derived treatment for acute spinal cord injuries; this program is currently in human clinical trials - the 1st ever stem cell trial approved in the United States
- For SMA, Dr. Keirstead has perfected a process that replaces lost motor neurons that characterize SMA with high purity human motor neurons derived from human embryonic stem cells
- All of the pre-clinical efficacy studies, including the pivotal animal safety studies, have been completed, demonstrating that the cells work
- They are now targeting a Phase I clinical trial in humans to begin in early 2010
- But, the program needs an additional ~$500,000 as a bridge to that phase for staffing and studies to support the requirements leading into that critical Phase I clinical trial in early 2010 and looking beyond to later phases
For more information about the SMA stem cell program or Dr. Hans Keirstead, please click here.
If you are interested in getting involved to see that this program is given a chance, please contact:
We can’t do this alone — but we can do it together! UNITED we can take funding out of the equation and give Dr. Keirstead, his team, and this groundbreaking science a chance to cure SMA once and for all.
