Panel, Conference Presentation
Editing the Genome: Promise and Peril
Milken InstituteJonathan Thomas, David Agus, David Baltimore, Amander Clark, Hank Greely, John Thomas
- Technological capabilities are projected to accelerate rapidly and become more controllable, necessitating that legal, ethical, and moral systems adapt within significantly less than 15 years per breakthrough to address unprecedented pace.
- Current medical practices are shifting toward sequencing cancer prior to treatment and utilizing somatic CRISPR-Cas9 for precision medicine, with complex, multi-gene disease applications clinically expected to be decades away.
- Gene editing for non-disease human enhancement, such as creating taller or smarter children, is anticipated to emerge, likely generating prolonged controversy compared to the relative acceptability of fixing diseases.
- Basic research leading to safe and effective human germline editing is projected to become possible within a five to 10-year timeframe, though immediate clinical trials for edited embryos face substantial constraints due to congressional riders.
- Regulatory frameworks for human germline editing rely on international moral force rather than binding law, as no single global body possesses the authority to enforce norms across different jurisdictions.
- The deployment of gene editing in non-human applications is expected to grow significantly, including agricultural improvements to prevent crop rot and pest resistance, and vector control efforts targeting mosquitoes for diseases like Zika, malaria, and dengue.
- Risks associated with gene editing include the potential for biological terrorism, such as converting benign pathogens into pathogenic forms, and the inability to fully control complex emergent systems, which may require evaluating outcomes 50 to 70 years in advance.
- A "flat" global enforcement environment allows individuals with minimal resources, such as $1,000 and a garage, to conduct gene editing experiments, raising concerns that regulatory gaps could lead to unintended consequences in developing nations.
- Congressional legislation, specifically riders in the Budget Reconciliation Act, prevents the FDA from receiving or approving clinical trial applications for edited embryos, creating a substantial constraint on research if the policy persists.
- The field of precision medicine aims to deliver the right drug to the right patient at the right time by identifying predisposition genes and signaling disruptions, though achieving fine-grained precision requires continued support for basic science.
- Public discourse and leadership are deemed essential for navigating ethical challenges, with calls for public education and normative behavior changes to prevent regulatory overreach driven by panic following catastrophic events.