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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?

#91

Post by codekilla »

netizen-936824 wrote: post #79
codekilla wrote: post #75

Earlier quoted context omitted.

There are a lot of modalities being integrated, things like spatial/temporal, ADT/protein, etc. Integrating all of this data is a computational challenge, and of course there are lots of methods for analyzing it that vary in computational demands. It's not simulation, but still a lot of processing.

So you're taking all wet bench data and analyzing or integrating it rather than modeling? That's interesting! Are there any possibilities that you see from your experience in using modeling or other in silico methods to reduce time in the lab, find new leads in drug development, or otherwise enhance research capabilities?

Yes, essentially, though you may create models of interactions etc., but the main idea is to extract information from various aspects of the cell.

As far as in silico, I think absolutely there are probably opportunities here. Generative models might be useful for some type of counterfactual (automated) reasoning with respect to disease course/treatment. I think we're in the relatively early days of collecting high resolution cellular data, so I think in silico approaches like this will be more and more relevant.

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

#93

Post by giantg2 »

I saw an article on here about a billionaire in Florida who makes donations. Perhaps you can find out who signed The Giving Pledge, what their philanthropic organizations are, and contact them. This should require the least capital. If one does donate, it could be a sizable amount.

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

#94

Post by zmmmmm »

Not quite on topic, but I do believe a way needs to be found to make development of targeted therapies feasible for these ultra rare genetic disorders. The current model seems to be that they go through the standard clinical drug development pathway which is never going to be economically feasible for something applicable to such small cohorts of patients.

The best I can see is to invest very heavily in the common methods underlying how the therapies get created - gene editing, knockout / interference etc. The better these work, the less risk there is that a drug is going to fail at clinical trials. It means, a lot more basic research as well as applied research to translate the findings.

Then we need to work on smoothing the clinical trials pathway as much as possible so that if a method is operating from proven principles it doesn't have to jump every single hurdle as if it is completely novel. Obviously, there are huge risks here if it goes wrong. But on the other hand, we can look at the cancer space and see that patients get very ready access to experimental drugs there, and overall it is enormously beneficial to overall progress. Rare disease sits somewhere in the middle - people are not (usually) dying but their life quality is so heavily compromised that there ought to be some allowance for a more accelerated access to trial drugs than if we are talking about routine medical use.

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

#95

Post by fartcannon »

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.

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

#96

Post by giantg2 »

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…

It would be cool if pharma companies had charitable rotation program for researchers to volunteer to research these types of things. So the researchers could still get paid and the company could claim the costs as a write-off (not sure if they actually need one) with the results being public use.

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

#98

Post by nicwilson »

halukakin wrote: post #57
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...

Derisking. Thats the term.

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

#99

Post by DoreenMichele »

bunwich wrote: post #37

Past discussion about a rare disease that found a cure, but due to economics and possibly efficacy it isn't produced any more. https://news.ycombinator.com/item?id=18475919 glybera

A more recent discussion about that:

https://news.ycombinator.com/item?id=29287200

It also includes a link to this:

https://www.resetera.com/threads/guy-cures-himself-of-lactos...

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

#100

Post by csee »

biols wrote: post #81
WalterBright wrote: post #69

Earlier quoted context omitted.

On the other hand, competition has given us what, 6 covid vaccines of varying effectiveness, and in record time. What if only the least effective one was developed, and took 18 months? The 1962 FDA effectiveness mandates have had the side effect of increasing drug development costs enormously, and that shuts down development of treatments for rare disorders.

I think the idea would have been that Pfizer and Moderna could have pooled resources and made a single optimal vaccine faster, though I'll admit I'm not sure it could have happened any faster than it did from my perspective. The Kefauver Harris Amendment you refer to was immensely important towards the development of safe and efficacious drugs -- I do not see the connection between that act and rare disease therapeut…

Competition is an incredible driver of outcomes, e.g. moon landing. It's also better from a risk perspective since it decorrelates efforts and we only need 1 to succeed. It also allows evolution to operate, where the incompetent and broken and corrupt die off and the productive are given more resources, which tends to lead to overall improvement.

Sure, competition also creates waste, which is your main point here, but don't discount the upsides.

Competition has proven itself in the real world. Having only one monolithic vaccine maker (whether for-profit, non-profit, or government) would be a very bad thing. What would happen if it falls to corrupt leadership, as one of many examples of how this could go wrong? There is no mechanism to escape badness here, because we only have 1 of them.

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