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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?

#41

Post by netizen-936824 »

codekilla wrote: post #40
netizen-936824 wrote: post #7

Earlier quoted context omitted.

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.

I have been reading a book recently: The Story of Taxol: Nature and Politics in the Pursuit of an Anti-Cancer Drug , and one of the most fascinating parts was the way they discovered this molecule. Long story short, Taxol is a molecule they isolated from the bark of the Pacific Yew. The interesting part for me was learning about the Cancer Chemotherapy National Service Center [1]. They went around collecting samples…

Out of curiosity, what does your work entail with single cell stuff from a computational perspective? I've been doing some research into molecular docking as a drug discovery method, but its all single protein.

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

#42

Post by t3po7re5 »

netizen-936824 wrote: post #7
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…

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.

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.

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

#43

Post by mattmight »

halukakin wrote: post #3
rsfern wrote: post #2

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/…

Thank you! Matt is a huge inspiration to all rare disease parents. I will read the NIH SBIR grant.

Feel free to reach out. Happy to help.

Also, I've put much of what I've learned online:

https://bertrand.might.net/articles/algorithm-for-precision-...

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

#44

Post by halukakin »

t3po7re5 wrote: post #42
netizen-936824 wrote: post #7

Earlier quoted context omitted.

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.

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 how to scale development.

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

#46

Post by nicwilson »

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 medical trial, it makes it much easier for Pharmaceutical companies to test repurposed drugs against that cohort.

* I think it was linked to here on HN. Apologies for the shoddy search terms but the only things I can remember about their story was: the parents both dropped out of software jobs to get biochem (or biomed?) PhDs to research the disease (don't remember what) and the ease of cohort accessibility was their number one takeaway for their retrospective blog post(?). If you can find that blog post its well worth a read for all the non-technical (i.e. logistical/financial etc.) aspects of rare disease research.

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

#47

Post by iamcreasy »

ricardolopes wrote: post #25

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…

That's nice! Do you happen to know any US based company that are working on similar objective?

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

#48

Post by WalterBright »

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 activities in the newspaper.)

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

#49

Post by danielmarkbruce »

You name three organizations who did the exact thing you are looking to do. Have you reached out to the relevant person (it's almost certain that 1 or 2 people at each of these orgs knew how to raise money) at each org to understand in detail how they did it?

There are tricks to this kind of stuff, and some people know the tricks. I guess the first step is knowing that it's a real skill, that people know how to do it well and you need to learn from them. I suspect they'd be willing to share them given the circumstances.

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

#50

Post by halukakin »

mattmight wrote: post #43
halukakin wrote: post #3

Earlier quoted context omitted.

Thank you! Matt is a huge inspiration to all rare disease parents. I will read the NIH SBIR grant.

Feel free to reach out. Happy to help. Also, I've put much of what I've learned online: https://bertrand.might.net/articles/algorithm-for-precision-...

Thank you Matt! Just so you know, "quickstart molecular biology" is my daily reading thanks to you.
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