Category: Health

Father Spends 20 Years Researching to Cure His Children’s Type 1 Diabetes

The development of a new drug to cure Type 1 diabetes is big news, a triumph of medicine. The process of developing the medicine and bringing it to market interests me for several reasons. One is that Doug Melton spent 20 years of effort on it. His passion was due to having skin in the game: he has two children with the disease. Another is that it took so many years “of painstaking, repetitive, frustrating work.” I emphasize the common importance of trial-and-error in many major medical discoveries. Another is that the trial-and-error was to develop a “chemical cocktail to turn stem cells into islet cells.” Several major medical advances have required nimble and persistent trial-and-error to adjust drug cocktails, in terms of components and doses. Examples include HIV, Hodgkin’s lymphoma, and childhood leukemia.

A final reason I am interested in the case is that Melton selected the Vertex company to bring the drug to market. Vertex is an interesting case of a large firm struggling to keep the innovative culture of its startup roots. I read a book about its struggles called The Antidote. I intend to read an earlier book about its early years called The Billion Dollar Molecule.

(p. 17) A single infusion of a stem cell-based treatment may have cured 10 out of 12 people with the most severe form of type 1 diabetes. One year later, these 10 patients no longer need insulin. The other two patients need much lower doses.

The experimental treatment, called zimislecel and made by Vertex Pharmaceuticals of Boston, involves stem cells that scientists prodded to turn into pancreatic islet cells, which regulate blood glucose levels. The new islet cells were infused and reached the liver, where they took up residence.

The study was presented Friday evening [June 20, 2025] at the annual meeting of the American Diabetes Association and published online by The New England Journal of Medicine.

“It’s trailblazing work,” said Dr. Mark Anderson, professor and director of the diabetes center at the University of California in San Francisco. “Being free of insulin is life changing,” added Dr. Anderson, who was not involved in the study.

. . .

The treatment is the culmination of work that began more than 25 years ago when a Harvard researcher, Doug Melton, vowed to find a cure for type 1 diabetes. His 6-month-old baby boy developed the disease and, then, so did his adolescent daughter. His passion was to find a way to help them and other patients.

He began, he said, with an “unwavering belief that science can solve the most difficult problems.”

It took 20 years of painstaking, repetitive, frustrating work by Dr. Melton and a team of about 15 people to find the right chemical cocktail to turn stem cells into islet cells. He estimated that Harvard and others spent $50 million on the research.

Dr. Peter Butler, a professor of medicine at the University of California, Los Angeles and a consultant to Vertex, said he was awed by the achievement of the Harvard team.

“The fact that it worked at all is just freaking amazing to me,” he said. “I can guarantee there were a thousand negative experiments for every positive one.”

When Dr. Melton finally succeeded, he needed a company to take the discovery into the clinic. He joined Vertex, which took up the challenge.

For the full story see:

Gina Kolata. “People With Severe Diabetes May Have Been Cured in a Small Trial of a New Drug.” The New York Times, First Section (Sun., June 22, 2025): 17.

(Note: ellipsis, and bracketed date, added.)

(Note: the online version of the story was updated June 21, 2025, and has the title “People With Severe Diabetes Are Cured in Small Trial of New Drug.” The online version says that the article appeared on page 24 of the New York edition of the print version. But the article appeared on page 17 of my National edition.)

The NEJM academic article co-authored by Melton and mentioned above is:

Reichman, Trevor W., James F. Markmann, Jon Odorico, Piotr Witkowski, John J. Fung, Martin Wijkstrom, Fouad Kandeel, Eelco J.P. de Koning, Anne L. Peters, Chantal Mathieu, Leslie S. Kean, Bote G. Bruinsma, Chenkun Wang, Molly Mascia, Bastiano Sanna, Gautham Marigowda, Felicia Pagliuca, Doug Melton, Camillo Ricordi, and Michael R. Rickels. “Stem Cell–Derived, Fully Differentiated Islets for Type 1 Diabetes.” The New England Journal of Medicine (published online on June 20, 2025), DOI: 10.1056/NEJMoa2506549.

