a16z Podcast: "America's Autism Crisis and How AI Can Fix Science with NIH Director Jay Bhattacharya"

Date: September 23, 2025
Guests: Dr. Jay Bhattacharya (NIH Director), Vanita Agarwala (a16z Health/Bio General Partner), Jorge Conde (a16z Health/Bio General Partner), other a16z contributors
Main Theme:
A deep dive into reforms at the NIH under Dr. Jay Bhattacharya, with a focus on America’s autism crisis, recent scientific initiatives, the replication crisis, public trust in science, balancing funding, and the transformative role of AI in biomedical research.

Executive Overview

In this episode, Dr. Jay Bhattacharya describes sweeping efforts at the NIH to address America’s autism crisis and restore trust and rigor to American science. He highlights a $50 million autism initiative, bold new drug studies (leucovorin, Tylenol caution), and systemic reforms to how science is funded, communicated, and made reproducible. The discussion delves into the challenges of the replication crisis, the conservative culture of research funding, and the vital role of public trust—especially post-pandemic. The future promise of artificial intelligence in drug discovery, clinical work, and the daily workflow of scientists is explored in detail. Throughout, the hosts and Dr. Bhattacharya keep an optimistic, practical, and self-critical tone, inviting a more entrepreneurial, risk-tolerant approach to making American biomedical research bolder and more accountable.

Key Discussion Points & Insights

1. Major NIH Autism & Other Announcements [01:16–05:01]

• $50M NIH Autism Initiative:

  • Spurred by Secretary Kennedy’s challenge to address the rising prevalence of autism (now 1 in 31 children per CDC).
  • Initiative funds 13 teams (selected from 250 applications) to accelerate autism data science.

• Leucovorin for Autism:

  • An old drug (folinic acid) shows promise in restoring speech in 20% and improving symptoms in up to 60% of autistic children with specific folate processing deficiencies.
  • Plans for wider access being coordinated with CMS, FDA, and other agencies.

• Tylenol (Acetaminophen) in Pregnancy:

  • New research links prenatal use to increased autism risk; still controversial, but NIH/FDA will revise guidelines to encourage more caution.
  • Dr. Jay Bhattacharya: "Just be careful. ... Use it only really when you really need it for high fevers. Just to think prudently about it. I don’t want to panic anybody." [03:38]

• Cross-agency coordination ensures reimbursement and updated guidance for both leucovorin and Tylenol.

• NIH preterm birth initiative also announced, responding to worse US outcomes compared to Europe.

2. The Replication Crisis & Scientific Rigor [05:01–09:04]

• US public health outcomes (including life expectancy) have stagnated despite investment—underscoring the need for excellent, reproducible science.
• Dr. Jay Bhattacharya underscores lack of incentives for replication:
  • "The standard for truth in science ought to be replication—independent teams. ... Publication standards are not high enough." [05:01–08:36]
• The volume and specialization of modern science have eroded communal checking among scientists.

3. The NIH’s Cultural and Structural Reforms [09:04–15:31]

• Auditing Foreign Collaborations: New systems to ensure NIH funds sent abroad (e.g., Wuhan Lab) are trackable and auditable.
• Centralizing Peer Review: The Center for Scientific Review is now the norm, improving fairness and transparency.
• Silicon Valley Inspiration:
  • Science funding should embrace risk, tolerate productive failure, and invest in bold, out-there ideas. Dr. Jay Bhattacharya credits Silicon Valley's willingness to fund big, risky bets—even if most fail—for driving breakthrough innovation:

    "We should stop punishing scientists who fail. If they fail productively, let them publish in a journal to explain what they learned from it. Like that Silicon Valley spirit, I think, needs to come to science a little bit more." [00:00, 09:04]

4. Decision-Making at NIH: Allocation and Execution [15:31–21:50]

• Allocation: How much funding for each disease area? Driven by both political (Congress/president) and scientific priorities.
• Execution: Within each area, funders must select, monitor, and incentivize the right projects and scientists.
• Dr. Bhattacharya highlights the need for input from both experts and the public, citing the HIV movement as an example of the political necessity for certain research focuses.

"Things that we focus on should reflect the real needs of the people that fund us. If we’re just doing science for science’s sake and we’re just wandering around without producing answers ... why should they fund us?" [15:43]

5. Overcoming Conservatism in Funding and Supporting Young Scientists [24:11–32:59]

• The age of first large NIH grant has crept up from 35 (1980s) to mid-40s.
• Older, established researchers often dominate panels and funding, stifling new ideas.
• Dr. Bhattacharya is shifting evaluation standards, empowering Institute Directors to diversify portfolios, and rewarding mentorship and support for early-career investigators.

"If you want the newest ideas, you have to let the young people have a try. ... The ideas that we support are just, they’re just older." [25:59]

6. The Stalled Life Expectancy Problem—and Rethinking NIH Priorities [32:59–34:39]

• Despite advances (e.g., cancer survival), US life expectancy has not improved for over a decade—NIH must directly address the biggest practical health needs.
• Refocusing on chronic diseases, diabetes, heart disease, and rising conditions (autism, cancer, kidney failure) is a strategic imperative.

