The biggest medical breakthroughs of 2024

Science and medicine usually move in spurts and stumbles. But every now and then a real breakthrough comes along and greatly improves the way doctors can manage or treat a particular health problem.

2024 has seen its fair share of these important advances, from new therapies for debilitating genetic diseases to a major vaccine-like drug for HIV. Here’s a rundown of this year’s drugs and innovations that are likely to change the rules going forward.

New weapons in the war against superbugs

Antibiotic resistance is one of humanity’s greatest public health challenges. Many disease-causing bacteria have steadily adapted to the most common drugs used to treat them, including those that cause urinary tract infections. In October, the FDA approved a drug designed to fight these persistent urinary tract infections: Iterum Therapeutics’ Orlynvah.

Orlynvah is only approved for certain urinary tract infections caused by Escherichia coli, Klebsiella pneumoniae or Proteus mirabilis bacteria – especially urinary tract infections that have not responded or are not expected to respond to other antibiotics. It is the first antibiotic of its kind, combining a drug that extends the life of antibiotics in the body with a compound from a subclass of antibacterial agents known as penems. Penems have shown promise in treating a wide variety of often resistant microbes, but Orlynvah is the first oral penem to be approved.

A robust pipeline of innovative treatments is essential to effectively combat antibiotic resistance, given how quickly bacteria evolve in response to new drugs. But drugs like Orlynvah, as long as they are managed carefully, are a crucial weapon in our eternal battle against superbugs.

A revolutionary drug for schizophrenia

In September, the FDA approved Bristol Myers Squibb’s Cobenfy – the first truly new schizophrenia drug seen since the 1950s.

Over the years, scientists have created less aggressive antipsychotics, but all of these drugs work on the same basic principle of targeting the neurotransmitter dopamine in our brain. However, dopamine is not the only neurotransmitter involved in schizophrenia, and frequently regulating this one lever is not enough to control people’s symptoms.

Cobenfy is the first schizophrenia drug to use a new mechanism of actionby specifically targeting the neurotransmitter acetylcholine. The drug is actually a combination of two drugs: xanomeline, which stimulates acetylcholine receptors in the brain, and trospium chloride, which is designed to block any off-target effects of xanomeline elsewhere in the body. In clinical trials, Cobenfy markedly reduced the symptoms of schizophrenia in humans without causing serious side effects.

As important as Cobenfy is for people with schizophrenia who have not responded to existing options, its value extends beyond that. Scientists and pharmaceutical companies will now be motivated to develop other acetylcholine-based drugs not only for schizophrenia but also for other neurological conditions such as Alzheimer’s disease.

A vaccine-like barrier against HIV

A widely viable drug for HIV is probably still many years away, if at all. But science has made incredible progress in both treating and preventing the infection, which was once universally fatal. Antiretroviral therapy has made it possible for people living with HIV to have a normal life expectancy, for example, often without the risk of spreading the infection to others. People can also take versions of these drugs as pre-exposure prophylaxis (PrEP), which greatly reduces their chances of catching the infection in the first place.

Today, PrEP is usually taken as a daily pill, although the FDA also approved injectable PrEP treatment to be taken every two months in 2021. (Viiv’s Aptitude). Last June, PrEP’s future got even brighter, with published results from Gilead’s PURPOSE 1 trial testing a twice-yearly injection of their antiretroviral lencapavir (the drug is already approved to treat HIV). The phase III study found that twice-yearly lencapavir performed as well or even better than daily PrEP options in preventing HIV infections in cisgender women over a one-year period. The preliminary results of the PURPOSE II trial, reported last fall also demonstrated the drug’s effectiveness among cisgender men, transgender men, transgender women, and nonbinary people who have sex with men. In both trials, twice-yearly lencapavir was estimated to be more than 99% effective in preventing HIV, with side effects similar to those of existing PrEP drugs.

Given the apparently impressive findings, lencapavir is almost certain to be approved as a new form of PrEP in the near future. Although oral PrEP is very effective at preventing HIV, it can be difficult for people to adhere to the daily regimen needed for optimal protection. Some people may also face stigma and discrimination if they are found to be taking or storing PrEP pills, experts noted. So the vaccine-like nature of lencapavir may provide a more convenient and safer option for HIV prevention, especially in parts of the world where HIV prevalence remains high.

Many outside scientists have already praised the arrival of twice-yearly lencapavir as research breakthrough of the year. And just this week, Gilead announced that it will soon trial a once-yearly formulation of lencapavir, making it even more like a typical annual vaccine (if all goes well, that version could be out by 2027). So it’s likely that this drug and others will help us bring HIV ever closer to the brink of eradication— already an achievable goal this once seemed impossible.

As with previous PrEP treatments, however, important questions remain about how accessible and affordable the therapy will be once it becomes available to the public.

The first drugs for life-shortening dementia

Niemann-Pick disease type C (NPC) is an incredibly rare but life-threatening genetic disorder that may affect about 1,000 people in the United States. People with NPC are unable to move cholesterol and other lipids around their cells, causing it to build up and eventually damage many organs, including the liver, spleen, and brain. The progression and symptoms of NPC can vary, but often include dementia, and the average life expectancy for sufferers is currently just 13. In September, the Food and Drug Administration approved the first-ever drugs to treat NPC, just days away : Zevra Therapeutics’ Miplyffa and Aqneursa of IntraBio.

Both drugs, taken as a pill, were found to slow the worsening of people’s symptoms compared to a placebo, but there were important differences between them. While Aqneursa is taken alone, Miplyffa will be prescribed along with an enzyme. Miplyffa also took longer to roll out as the drug was just getting started commercially available this month. It will take time to understand whether and to what extent these drugs can significantly alter the course of disease progression in humans. But given the lack of other options so far, these endorsements are monumental.

A new era of heat wave treatment

In August, drugmaker Bayer published the results of two successful phase III trials testing its experimental drug elinzanetant as a treatment for moderate to severe hot flashes in women over 40. By the end of the studies, women taking elinzanetant fared significantly better than those taking placebo, with over 80% of women on the drug seeing more than a 50% reduction in their symptoms.

The FDA has yet to formally approve the drug, with a final decision expected by next July. But given the convincing results, approval seems highly likely. If this happens, it would signal an important change in the treatment of this common and often devastating condition. Elinzanetant will become the second non-hormonal drug of its kind to treat symptoms by affecting certain neurons associated with hot flashes – after approval of fesolinetant in 2023. – and the first to do so by blocking two key receptors. While hormone therapy for hot flashes is safe and effective for most women, there are some who are unable or unwilling to take it. So the more opportunities there are for this condition, the better.

Future opportunities

In 2024 many scientific discoveries have been made, but there is much more to come in the coming years.

On the near horizon to watch out for: the potential approval of Vertex’s suzetrigina new, non-opioid treatment for moderate to severe acute pain; start of phase III trials new incretin-based drugs for obesity and diabetes, which may be even better than the already blockbuster drugs semaglutide (Ozempic, Wegovy) and tirzepatide (Mounjaro, Zepbound), and the possible occurrence of new and improved flu vaccines (including some that should be in combination with a covid-19 vaccine).

There are also potential innovations a little further afield that could have a huge impact in medicine if research continues to show promising results. Early studies this year found that gene therapy can significantly recovered the sight of people with certain hereditary visual impairments, for example. From 2024 scientists also began to successfully transplant organs of genetically modified pigs in living and terminally ill people, although so far these treatments have only extended life by a few months. And we may even someday be able to help paralyzed people with certain types of spinal cord injuries walk again using brain implants, based on early findings published this year.