New treatments for kidney cancer ... and VHL?

Happy Spring!

Even though it's still pretty blustery in Boston, the crocuses are beginning to bloom, bringing a sweet message of love and hope.

There are also some hopeful messages in the press -- we are cautiously optimistic with a stress on caution. We need your help to bring the hope to fruition.

New compound from Bayer

In December 2005, the FDA approved a Bayer compound known as Nexavar, a new anti-cancer medicine used to treat adults with advanced renal cell carcinoma, the most common type of kidney cancer. While in clinical trials, Nexavar was known by the code name BAY 43- 9006. FDA press release re Nexavar

New compound from Pfizer

In January the U.S. approved a new "targeted" therapy for use against two types of cancer: gastrointestinal stromal tumor (GIST), a rare form of stomach cancer, and advanced kidney cancer.

Approval for advanced renal cell carcinoma is based on partial response rates and duration of responses. There are no randomized trials demonstrating clinical benefit such as increased survival or improvement in disease-related symptoms in renal cell carcinoma. FDA press release re Sutent

What Does This Mean for VHL?

Both theses compounds are in a category that would be expected to be useful with VHL. At the moment, however, they are only approved for METASTATIC disease -- that has spread to other organs outside the kidney.

Very few people with VHL have participated in clinical trials up to this point. People with VHL generally have at least one tumor outside the kidney, making their cases difficult to add cleanly into a study of the effect of the drug on renal cell carcinoma (RCC). At this point, however, the key questions we all have are: will these drugs shrink VHL tumors and prevent metastasis? is it safe to take these drugs for more than a few weeks? what other side effects might there be when you take the drug for a longer period of time?

Reimbursement

There is another key question: will my insurance company reimburse for this treatment? Unless the product label says "approved for use with VHL", insurance companies may well consider its use with VHL to be "investigational."

The Road to Approval -- or not -- for VHL

If you and your physician feel that one of these compounds might be the thing for you to try, please help our entire community by making sure that what you and your doctor learn becomes part of a central collection of information about using these drugs with VHL. By compiling our learning together we will either learn their limitations, or we will gain approval for the drug "for VHL" and thus earn reimbursability. Because we are a small community, we need to hear from EVERYONE's experiences with the drugs, and we need that feedback to be structured in a way that we can add it all together and figure out what's going on.

Here's the plan:

• Ask your doctor to contact Dr. David F. McDermott, Clinical Director, Biologic Therapy Program, Dana Farber/Harvard Cancer Center Renal SPORE program. Dr. McDermott will be happy to discuss your case with physicians, but not with the patient directly. He is also in touch with a number of other clinical trials which might be even more appropriate for the patient's particular situation.
  

• If patients have questions, please ask them to call the VHL Family Alliance Hotline. We will do our best to answer questions, or will seek answers and reply.

Dr. McDermott is working with the pharmaceutical companies and other clinical researchers to set up a "protocol", a set of guidelines for the administration of the drug, so that everyone using it will be doing so in a consistent way, and our learning will be structured. For people outside North America, please give your physicians the same referral to the VHLFA or to Dr. McDermott. We will all need to work together globally to learn enough to gain approval for VHL for any drug.

Kidney Cancer Symposium

I also want to let you know about an upcoming Regional Kidney Cancer Symposium at the Boston Marriott Copley Place on April 7, 2006. This meeting is geared towards community medical oncologists, urologists, and their nursing staffs. It is hoped that our clinicians and researchers will be able to provide that group a comprehensive update on what is going on in the arena of kidney cancer treatment and research. You will find the brochure on the SPORE website. Medical professionals can earn up to 8.5 CMEs from Harvard Medical School.

Links:

See the Renal SPORE website

Program

Registration Form

We thank you very much for your help in helping us all learn from your experiences and finding a cure for VHL!

VHL Alert, March 2006 - register free to receive periodic alerts at www.vhl.org