ALS Advocacy

ALS Advocacy
Lou Gehrig's Disease - Motor Neuron Disease - Amyotrophic Lateral Sclerosis
Thought it had been cured by now? Still no known cause. Still no cure. Still quickly fatal. Still outrageous.

Wednesday, August 19, 2015

Say What?

It started on August 13 with a tweet from an ALSA executive, "#ALSIceBucketChallenge donations have led to a significant ALS research discovery! bit.ly/1DQByYZ "

Wow.  Impressive.  Holy cow.  That was fast.  Say what?

The press release was very carefully couched and didn't quite mesh with the cause-and-effect excitement of the tweet, but the seed was planted.

Then it continued with organizational tweets with wording that matched exactly.


Amazing. Remarkable. Cool.  Say what?

Then yesterday there was the ALSA mass emailing -- Subject line: "This is amazing news!"


Yes, amazing.  Fabulous. Incredible.  Say what?

The cause-and-effect implication is clear.  IBC donations to ALSA led to this discovery?  That's fast science.  How refreshing.  Say what?

We didn't get a good list of  #ALSicebucketchallenge checks written to researchers last year. We saw numbers related to committed funds.  In these lists of funded research, I don't see this project listed in the 2014 file but Dr. Wong is listed as a $160,000 grant recipient in 2015. 

The full research paper on this "significant #ALS research discovery" sits behind a paywall.  My curiosity won last night.  I bought access. Let's see how this breakthrough was funded with IBC funds, was discovered, was written up, was reviewed, and was submitted to a journal between August. 2014, and March, 2015.  That's cool fast science.  Say what?

Acknowledgements at the end of the paper included that the project was funded in part by
  • The Robert Packard Center for ALS Research
  • Muscular Dystrophy Association
  • The Amyotrophic Lateral Sclerosis Association
  • Target ALS
  • The Johns Hopkins University Neuropathology Pelda Fund
  • The Johns Hopkins University Alzheimer's Research Center (NIH P50AG05146)
  • The Samuel I. Newhouse Foundation

Unfortunately we don't know how many dollars were involved or how much each agency contributed or the dates of the grants.

The paper itself is complicated and involved experiments and evaluations on mice followed by more work on deceased PALS' brains.  There isn't a timeline but it would have taken a miraculous change in the ALS research paradigm for any substantial amount of this discovery work to have been carried out in Q4 of 2014.  I would be amazed if the illustrations alone could have been made and approved in six months, let alone all of the underlying work.

And there was even mention in the paper of a patent application filed by two of the authors. 

So the "Say what?" response keeps going off when we read the tweets and the emails.  How can this be?  Is it a deceitful way to make people feel good about their last year's IBC generosity so they'll do it again?  Or was there really a scientific funding-to-discovery-to-journal miracle in six months?

People with ALS and their caregivers have been pressing hard for the FDA to speed up approvals. They get pushback from organizations including ALSA that good science takes a long time.  Then we get self-serving fundraising messages that our donations led to a "breakthrough" in under six months in some remarkable fast science.  Say what?

People are dying.  They deserve some clarity.

If my "Say what?" reaction was out of line, I'm all ears.

2 comments:

  1. What a promising breakthrough. Great to see because it's been a long time without a ton of promise.

    I hope the part of Dr. Wong's comments that doesn't get lost is the fact that his lab has a long way to go. If we want to see that progress (and other valuable research) continue we've got to keep up the pressure and funding.

    I hope additional money from the IBC #1 and the on-going fund raising efforts like Round #2 can help there.

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  2. So, why don't we just get information on dollars budgeted, dollars spent, sources of funds, goal, and result for every project? Seems simple enough.

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