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

April 6, 2015 - als

The following blog post is combined by Kevin Lomangino, handling editor of He tweets as @Klomangino

The Washington Post featured a extraordinary story final week about an initial drug for amyotrophic parallel sclerosis (ALS), a on-going neurodegenerative commotion that is also famous as Lou Gehrig’s disease.

I contend “curious” since a story seemed infrequently naïve (or inexplicably silent) about a financial motivations concerned in drug development. Written by Pulitzer prize-winning health contributor Amy Ellis Nut and coauthored by Brady Dennis, a story leads with a apparently earnest impact seen in a little clinical trial.

Then in a fall, a tiny California biotech association named Genervon began extolling a advantages of GM604, a new ALS drug. In an early-stage hearing with 12 patients, a formula were “statistically significant,” “very robust” and “dramatic,” a association pronounced in news releases.

Such eager pronouncements are surprising for such a tiny trial.

That final matter gave me pause.

Is it unequivocally “unusual” for drug companies to surveillance their early formula as “statistically significant” and “very robust”?

In my experience, it’s all too common for companies to make arrogant claims about early formula from clinical studies. And that’s precisely since a FDA final information from many incomparable studies before awarding selling capitulation to new treatments.

The Post goes on to quote Winston Ko, one of a owners Genervon, denying any distant ground for seeking accelerated capitulation for BM604.

“The information is highly, frequency robust,” Winston Ko pronounced in an interview. “Why would we pull it if I’m not assured that a innovative, novel drug find is not effective?”

Perhaps a Post suspicion a answer to that doubt was too apparent to consequence a approach answer, though we suspicion a story could have finished some-more to inspect Mr. Ko’s claims to motivational purity.

As a co-owner of Genervon, Mr. Ko stands to reap financial rewards from GM604’s approval. But a story gives no guess of what a drug competence cost or a income that it competence beget for a association (surely tens if not hundreds of millions). It provides no guess of what a association competence save by skipping a large-scale clinical efficiency hearing (besides observant that such a hearing would be “costly”).

These are critical questions to ask of someone who warns that stream ALS patients “will all die” if they don’t they don’t obtain evident entrance to his company’s drug.

To a credit, a story does underline copiousness of doubt about a peculiarity of justification on offer from Genervon. It quotes ALS researcher Steve Perrin as to a complete scarcity of a 12-person investigate being touted by a company.

“The bottom line with a Genervon drug is there is positively no data,” he said. “There is no mathematical approach or statistical approach that they could magnitude a drug effect.”

Even one of a lead researchers on a investigate is brought brazen to expel doubt on a company’s claims.

“When [Genervon] started putting out things [about poignant results], we said, ‘That’s not true,’ ” Mitsumoto said, referring to himself and a other principal investigator, Merit Cudkowicz, executive of Massachusetts General Hospital’s ALS clinic. “I consider a vast investigate is needed, and they [the Kos] don’t understand.”

But a story’s disaster to try a business angle and a financial incentives during play is a poignant one. In a Post’s telling, a emanate here is radically one of reserve and miss of information vs. studious choice and a right to access.

But’s it’s also a story about money. And there’s lots of it to be finished by companies preying on a fake hopes of those who are suffering. The Post could have been clearer about that.

Social Media reaction:

This story was a theme of a energetic contention by health reporters over on Twitter. John Carroll, editor of FierceBiotech, seemed to be a initial to call courtesy to a story. He described a story as “ridiculous” and pronounced it was “riddled with absurd observations.”

Michael Hiltzik, a LA Times’s Pulitzer prize-winning columnist, responded that a Post charity a wilful “dismantling” of Genervon claims. He pronounced that a company’s viewpoint was cited merely so that a story could “knock it down.”


Meanwhile, Lisa Jarvis, a contributor with Chemical Engineering News, pronounced a story’s categorical problem was a disaster to support a emanate appropriately.


In my view, all 3 of these reactions are radically legitimate responses to a story. But my take is closest to Jarvis’s. Despite carrying some “absurd observations,” a story does go to good heedfulness to “knock down” Genervon’s claims about GM604. And nonetheless that bid is eventually dissatisfying since a topic of a story isn’t clear.

Despite carrying a right instincts, a story never questions a financial motivations behind Genervon’s pull for selling approval. Exploring a income angle would laid unclothed a fact that this is “bad scholarship preying on unfortunate patients.”


