A gallstone-related drug, already approved
in Asia and Europe, could enter human clinical trials soon as an
experimental way to block Type 1 diabetes before patients even develop
symptoms.
In a paper published Monday in the Journal of Clinical Investigation, researchers at Stanford University and Seattle's Benaroya Research Institute
at Virginia Mason Hospital said a study of the drug — called
hymecromone — blocked production of a substance that is essential for
the development of Type 1 diabetes. Although the study was in mice, they
believe the drug ultimately could be used to stop a disease that
afflicts one in 300 people in the United States.
Whether the
experimental drug is able to wend its way through the
multibillion-dollar, multi-year drug-approval process remains to be
seen. But the study, funded by the Juvenile Diabetes Research Foundation
and the National Institutes of Health, could lay the foundation for
preventing millions of so-called pre-diabetics from joining the ranks of
the 1.25 million Americans with Type 1 diabetes.
Type 1 diabetes,
also known as juvenile diabetes, is an autoimmune disease, where the
pancreas stops producing insulin, a hormone that converts sugars into
energy. More common Type 2 diabetes, in contrast, is a metabolic
disorder in which insulin still is produced but the body can't use it
effectively.
The lab of Dr. Paul Bollyky a Stanford assistant professor of infectious diseases who led the team
exploring hymecromone in Type 1 diabetes, is prepping a human clinical
trial of the drug. It also has received preliminary funding from SPARK, a
Stanford program designed to transform bench research into bedside
drugs and diagnostics.
Beyond the commercial possibilities, hypercromone is exciting scientifically.
Early in Type 1
diabetes, scattered clusters of human pancreas cells, called islets,
become inflamed when otherwise-protective immune cells attack. The
attack eventually spreads to beta cells, halting the production of
insulin.
By the time Type 1 diabetes can be identified, some 90 percent of pancreatic beta cells already have been killed off.
Ron Leuty, San Francisco BTs
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