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, a trial uses base editing to edit four genes in immune cells to amp up their cancer-hunting capabilities. Another study, BEACON, launched a few years back, is testing whether base-edited blood stem cells can tackle severe sickle cell disease, with initial results expected in February 2025.
In sickle cell disease, a genetic mutation transforms oxygen-carrying red blood cells from smooth, donut-like shapes into cells with sharp edges. The disease eventually destroys blood vessels and causes pain.
The BEACON trial base edits blood stem cells—dubbed HSCs (hematopoietic stem cells)—to correct the faulty genes. These cells eventually develop into all of our blood cells, including immune blood cells, and are critical for treating blood disease.
BEACON is open-label and single-arm, meaning all patients are getting treatment, and they know. During the trial, HSCs are taken from each person and given a gene variant that boosts fetal hemoglobin—a protein that carries oxygen in red blood cells. Increasing levels of the protein should improve symptoms.
The trial faces headwinds with a reported death in early results. But the death was attributed to side effects of busulfan, a drug used to create space in the bone marrow—a standard procedure before transplant—rather than the base editing itself. If successful, the trial opens the door to treating other inherited diseases and pushes the technology closer to clinical use.
A Cancer Throwdown With Radioactive Drugs
Prostate cancer creeps up. However, with screening, it can be detected early. Cancer cells are dotted with a protein dubbed PSMA, which has been a target for therapies tackling the disease.
After over a decade of research, one molecule stood out: lutetium-177. Also known as Pluvicto, the radioactive drug grabs onto PSMA once injected into the body and emits damaging levels of radiation directly onto cancerous cells. First approved by the FDA in 2022 for prostate cancer that has already spread, the drug significantly improved survival and quality of life.
Pluvicto was initially okayed for treatment after chemotherapy. Now, an ongoing trial, PSMAddition, is asking if early treatment may yield better results.
In over 1,100 patients with minimally treated prostate cancer that has spread, the trial is testing early treatment in a particular population of patients. Specifically, prostate cancer patients usually undergo hormone therapy to combat the disease, but in some people, the treatment could also lower their responsiveness to Pluvicto.
Positive results would be a “potential game-changer for hundreds of thousands of patients with prostate cancer globally,” Oliver Sartor, who’s leading the trial, wrote in Nature Medicine.
A Component of Weed Tackles Psychosis
Despite being federally illegal, psychedelics are having a moment. CBD, a component of both weed—which isn’t traditionally considered psychedelic, but can have similar effects—and hemp has already been approved by the FDA for treating seizures in kids two years or older.
A new clinical study called Stratification and Treatment in Early Psychosis (STEP) hopes the molecule could also help people with psychosis from schizophrenia or other disorders. Mostly based in the UK, the study consists of three placebo-controlled, double-blind randomized trials—the gold standard in clinical trials.
As a Phase 3 study, the final step before requesting approval, each trial will gauge the effect of CBD with or without anti-psychotics in people with different stages of psychosis.
One trial is working with people who’ve had just one episode; another includes those who’ve experienced psychosis resistant to drug treatments. The last trial is preventative, studying patients who are at high risk of developing psychosis. With blood tests, questionnaires, and brain imaging, the team aims to gauge how well the participants respond to CBD.
It’ll be one of the largest studies of CBD to date, coordinating 30 sites in 11 countries and recruiting roughly 1,000 participants. The study will also look for biomarkers that could potentially predict treatment success. Researchers expect first results in 2025, and hope the trial can shed a light on the potential therapeutic effects of CBD in severe psychiatric disorders.
Is Personalized Breast Cancer Screening Coming Your Way?
Breast cancer is far too common. For now, screening guidelines are one-size-fits-all. Generally, they’re based on age—beginning at roughly 50 years of age in most countries. But the tests have limited efficacy, reducing death risk by just 20 percent.
Part of this is due to family history. Each person has an individual risk depending on genetics, lifestyle, and other environmental factors. For people with potentially lower risk, mammograms may not be needed even if they fit the screening bill. Meanwhile, for women at high risk, more intensive screening could better capture cancerous cells.
One trial, called My Personal Breast Cancer Screening, is looking to make breast cancer screening more personalized based on risk. The largest global study to date, the trial has launched in six countries with over 53,000 women. It’ll compare the health outcomes of women that either follow current breast cancer screening recommendations or those that receive a personalized screen.
To tailor treatment, the team will use participants’ genetic data to assess risk, in combination with other factors, such as family history and breast density. They’ll follow the women and note whether, or when, they develop breast cancer four years after the screen. If successful, the strategy could help those at high risk while lowering unnecessary harm and screening burden for people with low risk.
These are just glimpses of medical therapies in the works. There’ll be plenty more to cover in 2025. As usual, it was a great year geeking out with you. Thanks for reading—and looking forward to sharing what this year has to offer!
Image Credit: Elsa Olofsson on Unsplash
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