Ashoka Fellow
Fellow Since 2009

Citation

This profile was prepared when Bruce Bloom was elected to the Ashoka Fellowship in 2009.
The New Idea
Dr. Bruce Bloom has invented a systemic way to transform medical research to focus on maximum patient impact by reusing or repurposing drugs for the benefit of new patient groups. By using approved medical drugs and therapies in new ways, this approach avoids a typically laborious and lengthy federal review process. Bruce acts on well-founded hypotheses of researchers and clinicians about simple, low-cost, effective ways to re-use treatments to demonstrate that existing therapies can dramatically improve quality and length of life for people suffering from life-altering diseases.

This idea takes advantage of significant unused resources to support patients. There are more than 4,000 safe and effective FDA-approved drugs being prescribed for patients, many with potential to treat other diseases. Research to discover these additional uses take far less time and cost than would be needed to develop a new drug. They can be affordably manufactured, and the regulatory cost of repurposing is a small fraction of the cost of new therapy development. Although these treatments are relatively quick and inexpensive, however, there is no incentive in the corporate or scientific community to research treatments for the more than 1,150 “orphan” diseases, which affect 20,000 people or fewer in the United States.

While government does not have repurposing programs, it does have policies that support research for repurposing. FDA rules allow this kind of research even without the permission of the drug and device manufacturers, and it allows physicians to prescribe new uses for old therapies once they are proven effective, even before a company seeks regulatory approval to market the new use. This speeds the delivery of new and less expensive treatments to patients. Further, tax incentives have been introduced for pharmaceutical companies who are willing to research potential treatments of orphan diseases using existing – but no longer profitable – drugs.

While the most direct benefit is improved quality and length of life for people suffering from these diseases and their families, the potential impact on the health care system more broadly is profound. Children suffering from rare diseases like ALPS (Autoimmune Lympho Proliferative Syndrome) often endure lengthy hospital stays and costly treatments, resulting in an average annual cost of care of more than $100,000. Even if only 500 children received the alternative, low-cost treatment developed with the support of Partnership for Cures, more than $50 million each year would be saved in hospital expenses, costly drugs and additional research. More importantly, the children would benefit from improved quality and length of life.

Further, many discoveries about orphan diseases have larger-scale implications. Researchers in Chicago were unable to get funding to research the benefits of a diabetes drug for Multiple Sclerosis patients because a generic equivalent was about to be released, so the manufacturer had no profit motive. With P4C funds, researchers demonstrated the benefits of repurposing the drug, which was oral rather than injectible, with much lower side effect risk. As a result, they published the study and applied for larger, follow-on grants from the MS Society and NIH to explore its impact on MS and other inflammatory conditions causing neuro-degeneration.
The Problem
The Strategy
The Person

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