Breakthrough Innovations Often Reach Success Through Incremental Improvements

After 130 days, the patient with the longest (so far) transplant of a pig kidney decided to have the transplant “explanted” and return to dialysis. Her surgeon explained that this should not be viewed as a failure of the basic innovation. The patient had the option to continue improvisations to keep the pig kidney alive, but decided that for her the risks had become too high.

The surgeon, Dr. Robert Montgomery said:

“All this takes time,” he said. “This game is going to be won by incremental improvements, singles and doubles, not trying to swing for the fences and get a home run.” (p. A24)

The pig kidney transplants follow the pattern of many other medical innovations, where on-the-fly adjustments, when the protocol allows them, lead to longer duration successes with fewer side effects. Emil Freireich found this with his chemo cocktails for childhood leukemia. Early human heart transplants also followed this pattern.

We should not allow early setbacks to push us to overregulate the incremental progress that can eventually leads to success.

My source is:

Roni Caryn Rabin. “Pig Kidney Is Removed From an Alabama Woman After Organ Is Rejected.” The New York Times (Sat., April 12, 2025): A24.

(Note: the online version of the article has the date April 11, 2025, and has the title “Pig Kidney Removed From Alabama Woman After Organ Rejection.”)

90% of Biomedical Articles Are “Either Misleading, Wrong or Completely Fabricated”

The right to health freedom is primarily an ethical issue. But the uncertainty and unreliability of much medical “knowledge” (as argued in the book reviewed in the passages quoted below) seems to strengthen the case for patient self-determination.

(p. A15) The largest repositories of biomedical research in the U.S. and Europe, PubMed and Europe PMC, contain 84 million articles between them, and add a million more each year. According to recent estimates, up to 90% of those papers—75 million total—contain information that’s either misleading, wrong or completely fabricated.

Over the past 20 years, certain branches of science have endured a so-called reproducibility crisis, in which countless papers have been exposed as shoddy if not bogus. Sometimes these revelations are merely embarrassing, but in biomedical research, incorrect publications can cost lives as doctors and drugmakers rely on them to treat patients.

In “Unreliable: Bias, Fraud, and the Reproducibility Crisis in Biomedical Research,” Csaba Szabo—a physician with doctorates in physiology and pharmacology—dissects the ways he’s seen research go wrong in his 30 years in academia and industry: data manipulation, poor experimental design, statistical errors and more.

. . .

The biggest problem, however, lies with scientists who strive to do good work but feel pressured to cut corners. Scientists cannot work without grant money, but of the 70,000 applications the National Institutes of Health receive each year, only 20% get funded. Leading journals reject up to 99% of papers submitted, and only one in 200 doctoral graduates ever becomes a full professor. Even with tenure, professors can suffer salary cuts or have their labs handed to higher-performing colleagues if they don’t keep pulling in cash. Some sadistic research professors even pit their graduate students against each other in “dogfights”—they run the same experiment, but only the first to get results publishes. No wonder researchers massage data or fudge images: Forget “publish or perish.” It’s “fib or forgo your career.”

. . .

Given this tsunami of mistakes, the author points out that cynical types have suggested we treat all biomedical research as fraudulent unless proved otherwise. The cost is staggering: The U.S. wastes tens of billions of dollars annually on useless research, shortening or even costing patient lives. Most scientists can’t even reproduce their own data half the time, and the number of papers retracted rose to 10,000 in 2023 from 500 in 2010.

. . .

Most importantly, Dr. Szabo calls for systematic changes in how science gets done.

. . .

Above all, he despises the broken status quo, where “everybody acts politely . . . keeps their mouths shut, and acts like the whole process is functioning perfectly well.”

For the full review see:

Sam Kean. “Bookshelf; Reaching For Results.” The Wall Street Journal (Tuesday, March 24, 2025): A15.

(Note: ellipses between paragraphs added; ellipsis internal to paragraph, in original.)

(Note: the online version of the review was updated March 24, 2025, and has the title “Bookshelf; ‘Unreliable’: Reaching for Results.”)

The book under review is:

Szabo, Csaba. Unreliable: Bias, Fraud, and the Reproducibility Crisis in Biomedical Research. New York: Columbia University Press, 2025.

Both Homocysteine and Cholesterol Are Actionable Causes of Atherosclerosis

Alan Donagan taught a thought-provoking graduate course on Action Theory when I was a philosophy student at the University of Chicago in the mid to late 1970s. Some of the course related to how we think about causes in the social sciences and in policy debates.

