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☆ Locks of Beethoven’s Hair Offer New Clues to the Mystery of His Deafness
▼ + stars: | 2024-05-06 | by ( Gina Kolata | ) www.nytimes.com time to read: +1 min

At 7 p.m. on May 7, 1824, Ludwig van Beethoven, then 53, strode onto the stage of the magnificent Theater am Kärntnertor in Vienna to help conduct the world premiere of his Ninth Symphony, the last he would ever complete. That performance, whose 200th anniversary is on Tuesday, was unforgettable in many ways. Ted Albrecht, a professor emeritus of musicology at Kent State University in Ohio and author of a recent book on the Ninth Symphony, described the scene. The movement began with loud kettledrums, and the crowd cheered wildly. At that moment, a soloist grasped his sleeve and turned him around to see the raucous adulation he could not hear.

Persons: Ludwig van Beethoven, strode, Ted Albrecht, Beethoven Organizations: Symphony, Kent State University, Ninth Symphony Locations: Vienna, Ohio

☆ First Patient Begins Newly Approved Sickle Cell Gene Therapy
▼ + stars: | 2024-05-06 | by ( Gina Kolata | Kenny Holston | ) www.nytimes.com time to read: 1 min

On Wednesday, Kendric Cromer, a 12-year-old boy from a suburb of Washington, became the first person in the world with sickle cell disease to begin a commercially approved gene therapy that may cure the condition. For the estimated 20,000 people with sickle cell in the United States who qualify for the treatment, the start of Kendric’s monthslong medical journey may offer hope. But it also signals the difficulties patients face as they seek a pair of new sickle cell treatments. For a lucky few, like Kendric, the treatment could make possible lives they have longed for. “Sickle cell always steals my dreams and interrupts all the things I want to do,” he said.

Persons: Kendric, ” Locations: Kendric Cromer, Washington, United States

☆ An Ozempic Relative Slowed Parkinson’s Disease in a Small Study
▼ + stars: | 2024-04-03 | by ( Gina Kolata | ) www.nytimes.com time to read: 1 min

In 1817, James Parkinson expressed a hope about the disease that is named after him. He thought that at some point there would be a discovery and “the progress of the disease may be stopped.”Now, nearly 200 years since Parkinson expressed his hope, and after four decades of unsuccessful clinical trials, a group of French researchers reports the first glimmer of success — a modest slowing of the disease in a one-year study. And the drug they used? A so-called GLP-1 receptor agonist, similar to the wildly popular drugs Ozempic, for diabetes, and Wegovy, for obesity. As many as half a million Americans have been diagnosed with Parkinson’s disease, a degenerative brain illness second only to Alzheimer’s in prevalence.

Persons: James Parkinson, Parkinson

☆ ‘A Chance to Live’: How 2 Families Faced a Catastrophic Birth Defect
▼ + stars: | 2024-03-28 | by ( Gina Kolata | Kim Raff | ) www.nytimes.com time to read: +1 min

Ashlee Wiseman, a waitress at a Sizzler in Idaho Falls, Idaho, was 10 weeks pregnant when a nurse phoned with crushing news: a test of fetal DNA in her blood had found that her baby girl had trisomy 18, a catastrophic genetic abnormality, and was unlikely to survive. Devastated, she called her partner, Clint Risenmay, who was at work. “I’m like, ‘I’m not going to listen to them. And there has to be someone who can help.’”A social media search led her to Dr. John Carey, a professor emeritus of pediatrics at the University of Utah, who has devoted his life to helping families dealing with trisomy 18. He supports pregnant women who chose abortion, but also helps couples who want to have babies with this rare condition, though most will be stillborn or die within a year.

Persons: Ashlee Wiseman, Clint Risenmay, ”, “, I’m, ’, John Carey Organizations: University of Utah Locations: Idaho Falls , Idaho

☆ Patients Hate ‘Forever’ Drugs. Is Wegovy Different?
▼ + stars: | 2024-03-24 | by ( Gina Kolata | ) www.nytimes.com time to read: +1 min

Most people, study after study shows, don’t take the medicines prescribed for them. It doesn’t matter what they are — statins, high blood pressure drugs, drugs to lower blood sugar, asthma drugs. Either patients never start taking them, or they stop. Though it’s still early days, and there is a paucity of data on compliance with the new drugs, doctors say they are noticing another astounding effect: Patients seem to take them faithfully, week in and week out. A national survey showed that when people were told they would gain weight back if they stopped taking the drugs, most lost interest in starting them.

