Activist Patient Foundations, Like the Children’s Tumor Foundation, Help Drive the Search for New Drugs

Patient groups have long been passionate advocates for their constituencies and many have become active investors in their sectors, spurring the drug discovery process with grants and other financial backing behind promising research. Increasingly, some foundations and associations, particularly in the rare disease space, are taking further steps, becoming significant drivers of the search for effective therapies.

The current work of the Children’s Tumor Foundation (CTF), for example, was born in part by frustration with the traditional, competitive drug development model where often, as Annette Bakker, PhD, the foundation’s president and chief scientific officer, observed, neither industry nor academia seemed quite suited to get the job done, while patients anxiously wait.

Bakker, a veteran of both pharma (six years at Janssen Pharmaceutica in Belgium) and biopharma (more than seven years as head of oncology research and development for Siena Biotech of Siena, Italy), said: “In academia, people’s careers depend on publications so what they need to do is publish as quickly as possible, to be able to get access to their funding. On the industry side, secrecy is needed to stay competitive – so the incentive is to keep discoveries secret for as long as possible” in order not to tip off competitors.” Bakker added that what she sought instead was “a kind of Switzerland situation. That’s why I left both academia and industry.”

At the Children’s Tumor Foundation, “we don’t need patents or publications,” Bakker said. “The only thing we urgently need is a creative business model that gets the fantastic academic discoveries as quickly as possibly translated into treatments for patients – from the research lab into the patient.”

The foundation has developed a multi-pronged strategy to help facilitate that goal, which circumvents the barriers within the traditional model of drug discovery and encourages instead a culture of collaboration to accelerate the cure for neurofibromatosis (NF), a set of genetic disorders that causes tumors to grow along the nerves. “In the rare disease space, we really don’t have the luxury to compete” says Bakker. The foundation’s “roadmap” to end NF in its three main forms – NF1, NF2 and schwannomatosis — includes the active engagement of all stakeholders in the cure: patients, patient advocates, researchers, physicians and the biopharma industry.

Two important planks to these efforts include the foundation’s collaborative research model, called Synodos, and the development of an NF tissue bank:

Synodos – This is the shared-science infrastructure developed by the foundation where all specialists – researchers,  patients and their physicians, – share their raw data in real time, and CTF’s partner Sage Bionetworks guarantees the full data integration. This all results in what is expected to be a faster and more efficient translation of top findings to clinical benefits. Synodos includes an incentive system that guarantees funding for scientists and clinicians involved.

Synodos for NF2 was established first, in 2014, with principal investigators from eight institutions involved. Synodos for NF1 followed, launching in 2015, with an expanded roster of 24 investigators from eight institutions and two companies. The foundation is starting for schwannomatosis in spring of this year.

The CTF Biobank – The foundation has developed a centralized, open access repository where patient tissue, such as blood and tumor samples, is stored for future use. The bank was born from a recognition that there were not enough high-quality NF tissue samples openly available, as the few institutions that had properly collected and stored samples were reluctant to share them. Protocols have been established so that patients with all NF types can also donate their bodies to science. The bank provides access to this “library” of samples, the foundation says, for biomarker discovery and development and other aspects of drug research.

Meanwhile, the foundation, from its home base in New York, continues with other critical roles, such as the creation and management of a patient registry, which has reached 7,500 participants. Its information has been used to connect patients, doctors and investigators, facilitating 18 clinical trials thus far.

Through 2016, CTF’s NF preclinical consortium has resulted in 115 preclinical studies which progressed to 16 clinical trials, including AstraZeneca’s MEK inhibitor, which shows encouraging results in shrinking tumors in patients. CTF’s support has contributed to more than 60 papers in peer-reviewed scientific journals. The $26.6M in investments made by the foundation in the five years from 2010 to 2015 attracted $38.4M in follow-up funding from other sources.

As the foundation continues to make progress towards its goal of ending NF, Clarivate Analytics has become a close partner to CTF, providing cutting-edge products and services, such as the pathway analytics tool Metacore, which have supported CTF in its vision. Since the foundation has moved into a more active role, the partnership with Clarivate has developed further, facilitating the foundation’s access to the knowledge required to be a resourceful contributor to the NF research community.

On Tuesday, April 11, the Children’s Tumor Foundation and Clarivate will host a webinar during which Annette Bakker and members of her team will speak about their pioneering work. They will also participate in a live Q&A session.

Join us to learn more about the foundation’s strategy and work.