Navigating through the complex web of clinical trials

Our stories and blogs about new treatments invariably raise questions that pertain to different clinical situations being experienced by our readers. Would this new treatment apply to my condition? How would I get access to this new drug through a clinical trial? The answer is complicated.The determination of who qualifies for an experimental trial, known as "eligibility criteria," can look like a series of roadblocks based on the stage of disease, prior therapies and a long list of blood tests that must return in a certain range. Some of the criteria are becoming even more complex because trials are being designed more from a biological standpoint. In fact, some trials are now addressing a particular gene mutation as opposed to a type of tumor. The rationale for this is that biology may trump the tissue of origin – we are getting more clues every year about specific tumor alterations that make them sensitive or resistant to targeted drugs. Also, genetic testing of tumors has become routine and quick.This trend is also spilling into clinical practice as newer gene and protein tests are becoming commercially available. In some cases, these test are being used "ahead of the data," or in other words, before we have definitive proof that a given treatment indicated by the test is really better that standard treatment. Imagine that a case of breast cancer that has not responded to the usual hormonal and chemotherapeutic drugs is found to express a protein found in gastrointestinal sarcoma tumor (GIST), which responds to the chronic myelogenous leukemia drug Gleevec. There are really no studies to verify that this strategy would work, but it certainly is very appealing. According to most guidelines, there are very few genetic tests that are actually "approved" as being proven to help with treatment decisions. Even BRCA testing for the breast and ovarian cancer susceptibility gene is not currently used to choose drugs in patients with cancer, but it is, of course being used to identify high-risk patients who need enhanced surveillance or even preventive surgery. For clinical trials, however, BRCA testing in patients with cancer is being used for some PARP inhibitor trials based on the biology of these drugs, which appear to inhibit DNA repair. These drugs might be more effective in patients who carry mutations in BRCA, which impairs DNA repair and might therefore predict extreme sensitivity to PARP inhibitors. However, we don't yet know that PARP inhibitors work only in BRCA-deficient cells, so this whole approach is investigational. So how is one in general supposed to navigate the complex web of choosing trials and knowing if one is eligible in the first place? CURE offers some resources to help with clinical trials, and websites are becoming better as search tools, such as www.clinicaltrials.org or www.breastcancertrials.org (specifically for breast cancer). You can search through various clinical trial websites in our Toolbox listing. You can also review our Patient's Guide to Clinical Trials for more information.It is probably not helpful to have unapproved protein or genetic tests performed on your tumor unless it is being done as part of a clinical trial to test for eligibility. However, it is very likely that newer tests will become more widely available and useful in the very near future.