The books that I mentioned about Vertex are:

Werth, Barry. The Antidote: Inside the World of New Pharma. New York: Simon & Schuster, 2014.

Werth, Barry. The Billion-Dollar Molecule: One Company’s Quest for the Perfect Drug. New York: Simon & Schuster, 1994.

Jarvik’s Father’s Heart Disease Drove Him to Persist in Developing First Permanent Artificial Heart

Robert Jarvik had skin in the game, had a sense of urgency, with his father suffering from severe heart disease. And he understood that the usual path toward an eventual breakthrough, is to keep “working it through so it can be better.”

(p. B10) Dr. Robert K. Jarvik, the principal designer of the first permanent artificial heart implanted in a human — a procedure that became a subject of great public fascination and fierce debate about medical ethics — died on Monday [May 26, 2025] at his home in Manhattan. He was 79.

. . .

In a 1989 interview with Syracuse University Magazine, Dr. Jarvik admitted that his belief that the Jarvik-7 was advanced enough to be used widely on a permanent basis was “probably the biggest mistake I have ever made.”

Still, he defended his work. Of the five recipients of the permanent Jarvik-7, he told the magazine, “These were people who I view as having had their lives prolonged,” adding that they survived nine months on average when some had been expected to live “no more than a week.”

“I don’t think that kind of thing makes a person in medicine want to stop,” he said. “It just makes you all the more interested in working it through so it can be better.”

. . .

From an early age, Robert was a tinkerer. As a teenager, he made his own hockey mask and began developing a surgical stapler. He attended Syracuse University from 1964 until 1968, intending to study architecture, but his interest turned to medicine after his father survived an aortic aneurysm, and he received a degree in zoology. Dr. Norman Jarvik died in 1976 after a second aneurysm.

“I knew that my father was going to die of heart disease, and I was trying to make a heart for him,” Robert Jarvik once said. “I was too late.”

. . .

According to a 2023 study of the artificial heart market, a descendant of the original Jarvik-7, now owned by another company, is called the SynCardia Total Artificial Heart. It is designed primarily for temporary use in patients who face imminent death while awaiting transplants. The study found that the device had been implanted in more than 1,700 patients worldwide.

For the full obituary, see:

Jeré Longman. “Robert Jarvik, a Designer of the First Artificial Heart, Is Dead at 79.” The New York Times (Friday, May 30, 2025): B10.

(Note: ellipses, and bracketed date, added.)

(Note: the online version of the obituary has the date May 29, 2025, and has the title “Robert Jarvik, 79, Dies; a Designer of the First Permanent Artificial Heart.”)

The Democratic Deep State Looked the Other Way as Fraudsters Stole 10 BILLION Healthcare Dollars

When DOGE fired federal workers we saw televised scenes where the fired workers expressed outrage at how taxpayers would be hurt by the loss of devoted civil servants. So where were the devoted civil servants in 2023? Were they doing their jobs to be alert to the Medicare, and personal identity, fraud that cost the public about 10 billion (that is “billion” with a “b”) dollars?

When Elon Musk’s DOGE uncovered myriad examples of major fraud, I saw Democrats on television complain that of course they were against fraud too, but it should be pursued more slowly and systematically, following traditional procedures. The Democrats were running the federal government in 2023. What procedure were the Democrats using, fast or slow, to protect taxpayers from the fraudulent loss of 10 billion (that is “billion” with a “b”) dollars?

Our jerry-rigged government-run-and-regulated health care system is rife with middlemen. In a true free-market healthcare system, patients would directly pay for healthcare, without middlemen. Patients would have the information, and the incentive to act on the information, to detect, report, and pursue fraudsters. Some fraud would exist under any system, but my hypothesis is that much less of it would exist under a free-market system.

