Refractory haematological malignancies carry poor prognoses when standard therapies fail. Ex-vivo drug sensitivity testing offers a functional approach to personalize salvage regimens. Clinicians at the University of Malaya evaluated the Optim.AI™ precision oncology platform in five heavily pretreated patients with refractory blood cancers. The cohort included cases of Burkitt lymphoma, B-cell acute lymphoblastic leukemia (B-ALL), diffuse large B-cell lymphoma, and Hodgkin lymphoma. Researchers isolated live tumor cells and tested them against drug combinations designed via an orthogonal array composite design. The Quadratic Phenotypic Optimisation Platform (QPOP) measured cell viability, delivering actionability scores within two weeks.
Key Clinical Outcomes
Treating physicians selected salvage regimens based on QPOP rankings, clinical status, and drug availability. The functional precision oncology platform guided significant clinical remissions:
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Burkitt Lymphoma Salvage Therapy: A 24-year-old patient achieved complete remission following a QPOP-guided regimen of gemcitabine, dexamethasone, cisplatin, and venetoclax prior to stem cell transplantation.
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Refractory B-ALL Remission: A 20-year-old patient achieved minimal residual disease (MRD)-negative complete remission using venetoclax-based combination therapy followed by allogeneic stem cell transplantation.
Across evaluable patients, median progression-free survival reached 108 days, with ongoing responses extending to 340 days.
Therapeutic Timing and Efficacy
Despite achieving complete remissions, two patients with advanced disease eventually experienced progression. As highlighted by the authors, “our findings suggest that greater clinical benefit may be achieved when Optim.AI™ recommendations can be implemented before exhaustion of available therapeutic options”. Functional ex-vivo testing provides vital strategic guidance, but early clinical integration remains essential to maximize patient benefit.