Category: Health

Pursuit of Slow Hunch Pays Off with Flu Drug

(p. B3) As Americans suffer through the worst influenza outbreak in almost a decade, a Japanese drugmaker says it has developed a pill that can kill the virus within a day.
. . .
“The data that we’ve seen looks very promising,” said Martin Howell Friede, who leads the World Health Organization’s advisory on vaccines, including for influenza. “This could be a breakthrough in the way that we treat influenza.”
. . .
Shionogi scientists began researching a novel flu drug more than a decade ago, shelving almost 2,500 compounds in the process. Then, the 140-year-old Osaka company, which has created blockbuster drugs used to treat HIV and high cholesterol, had a breakthrough.
Shionogi scientists knew from their research that an anti-HIV drug the company had developed with a joint venture of Pfizer Inc. and GlaxoSmithKline Co. worked by blocking a metallic enzyme that HIV uses as a weapon to hijack human cells. They found the flu virus was also exploiting a metallic enzyme.
“So we said, ‘why don’t we build on our HIV knowledge to find a way to treat the flu?’ And we did,” said Takeki Uehara, who led the compound’s development.

For the full story, see:
Preetika Rana. “Drugmaker: Pill Kills Flu in a Day.” The Wall Street Journal (Monday, Feb. 12, 2018): B3.
(Note: ellipses added.)
(Note: the online version of the article has the date Feb. 10, 2018, and has the title “Experimental Drug Promises to Kill the Flu Virus in a Day.”)

FDA Regulations Stop Vape Shop Innovations

(p. A19) After Kimberly Manor lost her husband to lung cancer, she was inspired to make a dramatic career change. Kimberly now owns and operates Moose Jooce in Lake, Mich., a “vape shop” that sells various electronic nicotine devices. These products use battery-powered coils to vaporize liquids, with differing levels of nicotine or none at all. Thus, vapers may inhale nicotine without the tar or other harmful chemicals in tobacco smoke, since there is no tobacco and no combustion. Scientific evidence suggests this is a much safer alternative to smoking.
Ms. Manor estimates that her business has helped more than 500 people quit smoking, most of them longtime smokers in their 50s or older. Yet the Food and Drug Administration is discouraging more such enterprises. In a regulation issued in 2016 known as the “deeming rule,” the agency ordered that vaping products would be subject to the same regulations developed for the cigarette industry under the Tobacco Control Act of 2009.
The deeming rule has been devastating to businesses like Ms. Manor’s. To give just one example, vape shop owners frequently experiment by mixing new flavors for the liquid “juice.” Now, each separate creation requires its own prohibitively expensive application for FDA approval, which means that vape shops have been forced to stop innovating.

For the full commentary, see:
Todd Gaziano and Tommy Berry, “Career Civil Servants Illegitimately Rule America; Leslie Kux has never been elected or confirmed by the Senate. She’s issued nearly 200 regulations.” The Wall Street Journal (Thursday, March 1, 2018): A19.
(Note: the online version of the commentary has the date Feb. 28, 2018.)

Proof of Concept for Regenerating Limbs and Internal Organs

(p. D3) Scientists have decoded the genome of the axolotl, the Mexican amphibian with a Mona Lisa smile. It has 32 billion base pairs, which makes it ten times the size of the human genome, and the largest genome ever sequenced.
The axolotl, endangered in the wild, has been bred in laboratories and studied for more than 150 years. It has the remarkable capacity to regrow amputated limbs complete with bones, muscles and nerves; to heal wounds without producing scar tissue; and even to regenerate damaged internal organs.
This salamander can heal a crushed spinal cord and have it function just like it did before it was damaged. This ability, which exists to such an extent in no other animal, makes its genes of considerable interest.
. . .
The researchers have identified some of the genes involved in regeneration, and some genes that exist only in the axolotl, but there is much work still to be done.

For the full story, see:
NICHOLAS BAKALAR. “TAKE A NUMBER; 32 Billion.” The New York Times (Tuesday, February 6, 2018): D3.
(Note: ellipsis added.)
(Note: the online version of the story has the date FEB. 1, 2018, and has the title “TAKE A NUMBER; The Smiling Axolotl Hides a Secret: A Giant Genome.”)