(If you are concerned that patients would not have enough funds to pay for healthcare themselves, we could adopt the much better insurance system once proposed by Susan Feigenbaum, combined with deregulation that would reduce healthcare costs–like no longer mandating Phase 3 clinical trials.)

And my secondary hypothesis is that if we have to have a jerry-rigged government-run-and-regulated system, the Republicans, a party full of former bourgeois entrepreneurs and business managers, will usually do a marginally better job of detecting and pursuing fraud.

I wonder if these hypotheses have ever been researched by any of those noble economists studying the field of Public Choice?

(p. A18) When hundreds of thousands of people enrolled in Medicare were billed for expensive medical equipment they never asked for in 2023, it was part of a $10.6 billion fraud, among the largest such schemes in the program’s history, federal prosecutors said this week.

. . .

Those involved in the fraud bought dozens of companies that were accredited to submit claims to Medicare and the program’s supplemental insurers, prosecutors say.

Then, using personal information stolen from more than a million Americans, the defendants filed billions of dollars in claims for equipment that had not been ordered by people enrolled in Medicare and was not delivered to them, according to the indictment.

Of the $10.6 billion that was fraudulently billed, the indictment says, the defendants collected more than $900 million, most of it coming from private “Medigap” insurers and the rest from the Medicare program itself.

Even if the patients themselves did not pay for the phantom supplies, which included urinary catheters, braces and other durable medical equipment, such schemes can affect Medicare recipients by causing premium costs to rise.

. . .

In 2019, the Justice Department uncovered a scheme that it said had defrauded the program of more than $1 billion with phony claims for back and knee braces. In April 2023, prosecutors charged 18 defendants in a nearly $500 million scheme that involved false billing for Covid-19 tests that were never administered.

For the full story see:

Santul Nerkar. “11 Accused of Medicare Fraud In Scheme Based in Russia.” The New York Times (Sat., June 18, 2025): A18.

(Note: ellipses added.)

(Note: the online version of the story has the date June 27, 2025, and has the title “U.S. Charges 11 in Russia-Based Scheme to Bilk Medicare of $10.6 Billion.”)

The better healthcare insurance system proposed by Susan Feigenbaum was proposed in:

Feigenbaum, Susan. “Body Shop’ Economics: What’s Good for Our Cars May Be Good for Our Health.” Regulation 15, no. 4 (Fall 1992): 25-31.

A.I. Hastens Search for Antibiotic Peptides in Extinct Species

In an earlier entry I commented on the use of A.I. to seek antibodies by George Church’s startup Lila. Now it appears that César de la Fuente is employing a similar approach. In both cases A.I. is being used to more efficiently do repetitive well-structured tasks. This is not the highest creative level of human intelligence, but it can free time for humans to exercise the highest level of human intelligence.

(p. A3) Buried in the DNA of the long extinct woolly mammoth is a compound that scientists hope will one day yield a lifesaving antibiotic.

In experiments, mammuthusin, as the compound is called, has eradicated superbugs—bacteria that are resistant to today’s antibiotics and cause infections that are hard to treat—says César de la Fuente, the bioengineer who helped discover the molecule.

. . .

To help combat superbugs, doctors say we need new antibiotics with novel chemical structures or mechanisms of action. But only a handful of such drugs has entered the market over the past several decades.

De la Fuente is banking on artificial intelligence to help end this dry spell. He and his collaborators have built deep-learning algorithms to comb through enormous genetic databases to find peptides, or protein fragments, that have antibacterial properties. They have used this method to analyze animal venoms, the human microbiome and archaea, an underexplored group of microorganisms. They have also mined the genetic codes from fossils of long-extinct animals and humans, including Neanderthals and Denisovans. “This deep-learning model has opened a window into the past,” de la Fuente says.

. . .

