Doctor With Rare Disease Crowdsourced His Own Cure, And Is Now Using That Network To Work On COVID-19

from the the-internet-to-the-rescue dept

A recent episode of NPR's Fresh Air ran an amazing interview with Dr. David Fajgenbaum, who was diagnosed years ago with the rare Castleman's Disease, about which very little information was known (and the general prognosis was grim). Fajgenbaum talks about how he ended up in hospitals believing that he was about to die five separate times (he even had his last rites read to him), but then set up his own organization to try to crowdsource a cure. He details the full story in his book that was published last fall, called Chasing My Cure.

The good news is through that crowdsourcing effort, called the Castleman Disease Collaborative Network (CDCN), they at least found a treatment that (for now...) appears to work for Fajgenbaum himself:

The biggest difference between this fifth time I nearly died and the previous four times is that, at this stage, I was engaged. And I had the ultimate date in mind, which is our wedding date, May 24, 2014 - in mind as the driver to say, I need to find something. I failed to respond to all these drugs. There's nothing left for me. But I have to make it to May 24, 2014. And so thankfully, this combination of seven chemotherapies saved my life.

And when I got out of the hospital, I was able to go back to all those samples I'd been storing on myself and performed a series of experiments where, from within my experiments, I found this pattern that suggested this one communication line in the immune system called the mTOR pathway was highly activated. And what was so exciting about finding this communication line turned on is that there is a drug that was developed 30 years ago that's really good at turning it off. It's called sirolimus.

And just knowing that this pathway was on did not guarantee that blocking it would work and that taking this drug would save my life. In fact, the immune system is a very finicky system. And basically, turning off this communication line could have actually caused even more problems. No one knew because this drug had never been given to a Castleman disease patient before.

But really, knowing that I needed to try something if I wanted to make it to our wedding date, I decided to take the leap of faith and to start taking this drug as the first patient with my disease ever to take sirolimus back in early 2014. And amazingly, thankfully, I was able to make it to Caitlin and I's wedding date. And you wouldn't think this is too important, Dave, but my hair grew back just in time.

He admits that the treatment that works for him has not been shown to work for everyone with Castleman's -- in fact, it appears to help only about 1/3 of those treated with it. But just the fact that it's been helping some is worth noting.

And here's the really interesting part: as we've gone into this whole pandemic thing, many of the participants in the CDCN have noticed some similarities between the issues with Castleman's disease, and with what people are reporting about COVID-19. So they've been repurposing the crowdsourcing effort to work on COVID-19:

DAVIES: So the collaborative that you formed to try and share information and leads about treating Castleman is now focused on COVID-19. I mean, this is obviously an urgent public health matter. Did you see similarities between Castleman disease and COVID-19 that made this a good fit?

FAJGENBAUM: That's right. So early on in this pandemic, it became clear that the most deadly aspect of COVID-19 is actually the cytokine storm that the virus ignites. And the cytokine storm that it ignites is almost identical.

While there are lots of different groups working on different ideas -- from vaccines to antibodies -- the CDCN is focused on what it does best: looking to see if there are FDA approved drugs out there that might have some useful effect here, and recognizing that the only way to really figure that out was to actually get the data (something very few others seemed set up to do):

And so with this similarity between - at the very basic mechanism, what drives the deadliness of COVID-19 is almost identical to what makes Castleman disease so deadly, it's these - the cytokine storm. That was one aspect of it. The second is that we know that drug repurposing is our best shot at identifying a drug that can help patients in the short-term, so a drug that's either already FDA-approved or a drug that is maybe experimental but is not yet approved for anything that could be repurposed for COVID-19. We knew that was our best shot.

And, Dave, I found myself, in early March, thinking to myself, I really hope that some research group out there that has experience studying cytokine storms and has experience doing drug repurposing will follow our blueprint and search for drugs that can be repurposed against this cytokine storm. And I was sitting there hoping that someone would do it.

And then I realized that I needed to listen to my own advice, and that if I'm going to hope that some research lab out there that has experience with cytokine storms and repurposing would turn their effort towards this, then I would need to turn my effort towards this. This is what we've been doing to chase my cure for these years. And we felt like we needed to do what we could in the fight against COVID-19.

