Sometimes funding research is not about testing the safety or effectiveness of a potential drug, device or biological therapy in animals or humans. Sometimes funding research is about determining if a particular protein can be produced in a cost-effective manner for treating patients.
The ALS Research Group at Wake Forest Baptist Medical Center, led by researcher Carol Milligan, Ph.D., neurobiology and anatomy, is looking for ways to treat Amyotrophic Lateral Sclerosis (ALS), more commonly known as Lou Gehrig's disease. Her recent work, using “heat shock” proteins to help control muscle movement, showed that their use in mice delayed the onset of ALS symptoms and extended survival times.
Heat shock proteins help prevent the destruction of motor neurons that can result in a number of neurological diseases including ALS.
ALS is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord. Motor neurons reach from the brain to the spinal cord and from the spinal cord to the muscles throughout the body. When the motor neurons die, the ability of the brain to initiate and control muscle movement is lost. With voluntary muscle action progressively affected, patients in the later stages of the disease may become totally paralyzed, unable to even breathe on their own. The progressive degeneration of the motor neurons in ALS eventually leads to death.
Last year, Milligan hit a roadblock when the cost to purchase heat shock proteins became too expensive to continue her promising research. She needed to find a way to easily and economically reproduce the heat shock proteins.
Most private companies won’t consider commercialization of a product that is not proven, even if the product shows promise in early research studies.
Milligan found a partner in Wake Forest Innovations, the commercialization arm of Wake Forest Baptist, which has the resources to produce the protein and determine the viability of a commercialized product, but she still needed the funding to do it.
And it would be costly. In spite of support from Wake Forest Baptist’s Department of Neurology and ALS Center, she still needed at least an additional $200,000 to begin to translate the basic science of animal study to clinical trial in humans.
Enter the Blazeman Foundation. The Massachusetts-based non-profit is a family foundation started in memory of the late Jonathan "Blazeman" Blais. Blais, who was diagnosed with ALS at age 33, was a triathlete who died of the disease.
The foundation’s mission is to raise awareness about ALS by leveraging the energy, commitment and compassion of the multi-sport community, to raise the necessary funds to be directed into cutting-edge scientific research to find treatments and an eventual cure for ALS. The foundation’s approach to research funding is unique. It funds promising projects that no one else is working on.
"Bob and I are excited to participate in this collaborative research project at Wake...having worked with Carol on a previous project we have all the confidence that the outcome will be valuable for ALS research," said Mary Ann Blais, Jon’s mom and vice president and event coordinator for the Blazeman Foundation.
Fortunately, Milligan’s research fit the family foundation’s vision, and it provided more than $200,000 in funding for the heat shock protein production project.
During the two-year project, Wake Forest Baptist’s ALS Research Group will develop a reliable and reproducible procedure to make and purify the protein. Once the protein is made, it will be tested in multiple assays to assure that it has the correct structure and function. Preliminary experiments will be performed at Wake Forest Baptist to confirm that the protein works as expected in the mouse model of ALS, and then the protein will be sent for independent confirmation at another laboratory. The work that results from this Blazeman-Wake Forest Baptist collaborative project will lay a foundation for large-scale protein production and clinical trial.
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