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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?

#131

Post by [deleted] »

JoshTriplett wrote: post #106
WalterBright wrote: post #48

Earlier quoted context omitted.

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…

> If you could do research that will save 10 lives, vs research that will save 1 life, which would you choose? Both. We're not even close to our limits on research capacity. If we actually were at the limits of research capacity, and we were actually forced to make decisions between livesaving treatments to research where we couldn't do both without sacrificing something else that saves lives, then yes, of course, ch…

[deleted]

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

#132

Post by human_person »

gardenfelder wrote: post #27
halukakin wrote: post #21

Earlier quoted context omitted.

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.

CZI's meta home page says that meta - the research arm - is sunsetting in March. Not clear how that will affect their funding plans.

Totally separate projects.

Meta aimed to build a knowledge graph of published research papers.

Patient driven research/rare is one is a separate program focused on supporting patients with rare diseases connect with and support scientists studying those diseases and the larger rare diseases network.

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

#134

Post by blfr »

JoshTriplett wrote: post #106
WalterBright wrote: post #48

Earlier quoted context omitted.

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…

> If you could do research that will save 10 lives, vs research that will save 1 life, which would you choose? Both. We're not even close to our limits on research capacity. If we actually were at the limits of research capacity, and we were actually forced to make decisions between livesaving treatments to research where we couldn't do both without sacrificing something else that saves lives, then yes, of course, ch…

> Both. We're not even close to our limits on research capacity.

Really? From the outside it seems to me like we are beyond our limits on research capacity.

The progress seems to be slowing down everywhere while the price per discovery of a new drug skyrockets with many having rather disappointing efficacy (high NNTs).

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

#135

Post by halukakin »

fartcannon wrote: post #95
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…

Just the fact that you get push back on work like that suggests to me that these companies will never cure anything. They will treat absolutely disease, but cure nothing.

Sometimes I share this feeling, but then I think about all the loved ones who were cured of some disease that would have been deadly 50 years ago. I don't think it's the pharma companies that are responsible for this. They are for-profit entities. Our governments should create the necessary business environment which will encourage these companies to work more for the people.

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

#136

Post by halukakin »

aloktayi wrote: post #118
halukakin wrote: post #35

Earlier quoted context omitted.

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.

hey halukakin. so very sorry to hear about your kid. our family experienced something similar when a loved one fell ill and therapeutic options were unavailable. feel free to ping me at atayi@vibebio.com - happy to connect and see how we can help. our vision is to realize every cure for every community. rare diseases is where we are starting. and thanks Sri for the shout-out!

Will certainly do. Thank you Alok!

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

#137

Post by halukakin »

danielmarkbruce wrote: post #74
halukakin wrote: post #62

Earlier quoted context omitted.

Considering they managed to raise close to $100M between them, if we could even get 1-2% cross donations from their existing donors that could give us a big jump start. This is an area I think we will definitely look into.

No no, I'm saying something slightly different. I'm saying there is a skill set that some human(s) at each of these places have. It's a real thing. It's almost like you are asking how to build rockets. The answer is: some people know how. Learn from them (or what they write in books). Now their existing donors may also be helpful, but you seem to be jumping a step.

Got it. Totally understand what you mean here. Will definitely try this.

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

#138

Post by halukakin »

pabs3 wrote: post #110

In countries with single-payer health care, it seems reasonable to use some funding from taxpayers on diseases that aren't considered to be profitable by the existing for-profit medical research companies. i.e. the medical research market is not providing what is needed by the population, so supplement that market using taxpayer funding. In the USA though, that doesn't seem feasible, but perhaps you could reach out t…

That is actually very smart. Right now we are limited to Netherlands and US mostly. Let me read about Canada.

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

#140

Post by sterlind »

biols wrote: post #59
WalterBright wrote: post #48

Earlier quoted context omitted.

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 z…

I wonder if you could run a pharma company that buys IP for failed cancer treatments and runs small trials in rare diseases. you have the advantage of having passed Phase I/II usually, so you know it's decently safe, and oncology hits a lot of diverse targets.

there are some examples, for instance enzastaurin being repurposed for vascular EDS, after washing out as an angiogenesis inhibitor for cancer. I don't know if it makes sense mechanistically, I sure hope it does.

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