Let's discuss this idea on Cure Odyssey tomorrow!
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?
#72Let's discuss this idea on Cure Odyssey tomorrow with Casey and other Gene Fixer regulars!
Re: Ask HN: How to raise funds for rare disease research?
#73I 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 want to preface this by saying I am not providing an opinion rather I am genuinely curious. When Martin Shkreli bought the rights to Daraprim, some of his rhetoric about pharmaceutical industry sounded fair. He said that he is willing to send the drug for free to anyone who wrote to the company and he was essentially making the insurance companies pay the absurd price of the drug. He claimed no patient would ever f…
not providing an opinion on your suggestion but there is no such a thing as "government and Insurance companies paying" though, It's taxpayers.
Re: Ask HN: How to raise funds for rare disease research?
#74You 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…
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.
Re: Ask HN: How to raise funds for rare disease research?
#75Earlier quoted context omitted.
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.
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.
Re: Ask HN: How to raise funds for rare disease research?
#76It'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.
Happy to expand on it if you wish.
Re: Ask HN: How to raise funds for rare disease research?
#77Earlier quoted context omitted.
I want to preface this by saying I am not providing an opinion rather I am genuinely curious. When Martin Shkreli bought the rights to Daraprim, some of his rhetoric about pharmaceutical industry sounded fair. He said that he is willing to send the drug for free to anyone who wrote to the company and he was essentially making the insurance companies pay the absurd price of the drug. He claimed no patient would ever f…
not providing an opinion on your suggestion but there is no such a thing as "government and Insurance companies paying" though, It's taxpayers.
Always has been. Through insurance,
cost-pass-thru, taxes, direct medication purchase, etc. So, now that it's established that it's always the individual paying into...which is the most efficient way of allocating funds out of the system? Profit? Lives saved? Less acne?
Re: Ask HN: How to raise funds for rare disease research?
#78Earlier quoted context omitted.
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…
The Broad Institute hosts a very interesting transcriptomic dataset called CMap [1] that was intended to facilitate rapid drug repurposing. Having studied this dataset and worked with the data generators and software teams, I can say that drug repurposing is NOT as straightforward as people think. However, I agree that as a strategy drug repurposing is a useful tool in the arsenal generally. [1] https://clue.io
Interesting....I'll have a close look at this, thank you. I didn't mean to imply drug repurposing was straightforward, certainly as you say this is very challenging. I guess my thinking was that there might be relatively lower hanging fruit here (if Pharma companies have very little incentive to exhaustively search for repurposing targets for off-patent meds, maybe only a non-profit would be willing to do this) than say de-novo development.
Re: Ask HN: How to raise funds for rare disease research?
#79Earlier quoted context omitted.
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.
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?
Re: Ask HN: How to raise funds for rare disease research?
#80Earlier quoted context omitted.
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…
This is what I'd like to see. There should be some kind of system where in-progress research being done by pharma companies can be published. This would reduce the massively redundant amount of studies (e.g. CRISPR screens, xenograft studies, etc.) and help scientists more quickly converge on the mechanistic underpinnings of disease and how best to address them therapeutically. Obviously this can't work in the curren…
I've thought about things like the patent/ip problem, the structure of biomedical research, Pharma research, etc. This is an area where I don't actually see competition as a net benefit, however....it's the reality. The only thing I can come up with is a version of 'data rental'. Rather than Pharma companies locking this data away from others indefinitely, is there a way they could profit from it somehow, while still retaining ownership and not divulging trade secrets? Maybe not.
I've thought that a type of cryptographic data commons based on multi-party communication [1] could possibly be deployed with some effect. Basically you need algorithms that can compute on encrypted data, and a way to securely communicate encrypted data. There might not be huge incentive to use something like this, but maybe a version of this idea could work.
[1] https://en.wikipedia.org/wiki/Secure_multi-party_computation