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I worked on and around aducanumab, an experimental treatment for Alzheimer’s, for years during my time at Biogen. I thought it was going to work. I wanted it to work — my father had died of Alzheimer’s disease. It was awful watching his mind disintegrate over the course of a decade, which made me even more hopeful for a positive outcome for aducanumab.

I had also worked on dexpramipexole, a molecule that Biogen and Knopp Biosciences were testing as a treatment for amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease. I wanted it to work, too, since one of my close friends had ALS. He was taking part in the trial for the drug, which made me even more hopeful for a positive outcome.


But dexpramipexole didn’t work any better than the placebo it was being tested against. I cried the day I found out. I remember sitting in a bathroom stall in Biogen’s building in Weston, Mass., with tears dropping onto my gray business casual trousers, crushed by the knowledge that my friend wouldn’t be helped, nor would countless others.

That day, George Scangos, who was the CEO of Biogen at the time, called an all-company meeting just after lunch. He asked that everyone stop what they were doing and come to the auditorium to talk about dexpramipexole.

George wasn’t a natural public speaker, and at times it showed. On that day, though, he walked to the podium without a discernible shred of discomfort. He stood calmly in front of the company, investors, and the media and, with a presence I had never before seen, told the crowd in no uncertain terms that dexpramipexole did not work and would not be a treatment for ALS.


He said the results made him profoundly sad, but made it clear that Biogen would do no fishing for false signals in subtypes or subgroups that, in the picked-over carcass of the dataset, may have appeared to have responded to the drug.

He said it was important to have the integrity to admit failure when failure was clear and how it was our responsibility to respect patients and their families by not giving them false hope. He then shared his resolution that Biogen would not stop fighting ALS and the company would focus its capital, time, and effort on the next generation of therapies.

Biogen’s stock got hammered, dropping more than 8% that day. Yet I’d never been more proud than I was that day to work for George and for Biogen, having seen for years companies hem and haw at negative results and claim that if you squint and look sideways that the molecule really did work, hoping that investors would have mercy on the stock price — and in the process ignoring the responsibility to be honest and forthcoming to patients and their families.

When I learned last week that Biogen had completed a retrospective analysis of the aducanumab data — the same data that seven months earlier the company had said justified stopping work on the drug — I thought of my father, my friend, George Scangos, and people around the globe with Alzheimer’s, ALS, and other diseases for which there are no cures.

I hope that Biogen made a mistake in its futility analysis back in March. But if it didn’t, and this is another example of the American public watching big pharma trying to spin bad data, then I can’t help but wonder if Biogen, and perhaps the industry, has lost its way.

Ted Whitford is a veteran of the biotech industry who now privately invests in the health care market.

  • Ted I really appreciate this article. I know it has generated a lot of backlash, but people on industry know that there’s reasons why protocols are written with endpoints in mind before trials begin.
    We need to get better at subtyping diseases and personalizing medicine, but realistically acknowledge when the group will be underpowered and has no counterfactual controls. Thank you for your piece, it’s a lovely succinct message.

  • This is a follow-up to my earlier comment.
    1. What was the age range and mean average of the patients in each trial, along with the same for the high dose and futility analysis participants?
    2. What was the educational level and diversity mix of the participants in each of the above groups?
    3. Will the FDA need more data to confirm “meaningful cognitive benefits” for Alzheimer’s patients?
    4. Will Biogen request approval of Aducanumab to treat Amyloidosis for Asymptomatic candidates without cognitive symptoms?

    Author Bruce Bauer’s web site:

  • No one “wins” when a drug doesn’t appear to work. We can only hope that there may be a subset of patients that perhaps derive benefit. Carefully looking at all of the data is the responsibility of researchers. Some drugs were initially rejected due to the futility of trials but had benefits that were not looked at in the trials (verapamil failed for the treatment of angina because the dose chosen in the trials was wrong, but it came back for high blood pressure and arrhythmias at the right dose). Having industry support further looks at why a trial failed may lead to additional knowledge and research. The many patients, nurses, researchers and physicians that spent time, donated their blood, went through additional testing, and had faith in an ethical pharmaceutical company hope that all of the information from the trial is looked at as carefully as is possible, with no attempt to “spin” the results for profit or fame. For those desiring a “right to try”, there must be an understanding that this desperation often is a right to experience side effects and exposure to failure without realistic possibility of benefit.

  • Thank you for your insights. It is well past time to declare the amyloid hypothesis to be misdirected. As Dr. Herbert B. Allen has stated “ignoring the likely microbial pathogenesis in AD could possibly become one of history’s greatest ethical calamities.” See, “Bioethical Challenges Arising from the Microbiology and Pathology of Alzheimer’s Disease,” Curr Neurobiol 2018; 9(1): 26-28 ISSN 0975-9042. Other possible treatments for AD should be more avidly investigated, such as described in US Pat. No. 10,010,568 entitled “A Method and System for Reducing the Likelihood of a Spirochetes Infection in a Human Being.” See also, US Pat. Publication No. 20180303658 – Method and System for Reducing the Likelihood of a Porphyromonas Gingivalis Infection in a Human Being (allowed)

  • The fact is that there isn’t a lot of good news in this arena and the information in Biogen’s press release indicates that 15-20% of the study patients benefited from the drug without significant side effects. It isn’t unusual for less than 100% of study patients to respond to any drug, so I surmise that denying access to 15-20% of Alzheimer’s patients was a greater evil than approving the drug in the FDA’s risk/benefit analysis.

  • I am so disappointed about the drug for Dimentia, my husband did the trial. I can’t believe you can’t find a cure for this debilitating disease. You watch them disappear right before your eyes.

  • I am the primary caregiver of my 67 year old fiancé and he has been battling Alzheimer’s for 9 yrs. He was in a trial study and received the placebo for weeks, then he was 64 and still golfing and enjoying life. When the drug went into phase 2 and he could not do a cognitive test, he was denied access to the phase 2 IV infusion. I tried to tell his Doctor about the right to try law, but he said no. My main problem is these drug companies only want stats that pay. Why not give anyone with Alzheimer’s or ALS any new drug out there ? They have nothing to lose but I think the drug co do.

  • Reading the comments here makes me realize how hard it is to do science when the emotions of people’s hopes for their loved ones are on the line. This is clearly and utterly an attempt at biogen to sugar coat a failed study. Looking at the data, one study’s results are horrid where the other one is promising. Now, why did this happen? Because the chaotic nature of the clinical endpoints chosen require an unbelievably large number of people to show that there is indeed, no effect. The FDA has rightly required two identical successful phase 3 studies and Biogen had delivered on this as mentioned in their earnings call. Yes, there was that protocol amendment difference but that could not have caused a successful trial and a failed trial. The time difference was just too low. To add insult to injury, the more the patients were on the trial, the less of an effect there was. (ITT vs OTC).

    Thank you for writing this. We need capital and resources to go to drugs that actually work.

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