When the algorithms identify a new peptide with antibiotic potential, de la Fuente and his team use robots to manufacture the compound in their lab and then test it in mice infected with bacteria. So far, a few hundred peptides made in de la Fuente’s lab have safely and effectively cured sick mice.

One of them was mammuthusin, identified in the genetic code of Mammuthus primigenius, a species of mammoth that last roamed the Earth about 4,000 years ago. The researchers discovered the peptide after mining a National Center for Biotechnology Information database of DNA sequencing data obtained from the fossils of extinct animals. In experiments, mammuthusin was as potent as polymyxin B, an antibiotic often used as a last resort for serious infections, according to a paper published in the journal Nature in June [2024]. The mammoth peptide effectively eradicated a type of bacterium that the World Health Organization has designated a critical pathogen because of its resistance to many common antibiotics.

For the full story, see:

Dominique Mosbergen. “Search for New Antibiotics Turns Back Time.” The Wall Street Journal (Weds., May 28, 2025): A3.

(Note: ellipses, and bracketed year, added.)

(Note: the online version of the story has the date May 24, 2025, and has the title “A Search for New Antibiotics in Ancient DNA.” In the original of both the online and print versions, Mammuthus primigenius appeared in italics.)

The academic article published in Nature Biomedical Engineering in June 2024, and mentioned above, is:

Wan, Fangping, Marcelo D. T. Torres, Jacqueline Peng, and Cesar de la Fuente-Nunez. “Deep-Learning-Enabled Antibiotic Discovery through Molecular De-Extinction.” Nature Biomedical Engineering 8, no. 7 (July 2024): 854-71.

One Third of Near-Death Multiple Myeloma Patients Are Cured by a New CAR-T Immunotherapy

Many consider immunotherapy to be the most promising current approach to curing cancer. One way to implement immunotherapy is to develop CAR-T cells. But there apparently are many ways to develop a CAR-T cell and which, if any, will work is a matter of trial-and-error.

It seems overly-cautious for regulators to require that the most innovative and promising therapies must first be tried on the patients nearest to death, and so least likely to respond. Why not allow patients at earlier stages to volunteer to try the new therapies earlier? They would be taking a bigger risk, but also would have the possibility of a bigger benefit. They would avoid the suffering from current treatments that are known to have major side-effects, and also are known to only extend life for short periods of time; and they would gain a shot at a real long-term cure.

(p. A18) A group of 97 patients had longstanding multiple myeloma, a common blood cancer that doctors consider incurable, and faced a certain, and extremely painful, death within about a year.

They had gone through a series of treatments, each of which controlled their disease for a while. But then it came back, as it always does. They reached the stage where they had no more options and were facing hospice.

They all got immunotherapy, in a study that was a last-ditch effort.

A third responded so well that they got what seems to be an astonishing reprieve. The immunotherapy developed by Legend Biotech, a company founded in China, seems to have made their cancer disappear. And after five years, it still has not returned in those patients — a result never before seen in this disease.

These results, in patients whose situation had seemed hopeless, has led some battle-worn American oncologists to dare to say the words “potential cure.”

. . .

The new study, reported Tuesday [June 3, 2025] at the annual conference of the American Society of Clinical Oncology and published in The Journal of Clinical Oncology, was funded by Johnson & Johnson, which has an exclusive licensing agreement with Legend Biotech.

. . .

The Legend immunotherapy is a type known as CAR-T. It is delivered as an infusion of the patient’s own white blood cells that have been removed and engineered to attack the cancer. The treatment has revolutionized prospects for patients with other types of blood cancer, like leukemia.

Making CAR-T cells, though, is an art, with so many possible variables that it can be hard to hit on one that works.

. . .

The . . . study took on a . . . challenge — helping patients at the end of the line after years of treatments. Their immune systems were worn down. They were, as oncologists said, “heavily pretreated.” So even though CAR-T is designed to spur their immune systems to fight their cancer, it was not clear their immune systems were up to it.

