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Ask HN: How to raise funds for rare disease research?

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Re: Ask HN: How to raise funds for rare disease research?

#51

Post by gardenfelder »

halukakin wrote: post #44
t3po7re5 wrote: post #42

Earlier quoted context omitted.

I wish there was a way to crowd fund or crowd source a push for new therapeutics. I have a rare cancer at the moment and the overwhelming majority of drugs used for it were developed for other cancers. I wish there was a way we could establish an open source community or project around creating novel drug targets as a small moon shot funded by donations from the lives that it effects.

From a software developer perspective: A github like service where every incremental research step is recorded&visible. A build management system like travis where each experiment is built and held accountable to unit tests. Something like github actions where you can trigger an automated lab trial instantly. Somehow opensource SW development communities have so much they can teach to medical researchers in terms of…

Sage Bionetworks Synapse https://www.synapse.org/ was conceived as a github-based research collaboration ecosystem.

Re: Ask HN: How to raise funds for rare disease research?

#52

Post by halukakin »

WalterBright wrote: post #48
biols wrote: post #5

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 pop…

If you could do research that will save 10 lives, vs research that will save 1 life, which would you choose? I understand that there are no easy choices here, and having to make a choice will always be heartbreaking. May I suggest contacting MacKenzie Scott (Jeff Bezos' ex) who seems to be looking for worthwhile endeavors to finance. (I have no connection to Ms Scott, I just read articles about her charitable activit…

I first heard about her efforts when she donated $40M to UCF. https://www.ucf.edu/news/scott-40-million-transformational-i...

I hope we can get in touch somehow. Thank you.

Re: Ask HN: How to raise funds for rare disease research?

#53

Post by teva »

nicwilson wrote: post #46

I can't remember where I read it, though I'm pretty sure that it was someone in your situation *: By far the easiest non-technical solution is to make it as easy as possible (logistically) for medical research companies to find a suitable cohort to test the intervention on. As you note with a patient pool of the order of hundreds, they are likely to be very scattered and so if you can get together a cohort for a medi…

Could someone link this blog post here?

Re: Ask HN: How to raise funds for rare disease research?

#54

Post by atdrummond »

I'd highly recommend reaching out to Matt Might and his team at UAB. (https://www.uab.edu/medicine/magazine/178-codebreaker-a-deep...)

This is what they specialize in. Feel free to e-mail me if you need help getting the conversation started there, I know a few of their MDs and researchers.

EDIT: Apologies, I see this has already been recommended and Matt has himself replied. Best of luck!

Re: Ask HN: How to raise funds for rare disease research?

#55

Post by MattGaiser »

nicwilson wrote: post #46

I can't remember where I read it, though I'm pretty sure that it was someone in your situation *: By far the easiest non-technical solution is to make it as easy as possible (logistically) for medical research companies to find a suitable cohort to test the intervention on. As you note with a patient pool of the order of hundreds, they are likely to be very scattered and so if you can get together a cohort for a medi…

> ease of cohort accessibility was their number one takeaway

A good friend of mine founded a startup to try and solve this and I was about to join her full time when the pandemic unfortunately crushed it, especially as they couldn't find a real business model.

It would be quite interesting as a non-profit entity though.

Re: Ask HN: How to raise funds for rare disease research?

#56

Post by ampdepolymerase »

Human trials for drugs is the expensive part. If the situation is urgent, look into compassionate use and right to try. If you have time, the fastest way to get it to production is to have the trials done in a lower cost country. At the end of the day if you control the experiment and manufacturing pipeline, then you can make a develop a drug for a much lower price. You absolutely cannot cut corners in the experimental and engineering stage even if you do it in a more compliant regulatory regime.

Re: Ask HN: How to raise funds for rare disease research?

#57

Post by halukakin »

nicwilson wrote: post #46

I can't remember where I read it, though I'm pretty sure that it was someone in your situation *: By far the easiest non-technical solution is to make it as easy as possible (logistically) for medical research companies to find a suitable cohort to test the intervention on. As you note with a patient pool of the order of hundreds, they are likely to be very scattered and so if you can get together a cohort for a medi…

I believe they call it "derisking" for the companies. It's a topic mentioned a lot on Ethan's genefixers clubhouse meetings. As you summarized, it's a very important topic. For anyone interested, the episode with Dr Allyson Berent is a masterclass about this. https://www.clubhouse.com/room/Md8njknG?utm_medium=ch_room_x...

Re: Ask HN: How to raise funds for rare disease research?

#58

Post by totoglazer »

morelandjs wrote: post #11

Maybe take a look at the orphan drug act for inspiration, https://www.fda.gov/patients/rare-diseases-fda

Definitely read about the orphan drug act, and look at the NORD https://en.wikipedia.org/wiki/National_Organization_for_Rare...

Re: Ask HN: How to raise funds for rare disease research?

#59

Post by biols »

WalterBright wrote: post #48
biols wrote: post #5

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 pop…

If you could do research that will save 10 lives, vs research that will save 1 life, which would you choose? I understand that there are no easy choices here, and having to make a choice will always be heartbreaking. May I suggest contacting MacKenzie Scott (Jeff Bezos' ex) who seems to be looking for worthwhile endeavors to finance. (I have no connection to Ms Scott, I just read articles about her charitable activit…

Major pharma companies are all constantly competing, and very often are duplicating work because they are not sharing major experimental results. The way it feels is that there's already "too many people" working in certain areas (e.g. in cancer), while almost no attention is paid to these rare diseases.

I think that more people studying rare diseases would result in a net gain of lives saved; I don't think it's as zero-sum as "either 1 person's life is saved or 10 are" in this instance.

Research also cross pollinates across disease areas. For example, understanding altered metabolism in cancer can yield insights for non-oncological metabolic disorders. Oftentimes, though, nobody's working on translating that work out of a cancer model, because the financial incentives are not there.

Re: Ask HN: How to raise funds for rare disease research?

#60

Post by jrowley »

nicwilson wrote: post #46

I can't remember where I read it, though I'm pretty sure that it was someone in your situation *: By far the easiest non-technical solution is to make it as easy as possible (logistically) for medical research companies to find a suitable cohort to test the intervention on. As you note with a patient pool of the order of hundreds, they are likely to be very scattered and so if you can get together a cohort for a medi…

Trial recruitment is definitely one the hardest parts of running a successful trial. With rare diseases reaching out with existing patient groups is one of the most successful recruitment mechanisms.
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