Our Mission

Precision medicine is starting to incorporate functional assays to evaluate anticancer agents on patient-isolated tissues or cells to select for the most effective. Among these new technologies, dynamic BH3 profiling (DBP) has emerged and extensively been used to predict treatment efficacy in different types of cancer. DBP uses synthetic BH3 peptides to measure early apoptotic events (‘priming’) and anticipate therapy-induced cell death leading to tumor elimination. This predictive functional assay presents multiple advantages but a critical limitation: the cell number requirement, that limits drug screening on patient samples, especially in solid tumors.

The story begins decades ago with a man named Stanley Korsmeyer, who led the molecular oncology program at Dana-Farber from 1998 until his death in 2005. He discovered that B-cell cancers like CLL over-produced a protein called BCL-2, and interfered with apoptosis, or programmed cell death. But how that went from an interesting discovery to a game-changing cancer drug is a story of persistence and momentum, and it’s the first episode of season two of Unraveled

The 4th Precision Oncology Symposium of the Comprehensive Cancer Center Zurich will bring together international leaders in the field of functional precision medicine including oncologists, cancer researchers and data experts to discuss latest developments and future directions.  Click the button below to register today!

Patients with non-Hodgkin’s lymphomas (NHLs) often relapse after frontline treatment, and interpatient heterogeneity make personalized combination treatment difficult. Goh et al. have developed a hybrid experimental-analytic method that they call quadratic phenotypic optimization platform, or QPOP, to identify personalized drug combination therapies using ex vivo patient samples to improve patient outcomes. In a prospective cohort, physicians were able to alter treatment according to drug combinations identified using QPOP after 6 days to achieve complete responses in 5 of 17 patients with NHL. This is a promising step for providing new hope for patients who have relapsed NHL and provides a foundation for further clinical trials.

A follow up to our October Seminar

The BCL2 inhibitor venetoclax has revolutionized the treatment of AML patients not benefiting from intensive chemotherapy. Nevertheless, treatment failure remains a challenge, and predictive markers are needed, particularly for R/R AML. Although, the ex vivo drug sensitivity testing has been used by several research groups, very few prospective trials have analyzed the correlation of drug sensitivity testing results to treatment outcome

VenEx is a prospective Phase 2 trial aiming to evaluate if ex vivo drug sensitivity testing could be used to identify AML patients who benefit from venetoclax + azacitidine therapy. This interim analysis of 39 first trial participants demonstrated that the experimental conditions significantly influenced predictive accuracy. Blast-specific venetoclax sensitivity measured in conditioned medium most accurately correlated with treatment outcomes; 88% of sensitive participants achieved treatment response. Median survival was significantly longer for ex vivo sensitive participants. This analysis illustrates the feasibility of integrating drug-response profiling into clinical practice and demonstrates excellent predictivity. The second stage of the trial is on-going to validate these results.

The American Society of Hematology welcomes SFPM President Dr. Tony Letai, Dr. Philipp Staber, & Dr. Caroline Heckman to discuss "Functional Precision Hematology" at their October 11 webinar.