Often we seek THE cause of what we want (or what we want to avoid). But most results have multiple causes. Which cause is most important, and so to some appears to be THE cause, depends largely on which cause is most easily actionable, which can change based on our knowledge or our constraints.

The obituary passages quoted below tell the sad story of how Kilmer McCully found that an amino acid called homocysteine was one cause of atherosclerosis, a cause that was actionable (could be countered) by eating foods containing various of the B vitamins. Kilmer’s career was canceled by powerful academics committed to the dominant view that cholesterol was THE cause of atherosclerosis.

McCully’s Harvard lab was moved to the basement, and eventually he was pressured out of Harvard.

Later studies, including the large, influential, and continuing Framingham study, eventually vindicated McCully’s claim.

We know the wrongly-cancelled pay a price for deviating from the dominant view. But how often do the cancellers pay a price for wrongly cancelling?

(p. B6) Kilmer S. McCully, a pathologist at Harvard Medical School in the 1960s and ’70s whose colleagues banished him to the basement for insisting — correctly, it turned out — that homocysteine, an amino acid, was being overlooked as a possible risk factor for heart disease, died on Feb. 21 [2025] at his home in Winchester, Mass. He was 91.

. . .

Dr. McCully didn’t think cholesterol should be ignored, but he thought it was malpractice to disregard the significance of homocysteine. His bosses at Harvard disagreed. First, they moved his lab below ground; then they told him to leave. He struggled to find work for years.

. . .

Presenting the case of homocystinuria in a 9-year-old girl, doctors mentioned that her uncle had died from a stroke in the 1930s, when he was 8 and had the same disease. “How could an eight-year-old have died the way old people do?” Dr. McCully wrote, with his daughter, in “The Heart Revolution” (1999).

When Dr. McCully tracked down the autopsy report and tissue samples, he was astounded: The boy had hardened arteries, but there was no cholesterol or fat in the plaque buildup. A few months later, he learned about a baby boy with homocystinuria who had recently died. He also had hardened arteries.

“I barely slept for two weeks,” he wrote.

In 1969, Dr. McCully published a paper about the cases in The American Journal of Pathology. The next year, in the same journal, he described what happened after he injected rabbits with high doses of homocysteine. “The aortas of all 13 of the animals injected with homocysteine were moderately thickened,” he wrote, “compared to the controls.”

. . .

The medical profession responded with “stony silence,” Dr. McCully told The Times.  . . .

. . .

“I felt for him, and I admired him,” J. David Spence, a professor emeritus at the University of Western Ontario who studies homocysteine, said in an interview. “He was neglected more than he ought to have been. It was sad.”

That began to change in the early 1990s, when large-scale, long-term studies of the risks for heart disease revealed that Dr. McCully had, in fact, been heading down the right path when Harvard relegated him to the basement.

. . .

As a teenager, Kilmer was enthralled by “Microbe Hunters,” Paul de Kruif’s 1926 book about Pasteur, Walter Reed, Robert Koch and others who investigated infectious diseases. He knew almost immediately that he wanted to become a scientist.

. . .

At a medical school reunion in 1999, his classmates presented him with a silver platter.

It was inscribed, “To Kim McCully, who saw the truth before the rest of us, indeed before the rest of medicine, and who would not be turned aside.”

For the full obituary see:

Michael S. Rosenwald. “Kilmer S. McCully Is Dead at 91; Fueled Debate on Heart Disease.” The New York Times (Monday, March 24, 2025): B6.

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

(Note: the online version of the obituary has the date March 21, 2025, and has the title “Kilmer McCully, 91, Dies; Pathologist Vindicated on Heart Disease Theory.”)

The book by McCully and his daughter, mentioned above, is:

McCully, Kilmer, and Martha McCully. Heart Revolution: The Vitamin B Breakthrough That Lowers Homocysteine Levels, Cuts Your Risk of Heart Disease, and Protects Your Health. New York: HarperCollins Publishers, 1999.

The book that inspired a teenage McCully to become a scientist is:

Kruif, Paul de. Microbe Hunters. New York: Harcourt, Brace and Company, 1926.

