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‘Home run’ gene therapy for Duchenne muscular dystrophy: Investor-oriented coverage pitches promise but …

Michael Joyce is a writer-producer with HealthNewsReview.org and tweets as @mlmjoyce

gene therapy for Duchenne muscular dystrophy

Sarepta stock (SRPT) price spike coincides with last Monday morning’s investor event.

Biotech investor magazines and websites were buzzing last week over the news that Boston-based Sarepta Pharmaceuticals released preliminary findings of an experimental gene therapy for Duchenne muscular dystrophy (DMD).

Various analysts dubbed it “spectacular,” a “home run,” and a “game-changer.”

One analyst with Baird, a financial company, went so far as to proclaim:

“Given today’s data, it is really hard to believe this study won’t be positive, ushering in a new paradigm that we expect will transform outcomes for patients diagnosed with this horrible disease.”

That’s called a guess. And these are called business analysts. Predicting transformed outcomes is not their purview.

This is disconcerting for a very important, but often neglected, reason. In a U.S. pharmaceutical industry that accounts for nearly half of the global market, and is valued at about $500 billion, it’s becoming increasingly common that the public’s first glimpse into a “promising” therapy comes from people whose job it is to size up the market potential of emerging therapies … not necessarily the potential for clear patient benefits, especially when weighed against potential harms and costs.

Why caution is warranted

Last year our publisher, Gary Schwitzer, wrote:

Gene therapies are still largely experimental–and the few that are FDA approved are enormously expensive.

“The flow of healthcare and research news to the public can be contaminated at various points along a polluted stream of information.”

These investor publications are frequently one of those points. What’s worrisome about that is they’re pretty far upstream; often basing their “analysis” on corporate news releases and extremely preliminary studies. In doing so they’re more reactionary than analytical and — as in this case — they often miss key information, such as:

  • This was a Phase I-II clinical trial of just three boys presented at an investors event.  The data have not been published nor peer-reviewed.
  • The follow-up was just three months. Whether the positive results will prove to be sustainable is unclear.
  • The boys were young (4, 5, and 6), so their disease was relatively mild compared to older patients whose disease has progressed to a more severe phase.
  • Two of the boys experienced elevated liver enzymes, which did improve with steroid therapy. The full extent of side effects is unknown.

We were encouraged that STAT included most of these cautionary points, but discouraged when they went on to write:

“… the effect of the gene therapy … is profound enough to suggest it may halt or even reverse the effects of Duchenne in certain patients.”

That’s rather speculative and somewhat at odds with their apt cautionary points.

Pitching promise

There are other issues raised by this investor-oriented coverage.

It’s compelling that in the dozen-plus such articles I read, only a few sought out commentary from someone not affiliated with the study.  If more reporters had done so they very well may have gotten a cautionary note–not just about the key information bulleted above, but that we’ve been down this road many times before with DMD.

In January, for example, Solid Bioscience had a record-setting initial public offering after very preliminary results of gene transfer in dogs with DMD showed enough promise to launch clinical trials in humans. But just two months later the FDA stopped the trial because of side effects.

In April, Pfizer launched a Phase I gene therapy clinical trial for DMD. Even before any results were in, the CEO of a DMD advocacy group said the therapy could “dramatically change outcomes” and be “a potentially transformational treatment option” for patients.

The Sarepta gene-modifying research creating a buzz this week is funded by several patient advocacy groups. This raises two more issues. First, that the default narrative of this investor-oriented coverage is one of advocacy. It’s opinion-based. Not evidence-based.

Susan Molchan, MD

Second, as Susan Molchan, MD reported for HealthNewsReview.org in 2016, there is often a two-way flow of money between Duchenne advocacy groups and drug companies developing treatments. Much of this money is not just for research, but also lobbying. She noted that very few advocacy groups refuse industry funding, and they aren’t required to disclose the money they accept. Molchan felt this raised an important question:

“Whose interests are being served when an advocacy organization advocates vigorously on behalf of a drug that could earn millions in profits for a drug company?”

And a related question could be: Who benefits from the inevitable rush of “me-too-drugs” that often get approved based on marginal studies showing questionable safety and/or effectiveness?

The long road ahead

“Duchenne muscular dystrophy is a horrible disease and we can only hope that Sarepta’s preliminary results will persist and be confirmed in more patients,” said Harold DeMonaco, MS, one of our longtime expert reviewers and a visiting scientist at the MIT Sloan School of Management.

But DeMonaco also feels that taking a long view provides some much-needed context.

Harold DeMonaco, MS

“There’s a stark reality that’s being overlooked in many of these overly optimistic reviews and that’s this: The majority of biologics never see the light of commercialization. The Biotechnology Innovation Tracker Organization report covering 2006-2015 provides insight into the long road ahead. The probability of success of proceeding from Phase 1 to FDA approval is 9.6%. If history is any measure, the gene therapies of Sarepta, Solid Bioscience, and Pfizer have a less than 1 in 10 chance of making it past the regulatory hurdles and becoming established patient therapies.”

Taking umbrage

It’s not unusual that when we point out such inadequacies of coverage — especially concerning diseases that are fatal — that some people might take umbrage with our criticism. They might say we are being insensitive to suffering. Or even, that we are somehow taking away hope.

On the contrary, our project has tracked journalism – and other media messages – that take patients and families on a roller-coaster ride of inflated promises, fostering false hope and — in doing so — can lead to more suffering in a group of vulnerable people who already have more than their fair share.


This post has been updated to clarify that analysts don’t necessarily profit when a stock they follow goes up.

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