The really incredible part here is that he notes that there's no official tracking of the various tests that doctors are doing, and that's a key aspect of what they've set up for doctors around the world:

I mean, you can basically think about the state that we're in right now is that doctors are trying all kinds of things - hydroxychloroquine, remdesivir and many other drugs. Yet there's no system in place to track what's working and what's not working. And so recognizing that this wasn't being done, we decided to build a database, what we called the CORONA database - COVID-19 Registry of Off-label & New Agents. So it's a database to track all of the drugs that have been used against COVID-19 to date 'cause we want to know everything that's been tried, and we want to see what's working and what's not working. And amazingly, almost 150 different drugs have already been tried against COVID-19. And of course, we hear about a handful of them, but there are a lot of others that have already been tried as well. And so we've created this giant database from - right now it's over 11,000 patients and growing - to collect data on every drug that's been used and so that we can really dig into what's working and what's not working.

And the second part of this equation is that you want to track what's being used, but then you also want to piece together all of the data that's emerging from labs around the world to try to map out what are maybe some new drugs that we could start trying to use? What are some new pathways from all of this data that we should start going after? And interestingly, from the state of - we're finding signals that are Castleman-like, basically. A number of the features that we're seeing in the COVID-19 data, these same features we see in Castleman disease.

Think about that first part for a second. In the past, if you wanted to have a database of how certain drugs were used to treat different diseases, and what the impacts of those treatments were, you'd probably need a government to set up a program -- with lots of bureaucracy and mess. But here, a doctor and some other interested researchers were able to set up their own such database on the fly and get a massive amount of data piped into it, from which they can do all sorts of (hopefully!) useful analysis.

This is not to say they're ignoring other approaches -- because these things work together. In the interview, it's mentioned that the crowdsourcing team at CDCN has combed through over 2,500 published papers to look for potential promising treatments.

Also important: they're being very open about all of this. While some keep insisting that we need to lock up successful treatments and ideas, Fajgenbaum recognizes the power of sharing information widely (the very root of crowdsourcing, after all):

You know, what we really want to do with this corona project is to map out everything that's being tried, to put in one place all of the studies that are being published, all of the data on every drug that's being tried so that other people can go to it and they can kind of decide for themselves what looks promising and what doesn't. We didn't build this to say this is the drug and that's not the drug; we built this to say this is where all the data is. If anyone wants to use the data, we have this very data-first approach. Anyone can use the data.

And from our perspective, we want to use the data to determine and to prioritize what drugs should go on to clinical trials. So the fact is, is that this drug is already being studied in randomized controlled trials, and that's all that we can really ask for. We want to use the database to say what's being given, what looks really promising and what should go forward to randomized controlled trial. We don't want to use the database to say this drug should be given or that drugs should not be given. So we're hopeful.

And actually, we put together a paper based on our first pass of analyses of the data and recently received favorable reviews. So hoping that that'll get published in peer-reviewed journal shortly and that we will be able to get the word out about this database. But the goal is not to say this is the drug that everyone should be on; the goal is to say these are the promising drugs. Let's make sure that we don't forget anything along the way because you're right - I think that we all have a tendency to jump on every major drug or every major headline. But we need to keep an eye on all the drugs that are being tried and make sure that we're doing this really systematically.

Once again, the ability for anyone to just setup and build something on the internet, without needing to ask for approval or go through some big bureaucratic process, may be helpful yet again, and hopefully the very open process of bringing in data, and sharing it outward, will lead to real breakthroughs.

Hide this

Thank you for reading this Techdirt post. With so many things competing for everyone’s attention these days, we really appreciate you giving us your time. We work hard every day to put quality content out there for our community.

Techdirt is one of the few remaining truly independent media outlets. We do not have a giant corporation behind us, and we rely heavily on our community to support us, in an age when advertisers are increasingly uninterested in sponsoring small, independent sites — especially a site like ours that is unwilling to pull punches in its reporting and analysis.

While other websites have resorted to paywalls, registration requirements, and increasingly annoying/intrusive advertising, we have always kept Techdirt open and available to anyone. But in order to continue doing so, we need your support. We offer a variety of ways for our readers to support us, from direct donations to special subscriptions and cool merchandise — and every little bit helps. Thank you.

