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It’s easy to forget that we live in an age of medical miracles. Cancerous tumors can be slowed or shrunk in ways unimagined by previous generations, using 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 results that would not have been possible twenty years ago.
So what has changed this year?
Below is an attempt to recognize new developments unveiled in 2024 that will be remembered in the future for changing the way medicine is practiced – and also the way people live. I preferred developments that have been well proven and are now becoming available to people, rather than basic research or proof-of-concept efforts. My expertise? I’m a reporter who has been covering medical innovation for twenty years.
GLP-1s beyond obesity
The drugs known as GLP-1 analogs have slowly changed the treatment of diabetes for decades, since the first one, derived from Gila monster venom, was introduced two decades ago. In recent years, new versions have become a phenomenon, both in terms of social impact and sales, as 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 research results have also shown that semaglutide can treat heart failure and also relieve pain from knee arthritis. Eli Lilly’s Tirzepatide was approved for the treatment of sleep apnea. The message: These drugs appear to have benefits for patients with many diseases for which obesity is a risk factor.
Now drug makers are trying to make oral versions of the drugs. Currently, injections containing protein fragments called peptides are difficult to manufacture. But Eli Lilly, Pfizer, Viking Therapeutics and Structure Therapeutics are among the many companies working on pills that could be much easier to manufacture — and that could make such drugs available to even greater 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 the spread of the disease has worked: giving HIV drugs to people at risk to prevent infection, a practice known as pre-exposure prophylaxis, or PrEP.
PrEP is common and generally involves the use of a combination pill called Truvada, which is made by Gilead Sciences and taken daily. A new drug from Gilead, lenacapavir, was found to be even more effective in a large clinical trial, in part because it is long-acting: the drug is an injection given twice a year.
In a study published in the New England Journal of Medicine5,338 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 with about two in 100 people treated with the other drugs for a year. Lenacapavir is approved as an HIV treatment, although it is not yet approved for PrEP in the US. One of the biggest problems is social, not scientific: people who need PrEP in less prosperous countries often don’t have access to it.
New drugs for schizophrenia, muscular dystrophy and other diseases
Those first two advances were based on new research on existing drugs, not new ones. This is often how medicine works, with research growing slowly and no sudden breakthroughs happening. Of course, making new medicines available also helps.
Until December 20, the FDA approved 50 brand new drugs, defined this year as proteins, small molecules or other chemicals. That is 9% less than in 2023, but still an impressive number. In the 2000s, the annual number never exceeded 36. Gene therapies and some other types of new treatments are also not included in this figure.
Some highlights:
Cobenfy, made by Bristol Myers Squibb, represents the first new class of treatments for schizophrenia in decades.
Winrevair is a medicine for the treatment of pulmonary arterial hypertension, where blood flow from the lungs to the heart is restricted. It helped patients walk an average of 41 meters further than patients given a placebo, in a test lasting an average of six minutes. It is manufactured by Merck.
Duvyzat was approved for the treatment of Duchenne muscular dystrophy in some patients. Gene therapies for Duchenne have made headlines and stirred controversy in recent years. This drug has largely come to light in the press. The FDA said it helped patients get into a standing position.
Progress in cancer
It has gotten to the point where advances in cancer, which would once have generated great public attention, are fading into the background. Nevertheless, progress is being made. Some examples:
Enhertu, a drug already used to treat breast cancer patients whose tumors test positive for a certain protein, HER2, was shown to work even when HER2 levels were very low – that is, in most patients. That’s because the drug links a chemotherapy load to a targeted agent, such as a smart bomb.
Tagrisso, used in non-small cell lung cancer that tests positive for a mutation called EGFR, is used in metastatic diseases and to prevent tumors that have been surgically removed from coming back. New results showed that the drug could also be used in patients with earlier stages of cancer, where the disease has spread locally. These kinds of victories, while not breakthroughs, add up to progress.
Sometimes it can take a long time before the impact of a new medicine is visible. It has been clear for years that the HPV vaccine can prevent cervical cancer, head and neck cancer and other forms of cancer caused by the human papillomavirus. Results from Scotland in January showed that not only had the vaccine dramatically reduced the rate of cervical cancer there, but also that for children vaccinated at age 12 or 13, as recommended, there were no cases of cervical cancer since the program started in 2008.
Even more developments to watch
There are many developments that have not yet reached patients and that are still promising or just very cool. Here are a few.
CAR-T therapy, in which white blood cells are genetically modified, is used to treat autoimmune diseases such as lupus. Early data has sparked a biotech gold rush.
Pig organs are implanted in humans in an attempt to increase the supply of organs available for transplantation. This year, for the first time, a gene-edited kidney from a pig was transplanted into a human. The man died two months later.
Electrical stimulation to help restore some function in people with spinal cord injuries has made great strides, including the technology’s first approval by the FDA.
The American healthcare system is riddled with problems. It is failing to control costs, ensure new breakthroughs are accessible to those who need them, and distribute 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.