Oncologists say that even though most patients did not clear their cancer, having a third who did was remarkable.

To see what the expected life span would be for these patients without the immunotherapy, Johnson & Johnson looked at data from patients in a registry who were like the ones in its study — they had failed every treatment. They lived about a year.

. . .

. . ., the hope is that perhaps by giving it earlier in the course of the disease, it could cure patients early on.

Johnson & Johnson is now testing that idea.

Dr. Kenneth Anderson, a myeloma expert at Dana-Farber Cancer Institute who was not involved with the study, said that if the treatment is used as a first-line treatment, “cure is now our realistic expectation.”

For the full story, see:

Gina Kolata. “From No Hope to Potential Cure for Deadly Blood Cancer, Study Shows.” The New York Times (Thurs., June 5, 2025): A18.

(Note: ellipses, and bracketed date, added.)

(Note: the online version of the story was updated June 5, 2025, and has the title “From No Hope to a Potential Cure for a Deadly Blood Cancer.”)

The academic article on the new cure is:

Jagannath, Sundar, Thomas G. Martin, Yi Lin, Adam D. Cohen, Noopur Raje, Myo Htut, Abhinav Deol, Mounzer Agha, Jesus G. Berdeja, Alexander M. Lesokhin, Jessica J. Liegel, Adriana Rossi, Alex Lieberman-Cribbin, Saad Z. Usmani, Binod Dhakal, Samir Parekh, Hui Li, Feng Wang, Rocio Montes de Oca, Vicki Plaks, Huabin Sun, Arnob Banerjee, Jordan M. Schecter, Nikoletta Lendvai, Deepu Madduri, Tamar Lengil, Jieqing Zhu, Mythili Koneru, Muhammad Akram, Nitin Patel, Octavio Costa Filho, Andrzej J. Jakubowiak, and Peter M. Voorhees. “Long-Term (≥5-Year) Remission and Survival after Treatment with Ciltacabtagene Autoleucel in Cartitude-1 Patients with Relapsed/Refractory Multiple Myeloma.” Journal of Clinical Oncology https://doi.org/10.1200/JCO-25-0076.

Public Should Beware of F.D.A.’s Lax Inspections of Drugs Made in India and China

On drug safety, the public puts its trust in the F.D.A. which frequently drops the ball. The public would be better off adopting caveat emptor, and seeking private means of assurance of safety. These would include certification (stamps of approval) from private organizations like the United States Pharmacopeial Convention (USP).

They might also choose to buy well-known name brands, since a well-known brand has more reputation capital to lose if they produce a bad batch, and thus can be expected to invest more in quality control. (Lester Telser made this argument–I need to seek where.)

(p. A10) A highly drug-resistant bacteria that was linked to eyedrops imported from India and that spread from person to person in a Connecticut long-term care center has prompted concerns that the strain could gain a foothold in U.S. health care settings, according to the Centers for Disease Control and Prevention.

. . .

The outbreak has renewed longstanding concerns about the quality and frequency of the F.D.A.’s overseas inspections.

In June 2020, Senator Chuck Grassley, Republican of Iowa, held an oversight hearing on the F.D.A.’s foreign inspection process, noting that the plants were given 12 weeks’ advance notice, “plenty of time to doctor up a facility to make sure that it passes inspection.” The agency has since received budget authority to conduct unannounced overseas inspections.

. . .

The F.D.A. paused overseas inspections during the height of the coronavirus pandemic, and the number of foreign inspections remained low last year, at 684 compared with 3,272 in 2019, according to agency data.

The F.D.A. has 4,000 overseas facilities to inspect, with about 20 percent in India; one of its six inspector positions in that country was vacant in late 2021, according to a report issued last year by the Government Accountability Office.

For over-the-counter drugs, the F.D.A. uses a system that essentially lists a medication recipe. Companies can make the products without express agency approval but are expected to follow agency rules for manufacturing quality products, said John Serio, a lawyer with Withers who has pharmaceutical clients.

