Medical Breakthroughs in 2024: Profound Impact Without the Flash
While 2024’s medical advancements may appear to lack the dramatic "wow factor" of some prior years, their profound impact is undeniable. Below, we’ve categorized the year’s breakthroughs into three key areas: global healthcare advances, research developments with broad promise, and rare-disease innovations, which are of special interest to LDA Research.
Global Healthcare Advances
- Advances in Obesity Medications
One of the most transformative medical advancements this year is the continued rapid development and application of obesity drugs. In 2024, Tirzepatide and Liraglutide joined Semaglutide as approved GLP-1 receptor obesity medications, with several other promising drugs in late-stage trials. These medications are demonstrating broader health benefits beyond weight loss and diabetes management, including improvements in cardiovascular, kidney, liver, inflammation, and sleep disorders, with anticipated benefits in areas like cancer, addiction, fertility, and mental health.
Dr. W. Timothy Garvey, a leading endocrinologist and professor at the University of Alabama at Birmingham, remarked: “The development of these new medications represents a paradigm shift in the treatment of obesity, offering hope to millions of patients who have struggled with weight management and disease.”
- Injectable PrEP for HIV
Another major breakthrough is the approval and rollout of injectable pre-exposure prophylaxis (PrEP) medications for HIV, such as the long-acting injectable lenacapavir. With its twice-yearly dosing schedule, lenacapavir significantly enhances adherence and accessibility, particularly in regions with high HIV prevalence. Its unique mechanism as a capsid inhibitor also reduces the risk of drug resistance.
Dr. Linda-Gail Bekker, Director of the Desmond Tutu HIV Centre at the University of Cape Town, emphasized the achievement: “The monumental results of the PURPOSE 1 trial demonstrate that lenacapavir is 100% effective in preventing HIV transmission… This remarkable achievement builds on decades-long investments in scientific research and will yield life-changing results for countless people.”
- A New Class of Antipsychotics
The FDA's approval of Xanomeline-Trospium (marketed as KarXT and Cobenfy) marks a breakthrough in antipsychotic treatment. Unlike traditional antipsychotics, which target dopamine receptors, Xanomeline works on muscarinic acetylcholine receptors. Initially developed in the 1990s for Alzheimer’s disease, it was found to reduce psychosis symptoms but was abandoned due to gastric side effects. Pairing it with Trospium, a muscarinic antagonist that does not cross the blood-brain barrier, resolved these issues.
Hannah Brown, Director of the WRAP program at Boston Medical Center, noted: “The real benefit of this new medication lies in its tolerability. People relapse because they stop taking their antipsychotic medications due to unbearable side effects.”
- Toothbrushing to Prevent Hospital-Acquired Pneumonia (HAP)
A 2024 meta-analysis revealed that daily toothbrushing in hospitalized patients significantly reduces the incidence and mortality of HAP, the most common healthcare-associated infection. By decreasing oral bacteria, this simple intervention reduces bacteria migration to the lungs, preventing infections.
Dr. Michael Klompas, professor of population medicine at Harvard Pilgrim Health Care Institute, remarked: “The signal that we see here towards lower mortality is striking. Regular toothbrushing in the hospital may save lives. It’s rare in preventative medicine to find something so effective and inexpensive.”
Research Developments with Large Promise
- Safer Non-Opioid Painkillers
The opioid crisis has fuelled intense efforts to develop non-opioid painkillers with lower addiction risks. Opioids alleviate pain by binding to mu-opioid receptors in the central nervous system (CNS), but their profound effects also make them highly addictive, posing significant risks to public health. In contrast, emerging non-opioid painkillers offer alternative mechanisms of action. Many of these drugs work by blocking the transmission of pain signals from the peripheral nerves to the CNS or by modulating other pain pathways, reducing the likelihood of addiction. Emerging options include oral drugs like Suzetrigine, FEM-1689, and Difelikefalin, as well as topical formulations such as capsaicin-based creams and cannabinoid-based ointments.
Dr. Deborah Dowell, Chief Medical Officer of the CDC, highlighted their importance: “Non-opioid therapies are preferred for chronic pain because they are associated with better long-term outcomes and fewer risks compared with opioids.”
