It’s easy to forget that we live in an age of medical wonders. Cancer tumors can be slowed or shrunk in ways previous generations couldn’t imagine, with everything from pills to genetically engineered white blood cells. Surgeons can transplant a face, or replace a heart valve without cracking a chest. These are outcomes that would not have been possible 20 years ago.
So what changed this year?
Below is an attempt to recognize new advances that were unveiled in 2024 and that will be remembered in the future as having changed the way medicine is practiced — as well as the way people live. I gave preference to advances that are well-proven and are becoming available to people now, not basic research or proof-of-concept efforts. My expertise? I’m a reporter who has been watching medical innovation for two decades.
GLP-1s beyond obesity
The drugs known as GLP-1 analogues have been slowly changing the treatment of diabetes for decades, ever since the first, derived from Gila monster venom, was introduced two decades ago. Over the past few years, new versions have become a phenomenon, both in their social impact and their sales, because they are perhaps the first truly effective and relatively safe obesity drugs.
But in 2024, the Food and Drug Administration approved semaglutide, made by Novo Nordisk, to reduce the risk of heart attacks and strokes. New trial results have also shown that semaglutide can treat heart failure and alleviate knee arthritis pain as well. Eli Lilly’s tirzepatide was approved to treat sleep apnea. The message: These medicines appear to have benefits for patients with many diseases for which being overweight is a risk factor.
Now drugmakers are trying to create oral versions of the drugs. Right now they are difficult-to-manufacture injections that contain protein fragments called peptides. But Eli Lilly, Pfizer, Viking Therapeutics, and Structure Therapeutics are among numerous companies working on pills that could be far easier to manufacture — and might make such drugs available to even larger numbers of people.
Preventing HIV
Experimental HIV vaccines have failed repeatedly, in one of the most frustrating efforts in medical research. But another approach to curbing spread of the disease has worked: giving HIV medicines to people who are at risk to prevent infection, a practice known as pre-exposure prophylaxis, or PrEP.
PrEP is common, and has generally involved use of a combination pill called Truvada, which is made by Gilead Sciences and taken daily. A new Gilead drug, lenacapavir, was found in a large clinical trial to be even more effective, in part because it is long-acting: The medicine is an injection that is given twice a year.
In a study published in the New England Journal of Medicine, 5,338 women in South America and Uganda were randomly assigned to receive Truvada, a newer form of that drug, or lenacapavir. No women in the lenacapavir arm developed HIV, compared to about two of every 100 people treated for a year with the other drugs. Lenacapavir is approved as an HIV treatment, although it is not yet approved in the U.S. for PrEP. One of the biggest issues is social, not scientific: People in need of PrEP in less well-off nations often don’t have access.
New drugs for schizophrenia, muscular dystrophy, and other diseases
Those first two advances were based on new research into existing drugs, not new ones. That’s how medicine often works, with studies slowly accruing, not sudden breakthroughs. Of course, making new drugs available also helps.
Through Dec. 20, the FDA approved 50 brand new medicines, defined as proteins, small molecules, or other chemicals this year. That’s 9% fewer than in 2023, but still an impressive tally. In the 2000s, the annual tally never rose above 36. The figure also doesn’t include gene therapies and some other types of new treatments.
Some standouts:
Cobenfy, made by Bristol Myers Squibb, represents the first new class of treatments for schizophrenia in decades.
Winrevair is a drug to treat pulmonary arterial hypertension, in which the flow of blood from the lungs to the heart is limited. It helped patients walk 41 meters farther than patients on placebo in a six-minute test, on average. It is manufactured by Merck.
Duvyzat was approved to treat Duchenne muscular dystrophy in some patients. Gene therapies for Duchenne have grabbed headlines while generating controversy in recent years. This drug has gone largely uncovered in the press. The FDA said it helped patients rise to a standing position.
Progress in cancer
It’s gotten to the point where advances in cancer that would have once generated huge public attention fade into the background. Still, progress is being made. Some examples:
Enhertu, a drug that is already used to treat breast cancer patients whose tumors test positive for a particular protein, HER2, was found to work even when HER2 levels were very low — that is, for most patients. That’s because the drug couples a chemotherapy payload with a targeted agent, like a smart bomb.
Tagrisso, used in non-small cell lung cancer that tests positive for a mutation called EGFR, is used in metastatic disease and to prevent tumors that have been removed surgically from coming back. New results showed the drug could also be used in patients with an earlier stage of cancer, in which the disease has spread locally. These types of wins, while not breakthroughs, add up to progress.
Sometimes it can take a long while for the impact of a new medicine to be visible. It’s been clear for years that the HPV vaccine, for example, can prevent cervical, head-and-neck, and other types of cancer caused by the human papillomavirus. Results from Scotland in January showed that not only had the vaccine dramatically reduced cervical cancer rates there, but also that for children who were vaccinated at age 12 or 13, as recommended, there had been no cases of cervical cancer since the program began in 2008.
More developments to watch
There are a lot of advances that have not reached patients yet that are still promising or just really, really cool. Here are a few.
CAR-T therapy, in which white blood cells are genetically modified, is being used to treat autoimmune diseases such lupus. Early data have set off a biotech gold rush.
Pig organs are being implanted into people in an effort to widen the supply of organs available for transplant. This year, a gene-edited kidney from a pig was transplanted into a human for the first time. The man died two months later.
Electrical stimulation to help restore some function to people with spinal cord injuries has made great strides, including with the first FDA approval of the technology.
The U.S. health care system is fraught with problems. It fails at controlling costs, at making sure that new breakthroughs are accessible to those who need them, and at distributing important new medicines to poorer parts of the world. It can also be inhumane. But the pace of innovation is real and fast — just look at the examples above.
Correction: An earlier version of this story mischaracterized the results of a clinical trial of lenacapavir.
