Alex Philippidis Senior News Editor Genetic Engineering & Biotechnology News

Translation-focused disease foundations could help bridge the gap between academia and industry.

Patients keep waiting longer and longer for research to translate into new drugs as development costs rise. Estimates range from over $1.3 billion (Tufts Center for the Study of Drug Development) to between $4 billion and $11 billion (InnoThink Center for Research in Biomedical Innovation).

Fueling those numbers is a clash of cultures: Academia typically focuses on generating knowledge for knowledge’s sake. Industry focuses on ever-increasing efficiency.

The best hope for bridging that gap—thus cutting time and money from drug development—rests with translation-focused disease foundations, David A. Shaywitz, M.D., Ph.D., co-founder of the Center for Assessment Technology and Continuous Health (CATCH), recently posited in The Atlantic.

A good example of straddling academia and industry, as Dr. Shaywitz noted, is the nonprofit Myelin Repair Foundation (MRF). The foundation says its Accelerated Research Collaboration (ARC) model bridges the logistical, cultural, and financial distance between academic labs and licensing by commercial drug developers, with the goal of bringing new medicines to patients 50%–70% faster than current drug development.

MRF is applying that model in developing a new myelin repair drug for multiple sclerosis (MS). In June, the foundation initiated a Phase I trial at Cleveland Clinic to test the efficacy of a myelin repair therapeutic pathway with mesenchymal stem cells. The trial is based on foundation-supported research by Robert Miller, Ph.D., of Case Western Reserve University.

“Our goal initially was to reach that goal in 2014. And we reached it at the end of 2011,” Jennifer Chang, a foundation spokeswoman, told GEN.

MRF’s drug trial was its second clinical trial launched this year. The first was a clinical proof-of-concept trial to determine safety and efficacy of a potential therapeutic pathway to desensitize the immune system to myelin in MS patients. Most approved MS therapies suppress the body’s immune system. That trial is based on MRF-backed research by Stephen D. Miller, Ph.D., of Northwestern University’s Feinberg School of Medicine.

“The biggest indications for this type of technology rest outside multiple sclerosis—juvenile diabetes, allergies, arthritis, any autoimmune disease. It’s really not in our sweet spot of myelin repair,” Jay Tung, Ph.D., MRF’s vp, drug discovery and research operations, said. “We are looking at innovative ways to uplicense this technology, where our motivation isn’t to get a pile of cash, but to be good stewards of this intellectual property and move it forward.”

MRF envisions a licensee that is not a biopharma: “There are some foundations with much, much deeper pockets that will enable the commercialization of this. And in doing so, the proof-of-concept within that initial indication would be very valuable to us,” Dr. Tung said.

MRF counts among its strengths its ability to surmount the academic-industry divide; an internal research team focused on clinical drug delivery; the expertise of researchers boosted by its advisory boards of academic and industry scientists; and its own laboratory, the MRF Translational Medicine Center, staffed by internal researchers and opened late last year. The lab fosters research with academic partners that include Case Western and Northwestern University, as well as Stanford University; University of California, San Francisco; University of Chicago; and The University of Melbourne.

“We pay for the research, we have a project manager go in, watch for intellectual property, and when something comes up, we file those invention disclosures at our cost. The university still owns the IP, but our foundation has exclusive rights to license it,” Dr. Tung said.

Dr. Tung, who left Eli Lilly for MRF six years ago, says the foundation is funding a clinician at NIH’s National Institute of Neurological Disorders and Stroke, allowing for access to human tissue, and enhancing the foundation’s ability to do clinical trials since the clinician’s work dovetails with its biomarker effort. However, MRF doesn’t rely on NIH to fund research proposals using traditional peer review.

“We actively seek out the best science. We have a scientific advisory board. We don’t use them to peer review what investigators are doing. Instead, we utilize them to help our group cooperatively refine our research strategy,” Dr. Tung said.

“That peer-reviewed process has some benefits, but also has some disadvantages. It’s an old-buddy system,” Dr. Tung said, echoing a long-standing criticism that NIH has long rejected. “It’s long and tedious. We feel we know exactly what we want in terms of filling the void of translation medicine, and we go out and get it. That’s how we expand our team.”

He said MRF is starting to partner with pharmaceutical organizations: “It’s still a work in progress, but we’re fostering half a dozen partnerships where we are testing their chemical matter for myelin repair potential.”

Earlier this year, the foundation signed its first such partnership with ENDECE Neural to advance myelin regeneration drug candidates for MS patients through preclinical and into Phase I studies. Dr. Tung confirmed MRF has had talks with Vertex Pharmaceuticals but wouldn’t identify other biopharmas.

While it’s too soon to tell if MRF or anybody else can develop a drug 50% to 70% below current costs, the attempt is laudable and should find the support needed among partners, since biopharmas, academia, and even foundations have an interest in saving money.

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