Healthcare Under ObamaCare’s “Affordable” Care Act Is Neither Popular Nor Affordable

In my Openness book, I argue that government regulations bind entrepreneurs and reduce innovation. As part of an antidote, I suggest that “sunset laws,” where regulations automatically expire, if not renewed. Later, at a small conference on Adam Thierer’s latest book, I was discouraged to hear a couple of participants grant the plausibility of the “antidote,” but report that in actual practice it does not work because almost all old regulations get renewed. Some hope returned when I read a report from James Broughel of a successful sunset review process:

(p. A17) Well, well. Progressives are at last acknowledging that ObamaCare is a failure. They aren’t doing so explicitly, of course, but their social-media screeds against insurers, triggered by last week’s murder of UnitedHealthcare CEO Brian Thompson, suggest as much. “We’ve gotten to a point where healthcare is so inaccessible and unaffordable, people are justified in their frustrations,” CBS News medical contributor Céline Gounder said during a Friday segment on the roasting of health insurers.

A Gallup survey released Friday [Dec. 6, 2024] affirms the sentiment, finding that only 44% of Americans rate U.S. healthcare good or excellent, down from 62% when Democrats passed ObamaCare in 2010. A mere 28% rate the country’s insurance coverage highly, an 11-point decline. ObamaCare may rank as the biggest political bait-and-switch in history.

Remember Barack Obama’s promise that if you like your health plan and doctor, you could keep them? Sorry. How about his claim that people with pre-existing conditions would be protected? Also not true. The biggest howler, however, was that healthcare would become more affordable.

Grant Democrats this: The law has advanced their political goal of expanding government control over insurers, in return for lavishing Americans with subsidies to buy overpriced, lousy products.

. . .

At the same time, ObamaCare’s perverse effects are fueling public rage against insurers and support for a single-payer system that would eliminate them. Mr. Obama and Peter Orszag, the law’s chief architect, must be smiling. Mr. Orszag, now CEO of the financial-services firm Lazard, has dined out on advising health insurers on mergers he says were spurred by the law’s regulations. How convenient.

. . .

If the goal were to help Americans with costly health conditions, it would have been far simpler and less expensive to boost subsidies for state high-risk pools. But that wouldn’t have accomplished Democrats’ actual goal, which is to turn insurers into de facto public utilities and jerry-rig a halfway house to single-payer healthcare. What a con.

For the full commentary see:

Allysia Finley. “Life Science; UnitedHealthcare and the ObamaCare Con.” The Wall Street Journal (Mon., Dec. 9, 2024): A17.

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

(Note: the online version of the commentary has the date December 8, 2024, and has the same title as the print version.)

The Gallup poll results mentioned above can be viewed at:

Brenan, Megan. “View of U.S. Healthcare Quality Declines to 24-Year Low.” Gallup, Inc., Dec. 6, 2024 [cited March 27, 2025]. Available from https://news.gallup.com/poll/654044/view-healthcare-quality-declines-year-low.aspx.

Doctors Burnout from Spending Less Time with Patients and More Time Arguing with Insurance Firms

Government policies have increased the paperwork that physicians must process and the time they must spend arguing with insurance companies on behalf of their patients. The policies have increased the need for back-office staff to handle the regulations, and so increased the overhead of private practice. So more and more physicians have given up private practice and become employees. They find their work less fulfilling and face burnout. Patients suffer when more of their physicians are bitter and burned-out.

(p. A1) There’s a question dividing the medical practice right now: Is being a doctor a job, or a calling?

. . .

(p. A2) Physicians work an average of 59 hours a week, according to the American Medical Association, and while the profession is well-compensated—the average physician makes $350,000, a recent National Bureau of Economic Research analysis found—it comes with high pressure and emotional strain.

. . .

Among physicians under age 45, only 32% own practices, down from 44% in 2012. By comparison, 51% of those ages 45 to 55 are owners.

Owners have more autonomy, but also increasing overhead costs. Vaughan, who sold his private practice in 2011, saw his malpractice insurance premiums increase to $65,000 a year.

Dr. Joseph Comfort, 80, sold his anesthesiology practice in 2003, frustrated by rising billing tussles with insurance companies. He now works part time as an internal medicine doctor at a small concierge clinic in Sanford, Fla.

“We’ve been ripped down off our pedestals,” he says.

For generations, Comfort says, doctors accepted being at the mercy of their pager and working long hours as the cost of doing business. “We took it because we considered ourselves to be masters of our own fate,” he says. “Now, everything’s changed. Doctors are like any other employee, and that’s how the new generation is behaving.”

They also spend far more time doing administrative tasks. One 2022 study found residents spent just 13% of their time in patient rooms, a factor many correlate with burnout.

. . .

