[HN Gopher] Ask HN: How to raise funds for rare disease research?
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Ask HN: How to raise funds for rare disease research?
If that's okay I would like to ask the HN community for their
advice on a personal matter. My beautiful daughter Nil (3yo), has
a rare genetic disorder called Kleefstra Syndrome (KS). She cannot
walk or talk at this point. Doctors believe she will walk
eventually, but speech they are not so sure about. KS involves
partial chromosome deletion (or sometimes mutation) of a particular
gene, EHMT1, which in turn causes a protein called GLP not to be
produced. Moderate to severe Intellectual disability,
limited/absent speech are some of the symptoms. KS was first
"discovered" in 2010. Thanks to "whole genome sequencing", it is
now possible to diagnose KS with a single draw of blood. Maybe
that's why we are hearing about KS kids more often in our
community. We have a non-profit foundation based in the US (
https://www.idefine.org ) to improve awareness and lead/fund
potential research for KS. Also, there are already two active
pieces of research that provide potential improvement for these
kids. One explores drug repurposing (
https://www.nature.com/articles/s41467-019-12947-3 ), the other is
about supplementing the missing proteins (
https://www.sciencedaily.com/releases/2021/09/210921100245.h... ).
Both have very promising results but are not close to clinical
trials yet. Still, several potential treatment modalities need to
be explored in depth. Antisense Oligonucleotide Therapy (ASO), gene
therapy (CRISPR), drug repurposing are a few modalities to name.
Known KS individuals sum up to only a few hundred patients so far.
When the patient count is so low, pharmaceutical companies are not
interested in pursuing research for that disease, so patient
organizations are forced to fund their research themselves. This
has been done by several rare disease patient organizations before.
Batten Disease (Beyond Batten Disease Foundation funded $35M
research), Angelman Syndrome (Fast Foundation funded $26M
research), SMA (Cure SMA funded $35M research), all funded
successful research and managed to reach clinical trial level.
Sorry about the extra-long intro, but I wanted to provide context
for this relatively "new" genetic disease which is hardly known.
Since HN has members with extensive digital marketing experience,
I'm hoping you would share your ideas with us. Long story short, if
we can manage to raise several million to kickstart multiple types
of research in parallel, then we can offer these kids a chance. My
first idea is about co-hosting a series of Instagram live streams
with celebrities to ask for donations for research. I'm not sure if
this is already a solid fundraising technique? Also, I don't know
the first thing about finding celebrities as well. A second idea
is, recording a youtube video and promoting it using google ads
grants. At this point, we want to leave no stone unturned about
fundraising. So here we are. Any advice would be greatly
appreciated.
Author : halukakin
Score : 56 points
Date : 2022-02-07 20:23 UTC (2 hours ago)
| piqufoh wrote:
| Hello!
|
| > How to raise funds for rare disease research?
|
| I'm not a marketing expert (though I do work with rare diseases).
| But a good starting point might be to checkout
| https://www.rarebeacon.org/ There certainly is help and there are
| options, but as you understand finding and accessing them isn't
| always easy. Good luck on your journey
| halukakin wrote:
| This looks very helpful. First time I heard about this
| organization. Apparently, they train patient groups like us.
| They even host their own drug repurposing conference. I'll
| signup on the email list and study their site. Thank you.
| ricardolopes wrote:
| My son (now 4yo) was diagnosed with two rare diseases, due to a
| mutation in a collagen production gene, and at first it wasn't
| clear if he was ever going to walk at all. His life will always
| be severely impacted.
|
| I feel your pain about wanting more R&D around treatments and
| potential cures but finding almost nothing because it wouldn't be
| commercially viable.
|
| My main strategy was trying to contribute with what I know best:
| software development. The company I joined (lifebit.ai) is
| building tooling to reduce medical/pharma R&D costs, in an effort
| to make it economically viable to research diseases that weren't
| viable before. This is a mission I can get behind and that feels
| like the best use of my time.
|
| I hope you get to find the best way to have the biggest impact.
| Good luck!