Persons: don’t, nonadherence, Wegovy, it’s

☆ Doctors Say Diagnosis of Catherine’s Cancer Is a Familiar Scenario
▼ + stars: | 2024-03-22 | by ( Gina Kolata | More About Gina Kolata | ) www.nytimes.com time to read: +1 min

Although it is not known what type of cancer Princess Catherine has, oncologists say that what she described in her public statement that was released on Friday — discovering a cancer during another procedure, in this case a “major abdominal surgery” — is all too common. “Unfortunately, so much of the cancer we diagnose is unexpected,” said Dr. Elena Ratner, a gynecologic oncologist at Yale Cancer Center who has diagnosed many patients with ovarian cancer, uterine cancer and cancers of the lining of the uterus. Often, Dr. Ratner says, the assumption is that the endometriosis has appeared on an ovary and caused a benign ovarian cyst. But one to two weeks later, when the supposedly benign tissue has been studied, pathologists report that they found cancer. In the statement, Princess Catherine said she was is getting “a course of preventive chemotherapy.”That, too, is common.

Persons: Princess Catherine, ”, Elena Ratner, Ratner Organizations: Yale Cancer Center

☆ Eclipses Injured Their Eyes, and the World Never Looked the Same
▼ + stars: | 2024-03-20 | by ( Gina Kolata | ) www.nytimes.com time to read: 1 min

A young woman visited New York Eye & Ear Infirmary of Mount Sinai Hospital shortly after the eclipse of Aug. 21, 2017. She told Dr. Avnish Deobhakta, an ophthalmologist, that she had a black area in her vision, and then drew a crescent shape for him on a piece of paper. When Dr. Deobhakta examined her eyes, he was astonished. She had looked at the sun during the eclipse without any protection. With every eclipse, ophthalmologists see patients who looked at the sun and complain afterward that their vision is distorted: They see small black spots, their eyes are watery and sensitive to light.

Persons: Avnish, Deobhakta, ” Organizations: Mount Sinai Locations: New, Mount

☆ A Blood Test Shows Promise for Early Colon Cancer Detection
▼ + stars: | 2024-03-13 | by ( Gina Kolata | ) www.nytimes.com time to read: 1 min

Early detection of colon cancer can prevent a majority of deaths from this disease, possibly as much as 73 percent of them. But just 50 to 75 percent of middle-aged and older adults who should be screened regularly are being tested. One reason, doctors say, is that the screening methods put many people off. There are two options for people of average risk: a colonoscopy every 10 years or a fecal test every one to three years, depending on the type of test. Gastroenterologists say such tests could become part of the routine blood work that doctors order when, for example, a person comes in for an annual physical exam.

Persons: Folasade Organizations: UCLA Health

☆ Memory Loss Requires Careful Diagnosis, Scientists Say
▼ + stars: | 2024-02-09 | by ( Gina Kolata | ) www.nytimes.com time to read: 1 min

A lengthy report by the Department of Justice on President Biden’s handling of classified documents contained some astonishing assessments of his well-being and mental health. Mr. Biden, 81, was an “elderly man with a poor memory” and “diminished faculties” who “did not remember when he was vice president,” the special counsel Robert K. Hur said. In conversations recorded in 2017, Mr. Biden was “often painfully slow” and “struggling to remember events and straining at times to read and relay his own notebook entries.” So impaired was Mr. Biden that a jury was unlikely to convict him, Mr. Hur said. Republicans were quick to pounce, some calling the president unfit for office and demanding his removal.

Persons: Biden’s, Biden, “, ”, Robert K, Hur, Mr Organizations: Department of Justice, Mr

☆ Experimental Drug Cuts Off Pain at the Source, Company Says
▼ + stars: | 2024-01-30 | by ( Gina Kolata | ) www.nytimes.com time to read: +1 min

Vertex Pharmaceuticals of Boston announced Tuesday that it had developed an experimental drug that relieves moderate to severe pain, blocking pain signals before they can get to the brain. Vertex says its new drug is expected to avoid opioids’ potential to lead to addiction. In its clinical trials, Vertex measured the drug’s effect with a standard pain scale in which patients rated pain severity from 1 to 10, with 10 the most severe. Those taking its drug had a statistically and clinically meaningful reduction in pain, it reports. A third study looked at safety and tolerability of the drug in people experiencing pain from a variety of conditions.

