Hope & hype on unpublished Huntington’s Disease trial splashy news

Some conditions like Huntington’s Disease are so bad and there are no treatments such that any bit of encouraging news can be cause for legitimate excitement, but at the same time things can go too far potentially, especially if there is news coverage not backed up by a published paper or some other definitive source of data. This kind of situation risks going from science over the line to hype.

The splashy news a couple days ago that there may have been a “major breakthrough” on Huntington’s Disease caught my eye. I was hoping that there was concrete reason for optimism such as a new paper and no hype here. Unfortunately, it feels to me like this Huntington’s Disease story was premature and hyped in the media in some cases. It’s not fair to patients to oversell scientific developments and especially to make bold statements before papers are published. Again, Huntington’s is a terrible disease. The patient advocacy community has been hard at work to advance new transformative therapies so they deserve balanced coverage.

A number of media outlets overdid it on this story about research out of University College London (UCL). The media buzz seems to have originated at least in part from a UCL press release. For instance, the BBC was too exuberant in my opinion with its header, “Huntington’s breakthrough may stop disease.” First, we can ask whether this is really a bona fide breakthrough. Who knows? Maybe that’s a judgement call and we will have to await eventual publication of the paper at the heart of this to be sure. The “may stop the disease” part of the BBC headline is questionable at this point too as there isn’t any evidence for that in humans.

In the long run, I hope the optimism turns out to be justified.

What’s the scientific approach here?

The unpublished study in question apparently uses a targeted approach with a product called Ionis-HTTRx to try to lower the levels of toxic mutant huntingtin, which it apparently did to some extent with what seems to be a good safety profile. Sounds pretty cool and important to me. But again no data were shown or even mentioned in detail and as best as I could tell there was no indication of the actual quantitative nature of the reduction in huntington. Small? Medium? Large? Furthermore, again there is no evidence that this lowering affected the clinical course of the disease in the patients. It’s just too soon to say.

Another problem here is that there is no particular logical basis that I can see for this story to be coming out now (update: a day later someone pointed me to this biotech transaction announced around the same time for the companies involved so that could have prompted the PRs and press attention). There is again no new paper published and as far as I can tell, no new speech at a conference describing the data. In the BBC piece we are told those will be coming sometime “next year.” So why announce this as a big breakthrough now? No reason is given that I saw.  It is concerning that this might be one of those events we sometimes call, “science by press release.”

One Alzheimer’s researcher quoted in the media on this “breakthrough” gushed about it:

“I really think this is, potentially, the biggest breakthrough in neurodegenerative disease in the past 50 years. That sounds like hyperbole – in a year I might be embarrassed by saying that – but that’s how I feel at the moment.”

In the past 50 years in all of neurodegenerative disease research? Really? I’m not so sure. Maybe he’s seen data that we haven’t?

On Facebook, the wonderful HD patient advocate Judy Roberson took an appropriately balanced view of this development:

“If this works, it will be the first treatment for HD. Hopeful time, but we’ll see” and “We need good news, so this is important to hear, but there’s a long way to go. That said, we can celebrate this advance and the hope it brings!”

I asked Hank Greely, Professor and Director of the Center for Law and the Biosciences at Stanford, about the splashy UK media coverage of this announcement and he voiced some concerns:

“Biohype is not confined to the US. This British report of a Huntington Disease’s “breakthrough” is based on an apparently unpublished study that showed levels of the huntingtin protein were lower in the participants’ cerebral spinal fluid – and discusses no evidence of any actual changes in those participants’ condition. Exciting – yes. A “breakthrough” – not yet.”

I agree with Judy’s and Hank’s takes on this.

I’m excited too as this sounds like a step forward, but going way over the top helps no one.

You can see a screenshot from Google News showing 3 different headlines on this Huntington’s Disease story above. Which one seems more responsible and balanced to you? The CNN headline in the middle strikes me as at least relatively more balanced.

In the somewhat breathless BBC piece, amongst all the upbeat language is one small section that serves as a caveat: “Doctors are not calling this a cure. They still need vital long-term data to show whether lowering levels of huntingtin will change the course of the disease.”

In other words, no one knows at all if this will be effective clinically. I hope it will, but it’s too soon to jump to conclusions. Keep in mind that even if this isn’t an outright cure, but were able to be a treatment that delays the course of the disease by say 10 years that’d be a major breakthrough development, but we just don’t know if there’ll be any efficacy at all.

In a piece on this development in The Guardian, which interestingly is somewhat more balanced, they also note limitations: “The trial was too small, and not long enough, to show whether patients’ clinical symptoms improved, but Roche is now expected to launch a major trial aimed at testing this.” The new trial will be exciting to watch, but this small initial media report sounds like just one important step of many needed in the path forward.

Overall, this clinical study sounds exciting to me and I can’t wait to see the eventual paper, but I think this media buzz with no paper or meeting report to support it at this time has risks to it.