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During the 2022 OTS annual meeting in Phoenix it was easy to listen to the talks announcing successful clinical trial results and assume that obviously favorable data were becoming routine.
Willeke van Roon-Mom, Chantal Ferguson, and Annemieke Aartsma-Rus have just published a commentary (Nucleic Acid Therapeutics, 33, 234, 2023) that reminds us that many clinical trials will have much more complex outcomes.
Specifically, the authors share the fascinating story behind the recent approval of Tofersen. High stakes – the desperate need for a treatment that can help a subset of ALS patients – confront the complexity of developing a drug for a disease that is both rare and incompletely understood. Doing nothing to help patients should not be an option, but achieving high confidence in a clinical trial outcome was not possible either.
Diseases with high unmet need have unmet need for a reason – there are daunting barriers to drug development. The article provides a fascinating glimpse into the evolving story of how to translate the potential of nucleic acid therapeutics into strategies for overcoming these barriers.