| halukakin wrote:
| Thank you. Great job btw, lifebit could do some real good! Same
| idea for me too. If I can manage to make a clean exit on my
| existing business, medicine R&D will be where I will spend the
| rest of my time.
| yeswecatan wrote:
| I'm sorry for what you are going through and can't offer any
| advice pertaining to your main question. Have you heard of
| AllStripes (https://www.allstripes.com/) by any chance?
| halukakin wrote:
| Thank you. Yes, idefine collaborates with All Stripes.
| Hopefully soon we will pass 100 registered participants there.
| Very important work.
| https://www.allstripes.com/program/kleefstra
| ISL wrote:
| One thing that has helped to make progress against progressive
| supranuclear palsy, not as rare as KS, but still quite rare, is
| that it has properties that are ideal for testing potential
| Alzheimers drugs. PSP patients have anomalous quantities of one
| of the two proteins that turn up in Alzheimers patients, so they
| are ideal for separating the effects of new therapies.
|
| So, a win-win emerges: PSP patients get well-funded clinical
| trials of therapies designed to address their affliction and the
| entire world gets improved understanding of therapies that target
| Alzheimers.
|
| If there is something special about KS that allows it to be a
| diagnostic for therapies in adjoining disease with broader
| impact, then suddenly an avenue opens for intense investment in
| therapies for KS.
| markdown wrote:
| experiment.com?
| halukakin wrote:
| First time I heard about this site. Maybe we can use this for
| preliminary research requiring small funds. I'll study this.
| biols wrote:
| I work at a medium-sized pharmaceutical company as a
| computational biologist. Diseases like KS sometimes come up as
| potential repurposing targets (or novel drug targets), but we get
| a LOT of pushback from finance / leadership because we're
| unlikely to turn a profit working on ultra-rare indications.
|
| It is a deeply frustrating position to be in, wanting to work on
| these rare diseases and help this rarified patient population and
| not being able to, even though me and my colleagues are poised to
| do so. I often get maligned for being a scientist in pharma;
| laypeople often assert that I "don't want to treat cancer / rare
| genetic disease / etc; because then I'd be out of business." I
| can assure those reading that all of us DESPERATELY would like to
| work in these indications, and often times it's tragically
| finance that dictates whether we are able to or not. The system
| feels broken.
| netizen-936824 wrote:
| Sounds like you and your colleagues could start your own
| company, there may be enough people. I wonder if a nonprofit
| pharma company is viable.
| halukakin wrote:
| I hope one-day things would turn around. If the regulators
| asked pharmaceutical companies to study for these drugs on the
| side so much could change. Probably less than 1% of their R&D
| budget would be enough to move things.
| TamDenholm wrote:
| In the world of business acquisitions, to which I'm no expert but
| know a little, it's not worth it for private equity firms to even
| look at businesses operating in less than probably 25 million in
| revenue, because it takes (near enough) the same amount of effort
| to acquire a business doing 25 million as a business doing 500k
| revenue.
|
| However, for a player like me, it's worth me operating in the
| 500k to 25mil range and buying businesses there because, first
| off I'm not a millionaire, and second, I can assemble a group of
| related small businesses which collectively would make 25 million
| which I can then sell to private equity for a larger multiple
| than I bought them for.
|
| My point being, is there a way to create some kind of collective
| of rare disease causes and raise money as a group, collectively
| funding the labs and salaries of scientists to work on these
| diseases, ideally in an efficient way so that the work done on
| one disease can, at least partially, be effective for a number of
| the diseases in the group.
|
| I don't know the answer, I just wanted to offer an idea from my
| perspective.
| AlexAndScripts wrote:
| I have no experience in this whatsoever. However, perhaps trying
| to make a mini celebrity of a small number of the patients,
| trying to go on TV talking about how there could be a treatment
| if there was just enough funding could help? Seeking to maximise
| empathy to get donations, pretty much.
| tootie wrote:
| You should try asking on https://reddit.com/r/nonprofit or even
| reaching out to people at those foundations you mentioned for
| other diseases.