–The Techdirt Team

Filed Under: castleman's disease, covid, covid-19, crowdsourcing, cytokine storm, david fajgenbaum
Companies: cdcn

Reader Comments

Subscribe: RSS

View by: Time | Thread

  1. icon
    Anonymous Anonymous Coward (profile), 27 May 2020 @ 6:02pm

    Money talks, bullshit walks, the right way not even considered

    While I endorse the concept of openness in this type of research there is still the problem of regulatory agencies sticking their noses into the process before any viable results emerge. And with a plethora of profit oriented entities vying to be 'the one' the chances that an open sourced (hopefully patented but not monetized), non profit oriented result to win has little or no chance.

    reply to this | link to this | view in thread ]

  2. This comment has been flagged by the community. Click here to show it
    Anonymous Coward, 27 May 2020 @ 6:03pm

    The only cure is still nerve regeneration from stem cell therapies that aren't physically possible with our current understanding of biology though at some point in the future more advances may be made.

    The use bleach joke is dump it in the semiconductor work bench to "fix" the source of the disease, possibly in the distilled purified water or hydrophilic or hydrophilic test chemical or the Photoresist chemical.

    reply to this | link to this | view in thread ]

  3. identicon
    Anonymous Coward, 27 May 2020 @ 6:25pm


    Uh. WTAF?

    reply to this | link to this | view in thread ]

  4. icon
    Anonymous Anonymous Coward (profile), 27 May 2020 @ 6:33pm

    Re: Re:

    Well, obviously you are responding to the smartest person in the world, as they have all the answers. You also know that they are a medical professional, you can tell by the MD appended to their username. So, no worries, they have stated that no cure or vaccine is possible as our current understanding of biology is lacking. BTW, we can take that as a serious prediction that 'we are all gonna die'.


    reply to this | link to this | view in thread ]

  5. identicon
    Anonymous Coward, 28 May 2020 @ 1:43am

    Crowdsourcing? Hamilton's not going to like this, is he?

    reply to this | link to this | view in thread ]

  6. icon
    PaulT (profile), 28 May 2020 @ 8:12am

    Re: Re:

    Well, those were words. They didn't make much sense in the order they were typed in, but the were definitely in the English language...

    reply to this | link to this | view in thread ]

  7. identicon
    Anonymous Coward, 28 May 2020 @ 9:17am

    'the ability for anyone to just setup and build something on the internet, without needing to ask for approval or go through some big bureaucratic process, may be helpful yet again, and hopefully the very open process of bringing in data, and sharing it outward, will lead to real breakthroughs'

    and there lies the problem! too many will want to stop this sort of thing from happening because they're afraid that they might lose the availability of earning something from it! it's the old story, 'i dont care about anything/anyone. i care about making myself money!'
    and those with this attitude are, obviously, not only big pharma but despicably, politicians/members of our government(s)!

    reply to this | link to this | view in thread ]

  8. identicon
    RD, 28 May 2020 @ 10:04am

    But not..

    But not Hydroxychrlooquine, no no no no no, we can't look into that one at all. That is a dangerous drug that kills you dead if you even take a single dose! It kills all in it's path and should never be used for anything under any circumstances. In no way are you to look into any possible efficacy of Hq, because it's already been reported and established as a de facto truth that it is dangerous and will not work at all. Silence and banning any discussion about it, as well as removal of the offending speech and poster, is the only acceptable answer. In no way should any theories be tested, discussed or attempt to be proven. Full embargo is the only solution.

    reply to this | link to this | view in thread ]

  9. identicon
    Anonymous Coward, 28 May 2020 @ 10:20am

    Re: Hydroxychloroquine

    It's been tested, it's been found actively harmful. There's no need to sacrifice more people to it.

    reply to this | link to this | view in thread ]

  10. icon
    PaulT (profile), 28 May 2020 @ 10:36pm

    Re: But not..

    Are you actually getting dumber in these threads? It seems like it.

    reply to this | link to this | view in thread ]

  11. identicon
    RD, 29 May 2020 @ 6:42am

    Re: Re: Hydroxychloroquine

    In tests that didn't administrate it properly, didn't include the zinc component, and delivered it too late in the process. Flawed "studies" that tested literally a handful of people incorrectly then pulled the plug when things went wrong (rightfully so), then decried the entire proposed treatment as invalid on a test with 8 or 20 people, and stopped any further because it's "settled." Yeah right.

