Tuesday, June 2, 2015

Clinical Trial Provides Access to Targeted Drugs To Patients Who Have Exhausted Conventional Therapy

This is very exciting and long awaited news. I have seen too many people who have exhausted all their options, and who were physically eligible for a new, targeted treatment, only to be turned down because it has not been approved for metastatic use or they are excluded because of all their previous therapies. 

When you have  no more choices, access to potentially life extending drugs is your only hope. Finally, ASCO understands this and five pharmaceutical companies are on board. This is a major breakthrough for advanced disease patients. All solid tumor and blood cancer patients are included. I have had friends with advanced lung and breast cancer beg for compassionate access only to be denied because of red tape. This trial will change that. Bravo ASCO and thank you.

ASCO Launches First-ever Clinical Trial: Aims to Learn from Patients with Advanced Cancer Who Lack Standard Treatment Options Five Leading Pharmaceutical Companies Will Support Innovative Study, Contribute Drugs

CHICAGO – The American Society of Clinical Oncology (ASCO) today announced its first-ever clinical trial that will offer patients with advanced cancer access to molecularly-targeted cancer drugs and collect “real-world” data on clinical outcomes to help learn the best uses of these drugs outside of indications approved by the Food and Drug Administration (FDA). Plans for the Targeted Agent and Profiling Utilization Registry (TAPUR) study, including the participation of major pharmaceutical companies that will contribute free drugs, were released in a news briefing at the Society’s 2015 Annual Meeting in Chicago.

The ASCO-sponsored prospective, non-randomized clinical trial will collect information on the anti-tumor activity and toxicity of commercially available, targeted cancer drugs in a range of cancer types, including any advanced solid tumor, multiple myeloma, or non-Hodgkin lymphoma with a genomic variation known to be a drug target.

“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,” said ASCO President Peter Paul Yu, MD, FACP, FASCO. “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.”
ASCO will organize the operational aspects of the study, including the participation of multiple collaborators that are central to TAPUR’s success. TAPUR will involve not only patients and physicians, but also ASCO oversight committees, pharmaceutical companies, technology firms, and community-based study sites—representing a uniquely innovative and inclusive approach to studying the use of molecularly-targeted cancer drugs.

“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,” said ASCO Chief Medical Officer Richard L. Schilsky, MD, FASCO. “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.”

TAPUR Next Steps
In the coming months, an Institutional Review Board will review the study protocol and consent form.  In addition, ASCO has established three oversight committees—each of which will include patient representatives, clinical oncologists, statisticians, and genomics specialists:

    •    Steering Committee to oversee study operations, establish data sharing and publication policies, review plans to add or remove drugs from the study, and approve participation of clinical study sites;
    •    Molecular Tumor Board to review the proposed drug-target match and suggest therapies on or off the study;
    •    Data and Safety Monitoring Board to regularly review study results to ensure that severe or unexpected adverse events are carefully monitored, to determine when enrollment of study cohorts should expand or cease, and to determine when to release data and to which parties.

Patient Participation
TAPUR is designed to include a broader patient population than is typically enrolled in clinical trials. It will accept patients who have any advanced solid tumor, multiple myeloma, or B cell non-Hodgkin lymphoma and are no longer responding to standard anti-cancer treatment or for whom no acceptable treatment is available. Patients will be screened to determine if they are healthy enough to participate based on broad inclusion/exclusion criteria.

If and when a patient meets the defined trial criteria, his or her treating physician will select a drug from among those available in the TAPUR study protocol that targets the identified genomic variation in the patient’s tumor. If a relevant drug-target match is not described in the protocol, a physician may consult the Molecular Tumor Board which will review the clinical and genomic features of the case and suggest potential therapies on or off the study. All patients who receive treatment through TAPUR will be monitored for standard toxicity and efficacy outcomes including tumor response, progression-free and overall survival, as well as duration of treatment.

Patients participating in TAPUR will receive the anti-cancer drugs at no charge. It is expected that routine clinical care costs will be covered by the patient’s insurance plan.

Participating Organizations ASCO has invited a number of pharmaceutical companies to provide marketed, targeted drugs and additional resources to support the development of the new study’s infrastructure. At the time of this announcement, ASCO reported that the following companies have signed memoranda of understanding agreeing to participate in the TAPUR study:

    •    AstraZeneca
    •    Bristol-Myers Squibb
    •    Eli Lilly and Company
    •    Genentech
    •    Pfizer

“At least 13 drugs that target more than 15 unique genomic variants will be provided by these companies. We are extremely grateful for the generosity of these companies without whose support TAPUR would not be possible,” said ASCO President Dr. Yu. “We anticipate additional companies will sign on, and are extremely encouraged with the level of interest we have received so far.”
ASCO will launch the TAPUR study at clinical sites that comprise the Michigan Cancer Research Consortium, the Cancer Research Consortium of West Michigan, and the Carolinas Healthcare System—existing research networks that run research trials for the National Cancer Institute and industry—with the ultimate goal of expanding nationally.

Two technology companies will provide key support to manage, analyze, and interpret the study data: Syapse will provide its Syapse Precision Medicine Platform to automate the study workflow and the Molecular Tumor Board process, and capture structured data from participating practices. Illumina will provide its NextBio knowledge base platform to support and inform the case review by the Molecular Tumor Board, as well as to support analysis of the TAPUR data by the study team.
Finally, the Society will collaborate and share data with the Netherlands Center for Personalized Cancer Treatment, which is conducting a clinical trial using a study protocol very similar to TAPUR.
“We are very fortunate that this leading cancer center has the same focus as TAPUR,” said Dr. Schilsky. “Technological advancements will allow us to pool our information in a seamless fashion and give us the ability to learn from the experience of a larger group of patients.”
Patient Advocates to Play Key Role in TAPUR

Patient advocates will play a central role throughout the study, providing guidance and oversight support. Jane Perlmutter, PhD, a cancer survivor and nationally recognized patient advocate, is lending her expertise in trial development and will help coordinate patient advocate recruitment and training for the study.

“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 Dr. Perlmutter. 

“I applaud ASCO for undertaking this important study and believe its findings will improve cancer care, especially for those with advanced cancer for whom traditional therapies are no longer working.”

For more information about TAPUR, please go to http://www.asco.org/TAPUR.

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