Persons: midyear Organizations: Pharmaceuticals, Boston, Food and Drug Administration

☆ Gene Therapy Allows an 11-Year-Old Boy to Hear for the First Time
▼ + stars: | 2024-01-23 | by ( Gina Kolata | ) www.nytimes.com time to read: +1 min

Last year, after moving to Spain, his family took him to a hearing specialist, who made a surprising suggestion: Aissam might be eligible for a clinical trial using gene therapy. On Oct. 4, Aissam was treated at the Children’s Hospital of Philadelphia, becoming the first person to get gene therapy in the United States for congenital deafness. His is an extremely rare form, caused by a mutation in a single gene, otoferlin. Otoferlin deafness affects about 200,000 people worldwide. The goal of the gene therapy is to replace the mutated otoferlin gene in patients’ ears with a functional gene.

Persons: Aissam, ” Aissam, “ Organizations: Children’s Hospital of Philadelphia Locations: Aissam, Morocco, Spain, United States

☆ King Charles’s Prostate Treatment Is Common Among Men His Age
▼ + stars: | 2024-01-17 | by ( Gina Kolata | More About Gina Kolata | ) www.nytimes.com time to read: +1 min

King Charles III will have a procedure to address an enlarged prostate at a hospital next week. The 75-year-old British monarch’s diagnosis is common among men his age, and experts say that typical treatments are not dangerous. An enlarged prostate, known also as benign prostatic hyperplasia, or BPH, is a noncancerous condition that occurs frequently among older men. By age 60, more than half of men have at least mild BPH symptoms, which include difficulty urinating and a sense of urgency to urinate. The same thing happens in men, Dr. Albertsen said, and at the same age.

Persons: King Charles III, Peter Albertsen, Albertsen, ” Organizations: University of Connecticut

☆ Cancer Deaths Are Falling, but There May Be an Asterisk
▼ + stars: | 2024-01-17 | by ( Gina Kolata | ) www.nytimes.com time to read: +1 min

The cancer society highlighted three chief factors in reduced cancer deaths: declines in smoking, early detection and greatly improved treatments. Breast cancer mortality is one area where treatment had a significant impact. That includes metastatic cancer, which counted for nearly 30 percent of the reduction in the breast cancer death rate. Breast cancer treatment has improved so much that it has become a bigger factor than screening in saving lives, said Ruth Etzioni, a biostatistician at the Fred Hutchinson Cancer Center. “The biggest untold story in breast cancer is how much treatment has improved,” said Dr. H. Gilbert Welch, a cancer epidemiologist at Brigham and Women’s Hospital.

Persons: “, ”, Donald Berry, Sylvia K, Plevritis, Ruth Etzioni, Mette Kalager, H, Gilbert Welch Organizations: University of Texas, Anderson Cancer Center, Stanford University, Fred Hutchinson Cancer Center, University of Oslo, Oslo University Hospital, Brigham, Women’s

☆ Innovative Cancer Treatment May Sometimes Cause Cancer, F.D.A. Says
▼ + stars: | 2023-11-28 | by ( Gina Kolata | More About Gina Kolata | ) www.nytimes.com time to read: +3 min

Cancer specialists said the treatments have saved the lives of thousands of patients with blood cancers. And, he said, “I haven’t seen a single one” develop a new T cell cancer. When patients’ T cells are engineered to make proteins that attack cancer cells, a virus helps slip new genes into T cell DNA. Even without chemotherapy or radiation, Dr. Maus added, patients with blood cell cancers are especially susceptible to developing other blood cell cancers. But Dr. DiPersio said, “it is more of a smoking gun.”The F.D.A.

Persons: Marcela V, Maus, John DiPersio, Louis, “, ”, DiPersio, —, . Maus Organizations: Massachusetts General Hospital, Washington University School of Medicine Locations: Massachusetts, St

☆ Sickle-Cell Treatment Created With Gene Editing Wins U.K. Approval
▼ + stars: | 2023-11-16 | by ( Gina Kolata | More About Gina Kolata | ) www.nytimes.com time to read: +1 min

Regulators in Britain on Thursday approved the first treatment derived from CRISPR, the revolutionary gene-editing method. Called Casgevy, the treatment is intended to cure sickle-cell disease and a related condition, beta thalassemia. The companies anticipate that the Food and Drug Administration will approve Casgevy for sickle-cell patients in the United States in early December. The agency will decide on approval for beta thalassemia next year. That treatment does not rely on gene editing, insteading using a method that inserts new DNA into the genome.