    Meanwhile other verifiable tests around the world, as well as many anecdotal and informal tests, done the right way, showed much improved results, but those are ignored and hand-waved away as invalid. I'm sure the people who survived and didn't die feel the treatment was "invalid"

    But you can't talk about that because its a "conspiracy theory" and "settled." So no one is ever allowed to broach the subject ever again nor do any further testing for efficacy (though the NIH is actually going to do another round, but again they are going to leave out some of the other components that would make it work to make it a flawed study so it will fail. probably only treat late-stage and/or the elderly as well just to ensure it)

    reply to this | link to this | view in thread ]

  12. identicon
    RD, 29 May 2020 @ 6:43am

    Re: Re: But not..

    Yep! I wear my "ask questions and don't just blindly accept what Big Brother tells you just because you are too lazy to think or research yourself" dumbness with pride.

    Enjoy your frog-march to the comforting arms of totalitarianism you are so willing to embrace without question.

    reply to this | link to this | view in thread ]

  13. icon
    PaulT (profile), 29 May 2020 @ 11:10pm

    Re: Re: Re: But not..

    "I wear my "ask questions and don't just blindly accept"

    Except, you're not just "asking questions". You're rejecting the sensible conclusions that have already been reached by professionals on the subject, and parroting the bullshit intended to convince people to to follow real medical advice. No study is perfect and there is room to return to them, but there are limited resources and limited time and if another effective solution is to be found elsewhere then it makes sense not to concentrate something that's already been checked. You may disagree, but then I'd ask for your credentials, which I believe should be higher than "I read stuff online".

    I'm sorry that Trump's momentary pet obsession didn't turn out to be the miracle cure he wanted it to be, but it's time to follow the doctors instead of the politicians. Like you should have done in the first place, since that would have saved lived and shortened the need for you to be in the situation you're in.

    "Enjoy your frog-march to the comforting arms of totalitarianism "

    Yeah, this is why I consider you to have turned into an idiot on this subject, and I wonder what sources have been lying to you in order for you to reach this position. Because some of the words you're parroting sound very much like words I've heard from certain others, and those people are not reliable sources for anything.

    But, that doesn't matter. I'm in a place where the options on the table that you're complaining about are not even being considered, and where I'm not surrounded by idiots who think that vaccination is some kind of a plot to do things that would already be possible without it.

    reply to this | link to this | view in thread ]

  14. icon
    PaulT (profile), 29 May 2020 @ 11:17pm

    Re: Re: Re: Hydroxychloroquine

    "But you can't talk about that because its a "conspiracy theory" and "settled.""

    It's not settled. However, it is the dumber side of the conspiracy theory echo chamber that's been obsessed specifically with zinc through this whole think - largely because they sell zinc supplements and are less likely to be sued over negative effects than they are over things like colloidal silver. For someone who claims to be free-thinking, you do seem to be repeating a lot of what those guys say.

    Also, thanks in part to gutting of the teams that would have been responsible for this stuff before the pandemic, funding and staffing are fairly limited, and when you have limited resources you follow new avenues rather than the ones that have already failed. I'm sure it will be looked at again, but I'd rather be because of medical data and by medical professions who is already second guessing the actual doctors because he jumped to some unqualified conclusions.

    reply to this | link to this | view in thread ]

Add Your Comment

Have a Techdirt Account? Sign in now. Want one? Register here

Subscribe to the Techdirt Daily newsletter

Comment Options:

  • Use markdown. Use plain text.
  • Make this the First Word or Last Word. No thanks. (get credits or sign in to see balance)    
  • Remember name/email/url (set a cookie)


Add A Reply

Have a Techdirt Account? Sign in now. Want one? Register here

Subscribe to the Techdirt Daily newsletter

Comment Options:

  • Use markdown. Use plain text.
  • Make this the First Word or Last Word. No thanks. (get credits or sign in to see balance)    
  • Remember name/email/url (set a cookie)

Follow Techdirt

Tech & COVID is a new project by
Techdirt, with sponsorship from

Essential Reading
Techdirt Insider Chat
Recent Stories

This site, like most other sites on the web, uses cookies. For more information, see our privacy policy. Got it

Email This

This feature is only available to registered users. Register or sign in to use it.