Impatient Patients: How Disease Organizations Are Accelerating Research

Panel Overview & Organizational Models

• Faster Cures Mission: A Milken Institute center based in Washington, D.C., operating for 10 years to accelerate the medical research system under the tagline "Time Equals Lives."
• Venture Philanthropy Defined: Characterized as "philanthropy with an opinion," this model involves targeted investment to solve specific problems rather than passive funding, aiming to eventually make the organization obsolete by achieving cures.
• Funding Scope: Disease-focused organizations represent a small fraction of total biomedical research funding but have driven disproportionate change through intense focus on organizational models and goals.
• Strategic Alignment: Panelists emphasized that success requires alignment across academia, biotech, pharma, the FDA, payers, and patients, rather than simply increasing capital.

Leukemia & Lymphoma Society (LLS)

• Leadership: Lou DiGennaro, Executive Vice President and Chief Mission Officer, leads an organization founded 65 years ago by parents who lost a son to leukemia.
• Disease Scope: Addresses ~160,000 new North American diagnoses annually for blood cancers (leukemia, lymphoma, myeloma), with ~140,000 annual deaths.
• Grant Portfolio: Funds ~90 new projects annually out of 1,000 applications; currently holds a portfolio of 350 projects with an $60 million annual investment.
• Therapy Acceleration Program: Created to harvest projects moving from basic research to product development (e.g., IND-enabling studies) that struggle to secure funding in academic settings.
• Biotech Partnerships: Deploys resources to small biotech companies with therapies stalled for economic reasons due to small patient populations.
• Financial Leverage: $17 million in LLS resources for small companies triggered $1.7 billion in potential out-year milestones, representing a 100x leverage on investment and a 30x cash return via acquisitions and licensing deals.
• Repurposing Success: Successfully repurposed an FDA-approved topical antifungal ingredient for leukemia treatment.

Prostate Cancer Foundation (PCF)

• Urgency Driver: Founded by Michael Milken after observing that prostate cancer death rates were projected to be 40% higher than current levels; currently the second most common cause of cancer death in men.
• Patient Activism Case Study: John Willey, a prostate cancer patient, participated in seven clinical trials, four remissions, and a posthumous rapid autopsy, providing genomic data on drug resistance.
• Scientific Insight: Whole genome sequencing of patients like Willey revealed mechanisms for resistance to drugs like abiraterone and hormonal therapy, contributing to broader cancer biology understanding.
• Cross-Disease Impact: Shared biological drivers identified in prostate cancer (e.g., bone growth) are relevant to leukemia, demonstrating the ripple effects of patient-driven research.
• Social Media Dynamics: Relies heavily on organic growth of communities, primarily driven by women (caregivers) on Facebook for a demographic mostly aged 60–70.
• Resource Constraint: PCF has invested minimally in social media infrastructure due to a priority on funding postdocs and young investigators, despite recognizing social media's power in trial accrual and advocacy.
• Public Policy: Urges nonprofits to lead in activating patient populations for clinical trials, citing government inaction despite the availability of social media tools.

Foundation for Fighting Blindness

• Leadership: Co-founded by Gordon Gunn, a retinal patient diagnosed in 1970, focusing on retinitis pigmentosa, Usher's disease, and age-related macular degeneration (AMD).
• Market Scope: Addresses diseases affecting 10+ million US residents, with AMD being the leading cause of blindness for those over 55.
• Funding Strategy: Raised over $550 million in 42 years to build a knowledge base and support early-stage investigators, leveraging NIH funding once traction is proven.
• Translational Capability: Established internal drug development capabilities to navigate the "Valley of Death" between laboratory breakthroughs and clinical delivery.
• Commercial Success: Funded the development of Second Sight's Argus 2 ("bionic retina"), an FDA-approved implant providing vision to the blind.
• Gene Therapy Milestone: Supported gene replacement therapy for Leber's congenital amaurosis, restoring significant vision in over 50 patients blind from birth.
• Event Innovation: Utilizes "Dining in the Dark" fundraising dinners to create immersive empathy regarding vision loss.

Melanoma Research Alliance (MRA)

• Leadership: Co-founded by Deborah Black, a melanoma survivor, with 100% of expenses covered by the founders to direct all raised funds to research.
• Public Health Goal: Aims to reduce mortality (one US death per hour) through early detection education, countering the misconception that melanoma is easily treatable at late stages.
• Targeted Demographics: Prioritizes outreach to underserved populations, including people of color, to combat the myth that darker skin cannot contract melanoma.
• Strategic Partnerships: Launched a campaign with L'Oreal where 100% of profits from a specific SPF product go to research; collaborated with Stand Up to Cancer for celebrity advocacy.
• Fundraising Campaign: Initiated a text-to-donate campaign ("Text Danny 50555") honoring Danny Federici of the E Street Band, raising funds for research at Sloan Kettering.
• Drug Development Impact: Funded anti-PD-1 research with robust results in lung cancer, demonstrating the cross-pollination of melanoma-funded therapies to other disease areas.
• Policy Advocacy: Urges Congress to end federal sequestration to protect research funding and young investigators.

Alpha-1 Foundation

• Leadership: John Walsh, a patient with Alpha-1 antitrypsin deficiency, co-founded the organization after the NIH ceased funding a longitudinal study in 1994.
• Disease Impact: Affects 3–5 million people globally; causes early-onset emphysema (ages 35–45) and liver failure, serving as a major reason for adult lung and infant liver transplants.
• AlphaNet Program: A non-profit health management company providing peer coaches and 24/7 support, achieving a 98% adherence rate to weekly IV augmentation therapy.
• Research Revenue: Generated over $35 million in recurring revenue via AlphaNet to fund $47 million in research grants at 96 institutions across North America and Europe.
• Clinical Trial Infrastructure: Maintains the world's largest registry, enabling recruitment for pivotal Phase III trials in 6–8 weeks despite a small patient population (~7,000 diagnosed).
• AlphaOne Project: Created a wholly-owned for-profit subsidiary to invest in biotech advancement, including a $350,000 grant to AGCT that helped secure $37 million in venture capital for gene therapy trials.
• Cultural Shift: Embedded a culture of clinical research participation within the patient community to secure treatments for future generations.

Key Trends & Consensus

• De-risking Science: Organizations are actively intervening to move high-value projects from basic research through the "Valley of Death" into clinical trials, often filling gaps where academia and big pharma hesitate.
• Patient as Partner: Patients are increasingly viewed as essential partners in defining research questions, accelerating trials via registries, and providing data through rapid autopsies.
• Ecosystem Leverage: Successful models leverage volunteerism, small initial investments to trigger large external capital (30x to 100x leverage), and cross-sector collaborations (pharma, biotech, FDA).
• Policy as Critical Path: Panelists stressed that funding research is insufficient without concurrent advocacy to ensure FDA approval efficiency and payer reimbursement.
• Open Source Collaboration: Organizations within Faster Cures share best practices, data, and infrastructure (via the TRAIN network) to prevent redundant work and accelerate collective progress.
• Democratization of Health: Social media and mobile technology are enabling patients to bypass traditional information barriers, driving trial accrual and public awareness.