Improving patients’ lives quickly and safely by targeting small research investments to repurpose existing FDA-approved pharmaceuticals, treatments, and medical devices.
Dr. Bruce Bloom has pioneered a way to improve patients’ lives quickly and safely by repurposing existing drugs, treatments and medical devices. Rather than waiting for elusive scientific breakthroughs, Bruce brings researchers, clinicians and philanthropic partners together to speed promising research into patient care to improve quality and length of life for patients with debilitating, but often rare, diseases.
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.
Our current medical research system does not reward the most effective methods to develop treatments and cures for patients with life-altering diseases quickly. Many patients, especially those with orphan diseases, live without effective treatments. Industry and government have used over $400 billion dollars for medical research since 1970, yet only a handful of cures have been created for even the most common life-threatening diseases. We still use insulin to treat type I diabetes; of the top 50 adult cancers, not one has been cured, and a majority of the current therapies for these cancers were developed over 10 years ago. There are no safe, effective, affordable treatments for MS, Parkinson’s diseases, ALS, lupus, or other common diseases.
Government, academia, industry and large, disease-specific non-profits play a vital role training researchers, creating cutting edge knowledge about diseases, formulating new drugs and improving technology. However, their structures and missions do not typically harness the available resources to impact patients quickly. Because FDA approval for new drugs typically takes more than $1 billion and 15 years to complete, corporations can’t profit from treatments for diseases affecting smaller numbers of people (generally less than 100,000 people), so large-scale research funding is focused on treatments that could affect more common diseases. Pharmaceutical companies aim to achieve the greatest market and profit for each drug. There is a gap in rare conditions, because there is little potential to fund pre-clinical or clinical work.
In 2006 the FDA only approved 17 new drugs. In addition to the extensive cost and time, there are strict regulatory processes designed to protect the public and the high cost of litigation when something goes wrong, so drug companies are wary of which drugs they bring to market. Last year more than 50 drugs were refused approval at the last step of the billion dollar process, adding to the overall cost of drugs and eliminating potential cures for patients.
There are more than 1.5 million medical researchers worldwide, and over $120 billion is spent worldwide on medical research each year. Billions of patients wait for better treatments and cures. There is a huge amount of scientific knowledge sitting unused, both in publications, and more importantly, in the minds of clinicians and researchers. A poll by Partnership for Cures found that 56% of disease specific researchers and 19% of clinicians had at least one potentially powerful research idea that could quickly, affordably and effectively touch patient lives.
Three critical factors stymie the creation of affordable, safe and effective new treatments. First, current therapy development requires the combined efforts of industry, academia and government, yet there is no coordinated system to support this process, and significant new economic and regulatory hurdles arise to further debilitate the process. Second, therapy development is driven by ineffective rewards systems: profits in industry, promotion in academia and program bureaucracy in government. These systems create expensive therapies, well trained scientists and expansive scientific knowledge, but not safe, affordable, and effective therapies and cures for patients. Third, there is no system to get clinical discoveries to patients – no way to find and validate anecdotal treatment successes that might benefit patients.
Further, researchers and research institutions are motivated by achieving large-scale breakthroughs, for their own notoriety, promotion, tenure and larger funding opportunities. Less than 5% of NIH funding is directed toward innovation, and even those programs don’t require that results that will touch patients. Industry continues to focus on large patient populations and drug/device therapies that can generate significant shareholder returns, not patient impact. Academia promotes scientists who generate high profile publications and durable funding streams but do not provide incentives to drive treatments to patients. Disease-specific non-profits add billions of dollars to this funding system, mostly to academic institutions in support of creation of new knowledge and long-term progress, but not immediate patient impact. In at least one instance, two scientists in different institutions had found the answers to the other’s research roadblocks, but to prevent one from achieving faster or greater notoriety, the institutions forbid the scientists from sharing vital information that could help achieve patient impact.
Finding treatments for orphan diseases is even more difficult. Thousands of small, disease-specific organizations exist, typically founded by parents of children with rare diseases, but no mechanism exists to address orphan diseases collectively, and few researchers are searching for treatments or cures for such small patient populations.
Time and money are luxuries that patients battling illness do not always have. Through the Two Years to Cures (2Y2C) Initiative, Partnership for Cures teams up philanthropists and researchers to find new uses for already FDA approved drugs and other therapies to quickly and inexpensively improve the quality and length of life of those who need help now. This applied research uses already available drugs, knowledge and technology created as large non-profits, industry and government support long term medical research in the search of cures.
