In the summer of 2021, the FDA approved Biogen’s novel gene therapy treatment for Alzheimer's Disease. Aduhelm (Aducanumab) works by selectively binding amyloid fibrils and oligomers that contribute to Alzheimer’s (AD) development (Cummings et al., 2021). During the human trials, about 165 patients with mild AD received the therapy intravenously. Results showed that amyloid plaques were decreased in all brain regions examined with a 22% decrease in cognitive decline (Haddad et al., 2022). Sounds promising right?
It was originally approved for all forms and stages of AD, but FDA had updated Aduhelm’s label a month later to only approve it for mild/early forms of AD. You may think this treatment will be a smash hit since Biogen is projected to have a $6 billion revenue by 2025, but physicians are very cautious about prescribing it to their patients. Was it because the research wasn’t compelling enough? Or is it due to tragic gene therapy cases that happened decades earlier?
You’ve probably heard about some failed gene therapy treatments in the late 1990’s and early 2000’s that resulted in a general halt of gene therapy research. In a quest to do no harm or non malfeasance, a gene therapy trial resulted in the death of a young Jesse Gelsinger, who was 18 at the time, and other young participants who developed cancer several years later (Lewis, 2021). The gene therapy trial involved using an engineered adenovirus as a vector to deliver a normal version of the ornithine transcarbamylase gene to his liver. Within days Jesse’s condition rapidly declined to organ failure and ultimately death (Lewis, 2021).
Since this tragic event, research for gene therapies for other conditions halted. The public and the scientific community was concerned about gene therapy’s negative effects because there were other failed cases that harmed the participants. Gene therapy research has made advancements and I believe that although it is a powerful tool to improve inherited disorders, there must be stronger regulation and screening to prevent adverse effects.
There is also the concept of justice when prescribing gene therapy. Some therapies in the market cost several hundreds of dollars over a few years of treatment while a single dose could be worth $2 million. Access to treatment is also scarce because some therapies can only be administered in certain medical centers. With the progress of gene therapy, there also needs to be a balance between its affordability and access to ordinary patients.
Cummings, J., Aisen, P., Apostolova, L. G., Atri, A., Salloway, S., & Weiner, M. (2021). Aducanumab: Appropriate Use Recommendations. The journal of prevention of Alzheimer's disease, 8(4), 398–410. https://doi.org/10.14283/jpad.2021.41
Haddad, H. W., Malone, G. W., Comardelle, N. J., Degueure, A. E., Poliwoda, S., Kaye, R. J., Murnane, K. S., Kaye, A. M., & Kaye, A. D. (2022). Aduhelm, a novel anti-amyloid monoclonal antibody, for the treatment of Alzheimer's Disease: A comprehensive review. Health psychology research, 10(3), 37023. https://doi.org/10.52965/001c.37023
Lewis, T. (2021, October 26). The quest to overcome gene therapy's failures. Nature News. Retrieved November 27, 2022, from https://www.nature.com/articles/d41586-021-02734-w
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