Breakthroughs in medicine are exciting. They promise to alleviate human suffering, sometimes on global scales. But it takes years, even decades, for new drugs and therapies to go from research to your medicine cabinet. Along the way, most will stumble at some point. Clinical trials, which test therapies for safety and efficacy, are the final hurdle before approval.
Last year was packed with clinical trials news.
Blockbuster medications Ozempic and Wegovy still dominated headlines. Although known for their impact on weight loss, that’s not all they can do. In an analysis of over 1.6 million patients, the drugs seemed to block 10 obesity-associated cancers—including those of the liver, kidney, pancreas, and skin cancers. Another trial over one year found that a similar type of drug slowed cognitive decline in people with mild Alzheimer’s disease.
Meanwhile, scientists dug into how psychedelics and MDMA fight off depression and post-traumatic stress disorders. The year was a relative setback for the psychedelic renaissance, with the FDA rejecting MDMA therapy . But the field is still gaining recognition for its therapeutic potential.
Then there’s lenacapavir, a shot that protects people from HIV. Named “ breakthrough of the year ” by Science , the shot completely protected African teenage girls and women against HIV infection. Another trial supported the results, showing the drug protected people who have sex with men at nearly 100 percent efficacy. The success stems from a new understanding of the protein “capsule” guarding the virus’ genetic material. Many other viruses have a similar makeup—meaning the strategy could help researchers design new drugs to fight them off too.
So, what’s poised to take the leap from breakthrough to clinical approval in 2025? Here’s what to expect in the year ahead. Base Editing Takes a Shot at Sickle Cell Disease
Base editing is a type of gene editor, like the genetic Swiss Army knife CRISPR-Cas9. Developed in 2016, base editing nicks a single DNA strand—rather than cutting both strands—making it far less likely to damage untargeted parts of the genome.
In previous years, base editing teamed up with CAR T therapy to destroy cancer cells. Led by Beam Therapeutics, […]
Four Clinical Trials We’re Watching That Could Change Medicine in 2025
