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Money angle is missing from Post’s coverage of a new ALS drug

The following blog post is written by Kevin Lomangino, managing editor of HealthNewsReview.org. He tweets as @Klomangino

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.”

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.”

 

Meanwhile, Lisa Jarvis, a reporter with Chemical & Engineering News, said the story’s main problem was a failure to frame the issue appropriately.

 

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.”

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Comments (22)

Please note, comments are no longer published through this website. All previously made comments are still archived and available for viewing through select posts.

Steven Walker

April 6, 2015 at 2:10 pm

It is no surprise, nor is it morally wrong, for there to be a profit motive behind the efforts of drug companies to develop new medicines for horrible diseases like ALS. Implying that just because a for-profit company (which is where virtually all of our medicines come from) wishes to cover its costs of development and manufacturing, plus make an eventual profit, makes the position of a company like Genervon regarding approval of its investigational drug a predator is just ridiculous. The discussion about whether data from an early clinical trial is sufficient to support a conditional approval (which is what Genervon is seeking from FDA), which comes with an enforceable commitment on the part of the company to continue testing of the drug in clinical trials to confirm safety and efficacy), is also an extremely important part of this story. Finally, this is about a weighing of the potential for benefit versus the potential for risk. ALS patients see this drug as their only option that might work in the time they have left, and that also might become available to them in time. The greatest risk they face by far is the risk posed by their disease. They want their chance to find out if it will work, and they have made it clear they are willing to participate in the collection of data regarding how well the drug works, or doesn’t work, as part of their getting it. The FDA’s Accelerated Approval pathway was created for situations like this, and has been used many times for other diseases. Why not for ALS? Do you also question the motives of, for example, a company that makes passenger airplanes because they have to make enough money to cover development costs and manufacturing, and to design the next generation of aircraft, a profit? There is much to debate about how much information we need before a new medicine for a disease like ALS gets first approval, but assigning malicious intent because the profit motive causes investors and companies to take the risk to develop new ones, isn’t one of them. There are two sides to the timing of an approval decision, and the one that rarely gets any coverage is the human cost of delaying a drug that works for a disease like ALS for years. If three or five years from now, the people opposing approval of GM604 finally agree, based on bigger trials, that the drug is safe and effective, what would you write about the ALS patients who want it now and never got their chance? I suggest you contact the thousands of Persons with ALS (PALS) and Caregivers with ALS (CALS) who are pressing for this drug to be approved now, and learn for yourself why they want it approved. It is a very important part of the story, and I strongly suspect, would change your view of this situation.

    Kevin Lomangino

    April 6, 2015 at 3:08 pm

    Steven,

    Thanks for taking the time to comment.

    I never suggested or implied that it was morally wrong for there to be a profit motive behind the efforts of drug companies to develop new medicines.

    I did suggest that the profit motive creates a conflict of interest. And I do think it was The Washington Post’s duty to call attention to that conflict in their reporting.

    This site is about journalism and the way media messages affect health care. We think people are better informed and make better decisions when news stories carefully vet story sources and alert readers to potential conflicts.

    The Post didn’t do a great job of that in this story, which is why I thought it was worth writing about.

    Best regards,

    Kevin Lomangino
    Managing Editor

Robert Cornish

April 6, 2015 at 3:22 pm

My reading of the accelerated approval process is that it allows approval for drugs that have shown a verifiable impact on a surrogate marker that is likely to correlate to a clinical outcome. The sponsor is obliged to continue study to verify the correlation between the surrogate and a meaningful clinical outcome. And if I read this situation correctly, the company has not shown very much of anything, based on the Post article and on the company’s press releases.

The earlier comment seems to say that companies should be able to sell their drugs without having to show any real or meaningful evidence that it does anything. Desire or need ought to be enough evidence that the drug needs to be approved.

Patients without effective therapies might feel it’s their only hope. But is that a false hope? America can’t afford to pay for untested drugs, to see if they work? If the drug does work, the company stands to make a justifiable fortune. It doesn’t deserve to make one on a drug that might not have any positive effect.

