Genomics-based clinical trials are speeding the development of new targeted therapies. They sort potential research participants by genetic criteria, and match them with the experimental therapy that targets the particular molecule associated with their disease. Genetic profiling shortens the time it takes to enroll patients and increases the chance that patients will benefit from the therapy being studied.
“Patients are waiting too long to enter clinical trials for drugs that may or may not be effective for their specific variation of cancer,” says Charis Eng, MD, PhD, chair of the Cleveland Clinic Genomic Institute. “End-stage cancer patients especially may not have that time. By identifying the molecular profile of a patient’s tumor or by looking for heritable mutations in the patient, we can get them to targeted clinical trials that have a higher chance of positively impacting their lives.”
In the traditional randomized control trial, researchers enroll a large number of patients and randomly assign them to receive the experimental therapy or a placebo (or current standard of treatment). This sturdy model has been the gold standard since 1961. But it is proving inadequate to the complex and fast moving world of genetics and gene-based therapies.
The development of genomic-based clinical trials is most advanced in the field of cancer treatment. A number of models have evolved to meet the needs of patients and researchers. One type studies the effects of targeted therapies on different genetic variations of a single cancer type (the basket approach). Other genomics-based trials may examine how targeted therapies affect one genetic variation across multiple cancer types (the umbrella approach). Another important category which is rarely recognized is offering trials based on the genetic change in the individual.
Biostatisticians and researchers are continually refining genomic-based clinical trials to increase their speed, specificity and flexibility. Genomics-based clinical trials are an innovation that not only enables other innovations to be tested and flourish as they deserve, but benefit patients directly by steering those with rare cancers or late-stage disease to the experimental therapeutic that gives them the best chance when they need it most – right now.
Where Are They Now
As of 2016, molecular characterization programs using next generation genomics-based technologies have been established in many cancer centers worldwie. The method of testing and treating based on genomics continues to gain popularity and has been expanded for a variety of applications.