In San Francisco, Dr. Christopher Domanski—a first-year resident who had his first child earlier this year—says he’s interested in pursuing a four-day workweek once he’s completed his training.

“I’m very happy to provide exceptional care for my patients and be there for them, but medicine has become more corporatized,” says Domanski, 29. Though he’s early in his medical career, he’s heard plenty of physicians complain about needing to argue with insurance companies to get their patients the treatments they need.

“It’s disheartening,” he says.

For the full story see:

Te-Ping Chen. “Younger Doctors Balk at Medicine’s Workaholic Culture.” The Wall Street Journal (Monday, Nov 04, 2024 [sic]): A1-A2.

(Note: ellipses added.)

(Note: the online version of the story has the date November 3, 2024 [sic], and has the title “Young Doctors Want Work-Life Balance. Older Doctors Say That’s Not the Job.”)

Vindication is Sweet, Even 60 Years Too Late

When I was a child my mother would stick an oral thermometer in my mouth. When she returned she would always be annoyed with me, saying that I didn’t have it in right, because my temperature was too low. She would say with irritation: ‘Now this time do it right!’ So I would feel discouraged and would give the thermometer a hard jab into my mouth until it hurt. But my temperature would still be too low.

The story below suggests, decades too late for me, that maybe it wasn’t my fault. Maybe the official mandated “normal” temperature of 98.6 was wrong!

(p. D6) We seem to be getting cooler. Since 1851, when the standard was set at 37 degrees centigrade, or 98.6 Fahrenheit, the average human body temperature has steadily declined.

. . . . The analysis is in eLife.

. . .

. . . improvements in sanitation and improved dental and medical care have reduced chronic inflammation, and the constant temperatures maintained by modern heating and air conditioning have helped lower resting metabolic rates. Today, a temperature of 97.5 may be closer to “normal” than the traditional 98.6.

For the full story see:

Nicholas Bakalar. “Is 98.6 No Longer ‘Normal’?” The New York Times (Tuesday, January 21, 2020): D6.

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

(Note: the online version of the story was updated Jan. 21, 2020 [sic], and has the title “Body Temperature 2.0: Do We Need to Rethink What’s Normal?”)

The academic paper in eLife, mentioned above, is:

Protsiv, Myroslava, Catherine Ley, Joanna Lankester, Trevor Hastie, and Julie Parsonnet. “Decreasing Human Body Temperature in the United States since the Industrial Revolution.” eLife 9 (2020): e49555.

See also:

Dana G. Smith. “We Are Running Cooler, on Average.” The New York Times (Tues., October 17, 2023): D7.

“Practice-Changing” Cancer Advance Adds Only 10 Months of Life

The subheadline of this article gushed that this drug “tripled life expectancy,” the article quoted one expert gushing that it was “a light after a long time,” and another expert gushing that it “will be practice-changing.”

Then you read more carefully and see that the average recipient of the drug has a gain in life expectancy from about four and a half months without the drug to 14 months with the drug–in other words a gain of only roughly 10 months, and with the major side effect of cytokine syndrome.

This illustrates the discouraging side of many ballyhooed cancer advances, they amount to only months of added life, and the added life comes at the cost of major side effects.

(p. D4) The Food and Drug Administration on Thursday [May 16, 2024] approved an innovative new treatment for patients with a form of lung cancer. It is to be used only by patients who have exhausted all other options to treat small cell lung cancer, and have a life expectancy of four to five months.

The drug tarlatamab, or Imdelltra, made by the company Amgen, tripled patients’ life expectancy, giving them a median survival of 14 months after they took the drug. Forty percent of those who got the drug responded.

After decades with no real advances in treatments for small cell lung cancer, tarlatamab offers the first real hope, said Dr. Anish Thomas, a lung cancer specialist at the federal National Cancer Institute who was not involved in the trial.

“I feel it’s a light after a long time,” he added.

Dr. Timothy Burns, a lung cancer specialist at the University of Pittsburgh, said that the drug “will be practice-changing.”

(Dr. Burns was not an investigator in the study but has served on an Amgen advisory committee for a different drug.)

The drug, though, has a side effect that can be serious — cytokine release syndrome. It’s an overreaction of the immune system that can result in symptoms like a rash, a rapid heartbeat and low blood pressure.

For the full story see:

Kolata, Gina. “Drug Approved for a Stubbornly Deadly Cancer.” The New York Times (Tuesday, May 21, 2024 [sic]): D4.