I wonder why more of the patients are not in a clinical trial to find out of the drug works. If there are thousands of patients waiting for this therapy, how come the company is saying it deserves approval on the basis of studying only 12. And of the 12, did all the patients get the drug? Did all of them respond?

I fall into the Jarvis camp too. This isn’t sounding like the company has done what it needs to do to show their drug works, or to what degree. And I’d sure hate to see this process become the pathway for drug approval in the United States.

    Jim Schmitt

    April 6, 2015 at 4:55 pm

    All ALS sufferers want is hope.

    Right now, there is none. There is no current treatment available to save them from this horrible death.

    My wife is 18 months into her Bulbar ALS diagnosis and she is rapidly deteriorating. She can no longer eat or speak. She eats through a feeding tube. It’s difficult for her to hold her head erect, and the muscles in her arms and legs are weakening to where she can’t lift anything weighing more than a pound or two, and she wears a brace on her left left leg. She now sleeps apart from her husband in a room set up with with a hospital bed and other myriad medical devices because she can’t easily navigate the stairs to our second floor bedroom. She has to use a device to suck saliva and flem from her throat, and she wears a device to help her breath while she sleeps and has had an experimental diaphragm pacemaker-type device installed to see if it can jolt her diaphragm into helping her breath.

    For her, it’s only going to get worse to where she’ll be mentally alert in a useless body that will entomb her during her final days.

    She’s very mentally sharp . . . and she wants to live. All she has left, aside from the constant care and love from her family, is hope. Period! And you pompously want to take that away from her because you suspect Genervon wants to make a profit from their treatment. You say Genervon stands to make tens if not hundreds of millions by offering this treatment. Really! How many millions will Genervon make if their treatment proves to be a dud? How much would the public trust Genervon with treatment they’re working on for other diseases? Your article, and others like it, will only serve to reinforce the FDA’s decision to not approve Compassionate Care usage of this treatment, if that’s the position it ultimately takes.

    My wife Kathy has said she would rather take an experimental treatment for ALS and die from lack of results in the name of science than simply sit around waiting to die a horrible death. I presume many of the 30,000 residents of the United States who have been diagnosed with ALS would agree. But most of all they want hope.

    When my wife was first diagnosed, we looked all over the world for a legitimate treatment, particularly a stem cell treatment. All we found was fraud by subhumans who want to get rich exploiting people like my wife who want to live. If Genervon wanted to get rich off a treatment for ALS that gives false hope, all they’d have to do is open a clinic in Mexico just a few miles south of their Pasadena headquarters. Trust me, ALS patients from all over the world, including my wife, would flock to them and pay on obscene amount of money for Genervon’s treatment.

    My wife is under the care of one of the nation’s foremost ALS medical complexes in the country and her doctor’s have no objection to her taking the treatment. Genervon is a company that has a good reputation in the medical research industry. .

    She has applied for many trials but none accept patients with the Bulber form of the disease. Furthermore, even if the researchers decide to eventually include Bulber ALS sufferers, she’s rapidly approaching the two year cut-off these trials adhere to. So what’s left for her? A rapid path to a horrible death.

    We’re all hoping the FDA will give Genevon approval to administer its treatment to ALS sufferers who volunteer, “volunteer” being the operative word. All they want is hope!

      Teresa OLeary

      April 7, 2015 at 7:07 am

      thank you Jim for pointing out the obvious. I often wonder if the same ” critics” would be writing these kind of articles if one of the big pharmaceutical companies had developed gm604? Highly doubt it.