Partnership for Cures uses a five-step process to identify these innovative treatments. First, P4C locates potential funding partners and demonstrates how partnering significantly increases the impact of their philanthropic dollars. After defining a project, P4C sends a “call for ideas” to a wide range of clinicians and researchers in the field, including those at major research institutions, those who have published on the topic, or a related issue, as well as those in their existing database. With a typical 30-40% response rate, Bruce and P4C’s Science Review Board evaluate the two-page briefs, rank them according to potential for impact, and send to a group of independent scientists for review. These scientists rank each proposal, and P4C asks the researchers submitting the top 6-8 entries for full proposals. P4C shares these with the funders and identifies which is the most promising to have direct impact on patients within two years. P4C provides small grants, typically less than $100,000, to fund this research.
While the donated funds go directly to research, the research institution contributes 10% of the research cost to cover P4C’s administrative costs. This contribution ensures quality and efficiency of the research, since the institution is equally invested. P4C stays very involved throughout the process to ensure business rigor to compress timelines, leverage investments, report widely and provide complete transparency. Especially within small disease communities, news of successes spreads rapidly, through medical journals and disease-specific support groups.
Since December 2006, more than 20 funding-research partnerships deploying nearly $2 million dollars invested through P4C and matched to more than $13 million dollars. P4C projects are clinically helping over 100,000 worldwide patients right now who have multiple myeloma, ALPS, traumatic brain injury and familial dysautonomia. They have partnerships with major hospitals and research centers including Mayo Clinic, Stanford and Massachusetts General.
P4C ultimately intends to increase research funding to $10 million annually, while establishing “franchises” in disease-specific US organizations and expanding the model to other countries. P4C also aims to develop an online portal to help researchers and funders find each other more efficiently. As more successes occur, P4C will engage government, academia and industry to participate, both by devising outcomes that fit into their current reward systems and changing those reward systems to focus on patients and healing. They will work with medical research publications to provide publication space for repurposing research.
The oldest sibling in a poor family, Bruce Bloom is inventive by both nature and nurture. His belief in the importance of contributing to one’s community was ingrained by his mother, who volunteered consistently, without expectation or desire for recognition. Bruce worked as a counselor at a camp that brought kids of all races and backgrounds together during the racially heated 50’s and 60’s. When he was 12 years old, his mother took him to observe the infamous 1968 Democratic National Convention in Chicago.
Drawn to dentistry by the kindness of his own dentist, who became a lifelong mentor and friend, Bruce created a job at the dental office. The dentist practiced in a wealthy, conservative suburb but was fiercely liberal and treated several members of the Black Panthers in his office. Bruce heard him telling them that while their ideals were important, their methods were ineffective because of their violence. Although the influence of the training and his mentor were great, he was ultimately unable to practice dentistry because of a severe eye condition.
With training in dentistry and law, Bruce conceived of, launched and run a series of business initiatives related to the field of dentistry. Each endeavor was undertaken based on an original insight Bruce developed after his persistent curiosity compelled him to find out how and why things were working in a particular way. When legislation passed in the 1980’s mandating that individuals, not doctors, were responsible for deciding their own treatment, he founded a dental claims insurance business and began investigating how dentists handled various patient groups. He discovered that insured patients received substantially more services than the uninsured, and that dentists often demonstrated illegal bias against openly gay and HIV-positive patients. Later, Bruce then founded a successful company creating original art for dental offices, recognizing that dentists consider themselves artists and would appreciate beautiful artwork.
After a successful career in the private sector, Bruce had a series of experiences that profoundly impacted his perspective and sense of purpose. His brother had committed suicide, leaving him devastated. He also watched a friend suffer from melanoma, going through painful and ineffective treatments that drained his spirit and ultimately killed him. A friend advised that he would benefit from expanding his perspective beyond what he had learned from his mother, that the two ways of doing things were “her way or the wrong way,” so Bruce took time and training to become more able to work with people to achieve a shared goal. Bruce shared this with his children, pioneering the idea in his community of sponsoring service projects instead of having the traditional elaborate mitzvah party. For his daughter’s bat mitzvah, his family invited 200 volunteers and invested $35,000 to build a new community playground! Bruce realized he should look to the social sector for involvement beyond board seats and volunteering.
At its inception, Goldman Philanthropic Partners was looking for an entrepreneurial leader to manage a $5 million foundation to fund new medical treatments. Bruce realized it was the ideal venue for his entrepreneurial spirit, love of research and passion for social impact. After two years, Bruce noticed that his smaller grants were having disproportionate impact on patients, while larger grants were not producing as much actual impact on patient lives, he called researchers and clinicians to ask why. More than 60% had anecdotal evidence that existing treatments could be used to touch patients in two years or less, but no one would fund the research to prove the cause and effect. The Goldmans had intended for the foundation to close when they money had been spent, but when only $400,000 remained, Bruce singlehandedly convinced the Board to invest remaining funds in Partnership for Cures. He took over leadership and led the transition to Partnership for Cures as an independent organization.
Bruce recognizes the value of the existing research with its scientific focus and long-term goals. “I don’t want to change the existing system all that much,” he says. “We need all of that science to inform what we do. I want to create an entirely new system that will make a difference in people’s lives right now.”