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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?

#31

Post by halukakin »

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…

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.

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

#32

Post by ISL »

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.

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

#33

Post by halukakin »

Fomite wrote: post #28

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 d…

That's a big tip for the down the road. Will try to understand this better. Thank you.

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

#34

Post by halukakin »

coderintherye wrote: post #24

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, bu…

These are great leads. I will study them. Thank you.

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

#35

Post by halukakin »

skosuri wrote: post #22

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 RareBa…

Thank you! Thanks to Ethan and Julia's weekly meetings on clubhouse, I've heard about some of these. Alok's DAO-based funding system seems very promising. I hope that gets traction. I thought I knew more about RareBase, but now I think I'll need to study their site in more depth.

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

#36

Post by halukakin »

wanderer_ wrote: post #23

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…

Glad to hear a good story. Sobering is a very good way to put it. For some of the parents, it just brings something else out of them. You lose the option to be a couch potato, forever.

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

#38

Post by halukakin »

BurningFrog wrote: post #30

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.

I wouldn't know the first thing about medical drugs. But I noticed, interestingly, Ketamine is a mentioned drug in the first research paper I cited above.

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

#39

Post by ImaCake »

halukakin wrote: post #8
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…

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.

Absolutely. A lot of funding gets poured into dead end alzheimers research or similar that will never work. A fraction of that redirected to rare diseases that get zero funding will do wonders. It is a shame that academia and research are so burdened with graft and politics.

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

#40

Post by codekilla »

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 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 of random plants, then tested them for anti-cancer properties very systematically. So in the U.S., at one point, we had a publicly funded drug discovery program targeted at a specific disease, and this is what jump started Pharma research in anti-cancer drugs. I would say we need to restart a program like this, and of course we should also focus on rare diseases--we stand to learn a tremendous amount, and it's difficult to convince industry to do it.

Personally, I'm a computational/mathematical biologist and I work on single cell data targeting multiple myeloma, I'd really like to see serious non-profit Pharma. Drug repurposing seems like the most feasible avenue. What I know of right now is open Pharma [2].

[1] https://dtp.cancer.gov/timeline/flash/milestones/M3_CCNSC.ht... [2] https://www.ospfound.org

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