“Experimental” Treatment—Finding Information and Making Decisions

Did you know that a treatment can be approved by the FDA and still be considered experimental? It happens more often than you might think and is a good example of why it is so important to ask your doctor questions.

After Lisa had a cancerous tumor removed from her breast, her surgeon performed a sentinel lymph node biopsy to determine if the cancer had spread from the primary site to her lymph nodes. This assessment is an important consideration in “staging” which is also based on the size of the tumor and whether it has metastasized or spread to other parts of the body. Staging helps guide treatment.

Lisa’s tumor was Stage 1, N0, M0—medical shorthand which means it was 3 centimeters (3/4 of an inch) or smaller across and had not spread to the lymph nodes or metastasized to other parts of her body. This was very good news since the survival rate for Stage 1 breast cancer is thought to be 100%.

Full breast radiation, 5 days a week for 5 to 7 weeks is standard treatment for this stage. And so I was surprised when Lisa called to ask if I knew anything about “balloon radiology,” a new treatment that delivers radiation seeds through a balloon catheter directly to the lumpectomy site, twice a day for only 5 days.

Apparently, this was something I had missed and I had a lot of questions. Was it approved or was her surgeon suggesting a clinical trial? How did it work? ? How effective is it compared with standard full breast radiation? Is it safe? What are the risks?

I started by searching the words “balloon radiology for breast cancer” on www.google.com. I found references to “brachytherapy” and “internal radiation” on the American Cancer Society site (www.cancer.org) and then used those terms to refine my search, looking for:

• “Clinical evidence for internal radiation in treating breast cancer”
• “Safety and efficacy of internal radiation in treating breast cancer”
• “Comparative safety and efficacy for internal radiation and full breast radiation”

I found what I was looking for on the websites of several major medical centers and other reputable cancer organizations. Along the way, I came across a New York Times article from October 2008 that discussed the product within the broader context of how some new treatments come to market.

Bottom line: The FDA approved the product several years ago based on limited clinical evidence that it is safe and effective. This fast track approval is given every year to thousands of products which the FDA says are mostly improvements of existing devices. In this case the rationale is that radiation is standard treatment for breast cancer and internal radiation has been used to treat other types of cancer.

I sent the information to Lisa with a note saying that the evidence on safety and effectiveness of the new device in a relatively small number of patients was promising but that the studies were ongoing. The true test will be how it compares with traditional full breast radiology, for which recurrence rates are 3 percent or less at five years.

I suggested that Lisa ask her doctor:

• How many of these procedures he has done over what length of time
• What he knows about data on comparative efficacy and the risks and benefits of internal radiation and full breast radiation
• Is he a clinical investigator for the product

My advice to Lisa was to have the discussion in person and to bring someone to take notes or record the conversation.

If it were me, I’d get a second or even a third medical opinion. But our circumstances are very different and in the end Lisa has to do what she thinks is best for her.

What would you do?

Here’s a link to a great tool for making difficult medical decisions: http://decisionaid.ohri.ca/docs/das/OPDG.pdf

The American Cancer Society also has a set of tools that are specific to various types of cancer: www.cancer.org/docroot/ETO/eto_0.asp

Later this week…more about the importance of asking your doctor questions and participating in decisions about your treatment.

Resources for Information About Breast Cancer

Susan G. Komen Breast Cancer Foundation: ww5.komen.org

MedlinePlus: http://www.nlm.nih.gov/medlineplus/breastcancer.html

National Cancer Institute: www.cancer.gov; telephone: 1-800-4-cancer

American Cancer Society: www.cancer.org; telephone: 1-800-ACS-2345