PONTE

Program Description

Implementing Precision Medicine in clinical practice remains challenging, as clinical trials have failed so far to demonstrate overall survival advantages in unselected patient populations.  

We have therefore elected to profile orphan tumours, defined as rare cancers for which orphan-drug status might apply, including: classical rare cancers, rare histologies of common cancers, malignancies that currently have no defined molecular diagnostic paths, thereby excluding common cancers for which a standard molecular profiling is indicated. Drug access for driver/matched therapy will be prioritized as follows:

1. clinical trials available at the proponent and/or collaborating Institutions
2. ad hoc designed clinical trials (including clinical trials designed for recurrent - i.e. encountered in >2% of the retrospective dataset - potentially driver aberrations, see below),
3. off label drug use.

We aim at demonstrating a prevalence of potentially actionable drivers in at least 30% of the orphan tumours patient population, as defined above. We plan to retrospectively profile 800 cases (which will also be used to define recurrent drivers) and prospectively profile 1200 cases. Clinical data will be collected for both the retrospective and prospective populations.

Program Goals and Expected Outcomes

The goal of the project is to identify new treatment options for patients with cancers that currently do not have standard treatments options. By testing cancers that currently do not have many options for treatment because of either their rarity or lack of identified molecular driven options, hopefully, these patients can be directed to clinical trials or to treatments that may be already in use for other cancers. This could then potentially create new standards of treatment for these “orphan” cancers.

What gaps in existing knowledge will be addressed by the study?

This work is important to help identify gaps in the treatment options for these “orphan” cancers. Precision medicine in clinical practice remains challenging for these cancer patients, as clinical trials have failed so far to demonstrate overall survival advantages in unselected patient populations. By studying these cancers as part of ICGC ARGO, the treatment and outcome of patients with these cancers can potentially be improved and new diagnostic and treatment processes can be identified for all patients with these cancers in the near future.

Program Team

Aldo Scarpa, MD

Lead Investigator

Funding Organisations

Further Information

PROGRAM WEBSITE