Evaluating an Investigational Treatment for ALS and Frontotemporal Dementia: Michael Panzara, MD, MPH


The potential of a new investigational treatment, WVE-004, was discussed by the chief medical officer of Wave Life Sciences, the company that developed the molecule for C9-ALS and C9-FTD.

“The study is designed to be adaptive and change depending on the data and, hopefully, accelerate as quickly as possible because these are 2 devastating diseases with a lot of impact on patients and their families.”

Dosing of WVE-004, an investigational treatment for C9orf72-associated amyotrophic lateral sclerosis (C9-ALS) and frontotemporal dementia (C9-FTD) recently began with the phase 1b/2a FOCUS-C9 trial (NCT04931862). The adaptive study will evaluate the safety and efficacy of the treatment, and investigators remain optimistic that WVE-004 may offer treatment routes for patients with ALS and FTD, both of which have limited options. Investigators will look to observe the impact of the intrathecal-administered oligonucleotide on toxic dipeptide repeat proteins, specifically polyGP.

In conversation with NeurologyLive, Michael Panzara, MD, MPH, chief medical officer, and head, therapeutics discovery and development, Wave Life Sciences, provided an overview of the trial design, which is unique due to its adaptive nature and basket-like design, as it will be studying both ALS and FTD. Dose escalation and frequency will be adjusted following the review of an independent safety monitoring board, and investigators are hoping to move forward similarly to how trials are advanced in the oncology space. Once treatments are seen to influence underlying pathology in oncology, they move forward at a quicker pace; the FOCUS-C9 study will be run in a similar manner, as Panzara stated, as both ALS and FTD can be just as devastating as cancer for patients and their families. 

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