Mike C

April 6, 2015 at 5:34 pm

As a person with ALS, I would like to note that we are not talking about high cholesterol here, but a disease that is 100% fatal, wholly untreatable and as cruel as they come. We have had 20 years of failed Phase 3 trials with all manner of risky surgery or noxious substances, to such an extent that the longest survival is observed in placebo groups of clinical trials. Given GM604’s demonstrated safety and statistically significant, if highly preliminary, effectiveness on biomarkers and clinical results, what is the rationale for withholding it, exactly? AZT (as 3TC) and Bevacizumab got Accelerated Approval in the past. This is the system working as intended. On what basis deny this to ALS patients? What value there is there in hammering in the blindingly obvious observation that Genervon is a for-profit company? One thing I guarantee is that had Lisa Jarvis had ALS, she would have been voting for GM604 Accelerated Approval Process with all the limbs she still could control. A slender straw, you say? We, the patients, know that! Nobody is advocating that AAP should be “the pathway for drug approval”.

    Kevin Lomangino

    April 6, 2015 at 7:38 pm

    Thanks to Mike C and Jim Schmitt for the comments. But I’d remind everyone that the purpose of our site is to review journalism — for all audiences. Your perspective as engaged, informed advocates is very different from that of the average news consumer reading the Post story. It may be blindingly obvious to you that Genervon officials are conflicted in their push for accelerated approval, and you’re obviously ok with that, but the typical news consumer needs and deserves to have those relationships and biases clarified. We welcome additional comments on the quality of the Post story or journalism-related issues. But this is not a forum for advocacy, definitive discussions of science, or deep discussion of the FDA approval process.

Jennifer Dean

April 6, 2015 at 11:48 pm

Like Jim Schmitt’s wife, I have bulbar ALS. In your article you talk of false hope. At the moment ALS sufferers have no hope, no effective treatment. We are offered assisted suicide as a way out of this nightmare. There is a glimmer of hope with this drug. It may not work but we should be given the opportunity to try. Ebola patients are given experimental drugs to help save their lives. I would be willing to be a guinea pig. If I die from taking this drug then so be it – I’m going to die anyway and I may have helped science in the process.

Tom Sharp

April 7, 2015 at 12:04 am

Your journalistic integrity is flawed. The WaPost article was merely to publicize that information showed hope to ALS patients. If you believe that only naive children read the Post then you could claim that the Post failed to note Genervon’s “conflict” as you see it. This “conflict” would certainly be mitigated if the drug wasn’t effective and Genervon would have blown 20 years of research good will. Did you research the findings yourself, or simply take other’s words for it? That’s what I thought.

    Kevin Lomangino

    April 7, 2015 at 5:47 am

    Tom,

    Thanks for the comment. I disagree that the WaPo piece was merely to publicize information that showed hope to ALS patients. Health journalism as we see it is supposed to inform and engage on a number of levels, not merely publicize. Please read our new story review criteria — two of which involve costs and discussion of conflicts of interest. And I would be happy to look at a peer reviewed study on the Genervon research, as would many other people. My understanding is that such research has not been published, obliging us to rely on Genervon press releases. From a journalist’s perspective, that is not an ideal situation.

Bill Ziese

April 7, 2015 at 10:52 am

I had symptoms in 2007 and was diagnosed with ALS in 2008. I take over 80 supplements a day, some multiple times. I have retarded the progression and have some minor improvements. The problem with drugs is they have side effects and every patient will react differently. I believe there are many types of ALS and a “cure” is in the distant future, if at all. Maybe prevention is the answer. I refuse any clinical trials because: 1. I would have to stop taking all my supplements; 2. There is a 50/50 chance I would get a placebo and 3. Even if a drug shows promise, it would be years before FDA approval and production. ALS patients simply don’t have that time.

Dante

April 7, 2015 at 11:09 am

I wonder how much $ plays into FDA’s decision?

The Big Chill: How Big Money Is Buying Off Criticism of Big Money

It’s bad enough big money is buying off politicians. It’s also buying off nonprofits that used to be sources of investigation, information, and social change, from criticizing big money.

Other sources of funding are drying up. Research grants are waning. Funds for social services of churches and community groups are growing scarce. Legislatures are cutting back university funding. Appropriations for public television, the arts, museums, and libraries are being slashed.

So what are non-profits to do?

