How will FDA regulate psychedelics?

How will FDA regulate psychedelics?

FDA has been taking a look at psychedelics as a potential treatment for psychiatric and substance abuse disorders, recently publishing a final guidance [https://www.fda.gov/media/169694/download] for drug sponsors conducting clinical trials of psychedelics, including psilocybin, LSD, and MDMA.  “This is of interest to psycho-oncologists that have been looking into this question for at least two decades around cancer-related anxiety, depression, and PTSD,” said Sara Willa Ernst, a reporter with The Cancer Letter. “Could psilocybin specifically be a treatment to help cancer patients and cancer survivors deal with psychological distress that comes from a cancer diagnosis?” However, regulatory challenges emerge when FDA is tasked with evaluating a treatment that may (or may not) rely on both a drug and a psychotherapy component to improve outcomes. In this episode of The Cancer Letter Podcast, Sara and Paul Goldberg, editor and publisher of The Cancer Letter, discuss the tricky regulatory landscape facing psycho-oncologists.  “FDA does not regulate psychotherapy,” Sara said. “I think a lot of the sources, the clinical trialists I talked to said, admittedly, ‘This is kind of a weird space for FDA to approve or not approve.’” But psycho-oncology experts with experience administering psychedelic therapy are almost uniformly convinced that the psychotherapy component is critical to the treatment’s efficacy. Most of Sara’s sources ended up “echoing the same thing—that the psychotherapy component is very crucial,” she said.  While FDA is staying in its lane and remaining agnostic to a question for which there is not yet satisfying data, Sara said her sources are concerned about what that might mean for the development of the field. “FDA has opened the door—at least in the final guidance—to applications that kind of de-emphasize the psychotherapy aspect. So, that might be a whole lot cheaper for a pharmaceutical company that is trying to bring a drug to market to go in that direction as opposed to the real hefty lift of a factorial design trial [to demonstrate the role of psychotherapy],” Sara said. “A lot of methodologists are having a lot of fun deciphering this,” Paul said. “How do you go forward with this? Could be very interesting.” This episode is sponsored by the University of Kentucky Markey Cancer Center. Learn more at ukhealthcare.com/markey [https://ukhealthcare.com/markey]. Stories mentioned in this podcast include: * The Directors: Two leaders in data science have a warp speed vision for AI, with cancer clinical trials leading the way Knudsen and Thoelke [https://cancerletter.com/podcastc/20260717_1/] * FDA final guidance on psychedelics opens the door to psycho-oncology research [https://cancerletter.com/regulatory-news/20260717_2/] * OMB proposal to sideline peer review sparks widespread opposition [https://cancerletter.com/regulatory-news/20260717_3/] * Cancer progress is not reaching everyone. Native Nations must be part of the solution. [https://cancerletter.com/guest-editorial/20260717_4/] * The future of oncology: Early-career scientists need mentorship and a new academic model [https://cancerletter.com/sponsored-article/20260717_5/] A transcript of this podcast is available: cancerletter.com/podcastc/20260722-psychedelics/ [http://cancerletter.com/podcastc/20260715-retraction/]

Two leaders in data science have a warp speed vision for AI, with cancer clinical trials leading the way

The United States has a translation problem. Scientific breakthroughs in oncology are accelerating, but clinical trial infrastructure hasn’t kept pace.  The solution: Speed up clinical trial enrollment by using AI to reduce costs and eliminate bureaucratic red tape. Tasks that ordinarily require hours of manual work for each patient in a single study can now be completed for 100,000 patients in a matter of minutes.  “If we really do have this problem of translating science to people because of the lack of resourcing and efficiency on the clinical trial side, then I don’t view innovation outside the U.S. as a bad thing. I actually think it’s a great thing, and it’s great for people who are getting access to these clinical trials,” Karen Knudsen, CEO of the Parker Institute for Cancer Immunotherapy, said on the July episode of The Directors.  For years, U.S.-based pharma and biotechnology companies have been offshoring clinical trials to manage labor costs, avoid regulatory burdens, and gain access to larger patient populations that can make recruitment easier.  In this episode of The Directors, Knudsen and Kent Thoelke, CEO of Paradigm Health, a technology company focused on using AI to enhance clinical trials, discuss the factors that are bottlenecking translation in the U.S. and the promising role of AI in modernizing the U.S. clinical trial infrastructure.  A transcript is available at https://cancerletter.com/podcastc/20260717_1/ [https://cancerletter.com/podcastc/20260717_1/]

