Mission Critical: How Translation-Focused Disease Foundations May Save Medical Research

Successfully translating scientific discoveries requires a primal sense of urgency, which some disease foundations seem to have, and many big pharmas appear to need.

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A still-frame from a Myelin Repair Foundation video.

Patients waiting expectantly for medical research to produce important new cures are finding bad news almost everywhere they turn.

Pharmaceutical companies are suffering from a much-discussed innovation crisis, as old drugs lose patent protection without new drugs to replace them; meanwhile, the small biotechs that could potentially bail big pharma out struggle to raise capital .

University scientists, for their part, are beset by an unseemly credibility crisis, as the intrinsic fragility of medical research is now vividly apparent from the soaring number of high-profile retractions, and the well-documented difficulty of reproducing many published findings outside the originator's lab.

At the heart of this crisis is the misalignment of two very different cultures.

Academic scientists tend to focus on publishing papers, and usually assume that the results will eventually be useful. They place a high value on novelty, and relatively less value on whether the data are robust, easily reproducible by others, or truly relevant to human disease. Captivating data from putative laboratory models of disease generate publications, even if the model is not very predictive of human disease - and unfortunately, most models aren't.

Conversely, big companies traditionally focus on generating efficiencies through scale, and on developing reproducible processes. This works very well for manufacturing, reasonably well for large late-stage clinical trials, and essentially not at all for early-stage (discovery) research.

The result has been two systems that operate nearly independently, yet fundamentally, should be seamlessly connected; industry is absolutely dependent on the creativity-driven research of university scientists, and these scientists in turn absolutely require industry to develop their preliminary ideas into reliable products (and could probably also benefit from the opportunity to pressure-test their results in a rigorous and systematic fashion).

The answer, I suspect, may come not from either industry or academia, but rather from an emerging third party: translation-focused disease foundations.

Consider the example of the Myelin Repair Foundation (MRF, with which I have no relationship). Trying to develop solutions to restore the myelin lost in multiple sclerosis, the MRF is interested in the entire drug development "value chain," from academic research to ultimate application, but has placed distinctive emphasis on the translational step, the part where promising academic advances are rendered sufficiently robust for industrial use. They strive to reduce the activation energy required for a company to study a drug, and have even developed their own lab to facilitate this - put together and staffed by biopharma vets.

In some ways, the MRF functions like a contract research organization for multiple sclerosis, capable of validating and robustifying preclinical assays, and developing and analyzing potential biomarkers for use in clinical studies. This approach represents a stark contrast from the more traditional disease-foundation route of simply plowing money into basic academic research and clinical trials, or the recent foundation trend of funding new companies (or specific projects within companies ). Here, the explicit goal is to enable translation by building out the capabilities required to achieve it.

MRF's research facility differs from an academic center in that no academic center would - or (arguably) should -- put in the effort to industrialize the assays to the level required by industry - it's not that interesting, and probably not publishable.

It's also somewhat different from a big pharma translational effort in that, as a non-profit, it's a lot easier to get academic assistance (as well as access to university-derived IP), and even more importantly, there's a clear and enduring sense of mission. While pharmas tend to switch therapeutic focus with each change in management (and each new influx of management consultants), disease-focused non-profits are steadfast about their mission - a dedication that will persist years down the line.

Presented by

David A. Shaywitz, MD, PhD, is a director of strategic and commercial planning at a biopharmaceutical company based in San Francisco, and the co-author of Tech Tonics: Can Passionate Entrepreneurs Heal Healthcare With Technology? More

Trained as a physician-scientist and management consultant, Dr. Shaywitz has experience in clinical drug development and strategic and commercial planning. Dr. Shaywitz is co-founder of the Center for Assessment Technology and Continuous Health (CATCH), a Boston-based initiative to use improved real-world measurement to improve care and drive science. He also is co-founder of the Harvard PASTEUR program, a translational research initiative at Harvard Medical School, and a founding advisor of Sage Bionetworks, a non-for-profit medical research initiative focused on open innovation. He works at a biopharmaceutical company in San Francisco; the views expressed in his postings are his own and do not represent the views of his employer. Dr. Shaywitz is an adjunct scholar at the American Enterprise Institute. His personal website is: http://davidshaywitz.wordpress.com.

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