New Trial to Expand Patient Access to Off-Label Use of Targeted Drugs for Many Types of Cancer


ASCO's first-ever clinical trial aims to match patients who have run out of treatment options with therapies that are FDA-approved to treat tumors with their genetic variant but for a different type of cancer.

Richard L. Schilsky,


When patients have run out of treatment options, but they have a known genetic variant that may be responsive to a drug that has been approved to treat a different tumor, these patients—and their healthcare providers—may understandably wonder, “How do patients get the drug that is suggested by their tumor’s profile?”

In its first-ever clinical trial, ASCO hopes to provide an answer to that question.

ASCO announced the trial, known as TAPUR (Targeted Agent and Profiling Utilization Registry), in a June 1st press briefing at its 2015 Annual Meeting. The nonrandomized clinical TAPUR trial will involve patients with any advanced solid tumor, multiple myeloma, or non-Hodgkin lymphoma that has a genomic variation known to be a drug target.

“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,” noted Jane Perlmutter, PhD, a cancer survivor and nationally recognized patient advocate, in an ASCO statement.

Perlmutter is helping to coordinate patient advocate recruitment and training for the initiative, which she said will be especially helpful “for those with advanced cancer for whom traditional therapies are no longer working.”

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.

“Increasingly, we find that patients with advanced cancer who no longer have any standard treatment options are having a genomic profiling test performed,” noted Schilsky in explaining the rationale for the initiative. “These tests are now readily available…and sometimes what’s known as ‘a potentially actionable variant’ is detected.” Estimates in the literature are highly variable, he added, but about 40% to 70% of the time these tests are likely to turn up something that the doctor might be able to act on.

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 patient—how 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.”

Schilsky said that ASCO will submit its completed trial protocol and consent form to an institutional review board in July, and the organization hopes to begin patient enrollment by the end of this year.

He said the patient eligibility criteria are intentionally broader than that of a typical clinical trial. The study will enroll patients with advanced solid tumors, B-cell non-Hodgkin lymphoma, and multiple myeloma who are not responding to standard anticancer treatment or for whom no acceptable treatment is available. Patients will be screened to determine if they have adequate organ function to participate based on broad inclusion/exclusion criteria.

Any genomic test that is available to the physician in clinical practice, as long as it is performed in a CLIA-certified/CAP-accredited laboratory, will be enough to qualify a patient to enter the study, Schilsky said.

If a patient meets the trial criteria, his or her physician can select a drug from among those available in the TAPUR study protocol that targets the genomic variation in the patient’s tumor. Physicians can consult one of ASCO’s three oversight committees set up for the initiative, the Molecular Tumor Board, for guidance on potential therapies on or off the study, if a relevant drug match is not described in the protocol. ASCO also will have a Steering Committee and Data and Safety Monitoring Board to oversee the effort.

All patients who receive treatment through TAPUR will be monitored for standard toxicity and efficacy outcomes, including objective response rate per RECIST criteria—the study’s primary endpoint—as well as time on treatment and progression-free and overall survival.

Schilsky explained that the unit of evaluation in the study will be a “tumor type genomic variant drug group.” The plan is to enroll eight patients per group; if no responses are observed, enrollment in that group will be stopped, but if there is at least one response, enrollment will be expanded up to a total of 24 patients.

“The idea here is to detect a signal of drug activity…we hope that signals of activity will result in pharmaceutical companies, the clinical oncology research community, or others pursuing those signals,” he continued.

TAPUR will be launched initially at three clinical sites—the Michigan Cancer Research Consortium, the Cancer Research Consortium of West Michigan, and the Carolinas Health System—with the ultimate goal of expanding the effort nationally.

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.”

More information about the program can be found at

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