Redesigning Clinical Trials
It feels as though it is time to post another geek-y blog. I greatly appreciate those of you who check in regularly and know that your interests are varied. This is from the NCI Cancer Bulletin and is a transcript of a conversation with Dr. Patricia Steeg on redesigning clinical trials to attempt to better prevent metasteses in breast cancer.
As we all know, primary (non-metastic) breast cancer is very treatable, often curable, and not a lethal disease in and of itself. It becomes a lethal disease if it spreads outside of the breast and axillary nodes. At that point, called metastatic or Stage IV or advanced breast cancer, it is treatable, but not curable. There is a tiny (two to three percent) group of women with advanced breast cancer who live for years and years with stable disease. I know one of them, a terrific woman who sometimes attends my weekly group for women with advanced cancer. She was initially diagnosed with Stage IV (it had spread to her bones) inflammatory breast cancer more than 14 years ago. Since then, she has been treated with weekly herceptin and Taxol, and her disease has been rock solid stable. A great story!
Generally, the goal of treatment for metastatic breast cancer is twofold: to prolong life as long as possible and to maintain the best possible quality of life. The strategy is serial treatments. That is, any one drug is given for as long as it is effective, and, when the cancer cells figure out how to resist it and to grow again, the treatment is changed to something different. There is a long list of possibilities, but some drugs have more side effects than others, and women are forced to adapt to a very different life than they would have hoped to have.
That is all background to this article. The goal of all breast cancer research is how better to treat (or even prevent) the disease. Once breast cancer has been diagnosed, the goal is clearly to prevent its spread. Dr. Steeg suggests that designing clinical trials in different ways would better succeed in learning how to prevent metasteses. Her proposal is that women who are at high risk for developing metastatic cancer be enrolled in trials to test new agents, not that these trials are reserved for women who already have Stage IV disease. It is, I think, an interesting perspective.
Here is the beginning and then a link to read more:
A Conversation with Dr. Patricia Steeg on Redesigning Clinical Trials to Test Therapies that Could Prevent Cancer Metastasis
In a perspective published May 30 in Nature, Dr. Patricia Steeg argues that the current system for clinical trials must be redesigned if there is to be a decline in breast cancer metastasis, which is the leading cause of death from the disease. Dr. Steeg, who heads the Women's Cancers Section in NCI's Laboratory of Molecular Pharmacology, recently spoke with the NCI Cancer Bulletin about the challenges in developing treatments that prevent metastasis and her proposal for a new clinical trial design to test such therapies.
How big a problem is metastatic breast cancer, and would your proposal apply to other cancers besides breast cancer?
This year, nearly 40,000 women will die of breast cancer, and metastatic disease is the largest contributor to these deaths. Although the annual number of breast cancer deaths is declining, it's not declining by much.
The proposal I've put forth should apply to a number of different cancers, particularly those where the majority of patients are diagnosed before they have full-blown metastatic disease, or if they have limited, treatable metastatic disease. One could imagine applying this to prostate, bladder, and colon cancers.
Why is treating metastatic cancer so challenging? Are there any effective, approved treatments?
First off, metastasis research is tough to do. There is no in vitro, or lab bench, model for metastasis, so you have to use animals and the experiments are long and complex. Most of our animal model data show that we can prevent the formation of metastases. Taking those preclinical findings and validating them in a clinical trial is just about impossible.
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