A brief update on the GVAX trial – and what’s next?

Dr. Lipson told us today that the final subject in the Phase 1 GVAX melanoma vaccine trial that Robert went through at Johns Hopkins was enrolled two weeks ago. Around the end of the year or early in 2014, the researchers will have all the data from the study and will begin to write a report that they hope will be published in the middle of next year. Although it’s too soon for them to begin speculating about the results of the study, they are noting an increase in the immune response when they look at the biopsies taken after the final injection. So ... something’s happening there! Whether it’s enough to provide a viable therapy for lots of melanoma patients remains to be seen – but at least it’s a start.

We talked briefly about the research presented at the June meeting of the American Society of Clinical Oncology. Researchers and product developers are making progress – particularly with therapies for Stage IV patients, those with active, advanced melanoma that has spread beyond the primary site and its surrounding area. Clinical oncologists are using ipilumimab (known as ipi and marketed by Bristol-Myers Squibb as Yervoy®), an approved therapy that works against cytotoxic T-lymphocyte antigen-4 (CTLA-4), to prolong the lives of melanoma patients with advanced or unresectable disease.

A number of researchers are studying products that work against programmed death protein 1 (PD-1). One study reported this summer in which BMS used ipi together with its anti-PD-1 antibody, nivolumab, appears to have produced  even better results than those reported for ipi when it was approved two years ago. Another anti-PD-1 therapy, lambrolizumab (under development by Merck), also has done well in Phase 1 studies, according to reports published in July.

Less publicized, Dr. Lipson said, is research that’s still in the proof-of-concept stage, looking for tests that could offer some relief to patients at Stage II or III who don’t have advanced disease and aren’t eligible for treatment with ipi or studies of anti-PD-1 agents. In a nutshell, here’s the problem those people (including Robert) face: no one knows whether they are cancer-free after their previous treatments, or whether instead there are some micro-metastases floating around in their bodies somewhere looking for an opportune time and place to grow. You don’t want to use toxic therapies to treat patients who don’t have active disease unless you know there’s a real threat their disease will progress. But so far, no one has come up with a test that says who should be treated and who can go on with life as before because the melanoma is gone.

This research doesn’t appear to offer the golden egg – a cure for metastatic melanoma, which could save the lives of so many people and save their loved ones the misery of watching them die. What it offers instead is real peace of mind for melanoma survivors who are cancer-free but have no idea whether the ugly beast will invade their lives again.

The costs of carrying out these studies are high, and funding for proof-of-concept research doesn’t come easily. But why shouldn’t a project like this receive attention from donors as well? After all, it offers very real relief to tens of thousands of melanoma patients who are already cured but don’t know it.