10 Fox Focus | Research
New Funding Approach Turbocharges De-risking Strategy
By Joe Nocera
It ’ s no secret that it takes a very long time to develop a drug — anywhere from 12 to 20 years , says Shalini Padmanabhan , PhD , vice president for drug discovery and translational research at The Michael J . Fox Foundation . “ Super long ,” she acknowledges with a small sigh . And of course , it ’ s also no secret that far more compounds fail than succeed . Which is why pharmaceutical companies have often needed an incentive — a friendly nudge , you might say — to work on a drug in its earliest stages , long before the clinical trials that are needed to get approval from the Food and Drug Administration .
When it comes to Parkinson ’ s disease , MJFF ’ s mission is , in no small part , to provide that nudge . It calls this crucial aspect of its work “ de-risking ,” a broad term that encompasses a variety of approaches . One approach , says Padmanabhan , is “ to solidify the biology around a target and link it to Parkinson ’ s in some way that makes it attractive to a company ” — in the hope that they will then grab the baton from MJFF and try to take it across the finish line . Another approach is funding research at small biotechs that are attempting to develop compounds the Foundation believes have
promise . The fact that three such biotechs that had received MJFF grants — Mitokinin , Cerevel and Caraway Therapeutics — were all acquired last fall by major pharmaceutical companies is proof of the strategy ’ s success .
Yet there remains a lot more de-risking MJFF can do . “ In the past we emphasized an investigatorinitiated approach where we would say , ‘ Hey , we ’ re interested in this activity ,’ and let the community come to us with ideas ,” says Padmanabhan . “ Over the years , we ’ ve spent an enormous amount of resources on developing 100 to 200 targets .” This activity was critical to seeding the pipeline . More recently , however , it became clear that the Foundation ’ s most successful efforts took place when it was more proactive — “ bringing together communities around a challenge and addressing what we think are the key gaps in funding ,” as Padmanabhan puts it .
Now MJFF is innovating again – with a new derisking initiative , one that is both more proactive and more targeted , which the Foundation hopes will accelerate the process of developing compounds that will mitigate and eventually cure Parkinson ’ s disease .