Did peer review fail? Nature Medicine’s chronotherapy paper retraction raises questions

The cover story in last week’s issue of The Cancer Letter featured a retraction of a Nature Medicine paper demonstrating that giving patients immunochemotherapy before 3:00 p.m. led to massive improvements in overall survival and progression-free survival. In this week's episode of The Cancer Letter Podcast, editor and publisher Paul Goldberg, and associate editor Jacquelyn Cobb revisit the high-profile retraction. The retracted study, one of the first to provide prospective, randomized data on the topic of chronomedicine in cancer immunotherapy, was retracted due to a litany of errors that began to be flagged by the scientific community nearly immediately after the study was published. “When you come up with results, which these folks did, that are really just unbelievably cool and immediately in principle applicable in the clinic, of course, everybody's going to check them. And if you have any booboos in there, they'll find them,” Paul said.  While the cover story in last week’s issue ended up including a sort of “love letter to the scientific process,” with sources Jedd D. Wolchok and Rachel Humphrey proclaiming that the snafu was in fact a signal that the scientific process is working, Paul had his own take on the issue. “My question with something like that always is the same. And I've done a lot of stories about these kinds of booboos and retractions,” Paul said. “The question is the process works, as you say, but sometimes the process really should be prevented from having to work, because if you've got ... What's the French word? Dreck, that should be obviously found in peer-review. Just leave the process, let the process concentrate on something bigger, which I think is what we had here. And as you guys were rhapsodizing about how wonderfully the process worked, my kishkes were grinding. And that's another French word, I'm sorry.” “Rachel talks about this, how you throw it out to the community. But I think there's a hygiene question. What do you throw out to the community? Sometimes you want to spare the community. I do believe that in this case we see ... This is just my opinion, I think we see a failure of Nature Medicine to properly peer review this thing and save embarrassment to the investigators who sound like reasonable people. And the community. I mean, of course, ultimately it's really sparing the patients the nonsense.” This episode is sponsored by City of Hope. Learn more at www.cityofhope.org [http://www.cityofhope.org]. Stories mentioned in this podcast include: * A chronotherapy study in Nature Medicine showed dramatic results. Then it was retracted. The phase III trial claimed a staggering boost to OS and PFS when immunochemotherapy was given before 3 p.m. [https://cancerletter.com/news-analysis/20260710_1/] * OMB’s plan to increase political control of science faces stiff opposition on Capitol Hill [https://cancerletter.com/capitol-hill/20260710_2/] * Saving Western Kenya’s pediatric blood cancer patients How flow cytometry is changing the diagnostic landscape [https://cancerletter.com/trials-and-tribulations/20260710_3/] * The challenge of making the move from conversational AI to autonomous scientific discovery Advancing from level II to level III AI agents in precision medicine [https://cancerletter.com/trials-and-tribulations/20260710_4/] * The long life of a clinical trial How JCO sustains landmark oncology research [https://cancerletter.com/sponsored-article/20260710_5/] * FDA halts public release of Complete Response Letters, following citizen petition [https://cancerletter.com/cancer-policy/20260710_6a/]  A transcript of this podcast is available: cancerletter.com/podcastc/20260715-retraction/ [http://cancerletter.com/podcastc/20260715-retraction/]

