In only four months and with fundraisers organized by over twenty families and their friends across the globe (literally), “Unite For The Cure” (Unite) met and surpassed their goal of raising $100,000 for Dr. Keirstead’s promising SMA research. In fact, over $145,000 — yes, one-hundred-forty-five-thousand-dollars — was donated to Families of SMA (FSMA) through Unite efforts earmarked specifically to accelerate Dr. Keirstead’s stem cell research to human clinical trial. This is an enormous accomplishment in and of itself in such a short period of time, but when you take into account that every single one of these remarkable families is either caring for a terminally ill child or grieving the loss of their precious baby — it is nothing short of miraculous!
Each of these inspiring Unite families has poured their heart and soul into fundraising, has shown tenacity and dedication, has asked friends and family to be involved, and has campaigned tirelessly to get Dr. Keirstead the money he needs. In November 2009, Dr. Keirstead’s SMA stem cell program was named by Esquire magazine as a “Project That Will Change Science Forever”. We truly hope that this will prove to be the case and that each of us will have had a material impact on accelerating this groundbreaking, game changing science into the history books.
A message from Kenneth Hobby, President, Families of SMA:
Thank you all for the tireless efforts involved here to support this program and our mission to develop a cure. Thanks to you we will be one step closer.
Reaching $100,000 raised is a tremendous milestone to be able to hit. I think we all have that additional bit of extra hope now in the community with the funds you have all raised to help move us forward.
The initial investment from Families of SMA in stem cell research was in 2000 and this funded efficacy studies using motor neurons from mouse stem cells. Over the following years additional funding lead to the first, highly-pure therapeutic population of human motor neurons for cellular replacement therapy for SMA. Click here for full details on the program.
The team involved in this collaboration includes the laboratory of Dr. Keirstead at The University of California, Irvine, the laboratory of Dr. Kerr at Johns Hopkins University, California Stem Cell, Inc., and FSMA.
In October of this year we completed a pre-Investigational New Drug (IND) Meeting with the FDA. Click here for details of this recent meeting. This meeting gave us guidance from the FDA on the clinical and regulatory pathway and requirements for submission of an IND application to be able to initiate human clinical trials.
The stage of the program we are now beginning, and the work and studies that all of your efforts are helping to support, is the preparation of the final IND application. This involves a lot of regulatory work and applications, and several experiments and tests showing that manufacturing of the motor neurons to be used in the clinical trials is reliable and scalable. The final IND application that is given to the FDA is meant to comprise all of the information that has been generated for the program to date. This can easily run into many thousands of pages in length. This application is submitted to the FDA, which then has 30 days to review the information and then give a final say on whether we are clear to start the first clinical trial.
At this final stage just before clinical trials nothing is yet guaranteed. Programs can and often do get put on hold by the FDA at this stage. This particular program looks to not only be the first new IND application put into the FDA for SMA, but also one of the first for a stem cell therapy for any disease! So, the unknowns and risks are still high, but so are our hopes that we will be successful in getting approval and then move forward into clinical trials with patients.
Thank you all so much for all the amazing energy you have put in to support this program and to help all in the community.
Kenneth Hobby, President
Families of SMA
And a message from Dr. Hans Keirstead:
I am excited to let you know that we have finished all of the animal work that is required by the FDA, covering both efficacy and safety. We are currently writing up all of the results in a manner that the FDA requires for submission of an IND. We are also completing work to document the cell preparation and shipping procedures, and conducting experiments to ensure that those procedures do not adversely affect the cells. Lastly, we are preparing the IND.
FSMA, and many families, have generously contributed to the funding of many of our research needs. However, many non-research tasks must be preformed before we can submit our IND. These studies include the cell preparation and shipping procedures mentioned above, and the IND ‘paperwork’ preparation costs.
Thank you so very much Unite families for all of your support. We simply could not have done this without you, and your ongoing support is nothing short of enabling this research to move forward into the clinic.
Hans S. Keirstead, Ph.D.
University of California, Irvine – Reeve-Irvine Research Center
Sue and Bill Gross Stem Cell Research Center
At this point, we can all know and be proud of the fact that, as one united community, we’ve helped accelerate this program. We now simply have to wait for the IND process to play itself out, the science to prove itself in humans, and — hopefully — pave the way for getting this treatment to children as safely and efficiently as possible.
From the bottom of our hearts, thank you Unite families. It has been an incredibly inspiring experience working with you and we hope that each of you found it to be motivating and uplifting. This has been a wonderfully successful campaign and we are so proud of every single effort that has helped make this such a huge success — a $145,000+ collective success!
As we intended Unite to be a one-off campaign, we will be closing Unite down (although this site will live on in perpetuity in its current form). However, we believe this type of earmarked, directed fundraising serves an enormous benefit to both fundraisers and researchers and in the coming months we plan to launch a similar program. After all, it is your money — you should decide where it goes. If you are interested in joining this effort, please contact us or stay tuned for the big launch in the coming months. Together we will see SMA cured!
The McClay Family
Raised over $16,700!
The Span Family
Raised over $10,700!
The Potter/Reilly Families
Raised over $10,000!
The Schmid Family
Raised over $9,500!
The Marshall Family
The Strong Family
Raised over $8,300!
The Gooden Family
Raised over $7,700!
The Pringle Family
Raised over $7,700!
The Butler/Trakas Families
Raised over $6,900!
The Gustafson Family
Raised over $6,700!
The Lunt Family
Raised over $6,600!
The Lucas Family
Raised over $6,000!
The Zahn Family
The Coleman Family
Raised over $5,200!
The Wright-Beasley Family
Raised over $5,000!
The Ragland Family
Raised over $5000!
The Howell Family
The Calafiore Family
Raised over $4,500!
The Felix Family
Raised over $4,000!
The Greene Family
Raised over $1,400!
The Lovelace Family
Raised nearly $1,000!
An additional $2,000 was raised in private donations sent directly to Unite from individuals, ranging from $25.00 to $500.