Organizations: Vertex Pharmaceuticals, CRISPR Therapeutics, and Drug, Bluebird Bio Locations: Britain, Boston, Switzerland, United States, Somerville, Mass

☆ V.A. Recruits Millionth Veteran for Its Genetic Research Database
▼ + stars: | 2023-11-15 | by ( Gina Kolata | More About Gina Kolata | ) www.nytimes.com time to read: +3 min

says its database offers a more diverse population: 175,000 people with African ancestry and 80,000 Hispanics joined the Million Veteran Program. For example, said Dr. Sumitra Muralidhar, director of the Million Veteran Program, researchers found genes linked to having flashbacks of traumatic events, a feature of post-traumatic stress. Although researchers can examine genetic and other data and links to medical records, fewer than 10 people at the V.A. Those records, Dr. Muralidhar said, are held at a facility in Boston that is “heavily secured.”What It Looks Like: Veterans hope the database will help. hospital told Octavia Harris, 60, of San Antonio, about the Million Veteran Program.

Persons: ”, Shereef Elnahal, “, Amit V, Sumitra Muralidhar, Muralidhar, Octavia Harris, Harris Organizations: Employees, Department sVeterans Affairs, Million, Massachusetts General Hospital, Million Veteran, Navy Locations: Europe, Massachusetts, Boston, San Antonio

☆ New Gene Editing Treatment Cuts Dangerous Cholesterol in Small Study
▼ + stars: | 2023-11-12 | by ( Gina Kolata | More About Gina Kolata | ) www.nytimes.com time to read: +1 min

The handful of patients had severe heart disease that had caused chest pain and heart attacks. After trying available cholesterol-lowering medications, they could not get their cholesterol as low as cardiologists recommended. So they volunteered for an experimental cholesterol-lowering treatment using gene editing that was unlike anything tried in patients before. Each had a genetic abnormality, familial hypercholesterolemia, that affects around one million people in the United States. In the United States alone, more than 800,000 people have heart attacks each year.

Organizations: Verve Therapeutics, American Heart Association Locations: Boston, United States

☆ F.D.A. Approves New Obesity Drug That Will Compete With Wegovy
▼ + stars: | 2023-11-08 | by ( Gina Kolata | More About Gina Kolata | ) www.nytimes.com time to read: 1 min

The Food and Drug Administration on Wednesday approved an obesity drug from the company Eli Lilly that will be a direct competitor to the wildly popular Wegovy. The drug is called tirzepatide and will be sold under the name Zepbound. Patients who used tirzepatide lost an average of 18 percent of their body weight, according to the F.D.A., when it was taken at its highest dose in a drug trial. That’s compared with Wegovy, manufactured by Novo Nordisk, which produced an average 15 percent weight loss. approved Zepbound for people with obesity and for those who are overweight and have at least one obesity-related condition.

Persons: Eli Lilly, tirzepatide Organizations: Drug Administration, Novo Nordisk

☆ Panel Says That Innovative Sickle Cell Cure Is Safe Enough for Patients
▼ + stars: | 2023-10-31 | by ( Gina Kolata | More About Gina Kolata | ) www.nytimes.com time to read: +1 min

Family members are affected too — they may need to take time off work during the most intensive phase of the treatment. Additionally, most Americans with sickle cell are Black and may not trust a health care system that has often failed to provide the most basic preventive and therapeutic care for those with the disease. Some with sickle cell are anxious about undergoing a medical treatment that is on the cutting edge of biotechnology. “We are finally at a spot where we can envision broadly available cures for sickle cell disease,” said Dr. John Tisdale, director of the cellular and molecular therapeutics branch at the National Heart, Lung and Blood Institute and a member of the advisory committee. Kyra is now in intensive care as doctors try to control her pain.

Persons: It’s, ”, John Tisdale, Dana Jones, San Antonio, Kyra Organizations: Blood Institute

☆ F.D.A. Experts Will Vote on Safety of a Cure for Sickle Cell Disease
▼ + stars: | 2023-10-30 | by ( Gina Kolata | More About Gina Kolata | ) www.nytimes.com time to read: +4 min

An estimated 100,000 people in the United States have sickle cell disease, most of whom have African ancestry. will decide on another application for sickle cell gene therapy made by Bluebird Bio. Two other companies and an academic center, Boston Children’s Hospital, are testing their own sickle cell gene therapies. While these therapies could reduce the suffering of sickle cell patients in the United States and other wealthy countries, there is an even greater need for them in some developing countries like Nigeria. One company, Beam, is testing a way to provide gene editing that requires nothing more than a single infusion in a doctor’s office.

