The ALS Association has been working with FDA to seek the meeting, Director of Public Policy Pat Wildman said in an interview. The organization wants FDA to hear about ALS, a rapidly progressing and fatal neurodegenerative disease also known as “Lou Gehrig’s Disease,” from the patient’s perspective, he said, as well as that of clinicians and researchers. The only drug currently approved for ALS, Sanofi’s Rilutek (riluzole), only delays disease progression in some by patients by roughly three months; there is no cure and patients typically live for three to five years following diagnosis.
“People with ALS have a different perspective about drug development than the average person, whether it’s a person with a different condition or a healthy person,” Wildman said. “They have a much different view of benefit/risk … [and are] much more willing to take risk, whether in a trial or taking the drug itself.”
“People with the disease want to make it clear to FDA what this disease truly is,” he added, “because it’s one thing to read about ALS and understand it from a clinical standpoint, but it’s another to actually see it and understand how it impacts someone’s life and destroys the person’s ability to do things that almost everyone else takes for granted.”
The association plans to call for expanded access and accelerated development and approval of ALS therapies at the meeting, Wildman said. It also will advocate for the use of biomarkers and surrogate endpoints “to speed development and let us know sooner whether something is working or not working,” he explained.
Lucie Bruijn, the association’s chief scientist, said she hopes the discussion will focus on whether current requirements in the clinical setting make trials take longer than they need to. “This is going to be a back-and-forth discussion with FDA and the clinicians but there’s clearly a need to consider different outcome measures that potentially might be effective, and discuss how we interpret clinical trials [and] whether multiple trials are needed,” she said.
While some of the current investigational therapies for ALS are stem cell approaches, Bruijn said the meeting is not necessarily about addressing hesitation toward testing such products in humans. Candidates of various types, including small molecules and antisense gene therapy, offer promise for the treatment of ALS, she noted. But Biogen’s decision to end development of pramipexole necessitated new discussion on clinical methodology.