Category: Health

Double-Blind Trials Are Not the Only Source of Sound Knowledge

(p. 1) . . . while all doctors agree about the importance of gauging the quality of evidence, many feel that a hierarchy of methods is simplistic. As the doctor Mark Tonelli has argued, distinct forms of knowledge can’t be judged by the same standards: what a patient prefers on the basis of personal experience; what a doctor thinks on the basis of clinical experience; and what clinical research has discovered — each of these is valuable in its own way. While scientists concur that randomized trials are ideal for evaluating the average effects of treatments, such precision isn’t necessary when the benefits are obvious or clear from other data.
Clinical expertise and rigorous evaluation also differ in their utility at different stages of scientific inquiry. For discovery and explanation, as the clinical epidemiologist Jan Vandenbroucke has argued, practitioners’ instincts, observations and case studies are most useful, whereas randomized controlled trials are least useful. Expertise and systematic evaluation are partners, not rivals.
Distrusting expertise makes it easy to confuse an absence of randomized evaluations with an absence of knowledge. And this leads to the false belief that knowledge of what works in social policy, education or fighting terrorism can come only from randomized evaluations. But by that logic (as a spoof scientific article claimed), we don’t know if parachutes really work because we have no randomized controlled trials of them.

For the full commentary, see:
PAGAN KENNEDY. “The Thin Gene.” The New York Times, SundayReview Section (Sun., NOV. 27, 2016): 1 & 6.
(Note: ellipsis added.)
(Note: the online version of the commentary has the date NOV. 25, 2016.)

The academic article calling for double-blind randomized trials to establish the efficacy of parachutes, is:
Smith, Gordon C. S., and Jill P. Pell. “Parachute Use to Prevent Death and Major Trauma Related to Gravitational Challenge: Systematic Review of Randomised Controlled Trials.” BMJ 327, no. 7429 (Dec. 18, 2003): 1459-61.

Science Can Learn Much from Outliers “Who Are Naturally Different”

(p. 1) Abby Solomon suffers from a one-in-a-billion genetic syndrome: After just about an hour without food, she begins to starve. She sleeps in snatches. In her dreams she gorges on French fries. But as soon as she wakes up and nibbles a few bites, she feels full, so she ends up consuming very few calories. At 5 feet 10 inches tall, she weighs 99 pounds.
Now 21 years old, she is one of the few people in the world to survive into adulthood with neonatal progeroid syndrome, a condition that results from damage to the FBN1 gene.
. . .
(p. 6) Dr. Chopra told me that, as far as medical science is concerned, Abby Solomon is worth thousands of the rest of us.
. . .
“Nothing comes close to starting with people who are naturally different,” he said. This is why he searches out patients at the extreme ends of the spectrum — those who are wired to weigh 80 pounds or 380 pounds. He said, “We have the opportunity to help a bigger swath of humanity when we learn from these outliers.”
In 2013, after hearing about Ms. Solomon’s unusual condition from another patient, he asked her to visit his clinic. Ms. Solomon warned him that she would be able to carry on a conversation for only 15 minutes before she needed to snack on chips or a cookie. That remark inspired a revelation. Dr. Chopra realized that “she had to eat small, sugary meals all day to stay alive, because her body was constantly running out of glucose,” he said.
The clue led Dr. Chopra and his colleagues to their discovery of the blood-sugar-regulating hormone, which they named asprosin. Ms. Solomon’s natural asprosin deficiency keeps her on the brink of starvation, but Dr. Chopra’s hope is that an artificial compound that blocks asprosin could be used as a treatment for obesity. He and his team have already tested such a compound on mice, and found that it can reverse insulin resistance and weight gain.

For the full commentary, see:
PAGAN KENNEDY. “The Thin Gene.” The New York Times, SundayReview Section (Sun., NOV. 27, 2016): 1 & 6.
(Note: ellipses added.)
(Note: the online version of the commentary has the date NOV. 25, 2016.)

“Patients Should Be the Owners of Their Own Medical Data”

(p. A21) THERE’S quite a paradox when it comes to our health data. Most of us still cannot readily look at it, but there’s been an epidemic of cybercriminals and thieves hacking and stealing this most personal information.
. . .
. . . , giving consumers control of their own medical data would revolutionize who owns medical data and how it is used. Concerns about researchers losing access to this amassed data are overstated. Patients have shown an overwhelming willingness to share their information for altruistic reasons (which far exceeds the track record of doctors and health systems when it comes to sharing data).
. . .
We need to move on from the days of health systems storing and owning all our health data. Patients should be the owners of their own medical data. It’s an entitlement and civil right that should be recognized.

