Podcast Summary
In this podcast, Dr. Nicole Paul, a professor of viral gene therapy at UCSF, discusses the evolution of medicine and the potential of gene therapy. She explains how viruses can be used as tools to deliver medicines and treat genetic disorders. The podcast also covers the challenges and potential applications of gene therapy, including its use in treating cancer, sleep disorders, and promoting longevity.
Key Takeaways
The Evolution of Medicine
- Chemical to Living Medicines: Dr. Paul outlines the evolution of medicine from chemical medicines, where compounds from natural sources were synthesized and mass-produced, to protein medicines, and now to living medicines. Living medicines use viruses, bacteria, and cells to impart changes in the body and can be engineered to perform precise drug delivery.
- Viruses as Tools: Viruses are not inherently bad and can be used as tools to deliver various medicines. They can be used in gene therapy to provide a functional copy of a missing or mutated gene, treating single gene genetic disorders.
Gene Therapy in Practice
- Gene Therapy for Rare Genetic Disorders: Dr. Paul’s lab and company are working on a project to use gene therapy to treat cancer, blending it with immunotherapy. They aim to create a universal gene therapy that can be used to treat millions of patients across different indications.
- Immunotherapy and Cancer: Immunotherapy aims to retrain the immune system to recognize and destroy tumors that have developed mutations making them invisible to the immune system. Dr. Paul’s company has developed a platform for universal gene therapies that can deliver cancer-announcing payloads to activate the immune system.
Future Applications and Challenges
- Future Applications: Dr. Paul discusses potential future applications of gene therapy, such as treating sleep disorders, promoting longevity by regenerating tissue in joints, and even enhancing human potential by altering skin color to withstand harsh environments or engineering gut bacteria to metabolize food in a Martian environment.
- Regulatory and Ethical Challenges: The regulatory hurdles for gene therapy development are significant, with a lengthy timeline for clinical trials and extensive paperwork required for FDA approval. There are also ethical considerations surrounding enhancements and augmentations that are not related to diseases.
Sentiment Analysis
- Bullish: Dr. Paul is optimistic about the potential of gene therapy to treat a wide range of diseases and conditions. She highlights the progress made in the field and the promising results from early trials.
- Bearish: Despite the optimism, Dr. Paul acknowledges the significant challenges facing gene therapy, including regulatory hurdles, ethical considerations, and the high cost of research and development.
- Neutral: While discussing the potential future applications of gene therapy, Dr. Paul maintains a balanced view, acknowledging that these applications are still in the development stage and may be several years away from being ready for use.
