Thursday, September 21, 2017
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RCF joins The 200 Commitments Program

As a community we need to raise the $1,000,000 needed to take the work of The SMA Gene Therapy program from Nationwide Children’s Hospital/Ohio State University out of the Lab and into the SMA Patient Population!

This revolutionary research has the potential to cure Spinal Muscular Atrophy, as well as various other Neurological diseases. The earlier we can get into the SMA patient population, the greater the chances of rescuing the remaining motor neurons. The longer we wait, the greater the motor neuron loss in our SMA patient population. The time is NOW to stop the progression of the disease. Based on initial non-binding talks with the Food and Drug Administration there is a realistic timeline of what is necessary to ensure this program reaches human clinical trials. This program can reach Phase 1 in 12 months if the funding gap is closed. Based on Pre Pre IND talks with the FDA and the studies they have deemed necessary roughly $1,000,000 is needed.

We are asking for 200 commitments, every commitment is $5,000. We need 200 commitments within the next 12 months- for a total of $1,000,000 - with 100% of the proceeds going directly to fund Dr. Kaspar and the Gene Therapy program at Nationwide Children’s Hospital/OSU. More info..

200 Commitments

The 200 commitments has an extremely simple, but critical goal:

To raise as a community the $1,000,000 needed to take the work of The SMA Gene Therapy program from Nationwide Children’s Hospital/Ohio State University out of the Lab and into the SMA Patient Population

This revolutionary research has the potential to cure Spinal Muscular Atrophy, as well as various other Neurological diseases. The earlier we can get into the SMA patient population, the greater the chances of rescuing the remaining motor neurons. The longer we wait, the greater the motor neuron loss in our SMA patient population. The time is NOW to stop the progression of the disease. Based on initial non-binding talks with the Food and Drug Administration there is a realistic timeline of what is necessary to ensure this program reaches human clinical trials. This program can reach Phase 1 in 12 months if the funding gap is closed. Based on Pre Pre IND talks with the FDA and the studies they have deemed necessary roughly $1,000,000 is needed.

We are asking for 200 commitments, every commitment is $5,000. We need 200 commitments within the next 12 months- for a total of $1,000,000 – with 100% of the proceeds going directly to fund Dr. Kaspar and the Gene Therapy program at Nationwide Children’s Hospital/OSU.

How you raise the money is up to you. Whether it is a run, a triathlon, a bake sale, a bowling event, a gala, a spaghetti fundraiser, the list of possibilities is endless. Make it fun, get your community involved. Do you know a company that is willing to sponsor a commitment? We will post all corporate sponsors on the front page for the entirety of this campaign! Can you raise $10,000? That would be amazing and would deduct 2 commitments from the goal! We welcome any and all other SMA organizations to join on board!

$1,000,000 seems like such a daunting task but it is a goal that is a achievable……. it will just take teamwork. We are not afraid of taking on a challenge so daunting……. we may not succeed, however we will try as hard as we can to make this trial a reality. Are you ready to become 1 of THE 200?

What is SMA?

  • SMA is the #1 genetic killer of children under the age of 2
  • SMA is estimated to occur in nearly 1 out of every 6,000 births.
  • 1 in every 40 people, or nearly 10 million Americans, unknowingly carries the gene responsible for SMA.
  • SMA is a pan-ethnic disease and does not discriminate based on race, ethnicity, or gender.
  • There is currently no treatment and no cure, but the National Institutes of Health (NIH) selected SMA as the disease closest to treatment of more than 600 neurological disorders.
  • Researchers estimate that a viable treatment and/or cure is attainable in as little as 5 years if provided the necessary funding.

Gene Therapy

Minimize

The TeamLead Researcher Dr. Brian K. Kaspar, Ph.D. Associate Professor The Research Institute at Nationwide Children’s Hospital /The Ohio State University working on Gene Delivery of SMN using AAV9 vector.

Co-Collaborator/ Co-Lead Dr Arthur Burghes, Ph.D Professor Ohio State University has contributed to 31 publications on Neuromuscular diseases.Burghes Labs at OSU have made significant contributions to Spinal Muscular Atrophy which include The Carrier Screening test for SMA, The Genetic Diagnostic test for SMA, and The Original SMA Mouse Model.

Jerry R. Mendell, M.D.- Dr. Mendell currently serves as Director of the Center for Gene Therapy at The Research Institute at Nationwide Childrens Hospital and holds Professorships in Neurology, Pediatrics, Pathology, Physiology and Cell Biology at the Ohio State University. Dr. Mendell has had a long history of partnering with the MDA on neurological diseases and brings an extensive track record of working with the FDA to successfully move programs from the lab and into clinical trials.The ProgramThe Kaspar Labs using AAV9 for Gene Delivery has progressed at an incredible pace.

Initial funding for Kaspar Labs using AAV9 first came in from the SMA organizations in 2010. This program has the potential to be in Human Clinical trials by 2012.

A  combined total of $600,000 has been donated to Kaspar Labs from multiple SMA Organizations and the amount of information that has come from that contribution is astounding. Similar programs in the pipeline have received between $2,000,000 and $15,000,000.

The Pre-clinical data for this program has been the most promising of any other therapy to date for Spinal Muscular Atrophy. In the most severe animal model of Spinal Muscular Atrophy early intervention has validated a full reversal of the disease. In larger animal models targeted motor neurons were hit successfully and efficiently suggesting the ability to stop progression in more advanced subjects.

AAV9 effectively crosses the blood brain barrier efficiently  which has been a major hurdle blocking the advancement of many SMA therapies. This separates AAV9 from other Gene Therapies such as rAAV and AAV that must be administered via cranial injections or intrathecal injections directly into the spinal fluid. AAV9 can be injected directly into the blood stream similar to an immunization shot.

AAV9 virus has already been introduced into thousands of human subjects through various clinical trials. The AAV9 vector has proven to be effective and well tolerated in humans.

The overwhelming positive results of pre-clinical efficacy and initial safety studies have provided the impetus for advancing this program towards the clinic. The initial talks with the FDA have given a clear path forward for studies that are necessary for a formal IND application.

For the very first time in over 20+ years of SMA Research here is a program that has overwhelming pre-clinical data that shows success on the most severe animal model of the disease. These pre-clinical studies suggest that Gene Delivery using AAV9 specifically is an effective and well tolerated approach for Spinal Muscular Atrophy. All research is a gamble and this program has shown the greatest odds of a successful outcome to date. To read well written published articles on The Kaspar Labs please click here: