Selfishly I'd like to do the same for Otosclerosis, a hearing disease that manifests in early 20s and 30s.
Ask HN: How to raise funds for rare disease research?
171–180 of 212 posts
Re: Ask HN: How to raise funds for rare disease research?
#172Earlier quoted context omitted.
This is such a fucked up incentive structure. Literally having the capacity to save lives but not doing it because otherwise people will pull out their money because Roblox is has a better ROI.
> This is such a fucked up incentive structure [..] It may well be(!), so let's assume you're right, what better incentive structure should we put in place instead? Increase general taxation and use that to fund more pharamaceutical R&D?
Break up the pharma companies and let them only be at-cost (!) manufacturers, move all R&D off to universities, and cooperate with other countries in said R&D efforts.
Re: Ask HN: How to raise funds for rare disease research?
#173Earlier quoted context omitted.
> This is such a fucked up incentive structure [..] It may well be(!), so let's assume you're right, what better incentive structure should we put in place instead? Increase general taxation and use that to fund more pharamaceutical R&D?
Break up the pharma companies and let them only be at-cost (!) manufacturers, move all R&D off to universities, and cooperate with other countries in said R&D efforts.
> Break up the pharma companies
For many politicians that would be seen as a "courageous decision" (hat-tip: Sir Humphrey Appleby)
> and let them only be at-cost (!) manufacturers
Won't it be hard to find investors if you do that?
> move all R&D off to universities
I think the "D" in "R&D" might be the problem in this approach. Universities are great at many things, including research, but based on my experiences (science PhD two decades ago in a research group which worked on anti-infectives) the scientists there aren't necessarily very good - or even actually interested - in development as such. We partnered fairly closely with $bigPharma at the time, and they funded a fair chunk of our work.
Re: Ask HN: How to raise funds for rare disease research?
#174Earlier quoted context omitted.
Break up the pharma companies and let them only be at-cost (!) manufacturers, move all R&D off to universities, and cooperate with other countries in said R&D efforts.
> Break up the pharma companies For many politicians that would be seen as a "courageous decision" (hat-tip: Sir Humphrey Appleby) > and let them only be at-cost (!) manufacturers Won't it be hard to find investors if you do that? > move all R&D off to universities I think the "D" in "R&D" might be the problem in this approach. Universities are great at many things, including research, but based on my experiences (sc…
> Won't it be hard to find investors if you do that?
Better that than paying negative interest for German 20 year bonds, if you ask me.
> Universities are great at many things, including research, but based on my experiences (science PhD two decades ago in a research group which worked on anti-infectives) the scientists there aren't necessarily very good - or even actually interested - in development as such. We partnered fairly closely with $bigPharma at the time, and they funded a fair chunk of our work.
Agreed, universities have historically not been involved into the development part. But that is not a given dogma that can't be changed - the government could fund the establishment of development departments.
Alternatively, international governments could establish cooperative efforts to develop pharmaceutical compounds. Rare diseases and the decline of available reserve antibiotics are a global problem affecting every country on Earth just the same.
Re: Ask HN: How to raise funds for rare disease research?
#175Look into EmCell.com - there's a free documentary on the clinic here: https://stemcellsmovie.com/ - under the round "Watch Movie Free" button.
They've not received much attention, nor do Western doctors/researchers have much to any knowledge at all in regards to what they're doing - because for the last 30 years they've been using fetal stem cells to successfully treat a variety of conditions; and last 25+ years offering treatment clinically.
Conditions like Multiple Sclerosis, which have "no cure" in the Western world, EmCell states and their experience is (which the documentary done by independent film maker follows at least one person treated for beginning MS symptoms) that if you treat MS earlier enough, before there's too much disease progression destroying the body's healing abilities, that the fetal stem cells will cause the dis-ease to stop progressive and even to regress (to heal) potentially fully (to be cured); multiple dystrophy, ALS, etc. are other "uncurable" treatments they offer treatment for.
As in the documentary it will be explained, they used fetal tissue that is 7-12 weeks of age, which is before the tissue begins to differentiate into that specific human tissue - before the immune system starts to develop, and so they argue it's safe to inject in anyone - as we all started from those base tissues.
I'd suggest looking at their website (which they updated recently so it's not as easy to navigate IMHO) but then also to email them to ask them specific about your daughter's issue. My guess is they at minimum would state that you could try to see if it helps - but maybe ask if they have treated other rare genetic disorders with any improvement seen.
