Experimental Drug Shows Promise for Some Patients with Advanced Prostate Cancer

Experimental drug may extend effectiveness of existing treatments in advanced prostate cancer patients. #ProstateCancer #MedicineNews

For men with metastatic castration-resistant prostate cancer, treatment options are limited as the disease progresses and standard therapies become less effective. A multi-institutional clinical trial led by researchers at the Medical University of South Carolina (MUSC) explored whether an experimental drug could extend the duration of existing treatments. The study, published in Cancer Medicine, focused on men whose cancer had already developed resistance to hormone-blocking drugs like abiraterone and enzalutamide.

"This is a very difficult-to-treat population," said co-author Besim Ogretmen, Ph.D., from MUSC Hollings Cancer Center. "These are patients whose cancer has become resistant to standard therapies, so there's a clear need for new options."

The experimental drug at the center of the study - opaganib - is based on foundational research led by Charles Smith, Ph.D., and advanced through years of laboratory and early-phase work at MUSC before transitioning into mid-phase clinical trials. This first-in-class therapy works differently from standard therapies; instead of targeting hormones, it blocks a pathway involved in sphingolipid metabolism - a process cells use to manage lipids that influence cell survival.

"Opaganib is not many drugs in the clinic," explained Michael Kucuk, M.D., who led the clinical trial. "That makes it very exciting and different from the treatments we currently use."

In a Phase 2 trial involving 66 patients, those receiving opaganib in combination with abiraterone or enzalutamide after their cancer had progressed showed some disease control at 16 weeks, although not reaching the study's primary goal. However, a closer look revealed that about 15% of patients taking opaganib with abiraterone and 9% with enzalutamide experienced clear biological signs of response, including drops in prostate-specific antigen (PSA) levels and periods of disease stabilization.

"These findings suggest the treatment may be slowing the disease and enabling patients to stay on the therapy longer," emphasized Ogretmen. "Even though it's a small percentage, those are real patients who are benefiting from this treatment when others have not worked."

The combination treatment was generally manageable with most side effects being mild to moderate. Some patients did experience more serious side effects, but these were often alleviated by adjusting the drug dose.

A key next step will be identifying biomarkers in blood samples that could predict response, allowing for a more precise approach tailored to individual patients - a hallmark of precision medicine.

"This work depends on strong collaborations between academic researchers, clinicians, and industry partners," Ogretmen added. "That's how we bring new therapies to patients who otherwise don't have effective treatments."

Looking ahead, the researchers are hopeful that refining this approach or developing next-generation drugs targeting the same pathway could expand treatment options, with growing optimism that these therapies could become part of future treatment strategies.

Publication details: Jacqueline T. Brown et al, Phase II Trial of Opaganib Addition in Metastatic Castration-Resistant Prostate Cancer After Disease Progression on Abiraterone or Enzalutamide, Cancer Medicine (2026).

Citation: Experimental drug shows promise for some patients with advanced prostate cancer (2026, May 15) retrieved from https://medicalxpress.com/news/2026-05-experimental-drug-patients-advanced-prostate.html

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