(Note: bracketed date added.)

(Note: the online version of the story has the date May 16, 2024 [sic], and has the title “F.D.A. Approves Drug for Persistently Deadly Form of Lung Cancer.”)

Public Health “Experts” Rebuffed Renegades Who Saw Covid Spread in Aerosols

Steven Johnson’s The Ghost Map shows how rigid adherence to the miasma theory of disease shut out alternatives. And an alternative was indeed needed to explain the spread of cholera. But the defeat of the miasma theory for cholera may have been too complete, prejudicing scientists to oppose theories of disease-spread through the air, which turn out to be important for some diseases, such as Covid-19.

(p. C9) In early 2020, as word spread of a frightening new respiratory outbreak in China, the World Health Organization and the Centers for Disease Control and Prevention were pressed for advice. Both initially counseled social distancing, guided by the assumption that the disease was spread by large, boggy droplets that fell rapidly to the ground after being expelled by coughing or sneezing.

By avoiding such projectiles and keeping surfaces clean, the reasoning went, infection could be avoided. Yet this advice ignored—with tragic consequences—nearly a century of science suggesting that many respiratory diseases can spread via microdrops that are exhaled during normal breathing and can remain suspended in the air for hours.

In “Air-Borne,” the New York Times science writer Carl Zimmer seeks to explain how public-health officials could have overlooked such an important mechanism of the Covid-19 contagion. He begins his meticulous history with the ancient Greek physician Hippocrates, who taught that illness could be caused by “an invisible corruption of the air,” which he termed a “miasma.”

. . .

While the field of aerobiology may have entered the new millennium stuck on a “stagnant plateau,” as one journal article lamented, hope was starting to emerge. Advances in technology led to a more complete characterization of the aerobiome. A range of scientists from around the world, meanwhile, re-examined the possibility of airborne transmission and discovered the evidence against it wanting.

Following the emergence of Covid-19, many of these researchers were appalled by the seemingly reflexive—“mind-boggling,” in the words of one scientist—rejection of airborne transmission by public-health agencies. At first, these renegades individually struggled to have their work published but were largely rebuffed.

After an early Covid-19 outbreak among a choir in Washington state was initially attributed to large-droplet spread, a more detailed analysis by a unified group of skeptical researchers suggested that airborne transmission was far more likely. On Dec. 23, 2021—nearly 21 months after tweeting “FACT: #COVID19 is NOT airborne”—the WHO “finally issued a clear public statement that the virus was airborne,” Mr. Zimmer writes. A triumph for persistent scientists, perhaps, but also a pointed reminder of the complexity, fragility and deeply human dependencies of evolving science.

For the full review see:

David A. Shaywitz. “Microbes in the Mist.” The Wall Street Journal (Saturday, March 15, 2025): C9.

(Note: ellipsis added.)

(Note: the online version of the review has the date March 14, 2025, and has the title “‘Air-Borne’: The Microbes in the Mist.”)

The book under review is:

Zimmer, Carl. Air-Borne: The Hidden History of the Life We Breathe. New York: Dutton, 2025.

E. Coli in Organically Grown Carrots Kill One and Sicken 39

Many years ago, when ConAgra was still headquartered in Omaha, I heard a speech by the then-C.E.O. in which he argued that big food producers have processes in place to make food safer than the processes often used by smaller independent local and organic farmers. Stories of contamination of organic food do not prove, but are consistent with, his argument. For instance it was reported in 2024 that organic carrots contaminated with E. Coli killed one person and made 39 others sick. Trader Joe’s was one of the stores where contaminated carrots were sold.

E. Coli in organically grown carrots was reported in:

Johnny Diaz. “Organic Carrots Behind Outbreak of E. Coli; 39 Sickened, 1 Dead.” The New York Times (Tues., November 19, 2024): B5.

(Note: the online version of the article was updated Nov. 18, 2024, and has the title “1 Dead and Dozens Ill in E. Coli Outbreak Linked to Organic Carrots.”)

A Fluoridate Water “Warning Sign”

When I was a child, my family opposed the government-mandated fluoridation of the water supply of South Bend, Indiana, my hometown. We were not anti-science. We believed fluoride was a poison and that individual citizens had a right to make their own judgement whether to consume it. I still believe we were right.

