Phase 2 International Clinical Trial
Multiple Centers (originally led by Memorial Sloan Kettering Cancer Center)
What began as a proposal for a clinical trial in four centers, has expanded into a worldwide clinical trial.
In 2016, the TLC Foundation, in collaboration with like-minded nonprofits A Kids’ Brain Tumor Cure and Solving Kids’ Cancer, provided the seed money for a phase 1 clinical trial for children with brain tumors using combination immunotherapy that targets controlling factors in the body’s immune system called checkpoints for a total investment of $185,000.
What began as a proposal for a four center US-based clinical trial of a promising combination therapy for children battling brain tumors, quickly expanded into a large-scale phase 2 clinical trial at 58 centers in 13 countries, bringing new hope to children across the globe who have run out of treatment options.
This collaborative funding model and potential impact of the new therapy attracted the attention of pharmaceutical giant Bristol Myers-Squibb, and led to a major expansion of the project into the International Phase 2 clinical trial that just completed enrollment in 2019, and is now in the process of analyzing the data with the goal of presenting the findings before the end of the year.
Checkpoint inhibitors are therapeutic antibodies that block the inhibitory receptor signaling and restore the capacity of T-cells to kill cancer. The trial targeted two types of checkpoint receptors – (PD1 and CTLA4) using a combination of antibodies that targeted the immune system rather than the cancer itself. Checkpoint combination therapy has been the most exciting breakthrough in cancer research this decade. Adults with previously untreatable cancers like metastatic melanoma and lung cancer have been cured in clinical trials.
Had it not been for these three nonprofits trusting in this idea, this clinical trial may have never come to fruition. By working together, nonprofits and medical institutions can move the needle forward faster. An update report from January 2019 can be found here. Information on the trial can also be found at clinicaltrials.gov.