For the full commentary, see:
KATHRYN HAUN and ERIC J. TOPOL. “The Health Data Conundrum.” The New York Times (Tues., January 3, 2017): A21.
(Note: ellipses added.)
(Note: the online version of the commentary has the date January 2, 2017.)

British Government Ignored Scurvy Cure

(p. C14) Scurvy, we know today, has a single and simple cause: lack of vitamin C. But between the years 1500 and 1800, when an estimated two million sailors died from the disease, it seemed to defy all logic.
. . .
The conventional medical narrative holds that the mystery was solved by James Lind’s announcement, in his “Treatise of the Scurvy” (1753), that it could be cured by drinking lemon juice. But in “Scurvy: The Disease of Discovery,” Jonathan Lamb, a professor at Vanderbilt University, shows that the story is nowhere near so simple and that scurvy was a much stranger condition than we imagine, with effects on the mind that neuroscience is only now beginning to elucidate. The result is a book that renders a familiar subject as exotic and uncanny as the tropical shores that confronted sailors in the grip of scurvy’s delirium.
James Lind was not the first person to recommend the lemon-juice cure. Contemporaries of Francis Drake had discovered it 150 years before, but the secret was lost and found again many times over the centuries. Some citrus juices were much more effective than others, and their efficacy was reduced considerably when they were preserved by boiling. The British admiralty ignored Lind’s researches, . . .

For the full review, see:
MIKE JAY. “The Disease of the Enlightenment.” The Wall Street Journal (Sat., December 10, 2016): C14.
(Note: ellipses added.)
(Note: the online version of the review has the date Dec. 9, 2016, and has the title “Scurvy: The Disease of the Enlightenment.”)

The book under review, is:
Lamb, Jonathan. Scurvy: The Disease of Discovery. Princeton, NJ: Princeton University Press, 2017.

Superagers Engage in “Strenuous Mental Effort”

(p. 10) Why do some older people remain mentally nimble while others decline? “Superagers” (a term coined by the neurologist Marsel Mesulam) are those whose memory and attention isn’t merely above average for their age, but is actually on par with healthy, active 25-year-olds.
. . .
Of course, the big question is: How do you become a superager? Which activities, if any, will increase your chances of remaining mentally sharp into old age? We’re still studying this question, but our best answer at the moment is: work hard at something. Many labs have observed that these critical brain regions increase in activity when people perform difficult tasks, whether the effort is physical or mental. You can therefore help keep these regions thick and healthy through vigorous exercise and bouts of strenuous mental effort. My father-in-law, for example, swims every day and plays tournament bridge.
The road to superaging is difficult, though, because these brain regions have another intriguing property: When they increase in activity, you tend to feel pretty bad — tired, stymied, frustrated. Think about the last time you grappled with a math problem or pushed yourself to your physical limits. Hard work makes you feel bad in the moment. The Marine Corps has a motto that embodies this principle: “Pain is weakness leaving the body.” That is, the discomfort of exertion means you’re building muscle and discipline. Superagers are like Marines: They excel at pushing past the temporary unpleasantness of intense effort. Studies suggest that the result is a more youthful brain that helps maintain a sharper memory and a greater ability to pay attention.

For the full commentary, see:
LISA FELDMAN BARRETT. “Gray Matter; How to Become a ‘Superager’.” The New York Times, SundayReview Section (Sun., January 1, 2017): 10.
(Note: ellipsis added.)
(Note: the online version of the commentary has the date DEC. 31, 2016.)

The passages quoted above are related to Barrett’s academic paper:
Sun, Felicia W., Michael R. Stepanovic, Joseph Andreano, Lisa Feldman Barrett, Alexandra Touroutoglou, and Bradford C. Dickerson. “Youthful Brains in Older Adults: Preserved Neuroanatomy in the Default Mode and Salience Networks Contributes to Youthful Memory in Superaging.” The Journal of Neuroscience 36, no. 37 (Sept. 14, 2016): 9659-9668.