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Steven Walker posted on Apr 6, 2015 during 2:10 pm

It is no surprise, nor is it implicitly wrong, for there to be a distinction ground behind a efforts of drug companies to rise new medicines for terrible diseases like ALS. Implying that usually since a for-profit association (which is where probably all of a medicines come from) wishes to cover a costs of growth and manufacturing, and make an contingent profit, creates a position of a association like Genervon per capitulation of a investigational drug a predator is usually ridiculous. The contention about either information from an early clinical hearing is sufficient to support a redeeming capitulation (which is what Genervon is seeking from FDA), that comes with an enforceable joining on a partial of a association to continue contrast of a drug in clinical trials to endorse reserve and efficacy), is also an intensely critical partial of this story. Finally, this is about a weighing of a intensity for advantage contra a intensity for risk. ALS patients see this drug as their usually choice that competence work in a time they have left, and that also competence turn accessible to them in time. The biggest risk they face by distant is a risk acted by their disease. They wish their possibility to find out if it will work, and they have finished it transparent they are peaceful to attend in a collection of information per how good a drug works, or doesn’t work, as partial of their removing it. The FDA’s Accelerated Approval pathway was combined for situations like this, and has been used many times for other diseases. Why not for ALS? Do we also doubt a motives of, for example, a association that creates newcomer airplanes since they have to make adequate income to cover growth costs and manufacturing, and to pattern a subsequent era of aircraft, a profit? There is many to discuss about how many information we need before a new medicine for a illness like ALS gets initial approval, though assigning antagonistic vigilant since a distinction ground causes investors and companies to take a risk to rise new ones, isn’t one of them. There are dual sides to a timing of an capitulation decision, and a one that frequency gets any coverage is a tellurian cost of loitering a drug that works for a illness like ALS for years. If 3 or 5 years from now, a people hostile capitulation of GM604 finally agree, formed on bigger trials, that a drug is protected and effective, what would we write about a ALS patients who wish it now and never got their chance? we advise we hit a thousands of Persons with ALS (PALS) and Caregivers with ALS (CALS) who are dire for this drug to be authorized now, and learn for yourself since they wish it approved. It is a really critical partial of a story, and we strongly suspect, would change your viewpoint of this situation.


    Kevin Lomangino posted on Apr 6, 2015 during 3:08 pm


    Thanks for holding a time to comment.

    I never suggested or pragmatic that it was implicitly wrong for there to be a distinction ground behind a efforts of drug companies to rise new medicines.

    I did advise that a distinction ground creates a dispute of interest. And we do consider it was The Washington Post’s avocation to call courtesy to that dispute in their reporting.

    This site is about broadcasting and a approach media messages impact health care. We consider people are improved sensitive and make improved decisions when news stories delicately oldster story sources and warning readers to intensity conflicts.

    The Post didn’t do a good pursuit of that in this story, that is since we suspicion it was value essay about.

    Best regards,

    Kevin Lomangino
    Managing Editor


Robert Cornish posted on Apr 6, 2015 during 3:22 pm

My reading of a accelerated capitulation routine is that it allows capitulation for drugs that have shown a verifiable impact on a broker pen that is expected to relate to a clinical outcome. The unite is thankful to continue investigate to determine a association between a broker and a suggestive clinical outcome. And if we examination this conditions correctly, a association has not shown really many of anything, formed on a Post essay and on a company’s press releases.

The progressing criticism seems to contend that companies should be means to sell their drugs though carrying to uncover any genuine or suggestive justification that it does anything. Desire or need ought to be adequate justification that a drug needs to be approved.

Patients though effective therapies competence feel it’s their usually hope. But is that a fake hope? America can’t means to compensate for untested drugs, to see if they work? If a drug does work, a association stands to make a pardonable fortune. It doesn’t merit to make one on a drug that competence not have any certain effect.

I consternation since some-more of a patients are not in a clinical hearing to find out of a drug works. If there are thousands of patients watchful for this therapy, how come a association is observant it deserves capitulation on a basement of study usually 12. And of a 12, did all a patients get a drug? Did all of them respond?

we tumble into a Jarvis stay too. This isn’t sounding like a association has finished what it needs to do to uncover their drug works, or to what degree. And I’d certain hatred to see this routine turn a pathway for drug capitulation in a United States.


    Jim Schmitt posted on Apr 6, 2015 during 4:55 pm

    All ALS sufferers wish is hope.

    Right now, there is none. There is no stream diagnosis accessible to save them from this terrible death.

