Category: Health

Dog Research on Muscular Dystrophy Can Lead to Cures for Both Dogs and Humans

(p. A13) Researchers used a gene-editing tool to repair a gene mutation in dogs with Duchenne muscular dystrophy, an important step in efforts to someday use the tool to edit DNA in people with the same fatal disease.
In a study published Thursday [Aug. 30, 2018] in the journal Science, researchers at UT Southwestern Medical Center in Dallas and the Royal Veterinary College in London reported that they used the Crispr gene-editing system in four dogs to restore production of dystrophin, a protein crucial for healthy muscle function.
. . .
“It’s like putting a good spare tire on a car. It’s not as good as the original, but it gets you where you want to go,” said Eric Olson, director of UT Southwestern’s Hamon Center for Regenerative Science and Medicine and senior author of the paper.
Dr. Olson, who is also founder and chief scientific adviser of Exonics Therapeutics Inc., which licensed the technology from UT Southwestern and helped fund the dog studies, said next steps involve testing Crispr in more dogs and observing them for a year or more. If the approach works in the dogs, he said researchers hope to try Crispr in a clinical trial with people with Duchenne.

For the full commentary, see:
Amy Dockser Marcus. “Gene Editing Shows Promise for Muscular Dystrophy.” The Wall Street Journal (Friday, Aug. 31, 2018): A13.
(Note: ellipsis, and bracketed date, added.)
(Note: the online version of the commentary has the date Aug. 30, 2018, and has the title “Crispr Used to Repair Gene Mutation in Dogs With Muscular Dystrophy.”)

The study in Science, that is mentioned above, is:
Amoasii, Leonela, John C. W. Hildyard, Hui Li, Efrain Sanchez-Ortiz, Alex Mireault, Daniel Caballero, Rachel Harron, Thaleia-Rengina Stathopoulou, Claire Massey, John M. Shelton, Rhonda Bassel-Duby, Richard J. Piercy, and Eric N. Olson. “Gene Editing Restores Dystrophin Expression in a Canine Model of Duchenne Muscular Dystrophy.” Science (Aug. 30, 2018), DOI: 10.1126/science.aau1549. [Epub ahead of print]

Cancer Cure Progress Has Been “Painfully Incremental”

(p. A15) Hopes were high in 1971 when President Richard M. Nixon called for a War on Cancer. The disease was as pernicious as it was mysterious, claiming more American lives each year in the 1960s than had perished in combat during all of World War II. Still, it wasn’t hard to imagine medical experts coming up with a cure. After all, hadn’t the country just put a man on the moon?
Almost 50 years later, the war rages on. Decades of hard work and grand promises have yielded more disappointments than breakthroughs. Reliable treatments remain elusive, and researchers still aren’t sure why some people get the disease and others don’t, why some die while others survive. In “Cancerland: A Medical Memoir,” David Scadden offers a personal account of the inspiring but often exasperating hunt for solutions to the profound problem of cancer.
. . .
. . . moving science forward “to create better clinical approaches,” Dr. Scadden writes, “is an almost painfully incremental affair.” This puts physicians in the awkward position of having to explain the slow pace of research to dying patients, many of whom hope that a miraculous new drug or therapy awaits them if they can just hold on for another year or two. This is not a crazy idea. Dr. Scadden’s own mother, who died of colon cancer in 1985, might have survived if certain studies were completed five years sooner. But most clinical trials come to nothing, particularly in cancer. Many patients are stuck with the same interventions that have been around for decades: surgery, radiation and toxic chemotherapy. The miserable side effects can sometimes make life only marginally better than death.

For the full review, see:
Emily Bobrow. “BOOKSHELF; Reason to Hope.” The Wall Street Journal (Thursday, Aug. 2, 2018): A15.
(Note: ellipses added.)
(Note: the online version of the review has the date Aug. 1, 2018, and has the title “BOOKSHELF; ‘Cancerland’ Review: Reason to Hope.”)

The book under review, is:
Scadden, David, and Michael D’Antonio. Cancerland: A Medical Memoir. New York: Thomas Dunne Books, 2018.