Environment Can Affect Which Genes Are Activated

(p. D5) In September 1944, trains in the Netherlands ground to a halt. Dutch railway workers were hoping that a strike could stop the transport of Nazi troops, helping the advancing Allied forces.
But the Allied campaign failed, and the Nazis punished the Netherlands by blocking food supplies, plunging much of the country into famine. By the time the Netherlands was liberated in May 1945, more than 20,000 people had died of starvation.
The Dutch Hunger Winter has proved unique in unexpected ways. Because it started and ended so abruptly, it has served as an unplanned experiment in human health. Pregnant women, it turns out, were uniquely vulnerable, and the children they gave birth to have been influenced by famine throughout their lives.
When they became adults, they ended up a few pounds heavier than average. In middle age, they had higher levels of triglycerides and LDL cholesterol. They also experienced higher rates of such conditions as obesity, diabetes and schizophrenia.
. . .
“How on earth can your body remember the environment it was exposed to in the womb — and remember that decades later?” wondered Bas Heijmans, a geneticist at Leiden University Medical Center in the Netherlands.
Dr. Heijmans, Dr. Lumey and their colleagues published a possible answer, or part of one, on Wednesday in the journal Science Advances. Their study suggests that the Dutch Hunger Winter silenced certain genes in unborn children — and that they’ve stayed quiet ever since.
While all cells in a person’s body share the same genes, different ones are active or silent in different cells. That program largely is locked in place before birth.
But scientists have learned that later experiences — say, exposure to a virus — can cause cells to quiet a gene or boost its activity, sometimes permanently.
The study of this long-term gene control is called epigenetics. Researchers have identified molecules that cells use to program DNA, but how those tools work isn’t entirely clear. One of the best studied is a molecular cap called a methyl group.
At millions of spots across our DNA, genes may carry a methyl group. They seem to silence genes — at least, researchers have found that silenced genes often have a collection of methyl groups lurking nearby.

For the full story, see:
Zimmer, Carl. “Dutch Genes Still Bear Scars of a Famine.” The New York Times (Tuesday, February 6, 2018): D5.
(Note: ellipsis added.)
(Note: the online version of the story has the date JAN. 31, 2018, and has the title “MATTER; The Famine Ended 70 Years Ago, but Dutch Genes Still Bear Scars.”)

FDR’s Coast Guard Denied Entry to Future Medical Visionary

(p. B12) Dr. Arno G. Motulsky, a former refugee from Nazi Germany who became a founder of medical genetics, recognizing the connection between genes and health long before mainstream medicine did, died on Jan. 17 [2018] at his home in Seattle.
. . .
“It was his vision to study how heredity could be involved in practically everything,” Dr. Francis Collins, a geneticist and the director of the National Institutes of Health, said in an interview. “The relationship between heredity and the response to drug therapy — nobody was thinking about that until he started, 60 years ago. He anticipated it decades before science made it possible to get the answers that he dreamed of.”
As technologies emerged to decode DNA, the fields that Dr. Motulsky helped originate came to the forefront of medicine, leading to improved diagnosis and treatments for a host of diseases.
. . .
Dr. Motulsky’s path to prominence began in harrowing fashion. He had been one of more than 900 Jewish refugees aboard the German liner St. Louis, which reached the Miami coast in 1939 but was turned away by the United States and sent back to Europe.
. . .
His parents tried to leave Germany with him and his younger siblings, Leah and Lothar, in 1939, before war broke out in Europe. In an account he gave to the Annual Review of Genomics and Human Genetics in 2016, Dr. Motulsky said his family had hoped to join his father’s brother in Chicago but headed for Cuba instead after hearing that a United States quota system was causing long delays in granting visas.
His father left first. His mother followed soon afterward, taking young Arno and his brother and sister with her aboard the St. Louis in Hamburg on May 13, 1939, bound for Havana. But Cuba refused to accept the refugees, as did other Caribbean countries.
“We asked to land in America, but were denied,” Dr. Motulsky said. “When we sailed close to Miami, U.S. Coast Guard cutters and planes shooed us off.”
Its passengers filled with dread, the ship headed back to Europe on June 6.
“Miraculously, a few days before we would have arrived back in Germany, four other countries — England, France, Holland and Belgium — each agreed to take one-fourth of the passengers,” Dr. Motulsky said.

For the full obituary, see:
DENISE GRADY. “Arno Motulsky, a Founder of Medical Genetics 60 Years Ago, Dies at 94.” The New York Times (Tuesday, January 30, 2018): B12.
(Note: ellipsis, and bracketed year, added.)
(Note: the online version of the obituary has the date JAN. 29, 2018, and has the title “Arno Motulsky, a Founder of Medical Genetics, Dies at 94.”)