Lately they've been diving into more specific research, like trying to improve fertility - injecting the testicles - which initial research showed a 30% increase in motility/health in sperm, etc.
They list a lot of success stories - https://emcell.com/success-stories/ - where they include (at least in their previous website they did) long-form video testimonials following them.
They also inject placental tissue into a patient's body, as placental tissue apparently has powerful anti-inflammatory and immune regulation mechanisms - so it's possible that a large amount of benefit may come from re-balancing/re-regulating the immune system however the placental tissue works to do so.
Re: Ask HN: How to raise funds for rare disease research?
#176>few hundred patients so far.
Did you consider non-US companies? few_hundrend x $500/month...
Did you talk with universities/academia? They have laboratories and students who may be will be glad to help.
Re: Ask HN: How to raise funds for rare disease research?
#177Earlier quoted context omitted.
This is such a fucked up incentive structure. Literally having the capacity to save lives but not doing it because otherwise people will pull out their money because Roblox is has a better ROI.
> This is such a fucked up incentive structure [..] It may well be(!), so let's assume you're right, what better incentive structure should we put in place instead? Increase general taxation and use that to fund more pharamaceutical R&D?
> Increase general taxation and use that to fund more pharamaceutical R&D?
Take the US military budget and give it to life saving research instead of life taking.
Edit: Also implement a wealth tax instead of income tax. Discourage hoarding of wealth and concentration of power in the hands of top 1%.
Re: Ask HN: How to raise funds for rare disease research?
#178Earlier quoted context omitted.
Break up the pharma companies and let them only be at-cost (!) manufacturers, move all R&D off to universities, and cooperate with other countries in said R&D efforts.
> Break up the pharma companies For many politicians that would be seen as a "courageous decision" (hat-tip: Sir Humphrey Appleby) > and let them only be at-cost (!) manufacturers Won't it be hard to find investors if you do that? > move all R&D off to universities I think the "D" in "R&D" might be the problem in this approach. Universities are great at many things, including research, but based on my experiences (sc…
Can you illustrate for a lay person the difference between research and development in pharma context?
Re: Ask HN: How to raise funds for rare disease research?
#179Earlier quoted context omitted.
> This is such a fucked up incentive structure [..] It may well be(!), so let's assume you're right, what better incentive structure should we put in place instead? Increase general taxation and use that to fund more pharamaceutical R&D?
> Increase general taxation and use that to fund more pharamaceutical R&D? Take the US military budget and give it to life saving research instead of life taking. Edit: Also implement a wealth tax instead of income tax. Discourage hoarding of wealth and concentration of power in the hands of top 1%.
Ah yes so China or Russia can invade and establish a totalitarian regime. Excellent idea.
Re: Ask HN: How to raise funds for rare disease research?
#180Earlier quoted context omitted.
> Break up the pharma companies For many politicians that would be seen as a "courageous decision" (hat-tip: Sir Humphrey Appleby) > and let them only be at-cost (!) manufacturers Won't it be hard to find investors if you do that? > move all R&D off to universities I think the "D" in "R&D" might be the problem in this approach. Universities are great at many things, including research, but based on my experiences (sc…
Can you illustrate for a lay person the difference between research and development in pharma context?
Research = determining a candidate compound, e.g. a new antibiotic, (e.g. by taking and tweaking an existing compound, by basing off of the actual viral/bacterial genetic code such as with the Biontech/Moderna CoV vaccines or by isolating a compound found in a plant, fungus or other natural agent which has been determined to have the effect you want), and initial checking against cell cultures and/or lab animals for effectivity.
Development = taking a lot of candidate compounds and evaluating them in the classic three-stage procedure - phase 1: determine how the compound is processed in actual human bodies to check if it is actually safe to ingest and what side effects can already be observed (n ~ 20-80), phase 2: determine effective dosage (n ~ 100-500), phase 3: check effectivity in a double-blind trial (n ~ 1k-10k), as well as the fourth phase (after-license monitoring) [1][2].
The more a compound progresses, the more expensive the trials get to conduct, not just because the participants usually get some compensation for their risk, but also because all the data has to be tracked and processed. And a lot of candidate compounds fail somewhere along the path (either because they are ineffective or because the side effects are too severe), which makes the money invested effectively wasted (from a capitalist point of view, not from a scientific!).
[1]: https://en.wikipedia.org/wiki/Clinical_trial
[2]: https://studienteilnehmergesucht.de/ratgeber/die-vier-phasen...