Conference Presentation, Panel
Impatient Patients: How Disease Organizations Are Accelerating Research
Milken InstituteMargaret Anderson, Lou DiGennaro, Jonathan Simons, Gordon Gunn, Deborah Black, John Walsh
- Faster Cures aims to accelerate research by analyzing cross-disease ripple effects, with its member organizations expected to remain a source of optimism despite representing a small fraction of total biomedical funding.
- The Leukemia and Lymphoma Society seeks to eliminate blood cancer by leveraging resources for up to 100 times its investment based on out-year milestones and supporting small biotech firms in accessing capital markets or partnering with large pharmaceutical companies.
- The Prostate Cancer Foundation projects a historical death rate 40% higher than current levels and plans to drive patient-partnered science into the public domain, anticipating that shared biology will allow one cancer's experience to benefit others.
- PCF expects social media to dominate scientific agendas, plans to expand its social media presence to a new order of magnitude despite current budget constraints, and anticipates that new genetic understanding will soon enable primary care physicians to counsel high-risk young men on diet and lifestyle changes to avoid the disease.
- The Prostate Cancer Foundation believes nonprofits must fill the gap left by government policy in activating patient populations and that patient-generated data and trials will drive better science.
- The Foundation for Fighting Blindness projects age-related macular degeneration will become an epidemic affecting over 10 million people in the United States and anticipates commercial availability of the Argus 2 bionic retina following FDA approval.
- FFB expects a platform for gene therapy to emerge following the success of treating over 50 individuals with Leber's congenital amaurosis and plans to leverage National Eye Institute support through early-stage funding and research translation capabilities.
- The Melanoma Research Alliance cites a 75% increased risk of melanoma for regular tanning bed users and plans to utilize L'Oreal's "Sublime Sun" partnership to reach underserved people of color.
- MRA expects its anti-PD-1 drug funding to continue yielding results for lung cancer due to shared pathways and plans to fund research via a texting campaign, while urging stakeholders to collaborate to prevent government sequestration from harming research and early-career scientists.
- The Alpha One Foundation plans to recruit for a phase three pivotal clinical trial within six to eight weeks, a timeframe described as exceptional for the rare disease space, and expects its AGCT investment to help the company raise $37 million.
- AO1 intends to embed a culture of research participation within its community to benefit future generations affected by the familial disease.
- General strategic outlooks suggest research acceleration requires ecosystem alignment among academia, biotech, pharma, the FDA, payers, and patients, and that patient mobilization is critical for creating treatments and cures.
- Panelists anticipate that the TRAIN network will continue sharing best practices and infrastructure to prevent resource waste, while patient-generated ideas and incentives created by funding in previously stagnant fields like melanoma are expected to drive scientific progress.