    My mother is 18 months into her Bulbar ALS diagnosis and she is fast deteriorating. She can no longer eat or speak. She cooking by a feeding tube. It’s formidable for her to reason her conduct erect, and a muscles in her arms and legs are weakening to where she can’t lift anything weighing some-more than a bruise or two, and she wears a prop on her left left leg. She now sleeps detached from her father in a room set adult with with a sanatorium bed and other innumerable medical inclination since she can’t simply navigate a stairs to a second building bedroom. She has to use a device to siphon spit and flem from her throat, and she wears a device to assistance her exhale while she sleeps and has had an initial diaphragm pacemaker-type device commissioned to see if it can jar her diaphragm into assisting her breath.

    For her, it’s usually going to get worse to where she’ll be mentally warning in a invalid physique that will imprison her during her final days.

    She’s really mentally pointy . . . and she wants to live. All she has left, aside from a consistent caring and adore from her family, is hope. Period! And we seriously wish to take that divided from her since we consider Genervon wants to make a distinction from their treatment. You contend Genervon stands to make tens if not hundreds of millions by charity this treatment. Really! How many millions will Genervon make if their diagnosis proves to be a dud? How many would a open trust Genervon with diagnosis they’re user on for other diseases? Your article, and others like it, will usually offer to strengthen a FDA’s preference to not approve Compassionate Care use of this treatment, if that’s a position it eventually takes.

    My mother Kathy has pronounced she would rather take an initial diagnosis for ALS and die from miss of formula in a name of scholarship than simply lay around watchful to die a terrible death. we assume many of a 30,000 residents of a United States who have been diagnosed with ALS would agree. But many of all they wish hope.

    When my mother was initial diagnosed, we looked all over a universe for a legitimate treatment, quite a branch dungeon treatment. All we found was rascal by subhumans who wish to get abounding exploiting people like my mother who wish to live. If Genervon wanted to get abounding off a diagnosis for ALS that gives fake hope, all they’d have to do is open a sanatorium in Mexico usually a few miles south of their Pasadena headquarters. Trust me, ALS patients from all over a world, including my wife, would group to them and compensate on pornographic volume of income for Genervon’s treatment.

    My mother is underneath a caring of one of a nation’s inaugural ALS medical complexes in a nation and her doctor’s have no conflict to her holding a treatment. Genervon is a association that has a good repute in a medical investigate industry. .

    She has practical for many trials though nothing accept patients with a Bulber form of a disease. Furthermore, even if a researchers confirm to eventually embody Bulber ALS sufferers, she’s fast coming a dual year cut-off these trials belong to. So what’s left for her? A fast trail to a terrible death.

    We’re all anticipating a FDA will give Genevon capitulation to discharge a diagnosis to ALS sufferers who volunteer, “volunteer” being a user word. All they wish is hope!


Mike C posted on Apr 6, 2015 during 5:34 pm

As a chairman with ALS, we would like to note that we are not articulate about high cholesterol here, though a illness that is 100% fatal, unconditionally untreatable and as vicious as they come. We have had 20 years of unsuccessful Phase 3 trials with all demeanour of unsure medicine or noxious substances, to such an border that a longest presence is celebrated in remedy groups of clinical trials. Given GM604′s demonstrated reserve and statistically significant, if frequency preliminary, efficacy on biomarkers and clinical results, what is a motive for self-denial it, exactly? AZT (as 3TC) and Bevacizumab got Accelerated Approval in a past. This is a complement user as intended. On what basement repudiate this to ALS patients? What value there is there in hammering in a blindingly apparent regard that Genervon is a for-profit company? One thing we pledge is that had Lisa Jarvis had ALS, she would have been voting for GM604 Accelerated Approval Process with all a limbs she still could control. A slim straw, we say? We, a patients, know that! Nobody is advocating that AAP should be “the pathway for drug approval”.


    Kevin Lomangino posted on Apr 6, 2015 during 7:38 pm

    Thanks to Mike C and Jim Schmitt for a comments. But I’d remind everybody that a purpose of a site is to examination broadcasting — for all audiences. Your viewpoint as engaged, sensitive advocates is really opposite from that of a normal news consumer reading a Post story. It might be blindingly apparent to we that Genervon officials are conflicted in their pull for accelerated approval, and you’re apparently ok with that, though a standard news consumer needs and deserves to have those relations and biases clarified. We acquire additional comments on a peculiarity of a Post story or journalism-related issues. But this is not a forum for advocacy, decisive discussions of science, or low contention of a FDA capitulation process.


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