E.U. Regulations Protect Paris Rats

(p. A4) PARIS — On chilly winter mornings, most Parisians hurry by the now-locked square that is home to the beautiful medieval Tour St. Jacques. Only occasionally do they pause, perhaps hearing a light rustle on the fallen leaves or glimpsing something scampering among the dark green foliage.
A bird? A cat? A puppy?
No. A rat.
No. Three rats.
No. Look closer: Ten or 12 rats with lustrous gray-brown coats are shuffling among the dried autumn leaves.
Paris is facing its worst rat crisis in decades. Nine parks and green spaces have been closed either partly or entirely
. . .
In the 19th century, rats terrified and disgusted Parisians who knew that five centuries earlier, the creatures had brought the bubonic plague across the Mediterranean.
The plague ravaged the city, as it did much of Europe, killing an estimated 100,000 Parisians, between a third and half the population at the time. It recurred periodically for four more centuries. Not surprisingly, the experience left Paris with a millennium-long aversion to rodents.
. . .
. . . why are they proliferating? Could it be everybody’s favorite scapegoat — the European Union and its faceless, unaccountable bureaucrats?
Yes, it could.
New regulations from Brussels, the European Union’s headquarters, have forced countries to change how they use rat poison, said Dr. Jean-Michel Michaux, a veterinarian and head of the Urban Animals Scientific and (p. A14) Technical Institute in Paris.
. . .
While the poison could be a risk to human beings, so are the rats — potentially, although no one is suggesting that the bubonic plague is likely to return.

For the full story, see:

ALISSA J. RUBIN. “PARIS JOURNAL; The Rats Came Back. Blame the E,U.” The New York Times (Fri., DEC. 16, 2016): A4 & A14.
(Note: ellipses added.)
(Note: the online version of the story has the date DEC. 15, 2016, and has the title “PARIS JOURNAL; Rodents Run Wild in Paris. Blame the European Union.”)

Failed “War on Cancer” Gets Repackaged as “Moonshot”

(p. A15) Last Friday [January 8, 2016] a group of 15 cancer researchers cut short a meeting at the Food and Drug Administration. The reason: They had been invited to Vice President Joseph R. Biden’s office to discuss his “moonshot” to cure cancer.
. . .
The idea that a concerted government push can lead to a “cure” for cancer is nearly a half century old, stretching back to President Nixon’s failed “War on Cancer.” The latest, which President Obama formalized in his State of the Union address on Tuesday, has a deeply emotional tinge. Mr. Biden’s son Beau died of brain cancer in May, and the vice president’s very public mourning and call for a “national commitment to end cancer as we know it” as he announced his decision not to run for president has moved and captivated Washington.
. . .
Unlike in 1971, when President Nixon launched his cancer war, researchers now understand that cancer is not one disease but essentially hundreds. The very notion of a single cure — or as Mr. Obama put it, making “America the country that cures cancer once and for all” — is misleading and outdated.
“Cancer is way more complex than anyone had imagined in 1970,” said Dr. Jose Baselga, the president of the American Association for Cancer Research and physician in chief and chief medical officer at Memorial Sloan Kettering Cancer Center.
. . .
Commitments by powerful Washington figures to cure cancer seem to come along about every decade.
Dr. Andrew von Eschenbach, the director of the National Cancer Institute, announced in 2003 that his organization’s goal was to “eliminate suffering and death” caused by cancer by 2015.
During an appropriations hearing, Dr. von Eschenbach got into a public bargaining session with Senator Arlen Specter, then a Republican from Pennsylvania, about how much money Dr. von Eschenbach would need to advance the date of the cure.
“I asked you what it would take to move that date up to 2010,” Mr. Specter asked.
“We have proposed a budget that would support those initiatives that would amount to approximately $600 million a year,” Dr. von Eschenbach answered.
“Six-hundred million a year?” Mr. Specter asked. “And you can move the date from 2015 to 2010?”
“Yes, sir,” Mr. von Eschenbach said.
Mr. Specter died of cancer in 2012.

For the full story, see:
GINA KOLATA and GARDINER HARRIS. “‘Moonshot’ to Cure Cancer, to Be Led by Biden, Relies on Outmoded View of Disease.” The New York Times (Thurs., JAN. 14, 2016): A15.
(Note: ellipses, and bracketed date, added.)
(Note: the online version of the story has the date JAN. 13, 2016.)

