Outstanding Medically Significant Advancements of 2024
Science and medicine often progress in fits and starts, but every so often, a genuine breakthrough emerges that significantly impacts how doctors manage or address specific health issues. 2024 has been a year full of such advancements, from revolutionary treatments for persistent genetic conditions to a groundbreaking medication for HIV. Let's explore some of this year's groundbreaking drugs and innovations that are poised to revolutionize healthcare.
Introducing New Tools in the Battle Against Antibiotic-Resistant Bugs
Antibiotic resistance continues to be a major public health concern. Over time, various bacterial strains have developed resistant characteristics against commonly used antibiotics, including those that cause urinary tract infections (UTIs). In October, the FDA granted approval to Iterum Therapeutics' Orlynvah, a unique drug designed to combat resistant UTI bacteria.
Orlynvah is approved for specific UTIs caused by Escherichia coli, Klebsiella pneumoniae, or Proteus mirabilis bacteria, particularly those that have shown resistance or are anticipated to be unresponsive to other antibiotics. The drug combines a substance that extends the duration of antibiotics in the body with a compound from the penems subclass of antibacterials. Penems have been shown to have immense potential against a broad range of resistant strains, and Orlynvah is the first oral penem to receive approval.
While a steady flow of innovative treatments is essential to combating antibiotic resistance effectively, drugs such as Orlynvah, utilized responsibly, are invaluable weapons in our ongoing crusade against superbugs.
A Pioneering Drug for Schizophrenia
In September, the FDA approved Bristol Myers Squibb's Cobenfy, the first truly innovative drug for schizophrenia since the 1950s. Most antipsychotics developed to date target the neurotransmitter dopamine in the brain, believing this is the primary driver of schizophrenia. However, dopamine is not the only neurotransmitter involved, and unilaterally adjusting this lever often fails to fully control the symptoms.
Cobenfy employs a new mechanism of action by specifically targeting the neurotransmitter acetylcholine. The drug is a fusion of two medications: xanomeline, which stimulates acetylcholine receptors in the brain, and trospium chloride, designed to block any unintended effects of xanomeline elsewhere in the body. In clinical trials, Cobenfy demonstrated significant reductions in schizophrenia symptoms without causing major side effects.
The impact of Cobenfy will be significant for those with schizophrenia who have not responded to existing options. Further, it may motivate researchers and drug companies to develop other acetylcholine-based drugs for not only schizophrenia but also various neurological conditions, such as Alzheimer's disease.
A Vaccine-Like Barrier Against HIV
While a universal HIV cure may still be several years away, if attainable, science has made notable progress in both treating and preventing the infection. Antiretroviral therapy has enabled people with HIV to enjoy normal lifespans and, with proper use, significantly lowers the risk of spreading the infection to others. People can also take daily PrEP to drastically reduce their risk of contracting HIV in the first place.
This year, the FDA approved an injectable PrEP treatment (Viiv's Apretude) to be administered every two months in 2021. In June, the potential of a once or twice-yearly injectable PrEP option became even brighter, following the release of Gilead's PURPOSE 1 trial results. This study demonstrated that Gilead's lenacapavir was just as effective or even more so at preventing HIV infections over a year in cisgender women. Preliminary findings from the PURPOSE II trial, reported last fall, also indicated lenacapavir's effectiveness across various populations, including cisgender men, transgender men, transgender women, and non-binary individuals. Across both trials, lenacapavir proved to be over 99% effective at preventing HIV, with side effects similar to those of existing PrEP medications.
Lenacapavir's exceptional findings make it highly likely to receive FDA approval as a new type of PrEP in the near future. Daily oral PrEP is highly effective at preventing HIV, but maintaining a daily regimen can be challenging for some. And in certain regions, taking or storing PrEP pills may elicit stigma and discrimination. As a vaccine-like option, lenacapavir could supply a more convenient, safer way to prevent HIV.
