Announcing Smackdown Grants
The Lowdown on Smackdown Grants:
- Smackdown Grants are $500 - $5,000 seed grants for projects, papers or posts that help speed CF research, innovation, awareness, and general smacking down.
- These projects are all over the map, we know. Some may involve things you wouldn't necessarily associate with "advancing CF research" or "finding a cure." Some may be specific, some vague, some simple, some complex. One of the projects is simply about making an existing online CF database easier to use. Over time, Smackdown Grants may be better organized, but for now, we just want to get started. To our knowledge, no one else is doing anything like this, so we don't have anything to model off of.
- Some of these are "projects" and some are more like be opinion or summary pieces. For example, there may be subjects (e.g., "mild CF", "future treatments") where there's been so much study and writing that folks could use a solid wrap-up piece, with some authoritative color, to make sense of it all. Maybe not "advancing research" but if the Smackdown can elicit such work, it will certainly help to advance CF awareness and understanding.
- If this looks like it will work, we can consider accepting additional projects that anybody might want to see done. Ideally those who submit projects would also put up the funding, or at least help raise it. Which leads to the third point:
- Interested in funding a project? Currently, the CF Smackdown team is going to fund these projects out of our own pockets, but please let us know if you're interested in funding, or co-funding one of these projects.
- Interested in applying for one of these grants? The process is simple (for now), and we will lean on you to provide an all-around solid pitch. Start by telling us (in a 1+ page document sent to firstname.lastname@example.org), what you're interested in working on, why you're well qualified to work on it, and anything else we should know (like the milestones you'll need to reach to accomplish your goal). Here's an example of a non-research-oriented grant request that was recently awarded $5,000. Eventually we'll have a more formalized process, but not yet, so get in while the gettin's good.
Improve CFTR2's UI/UX
Background - the CFTR2 website hosts a fantastically ambitious and practical project "to provide information about specific cystic fibrosis (CF) mutations to patients, researchers, and the general public." It is much needed, and everyone who has helped put this thing together should be knighted.
The issue - The CFTR2 website user interface is, some might say, "janky"; as in, not particularly intuitive or easy to use, at least for regular people on the web. Since so much work has gone into collecting and organizing the data, if there is a chance we can help get more people to use it, and benefit from it, that's a chance worth taking.
The call to action - Let us know if you think you can help. We realize Johns Hopkins may not need or want random strangers working on their website, so if you work for Johns Hopkins, or can collaborate with them, all the better. $5000 up for grabs here.
5T's Big Data - make it useful
(possible $10k grant)
Background - The CFTR 5T variant, aka IVS8(5T) is not well understood, but it is getting increasing attention. It is carried by up to 10% of the U.S. population (yes, 10%), and, especially when found in trans to a major CF mutation, and when associated with a higher TG repeat sequence (e.g., TG12 or TG13), appears to contribute to a range of phenotypes, from CBAVD to sinusitis to full-blown CF. To some (e.g., Europe), the 5T (associated with (TG)13) is "CF-Causing"; to others, its effects are less certain.
The issue(s) - Because it is so common, and increasingly prevalent in newborn screening, there will be more and more patients identified with the 5T and with an "uncertain" prognosis. Sweat tests are often borderline, penetrance can be minimal or significant, organ-specific, or delayed over time. Those affected by the 5T could benefit greatly from a comprehensive analysis of known and likely outcomes in patients with the TG/13-5T/M and TG/12-5T/M genotype. Given the range of symptoms potentially attributable to the 5T, and its prevalence in the population, the opportunities for better understanding are promising. The fact that Ivacaftor may be an effective potentiator of the 5T's residual CFTR makes things all the more interesting...
Call to action - We have several hundred papers available on the subject. To get started, here's one paper to look at, and here's a simple spreadsheet, both with assumptions that could certainly be refined. If this project is up your alley, contact us, as we are eager to help you get going. Depending on your potential scope, we could double our grant offering to $10,000.
Dig deeper into "CRMS"
Background - There's lots of talk about "mild" or "atypical" CF, "CRMS", and "CFTR-related disorders". Increasingly thorough administration of newborn screening programs (NBS) are resulting in more and more documented cases with such vague-ish labels.
The issue(s) -
- There's a whole lot of ambiguity as to the meanings of these terms.
- Aside from somewhat bright-line delineations such as PS and PI, some would say there is no "mild" CF, only "delayed" CF. That's kind of a big question just begging for a clearer answer.
- Some argue that genotype and biomarkers such as sweat tests fall upon a somewhat smooth curve of association with likely CF phenotype (e.g., CBAVD, mild pulmonary disease, bronchiectasis, etc). Others point to mutations that may throw off misleading readings (3849 + 10 kb C --> T, IVS8-5T, etc)
and, for example, have organ-specific penetrance. There's some room for more understanding here.
- Could *preventative* treatment regimens play an important role in CRMS, vs waiting for clinical (and potentially irreversible) symptoms? (see Project #4)
Call to action - There's a lot here. Can you clarify the issue(s)? If so, let us know. There's an argument to be made that these (and more CRMS-related questions) could be broken out into separate projects.