Prehistoric Hunter Suffered from Ulcer-Causing Microbe

(p. A7) Microbes that once troubled the stomach of a prehistoric hunter known as “Otzi the Iceman,” who died on an Alpine glacier 5,300 years ago, are offering researchers a rare insight into the early settlement of Europe.
In findings reported Thursday [January 7, 2016] in Science, an international research group analyzed remnants of ulcer-causing microbes called Helicobacter pylori exhumed from the well-preserved mummy of the Neolithic nomad. With modern DNA sequencing technology, they reconstructed the genetic structure of this ancient microbe–the oldest known pathogen sequenced so far.
. . .
“We know he had a rough lifestyle,” said Frank Maixner at the European Academy Institute for Mummies and the Iceman in Bolzano, Italy, who led the team of 23 scientists. “We found a lot of pathological conditions.”
. . .
The researchers also determined that the bacteria had inflamed his stomach lining, indicating that the prehistoric hunter, fleeing into the icy highlands where he was shot in the back with an arrow and beaten, may have been feeling ill on the day he was murdered.

For the full story, see:
ROBERT LEE HOTZ. “Iceman’s Gut Sheds Light on Human Migration.” The Wall Street Journal (Fri., Jan. 8, 2016): A7.
(Note: ellipses, and bracketed date, added.)
(Note: the online version of the story has the date Jan. 7, 2016, and has the title “Otzi the Iceman’s Stomach Sheds Light on Copper-Age Migration to Europe.”)

The research summarized in the passages quoted above, was more fully reported in:
Maixner, Frank, Ben Krause-Kyora, Dmitrij Turaev, Alexander Herbig, Michael R. Hoopmann, Janice L. Hallows, Ulrike Kusebauch, Eduard Egarter Vigl, Peter Malfertheiner, Francis Megraud, Niall O’Sullivan, Giovanna Cipollini, Valentina Coia, Marco Samadelli, Lars Engstrand, Bodo Linz, Robert L. Moritz, Rudolf Grimm, Johannes Krause, Almut Nebel, Yoshan Moodley, Thomas Rattei, and Albert Zink. “The 5300-Year-Old Helicobacter pylori Genome of the Iceman.” Science 351, no. 6269 (Jan. 8, 2016): 162-65.

Serendipitous Discoveries “Happen in Medicine All the Time”

(p. 18) In the late 1950s, Dr. Jude was a resident at the Johns Hopkins University School of Medicine in Baltimore, experimenting with induced hypothermia as a way to stop blood flow to the heart by cooling it down and allowing surgical procedures to be performed without fatal loss of blood.
In experiments with rats, he found that hypothermia often caused cardiac arrest, a problem that two electrical engineers down the hall were addressing in experimental work on dogs, using a defibrillator to send electrical shocks to the heart. William Kouwenhoven, the inventor of a portable defibrillator, and G. Guy Knickerbocker, a doctoral student, had seen that the mere weight of the defibrillator paddles stimulated cardiac activity when pressed against a dog’s chest.
Dr. Jude immediately saw the potential for human medicine and began working with the two men.
In July 1959, when a 35-year-old woman being anesthetized for a gall bladder operation went into cardiac arrest, Dr. Jude, instead of using the standard technique of opening the chest and massaging the heart directly, applied rhythmic, manual pressure.
“Her blood pressure came back at once,” he recalled. “We didn’t have to open up her chest. They went ahead and did the operation on her, and she recovered completely.”
. . .
Dr. Jude played down his importance in developing CPR, a breakthrough that The Journal of the American Medical Association had recently compared to the discovery of penicillin.
“It was just serendipity — being in the right place at the right time and working on something for which there was an obvious need,” he told the alumni newsletter of the University of St. Thomas in 1984. “Things like that happen in medicine all the time.”

For the full obituary, see:
WILLIAM GRIMES. “Dr. James Jude Dies at 87; Helped Develop Use of CPR.” The New York Times, First Section (Sun., AUG. 2, 2015): 18.
(Note: ellipsis added.)
(Note: the online version of the obituary has the date AUG. 1, 2015, and has the title “Dr. James Jude, Who Helped Develop Use of CPR, Dies at 87.”)