“There’s really no choice,” a university dean told me. “We’ve got to go where the money is.”

http://robertreich.org/post/115695610915

If the author of this article had ALS or maybe one of his children, would he want access to it? Yes or no? Simple question.

    Kevin Lomangino

    April 8, 2015 at 11:37 am

    Dante,

    What I would choose to do is nobody’s business but mine. We don’t give advice. We don’t try to influence others with our own choices. And one individual’s choice is not a sound basis for a national health policy.

    Thanks for your comment.

      Dante

      April 9, 2015 at 10:25 am

      Kevin,
      You brought up the money angle regarding Genervon. You polluted the argument to distract from need to get help for people that have no options. National health policy is based on money but the guys collecting are setting policy. Your answer to my question tells me your like everyone else in this country
      …SELF ABSORBED!

      Study Warns of Diet Supplement Dangers Kept Quiet by F.D.A.

      Daniel Fabricant, who ran the agency’s division of dietary supplement programs from 2011 to 2014, had been a senior executive at that trade group, the Natural Products Association, which has spent millions of dollars lobbying to block new laws that would hold supplement makers to stricter standards. He left the F.D.A. last year and returned to the association as its chief executive. His current replacement at the F.D.A.’s supplement division also comes from the trade group.

      “To have former officials in the supplement industry become the chief regulators of that industry at the F.D.A. is like the fox guarding the hen house,” said Michael F. Jacobson, the executive director of the Center for Science in the Public Interest, a consumer advocacy group.

      Dr. Fabricant disagreed, saying in an interview that having industry officials run the F.D.A.’s supplement division was “an incredible benefit to the agency.” He said that during his tenure, the division worked to get unsafe products off the market and issued a mandatory recall of a supplement involved in a deadly multistate outbreak of liver disease. “I did more there in three and a half years than was done in the 16 years prior,” he said.

      The controversy comes at a time when the supplement industry is under increased scrutiny. Last week, 14 state attorneys general, led by Eric T. Schneiderman of New York, called on Congress to provide the F.D.A. with more power to regulate supplements. Mr. Schneiderman’s office in February accused four major retailers of selling contaminated herbal supplements, and one of the companies, GNC, has agreed to extensive new testing and quality control procedures for its store-brand herbal products.

      http://well.blogs.nytimes.com/2015/04/07/study-warns-of-diet-supplement-dangers-kept-quiet-by-f-d-a/

      somethings get pass, others can’t get pass go..

      money talks, cures balk?

      how many billions at stake for status quo drugs and research industry?

      Kevin Lomangino

      April 9, 2015 at 12:41 pm

      Dante,

      There are, of course, many different ways that financial considerations might impact the development and approval of new drugs. I wrote about one of the conflicts that I thought was most directly relevant in this case, and which I thought The Post could have covered more thoroughly.

Amy Ellis Nutt

April 7, 2015 at 1:13 pm

Kevin,
First of all, please note the correct spelling of my last name. Your criticism that we failed to cover the financial angle of Genervon’s interest in GM604 is well taken, the problem is, Genervon is a private company and the Kos refused to answer all questions about who manufactures the drug for them, how much it costs, how long it takes to manufacture, how much money they’ve invested in the clinical trial and whether or not they have been soliciting investors or would like to sell the company to a larger biotech. Hence, we not only couldn’t report on what the Kos stand to gain financially, except in the most general terms, we couldn’t speculate on their motivations, except for what they told us. That wasn’t our place as reporters.

    Kevin Lomangino

    April 7, 2015 at 3:14 pm

    Amy,

    I am thrilled that you took the time to comment, and I apologize for the misspelling of your name (the error has been corrected).

    Your comment about the lack of availability of cost and financial data is one we often hear from journalists whose stories are flagged in our reviews (some 70% of the ~2000 stories we’ve reviewed are judged “not satisfactory” by our reviewers on that criterion).

