top of page


La Jolla Light and San Diego Business Journal recently reported upon the approval of Qalsody (tofersen). See links below or the pdf file.

La Jolla Light:

San Diego Business Journal:

FDA OK’s New ALS Drug - San Diego Business Journal
Download PDF • 143KB

Tofersen, a drug pioneered by Dr. Smith alongside Ionis Pharmaceuticals (Frank Bennett), and UCSD (Don Cleveland), was approved by the FDA for the treatment of one form of familial ALS. The approval of Tofersen is not only a milestone in the history of ALS, but also a milestone for the Center which has now been involved in the development of 2 out of the 5 drugs available for ALS.

Pictured above is the UCSD and CNS SOD1 Antisense team. Not pictured: Dr. Bennett (Ionis Pharmaceuticals). From left to right: Tim Miller, Dr. Smith, Don Cleveland, Melissa McAlonis-Downes.

Above is an early photo of the antisense team that was taken outside of Ionis's original laboratory in Carlsbad. Pictured from left to right: Tim Miller, Don Cleveland, Brett Monia, Tom Condon, Dr. Smith, Frank Bennett.,FDA%20Grants%20Accelerated%20Approval%20for%20QALSODY%E2%84%A2%20(tofersen)%20for%20SOD1,a%20Genetic%20Cause%20of%20ALS&text=CAMBRIDGE%2C%20Mass.%2C%20April%2025,GLOBE%20NEWSWIRE)%20%2D%2D%20Biogen%20Inc.

Alzheimers & Dementia: The Journal of the Alzheimer's Association has accepted "Pathogenesis of Alzheimer's Disease: Involvement of the Choroid Plexus" for publication. This paper is further support for a novel idea proposed by Dr. Smith and his colleagues that was published in 2019.

bottom of page