“If you’re not out there inspecting facilities,” Mr. Serio said, “these sorts of problems will crop up because there’s no threat that if you’re out of compliance that the inspector will come knocking at your door.”

For the full story see:

Christina Jewett and Andrew Jacobs. “Drug-Resistant Bacteria Linked To Eyedrops Can Easily Spread.” The New York Times (Mon., April 3, 2023 [sic]): A10.

(Note: ellipses added.)

(Note: the online version of the story has the same date as the print version, and has the title “Drug-Resistant Bacteria Tied to Eyedrops Can Spread Person to Person.”)

The F.D.A.’s lax inspections of generic drugs made in India is documented in:

Eban, Katherine. Bottle of Lies: The Inside Story of the Generic Drug Boom. New York: Ecco, 2019.

Electricity May Be a Pellet in the “Magic Buckshot” Against Cancer

In a recent entry I claimed that the cure for many diseases may not be Paul Ehrlich’s one “magic bullet” but may instead be “magic buckshot.” A recent article in The Wall Street Journal suggests that one pellet in the magic buckshot against cancer is electricity. As proof of concept, the article claims that after surgery, radiation, and chemotherapy for a glioblastoma brain cancer, adding electrodes to the skull that deliver low-intensity electricity to the brain, will add a median of 4.9 months to the patient’s lifespan.

The Wall Street Journal article mentioned above is:

Brianna Abbott. “Next Hope in Treating Cancer: Electricity.” The Wall Street Journal (Tues., May 20, 2025): A10.

(Note: the online version of the article has the date May 16, 2025, and has the title “The Next Frontier to Treat Cancer: Electricity.”)

The Wall Street Journal article links to the following article in JAMA:

Stupp, Roger, Sophie Taillibert, Andrew Kanner, William Read, David M. Steinberg, Benoit Lhermitte, Steven Toms, Ahmed Idbaih, Manmeet S. Ahluwalia, Karen Fink, Francesco Di Meco, Frank Lieberman, Jay-Jiguang Zhu, Giuseppe Stragliotto, David D. Tran, Steven Brem, Andreas F. Hottinger, Eilon D. Kirson, Gitit Lavy-Shahaf, Uri Weinberg, Chae-Yong Kim, Sun-Ha Paek, Garth Nicholas, Jordi Bruna, Hal Hirte, Michael Weller, Yoram Palti, Monika E. Hegi, and Zvi Ram. “Effect of Tumor-Treating Fields Plus Maintenance Temozolomide Vs Maintenance Temozolomide Alone on Survival in Patients with Glioblastoma: A Randomized Clinical Trial.” JAMA 318, no. 23 (Dec. 19, 2017): 2306-16.

During Covid-19 “Bureaucratic Authorities Erred in Pretending . . . Certainty”

(p. A13) Adam Kucharski, a professor of epidemiology at the London School of Hygiene & Tropical Medicine, takes the reader on a fascinating tour of the history of what has counted as proof.

. . .

What should we do, . . ., when a mathematical proof of truth is unavailable, but we must nonetheless act?

This leads us to a discussion of probability and statistics, and of pioneers such as William Gosset, a brewer at Guinness who figured out how to quantify random errors in experiments, and Janet Lane-Claypon, an English scientist who first thought to investigate confounding factors while analyzing children’s health. Some innovations, though, have hardened into unhelpful dogma. The scientific notion of “statistical significance” relies, Mr. Kucharski explains, on a wholly arbitrary cutoff, which incentivizes researchers to massage their data. Such issues, he says, can be hard for scientists, let alone the laity, to understand.

Mr. Kucharski speaks from experience, since he was one of the experts first called upon by the British government for advice on the Covid-19 pandemic. He explains brilliantly the fragmentary and confusing nature of the data then available, and the provisional conclusions they led to. As a public face of this effort, Mr. Kucharski was bombarded daily with abusive and threatening messages from angry citizens who simply didn’t believe what they were being told.

