Because Research Matters………..

So what’s next you guys?………….

As soon as we wrapped up funding on the Gene Therapy Program that was the first question asked by so many of our supporters. The day after we received many phone calls, emails and instant messages asking us where we go from here. We cannot wait to share our vision for The Sophia’s Cure Foundation and how we can further the Spinal Muscular Atrophy research community..but first………….

We are so excited that SCF has wrapped up such a long and focused commitment to advance AAV9 towards a clinical trial. What was necessary to advance Gene Therapy towards a human clinical trial was sustained support at extremely high levels. We most certainly take a different approach to SMA research and we are absolutely ecstatic that we have pushed  a very promising research program along at a pace that has never been accomplished before in SMA for a novel therapy. We are also very proud to say that the translational team did not shy away from the most severe phenotype. Instead this team decided to try to start in the most aggressive form of the disease. There are many questions that must be resolved and the only way to answer these questions is by attacking the most difficult problems first. It was amazing to watch the initial doubt, turn into support over the years and eventually that support turned into excitement. Our programs have raised an eye opening $2.3 Million dollars for Kaspar Labs at Nationwide Children’s Hospital. The very generous support SCF received from our donors enabled us to leverage their donations and multiply those numbers into great sums. Quite frankly without that multi-million dollar investment, Dr Brian Kaspar’s Gene Therapy Program would be many years away from where this program is today. Years in SMA are in fact lifetimes. When programs take 10 -15 years to reach a clinical trial in SMA, that means 5 – 7 generations of SMA children will pass before ever trying a single therapy on the patient population. Our goal in an aggressive disease such as SMA was to take the 15 year time frame for potential drug therapies and shorten that as much as possible. We are ecstatic that this program is marching towards an IND application in just 3 years. So Thank You to our donors!

So what is next for Sophia’s Cure Foundation? We have already been working on some great programs and strategies that will answer some very critical questions for Spinal Muscular Atrophy and especially the older patient population. While a Larger SMA Animal model was not necessary to advance Gene Therapy to the clinic, we agree that it is an important tool for SMA research. A larger Animal Model is not a new idea, one can find grant proposals online as far back as 2007 such as this link here. From our perspective if the SMA research community was asking for a larger animal model, we were going to help out. As an organization we had the confidence in the researchers we had been working with and came to an agreement to lend out some of the virus we had produced for the AAV9 studies to create a model. We are ecstatic that in just 9 months Dr. Arthur Burghes and Dr. Brian Kaspar teamed up to create not only an SMA Larger Animal but the first animal to present a clear phenotype of SMA. The PCR on the animal confirmed it had SMA.

We were simply not happy just creating an SMA Larger Animal, we wanted to take things a step even further and ask some critical questions. A couple of animals were created and there was a clear understanding of the progression of the disease. The next logical question was can we rescue this animal? …………That answer was yes. An additional cohort of animals was created and half of them were re-administered AAV9. The animals receiving the AAV9 SMN were rescued while the animals left untreated degenerated and progressed in the disease just as the first set of animals did. The future for Sophia’s Cure is to fund further studies in this SMA Pig Model and to fund revolutionary ideas in the hopes of improving the lives of those that are more advanced in the disease. One of the things we are looking into is if we can re-innervate axons of motor neurons that are potentially still alive. We appreciate your support in our future endeavors and our goals of gaining the greatest improvements possible for those suffering from Spinal Muscular Atrophy.

We would love to share with the SMA community the amazing advancements we have funded for Spinal Muscular Atrophy. Here is a temporary link where you can watch the SMA Larger Animal and phenotype created with the support of  The Sophia’s Cure Foundation and National Institutes of Health.

Thank you once again for your support!

The Sophia’s Cure Foundation…………because research matters!

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