What is the TAPUR (Targeted Agent and Profiling Utilization Registry) Study, with Richard Schilsky, MD, FACP, FASCO

Cancer.Net Podcast

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ASCO’s first clinical trial is the Targeted Agent and Profiling Utilization Registry, or TAPUR Study. This clinical trial is intended for people with advanced cancer without other treatment options available, and whose cancer has at least one genomic variation that can be targeted with specific drugs.

In this podcast, Dr. Richard Schilsky discusses the TAPUR study and explains why it is significant. He also discusses what participants can expect. Dr. Schilsky is the Principal Investigator for the TAPUR study. He is also the former Chief Medical Officer for ASCO and Professor Emeritus at University of Chicago.

View Dr. Schilsky’s disclosures at Cancer.Net.

Dr. Schilsky: Hi, everyone. My name is Richard Schilsky and I'm the principal investigator of the ASCO TAPUR Study and the former Chief Medical Officer of ASCO. I'm happy to give you an overview and update about the study today. By the way, TAPUR is an acronym that stands for Targeted Agent and Profiling Utilization Registry. Hopefully, the reason for naming it that will become clear as you listen. The TAPUR study was conceived in 2013 and launched in 2016, and was based on the observation that there was a rapid increase in testing the tumors of patients with advanced cancer for gene mutations that might be contributing to the growth of the tumor, so-called genomic profiling, in the hope of finding a genomic alteration that could potentially be treated by a drug that was already FDA-approved for a different tumor type than what the patient had.

Meaning, in order for the patient to receive the drug, it would have to be prescribed off-label. The challenge with prescribing the off-label use of a drug is that most insurance plans don't cover the cost of treatment. Additionally, even if the patient were able to receive the drug, there was no mechanism for the oncology community to learn from the patient's treatment experience.

The TAPUR study has managed to address these challenges by providing access to FDA-approved drugs at no cost to the patient and providing treatment results to the oncology community regarding the effects of off-label use of the treatments being studied. Now, TAPUR is a clinical trial, and its primary objective is to describe the anti-tumor activity and toxicity of commercially available targeted anti-cancer drugs prescribed for treatment of patients whose tumors have a genomic alteration known to be a drug target or to predict sensitivity to a drug.

TAPUR was designed to be simple for providers and patients. It's a phase 2 study, meaning that we're aiming to learn about efficacy and safety. It’s prospective, that is, it enrolls patients going forward. It is not randomized. Everybody gets a treatment based on the genomic profile of their tumor and the available treatments in the study. It's a multi-basket study. That is to say, multiple therapies are available on the study that are targeting multiple genomic alterations. And it's a pragmatic study. TAPUR attempts to replicate routine clinical care. It's exempt from FDA oversight. It provides oral drugs that can be shipped directly to the patient's home after the first visit.

Now, as I said, the TAPUR study was launched in March of 2016. And as of this month, it's still going strong, with more than 2,700 patients having been enrolled at 267 locations in 28 states. So how does the study work? Well, a patient's physician has results of a genomic profile of the patient's tumor and determines that a study drug might benefit the patient. The patient then decides to participate in TAPUR and gives their informed consent. A molecular tumor board, which is a group of experts convened by ASCO, is available to consult regarding the proposed treatment or to provide alternative treatment options for the patient. A participating pharmaceutical company, and there are 10 right now, provides the study treatments at no cost to the patient.

The patient is cared for by their own oncologist, receives a standard dose of the drug, and is evaluated at standard intervals to see if the treatment is working and if they're having any side effects. ASCO has convened an independent data and safety monitoring board of cancer experts that periodically reviews results and determines whether treatment is promising for a particular cancer type and genomic alteration. That's what we call a cohort in the study. Once the data are finalized, ASCO publishes the study findings in peer-reviewed journals to inform clinical practice and future research.

So let me give you an example. There are specific molecular alterations that often appear in tumor cells that are important for driving the growth and progression of the cancer and can be targeted with specific drugs that interrupt those abnormal molecular pathways. Many of these alterations occur at low frequency, meaning in less than 5% of tumors of any given type. The benefit of the TAPUR trial having a basket design is our ability to evaluate multiple therapies simultaneously to target multiple low-frequency alterations, which ultimately offers more treatment options to patients who wish to participate in the study.

If the TAPUR study were set up looking to target only a single genomic alteration, we would potentially have to screen hundreds of patients in order to find one who is appropriate for the trial, which also means hundreds more would still be left without treatment options. But because TAPUR evaluates multiple treatments and multiple genomic alterations simultaneously, we found that about two-thirds of patients who were screened for the trial ultimately enroll.

A specific example of a drug and targeted gene alteration on TAPUR is the use of the treatment combination pertuzumab plus trastuzumab in tumors with ErbB2 amplification or mutation. Now, you may be aware that ErbB2 is a gene that is synonymous with the HER2 gene that is frequently amplified or overexpressed in patients with breast cancer. And this drug combination, pertuzumab and trastuzumab, is FDA-approved for treatment of patients with breast cancer. But in the TAPUR study, we found multiple tumor types outside the FDA-approved label that can benefit from this treatment if an ErbB2 alteration is detected, including patients with colorectal cancer, endometrial [uterine] cancer, biliary tract cancer, and lung cancer.

To learn more about TAPUR, please follow our progress at the ASCO website. In an effort to provide up-to-date information about cohorts that are available for enrollment on the TAPUR study, ASCO launched a public-facing status report in March of 2023. So first click on www.tapur.org. Click on the link to the ASCO website. From there, select study participation at the bottom of the page. Once at the study participation page, click on the link to see a list of study cohorts that are currently enrolling. The report updates daily, providing viewers with an up-to-date list of available study cohorts based on their genomic alterations. It's important to note that study cohorts are available on a first-to-enroll basis. You can also find information about current results from the TAPUR study on the study results page.

So what have we learned so far? Thus far, we've publicly reported results on 29 cohorts of patients. 17 gave a positive signal of treatment activity, 12 were negative. Now we feel it's just as important to report on the negative results as the positive results. If the treatment is unlikely to be effective for patients, it's important to inform the oncology community because all of the drugs in the study are commercially available and could be prescribed to a patient.

Enrollment to patients on TAPUR is very representative of the U.S. population. The study has broad eligibility criteria that allows more patients to enroll, including patients with an ECOG performance status of 0 to 2 and younger patients. Some treatments allow for adolescent patients as young as age 12 to be enrolled in the study.

We hope the oncology community finds value in the TAPUR study. Physicians have the opportunity to contribute to research and participate in publications and to contribute more knowledge in the field of oncology. TAPUR provides guidance on interpreting genomic reports via the molecular tumor board and provides additional treatment options for patients. Institutions obtain insights on potential new uses of existing drugs and their side effects, and TAPUR data can inform updates to clinical practice guidelines. And patients receive access to drugs not available as standard of care. Patients may be able to receive oral drugs at their home and limit their commute to clinic.

And of course, participation in the study provides an opportunity for patients themselves to contribute to knowledge about cancer treatments. To find a clinical site offering the TAPUR study, please visit the TAPUR website again, www.tapur.org and select “Participating Centers.” This will lead to a searchable map of participating sites and includes the site-specific contacts. Contact the primary contact listed for that site. Thank you for listening to this update on the ASCO TAPUR study and enjoy the rest of your day.

ASCO: Thank you, Dr. Schilsky. Learn more about clinical trials, including the TAPUR Study, at www.cancer.net/clinicaltrials.

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