Many outside specialists have already hailed lenacapavir as the leading research breakthrough of the year. And merely this week, Gilead announced plans to test a once-a-year formulation of lenacapavir, which could make it even more analogous to routine vaccinations (if all goes well, this variant is expected to become available by 2027). Lenacapavir and other comparable drugs may go a long way to pushing HIV closer to eradication – a goal that was once considered unfeasible.
Gone are the days of limited treatment options for certain genetic disorders and conditions, as recent developments in the medical field have opened up new avenues.
The dawn of NPC treatment
NPC, or Niemann-Pick disease type C, is an uncommon but debilitating genetic condition that affects around 1,000 individuals in the U.S. It impairs an individual's ability to transport cholesterol and other lipids within their cells, leading to damaging accumulations in various organs, such as the liver, spleen, and brain. Symptoms can vary, but dementia is often present, and the current median life expectancy for patients is just 13. In September, the FDA granted approval to two revolutionary medications designed to combat NPC: Zevra Therapeutics’ Miplyffa and IntraBio’s Aqneursa.
Both medications, administered as tablets, were discovered to slow down the deterioration of symptoms compared to a placebo in clinical trials. While Aqneursa can be taken alone, Miplyffa will be prescribed in conjunction with an enzyme. It's only been recently that Miplyffa has become commercially accessible. The future impact of these drugs on NPC patients is yet to be seen, but given the scant availability of alternatives until now, these approvals can be considered groundbreaking.
The future of hot flash management
In August, Bayer released the results of two successful Phase III trials that proved its experimental drug, elinzanetant, as an effective treatment for moderate to severe hot flashes in women over 40. By the conclusion of the studies, women taking elinzanetant had a noticeable improvement in their symptoms compared to those on a placebo, with over 80% of women experiencing over a 50% reduction in symptoms.
The FDA has yet to give its final approval to the drug, but given the promising results, it's likely to do so by July 2024. If it does, it would signify a significant shift in the management of this common and frequently disruptive medical condition. Elinzanetant would become the second non-hormonal medication of its kind, following the approval of fezolinetant in 2023, to treat symptoms by impacting neurons associated with hot flashes. What makes elinzanetant unique is its ability to block two key receptors, making it the first drug of its kind to do so. For women who are unable or unwilling to receive hormonal therapy for hot flashes, more treatment options are always beneficial.
Horizon of possibilities
2024 saw numerous scientific advancements, but there's much more to come in the near and distant future.
Upcoming milestones on the radar: the potential approval of Vertex’s suzetrigine, a non-opioid drug for moderate to severe acute pain; the initiation of phase III trials to test out new incretin-based drugs for obesity and diabetes, which may be more effective than current blockbuster drugs like semaglutide (Ozempic, Wegovy) and tirzepatide (Mounjaro, Zepbound); the advent of new and improved flu vaccines, including those created to be combined with a COVID-19 vaccine.
Further down the line, potential innovations that could significantly impact the medical field if research continues to yield positive results include: the ability to substantially restore the sight of individuals with certain inherited vision disorders through gene therapy; the commencement of live organ transplants from genetically modified pigs into living and terminally ill patients, though treatments have only resulted in extended lifespans of a few months so far; and the possibility of using brain implants to enable individuals with certain types of spinal cord injuries to walk again, based on early findings published this year.
In the realm of technology and science, 2024 has also seen significant strides in potential treatments for vision disorders. For instance, companies are working on gene therapies that could significantly restore vision for individuals with inherited conditions such as retinitis pigmentosa. Similarly, advancements in genetic engineering have brought us closer to the possibility of live organ transplants from genetically modified pigs into terminally ill patients, although current results have only extended lifespans by a few months.
Furthermore, the future of healthcare could be revolutionized by brain implants, as researchers are exploring their potential to help individuals with certain types of spinal cord injuries walk again. Initial findings published this year have been promising, offering hope for a future where paralysis may no longer be a permanent condition. These advancements demonstrate the limitless potential of technology and science in improving health and addressing various health issues in the future.
