Clinical Trials

Early Results

There are no published results yet from the clinical trials of the new angiogenesis inhibitors, but nonetheless early responses are encouraging.

One patient has had shrinkage of kidney tumors. One patient saw his brain tumors shrink nearly 50% in volume in three months.

The most exciting response has been the lessening of edema around some brain tumors. VHL tumors have a tendency to “recruit fluid” around them, causing the tissues to soak up fluid like a sponge. Since it’s not a cyst, and you can’t just drain off the fluid, the doctors really have no treatment for it. It causes increasing pressure and increasing symptoms even though the tumor remains the same size. One patient in the PTK trial had significant reduction in this edema, with an accompanying improvement in his symptoms.

Side effects reported remain quite tolerable -- some people experience fatigue, a little wooziness, and sometimes some nausea which can be controlled with medication.

This is the only clinical trial specifically designed to test the effectiveness of a new drug with VHL. Over the past four years, PTK has been tested with nearly 1000 patients in trials for various types of cancer. It is doing very well with kidney cancer, and is now in Phase III testing in colorectal cancer. It is being combined with additional agents in early Phase I and II studies. The fact that it works on both the VEGF and PDGF pathways is making it much more effective than earlier drugs, and the hope is that by adding agents that work on a third or fourth associated pathway, the combination will be even more effective.

To Inquire:

Contact Dr. Patrick Wen, +1 (617) 632 2166, or ask a local oncologist about access to trials involving Novartis PTK-787, Bayer 43-9006, or other inhibitors of VEGF, PDGF, and HIF-1-alpha. Currently the only trial specifically for VHL is in Boston with Dr. Wen. A second site at Duke University is expected to open by June 2004. Participants can begin in Boston and transfer to Duke when it opens.