In April’s issue of NonProfit Pro, Annette Bakker, President and Chief Scientific Officer of the Children’s Tumor Foundation, shares how they are taking a non-traditional approach to expedite finding treatments and a cure. At Beyond Batten Disease Foundation, we share many of these same beliefs and this is why we can envision wholeheartedly a world beyond Batten.
Uprooting the Foundation: Innovation and Collaboration are the Only Way to Success
A lot of phrases—some positive, some negative—are being bandied about these days as those of us who work in the nonprofit medical research sector seek to actualize our missions. Well-meaning commentators needlessly snipe back and forth while espousing their analyses of the pros and cons of either the traditional nonprofit model or those more aligned with a business-oriented approach.
Often, the heart of the matter is lost during this war of words. Medical research foundations, especially those specializing in rare disease research, are focused on ensuring that safe and effective treatments and cures reach those living with the disease. These patients and their families seek answers to their problems now—not 20 years from now, but today.
The steps a foundation takes to achieve its goals are likely to be as individualized as the diseases and disorders each confronts. Rare diseases and disorders, for example, are at a disadvantage when it comes to receiving high levels of federal funding or strong interest from the pharmaceutical industry. With a perception of less public demand, it is all too easy for research into rare diseases to become a low priority. A low priority to everyone, that is, except those who are affected.
At Children’s Tumor Foundation, we work to find cures for the approximately one in 3,000 people living with neurofibromatosis (NF), a title that encompasses three distinct genetic disorders (NF1, NF2 and schwannomatosis). NF can cause tumors to grow along various types of nerves and, in addition, can affect the development of non-nervous tissues such as bones and skin. It can cause tumors to grow on or in the body, and it manifests itself differently in every single patient—from blindness in one, to deafness in another, to intense pain or even possibly cancer.
We have realized that the traditional funding model—one that rather passively hands out grants to promising scientists—simply was not resulting in expedient effective treatments. Too many institutional barriers were in place, too many limitations existed, and we simply could not sit around hoping someone else would tear those barriers down. We turned our funding model inside-out in order to build new mechanisms, create strategic partnerships, and foster a spirit of collaboration between the many different stakeholders inside and even outside the NF landscape: patients, researchers, clinicians, pharmaceutical companies and the biotechnology sector. And while we continue to provide grants to promising scientists for interesting research, we have rebuilt our foundation to realize optimal results. All of these choices were made with one goal in mind: to not merely activate the drug development process, but to set in motion a process that promises to get treatments (and one day a cure) into the hands of our patients.
What does this look like? It looks like tearing down barriers for all of our stakeholders in order to make the search for treatments faster, easier and more effective. We are inviting people from all sectors to collaborate, to share resources and to work together rather than separately find solutions. We are doing whatever it takes to facilitate cross-institutional data sharing. This means providing open access to animal and cellular models, and creating a tissue biobank with open access for all scientists who agree to share their research with others. (This open-source model, while it may sound just like common sense, is quite revolutionary in our world of litigious and overprotective entities.) It involves building and growing a patient registry, an anonymized patient-entered database that can be used to match willing patients to clinical trials. We now offer full drug research and development services, compound scouting services, and even the support of a patent attorney for those who need it. We simply cannot hear excuses anymore.
We have also redefined the very landscape of NF research by enhancing collaboration in a deeply meaningful way, by building teams of multidisciplinary scientists from world-class labs into consortia that we call synodos. These multiyear, multimillion-dollar collaborations are redefining the way research is done. In fact, we chose the word synodos because it is derived from the ancient Greek, and it means “to be on the same path together.” And this is what the NF synodos team is about—not just about competition, but about attracting courageous researchers across disease disciplines who are willing to share their unpublished data and information with others, in order to accelerate the drug discovery process.
In doing this, we envision a future where the roadblocks to drug discovery are removed. First and foremost, a world where patients are properly diagnosed and receive good treatment options. A world where industry is open to clinical trials for even smaller disease areas, but people have access to the tools they need to make their work efficient. And a world where researchers don’t feel restrained by contracts and publishing deadlines, and are willing to share their discoveries with others, early and often.
Let’s face it: Effective medical research nonprofits must continuously strive for innovation. We must constantly review methodologies and find areas for improvement—just like any nonprofit. Above all, in our specific field, we must find ways to put treatments into the hands of those who need them. Commentators can call it what they want; we call it effective drug development success.
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