Uncredentialed Entrepreneur Innovated to Save Babies

(p. 1A) He showed up in Omaha 120 summers ago, another unknown showman hoping to make a name for himself at this city’s biggest-ever event, its world’s fair.

He gave his name as Martin Couney, or sometimes Martin Coney. It wasn’t, at least not yet.
He said he was a doctor, a European doctor, a protégé of the world’s finest doctors. He was none of these things.
And yet in Omaha, Dr. Couney set up shop in a little white building on the east midway, not far from the Wild West Show, the Middle Eastern dancers, the roaming fortune tellers and the Indian Congress starring a Native American chief named Geronimo.
The fair, officially known as the Trans-Mississippi and International (p. 2A) Exposition, showcased all manner of things seen as strange, exotic and otherworldly to the 2 million Nebraskans and visitors paying the 50-cent admission to have their minds blown in the summer of 1898.
Couney thought he had just the thing to blow their minds.

“Infant Incubators with Living Infants” read the sign above the entrance.

“A Wonderful Invention … Live Babies” said another.
. . .
Usually the experts are right. That’s why they are experts,” says Dawn Raffel, author of the “The Strange Case of Dr. Couney,” a new biography seeking to save this once-famed faux doctor from history’s trash bin. “But occasionally you get an outlier like this. Someone who is extraordinarily inventive. Who brings us something incredible.”
What Dr. Couney gave us, through decades of work and tireless promotion, was an understanding that we could save babies that since the beginning of time had died before they crawled. We could save them using a piece of equipment designed by a French engineer who realized that if an egg could be nurtured in an incubator, then so could a newborn.
. . .
Newspapers, including The World-Herald, largely ignored the exhibit, Raffel says. The public didn’t seem particularly bothered that a “doctor” had decided to house anonymous newborns on the fairgrounds and put them on public display.
They also didn’t seem particularly interested, either.
. . .
Raffel estimates that Couney and his doctors and nurses saved between 6,500 and 7,000 premature babies all on their own during decades of midway work. But they saved countless thousands more by raising the profile of premature babies. By raising the hope that they could grow into healthy, happy adults.
. . .
“I find him fascinating because he was such a complicated man,” Raffel says. “He deserves more credit.”

For the full story, see:
Hansen, Matthew. “Tech Costs Force Honda To Let Go of Engineering Legacy.” Omaha World-Herald (Friday, Aug. 3, 2018): 1A-2A.
(Note: ellipses between paragraphs, added; ellipsis internal to sentence, in original.)

The Raffel book on which the passages quoted are partially based, is:
Raffel, Dawn. The Strange Case of Dr. Couney: How a Mysterious European Showman Saved Thousands of American Babies. New York: Blue Rider Press, 2018.

Cancer Five-Year Survival Rates Still Discourage

I quote the discouraging cancer survival numbers below because too often “Cancer Inc.” allies itself with government regulators to slow the disruptive medical entrepreneurs who who would otherwise quickly make those numbers less discouraging.

(p. A15) Cancer Treatment Centers of America– . . . –has long raised eyebrows with its marketing. Currently, the group touts its “genomic testing,” which guides patient-specific chemotherapy. Unmentioned is the dismal success rate of such tests in trials: Only 6.4% of patients were successfully matched with a drug, according to a 2016 article in Nature.
Here, from the American Cancer Society, are five-year survival statistics for various cancers: cervical, 69%; leukemia, 63%; ovarian, 46%; brain and nervous system, 35%; lung, 19%; liver, 18%; pancreatic, 9%.
One wonders how such numbers justify the blue sky seen in today’s advertising.
. . .
. . . the war on cancer is not the place for pep talks and poetic license. We could do with more disclosure, less delusion.
Nor is this a question of depriving patients of hope. On the contrary, it’s about depriving Cancer Inc. of the ability to exploit false hope.