Blobel Pursued a Slow Hunch for Over 30 Years

(p. B19) Günter Blobel, a molecular biologist who was awarded the 1999 Nobel Prize in Medicine for discovering that proteins in any living cell have virtual ZIP codes that guide them to where they can help regulate body tissues, organs and chemistry, died on Sunday [February 18, 2018] in Manhattan. He was 81.
. . .
The cause was cancer.
. . .
He spent nearly all his working life at Rockefeller University, what he regarded as the Valhalla of research.
Like many scientific advances, Dr. Blobel’s had no moment of “Eureka!” It unfolded over 30 years of painstaking, often frustrating, but occasionally thrilling investigation: a process of building on others’ work, intuitive thinking to form new hypotheses, and testing, using the results to modify his theories, and then testing and modifying again and again.
Driven to find underlying causes of diseases that were being treated for symptoms, and funded by the National Institutes of Health and the Howard Hughes Medical Institute, he successively developed five models of his original “beautiful idea.” Along the way he won many prestigious awards, some for essentially the same insights recognized later by the Nobel committee.

For the full obituary, see:
ROBERT D. McFADDEN. “Günter Blobel, Nobel Laureate Who Found Cell ‘ZIP Codes,’ Dies at 81.” The New York Times (Saturday, Feb. 20, 2018): B19.
(Note: ellipses, and bracketed date, added.)
(Note: the online version of the obituary has a date of Feb. 19, 2018.)

Regulations Threaten Precision Medicine Innovations Against Cancer

(p. A15) The federal government is threatening to limit treatment options for doctors fighting cancer.
. . .
At issue is whether reimbursements will be available to most physicians, hospitals and patients for a diagnostic technology known as next-generation sequencing. A cornerstone of the emerging field of precision medicine, NGS tests analyze molecular changes that occur in cancerous tumors and show up in biopsies.
. . .
Under the proposed policy, only one of hundreds of laboratories that currently offer NGS testing would meet all the new reimbursement requirements. The policy would in effect force clinicians and institutions to send all NGS testing to a single vendor, Foundation Medicine .
This is unfair to cancer patients. The proposal would result in a monopoly, allowing price manipulations, decreasing quality, and potentially contributing to market failure. It would turn the entire genomic-testing industry upside-down. The FDA is already unable to keep up with advances in precision medicine. Restricting access to cutting-edge molecular testing would stifle growth in precision medicine at approved testing sites nationwide. The limits could prevent desperately needed innovation, setting back progress in genomic testing and oncology by at least a decade.

For the full commentary, see:
Olivier Elemento. ”A New Regulatory Threat to Cancer Patients; Washington may impose needless limits on genetic testing.” The Wall Street Journal (Mon., Feb. 26, 2018): A15.
(Note: ellipses added.)
(Note: the online version of the commentary has the date Feb. 25, 2018.)

Over-Regulated, Quasi-Governmental Health Sector Is Often Slow in Face of Crisis

The nurse interviewed in the passages quoted below, also appeared at about the same period, on Anderson Cooper’s CNN 360 show. On that she had a wonderful riff on how the hospital was irresponsible in taking so long to get the right protective gear. She says that they could, and should, have gotten it overnight through Amazon Prime.

(p. B4) DALLAS — A nurse who observed and participated in the care of Ebola patients at Texas Health Presbyterian Hospital spoke out publicly on Thursday about what she characterized as inadequate training and infection control there.
. . .
Ms. Aguirre said she and other nurses were “horrified” at the protocols used to care for Ms. Pham. She said they received instruction only once about the proper use of personal protective equipment — gloves, masks, gowns, hoods and shields — before entering Ms. Pham’s room, and then were shown how to remove the potentially contaminated gear while in the room. The garb left a triangle of skin exposed on the front of her neck.
“The very first time I was being instructed to put the stuff on I immediately voiced my concerns,” Ms. Aguirre said. “Why would I be wearing two pairs of gloves, three pairs of bootees, have my entire body covered in plastic, have two hoods on and have an area so close to my mouth and my nose exposed? And they said, ‘We know, we’ve addressed it and basically our verdict on that at this time is we’re taping that area closed.’ “

For the full story, see:
KEVIN SACK. “Controls Poor at Hospital, Nurse Says.” The New York Times (Fri., October 17, 2014): A14.
(Note: ellipsis added.)
(Note: the online version of the story has the date OCT. 16, 2014, and has the title “WHEELS; The Internal Combustion Engine Is Not Dead Yet.” The online version says that the New York print version was on p. A14. My paper, probably the midwest version, was on p. A18.)

