Update on Clinical Trials

See updates marked in red below

So far there is still no “magic bullet,” but we are all searching!

The trials I know about are the following. If you know of another one, please let me know.

(1) Novartis PTK-787, trial headed by Dr. Patrick Wen at Dana Farber Cancer Research Institute in Boston. To qualify, you have to have at least one measurable lesion of the brain, spinal cord, or eye, which cannot be treated by normal treatments without causing you harm. This drug has had good response in the kidney, but this particular trial is designed to measure the effectiveness of PTK with tumors of the central nervous system. This trial is currently OPEN and recruiting patients. Contact Dr. Wen +1-617-632-2166 or one of his nurses Debra Conrad Gigas, RN +1-617-632-6327, Lisa MacDonald, NP +1-617-632-5925.

(2) Novartis PTK-787, headed by Dr. Daniel George at Duke University in North Carolina. To qualify, you have to have at least one measurable lesion of the brain, spinal cord, or eye, which cannot be treated by normal treatments without causing you harm. This drug has had good response in the kidney, but this particular trial is designed to measure the effectiveness of PTK with tumors of the central nervous system. This trial is currently OPEN and recruiting patients. Contact Trish Creel, Clinic Research Coordinator, 919-668-7531, fax, 919-668-7117, or paticia.creel@duke.edu. The trial requires a 3-4 day stay in the Duke area for screening, a 24 hour stay to start the trial, and every 2 week visits for the first two months, when visits them become every 4 weeks. Going into the 3rd month, there is a 2 day stay required every four weeks.

(2) Novartis PTK-787, headed by Dr. Daniel George at Duke University in North Carolina. To qualify, you have to have at least one measurable lesion of the brain, spinal cord, or eye, which cannot be treated by normal treatments without causing you harm. This drug has had good response in the kidney, but this particular trial is designed to measure the effectiveness of PTK with tumors of the central nervous system. This trial is currently OPEN and recruiting patients. Contact Trish Creel, Clinic Research Coordinator, 919-668-7531, fax, 919-668-7117, or paticia.creel@duke.edu. The trial requires a 3-4 day stay in the Duke area for screening, a 24 hour stay to start the trial, and every 2 week visits for the first two months, when visits them become every 4 weeks. Going into the 3rd month, there is a 2 day stay required every four weeks.

(3) Bayer 43-9006 is doing well in trials with kidney cancer. Patients with brain lesions have been excluded from the kidney cancer trials. There is a proposal to open a study of Bayer 43-9006 with other VHL tumors in Philadelphia and Boston which is wending its way through the forest of approvals. This trial is NOT CURRENTLY OPEN but is expected to open by early 2005. People being seen at the CCC’s at University of Pennsylvania (+1 (215) 662-4740) or Mass. General Hospital (+1 617-724-1971) may wish to discuss this possibility with those CCC’s.

(4) The Urologic Oncology Division of NIH under Dr. W. Marston Linehan has now opened a pre-surgical therapy for kidney cancer. This initial study will be limited to a small number of people with VHL who need kidney surgery. Patients will receive 17AAG from InvivoGen, an analog of geldanamycin, once a week for three months intravenously in Bethesda, then have a month off, and then the surgery will help us learn whether the drug is achieving the right response within the tumor. If this phase is successful, the study will be opened more widely. To inquire, contact Dr. Marston Linehan, +1.301.496.6353. The time commitment is significant, but NIH will pay travel costs for U.S. residents accepted into the trial. People from other countries may apply, but would be responsible for their own travel expenses. Click for more information

Most of the other kidney cancer trials are currently limited to people with metastatic kidney cancer, as we are still learning safety information about many of these drugs.

(5) Avastin from Genentech is in the category of angiogenesis inhibitors expected to have some usefulness with VHL. It has recently been approved for use with colon cancer, but only in combination with certain chemotherapy drugs. A trial of Avastin and Interferon compared to Interferon alone has opened within CALGB institutions, and trials of Avastin and Interleukin-2 are being discussed but will be limited to patients with metastatic (Stage IV) kidney cancer. See www.calgb.org for participating institutions.

Eye Lesions:

(7) Lucentis (previously named rhuFab) from Genentech, a drug being used for Age-related Macular Degeneration (AMD) is in the process of gaining approved for use with AMD. It is currently being controlled carefully for use ONLY with AMD, in order not to complicate its approval for AMD. Dr. Emily Chew at NIH is opening a trial of Lucentis for VHL. This trial is OPEN AND RECRUITING PATIENTS and you are welcome to contact Dr. Chew about the trial. This is an angiogenesis inhibitor, injected directly into the eye, and the study is limited to testing the effectiveness of the drug on eye tumors. Because of the way it is administered, it will likely have little or no effect on other tumors. Inquire with Dr. Emily Chew or her coordinator, Katherine Shimel at +1.301.402.2863 See newer information

(8) Macugen from EyeTech is also an injectable into the eye, being tested for AMD. Once this is approved for AMD, it is expected to be made available for trial with other conditions. The “wet” form of AMD is caused by leaky blood vessels under the retina , similar in many ways to the process experienced in VHL. See article page 12. This trial has been completed at NIH and publications are emerging. Lucentis is expected to be more effective.

Clinical Trials index

As printed in the VHL Family Forum  12:1, March/April 2004.  For permission to reprint, please contact VHL Family Alliance, editor@vhl.org. Further information is available from the VHL Family Alliance, info@vhl.org.