For the full commentary, see:
Steve Salerno. “In the War on Cancer, Truth Becomes a Casualty; The multibillion-dollar treatment industry appeals to emotion in misleading ads.” The Wall Street Journal (Saturday, April 21, 2018): A15.
(Note: ellipses added.)
(Note: the online version of the commentary has the date April 20, 2018.)

Entrepreneur Was Frustrated by Patients’ Pill Confusion

(p. B2) TJ Parker grew up working the counter for his father’s pharmacy in Concord, N.H., where he became frustrated by how much customers struggled to keep track of their medications.
He went to pharmacy school but rather than take up the family business, he and a friend set out to change it. In 2013, they launched an online pharmacy from Manchester, N.H. On Thursday, the 32-year-old CEO said he sold his startup to Amazon.com Inc. It was a roughly $1 billion deal, according to people familiar with the deal. Mr. Parker is expected to stay involved after the deal, said a person familiar with the matter.
. . .
One of the company’s earliest investors, David Frankel of Boston-based Founders Collective, wrote in a post on the website Medium Thursday that the company showed promise with two founders that complement each other.
“TJ cherishes beautiful design but has the bearing of a doctor,” he wrote of Mr. Parker, while Mr. Cohen was able to master the technical challenges behind an “indispensable pill dispensing solution.”
. . .
While attending the Massachusetts College of Pharmacy and Health Sciences in Boston, he started taking fashion-design classes at the nearby Massachusetts College of Art. “Pharmacy school was sooo boring,” he said in the interview.
His design-school stint was short-lived, but the expertise, he said, inspired PillPack’s concept of simplifying medication regimens by sorting pills into so-called “dose packets,” dispensed from a small box in baggies marked with the date and time they are to be taken.
It turned out to be a billion-dollar idea.

For the full story, see:
Eliot Brown and Sharon Terlep. “Frustrated Pharmacist Came Up With PillPack.” The Wall Street Journal (Friday, June 29, 2018): B2.
(Note: ellipses added.)
(Note: the online version of the story has the date June 28, 2018, and has the title “Behind PillPack’s $1 Billion Sale, a Frustrated 32-Year-Old Pharmacist.”)

“It’s Time for the FDA to Get with the Program”

(p. A14) Are eggs good for you or not?
It’s never been more confusing for consumers to answer that seemingly simple question. Vilified for years for their high cholesterol content, eggs more recently have broken back into dietary fashion. Nutrition experts today are touting eggs’ high levels of protein, essential vitamins and nutrients like brain-booster choline.
Government guidelines sometimes contradict nutrition experts’ advice as they play catch up with the latest scientific findings. Dietary advice from the U.S. departments of agriculture and health and human services includes eggs as part of a healthy diet, but also says cholesterol intake should be as low as possible. And the Food and Drug Administration says that eggs are too high in total fat, saturated fat and cholesterol to be labeled “healthy” by food marketers.
It’s such a scrambled issue that one egg brand is petitioning for an official government reassessment of eggs. “There’s so much new science out there about eggs, it’s time for the FDA to get with the program,” says Jesse Laflamme, chief executive of Pete and Gerry’s Organics, who filed a citizen’s petition urging the agency to rethink its ban on calling eggs “healthy.”

For the full commentary, see:
Ellen Byron. “The Great Egg Conundrum.” The Wall Street Journal (Wednesday, June 13, 2018): A14.
(Note: the online version of the commentary has the date June 12, 2018, and has the title “The Great Egg Debate: Are They Healthy or Not?”)

