First-Ever Phase 1 Clinical Trial for Choroid Plexus Carcinoma (CPC) Opens for Enrollment

Dr. Mark Souweidane’s Phase 1 clinical trial using intra-arterial (IA) chemotherapy prior to second-look surgery is open for enrollment for patients diagnosed with choroid plexus carcinoma (CPC), the first such trial to specifically address this rare diagnosis. (See Intra-Arterial (IA) Chemotherapy for Newly Diagnosed, Residual, or Recurrent Atypical Choroid Plexus Papilloma (ACPP) and Choroid Plexus Carcinoma (CPC) Prior to Second-Look Surgery.) Intra-arterial chemotherapy is a minimally invasive procedure that delivers chemotherapy directly to the tumor via the dominant artery, thus greatly reducing systemic toxicity while enabling higher drug concentrations.

Choroid plexus carcinoma is a rare malignant brain tumor that develops in the ventricles of the brain, most often in children. Successful resection surgery directly correlates with more successful outcomes among the patient population, which is why this clinical trial explores an alternative for those children who end up requiring follow-up surgery in cases where gross total resection is not achieved, or who experience recurrence. This approach has proven to simplify second-look surgery for many other solid tumor types, the most comparable of which is now the standard of care for infants and children with ocular retinoblastoma (Rb). This IA approach is perfectly suited for CPC, which parallels Rb in its anatomy and potentially shares similar biological mechanisms with respect to their chemosensitivity to drugs. The drug combination being used in the clinical trial has already been drug screened against a CPC cell line in collaboration with the NIH and showed much promise, and it proves to have a low toxicity profile when delivered intra-arterially.

Principal investigator Dr. Mark Souweidane leads this concentrated effort to bring therapeutic drug delivery alternatives to clinic. His renowned success with leveraging alternative drug delivery methods, compounded with his preclinical work to identify drug combinations for the CPC clinical trial, puts WCM in an optimal position to host this trial.

Update: First patient treated in choroid plexus clinical trial

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