Using Stem Cells to Cure Autism, Epilepsy & Schizophrenia | Dr. Sergiu Pașca

Overview

This episode explores groundbreaking advancements in stem cell research and their application to understanding and treating profound autism, epilepsy, schizophrenia, and other brain disorders. Dr. Sergiu Pașca, a pioneer in the development of organoids and assembloids, explains how these stem cell-derived tools are revolutionizing neuroscience. The conversation also delves into the ethical considerations of these technologies, the rise in autism diagnoses, and the future of personalized medicine.

Notable Quotes

- You think about psychiatric disorders as esoteric and complex, but we went all the way down to a molecular defect and are now on the verge of potentially reversing it. — Dr. Sergiu Pașca

- The brain builds itself. There’s no blueprint—it’s self-organizing, and that’s the power we’re leveraging with assembloids. — Dr. Sergiu Pașca

- Not all superheroes wear capes. You’re doing God’s work. — Andrew Huberman

🧠 Autism and Its Rising Prevalence

- Autism is a spectrum disorder defined by behavioral observations, with no definitive biomarkers.

- The prevalence has risen to nearly 3%, partly due to better diagnostic criteria and awareness.

- Genetic factors play a significant role, with twin studies showing high heritability. Environmental factors, such as prenatal exposures, may also contribute.

- Profound autism, the most severe form, often involves intellectual disability and epilepsy, requiring lifelong care.

🧬 Stem Cells, Organoids, and Assembloids

- Dr. Pașca’s lab uses induced pluripotent stem cells (iPSCs) to create organoids—3D clusters of brain cells—and assembloids, which mimic brain circuits.

- These tools allow researchers to model human brain development, study diseases, and test therapies without relying on animal models.

- Organoids replicate developmental timelines, such as the switch in NMDA receptor subunits at nine months, demonstrating their biological accuracy.

- Assembloids enable the study of complex neural circuits, such as sensory pathways and motor control loops.

🩺 Therapeutic Breakthroughs and Applications

- Timothy Syndrome, a rare genetic disorder linked to autism, has been a key focus. Dr. Pașca’s team developed a nucleic acid-based therapy that reverses cellular defects in patient-derived neurons.

- Assembloids are being used to study epilepsy, schizophrenia, and dystonia, offering insights into circuit-level dysfunctions.

- The potential for personalized medicine is immense, with therapies tailored to specific genetic mutations.

⚖️ Ethical Considerations in Stem Cell Research

- Ethical concerns include the use of human cells, animal transplantation, and the potential for emergent properties like sentience in organoids.

- Dr. Pașca emphasizes the importance of precise language, avoiding terms like mini-brains, which can mislead the public.

- Collaborative efforts among scientists have led to standardized nomenclature and ethical guidelines for the field.

🌍 The Future of Neuroscience and Medicine

- Advances in gene therapy and CRISPR hold promise for correcting genetic defects, but challenges remain in delivery and timing.

- The concept of rejuvenating or immortalizing tissues is being explored, though it raises complex technical and ethical questions.

- Dr. Pașca’s work exemplifies the shift toward human-relevant models in neuroscience, paving the way for transformative treatments.