The lesson Mr. Kucharski draws isn’t that he and his colleagues were right (though they largely were), but that bureaucratic authorities erred in pretending there was certainty when all that was possible at the time was messy and provisional. Notoriously, in March 2020 the World Health Organization tweeted “FACT: #COVID19 is NOT airbone.” (As it turns out, it was, and it is.) The author regrets, too, that politicians claimed to be “following the science,” because science can never tell you what you should do.

For the full review see:

Steven Poole. “Bookshelf; Finding Truth In Numbers.” The Wall Street Journal (Friday, June 6, 2025): A13.

(Note: ellipses added.)

(Note: the online version of the review has the date June 5, 2025, and has the title “Bookshelf; ‘Proof’: Finding Truth in Numbers.”)

The book under review is:

Kucharski, Adam. Proof: The Art and Science of Certainty. New York: Basic Books, 2025.

Dream of the Magic Buckshot

Paul Ehrlich in the early 1900s sought a “magic bullet” for each disease (including cancer). Given the alternatives at the time, when he found Salvarsan it could be considered a magic bullet against syphilis. Today Dr. Dale Bredesen has replaced “magic” with “silver” and “bullet” with “buckshot.” In his effort to cure Alzheimer’s “Bredesen believed in firing a “silver buckshot” (a reference to the sprayed pellets that come out of shotgun shells) by modifying 36 factors simultaneously” (Gellman, p. 18).

I have not investigated Dr. Bredesen’s “cure” for Alzheimer’s and express no opinion on it. I will express the opinion that I do not like the arrogantly dismissive tone of Lindsay Gellman’sThe New York Times article, bowing to “experts,” but calling Bredesen “Mr.” instead of the “Dr.” he has earned.

And I do like the idea that sometimes what is effective against a disease (including cancer) is not a single drug or therapy, but several taken together. For example, multi-drug cocktails have been effectively used against HIV, childhood leukemia, and Hodgkin’s lymphoma.

Ehrlich’s big dream was to find a magic bullet for each disease. But maybe it is mostly more promising to dream of the magic buckshot.

[N.B., the “Paul Ehrlich” I refer to is not the contemporary environmental alarmist “Paul Ehrlich” who famously lost his bet with the heroic heretic Julian Simon.]

The NYT article quoted above:

Lindsay Gellman. “Lifestyle to Reverse Alzheimer’s Carries High Costs and, Many Say, False Hope.” The New York Times, First Section (Sun., May 25, 2025): 1 & 18.

(Note: the online version of the NYT article was updated May 31, 2025, and has the title “An Expensive Alzheimer’s Lifestyle Plan Offers False Hope, Experts Say.”)

‘Getting Old Sucks, but It’s Still Preferable to Getting Cremated’

Apparently Billy Joel was unable to attend the celebration of a new documentary on the life of Billy Joel, since he had acquired a malady of the brain called “normal pressure hydrocephalus.” But Joel sent his greetings to the crowd: ‘Getting old sucks, but it’s still preferable to getting cremated’ (Zuckerman, p. C3).

“The audience roared in laughter” (Zuckerman, p. C3).

NYT article quoted above:

Esther Zuckerman. “When Billy Joel Started Singing Us Songs.” The New York Times (Sat., June 7, 2025): C3.

(Note: the online version of the NYT article has the date June 5, 2025, and has the title “‘And So It Goes’ Traces Billy Joel’s Dramatic Early Days: 5 Takeaways.”)

Kennedy Defends Patients’ Freedom to Try “Experimental Therapies”

I agree with Kennedy that people with diseases or conditions that cannot be cured by mainstream medicine have the right try experimental therapies, and should be allowed what some are calling “health freedom.” For those who want certification of safety, private labels would emerge if the government exited from its role of allowing or banning. In the unregulated supplement business, multiple such organizations exist, e.g., the United States Pharmacopeial Convention (USP).

