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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?

#121

Post by eby »

You might want to re-examine prior assumptions: there are in fact companies very interested in pursuing rare diseases. This article[1] discusses why: they can be highly lucrative opportunities. Despite there being very few patients, if any treatment at all exists then many national health care systems or insurance companies are forced to pay for it. That article is ten years old but a basic web search for "world's most expensive drugs" will turn up a number of similar articles. Here's a more recent one[2] and it seems the prices have gone up, as have the size of the companies.

As for how to find/approach such companies, that's a tougher question. It's a great boon for a company to be able to get Orphan Drug designation[3][4], for which KS may qualify. Hurdles are lower and it can help them bring that treatment to approval.

One thought would be to examine company pipelines for drugs targeting this gene/pathway, even if it's for a different condition. For example, here's a press release[5] on a company with preclinical results for Sickle Cell Disease on a EHMT1 inhibitor (probably not what you need but at least related) and here's another[6] on a CDMO contracting to produce GLP protein for a client (it doesn't say who but at least indicates interest).

Another thought would be to look for companies targeting related conditions because at least they have the expertise and may even have a candidate (failed or active!) that could provide some benefit. The idea of a repurposed drug search is a good one if you can find something still under patent since you'll need someone with deep pockets to reach approval.

Note that these might not be pharmaceutical companies but biotechs instead, the distinction being small-molecule (chemical) vs. large-molecule (biologic), respectively, as it's much more difficult for others to make a generic copy of the latter. To that end, you might add monoclonal antibodies to your list of potential modalities.

Sorry, I have no clue on the funding question. You mentioned several other foundations that had success; I'd suggest reaching out to them for ideas and strategy if you haven't already. I do know that some states have Life Science initiatives of various kinds which might offer grants/funding and may have associated incubators and whatnot where smaller players can "band together" to get shared access to equipment, lab space, expertise, etc. Unfortunately I don't have a link for that at hand but can try to dig up something if it's useful.

[1] https://www.forbes.com/2010/02/19/expensive-drugs-cost-busin... [2] https://healthcareglobal.com/top10/top-10-most-expensive-dru... [3] https://en.wikipedia.org/wiki/Orphan_Drug_Act_of_1983 [4] https://www.npr.org/sections/health-shots/2017/01/17/5095068... [5] https://www.globenewswire.com/news-release/2017/12/11/125070... [6] https://www.contractpharma.com/contents/view_breaking-news/2...

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

#122

Post by kraig911 »

This probably isn't any help but as a father to a severely disabled autistic girl it feels the entire medical system is broken. There's so much future squandered in the hopes of short term profit paid for at the cost to the next generation.

I know people say "Autism can't be cured" etc. But having met other families in this process and visiting group homes and the potential for a life of happiness for kids like ours my hope grows bleak and I honestly catch myself crying when alone. Especially seeing neurotypical children playing or how other parents don't see the gift of a natural life their families have. I too turned to all sorts of exotic ideas of diets, behavioral therapies, vitamins and it was out of desperation. I put my daughter through a stem cell study at Duke that seemed promising but I then saw danger of losing potentially my daughter to experimentation. I was told by neurologist and psychiatrist that my daughter would never talk, never interact with the world. Today she's talking. I credit ABA, stem cell infusions, and a diet focused on fat consumption (basically keto) but in that same vein I've also learned to accept our fate.

Now I've cut way back and I just enjoy the gift of being with her. Her gains and triumphs whilst they pale in comparison to a neurotypical kid are harder but just as monumental. I know now the best thing parents or families in our position is to create a world that would accept our kids, take care of them, and create for them a place where individually they could have a fulfilling life. Yes a cure would be great but there are so many kids with different conditions, illnesses, accidents and circumstances that will have denied them a future. Modern day society doesn't adequately cover what children like ours will need when we are gone.

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

#123

Post by robotresearcher »

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.

Have you not had a family member or close friend cured of disease that would have killed them a hundred years ago, thanks to modern pharma drug? I sure have.

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

#124

Post by HWR_14 »

WalterBright wrote: post #69
biols wrote: post #59

Earlier 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 beyond puzzled. The multiple COVID vaccines weren't developed so quickly because of competition. They were developed by an unprecedented worldwide effort and government support.

