The Washington Post featured a curious story last week about an experimental drug for amyotrophic lateral sclerosis (ALS), the progressive neurodegenerative disorder that is also known as Lou Gehrig’s disease.
I say “curious” because the story seemed oddly naïve (or inexplicably silent) about the financial motivations involved in drug development. Written by Pulitzer prize-winning health reporter Amy Ellis Nutt and coauthored by Brady Dennis, the story leads with the apparently promising impact seen in a tiny clinical trial.
Then in the fall, a small California biotech company named Genervon began extolling the benefits of GM604, its new ALS drug. In an early-stage trial with 12 patients, the results were “statistically significant,” “very robust” and “dramatic,” the company said in news releases.
Such enthusiastic pronouncements are unusual for such a small trial.
That last statement gave me pause.
Is it really “unusual” for drug companies to tout their early results as “statistically significant” and “very robust”?
In my experience, it’s all too common for companies to make inflated claims about early results from clinical studies. And that’s precisely why the FDA demands data from much larger studies before awarding marketing approval to new treatments.
The Post goes on to quote Winston Ko, one of the owners Genervon, denying any ulterior motive for seeking accelerated approval for BM604.
“The data is highly, highly robust,” Winston Ko said in an interview. “Why would I push it if I’m not confident that our innovative, novel drug discovery is not effective?”
Perhaps the Post thought the answer to that question was too obvious to merit a direct answer, but I thought the story could have done more to examine Mr. Ko’s claims to motivational purity.
As the co-owner of Genervon, Mr. Ko stands to reap financial rewards from GM604’s approval. But the story gives no estimate of what the drug might cost or the revenue that it might generate for the company (surely tens if not hundreds of millions). It provides no estimate of what the company might save by skipping a large-scale clinical efficacy trial (besides noting that such a trial would be “costly”).
These are important questions to ask of someone who warns that current ALS patients “will all die” if they don’t they don’t obtain immediate access to his company’s drug.
To its credit, the story does feature plenty of skepticism about the quality of evidence on offer from Genervon. It quotes ALS researcher Steve Perrin as to the utter deficiency of the 12-person study being touted by the company.
“The bottom line with the Genervon drug is there is absolutely no data,” he said. “There is no mathematical way or statistical way that they could measure a drug effect.”
Even one of the lead researchers on the study is brought forward to cast doubt on the company’s claims.
“When [Genervon] started putting out things [about significant results], we said, That’s not true,’?” Mitsumoto said, referring to himself and the other principal investigator, Merit Cudkowicz, director of Massachusetts General Hospital’s ALS clinic. “I think a large study is needed, and they [the Kos] don’t understand.”
But the story’s failure to explore the business angle and the financial incentives at play is a significant one. In the Post’s telling, the issue here is primarily one of safety and lack of data vs. patient choice and the right to access.
But’s it’s also a story about money. And there’s lots of it to be made by companies preying on the false hopes of those who are suffering. The Post could have been clearer about that.
Social Media reaction:
This story was the subject of a spirited discussion by health reporters over on Twitter. John Carroll, editor of FierceBiotech, appeared to be the first to call attention to the story. He described the story as “ridiculous” and said it was “riddled with absurd observations.”
— John Carroll (@JohnCFierce) April 4, 2015
Michael Hiltzik, the LA Times’s Pulitzer prize-winning columnist, responded that the Post offered a decisive “dismantling” of Genervon claims. He said that the company’s view was cited merely so that the story could “knock it down.”
.@JohnCFierce Absurd how? I read piece as dismantling Genervon’s claims pretty decisively
— Michael Hiltzik (@hiltzikm) April 4, 2015
Meanwhile, Lisa Jarvis, a reporter with Chemical & Engineering News, said the story’s main problem was a failure to frame the issue appropriately.
— Lisa Jarvis (@lisamjarvis) April 4, 2015
In my view, all three of these reactions are essentially legitimate responses to the story. But my take is closest to Jarvis’s. Despite having some “absurd observations,” the story does go to great pains to “knock down” Genervon’s claims about GM604. And yet that effort is ultimately dissatisfying because the thesis of the story isn’t clear.
Despite having the right instincts, the story never questions the financial motivations behind Genervon’s push for marketing approval. Exploring the money angle would laid bare the fact that this is “bad science preying on desperate patients.”
Follow us on Twitter: