Some call it “Phase 4,” the moment of truth for any new therapy. These are clinical trials, the last crucial step before a promising treatment can become a reality for patients. And 2025 promises to be a particularly rich year of clinical trials with disruptive potential, from the treatment of genetic diseases like sickle cell anemia to new weapons against tumors, to the use of psychedelics to treat mental disorders.
Futuro Prossimo has its own watchlist, and the 4 searches I'm going to show you are among those at the top of the list: shall we take a look together?
BEACON: Gene editing against sickle cell anemia
The first of the clinical trials to watch in 2025 is BEACON, which is testing a pioneering gene therapy for sickle cell anemia. This genetic disease, caused by a mutation in the hemoglobin gene, turns red blood cells into sickle shapes, causing painful crises and progressive organ damage.
The experiment uses a technique of genetic editing call "basic editing” to correct the genetic defect directly in the patients’ own hematopoietic stem cells. Taken from the blood, these cells are modified in the laboratory to produce an “anti-sickle cell” form of hemoglobin, and then reinfused into the patients.
The first results are expected in February: if positive, they will pave the way for a potential one-shot cure for this serious disease, and more generally demonstrate the feasibility of gene editing to treat inherited diseases.
Lutetium-177: The Future is in Radioactive Drugs?
The second one clinical trial to be followed carefully is the one that is evaluating the effectiveness of the Lutetium-177, an innovative radioactive drug for the treatment of prostate cancer. Already approved as a “last line” therapy, it is now also being tested in the early stages of metastatic disease.
How does it work: Lutetium-177 is linked to a molecule that selectively binds to an antigen (PSMA) expressed on prostate cancer cells. Once injected, the drug accumulates in tumors and “bombards” them with targeted radiation, sparing healthy tissue.
The study in progress, on over 1.100 patients, could lead to a more widespread use of this promising therapy, and more generally confirm the potential of ultraspecific “oncological radiopharmaceuticals”. New weapons that are emerging as one of the most promising frontiers in the war on cancer.
Schizophrenia and psychosis, the CBD gamble
Turning to psychiatric diseases, the third of the “hot” clinical trials of 2025 is STEP, a multicenter study which is evaluating the efficacy of cannabidiol (CBD) in the treatment of psychosis in schizophrenia.
CBD, a non-psychoactive compound derived from cannabis, has already demonstrated anticonvulsant and anxiolytic properties. Now, three parallel clinical trials will test whether it can also alleviate psychotic symptoms., in combination or as an alternative to traditional antipsychotics, in patients at different stages of the disease.
With nearly 1.000 participants in 11 countries, it will be one of the largest studies ever conducted on the therapeutic potential of CBD in serious mental disorders. And it could pave the way for safer, more targeted treatment options for a highly disabling condition.
Tailored Breast Cancer Screening
Finally, I cannot fail to mention “My Personal Breast Screening“, the first large-scale clinical trial to evaluate the effectiveness of personalized breast cancer screening based on individual risk.
Currently, screening programs are based only on age, with suboptimal results: many false positives, few lives saved. The idea is to “tailor-make” surveillance, using genetic data, family history and breast density to stratify women based on risk and modulate the frequency and type of tests accordingly.
The study, involving over 50.000 women in 6 countries, could revolutionize prevention strategies for the most frequent tumor in the female population. A paradigm shift in line with precision medicine: no longer “one size fits all”, but “the right therapy (and screening) for the right person at the right time”.
Clinical trials, these and others: fingers crossed and eyes wide open
All that remains is to carefully follow the results of these pioneering experiments. Four small, great revolutions that, if they keep their promises, could change the lives of millions of patients for the better, and perhaps inspire a new generation of clinical trials increasingly innovative and ambitious.
Research, after all, never stops: who knows what other surprises the future of medicine will have in store for us this year. We'll be here to tell you about them.
