For years, Sonia Vallabh has been working to save her life. Vallabh has a type of brain illness called a prion disease — specifically, one called fatal familial insomnia.
Since the illness killed her mother and a genetic test showed she carried the same mutation, she and her husband, Eric Minikel — both prion researchers at the Broad Institute — have been working to find a possible treatment. But details about their work had been kept quiet until last week, when they revealed their years-long partnership with Ionis Pharmaceuticals (IONS).
What the California-based company is giving Vallabh and Minikel are a bunch of potential treatments. Ionis has been sending them small strands of RNA sequences known as antisense oligonucleotides. The therapeutic potential of these strands comes from their ability to change how proteins are handled in cells; they can prevent the body from producing a malfunctioning protein or encourage it to produce more of a necessary one. Vallabh and Minikel have been taking these strands and testing them in a standard mouse model of Vallabh’s illness.
This article is exclusive to STAT+ subscribers
Unlock this article — plus in-depth analysis, newsletters, premium events, and news alerts.
Already have an account? Log in
About the Author Reprints
To submit a correction request, please visit our Contact Us page.
