Scanning Some of 2024’s Biggest Medical Breakthroughs

Today’s era of medical breakthroughs transforms lives daily. In 2024, advancements redefined patient care worldwide. Cancer treatments now shrink tumors with pills or engineered white blood cells. Surgeons perform face transplants and heart valve replacements without major surgeries. These achievements were unimaginable 20 years ago. Yet, this year brought even more groundbreaking innovations. The biggest medical advances of 2024 are already improving lives and shaping the future of healthcare.

Advancements in Cancer Treatment: Enhertu Expands Targeting Options and Tagrisso Shows Promise in Early-Stage Lung Cancer

Advances in cancer care, which once would have attracted widespread attention, now often fade into the background. Yet, progress continues. For instance, Enhertu, a drug already used for breast cancer patients with high levels of the HER2 protein, has shown effectiveness even when HER2 levels are low. The drug combines a chemotherapy agent with a targeted treatment, acting like a smart bomb. Specifically, Enhertu, or fam-trastuzumab deruxtecan-nxki is a targeted therapy known as an antibody-drug conjugate. It combines a monoclonal antibody with deruxtecan, a drug that inhibits topoisomerase I. The antibody attaches to HER2, a protein found in high amounts on certain cancer cells, triggering their growth. Once inside the cells, deruxtecan causes DNA damage, leading to their destruction.

Tagrisso, a medication for non-small cell lung cancer (NSCLC) with the EGFR mutation, has been used to treat metastatic disease and prevent recurrence after surgery. New findings suggest it may also benefit patients with earlier stages of cancer where the disease has spread locally. These incremental successes contribute to ongoing progress.

While progress in cancer treatments is often gradual, breakthroughs like these are paving the way for long-term improvements in prevention and care. Sometimes the impact of new treatments sometimes takes time to show. For example, the HPV vaccine has long been known to prevent cancers like cervical and head-and-neck cancer. A January report from Scotland showed the vaccine significantly reduced cervical cancer rates. Notably, children vaccinated at ages 12 or 13 since 2008 have not developed cervical cancer.

GLP-1 Medications Soared to New Heights in 2024

GLP-1 analogues have revolutionized diabetes treatment for years, starting with a drug derived from Gila monster venom. These medications gained global attention recently as effective and relatively safe options for treating obesity.

In 2024, shifting away from the standard weight-loss focus, the FDA approved Novo Nordisk’s semaglutide to lower the risk of heart attacks and strokes. New research showed semaglutide also treats heart failure and reduces knee arthritis pain. Meanwhile, Eli Lilly’s tirzepatide earned the first ever approval for treating sleep apnea. These developments highlight the wide-ranging benefits of these drugs for conditions linked to excess weight.

Now, drugmakers are racing to develop oral versions of these medications. Currently, they are injections made with peptides, which are hard to manufacture. Companies like Eli Lilly, Pfizer, Viking Therapeutics, and Structure Therapeutics are among numerous runners in the race to create easier-to-produce pills that can give them a significant advantage in the industry. Markets aside, such advancements could make these life-changing drugs accessible to even more people.

Lenacapavir Proves 100% Effective in Preventing HIV in Large Trial with 5,338 Participants

Experimental HIV vaccines have repeatedly failed, making it one of the most challenging areas in medical research. However, another approach to preventing the spread of HIV has proven successful: pre-exposure prophylaxis, or PrEP, which involves giving HIV medicines to people at risk.

PrEP is widely used and typically involves a daily combination pill called Truvada, made by Gilead Sciences. A new drug from Gilead, lenacapavir, showed even greater effectiveness in a large clinical trial. The key advantage of lenacapavir is its long-acting nature, as it is administered via injection only twice a year.

In a study published in the New England Journal of Medicine, 5,338 women from South America and Uganda were randomly assigned to receive Truvada, a newer form of the drug, or lenacapavir. No women in the lenacapavir group contracted HIV, compared to about two out of every 100 women taking the other drugs for a year. While lenacapavir is approved as an HIV treatment, it has not yet been approved for PrEP in the U.S. One of the main challenges is social rather than scientific: people in lower-income countries often lack access to PrEP.

Precision Medicine Gains Ground with New Drug Developments for Schizophrenia, Muscular Dystrophy, and More

While some recent advances stem from new research on existing drugs, new treatments are also making their way into the market. As of December 20, the FDA approved 50 brand-new medicines, including proteins, small molecules, and other chemicals. Although this number is 9% lower than 2023, it still marks a significant achievement. In comparison, the annual tally never exceeded 36 in the 2000s, and this figure excludes gene therapies and other new treatments.

Some standout approvals include:

• Duvyzat, approved for Duchenne muscular dystrophy, which has garnered little press attention despite its success in helping patients rise to a standing position. Duvyzat is the first nonsteroidal medication approved to treat patients with all genetic variants of DMD. As a histone deacetylase (HDAC) inhibitor, it targets harmful processes to decrease inflammation and prevent muscle loss. This approval comes amid ongoing debates around gene therapies for the condition.
• Cobenfy by Bristol Myers Squibb, which introduced the first new class of treatments for schizophrenia in decades. In September, the U.S. FDA approved Cobenfy (xanomeline and trospium chloride) capsules for oral use to treat schizophrenia in adults. It is the first antipsychotic medication approved to target cholinergic receptors, in contrast to the long-established practice of targeting dopamine receptors.
• Winrevair by Merck, was FDA approved to treat pulmonary arterial hypertension, a rare, progressive, and life-threatening lung condition. This approval marks a major advancement for the approximately 40,000 U.S. patients living with the disease, as Winrevair is the first drug to target the condition’s root cause. It also represents a strategic win for Merck, which is working to diversify its revenue as its cancer drug Keytruda nears the loss of market exclusivity in 2028.

Eyes Open for More Developments: From CAR-T Therapy to Pig Organ Transplants

There are many exciting medical developments still in the works, offering hope for the future. The famous CAR-T therapy, which involves genetically modifying white blood cells, is now being tested to treat autoimmune diseases like lupus. Early results have sparked a rush of interest in biotech and this area.

Efforts to address the organ shortage have led to groundbreaking trials involving pig organs. This year, a gene-edited pig kidney was transplanted into a human for the first time. Unfortunately, the recipient passed away two months later. Advances in spinal cord injury treatment are also making waves. Electrical stimulation technology has shown significant progress, with the first FDA-approved device by ONWARD Medical offering new hope for patients.

Despite challenges, including high costs and unequal access to treatments, the rapid pace of medical innovation continues to impress. These breakthroughs show just how fast science is moving.

Author

Bernice Lottering