Tales from the bench Inside UCSFs new public lectures series

Professor Joseph DeRisi discussed the future of rare disease research at UCSF's inaugural public lecture series, focusing on Balamuthia mandrillaris, a brain-eating amoeba. He highlighted the challenges of diagnosing and treating this rare but fatal infection, which often goes unnoticed. DeRisi's lab employs genetic sequencing and metagenomics to identify pathogens in patients' samples, which can lead to targeted treatments. He emphasized the lack of funding for rare diseases, as organizations like the CDC are disincentivized to invest in conditions affecting few individuals. To address this, researchers often conduct drug repurposing screens to find existing medications that may be effective.

DeRisi's lab found that nitroxoline, although not approved in the U. S. , shows promise against Balamuthia. The lecture attracted about 50 attendees, including one-third without a scientific background, fostering community engagement. DeRisi's insights aim to bridge the gap between scientific research and public understanding.