Innovative Drug Delivery System Shows Promise for Treating Rare Eye Cancer

A groundbreaking study led by Moffitt Cancer Center reveals that a novel drug delivery system may significantly improve treatment outcomes for patients suffering from a rare form of eye cancer known as metastatic uveal melanoma, particularly when it has metastasized to the liver. This form of cancer has historically been challenging to manage, often resulting in poor prognoses for affected individuals.

The research, part of a phase 3 clinical trial dubbed the FOCUS trial, was published in the Annals of Surgical Oncology. The trial compared the efficacy of a melphalan hepatic delivery system against standard treatment protocols. Patients receiving the melphalan treatment demonstrated markedly better results compared to those on conventional therapies. Specifically, the median progression-free survival for patients treated with the melphalan system was recorded at 9.1 months, a substantial improvement over the 3.3 months experienced by those receiving standard care.

In addition to improved survival rates, the objective response rate for the melphalan group was 27.5%, nearly three times higher than the 9.4% seen in the control group. The disease control rate also showed significant improvement, rising from 46.9% to 80% for patients utilizing the new treatment. Furthermore, the median overall survival for patients treated with the melphalan delivery system was 18.5 months, compared to 14.5 months for those undergoing traditional treatment methods. Although some side effects were observed, primarily related to blood cell counts, these were manageable through standard outpatient care and typically resolved with simple observation.

According to Dr. Jonathan Zager, a surgical oncologist at Moffitt and lead author of the study, the results of this trial provide renewed hope for patients facing this difficult-to-treat cancer. This marks the second publication concerning the FOCUS trial in the Annals of Surgical Oncology, reinforcing the potential of the melphalan hepatic delivery system in managing liver metastases in uveal melanoma.

Approved by the U.S. Food and Drug Administration in August 2023, the melphalan hepatic delivery system distinguishes itself from conventional chemotherapy by administering a concentrated dose of the drug directly into the liver. This process involves isolating the liver using a series of catheters and balloons during percutaneous insertions, allowing for the filtration of the chemotherapy agent before it enters the bloodstream.

This targeted methodology significantly minimizes adverse effects on the rest of the body, offering a more tolerable treatment option for patients. Ongoing research is planned to further explore the benefits of this delivery system, including potential combinations with other emerging therapies.