The Myelin Repair Foundation is more than just a policy precedent — it’s also emerging as the stimulus behind a future cure for MS.
The decades-old movement for “patient power” is evolving in unexpected directions, as a few vanguard organizations explicitly abandon that vague agenda around “advocacy” for something more granular and concrete. Yes, get ready for a new era, one where patient groups are no longer just a touching symbol of unmet medical need but lead instigators in revitalizing R&D pathways that lie dormant because Big Pharma’s money isn’t connecting to the cure.
An exemplar of this trend is the Myelin Repair Foundation, founded 12 years ago by Scott Johnson, a former Silicon Valley venture capitalist whose professional life has been defined by the diagnosis of multiple sclerosis (MS) he received at the early age of 20. Johnson launched the Foundation after noting that little, if any, research by the pharma majors addressed the damage the disease does to the protective myelin coating that encases nerves in the body; MS erodes this coating and prevents its repair, leading to the progressive loss of the motor control and basic cognitive abilities controlled through neural signaling.
Instead, the focus of both public and private-sector R&D has been on creating drugs that attack MS by mobilizing the entire immune system, a scattershot response with significant side-effects and which Johnson believed did little to slow deterioration of the myelin properties essential to healthy neural function – the root of what actually ails patients. With two million people living today with the disease, at varying stages of progression, the therapy response seemed unnecessarily attenuated and conservative.
According to Johnson, the situation has changed, largely because of his group’s single minded focus on expanding the boundaries of MS research, particularly in the key area of myelin repair. In a recent discussion with Pharm Exec, Johnson described his role less as a patient adv
ocate than as a “coordinator and facilitator of research geared to widening the investigatory horizons for an eventual cure for MS.” The Foundation is organized as a 501c3 non-profit and thus has the independence and flexibility – no shareholders – required to identify gaps in the science and to work with multiple partners, avoiding the bureaucratic conflicts that plague liaisons between profit-seeking commercial organizations as well as with the government agencies that set the complex criteria for basic research.
“When we started, I had no background in industry or academic research; had this been the case, I would have been too aware of the challenges and most likely would never have taken the risk of launching the Foundation,” Johnson said. What Johnson saw that others missed was the silo thinking that provided little incentive to pursue leads in new areas of science, using multi-disciplinary tools like test assays, cell-based drug screens, animal models and biomarkers that translate basic research into a clear pathway to clinical development. “Our niche is that through the Foundation’s investments in translational research we can de-risk what the profit-seeking pharma companies have to do when putting their own money into new investigational platforms on MS.”
Three ways to succeed
The Foundation has pursued three simple objectives since its start in 2002. The first is to raise the profile and investment in R&D programs specifically focused on myelin repair. A key vehicle here is the formation of multiple partnerships with academia, government entities like the NIH, disease organizations and other external interests. The second is to lure Big Pharma to the research table by lowering their risk exposures and to validate new research investments in myelin repair. An example is the Foundation’s Accelerated Research Collaboration (ARC) model, which sets time-efficient guidelines for moving a compound quickly beyond proof of concept to actual testing in the human population. The third – and biggest – hurdle is contributing to the successful registration and launch of a new drug centered on the repair and rebuilding of myelin damaged by progression of the disease.
In gauging where the Foundation is today on these three objectives, Johnson says its biggest win has been to expand research on MS toward a greater diversity of approaches. He notes that a dozen pharma companies are now funding work specifically on myelin repair, while the Foundation has forged productive partnerships with more than 70 scientists affiliated with top academic institutions like Stanford, University of Chicago, Northwestern, and Case Western Reserve. In addition, its ARC model has begun to demonstrate real worth to the R&D community, with the ARC’s automated throughput technology producing over the last few years a tenfold increase in the number of therapeutically relevant compound targets for myelin repair.
As proof of the appeal of its research collaborations, Johnson cites the sublicense the Foundation granted to Biogen-Idec last year to build a mouse model for testing against MS and other health conditions that produce damage to the myelin coating. The two will work together in applying the model to test compounds that may reverse this effect and lead to clinically meaningful and faster treatments for patients.
Although the timing of the third goal – an approved new drug for myelin repair – has been put off to 2019, the Foundation can point to progress in initiating Phase I clinical trial work. Last month, the Foundation and the National Institute of Neurological Disorders and Stroke (NINDS), a unit of the NIH, announced a Cooperative Research and Development Agreement (CRADA)to test and develop a Foundation-sponsored compound, MRF-008, as a possible neuro-protective therapy to restore myelin in MS patients.
Repurposed for repair – is R&D ready?
Interestingly, MRF-008 is a generic originally registered to treat hypertension but has been identified by Foundation researchers for the stimulus it provides to myelin repair. Johnson told Pharm Exec that repurposing of older generics is a promising field in MS as well as other unrelated disorders, but it remains starved for attention due to the lack of incentives for the industry to put money behind any product lacking patent protection. “Few companies want to invest in a trial where there is zero prospect to recoup that investment around a generic. That may make sense commercially, but is it a good thing for the patient?”
Thus, it falls to the Foundation to help answer that question – this time, with a solution. It may seem modest, but there is merit in this strategy to move research just far enough to get a cash-rich pharma company interested in taking on myelin repair to full registration. Otherwise, you’d have to conclude that the critics are right in claiming we have a drug system that is broken. For that reason alone, The Foundation is worth keeping an eye on.