    I would offer a few of the suggestions that we’ve made to others in that regard:

    – Could you have cited the costs of existing alternatives — e.g. riluzole — to give readers some sense of the numbers we’re talking about?
    – Could you have tapped a pharma analyst to provide a ballpark projection?

    Failing either of those, I for one would have been satisfied with an acknowledgment that you had raised these issues with the Kos and that they would not respond. That at least would have gotten the point across that there are business as well as health issues at stake here.

    Again, thanks very much for engaging us. This is meant to be a dialogue, not a lecture — and I appreciate you providing some insight into your thinking.

    Kevin

Anonymous

April 7, 2015 at 1:36 pm

I posted this as a comment on the Washington Post websites.

I work in pharmaceutical regulatory affairs and see things in here that don’t make sense. Patients do not need accelerated approval to get access.

First off, there is Expanded Access and Treatment INDs and compassionate use, where the doctor petitions the FDA for use in that patient, or a group of patients, and as long as the drug company agrees, then FDA can grant access to the drug. Who pays for it, is another story. Here’s a link to the FDA guidance on it: http://www.fda.gov/downloads/drugs/guidancecomplia

Regarding this accelerated approval, this is NOT new, and this is not the first drug. Accelerated approval just means approval is based on a surrogate endpoint, instead of a real clinical effect. It may not mean any faster. Rather, Priority Approval is where the FDA is asked to approve something faster, yet that isn’t mentioned anywhere in this article (see this FDA guidance: http://www.fda.gov/downloads/drugs/guidancecomplia… If the drug is approved, by either of these means, then insurance companies would be hard pressed to refuse it. And THAT may be why the company is pushing for it.

So for those who want this drug, ask the company to give access via Expanded Access / Compassionate Use / Treatment IND. They should be able to do it, but who pays for it is another story. The fact this is not mentioned anywhere in this article, nor Priority Approval, makes me very suspicious of what the company is passing around and/or their knowledge of regulatory affairs.

Nick Grillo

April 7, 2015 at 2:12 pm

Kevin Lomangino: First of all, no one is praying on “false hopes” of those with ALS. The movement urging AAP for GM604 is a grassroots effort and none of us have any connection to Genervon. Second, the conflict of interest in getting a viable treatment to market is simply innate. It doesn’t matter if it is big pharma or a small biotech, someone will eventually be making money You do your readers a disservice by not disclosing that in your article.Is that perhaps because then the subject becomes irrelevant? It appears you are making a story where none exists.

Anonymous

April 7, 2015 at 9:10 pm

The company has put out a press release saying that they won’t provide expanded access because it’s too expensive. There are cost recovery rules under FDA’s regulations. The company can reclaim the cost of providing the drug, but can’t make a profit on the deal http://www.fda.gov/OHRMS/DOCKETS/98fr/06-9685.htm.

But the company would be in a precarious position if they made their product available through expanded access, and patients found that it was not effective. Where would they company be then? This way, they can reap the profits of drug sales, and if the drug proved it would be great. If it proved ineffectual, even if patients stopped using it, they’d be on their way to the bank with their profits, never having had to really prove anything at all. And in the process, they would have compromised the drug development process, and drug approval in the U.S.

Patients do not have time to wait if there is an effective drug. However, if the drug is ineffective – and there is pretty shaky data at this point – regardless of the claims of the company owners, then the patients are doing a great disservice to future patients who will coming behind them.

Mike C

April 11, 2015 at 6:15 pm

The Anonymous: So *if* GM604 is ineffective, P4 study unearths that in fairly short order, and GM604 is withdrawn. There is some cost to insurance companies — not a huge cost compared to $30-100M of a Phase 3 trial. But if GM604 should prove effective, you have just killed about 10,000-30,000 people. Your blithe exhortation for social responsibility at the cost of our lives is not only in poor taste, but is just wrong. PALS will benefit from broader access, which would permit data mining to determine responder sub-types of ALS, which is quite likely not one disease but many. It will permit exploring effects of cocktail approaches to treatment. It will answer questions like: does riluzole actually negate the effect of therapies?