Talking to me with the assistance of a speaking valve, Chris Simon made it clear what he thought of Radicava, a newly approved drug for patients who have amyotrophic lateral sclerosis (ALS).
“It’s more fool’s gold than it is gold,” said Simon, who has ALS and lives in California. He doesn’t plan to take the drug, especially since he has a friend with ALS who wasn’t helped by it–even though his friend flew to Japan to get the treatment before it was available stateside.
ALS patient Chris Simon
“They’re selling hope,” Simon said of marketing efforts around this drug. These include the company’s website for the drug and public relations efforts, such as this news release that we reviewed that describes Radicava as an “uplifting milestone” offering “new hope,” signaling a “new era” of treatment.
This hopeful framing also has found its way into talking points from ALS patient groups and recent news stories on Radicava–such as this one from Richmond Times-Dispatch and this report from WFMZ-TV in Allentown, Penn. These stories sometimes discuss the cost (~$150,000/year) and the restrictions around prescribing the drug to patients–often casting insurers in a negative light–but stop short of explaining why these restrictions exist.
What you are unlikely to find in most of these stories are viewpoints like Simon’s, yet he is onto something: While Radicava was FDA-approved in May for all ALS patients to help slow disease progression, it will probably only help a small percentage of them–roughly 7% of patients–who fall into a specific set of symptom characteristics.
And of those who do stand to benefit, the improvements are likely to be so modest they’re outweighed by the rigor of receiving infusion therapy–which isn’t easy for anyone, much less for patients who are often immobile and may be on ventilation.
In other words, for most ALS patients, it’s likely to be a mediocre and costly treatment. 
That’s not a headline we want to show ALS patients, but I’d argue that above all else, ALS patients deserve the truth. When measuring their life in months, they deserve a fully informed presentation of the benefits and the drawbacks before starting any treatment, especially one like Radicava. (It will also go long way helping them–and all of us–understand why insurance companies are reluctant to pay for the drug.)
Findings apply to about 7% of patients
To its credit, ABC News came close to cracking open this part of the story. It was the only U.S.-based story I read that discusses how pessimistic some doctors are about the drug. 
Underpinning the FDA’s approval is just one small phase 3 study published in May in Lancet Neurology. In the study of 137 patients, the researchers determined that the drug led to a 33 percent reduction in the rate of decline in physical function among ALS patients.
Yet, this benefit wasn’t found in a general ALS patient population. It was found in a group of Japanese ALS patients who had been diagnosed less than two years and had rapid progression of symptoms.
A previous phase 3 study by the drug maker, Mitsubishi Tanabe Pharma, found no statistically significant benefits for general ALS patients. However, a later analysis of that same data hinted that perhaps the newly diagnosed/rapid onset subset of patients would benefit–and that’s how the Lancet study came about.
As we explain in our review of the ABC News story, it “should have made it very clear that this new study was limited to patients who had a specific type of ALS disease process. In a commentary that accompanied the study, we learn that “less than 7% of patients with ALS would be eligible for enrollment.”
And about the 33 percent reduction in symptoms (among this small subgroup of patients): This was measured using the ALS Functional Rating Scale, with 48 being the best score a patient can get. The group receiving Radicava progressed less slowly on this scale–losing about 5 points–whereas the placebo group lost 7.5 points.
It’s unclear if this difference — 2.5 points on a 48-point scale — actually translates into meaningful improvements that patients themselves will notice, noted Dr. Orla Hardiman, who authored the commentary mentioned above. Hardiman is a clinical professor of neurology at the Trinity College Institute of Neuroscience in Ireland, where she is also the director of the National ALS Clinic and Irish ALS Research Group.
Since the FDA approved it for all ALS patients, and not the subgroup of patients most likely to benefit, she said it’s probable that Radicava “will be prescribed by physicians–non ALS experts–who will prescribe it for everybody, and the vast majority of people won’t respond.”
Was the drug ready for approval?
But because there are so few drugs for ALS, some in the field argue that experimental drugs such as this should be made widely available, regardless of the lackluster evidence.
While this may please advocates, this comes at a cost, of course. Radicava is very expensive, requires frequent and intensive IV-infusion therapy, and has known risks like respiratory failure. 
It also could be a setback to ALS research, Hardiman said. Radivaca has potential and research should continue, she said, especially to study potential biomarkers, genomic signatures and other factors that better pinpoint who will benefit most. 
Yet, already she has been turned down for grant funding involving Radicava, because it’s already approved and being used in the U.S. And since the drug is being used too broadly, it may end up looking ineffective, which could further impact future research. 
“I think it’s a pity that it might prevent investment in studies that allow us to find out who really benefits and what the biomarkers are.”

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