|
| I'd start with a simple campaign page with your mission, your
| goals, some cute pics and some way to collect donations and
| capture emails. There's loads of fundraising CRMs out there.
| Givelively is a decent one and costs nothing but there's plenty
| of options. Even just a Facebook page could work.
|
| As for how to acquire leads from scratch that's a tough one. Ads
| can work, you can also try reaching out to media orgs to try to
| get some airtime to talk about your campaign.
| halukakin wrote:
| Thank you. I wasn't aware of a nonprofit sub on reddit. So many
| members. Definitely will post there.
| human_person wrote:
| You might be eligible for some of the grants made by CZI for
| their rare as one program focused on supporting patient driven
| research for rare diseases.
|
| https://chanzuckerberg.com/science/programs-resources/rare-a...
| halukakin wrote:
| We couldn't get in last year's batch. I'm thinking we will re-
| apply this year. If any CZI team members read this at some
| point. Any pointers for the future would be a great help.
| gardenfelder wrote:
| CZI's meta home page says that meta - the research arm - is
| sunsetting in March. Not clear how that will affect their
| funding plans.
| Fomite wrote:
| It's a long way down the road, but if you're ever interested in
| funding university-based research (in the US):
|
| Universities have something called an indirect rate. This is
| basically how they pay for things that a private company would
| build into the overall cost of the project. Things like
| administrative folks, and lights, and heat, and IT support for
| the internet, etc., because "direct costs" are only what applies
| directly to your project.
|
| These are often 50%+, and tend to come as a shock to people, even
| when the total cost is well less than private research firms.
|
| The thing that doesn't get talked about as much is if you're a
| funder, if you have somewhere on your site, the funding
| announcement, etc. that you're capping indirects at say, 15%,
| that it's _much_ easier for researchers to convince university
| administration to use that lower rate (in that if you don 't have
| it, we're SOL).
|
| So if you do ever manage to head that direction, please do make
| sure to include something like that.
| halukakin wrote:
| That's a big tip for the down the road. Will try to understand
| this better. Thank you.
| [deleted]
| rsfern wrote:
| If you're not familiar with Matt Might, look into his work on the
| rare genetic disease that his son has. His story is really quite
| inspiring, and maybe there will be a strategy that you can apply.
|
| Here's a good post, he's a prolific blogger:
| https://matt.might.net/articles/rare-disease-internet-matchm...
|
| (Edit: this is a bigger picture post detailing the whole process:
| https://matt.might.net/articles/my-sons-killer/)
|
| Depending on the kind of research you are looking to get funding
| for, you might also look into an NIH SBIR grant or something.
| https://rarediseases.info.nih.gov/tips/pages/124/
| halukakin wrote:
| Thank you! Matt is a huge inspiration to all rare disease
| parents. I will read the NIH SBIR grant.
| gardenfelder wrote:
| In relation to that story is this
| https://www.science.org/content/article/researchers-turn-
| vol... where the Su Lab enlisted crowd sourcing in an open
| source online platform to assist in reading large amounts of
| literature on NGLY1. Something similar might be possible
| here. At the same time, there are emerging platforms in the
| social network space which focus on structured conversations,
| Quests; in that case game mechanics and the social dynamics
| of guilds in MMOs come into play; I cannot say whether that
| would necessarily be of immediate value, but there are
| arguments for exploring that space as well.
| morelandjs wrote:
| Maybe take a look at the orphan drug act for inspiration,
| https://www.fda.gov/patients/rare-diseases-fda
| skosuri wrote:
| I imagine how hard this must be on you and your family and kudos
| for having the energy and wherewithal to try to organize. I think
| there are a few people/places that might be worth reaching out to
| in order to learn more.
|
| 1. AllStripes (https://www.allstripes.com/) -> It might be worth
| reaching out to them to get put in contact with other foundations
| that might be working on the same thing.
|
| 2. Reaching out to RareBase (https://www.rarebase.org/) or Ethan
| Perlstein (https://mobile.twitter.com/eperlste) to talk about how
| they work with similar foundations.