Persons: “, Mariah Jacqueline Scott, Scott, ”, Stephan Grupp, What’s Organizations: Institute for Clinical, CRISPR Therapeutics, Children’s Hospital of Philadelphia, Bluebird, Boston Children’s Hospital Locations: United States, Highland Park, N.J, Boston, Nigeria

☆ Ozempic and Wegovy Don’t Cost What You Think They Do
▼ + stars: | 2023-10-22 | by ( Gina Kolata | More About Gina Kolata | ) www.nytimes.com time to read: +1 min

A recent paper published by the American Enterprise Institute revealed that the net prices for the new obesity drugs are just a fraction of the published annual list prices. And while the drugs’ prices remain out of reach for many, economists anticipate they will soon be driven down. More than a dozen companies are developing obesity drugs. As they enter the market, greater choice is expected to make prices plummet, as has happened with other expensive drugs. Those revenues are based on the net prices.

Persons: ”, Jalpa Doshi, Ippolito, Joseph F, Levy Organizations: American Enterprise Institute, University of Pennsylvania, Investors, Novo Nordisk, Johns Hopkins Bloomberg School of Public Health, Health Locations: Denmark, Novo

☆ Windows Installed in Skulls Help Doctors Study Damaged Brains
▼ + stars: | 2023-09-16 | by ( Gina Kolata | More About Gina Kolata | ) www.nytimes.com time to read: +1 min

He’d fractured his skull, and a large blood clot formed on the left side of his head. Surgeons had to remove a large chunk of his skull to relieve pressure on his brain and to remove the clot. “Getting a piece of my skull taken out was crazy to me,” Mr. Marr said. “I almost felt like I’d lost a piece of me.”But what seemed even crazier to him was the way that piece was restored. His prosthesis, which is covered by his skin, is embedded with an acrylic window that would let doctors peer into his brain with ultrasound.

Persons: Tucker, He’d, ” Mr, Marr, “, I’d, . Marr Organizations: Surgeons, Deloitte

☆ We Know Where New Weight Loss Drugs Came From, but Not Why They Work
▼ + stars: | 2023-08-17 | by ( Gina Kolata | More About Gina Kolata | ) www.nytimes.com time to read: +1 min

Every so often a drug comes along that has the potential to change the world. Medical specialists say the latest to offer that possibility are the new drugs that treat obesity — Ozempic, Wegovy, Mounjaro and more that may soon be coming onto the market. Obesity affects nearly 42 percent of American adults, and yet, Dr. Engel said, “we have been powerless.” Research into potential medical treatments for the condition led to failures. While other drugs discovered in recent decades for diseases like cancer, heart disease and Alzheimer’s were found through a logical process that led to clear targets for drug designers, the path that led to the obesity drugs was not like that. Researchers discovered by accident that exposing the brain to a natural hormone at levels never seen in nature elicited weight loss.

Persons: —, ”, Jonathan Engel, Engel Organizations: Baruch College Locations: New York

☆ 3 Factors Keeping Americans From New Weight-Loss Drugs
▼ + stars: | 2023-08-04 | by ( Gina Kolata | More About Gina Kolata | ) www.nytimes.com time to read: +3 min

That’s the median weight loss experienced by people who take Wegovy, a drug from Novo Nordisk. Many patients started by taking Ozempic, a diabetes drug also by Novo Nordisk that led to weight loss as a side effect. Mounjaro, made by Eli Lilly and approved for treating diabetes, is expected to be approved soon for obesity. While price and insurance coverage pose problems for patients, health economists expect prices to come down as more drugs are approved and companies face competition. Medicare is forbidden by law to pay for weight-loss drugs, although there is an intense lobbying effort to change that.

Persons: Eli Lilly, David A, Eli Lilly’s, “, ”, Ania, Robert F, Kushner Organizations: Novo Nordisk, Duke University, Yale University, Northwestern University Feinberg School of Medicine, “ Locations: Novo

☆ Flipping a Switch and Making Cancers Self-Destruct
▼ + stars: | 2023-07-26 | by ( Gina Kolata | More About Gina Kolata | ) www.nytimes.com time to read: +1 min

Within every cancer are molecules that spur deadly, uncontrollable growth. What if scientists could hook those molecules to others that make cells self-destruct? Could the very drivers of a cancer’s survival instead activate the program for its destruction? “It’s very cool,” said Jason Gestwicki, professor of pharmaceutical chemistry at the University of California, San Francisco. “It turns something the cancer cell needs to stay alive into something that kills it, like changing your vitamin into a poison.”

Persons: Gerald Crabtree, ”, Crabtree, Nathanael S, Gray, “, Jason Gestwicki Organizations: Stanford, redwoods, Foghorn Therapeutics, University of California Locations: Santa Cruz, San Francisco

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