Inside the ALS Breakthrough: 20-Year Journey to FDA Approval with Dr. Joseph Palumbo

🧠 From Inflammation to Innovation: Dr. Joseph Palumbo on Systems Neuroscience and the Future of Brain Medicine

🎙️ Guest: Dr. Joseph Palumbo, Executive Vice President , Chief Medical Officer, and Head of R&D, BioVie Pharma

“It’s never been a more exciting time to be a neuroscientist.” – Dr. Joseph Palumbo

👋 Episode Overview

In this episode, Brandon Li sits down with Dr. Joseph Palumbo, psychiatrist, neuroscientist, and biotech leader whose career spans academic medicine, Big Pharma, and entrepreneurial science. From leading global neuroscience at Janssen to pioneering new treatments for ALS and neuroinflammation at BioVie Pharma, Dr. Palumbo shares what he’s learned about translating brain science into real-world therapies.

They explore how inflammation connects brain and body, why systems biology is the next frontier in mental health, and what it takes to bring a drug from hypothesis to approval. With reflections from a career spanning Yale, J&J, Mitsubishi Tanabe, and BioVie, this episode is a masterclass in neuroscience translation—and a window into the future of psychiatry as integrated brain medicine.

🧭 Top Takeaways

✅ The next era of neuroscience is integrative. Advances in immunology, metabolism, and systems biology are reshaping how we understand the brain.
✅ Inflammation is a common denominator—linking disorders from depression to Parkinson’s and Alzheimer’s.
✅ Smaller biotechs must be nimble. Success comes from focus, collaboration, and clarity—“delivering to the next decision point.”
✅ Clinical relevance beats statistical significance. It’s not enough for a drug to be significant—it must make a meaningful difference for patients.
✅ Physician-scientists must think like engineers. The future belongs to clinicians who understand circuits, systems, and computational biology.

🔑 Key Topics Covered

Guest Background

• Trained in psychiatry at Yale after medical school at George Washington University.
• Early academic roles at Yale, Cornell, and the University of Pennsylvania.
• Industry leadership at Cephalon, Janssen (J&J), and Mitsubishi Tanabe, where he helped bring the ALS drug edaravone to global approval.
• Currently CMO at BioVie Pharma, advancing small molecules that modulate neuroinflammation.

The Problem He’s Solving

• Inflammation as a driver of brain dysfunction. Psychiatric and neurological disorders are deeply intertwined through inflammatory pathways.
• Gaps in translation. Too many promising discoveries fail between animal models and human application.
• The need for simplicity in clinical trials. Overly complex designs bury real therapeutic signals.

His Innovation or Contribution

• Advocating for systems neuroscience—bridging psychiatry, neurology, and internal medicine.
• Applying inflammation modulation to conditions like Parkinson’s, Alzheimer’s, and long COVID.
• Integrating clinical insight with molecular science—using patient function and quality of life, not just p-values, as endpoints.

Challenges & Barriers

• Translating preclinical success into human outcomes remains unpredictable.
• Balancing investor urgency with scientific diligence requires clear milestones.
• Measuring true brain change—via biomarkers like DNA methylation—is still in early stages.

Vision for the Future

• Neuroscience will merge with internal medicine. What’s good for your heart is good for your brain.
• Inflammation will become a core treatment target across psychiatry and neurology.
• AI and data science will redefine diagnostics and help track functional improvement.
• Next-generation clinicians will be systems thinkers—combining bedside empathy with computational literacy.

💬 Speaker Spotlights

On biotech vs. Big Pharma:

“In a small company, you have to be fast and accurate—like flying a drone instead of steering an aircraft carrier.”

On ALS drug development:

“We didn’t want to create false hope. We wanted to be certain that what we saw scientifically and clinically was real.”

On trial design:

“Go as fast as you can—and no faster. Development is about reaching your next decision point, not promising everything at once.”

On inflammation as a unifying mechanism:

“If you block inflammation, you can treat depression. You can slow Parkinson’s. Maybe even alter aging itself.”

On training the next generation:

“Be a clinician first. Then learn systems biology. The best physician-scientists ask how things work before they ask why.”

💡 Keep Exploring

🎧 Listen to the full episode on:
👉 Spotify
👉 YouTube

Subscribe to Power to the Patients for more conversations at the intersection of neuroscience, innovation, and patient impact.

Learn more about Dr. Joseph Palumbo and his work advancing neuroinflammatory therapeutics at BioVie Pharma and beyond.

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