The PIONEER Initiative stands for Precision Insights On N-of-1 Ex vivo Effectiveness Research. The PIONEER Initiative is designed to provide access to functional precision medicine to any cancer patient with any tumor at any medical facility. Tumor tissue is saved at time of biopsy or surgery in multiple formats, including fresh and cryopreserved as a living biospecimen. SpeciCare assists with access to clinical records in order to provide information back to the patient and the patient's clinical care team. The biospecimen tumor tissue is stored in a bio-storage facility and can be shipped anywhere the patient and the clinical team require for further testing. Additionally, the cryopreservation of the biospecimen allows for decisions about testing to be made at a later date. It also facilitates participation in clinical trials. The ability to return research information from this repository back to the patient is the primary end point of the study. The secondary end point is the subjective assessment by the patient and his or her physician as to the potential benefit that this additional information provides over standard of care. Overall the goal of PIONEER is to enable best in class functional precision testing of a patient's tumor tissue to help guide optimal therapy (to date this type of analysis includes organoid drug screening approaches in addition to traditional genomic profiling).

Population

The primary target study group is all patients with a cancer serious enough to put the patient at risk of morbidity or death.

Brief Title

The PIONEER Initiative: Precision Insights On N-of-1 Ex Vivo Effectiveness Research Based on Individual Tumor Ownership (Precision Oncology)

Official Title

The PIONEER Initiative: Precision Insights On N-of-1 Effectiveness Research. Tissue Ownership by the Individual With the Return of Actionable Information to the Individual Patient and Physician (Precision Oncology)

Brief Summary

The PIONEER Initiative stands for Precision Insights On N-of-1 Ex vivo Effectiveness <br /> Research. The PIONEER Initiative is designed to provide access to functional precision <br /> medicine to any cancer patient with any tumor at any medical facility. Tumor tissue is saved <br /> at time of biopsy or surgery in multiple formats, including fresh and cryopreserved as a <br /> living biospecimen. SpeciCare assists with access to clinical records in order to provide <br /> information back to the patient and the patient's clinical care team. The biospecimen tumor <br /> tissue is stored in a bio-storage facility and can be shipped anywhere the patient and the <br /> clinical team require for further testing. Additionally, the cryopreservation of the <br /> biospecimen allows for decisions about testing to be made at a later date. It also <br /> facilitates participation in clinical trials. The ability to return research information from <br /> this repository back to the patient is the primary end point of the study. The secondary end <br /> point is the subjective assessment by the patient and his or her physician as to the <br /> potential benefit that this additional information provides over standard of care. Overall <br /> the goal of PIONEER is to enable best in class functional precision testing of a patient's <br /> tumor tissue to help guide optimal therapy (to date this type of analysis includes organoid <br /> drug screening approaches in addition to traditional genomic profiling).

Detailed Description

The PIONEER Initiative is a lead clinical trial designed to provide the foundation for
subsequent adaptive trials. The mission with this and subsequent trial activity will be to
demonstrate the utility of carrying out functional precision medicine in that cohort of
patients who receive local cancer care at institutions that do not have significant research
capabilities or are not NCI-designated cancer centers. As discussed above, the vast majority
of cancer patients (~85%) are in this category. It is imperative to open up the possibility
of best-in-class functional precision medicine testing to these patients. Core aspects of the
PIONEER Initiative include recruiting a cadre of up to 200 patients, showing that proof of
concept in a more limited set of patients can ultimately scale to arbitrarily large numbers
of patients. It is anticipated that PIONEER will continue as the foundation for subsequent
clinical trials into the indefinite future. This evolving trial structure fills an important
unmet need within the broader cancer community since the primary mission is the return of
actionable information in order to positively impact care.

The underlying basic assumption of the PIONEER Initiative is that the ability to receive the
best in cancer care should not be restricted as to location, age, or medical condition. The
PIONEER Initiative design facilitates inclusion of subjects across all these divides, thus
providing beneficence to all participants. A key aspect of the PIONEER Initiative is to
provide patient benefit through a data and tissue biorepository to be utilized by researchers
to discover new cancer diagnostics, treatment therapies and preventive strategies.
Additionally, the data and samples will be used to improve existing treatments and to
understand, at the molecular level, changes occurring during transition from illness to
remission and/or relapse. SpeciCare, through the PIONEER Initiative, will store de-identified
self-reported data, medical records, and biological samples from individuals who consent to
participate in PIONEER. PIONEER will obtain further study and testing as appropriate and
return results, through CLIA certified labs, through research vetted by CLIA testing, and, as
enabled by current regulatory standards, through delivery of research results back to the
patient and patient's clinical care team.

SPECIFIC OBJECTIVES

1. To establish a data and tissue biobank comprised of high quality biological specimens
and associated clinical data that will support drug discovery, diagnostic assay
development, oncology biomarker discovery, molecular biology, immuno-oncology therapies,
translational medicine and other future discoveries as novel methods become available.

2. To facilitate and accelerate oncology research, including genetic research (e.g. whole
genome sequencing), by providing a pipeline of high quality specimens and associated
clinical data for researchers to access.

Outcome Measures

Outcome measures are the tests that investigators perform to prove whether or not a treatment being tested in a clinical trial is having any effect.