|
| 3. There is a new company called (https://www.vibebio.com/) that
| are working on helping fundraise for patient communities using
| DAOs. I think Alok Tayi is one of the founders there.
|
| 4. I know there are some tech founders with family members of
| rare disease that have gone through similar experiences. For
| example, I think Rohan Seth at clubhouse has one
| (https://www.lydianaccelerator.org/). They might be good
| resources to reach out too.
| BurningFrog wrote:
| If I was a billionaire looking to Do Some Good, I would look into
| financing medical trials for non profitable diseases like this
| one.
|
| The other kind is about drugs that are out of patent, like
| Ketamine, which very likely can cure important diseases, but
| since no one stands to make back the money it costs to get them
| approved, they remain illegal.
|
| Of course, I'm not at all a billionaire, but some of them read
| HN.
| wanderer_ wrote:
| Unfortunately I cannot help, as I have no experience with this.
| It is a very sobering thing to consider, and one my family is not
| altogether unfamiliar with. I had an uncle that was diagnosed
| with a very rare form of cancer, and through a very unlikely but
| not unwelcome turn of events involving a kind nurse and flagging
| health insurance, his life was saved at the St. Jude Research
| hospital (in TN).
|
| About twenty years later, he was grown up and was soon to have a
| family, when the son of a family friend was diagnosed in infancy
| with the same form of cancer - except it was even more rare. This
| strain is almost always manifested below the neck (like my
| uncle), but in this child, it was in his brain. It has been a
| battle, but the boy was able to fight through it several times.
| Multiple resurgences later, he is finally recovered: thanks to
| the research done on my uncle.
|
| All that to say, research on rare diseases is extremely important
| and something I am very passionate about.
| kingsloi wrote:
| Hi, your efforts to better understand your daughter's diagnosis
| are remarkable, and appreciated. A rare disease lights a fire
| that's almost impossible to extinguish, you want every question
| answered, there often are none, but it doesn't stop you from
| searching anyway. Your situation resonates with me a lot, my
| little girl was diagnosed with another KS - Kabuki syndrome,
| specifically a mutation with the KMT2D gene (thanks to whole
| genome sequencing), which is rare, but common enough for their to
| be a few decent resources out there now, specifically ones like
| https://www.allthingskabuki.org and
| https://www.kabukisyndromefoundation.org which helped my wife and
| I better understand our daughter's diagnosis. Kabuki is multi-
| system affecting, often accompanied by heart disease, feeding,
| hearing, hypotonia, endocrine, limb/spinal/skeletal/organ
| abnormalities, and similar to Kleefstra, intellectual, speech,
| and mobility difficulties.
|
| Your ideas all sound fantastic, and definitely on the right path,
| maybe reaching out to foundations of other rare syndromes may
| offer some help, likewise with Facebook groups, and the like. I
| run my own non-profit https://sterlingstrong.foundation to bring
| awareness to KS, CHD, isomerism, etc, and to help families during
| long ICU stays, and further research by donating to other
| research centers such as https://www.kennedykrieger.org, maybe
| they, or orgs like Center for Rare and Neglected Diseases
| https://crnd.nd.edu/directors-page may help? I wish you and your
| family all the best!
| halukakin wrote:
| Thank you so much. First time I hear about Kennedy Krieger.
| Their "feeding disorder" service caught my eye. That's a big
| issue for us. They post some pretty good results.
| https://www.kennedykrieger.org/outcome-data-2019/feeding-dis...
| . I'll study the CRND site as well.
| coderintherye wrote:
| https://seed.nih.gov/
|
| NIH provides funding and accelerator programs for medical
| startups. If you have the expertise, apply yourself, or find a
| grant writer to help you fill it out all out. The programs
| themselves are great and they provide a ton of support. Funding
| is in steps (e.g. step 1 - $200k, step 2 - $1mil).
|
| I'd also recommend https://wefunder.com/ of who I know many of
| the folks running it and it is great, but the NIH route is more
| specific to your case.
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