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Ask HN: How to raise funds for rare disease research?
111–120 of 212 posts
Re: Ask HN: How to raise funds for rare disease research?
#112Earlier quoted context omitted.
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…
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’m not sure that history is going to be kind to Trump’s shortcutting[1] regulatory approval on the current vaccines.
[1] “Operation Warp Speed”
Re: Ask HN: How to raise funds for rare disease research?
#113I 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.
Out of curiosity, what business model were they trying? A few years back we were looking into virtual trial site companies (e.g. Science 37), and the pitch seemed to make sense at the time (focus on studies which require minimal site visits, use telemedicine for routine visits, coordinate with local labs and pharmacies as needed). That structure might be a good fit for rare patient cohorts.
Re: Ask HN: How to raise funds for rare disease research?
#114Quoted post unavailable.
For example, to fight inflammation, high dose vitamin C may be helpful (and very safe as well).
Potentially very safe if vitamin C is actually needed for some reason.
I'm not familiar with the term orthomolecular but eating right as a primary modality of treatment is one of the things I do for my genetic disorder.
Re: Ask HN: How to raise funds for rare disease research?
#115I 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.
Ethan Perlstein uses ethnic stereotypes to market his company. He opportunistically uses rare disease kids to shield against complaints. I would steer clear.
Re: Ask HN: How to raise funds for rare disease research?
#116Hmmm… what does the average KS patient cost to their insurance company?
What is the likelihood of research finding treatments that will reduce that cost, and by how much?
I wonder if a financial instrument could be crafted that captures a potion of future insurance company savings in return for funding research now.
Probably would require some concessional, first loss funding, or perhaps a philanthropic evergreen fund.
Re: Ask HN: How to raise funds for rare disease research?
#117Glp and G9a are ubiquitous as they are import epigenetic regulators (histone methyltransferases). Sounds difficult to work on, and hard to supplement if they play an important role in many cell types
Re: Ask HN: How to raise funds for rare disease research?
#118I 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.
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!
Re: Ask HN: How to raise funds for rare disease research?
#119My cofounder and I are working on a synthetic biology startup concept targeting rare diseases. We're both genetic and microbiology generalists with a desire to dedicate ourselves to improving the human condition. I find it serendipitous that we just had a two hour call today discussing our desire to work on creating a treatment for one or more rare diseases. The one area we lack expertise in is the regulatory and com…
The challenge is to prove you have something that works. Animal studies are helpful here. Because once you have something, you can partner with the big pharmaceutical co’s for the regulatory hurdles.
Re: Ask HN: How to raise funds for rare disease research?
#120I 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…
Thanks for your comment. I'm also a computational biologist by training, but have never worked in the private sector.
I'm familiar with the idea of re-purposing drugs for rare disease treatments (most of my adjacent work has been in very early-stage academic research), but I'm curious about the financials here. Could some of the financial risk here be minimized by aggregating multiple groups of patients, all suffering from different rare diseases? From what I know about the process, the answer is yes, but I'd be curious to hear from somebody closer to the process.