Never Say Die

(p. A7) LONDON — During the last months of her life, a terminally ill 14-year-old British girl made a final wish. Instead of being buried, she asked to be frozen so that she could be “woken up” in the future when a cure was found — even if that was hundreds of years later.
“I want to have this chance,” the teenager wrote in a letter to a judge asking that she be cryogenically preserved. She died on Oct. 17 from a rare form of cancer. “I don’t want to be buried underground,” she wrote.
The girl’s parents, who are divorced, disagreed about the procedure. The teenager had asked the court to designate that her mother, who supported her daughter’s wishes, should decide how to handle her remains.
The judge, Peter Jackson, ruled in her favor. Local news reports said he was impressed by the “valiant way in which she was facing her predicament.” He said she had chosen the most basic preservation option, which costs about £37,000, or nearly $46,000, an amount reportedly raised by her grandparents.
“I want to live and live longer and I think that in the future they might find a cure for my cancer and wake me up,” the teenager wrote in her letter to the judge. Local reports said she had told a relative: “I’m dying, but I’m going to come back again in 200 years.”
. . .
“The scientific theory underlying cryonics is speculative and controversial, and there is considerable debate about its ethical implications,” the judge said in a statement.
“On the other hand, cryopreservation, the preservation of cells and tissues by freezing, is now a well-known process in certain branches of medicine, for example the preservation of sperm and embryos as part of fertility treatment,” the statement said. “Cryonics is cryopreservation taken to its extreme.”
Zoe Fleetwood, the girl’s lawyer, said her client had called Judge Jackson a “hero” after being told of the court’s decision shortly before her death. “By Oct. 6, the girl knew that her wishes were going to be followed,” Ms. Fleetwood told BBC Radio 4. “That gave her great comfort.”

For the full story, see:
KIMIKO DE FREYTAS-TAMURA. “Wish of Girl, 14, to Be Frozen, Is Granted by British Judge.” The New York Times (Sat., NOV. 19, 2016): A7.
(Note: ellipsis added.)
(Note: the online version of the story has the date NOV. 18, 2016, and has the title “Last Wish of Dying Girl, 14, to Be Frozen, Is Granted by Judge.”)

Uncredentialed Loner Saved Lives with Respirator Invention

(p. B9) When the fraternity of inventors celebrate the geniuses who came up with super glue, kitty litter and the cellphone, they sometimes talk about Dr. Bird, an American original who began tinkering with gizmos concocted out of strawberry-shortcake tins and doorknobs and eventually developed four generations of cardiopulmonary devices that came to be widely used in homes and hospitals.
. . .
Dr. Bird was inducted into the National Inventors Hall of Fame in 1995 for developing the first low-cost, mass-produced pediatric respirator, known as the Baby Bird, which has been credited by medical experts with significantly reducing the mortality rates of infants with respiratory problems.
The device, he said, saved two Idaho neighbor boys born with breathing distress. Among those aided by his inventions was his first wife, Mary, who learned she had pulmonary emphysema in 1964; his respirators, including one that used percussion to loosen secretions in her lungs, helped prolong her life until 1986.
Dr. Bird, who received the Presidential Citizens Medal from George W. Bush in 2008 and the National Medal of Technology and Innovation from President Obama in 2009, lived a self-contained but busy life on a remote, 300-acre compound on Lake Pend Oreille, surrounded by majestic mountains and forests 50 miles from the Canadian border.
On the estate was his home; the headquarters of his Percussionaire Corporation, with dozens of employees who develop and market his inventions; a working farm that sustained all the residents; an airfield and hangars for his scores of restored vintage airplanes, seaplanes, helicopters, cars and motorcycles; and the Bird Aviation Museum and Invention Center, which he opened in 2007.
. . .
His first prototype, cobbled together from shortcake tins and a doorknob in 1953, was revised often and tested on volunteer patients with limited success. But in 1958, he introduced the Bird Universal Medical Respirator, a green box that reliably assisted breathing and sold widely to patients and hospitals. He later developed improved versions, as well as his Baby Bird ventilator.
Much of Dr. Bird’s formal higher education came after his successful inventions. His curriculum vitae includes a doctorate in aeronautics in 1977 from Northrop University in Inglewood, and a medical degree in 1979 from the Pontifical Catholic University of Campinas in Brazil.

For the full obituary, see:
ROBERT D. McFADDEN. “Forrest M. Bird, Inventor of Respirators, Dies at 94.” The New York Times (Tues., AUG. 4, 2015): B9.
(Note: ellipses added.)
(Note: the online version of the obituary has the date AUG. 3, 2015, and has the title “Dr. Forrest Bird, Inventor of Medical Respirators and Ventilators, Dies at 94.”)