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

#125

Post by HWR_14 »

t3po7re5 wrote: post #42
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 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.

It's hard to imagine that working in a way that was free from scammers. People with rare conditions are already targeted by con men, internet fundraising is targeted by con men, I think it would implode.

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

#126

Post by efitz »

I found out a bit about this when a good friend of mine had two daughters that were born with a rare genetic disease (Morquio).

The US government, since 1983, provides grants and streamlines drug development processes for development of drugs for rare diseases. The government calls them “Orphan Drugs” and defines them as drugs that treat diseases that affect less thank 200k people annually, and are not expected to be profitable within 7 years.

I believe that my friend said that pharmaceutical companies are actually required or strongly incentivized to research some number per year but I can’t find a reference right now.

https://rarediseases.info.nih.gov/files/fda%20orphan%20drugs...

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

#127

Post by anyfactor »

not2b wrote: post #109
anyfactor wrote: post #107

Earlier quoted context omitted.

How does near-welfare state do innovation and research in pharmaceuticals? How do countries with universal medical care performs in terms of research and innovation and treating rare diseases? Shkreli's ideology was when it comes to innovation in Pharma and America's patent driven capitalist nature towards it is the reason why America leads the way in innovation.

Basic research is mainly government funded. Pharma companies build on that research to do the final phases of drug development and they fund the trials in exchange for a monopoly if the drug pays off. Shkreli wasn't interested in funding research. He looked for drugs he could buy and jack up the price of, so he was just a parasite.

This is very interesting. On a tangential note about Shkreli. I took a break and I remembered in the Facebook senate hearing a senator said to Zuck, "Who elected you to make decisions about what stays on the internet and what doesn't?" . This is tangentially related in the matters of Shkreli. Why it would be ethical for Shkreli to take this "moral burden" of doing the right thing and invest in innovation by essentially indirectly taking money from taxpayers? I am using the word ethical because the US government doesn't think price gouging illegal. He is nobody's champion and surely he is not elected. It is a very interesting idea.

I guess for decisions about pharma research like this having an elected body or representation of an elected body is a good thing.

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

#128

Post by csee »

Coding_Cat wrote: post #108
csee wrote: post #100

Earlier quoted context omitted.

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, bu…

It is possible to maintain the benefits of competition without the level of duplication and profit motives that we struggle with today. Large scale collaborative scientific endavours like CERN show us that it is possible to both publically share knowledge and still explore multiple avenues and competing designs. There's also no financial profit motive and while CERN receives a lot of public funding, it has to pump th…

I agree that it's possible to preserve many of the benefits of competition under a more centralized and explicitly cooperative structure. You see this sometimes within large for-profit companies that have competing products, which are siloed from each other in the workplace. This works fine, because there is still a profit motive at work and the identical evolutionary forces that will kill a particular silo if it's underperforming, and the same competitive pressure to perform.

Much larger cooperative structures are less proven to work and are more hypothetical, though, even if CERN is an example of such a structure working. The risk, mainly, is that there isn't a good corrective mechanism if the whole thing becomes corrupted or rotten from the top. The other risk is that the cooperation is actually detrimental to progress because it correlates outcomes via group think. Some decorrelation is nice. I am happy that Musk et al. weren't forced to become cogs at NASA, and could explore their own ideas, which was easier to achieve by then being explicitly separate entities (even if they were reliant on contracts).

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

#129

Post by rronalddas »

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!

Hi on a side note, I am curious about how did you get to know that your company was mentioned in this thread? Did the OP reach out to you first? Or did you find this comment while reading HN?

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

#130

Post by fartcannon »

robotresearcher wrote: post #123
fartcannon wrote: post #95

Earlier quoted context omitted.

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.

Have you not had a family member or close friend cured of disease that would have killed them a hundred years ago, thanks to modern pharma drug? I sure have.

No, not personally, I do have 2 on life long treatments. But I was being too hyperbolic and I don't doubt your story.

I'm just extrapolating incentives. Is there any incentive to cure (invoice once), when they can treat (life long invoices)? Certainly scorn is irrelevant as OPs comment suggests they're unwilling to work on rare diseases. And these companies are public. They have shareholders that expect them to constantly grow.

I don't believe it's a stretch to say, at the very least, the incentive is there.

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