Conference Presentation, Panel
Sustaining the New 'Golden Age' of Medical Research
Milken InstituteMargaret Anderson, Francis Collins, Kathy Giusti, Michael Milken, Kevin Yoder, Elias Zerhouni
- The 19th annual Milken Institute Global Conference features over 200 sessions and more than 750 speakers, including this opening panel on "Sustaining the New Golden Age of Medical Research."
- The panel is organized by Faster Cures, a Milken Institute center founded 13 years ago to increase efficiency in the biomedical research system and accelerate the path to cures.
- Kathy Hudson, diagnosed with multiple myeloma in 1996, founded the Multiple Myeloma Research Foundation (MMRF) after being told she had less than three years to live.
- Nearly 20 years after Hudson's diagnosis, multiple myeloma treatment has seen 10 drugs approved in the last year alone, including two antibody therapies, effectively tripling patient lifespans.
- Hudson's MMRF strategy involved building a coordinated ecosystem of scientists, pharmaceutical companies, biotech firms, and payers to drive precision medicine faster than other disease areas.
- Elias A. Zerhouni, former NIH Director and mathematician/physicist, noted that the U.S. was the only country in the mid-1970s capable of advanced computerized imaging research.
- Zerhouni warns that current NIH funding levels mean young investigators will not receive their first grant until age 42, threatening the pipeline since 95% of scientists make their contributions by that age.
- Congressman Tom Yoder argues that the 600,000 annual cancer deaths should be treated with the same urgency as a terrorist attack, framing biomedical research as a national security priority.
- Yoder contrasts the $700 billion annual U.S. defense budget with the roughly $30 billion spent on all disease cures, arguing that the human cost of inaction far exceeds the defense budget's scope.
- Yoder proposes a fiscal argument against short-term deficit concerns, citing a projected $1 trillion annual cost for Alzheimer's disease by 2050, suggesting early investment could prevent future economic devastation.
- Francis Collins, current NIH Director, highlighted that while NIH funding doubled from 1998–2003, it has remained stagnant in real terms since 2003, with a significant drop due to the 2013 sequestration.
- Collins noted a 7% ($2 billion) budget increase in the current fiscal year but emphasized the lack of long-term budgetary certainty hinders multi-year research planning.
- Collins stated that the current grant success rate at NIH is approximately 16%, the lowest in history, effectively scaring away talented young researchers.
- Mike Milken, diagnosed with prostate cancer in 1993 with a 12-to-18-month life expectancy, attributes the survival of over 16 million Americans to research-driven advancements.
- Milken asserts that medical research and public health accounted for more than 50% of global economic growth over the last 200 years and that life expectancy increased by 41 years in the last 115 years.
- Milken highlighted a technological leap where modern machines can perform 5 million times more work in genetic research than researchers could in 1993.
- Milken criticized the current system for taking its foot off the accelerator despite a $300 billion incremental investment in technology, arguing the field is in the "last quarter" rather than the "seventh inning."
- The Precision Medicine Initiative (PMI) aims to enroll 1 million volunteers by 2019 to create a longitudinal, data-rich cohort including genomic sequences and environmental sensor data.
- Eric Dishman, a cancer survivor and former Intel executive, was hired to lead the PMI, bringing an informatics-focused, fast-moving approach to the initiative.
- Elias Zerhouni advocates for "parallel processing" in biomedical research to replace the current linear, siloed model, arguing that simultaneous data sharing and collaboration can double productivity.
- The Accelerating Medicine Partnerships (AMP) were launched by the NIH to create pre-competitive spaces where industry and academia share data to validate biomarkers, aiming to reduce the 95% drug failure rate.
- The Cancer Moonshot initiative has established a blue-ribbon panel of 28 experts to define scientific goals, focusing on overcoming challenges in immunotherapy and moving from monotherapies to combination trials.
- Hudson reported that the MMRF successfully de-risked the multiple myeloma sector, transforming it from an obscure diagnosis into a multi-billion dollar market.
- Hudson is partnering with Harvard Business School to convene small, transparent groups of business, science, and technology leaders to solve data-sharing and business model issues.
- The MATCH trial, a pan-cancer study using genomic analysis to assign patients to targeted therapies, exceeded all previous enrollment expectations, forcing organizers to pause due to high volume.
- Clinical trial participation remains low, with less than 1.5% of Parkinson's patients and only 5-6% of cancer patients participating, creating a major bottleneck for validation.
- Hudson emphasized that patients must be viewed as partners rather than "human subjects," noting that current models of consent and data engagement are outdated.
- Collins stated that NIH will mandate open data access as a condition for grants, asserting that "we can't herd the cats, but we can move their food."
- Zerhouni identified the lack of understanding of human disease biology in living humans as a primary bottleneck, which the PMI aims to address through large-scale data collection.
- Collins noted that the FDA is now more willing to engage early in trial design for combination therapies and phase 2 approvals for high unmet medical needs.
- The panelists agreed that social media and celebrity advocacy (e.g., Ice Bucket Challenge) are critical for creating the social movement necessary to increase patient participation in research.
- Congressman Yoder called for a national conversation where political leaders and presidential candidates prioritize biomedical research as a top-tier national issue.
- Milken argued that AI and machine learning, when fed sufficient data, will soon dictate medical interventions, but this requires the creation of open, global data repositories.
- Hudson highlighted the "business leader" gap in academia and science, noting that many researchers lack the operational experience to commercialize or rapidly scale discoveries.
- The panel concluded with a consensus that while technology has outpaced science, the integration of AI, open data, and parallel processing can accelerate cures if systemic barriers are removed.