7. Academic Freedom and Scientific Publishing [34:39–37:22]

• Dr. Bhattacharya has abolished NIH internal publishing permissions, promoting freedom for its researchers.
• He criticizes the expensive, restrictive scientific publishing model and advocates for more open access.

"No more permission. If you’re an NIH researcher, you have a scientific paper, you don’t have to get permission from me." [35:35]

8. Restoring Trust in Science and Public Health [37:22–44:06]

• The pandemic deepened mistrust due to weak evidence behind public health mandates (plexiglass, masking rules, school closures).
• Restoring trust requires:
  • Gold-standard, reproducible science.
  • Institutional humility: admitting uncertainty openly and treating the public as partners, not inferiors.
  • Transparent, honest communication.

    "We … have to convey that uncertainty and we can’t blame the public. … The problem is that scientists conveyed certainty about things that they had no business conveying … during the pandemic." [41:28–44:06]

9. Applying AI to Healthcare and Scientific Discovery [47:52–53:08]

• AI has vast potential: protein folding (AlphaFold), drug development, streamlined workflows (medical records), radiology.
• There’s an emphasis on AI as augmenting scientists—not replacing them.
• Policies are being put in place to handle the flood of AI-generated grant submissions and avoid "a system of noise."

"AI is really good at summarizing existing knowledge … but really developing brand new ideas that challenge existing paradigms ... they’re not quite as good at that." [51:27]

10. Reflections and Inspirational Messages for Scientists [53:08–57:08]

• Dr. Bhattacharya urges scientists to persist with original ideas, citing the story of Max Perutz.
• He wants to reform NIH so that future Perutzes can survive, thrive, and revolutionize their fields.

"Science is incredible. ... It’s the individual scientist who believes in their idea, keeps knocking on the door even when the door is closed ... That’s who really makes a big difference in this world." [53:50]

11. The Future of Health: Portfolio Approach [55:28–57:08]

• When asked where the "next big gains" will come from—patient management, new drugs, or lifestyle—Dr. Bhattacharya’s answer is resounding: all of the above.
• Emphasis on maintaining a diversified "portfolio" of approaches to maximize the chance of breakthrough.

Notable Quotes & Memorable Moments

• On Scientific Failure and Productive Risk:

  "We should stop punishing scientists who fail. If they fail productively, let them publish in a journal to explain what they learned from it."
  — Dr. Jay Bhattacharya [00:00, 09:04]

• On Public Trust:

  "The American people are not stupid. ... When we talk to them ... with data, allow people to disagree, but then have the evidence right there ... I think people will respond with trust where the evidence actually leads."
  — Dr. Jay Bhattacharya [00:00, 45:47]

• On NIH’s Role:

  "Things that we focus on should reflect the real needs of the people that fund us. ... If we’re just doing science for science’s sake ... why should they fund us?"
  — Dr. Jay Bhattacharya [15:43]

• On AI’s Limitations:

  "AI’s really good at summarizing existing knowledge ... really developing brand new ideas that challenge existing paradigms ... they’re not quite as good at that."
  — Dr. Jay Bhattacharya [51:27]

• Inspiration for Rising Scientists:

  "It’s the individual scientist who believes in their idea, keeps knocking on the door even when the door is closed ... That’s who really makes a big difference in this world."
  — Dr. Jay Bhattacharya [53:50]

Timestamps for Key Segments

• NIH’s New Autism Initiatives: 01:16–05:01
• Replication Crisis/Scientific Rigor: 05:01–09:04
• Systemic Reforms & Silicon Valley Mindset: 09:04–15:31
• Funding Allocation and Execution: 15:31–21:50
• Early-Career Scientists & Cultural Change: 24:11–32:59
• Life Expectancy/Missed Health Gains: 32:59–34:39
• Academic Freedom & Publishing: 34:39–37:22
• Rebuilding Public Trust after COVID: 37:22–44:06
• The Science of Uncertainty/Honest Communication: 41:28–47:52
• AI's Role in Science & Policy: 47:52–53:08
• Inspiration & Future Vision for Scientists: 53:08–57:08
• Portfolio Thinking in Health Advances: 55:28–57:08

Summary Tone and Style

• Open, candid, and pragmatic discussion
• Optimistic, solution-focused, and often self-critical
• Balancing deep respect for the public with urgency to reform science for societal benefit
• Borrowing the entrepreneurial, risk-tolerant ethos of Silicon Valley and applying it to biomedical science

For listeners interested in public health policy, scientific reform, biomedical research, and the future of AI in healthcare, this episode is a must-listen—a roadmap for how the world’s biggest biomedical science funder is re-examining everything from peer review to drug discovery to restore U.S. health and global scientific leadership.