Dr. Bruce Bloom
January 17, 2012
Partnership for Cures Patient Impact Initiative Funds 4 Rediscovery Research™ Projects for 2011
Chicago —Philanthropists and Partnership for Cures (PFC), a public charity that repurposes existing therapies for new uses in new diseases, have funded four Rediscovery Research projects as part of the Patient Impact Initiative. Each project has the potential to impact patients in the next 3 years, either by completing a clinical trial that could establish a better standard of care for treatment, or by creating a clinical trial that will enroll patients to test a “new” therapy.
The four projects include a project testing whether repurposed drugs can be used to make radiation therapy more effective in defeating metastatic cancer. High dose radiation can wake up the body’s immune system to eliminate metastases, but the dose needed is often too great for patient safety. Certain drugs can sensitize the cancer cells to the radiation, allowing a lower dose to be lethal to the cells and still wake up the immune system. This project is the last research necessary before applying for approval to conduct a human clinical trial for patients who right now have no effective therapy.
A second project is testing a repurposed drug to help fight a wide variety of rare and deadly childrens autoimmune diseases, building on successful research that showed that the drug sirolimus could be effective in reversing the symptoms of the genetic disease Autoimmune Lymphoproliferative Syndrome (ALPS). Patients are already being enrolled in this clinical trial, and initial response has showed that more than half of the patients have had a complete response to therapy. Considering this trial is only for patients who have failed all prior therapies, the results are very encouraging. This drug is relatively safe and very inexpensive.
The last two projects involve improving patient compliance in children newly diagnosed with insulin dependent type 1 or type 2 diabetes. These young kids are asked to instantly take on very adult like responsibilities, checking their blood, injecting insulin, monitoring their food intake, understanding the changes in their body functions. This Rediscovery Research project is designed to give them an alternative way to exercise this kind of adult like responsibility, so they can see the impact it has. The hypothesis is that seeing the impact on something else will alert the kids to the self-impact of taking on these personal responsibilities.
About Partnership for Cures:
Partnership for Cures raises funds for Rediscovery Research™ that tests already approved drugs, devices and other therapies to quickly, safely and affordably create new treatments for patients with catastrophic diseases. Why do we focus on repurposing?
Compared to New Drug Research - Repurposing is 1,000 times more likely to succeed (some stats actually suggest 3,000 times), 3,000 times cheaper to bring to human use (albeit comparing a repurposed device that can be immediately used off label vs. going through FDA clinical trials), and much safer (drugs used are often 40 years old and side effects and drug to drug interactions are well known).
About the Patient Impact Initiative:
Drugs and devices that might provide treatments for diseases are not pursued when they don’t fit the for profit pharmaceutical model. This includes FDA approved drugs/devices that might have new indication but have no patent protection, patent protected therapies that could target a tiny disease market, and drugs that “failed” during research for one disease but could succeed for a different disease.
The Patient Impact Initiative is public-private partnership to source, fund, test, and market these drugs and devices for new indications. The Patient Impact Initiative funders, researchers and industry partners have done every part of this Rediscovery Research™ process as individual organizations-
Success may simply be publication of a proof of concept clinical trial that allows physicians to decide if off-label use could help their patients. In other cases market potential may be sufficient to cover the costs of a very efficient FDA approval process for a new indication. Either way, Rediscovery Research quickly, safely and affordably creates “new” treatments and saves significant research and healthcare dollars.
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Partnership for Cures raises funds for Rediscovery Research™ that tests already approved drugs, devices and other therapies to quickly, safely and affordably create new treatments for patients.