Why We Chose A Pre-Clinical Model As Our Next Research Focus

March 04, 2011 by Bill Strong

Taking a step back. Looking at the big picture. Making sure our direction is sensible and methodical. That is why we chose a pre-clinical model as our next research focus.

Over the past two years, the Gwendolyn Strong Foundation (GSF) has taken a laser-guided missile approach to funding research, primarily funding specific portions of two promising cure focused spinal muscular atrophy (SMA) research programs:

  • Stem Cell-Derived Motor Neuron Transplantation Therapy: In 2009 and early 2010, our fundraising programs provided over $235,000 to Dr. Hans Keirstead's stem cell-derived motor neuron transplantation therapy. In December 2010, this program filed an investigational new drug application (IND) with the US Food and Drug Administration (FDA) and is currently on clinical hold pending completion of more studies before hopefully commencing a Phase I safety study in the near term.
  • Gene Therapy: In 2010 and early 2011, our fundraising programs provided $250,000 to Dr. Brian Kaspar and his team working on a promising gene therapy based SMA program at Nationwide Childrens Hospital and The Ohio State University. GSF's support funded the initial safety and toxicity studies for the program, both critical hurdles to the first step in proving safety in the human population and in anticipation of the pre-IND and IND.

We're extremely proud of both of these programs and know that our fundraising efforts, and those of our partners, supporters and donors, have achieved their intended goal: using dollars to answer key questions and target specific portions of research necessary to push SMA closer to a cure. Time will tell how these therapies translate into humans and what their impact will or will not be. But it's clear that GSF's programs had a material, positive impact on moving them, and therefore SMA research in general, forward. And that's wonderful. With our next commitment we decided to step back, look at the big picture, and make sure our direction is sensible and methodical and will provide the best return on our investment and yours.

As you know, our daughter, Gwendolyn, has SMA type 1, the most severe form of the disease. At 3-and-a-half-years-old, she's an outlier living with SMA and, as parents, we often find it difficult to not become myopically focused on one research program hoping it unlocks the key to the SMA puzzle. But, we've learned that we must resist the temptation to hear only what we want to hear and make research funding decisions based on sound bites and in the process neglect to fund programs that will have a broad impact on unraveling this complex disease. That is why we choose to work closely with seasoned organizations with respected and highly educated scientific advisors -- because no matter how many research studies or books we read we are not scientists or SMA research experts ourselves -- nor would we purport to be -- and will always be parents first.

Now, this new commitment doesn't mean we won't return to funding Dr. Kaspar's program directly or other cure focused programs in the future, but we feel that other organizations already have those bases covered and our dollars are better served, at this point, in a different direction. It is a very exciting time for SMA research with interesting studies taking place around the world and we are so grateful to see SMA being attacked from all angles. We'd hate to get to the finish line and not have the tools ready to help us truly cross over it.

Thus, we've chosen to once again enthusiastically fund very exciting research with our next targeted commitment: the development of the first large SMA pre-clinical animal model -- the first-ever-in-the-world! Like the SMA mouse model advancement years ago, the pre-clinical large animal model that we will be funding is expected to become the standard by which future treatments will be studied and tested and is creating great excitement and enthusiasm among researchers and drug companies globally. The funding commitment is $200,000 and the program will take place over the next two years in the Lorson Lab of the University of Missouri, led by Dr. Monique Lorson and her colleagues. In short, we're very excited about this program and its impact on many facets of SMA research, including gene therapy, for years to come.

So, what exactly is this pre-clinical model and what does it mean for SMA research? In simple terms, this pig based SMA model should:

  • More closely mimic the human condition
  • Help researchers to identify the most effective therapies for SMA
  • Prove invaluable to two of the most promising SMA therapeutic strategies: gene therapy (e.g. Dr. Kaspar's program and others) research and the oligonucleotide studies

Here's what Dr. Brian Kaspar had to say on March 3, 2011 about the pre-clinical model:

"The pig model is a long term vision for not only gene therapy but also drugs, oligos, and stem cells for the treatment of SMA. The development of this larger model of the disease will provide a very powerful tool in our toolbox for understanding SMA and developing new and improved therapies. Therefore, I remain enthusiastic and in full support for developing additional models to study SMA and look forward to the continued momentum in research and therapeutic development in SMA. - Dr. Brian K. Kaspar, Ph.D. Associate Professor The Research Institute at Nationwide Childrens Hospital The Ohio State University

We couldn't agree more Dr. Kaspar and we're extremely excited about this next phase of GSF funded research. And we are so glad you all are, too. We already have fundraisers planned and we hope you will help make this exciting research a reality with us. If you are interested in getting involved to help us fundraise for this program, please contact Victoria Strong at

Together we can end SMA -- one person and one dollar at a time!

Related Campaigns

$200K For SMA—Phase Two | Funding the pre-clinical sma model $200K For SMA—Phase Two | Funding the pre-clinical sma model

On the heels of the success of the "$200K For SMA -- Phase One" campaign, in March 2011 theGSF launched "$200K For SMA -- Phase Two" to fund the first ever SMA pre-clinical animal model. theGSF...

View More Campaigns

Talk About It

Exciting!!! And if anyone can propel this at breakneck speed, it is the GSF and the SMA family smile 'Lucy'
Posted by Helen on 2011-03-04 13:32:53
[...] The inspiring Jadon’s Hope and Jim Baker are at it again! And with them you know it’s going to be something BIG. As if�climbing the 23,000 foot Mt. Aconcagua and raising over $45,000 for GSF’s “$200K For SMA” fundraising campaign wasn’t impressive enough, Jim Baker will be tackling the famous Mt. Rainier in Washington in June 2011. And he won’t be climbing alone this time. Jim will be joined by four other men for the challenging alpine ascent:�Tucker Trotter,�Justin Wood,�Garrett Ochs, and�Bill Spillar. And they’ll all be using their training and trip to fundraise for Jadon’s Hope and GSF’s new�“$200K For SMA” fundraising program focused on funding the first large SMA pre-clinical animal model. [...]
Posted by Climbing Again for SMA � Gwendolyn Strong | Gwendolyn Strong on 2011-03-30 12:42:28

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