Finding the right patients for the right treatments and vice versa.
Rare cancers are in fact not so rare. 20-25% of all cancers are rare, with over 500 rare cancers affecting more than 7 million people in the EU and US alone. The major challenges for patients and doctors are finding the right treatments and clinical trials, while for pharma companies it's a major challenge to find the right patients for clinical trials, and understanding how existing drugs can be repositioned, or identifying new drug targets. Most rare cancers do not have approved treatments, and many are treated with off-label or non-approved drugs. Information locked inside the patient's medical records when combined with BioXplor's broader oncology knowledgebase can be used to find new insights on rare cancer patients and there response to existing treatments, to better select the right patients for clinical trials, and to support repositioning of existing drugs or discovery of new drug targets for rare cancers with unmet medical needs.
BioXplor applies it's data-driven platform to accelerate and derisk drug repositioning including combination drug candidates. Patient data is required to match treatments to responder populations. Drugs are identified and assessed for licensing potential before being prioritized for preclinical validation in cell and animal models. To achieve this, BioXplor partners with rare disease patient groups, academic teams and pharmaceutical companies. BioXplor works with patients to collect data from electronic medical records, genomics sequencing results, patient reported insights and wearables data via a custom designed rare disease-specific patient app.
As part of an ongoing collaboration with Prof. Jody J. Haigh's cancer research group at the Department of Therapeutics and Pharmacology at the University of Manitoba / CancerCare Manitoba. We are studying a rare and particularly aggressive form of Leukemia (ETP-ALL). BioXplor set out to analyse the ZEB2 pathway to identify optimal drug combinations. Our team identified a novel drug combination for ETP-ALL with strong supporting evidence to modulate the known disease causing proteins, and the combination has previously shown safety and efficacy in another indication. This drug combination is being validated in vitro and in vivo using patient tissue and patient derived tumor models.
Case Study - Rare Leukemia Drug Combinations