A new diagnostic lens—and a lasting scientific legacy

In this week's episode of The Cancer Letter Podcast, editor and publisher Paul Goldberg, and associate editor Jacquelyn Cobb revisit two of last week’s most-read stories: the growing recognition that early-onset colorectal cancer requires a new diagnostic mindset, and the life and legacy of Maura Gillison, the scientist whose discovery that most head and neck cancers are driven by HPV transformed the field.  The cover story follows Jenna Scott, who was diagnosed with stage 4 colorectal cancer a year after giving birth to her son. Her cancer was repeatedly missed because its symptoms—rectal bleeding, abdominal pain, nausea—overlapped with those of pregnancy and the postpartum period. Researchers say her experience is indicative of a larger phenomenon: early-onset colorectal cancer increasingly appears to be a biologically distinct disease, affecting younger, otherwise healthy patients and often presenting at a more advanced stage.   “It's a time of patient advocacy, which has been going on really through most of my career, but patient stories really need to get out and be told,” Paul said. “I think that is a bit of a change now because at this time when science is in jeopardy, hearing from patients is very, very important.” This episode of the podcast also covers the legacy of Maura Gillison, who died on June 21 at age 61. Her groundbreaking research established that HPV causes the majority of head and neck cancers, fundamentally changing how those cancers are understood and treated.  “I'm going to steal a factoid that you mentioned earlier, Paul, but it's like 70% of head and neck cancers are caused by HPV. So, the magnitude of the effect is really huge here. The numbers are huge,” said Jacquelyn. Stories mentioned in this podcast include: * Early-onset colorectal cancer is behaving like a new, biologically distinct disease, researchers note. One woman’s pregnancy shows how this distinction matters [https://cancerletter.com/clinical/20260702_1/] * Maura Gillison, who identified the role of HPV in head and neck cancer, changing the standard of care, dies at 61 [https://cancerletter.com/obituary/20260702_2/] * Is it time to re-assess the practice and practicality of screening colonoscopy? Modeling data in ACS guideline support greater use of noninvasive tests to ease system strain, save lives [https://cancerletter.com/trials-and-tribulations/20260702_3/] * More than 50 million Americans remain unscreened for colon cancer. New guidelines offer another option [https://cancerletter.com/trials-and-tribulations/20260702_4/] A transcript of this podcast is available: cancerletter.com/podcastc/20260708-legacy/ [http://cancerletter.com/podcastc/20260708-legacy/]

Breaking down Operation Trailblazer: Rolling INDs, outside expert submission help, and one-trial requirement

In this week’s episode of The Cancer Letter Podcast, Paul Goldberg, editor and publisher of The Cancer Letter, and Sara Willa Ernst, reporter, break down HHS’ latest vision for the future: Operation Trailblazer.  The report [https://cancerletter.com/regulatory-news/20260626_2/] is titled: “HHS Roadmap to Maintaining U.S. Leadership in Early Clinical Research and Development.” “The U.S. is apparently losing its leadership in INDs and early phase research,” Paul said. “Some of it is true and also it does not help that the whole enterprise of cancer research right now is in, if not disarray, then at least a kind of mental and spiritual discombobulation.” Most notable were proposed changes to FDA, including an expedited Investigational New Drug program, which would use expertise outside FDA to help prepare INDs before submission, and a lower threshold—just one high-quality late-stage—for confirmatory evidence, which is already common practice in oncology.  Paul checked in with sources about the report after it was released. What he heard with his ear to the ground: It’s unclear whether the plans in the report will amount to policy.  “We don't know whether it's going to happen and there is a problem right now in that FDA really took a hit in terms of staffing,” Paul said. “At the moment, there is no permanent FDA commissioner. Having leadership that's deeply destabilizing was really a problem.” “FDA really right now needs to get its house in order or as the industry says, ‘right the ship,’” he continued. “A rudderless FDA for a while there was a step in the wrong direction. So, they're going to have to clean this up and maybe they will. Let's hope they do. “That's what I'm hearing from folks as I call them. But it was important to get [the news] out on paper even though the story certainly has a large number of caveats.” Stories mentioned in this podcast include: * The Directors: Directors of two Midwestern cancer centers tell us about the challenging healthcare economics in rural areas [https://cancerletter.com/podcastc/20260626_1/] * To keep the early clinical trial enterprise in the U.S., HHS pledges to simplify regulatory requirements [https://cancerletter.com/regulatory-news/20260626_2/] * Joe Fraumeni, founder of molecular epidemiology, dies at 93 [https://cancerletter.com/obituary/20260626_3/] * Karen Huntsman’s vision gave shape to the Huntsman Cancer Institute [https://cancerletter.com/obituary/20260626_4/] * Robert Mayer tells us about pancreatic cancer’s hopeless past as new data shows promise [https://cancerletter.com/in-the-archives/20260626_5/] A transcript of this podcast is available: https://cancerletter.com/podcastc/20260701-operation-trailblazer/