Audacious Heart Surgery During WW II Was Proof of Concept

(p. C9) The battle to operate meaningfully within the heart was a source of wonder and inspiration. Innovative in the extreme, brave to the point of recklessness, only exceptional characters could succeed. Some people claimed that only psychopaths could thrive in this environment. They were correct. More sensitive souls, like John Gibbon, who launched open-heart surgery in 1953, gave up after a spate of child deaths.
Thomas Morris tells this history well. “The Matter of the Heart” provides a thoroughly researched and detailed account of the major advances in cardiac surgery as derived from surgical literature, media reports and textbooks.
. . .
On Feb. 19, 1945, the courageous U.S. military surgeon Dwight Harken was attempting to remove bullets and shrapnel from in and around wounded soldiers’ hearts as a group of senior British surgeons looked on. His operating theater consisted of a ramshackle hut with corrugated iron roof in the English Cotswolds. “Working as quickly as he could, Harken now made a small incision in the heart wall and inserted a pair of forceps to widen the opening,” Mr. Morris recounts. “Through this aperture he introduced a clamp and fastened it around the elusive piece of metal. For a moment all was quiet. And then . . . ‘suddenly, with a pop as if a champagne cork had been drawn, the fragment jumped out of the ventricle, forced by the pressure within the chamber. Blood poured out in a torrent.’ . . . Harken put a finger over it, and picking up a needle started to sew it shut. . . . He discovered that he had sewn his glove to the wall of the heart. Finally his assistant cut him loose, and the job was done. Opening the heart, removing the shell fragment and repairing the incision had taken three minutes. His distinguished guests were deeply impressed: this was surgery of a sophistication and audacity which none had seen before.” This was the case that persuaded the English and American allies that heart surgery was indeed a possibility.

For the full review, see:
Stephen Westaby. “How the Beat Goes On; A daring attempt to pick shrapnel from a soldier’s heart opened the door to cardiac surgery.” The Wall Street Journal (Saturday, Jan. 27, 2018): C9.
(Note: ellipsis between paragraphs, added; ellipses internal two second quoted paragraph, in original.)
(Note: the online version of the review has the date Jan. 26, 2018, and has the title “Review: How the Beat Goes On in ‘The Matter of the Heart’; A daring attempt to pick shrapnel from a soldier’s heart opened the door to cardiac surgery.”)

The book under review, is:
Morris, Thomas. The Matter of the Heart: A History of the Heart in Eleven Operations. New York: Thomas Dunne Books, 2018.

Knowledge from Self-Experimentation Should Be Publishable

(p. D4) When Bob Hariri developed a product he thought could be useful as a human-skin replacement for burn victims, he had no trouble finding a subject willing to test it–himself.
An entrepreneur and a neurosurgeon with both a medical degree and a doctorate, Dr. Hariri is one of a number of scientists who have experimented on themselves with new or yet-to-be approved medical products or technologies, and who say such practice can be indispensable in the development of innovative biomedical treatments.
Some scientists are pushing for self-experimentation data to be reported publicly and more systematically to aid scientific progress. Alex Zhavoronkov, chief executive of an aging-research company called InSilico Medicine Inc., and others hope to start a peer-reviewed journal on self-experimentation, where scientists and other qualified individuals would publish high-quality case studies of tests performed on themselves. He plans to launch a crowdfunding operation in the next few months to fund it.
The idea is “to unlock the knowledge [of self-experimentation] that resides there anyway,” says Dr. Zhavrononkov, who takes an old diabetes drug called metformin that is supposed to have antiaging properties, even though it hasn’t been approved for that purpose.
. . .
Advocates say self-experimentation can yield information that is hard to get from a clinical trial. The experimenter feels what it’s like to be the patient and gets insight into how to improve testing procedures. Also, a number of individual reports, when cobbled together, can start to yield a picture of whether a new treatment is likely to work or not, though one wouldn’t rely on those reports alone to conclude safety or effectiveness.

For the full story, see:
Wang, Shirley S. “Why Medical Researchers Experiment on Themselves.”The Wall Street Journal (Tues., January 26, 2016): D4.
(Note: ellipsis added.)
(Note: the online version of the story has the date Jan. 25, 2016, and has the title “IN THE LAB; More Medical Researchers Engage In Self-Experimentation.”)