by Katie Hunt CNN, November 16, 2023
The United Kingdom has become the first country to give regulatory approval to a medical treatment involving the revolutionary CRISPR gene editing tool.
by Joseph Walker, Wall Street Journal, December 8, 2023
The landmark decision by the Food and Drug Administration heralds a powerful new kind of medicine, one that turns off or replaces genes to tackle conditions that have long confounded doctors and researchers.
Vertex’s Casgevy and Bluebird Bio’s Lyfgenia, both CRISPR-based treatments for sickle cell disease and beta thalassemia, have been approved by the United Kingdom and the United States based on remarkably successful clinical trials. According to Vertex, 94% of clinical trial participants went at least one year without having an episode of severe pain that required medical attention, and one was hospitalized.
Sickle cell disease and beta thalassemia involve a single gene mutation that affects hemoglobin in red blood cells. Casgevy edits the patient’s bone marrow stem cells so their bodies will produce a fetal version of hemoglobin. However, the treatment is grueling. The first step is to harvest the patient’s stem cells from their bone marrow. These cells are then edited in the lab rather than in the body, which decreases the risk of off-target edits. Before the genetically modified cells are re-inserted into the body, the patients must undergo high-dose chemotherapy or radiation therapy as a kind of “conditioning treatment” or preparation for the cells. Once the cells are re-introduced, the patient must remain in the hospital for a month for observation.
A report in STAT news addresses the difficult decision people undergoing this treatment will have to make because the treatment causes infertility (“New Gene Therapies Confront Many Sickle Cell Patients with an Impossible Choice: A Cure or Fertility”). Participants in the clinical trials were offered reproductive counseling, and many chose to freeze their eggs or sperm for future use.
Another ethical issue with these treatments is the price and availability to the populations most affected by sickle cell disease. Vertex’s Casgevy is priced at $2.2 million, and Bluebird Bio’s Lyfgenia costs $3.1 million. Seventy-five percent of the world’s sickle cell population lives in sub-Saharan Africa (“New Sickle Cell Therapies Will Be out of Reach Where They Are Needed Most,” New York Times). India makes up another significant portion of the sickle cell population, yet many of the locations in India and sub-Saharan Africa do not have either the infrastructure to provide a complex medical procedure or the resources to pay for it (“Sickle Cell Affects More Families in Africa and India, But New Gene Therapies Are out of Reach,” Associated Press).
by Casey Ross and Bob Herman, STAT News, November 14, 2023
The nation’s largest health insurance company pressured its medical staff to cut off payments for seriously ill patients in lockstep with a computer algorithm’s calculations, denying rehabilitation care for older and disabled Americans as profits soared, a STAT investigation has found.
by Casey Ross and Bob Herman STAT News, November 14, 2023
The investigation, cited by the lawsuit, found UnitedHealth pressured medical employees to follow an algorithm, which predicts a patient’s length of stay, to issue payment denials to people with Medicare Advantage plans. Internal documents revealed that managers within the company set a goal for clinical employees to keep patients rehab stays within 1% of the days projected by the algorithm.
STAT News found that U.S. insurance giant UnitedHealth pressured its employees to follow the company’s algorithm to deny payments for seriously ill patients. Journalists Casey Ross and Bob Herman wrote a four-part series on their investigation into the use of algorithms by insurance companies to cut off benefits, particularly to Medicare patients, and deny claims. This has led to a class-action lawsuit against UnitedHealth and Humana on behalf of deceased patients who were denied payments through Medicare Advantage Plans. The companies used the NaviHealth algorithm, nH Predict, a proprietary algorithm that the companies reportedly knew had a 90% error rate but used anyway because few people on Medicare appeal.
As a result of the investigation, the U.S. Senate launched a formal investigation while the House urged the Centers for Medicare and Medicaid Services to do a better job overseeing the use of algorithms, which resulted in investigations and additional regulations on proprietary algorithms (“How They Did It: STAT Reporters Expose how Ailing Seniors Suffer when Medicare Advantage Plans Use Algorithms to Deny Care,” The Journalist’s Resource).
by Carly Mallenbaum, Axios, November 7, 2023
As doctors deal with overwhelming demand for fertility services, machine learning tools are offering ways to help streamline the IVF process.