Fewer Regulations Allow Faster Chinese Cancer Innovation

(p. A1) HANGZHOU, China–In a hospital west of Shanghai, Wu Shixiu since March has been trying to treat cancer patients using a promising new gene-editing tool.
U.S. scientists helped devise the tool, known as Crispr-Cas9, which has captured global attention since a 2012 report said it can be used to edit DNA. Doctors haven’t been allowed to use it in human trials in America. That isn’t the case for Dr. Wu and others in China.
In a quirk of the globalized technology arena, Dr. Wu can forge ahead with the tool because he faces few regulatory hurdles to testing it on humans. His hospital’s review board took just an afternoon to sign off on his trial. He didn’t need national regulators’ approval and has few reporting requirements.
Dr. Wu’s team at Hangzhou Cancer Hospital has been drawing blood from esophageal-cancer patients, shipping it by high-speed rail to a lab that modifies disease-fighting cells using Crispr-Cas9 by deleting a gene that interferes with the immune system’s ability to fight cancer. His team then infuses the cells (p. A10) back into the patients, hoping the reprogrammed DNA will destroy the disease.
In contrast, what’s expected to be the first human Crispr trial outside China has yet to begin. The University of Pennsylvania has spent nearly two years addressing federal and other requirements, including numerous safety checks designed to minimize risks to patients. While Penn hasn’t received final federal clearance to proceed, “we hope to get clearance soon,” a Penn spokeswoman said.
“China shouldn’t have been the first one to do it,” says Dr. Wu, 53, an oncologist and president of Hangzhou Cancer Hospital. “But there are fewer restrictions.”

For the full story, see:
Rana, Preetika, Amy Dockser Marcus and Wenxin Fan. “China Races Ahead In Gene Editing.” The Wall Street Journal (Monday, January 22, 2018): A1 & A10.
(Note: the online version of the story has the date Jan. 21, 2018, and has the title “China, Unhampered by Rules, Races Ahead in Gene-Editing Trials.”)

Canada’s Single-Payer System Causes “Suffering and Deaths of People on Wait Lists”

(p. A17) Canada’s single-payer health-care system, known as Medicare, is notoriously sluggish. But private clinics like Cambie are prohibited from charging most patients for operations that public hospitals provide free. Dr. Day is challenging that prohibition before the provincial Supreme Court.
. . .
People stuck on Medicare waiting lists can only dream of timely care. Last year, the median wait between referral from a general practitioner and treatment from a specialist was 21.2 weeks, or about five months–more than double the wait a quarter-century ago. Worse, the provincial governments lie about the extent of the problem. The official clock starts only when a surgeon books the patient, not when a general practitioner makes the referral. That adds months and sometimes much longer. In November [2017] an Ontario woman learned she’d have to wait 4½ years to see a neurologist.
. . .
Dr. Day’s lawsuit aims to overturn these provisions. It alleges that the government’s legal restrictions on private care are to blame for the needless “suffering and deaths of people on wait lists.” Dr. Day argues that the current system violates citizens’ rights to “life, liberty, and security of the person,” as guaranteed by the Canadian Charter of Rights and Freedoms, the equivalent of the U.S. Bill of Rights.

For the full commentary, see:
Sally C. Pipes. “Single-Payer Health Care Isn’t Worth Waiting For; An orthopedic surgeon challenges Canada’s ban on most privately funded procedures.” The Wall Street Journal (Monday, January 22, 2018): A17.
(Note: ellipses, and bracketed year, added.)
(Note: the online version of the commentary has the date Jan. 21, 2018.)

No Known Maximum Life Span

(p. D3) Since 1900, average life expectancy around the globe has more than doubled, thanks to better public health, sanitation and food supplies. But a new study of long-lived Italians indicates that we have yet to reach the upper bound of human longevity.
“If there’s a fixed biological limit, we are not close to it,” said Elisabetta Barbi, a demographer at the University of Rome. Dr. Barbi and her colleagues published their research Thursday [sic] in the journal Science.
. . .
Dr. Barbi and her colleagues combed through Italy’s records to find every citizen who had reached the age of 105 between 2009 and 2015. To validate their ages, the researchers tracked down their birth certificates.
The team ended up with a database of 3,836 elderly Italians. The researchers tracked down death certificates for those who died in the study period and determined the rate at which various age groups were dying.
It’s long been known that the death rate starts out somewhat high in infancy and falls during the early years of life. It climbs again among people in their thirties, finally skyrocketing among those in their seventies and eighties.
. . .
Among extremely old Italians, they discovered, the death rate stops rising — the curve abruptly flattens into a plateau.
The researchers also found that people who were born in later years have a slightly lower mortality rate when they reach 105.
“The plateau is sinking over time,” said Kenneth W. Wachter, a demographer at the University of California, Berkeley, who co-authored the new study. “Improvements in mortality extend even to these extreme ages.”
“We’re not approaching any maximum life span for humans yet,” he added.