Revisit the "Preventative" Approach
Background - Is it necessary to wait for clinical symptoms to treat an underlying condition? Lung disease and damage can begin "silently" and subclinically through progressive mucus plugging, and a subsequent cascade of other events (infection, inflammation, scarring, etc).
Issue - Some believe treatments should begin only when clinical symptoms develop. But might some symptoms of CF (late onset, mild, etc) be outright preventable with more pre-emptive treatment regimens? E.g., saline, dornase alpha, even Kalydeco/Ivacaftor in the presence of gating mutations or residual CFTR?
Call to action - this one is controversial with clinicians, researchers, and insurers alike, but is not going away, particularly as we better understand the potential of recent breakthrough treatments. Anyone care to make a case for or against more aggressive pre-emptive treatment?
Make CFsmackdown.com more helpful
1 Grant awarded
Background - CF sucks in so many ways, so the more CFsmackdown can help people affected by it, perhaps in ways they're not being helped with already, all the better.
Issue: Some ways this site can help include the projects listed in the Smackdown grants on this page. Some other ways could include offering helpful online tools to:
- More easily keep track of and view test results (lung function, sweat, vitamins, etc) over time
- Better search through research and related papers. CF Smackdown has about 1000 papers in PDF form that it plans to post, likely grouped into "anyone with the link" Google Drive folders. This feature, however, does not provide search. Some way to search these files could be very helpful.
- Ability to rate and view curated ratings for different CF-related products (nebulizers, etc) and services (clinics, etc)
- Anything else that might help a patient or family on this website.
Call to action - Are you a developer who who has ideas for tools, apps, or other resources we could employ on CFsmackdown.com which could help a lot of people? If so, please contact us.
Mapping out (and developing) technologies that will help CF patients in the future
1 Grant awarded
Background - Most of us are aware that promising treatments like Kalydeco are now emerging from the pipeline. What about other technologies that might improve lives -- self-administered sputum cultures, home-based lung function tests, smartphone apps that sync with your clinician, etc. What other technologies are coming down the pike that might improve lives?
Issue - Some of this technology already exists, but it isn't well publicized, understood, or distributed.
Call to action - are you tracking this stuff? If so, CF Smackdown would like to help you share this info with others. Are you creating any of this stuff? Contact us.
Holistic or alternative medicine and what you read on the web - separating the true from the poo
Background - if you've spent any time in online CF forums, you will have read a lot of, well, crap.
Issue - deeply embedded in that mountain of crap are occasional nuggets of actual wisdom or utility (e.g., about diet, exercise, even supplements). With that said, it is hard to sift through a mountain of crap.
Call to action - calling all level-headed, non-alarmist, non-conspiracy theorists who aren't being paid to promote something-- or perhaps a clinician who has examined alternative treatments with an open mind. Can you offer a solid summary of what's proven to help, what's suspected to help, and what should remain a part of the mountain of crap? Let us know.
Background - Granted, modifier genes (TGFbeta1, MBL2, and more) play a role in disease penetrance.
Issue - OK, that's cool. Which ones, and how much? The decreasing costs of genetic testing will make this information all the more in demand.
Call to action - clarify what we know, don't know, and what we suspect. Contact us if you're interested in this project.
What the heck are the rules?
Background - OK. Clearly exposure to another infected CF patient, and smoking, are not cool for a CFer (smoking is actually not cool for anyone, but that's another project). There are plenty of other potential hazards, or at least things to be careful around, and it's hard to keep track.
Issue - For example, hot tubs, heated pools, bath and sink drains, even horses may be threats. What are the top 10 things to avoid?
Call to action - can anyone provide a fresh take on a list of hazards to a CF patient that is down-to-earth and practical? If so, contact us.
Background - CF research, with a lot of help from the absolutely ass-kicking CF Foundation along with private companies, has gained momentum in recent years, and Kalydeco is an example of a breakthrough.
Issue - There's a lot of other stuff coming down the pipeline - some reasonably well known (e.g., it may appear in the CFF's "Pipeline", but it may not). Much is dedicated to the most common genotypes, though everyone's goal is 100% coverage. Vertex has taken the lead, but AbbVie, Galapagos, Pfizer and others are nipping at their heels. And there's stuff going on in labs that would probably blow our mind.
Call to action - could you create an original paper to help the Smackdown summarize, comprehensively, what is coming, from relative sure-things, to speculative approaches, from treatments for the majority of mutations, to treatments for the rarer mutations? If so, please let us know. There are already some pretty good summaries out there, like this, but there's so much to look forward to, and we hope to help people better know and understand what good may be coming.
Gamification of CF
Background - Yes, this sounds inappropriate and ridiculous at first. But for those fighting CF, very little is inappropriate or ridiculous, especially if it helps.
Issue - CF patients have more control over their own health than many might think. Compliance with treatment protocol is a big part of it, but "compliance with treatment protocol" doesn't sound as fun as it should. What if there were a way of "gamifying" - not only compliance, but also extra credit for stuff like exercising, etc, allowing patients (and potentially parents or partners of patients)?
Call to action - Create an app - a good app - that makes it happen. This could be really cool.