Developing pediatric drugs for clinical trials represents a challenging undertaking. Most commercial drug developers steer clear of pediatric research due to business risk, not to mention an aversion to rare disease if the commercial upside isn’t robust enough. Academic medical researchers often don’t have sufficient infrastructure or capital to commercialize pediatric therapies for rare diseases. Enter the “scientific altruists” movement and SPARK, a Stanford University-based nonprofit organization.
A Compelling Story
Recently Belatina covered the story of Teresa Purzner and her husband, both neurosurgeon residents at the University of Toronto. On a mission to develop treatments to help children with medulloblastoma, they stepped aside to focus on research. They found the collective of altruistic scientists at SPARK.
What is SPARK?
SPARK offers primary funding for research projects and connects academic researchers to research professionals in the industry to develop solutions that are driven by patient demand, not necessarily by market forces. That is because SPARK is inspired by “scientific altruism,” which acknowledges a society’s basic need for medical solutions for pediatric conditions and rare illnesses, regardless of whether a profit can be produced.
SPARK bridges the gap between discovery and drug treatment through a unique partnership between the university and industry experts. With modest support of $50K/year per project and comprehensive weekly coaching by over 100 volunteer experts from industry, SPARK has achieved unprecedented success in translating early academic discoveries to solutions addressing unmet needs.
The program educates many hundreds of influential scientists and researchers on the translational process. A majority of focus for SPARK centers on neglected areas of child and maternal health, global health, and orphan diseases. Excitingly, the SPARK model is spreading and is now embraced by over 60 academic institutions worldwide.
A unique partnership between altruistic-driven researchers in universities and the community and industry experts, it was founded by Chemical and Systems Biology Professor Daria Mochly-Rosen in a quest to generate proof of concepts using out-of-the-box academic approaches combined with industry standards.
It is based on five principles, including 1) multiple opinions, 2) no need for consensus, 3) open exchange and no hierarchy 4) on university campus activity, and ongoing two-year programs.
From graduate level courses centering on drug development processes to a program for SPARK Scholars —opening up funding opportunities for researchers such as Teresa Purzner to mentoring services from industry insiders, new research endeavors including product proposals are reviewed by a panel of experts ongoing. SPARK Scholars (AKA “SPARKees”) are funded for an average of two years and participate in weekly seminars with industry and academic experts. SPARK offers participation to professors, clinicians, postdocs scholars, and graduate students. The network has spread worldwide.
A SPARK Success Story: Medulloblastoma Therapy to Clinical Trials
Teresa Purzner received FDA approval this past summer for her team to run clinical trials on a drug that her group believes will cure medulloblastoma in children, despite what appeared to be at time overwhelming challenges. In what can typically take a billion dollars and a decade of research, utilizing SPARK Ms. Purzner was able to accomplish in 5 years and half a million dollars thanks to SPARK. She noted recently for The Tot “Getting a drug into a pediatric clinical trial is extremely difficult and there were many movements in the years that it took to do it where the entire effort should have failed.” Ultimately having three children herself and the empathy and understanding of what parents go through when their children are afflicted with rare cancers represented a powerful altruistic-driven force propelling her forward.
SPARK has been instrumental in the process of helping this prospective medulloblastoma investigational treatment get over the ‘valley of death’—that is the gap between scientific discoveries and clinical trials. Most therapies that don’t have a commercial driver don’t make it.