SEEDING BPAN RESEARCH AT STANFORD
In November of 2024, Isabel’s Chance made a $125,000 grant to the Stanford School of Medicine to fund a pilot BPAN research project carried out by Dr. Juliet Knowles, MD, PhD, Dr. Rebecca Levy, MD, PhD and their dedicated team of researchers and clinicians. We are proud to be among the very first supporters of the Knowles Lab’s new beta-propeller protein-associated neurodegeneration (BPAN)-focused initiative, which will span years of research and include work to identify specific disease biomarkers (or biological sign posts) that will measure essential cell functions like autophagy (how cells replace themselves and manage waste) and ferroptosis (how cells react to stress), as a means of better understanding the broad spectrum of BPAN severity seen in kids suffering from the disease. This can, in turn, help us predict progression of the disease and determine whether potential treatments or other interventions are working.
This work will also include high throughput screening of existing drugs, including some which are already in use for children, to identify those that may hold benefit for BPAN patients. Using the results of these screenings, select drugs will be applied to BPAN cells to test for improved cellular health and function. This “off label” use of existing approved medicines to improve outcomes for BPAN patients would then be tested on mice.
The Stanford School of Medicine will then utilize mouse models to test promising drugs from the high throughput screen on BPAN-affected mice in an effort to determine what interventions may slow or even halt disease symptoms.
This critical work will aim to identify biomarkers and potential drug treatments and validate findings in mouse models in an effort to pave the way for future clinical trials in children with BPAN. It represents the only multiple-year study focused entirely on BPAN with an end goal of facilitating clinical trials in the western United States, of which we are aware, and Isabel’s Chance could not be more proud to help bring this work to fruition at a world-class private research university like Stanford.
