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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?

#61

Post by biols »

halukakin wrote: post #44
t3po7re5 wrote: post #42

Earlier quoted context omitted.

I wish there was a way to crowd fund or crowd source a push for new therapeutics. I have a rare cancer at the moment and the overwhelming majority of drugs used for it were developed for other cancers. I wish there was a way we could establish an open source community or project around creating novel drug targets as a small moon shot funded by donations from the lives that it effects.

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 current pharma industry configuration; what financial incentive is there for big pharma companies to publish their results for another company to beat them to a new drug? I don't have a solution to this problem, but I hope someday we as a society can find one. This would absolutely revolutionize biopharmaceutical science.

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

#62

Post by halukakin »

danielmarkbruce wrote: post #49

You 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.

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

#63

Post by biols »

codekilla wrote: post #40
netizen-936824 wrote: post #7

Earlier quoted context omitted.

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…

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

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

#64

Post by archarios »

I don't have any expertise that can help here, but I want to chime in and say I feel you. My partner has a rare genetic disorder as well that has put them in the hospital many times in the past few years. We have been VERY lucky because a new drug that treats their condition fairly well was approved by the FDA just MONTHS before my partner started experiencing symptoms (https://en.wikipedia.org/wiki/Givosiran if you're interested). It's an extremely expensive medicine. Fortunately our insurance covers it! Best of luck to you and your family.

PS: I'm actually going to try apply to work for AllStripes soon.. Wish me luck!

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

#65

Post by halukakin »

archarios wrote: post #64

I don't have any expertise that can help here, but I want to chime in and say I feel you. My partner has a rare genetic disorder as well that has put them in the hospital many times in the past few years. We have been VERY lucky because a new drug that treats their condition fairly well was approved by the FDA just MONTHS before my partner started experiencing symptoms ( https://en.wikipedia.org/wiki/Givosiran if you…

Wow. So glad for you! These rare disease stories with a positive ending give us all the hope. Best of luck!

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

#67

Post by labster »

WalterBright wrote: post #48
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…

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…

You don’t know in advance how many lives will be saved by research. You may not know what you are researching at first; diphenhydramine is a moderately effective sedative, but turned out to be a strong antihistamine.

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

#68

Post by joshuamcginnis »

My 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 commercialization pipeline. If someone reads this and is interested in speaking with us about how we can work on this (or other) rare disease and could offer the mentorship we need to ensure our work can actually reach the hands that need it, please email me: josh@everymanbio.com

We'd consider a non-profit model if it makes sense.

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

#69

Post by WalterBright »

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…

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.

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

#70

Post by anyfactor »

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 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 financially suffer for the drug. He said the needed the money to pay for new research and better drugs and it was one of of lesser of evil thing he can do to R&D.

Ignoring the trickle down effect, if the government and Insurance companies in most cases ultimately pay for the price of medication wouldn't it be valid motivator to research rare diseases?

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