Jeffrey Beekman Ph.D.

University Medical Center Utrecht

Dr. J.M. Beekman leads a research group that focuses on the development and validation on individual readouts for therapy in CF, and identification of mechanisms controlling clinical heterogeneity between individuals. He has been trained in molecular and cellular biology within an immunological context, and became principle investigator in 2010, focussing of CF. His lab developed novel readouts to analyse CFTR protein expression and function in easily accessible patient tissues such as nasal airway epithelium, and recently developed a highly innovate functional CFTR assay in intestinal organoids that provides a patient-specific platform for analysis of CFTR residual function and CFTR-targeting drugs. His current work focusses on the use of this intestinal model for CFTR drug development and the application of this patient-specific model in understanding relations between the CFTR genotype, residual function and response to therapy. Currently, several subjects in the Netherlands with rare CFTR mutations now receive VX770 therapy because of the preclinical analysis of drug response in their intestinal organoids, indicating that a primary cell model from individuals can help to select treatments and obtain reimbursement by health care insurance companies. We currently move forward to develop biobanks of intestinal organoids of subjects with rare mutations and integrate such biobanks into clinical care by implementing individual drug efficacy screening programs. Another main goal of the lab is to investigate whether individual CFTR function measurements and drug responses in subjects with similar CF-causing mutations have complementary value next to CFTR genotyping as to more precisely indicate severity of disease or response to therapy.