by Stacey DeFilippo
Dr. Bruce Bloom fascinated an HBA audience with stories of how repurposed drugs have changed the lives of children and adults with rare or neglected diseases. At Cures Within Reach, Dr.Bloom, improves patients' lives quickly and safely by finding new uses for FDA approved drugs, nutraceuticals and medical devices in treating rare diseases with no effective known treatment.
Rediscovery research has created cost-effective and quick solutions for patients worldwide. Currently there are half billon patients with diseases not being treated by larger pharmaceutical companies. Cures within Reach is filling a very important need in this world-finding treatments for these patients suffering from catastrophic diseases.
Dr. Bloom explained that “new discovery medical research” looks forward, answering the question, “What new things can I discover that might someday help patients?” Answering this question creates a significant amount of knowledge, but it takes a long time and significant dollars to impact patients. “Rediscovery research” asks, “How can we use what we already know and have to help patients now?” Answering this question quickly and affordably drives safe patient impact and it often quickly translates to other diseases and patients.
Dr. Bloom shared the story of Nicole, a twelve year-old girl with autoimmune lymphoproliferative syndrome. The drug sirolimus was repurposed to alleviate symptoms of the disease in less than 30 days. The rediscovery research cost $185,000 and the timeline was 30 months. After only two months on sirolimus, Nicole went from requiring blood transfusions every three days and spending 50% of her life in the hospital to being completely normal.
The funding for these small research endeavors comes from private philanthropy. Because rediscovery research focuses solely on the patient instead of profit, publication, promotion or protection of IP, costs are lower and timelines are significantly reduced.
Cures Within Reach has worldwide funders and institutional partnerships. Research partners include corporations, disease specific nonprofits and medical institutions. Funding partners specify their disease of interest, set the budget between $25,000-$500,000, receive $1 in actual research for each $1 donated, and see patients get better.
In summary, drug repurposing finds inexpensive and safe ways exist to increase quality and length of life for patients in less than 3 years. Dr. Bloom fulfills a very important need in our medical world by helping those suffering from rare diseases. Learning about Dr. Bloom’s work inspired all of us to continue to strive to make a difference in this world.
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