If the public decides the Kennedy/Diamond position is too radical, a step in the right direction would be for the government to provide informational certification of therapies or providers, but not to mandate that patients use only those therapies and providers.

(p. A1) Health Secretary Robert F. Kennedy Jr. recently declared that he wanted to expand access to experimental therapies but conceded that they could be risky or fraudulent.

In a podcast with Gary Brecka, who describes himself as a longevity expert, Mr. Kennedy vowed to end what he called the Food and Drug Administration’s war with alternative medicine. He said that would include stem cells, vitamins, peptides and chelation therapy, which involves removing heavy metals from the blood.

“If you want to take an experimental drug — you can do that, you ought to be able to do that,” Mr. Kennedy said.

“And of course you’re going to get a lot of charlatans, and you’re going to get people who have bad results,” he added. “And ultimately, you can’t prevent that either way. Leaving the whole thing in the hands of pharma is not working for us.”

Mr. Kennedy cited his own experience at a clinic in Antigua, where he said he received a stem cell treatment that “enormously” eased his neurological condition, spasmodic dysphonia, which affects his voice and has few treatment options.

. . .

(p. A11) Neither Mr. Kennedy nor the F.D.A. has released a formal plan to change agency standards for stem cell treatments, which have typically been reviewed by the agency as individual therapies to treat a specific disease.

Widening overall access could also happen informally if the agency decided to relax enforcement, an approach the F.D.A. used in the past to indicate that it wouldn’t crack down on unauthorized products. During the pandemic, for example, the agency allowed providers to retrofit infusion pumps and ventilators to treat hordes of sick patients.

. . .

Dr. Moreno and his colleagues found that the trials with funding from the red meat industry were nearly four times as likely to report favorable or neutral cardiovascular results after eating unprocessed red meat when compared with the studies with no such links.

. . .

“We don’t want to have the Wild West,” Mr. Kennedy said. “We want to make sure that information is out there. But we also want to respect the intelligence of the American people — the capacity of people who explore the outcomes that are going to benefit them the most.”

. . .

The field of stem cell treatments is so complex that the Harvard Medical School created a free course to help doctors navigate patient questions, said Insoo Hyun, the director of life sciences at the Museum of Science in Boston.

. . .

Some providers sidestep the costly, yearslong process of careful work that can lead to an F.D.A. approval. Among them is Dr. Chadwick Prodromos, a Chicago doctor who offers stem cell treatments in Antigua. Mr. Kennedy welcomed him warmly at the March meeting, Dr. Raizman recalled. Reached for comment, Dr. Prodromos’s office said that he was in Antigua doing treatments and was not available.

. . .

A website for Dr. Prodromos’s clinic says that he and colleagues offer injections in Antigua into the joints, back, neck, scalp, penis and pelvic floor for an array of conditions including autism, thinning hair and lupus. He uses AlloRX cells, which are derived from the umbilical cord, in a manner that in the United States would require an F.D.A.-cleared clinical trial.

People can seek out unregulated treatments using their own cells that are processed, purified and amplified in different ways. They can also find treatments using others’ cells that vary widely in quality and sterility.

Some low-quality clinics process cells in a back room, which is the opposite of a clinical-grade cell processing site. Dr. Hyun said he recently toured one in the Netherlands that used specialized air filtering, layers of gowns and a ban on bacteria-laden cellphones in their sterile area. “It’s kind of like you’re entering a space station,” he said.

For the full story see:

Christina Jewett. “‘Charlatans’ No Reason to Curb Untested Drugs, Kennedy Says.” The New York Times (Fri., June 6, 2025): A1 & A11.

(Note: ellipses added.)

(Note: the online version of the story has the date June 5, 2025, and has the title “Kennedy Says ‘Charlatans’ Are No Reason to Block Unproven Stem Cell Treatments.”)