For the full story, see:
Zimmer, Carl. “What Is the Limit of Our Life Span?” The New York Times (Tuesday, July 3, 2018): D3.
(Note: ellipses added.)
(Note: the online version of the story has the date June 28, 2018, and has the title “How Long Can We Live? The Limit Hasn’t Been Reached, Study Finds.” The NYT article says the Science article was published on “Thursday,” but the citation for it that I found says it was published on Fri., June 29, 2018.)

The Science article mentioned above, is:
Barbi, Elisabetta, Francesco Lagona, Marco Marsili, James W. Vaupel, and Kenneth W. Wachter. “The Plateau of Human Mortality: Demography of Longevity Pioneers.” Science 360, no. 6396 (June 29, 2018): 1459-61.

“A Big Step Toward Regenerative Medicine”

(p. C9) Mr. Zimmer, a New York Times science columnist and author, is careful and well-informed. So when he says that research is overturning things you were taught in biology classes, he’s worth heeding. Acquired traits can be inherited. Biological time can turn backward.
. . .
The bigger breakthroughs are more fundamental. One is the development of induced pluripotent stem cells. By adding four proteins to adult cells, scientists have learned how to make them embryonic–“turning back developmental time,” as Mr. Zimmer puts it. This is a big step toward regenerative medicine, which can grow spare parts customized for your body. It also creates new ways of making babies.
. . .
Another breakthrough is gene editing. Through a process called Crispr, which tags DNA segments for deletion, we’re learning how to program cells to make specific changes to their genomes. We’re also learning how to program organisms to pass down these editing instructions to their progeny. Experiments have shown that this technology could, at some point, cure hereditary diseases such as cystic fibrosis. In addition, scientists think it could wipe out destructive rodents and malaria-carrying mosquitoes.

For the full review, see:
William Saletan. “‘Biology’s Strange New World. Acquired traits can be inherited. Biological time can turn backward. And monsters are real.” The Wall Street Journal (Saturday, June 30, 2018): C9.
(Note: ellipses added.)
(Note: the online version of the review has the date June 28, 2018, and has the title “”She Has Her Mother’s Laugh’ Review: Biology’s Strange New World. Acquired traits can be inherited. Biological time can turn backward. And monsters are real.”)

The book under review, is:
Zimmer, Carl. She Has Her Mother’s Laugh: The Powers, Perversions, and Potential of Heredity. New York: Dutton, 2018.

Experimental Immunotherapy Holds Hope of Curing Metastasized Solid Tumor Cancers

(p. B3) WASHINGTON–A novel immunotherapy method led to complete regression of breast cancer in a metastatic patient unresponsive to all other treatments, National Cancer Institute researchers said, suggesting a way to reverse some other internal-organ cancers.
The findings, from the NCI laboratory headed by Steven A. Rosenberg, were published Monday in the medical journal Nature Medicine.
The scientific report largely focused on the course of treatment for one patient, Judy Perkins of Port St. Lucie, Fla.
. . .
The research remains experimental, Dr. Rosenberg said in an interview.
“But because this new approach to immunotherapy is dependent on mutations, not on cancer type, it is in a sense a blueprint we can use for the treatment of many types of cancer,” he said. “This could be the future of immunotherapy.”
Dr. Rosenberg, who has investigated for three decades how the immune system can be employed to fight cancer, said he is hopeful this approach “holds the best opportunities for finding effective immunotherapies for patients with the solid tumors that last year caused over 500,000 deaths in this country.”

For the full story, see:
Thomas M. Burton. “Cancer-Fighting ‘Blueprint’ Seen.” The Wall Street Journal (Tuesday, June 5, 2018): B3.
(Note: ellipsis added.)
(Note: the online version of the story has the date June 4, 2018, and has the title “Novel Immunotherapy Method Led to Complete Regression of Breast Cancer in Patient.”)