Axios reported in November that Alife Health’s AI software is used in 15 fertility clinics to make suggestions on hormone dosage for ovarian stimulation, embryo grading for successful transfer, and evaluation of the “overall IVF process.” The impetus behind using algorithms to aid in fertility treatment is to make the process more efficient so more people who want fertility treatments can get them. One way this streamlines the process is rather than biopsy or genetic testing, the algorithm can pick out “high-quality” embryos.
As with all algorithms, though, there is an inherent bias built into the program based on the data that it was trained on. Each proprietary algorithm has its own code and is trained on some data set. Because it is proprietary, there is no way to check which factors the algorithm considers when determining a “high-quality” embryo, which can amount to a kind of machine-guided eugenics. Other companies that are developing AI-based fertility software are Embryonics, AIVF, Life Whisperer, ImVitro, Fairtility, and Oova.
by Chad Terhune and Robin Respaut, Reuters, December 1, 2023
Drugmaker Novo Nordisk paid U.S. medical professionals at least $25.8 million over a decade in fees and expenses related to its weight-loss drugs, a Reuters analysis found. It concentrated that money on an elite group of obesity specialists who advocate giving its powerful and expensive drugs to tens of millions of Americans.
To strike pharmaceutical gold, companies need three key ingredients: a drug that addresses a pervasive health issue, like obesity; a drug that is effective and acts quickly; and a drug that patients must take indefinitely. Semaglutide-based drugs like Ozempic and Wegovy check all these boxes. However, until weight-loss drugs are covered by insurance, particularly Medicare, their reach will be limited to those who can afford its $1,000-plus monthly cost.
In November, a study came out indicating that Ozempic can also improve heart health (“The Future of Obesity Drugs Just Got Way More Real,” The Atlantic), opening the door for possible insurance coverage and adding to the growing list of benefits beyond curbing food craving and insulin management that semaglutide drugs seem to have (“Ozempic Could Also Help You Drink Less Alcohol,” Wired).
There are some questions about Novo Nordisk’s tactics in promoting Wegovy, the weight-loss drug equivalent to their diabetes drug Ozempic. While Novo Nordisk’s profits have soared because of the demand for weight-loss drugs, a Reuters investigation found that the company spent millions of dollars courting doctors who can serve as spokespersons for the drug. The Danish company focused primarily on the U.S. market because of the number of people who are overweight or obese as well as the high price of drugs in the U.S.
by Carl Zimmer, New York Times, December 4, 2023
Five people with moderate to severe brain injuries had electrodes implanted in their heads. As the electrodes stimulated their brains, their performance on cognitive tests improved. If the results hold up in larger clinical trials, the implants could become the first effective therapy for chronic brain injuries, the researchers said.
by Daniela Hernandez, Wall Street Journal, November 17, 2023
This tech could bust out of the lab and begin going mainstream as soon as the next year. Among the initial goals: improving concentration and helping consumers boost productivity and reduce burnout by alerting users when their attention wanders. Boston-based neurotech startup Neurable expects to sell a pair of headphones with brain-activity monitors to track focus in early 2024.
Elon Musk’s company, Neuralink, announced in November that it was looking for volunteers to undergo robotic-assisted surgery to implant a brain-computer interface (BCI) [“Elon Musk’s Brain Implant Startup Is Ready to Start Surgery,” Bloomberg]. Even though Neuralink received FDA approval for clinical trials earlier this year, the company has had to address reports from Wired and others that several of the monkeys who were implanted with the device at UC Davis had horrific reactions (“The Gruesome Story of How Neuralink’s Monkeys Actually Died”).
For several reasons, implantable BCI’s are not going to go mainstream; however, wearables might. A recent trial, reported in The New York Times, showed that people who had suffered from traumatic brain injury showed marked improvement in their ability to concentrate after using the wearable devices.
Even if implantable seems a long way off, Elon Musk has no qualms about his ultimate goal of ubiquitous AI-assisted BCIs that will improve cognition in everyone. BCIs, whether implantable or wearable, could be the next frontier in enhancement technologies.
Heather Zeiger, "Bioethics News Stories (November–December 2023)," Dignitas 30, no. 4 (2023): 19–21, www.cbhd.org/dignitas-articles/bioethics-news-stories-november-december-2023.
