Science and medicine often stumble and stumble. But every once in a while, a real breakthrough comes along and greatly improves the way doctors can treat or treat a particular health problem.
The year 2024 has seen its fair share of these important advances, from new treatments for degenerative genetic conditions to important drugs like the HIV vaccine. Here’s a look at this year’s drugs and innovations that could change the game going forward.
New weapons in the war against superbugs
Antibiotic resistance is one of the biggest public health challenges. Many pathogens have gradually adapted to common medications used to treat them, including those that cause urinary tract infections. In October, the FDA approved a drug designed to fight these stubborn UTI bugs: Iterum Therapeutics’ Orlynvah.
Orlynvah is only approved for certain UTI causes Escherichia coli, Klebsiella pneumoniae, or Proteus mirabilis bacteria—especially UTIs that have not responded or are not expected to respond to other antibiotics. It is the first antibiotic of its kind, which includes a drug that extends the duration of antibiotics in the body with a compound from a subclass of antibiotics known as penems. Penems have shown promise in treating a variety of resistant bacteria, but Orlynvah is the first oral penem to be approved.
A strong pipeline of new drugs is essential to successfully combating viruses, given how quickly viruses change in response to new drugs. But drugs like Orlynvah, as long as they are administered carefully, are an important weapon in our eternal war against superbugs.
A basic drug for schizophrenia
In September, the FDA approved Bristol Myers Squibb’s Cobenfy—the first real schizophrenia drug seen since the 1950s.
Over the years, scientists have developed less harsh antipsychotics, but all of these drugs work with the same basic principle of targeting the neurotransmitter dopamine in our brain. Dopamine is not the only neurotransmitter involved in schizophrenia, however, and simply adjusting this one lever is often not enough to keep people’s symptoms in check.
Cobenfy is the first drug for schizophrenia to use a new mechanism of action, by directly 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 unintended effects of xanomeline elsewhere in the body. In clinical trials, Cobenfy significantly reduced people’s symptoms of schizophrenia without causing major side effects.
Since Cobenfy will be useful for people with schizophrenia who have not responded to existing options, its value goes beyond that. Scientists and drug companies will now be encouraged to develop other acetylcholine-based drugs that are not limited to schizophrenia but also other neurological conditions, such as Alzheimer’s disease.
A barrier like a vaccine against HIV
An effective cure for HIV is probably years away, if possible. But science has made incredible strides in treating and preventing the infection, which has been killing the entire world. Antiretroviral therapy has made it possible for people living with HIV to live normal lives, for example, often without the risk of spreading the infection to others. People can also take versions of these drugs such as pre-exposure prophylaxis (PrEP), which greatly reduces their chances of getting the infection in the first place.
Today, PrEP is usually taken as a daily pill, although the FDA also approved an injectable PrEP treatment to be taken every two months in 2021 (Viiv’s Apretude). This past June, the future of PrEP became even brighter, with the results released from Gilead’s AIM 1 study, which tested the annual injection of their antiretroviral lenacapavir (the drug is already approved to treat HIV). A Phase III trial found that twice-yearly lenacapavir performed as well or better than daily PrEP options in preventing HIV infection in cisgender women over a one-year period. Preliminary results from the PURPOSE II study, reported last fall, also showed the drug’s effectiveness among cisgender men, transgender men, transgender women, and non-binary people who sleep with men. In both trials, lenacapavir for two years 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 results, lenacapavir will likely 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 stick to the daily regimen needed to be fully protected. Some people may face stigma and discrimination if they are found to be taking or keeping PrEP pills, experts noted. So a vaccine like lenacapavir could provide a simple and safe option for HIV prevention, especially in parts of the world where HIV prevalence remains high.
Many foreign scientists have hailed the arrival of twice-yearly lenacapavir as the research breakthrough of the year. And just this week, Gilead announced that it will soon be testing a once-a-year formulation of lenacapavir, which will make it more like an annual vaccine (if all goes well, this version could be out by 2027). So it’s possible that this drug and others will help bring HIV closer to the brink of eradication—a goal that now seemed impossible.
As with previous PrEP treatments, however, significant questions remain about how affordable and accessible the treatment will be once it becomes available to the public.
The very first life-shortening dementia drugs
Niemann-Pick disease type C (NPC) is a rare but life-threatening genetic disease, probably affecting about 1,000 people in the US. damage many organs, including the liver, spleen, and brain. The progression and symptoms of NPC can vary, but they usually include dementia, and the average life expectancy of sufferers is currently only 13. In September, the Food Drug Administration approved the first-ever drugs designed to treat NPC, a few days apart: Zevra Therapeutics’ Miplyffa and IntraBio’s Aqneursa.
Both drugs, taken as a pill, have been found to slow the progression of people’s symptoms compared to a placebo, but there are important differences between them. While Aqneursa is taken alone, Miplyffa will be placed alongside an enzyme. The release of Miplyffa also took a long time, as the drug was only available for sale this month. It will take time to know whether and to what extent these drugs can significantly alter the progression of people’s disease. But given the lack of other options so far, these concessions are monumental.
A new era of hot flash therapy
In August, drugmaker Bayer published the results of two successful Phase III trials testing its investigational drug elinzanetant as a treatment for moderate to severe hot flashes in women over 40. At the end of the study, the women who took elinzanetant were significantly better than those who drank. placebo, with more than 80% of women on the drug seeing more than a 50% reduction in their symptoms.
The FDA still has to officially approve the drug, and a final decision is expected next July. But given the compelling results, approval seems more likely. If it does, it could mark an important change in the treatment of this common and often disturbing condition. Elinzanetant will be the second non-hormonal drug of its kind to treat symptoms by affecting specific heat-related receptors—following the approval of fezolinetant in 2023—and the first to do so by blocking two key receptors. Although hormone therapy in hot light is safe and effective for most women, there are those who are unable or unwilling to accept it. So the more options available for this situation, the better.
Future opportunities
2024 has seen many breakthroughs in science, but more is set to come in the coming years.
On the near horizon to watch: the potential approval of Vertex’s suzetrigine, a novel, non-opioid treatment for moderate to severe pain; the start of a phase III trial to test new incretin-based drugs for obesity and diabetes, which may be even better than the blockbuster drugs semaglutide (Ozempic, Wegovy) and tirzepatide (Mounjaro, Zepbound), and the possible arrival of new . and improved flu vaccines (including those intended to be combined with the covid-19 vaccine).
There are also potential novelties that could have a major impact on medicine if research continues to show promising results. Early trials this year found that gene therapy could significantly restore vision in people with inherited vision problems, for example. As of 2024, scientists have also begun to successfully restore organs from genetically modified pigs to living and terminally ill people, although, so far, these treatments have only extended lives by a few months. And we may even be able to help people disabled by certain types of spinal cord injuries to walk again using brain implants, based on the first findings published this year.
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