ASCO has revealed its first clinical trial, which will provide patients with limited options an opportunity to receive a potentially beneficial targeted therapy that matches their distinct genetic make-up and is FDA-approved for another type of cancer.
Richard L. Schilsky, MD
ASCO has revealed its first clinical trial, known as TAPUR, which will provide patients with limited options an opportunity to receive a potentially beneficial targeted therapy that matches their distinct genetic make-up and is FDA-approved for another type of cancer. The new initiative was announced during a press conference at the 2015 ASCO Annual Meeting.
The study hopes to simplify patient access to targeted therapies across many tumor types, including advanced solid tumor, multiple myeloma, or non-Hodgkin lymphoma that has a genomic variation known to be a drug target. TAPUR (Targeted Agent and Profiling Utilization Registry) focuses on community-based research programs where the majority of patients are treated.
"Oncologists often use therapies approved for a specific cancer indication to treat people with other types of advanced cancer, but we very rarely learn from that experience to benefit other patients," ASCO president Peter Paul Yu, MD, FACP, FASCO, said in a statement. "TAPUR will document the real-world experience of patients who receive commercially available targeted anti-cancer drugs and will describe the effectiveness and side effects of a range of targeted agents available in this study."
The primary objectives of TAPUR are twofold, explained ASCO’s chief medical officer, Richard L. Schilsky, MD, FACP, FASCO, at the press briefing: to improve access to potentially effective therapies for a much broader population than is typically enrolled in clinical trials, and to gather important information on the antitumor and toxicity of targeted drugs across multiple cancers.
"We are leveraging ASCO's unique ability to bring together a diverse group of collaborators to undertake something that's never been done before, all while simplifying access to multiple cancer treatments across many tumor types," Schilsky said in a statement. "Perhaps even more importantly, TAPUR will involve community-based research programs, where the majority of cancer patients receive treatment and will provide education and support to community oncologists to help them interpret complex genomic tests."
Although in some cases, the drug will be investigational and thus best administered in the context of a conventional clinical trial, “in other cases, given the large number of targeted therapies that are now commercially available, the best option for the patient might be to receive a commercially available targeted drug, but one that would have to be prescribed outside of its FDA-approved indication,” a process that can be very complicated and burdensome.
Even when patients do get access to these agents, said Schilsky, “we have no mechanism right now to learn the experience of that patienthow the patient did, whether they responded or not, whether they had side effects—that information is never captured in any organized way that we as an oncology community can learn from.”
Five pharmaceutical companies (AstraZeneca, Bristol-Myers Squibb, Eli Lilly and Company, Genentech, and Pfizer) have signed on to provide their currently marketed targeted drugs at no charge to participants, and more are expected to join the effort, noted ASCO President Peter Paul Yu, MD, FACP, FASCO, in a statement. He added that “at least 13 drugs that target more than 15 genomic variants will be provided by these companies.”
Two technology companies, Syapse, who will automate the study workflow, and NextBio, who will support and inform the case review, will provide data analysis.
Patient advocates also will be providing guidance and oversight support for TAPUR. Jane Perlmutter, PhD, a cancer survivor and nationally recognized patient advocate, will help coordinate patient advocate recruitment and training for the initiative.
“TAPUR has enormous potential to improve our understanding of the effectiveness of currently available therapies in treating cancers with genomic variations and to learn from patients who are treated with off-label drugs,” said Perlmutter in an ASCO statement, adding that the program will be especially helpful “for those with advanced cancer for whom traditional therapies are no longer working.”
ASCO also is collaborating with the Netherlands Center for Personalized Cancer Treatment, which is conducting a clinical trial using a study protocol very similar to TAPUR.