- Repurposing Drugs for Late-Stage Cancer
Repurposing drugs like Osimertinib for advanced-stage non-small cell lung cancer (NSCLC) has shown remarkable promise. Originally approved for early-stage NSCLC, Osimertinib’s use in later stages has proven effective at inhibiting tumor growth and reducing metastasis risks, particularly to the brain.
Dr. Roy S. Herbst, a leading oncologist, emphasized: “The results of this trial represent a substantial advancement in treating late-stage EGFR-mutated lung cancer, offering improved quality of life and prognosis.”
- Pollution’s Impact on Fertility
Highlighting the importance of gender-specific analysis, a Danish cohort study revealed significant links between environmental pollutants and infertility risks. Air pollution increases male infertility, while noise pollution elevates infertility risks in women. This study highlights that increases in environmental pollution may be one of the factors behind increased global infertility.
Professor Jill Belch of the University of Dundee commented: “Childlessness can be catastrophic... This study underscores the urgent need for stricter air quality standards and soundproofing regulations in urban planning.”
Rare Disease Innovations
- Restoring Hearing Through Dual-Vector Gene Therapy
Dual-vector gene therapy targeting the Otoferlin (OTOF) gene has restored hearing in one 11-year-old boy with OTOF gene-related hearing loss. This innovative approach splits the large OTOF gene into two segments, which are delivered separately into cells and reassembled within them.
Professor John Germiller, study lead, remarked: “Gene therapy for hearing loss is something scientists have been working toward for over 20 years. These results exceed expectations and could be applied to other conditions involving large genes.”
- Stem Cell Advances for Vision Restoration
Corneal epithelial stem cell deficiency, a condition that will lead to vision loss and can lead to blindness, saw a breakthrough with the use of induced pluripotent stem cells (iPSCs). In a small trial, researchers successfully restored vision in patients by creating corneal cells from iPSCsderived from umbilical cord blood
Professor Kohji Nishida from Osaka University shared: “We believe iPSCs can be used to treat other eye diseases, such as corneal endothelial disease and retinitis pigmentosa. Larger clinical trials are already in the works.”
- First Direct-to-Brain Gene Therapy for AADC Deficiency
The FDA’s approval of Kebilidi (Upstaza) offers new hope for patients with Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency. The inability to produce the AADC enzyme disrupts production of dopamine and serotonin, and interferes with proper motor function. This one-time gene therapy addresses the root cause of the condition by restoring dopamine production in the putamen.
Dr. Stuart Peltz, CEO of PTC Therapeutics, celebrated the achievement: “This approval represents a transformative milestone, providing patients with a life-changing cure for this devastating disease.”
What to Watch for in 2025
- Expansion of Obesity Drug Applications.
The development and rollout of new obesity drugs will continue as will further exploration of their associated health benefits beyond diabetes and weight loss, including potential improvements in cancer, addiction, fertility, and mental health.
- Monitoring the Spread of Avian Flu
As avian flu has recently spread to dairy cattle, other livestock, and humans through these vectors – resulting in the first human deaths – close monitoring of the virus’s evolution and spread will be critical in 2025.
- AI in Drug Development and Repurposing
Artificial intelligence is expected to accelerate breakthroughs in drug discovery and the repurposing of existing medications, offering the potential for faster, more cost-effective healthcare innovations.
- Advances in Genetic Sequencing with ‘Bridge RNA’
The novel “Bridge RNA” technology, which enables the rearrangement of genetic sequencing, will pave the way for treating a broader range of genetic conditions, moving beyond the single-gene focus of CRISPR technology.
- Progress in Liquid Biopsies
Following their successful use in detecting diseases like Parkinson’s and Alzheimer’s, further refinement and application of ‘liquid biopsies’ are expected, enhancing early disease treatment through detecting biomarkers in blood, urine, and sputum.
- Improving Wound Care with Biofilm Disruption
The development of plant-based compounds and enzymes targeting biofilms is anticipated to revolutionize wound care and topical disinfection, addressing one of the most persistent challenges in infection control.