(p. D6) A small study published Monday [May 20, 2024] suggested that higher levels of fluoride consumed during the third trimester of pregnancy were associated with a greater risk of behavioral problems in the mothers’ children at 3 years old. The authors of the study, which was funded in part by the National Institutes of Health and the Environmental Protection Agency and published in the journal JAMA Network Open, believe it is the first to examine links between prenatal fluoride exposure and child development among families living in the United States, where fluoride is often added to community water supplies to prevent dental cavities.

The study’s authors and some outside researchers said that the findings should prompt policymakers to evaluate the safety of fluoride consumption during pregnancy.

“I think it’s a warning sign,” said Dr. Beate Ritz, an environmental epidemiologist at the U.C.L.A. Fielding School of Public Health.

For the full story, see:

Alice Callahan and Christina Caron. “Fluoridated Water and Pregnancy.” The New York Times (Tuesday, May 28, 2024): D6.

(Note: bracketed date added.)

(Note: the online version of the story has the date May 22, 2024, and has the title “Is Fluoridated Drinking Water Safe for Pregnant Women?”)

The small published study mentioned above is:

Malin, Ashley J., Sandrah P. Eckel, Howard Hu, E. Angeles Martinez-Mier, Ixel Hernandez-Castro, Tingyu Yang, Shohreh F. Farzan, Rima Habre, Carrie V. Breton, and Theresa M. Bastain. “Maternal Urinary Fluoride and Child Neurobehavior at Age 36 Months.” JAMA Network Open 7, no. 5 (2024): e2411987-e87.

Off-Label Drug Use Shows F.D.A. Phase 3 Trials Could Be Dropped, Adding New Cures and Lowering Costs

The F.D.A. allows physicians to prescribe drugs for “off-label” use. These drugs were originally approved for a different “on-label” use. For that approval the drugs had to pass through Phase 1 and Phase 2 clinical trials, mainly to show safety, and massively expensive Phase 3 clinical trials to show efficacy for the on-label use.

When off-label use is allowed, that shows that the F.D.A. is accepting drugs for a use where efficacy has NOT been shown.

This is a proof of concept for my suggestion (that I originally heard from Nobel-Prize-winner Milton Friedman) that F.D.A. regulation should be pared back to just Phase 1 and Phase 2, for safety. Since the Phase 3 trials are usually far more expensive than the Phase 1 and Phase 2 combined, this would allow far more new drugs to be developed.

If the development of new drugs was cheaper, Fajgenbaum and others would not need to spend N.I.H.’s 48 millions of taxpayer dollars to comb through already-approved drugs to see if one can be jury-rigged as a therapy for a different disease.

(p. A6) [Dr. David Fajgenbaum, an immunologist at the University of Pennsylvania and . . . Castleman patient who studies the disease] . . . has matched rare-disease patients with drugs that are already in pharmacies for other conditions for over 10 years, starting with himself.

. . .

Every Cure, a nonprofit Fajgenbaum helped found in 2022, received funding on Wednesday [Feb. 28, 2024] that could surpass $48 million from the federal Advanced Research Projects Agency for Health. Fajgenbaum and his team will spend the money to build a drug-repurposing database and algorithms that patients, doctors and researchers can use to find drugs for untreated diseases.

There are over 10,000 known rare diseases and most don’t have a drug approved to treat them. The FDA said it has approved over 19,000 prescription drugs.

The notion of finding new uses for existing drugs has been around for a long time. Once the FDA approves a drug, doctors can prescribe it off-label to patients with other conditions they think it will help. Ozempic, originally approved for people with Type 2 diabetes, is now used by millions of people without the disease for weight loss.

The National Institutes of Health and research institutions have invested over the years in drug repurposing, hoping it would be faster and less costly to find new uses for drugs that have already made it to market, a process that can take more than a decade and cost $2 billion. But systematically matching approved treatments to unmet needs has been hard.

. . .

“Our end goal is not FDA approval. Our end goal is giving patients maximum access to medications,” said Tracey Sikora, co-founder of Every Cure.

For the full story see:

Amy Dockser Marcus. “Repurposed Drugs Give People With Rare Diseases New Hope.” The Wall Street Journal (Thursday, Feb. 29, 2024 [sic]): A6.

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

(Note: the online version of the story has the date February 28, 2024 [sic], and has the title “This Doctor Found His Own Miracle Drug. Now He Wants to Do It for Others.”)

For more on Fajgenbaum’s story, read his autobiographical account:

Fajgenbaum, David. Chasing My Cure: A Doctor’s Race to Turn Hope into Action; a Memoir. New York: Ballantine Books, 2019.