Project Marilyn Part I: Non-Patented Cancer Pharmaceutical
crowdtilt.com
crowdtilt.com
http://www.forbes.com/sites/matthewherper/2012/02/10/the-tru...
Not sure what the idea is here. Going by cold statistics, this will cost orders of magnitude more than they are seeking from crowd funding. And even then, with overwhelming likelihood, it will fail in clinical trials.
The actual cost of getting a chemotherapeutic developed is about 1-2 million in preclinical, and 12-20 million through clinical. Counting lots of inefficiencies, and, as I discuss downthread, we're stacking the deck in our favor.
Indeed, though your broader point is correct, clinical is more expensive and I think ultimately that should be done by a for-profit entity. But crowdfunding can nudge things in that direction by completing the preclinical.
"The actual cost of getting a chemotherapeutic developed is about 1-2 million in preclinical, and 12-20 million through clinical. Counting lots of inefficiencies, and, as I discuss downthread, we're stacking the deck in our favor."
That's only part of the equation. What's the probability that it fails preclinical or clinical trials? What are the historical success rates for chemotherapeutics? (Serious question)
edit: I've Googled up what I think is a relevant statistic? According to this source, of cancer drug candidates which completed US clinical trials between 2004-2011, 6.7% of the ones which entered phase I ended up as approved drugs (the rest failing out). Apparently this is much worse than non-cancer drugs, which have a 12.1% rate.
http://www.biotech-now.org/business-and-investments/2012/02/...
Are we really just donating money and time to people who end up over-charging us for pharmaceuticals and procedures? I'm not saying we are but I scratch my head when I hear about the astronomical cost of cancer treatment.
Clearly, software development was expensive and difficult.
In 1992 could an open-source OS on a shoestring budget contribute something of real value?
*adjusted for inflation: $150 million in 1992 = $250 in 2013.
Q: Which cancers will 9DS work on?
A: We don't have a definitive answer, but initial tests show that it's strongest against some forms of renal (kidney) cancer, melanoma(skin) cancer, and triple-negative breast cancer. We have reason to believe that variants of 9DS will target different cancer types, as well.
[1]: http://www.indysci.org/details.html "Details"
That's a great question. And I don't have a good answer for you. Any suggestions would be welcome.
Biology is complicated, which makes layman explanations a challenge.
Perhaps the best you can do is lay out in clear terms what you know and what you don't. This compound has been shown to be XXX±XXX% effective against these three cancers. It has also been shown to kill 90% of test animals. These three respected theory papers [1,2,3] have highlighted the fact that, if only the animals hadn't died, this compound would be more promising than YYY,ZZZ, and QQQ. Furthermore, in these references [4-10], making the modification we propose to make (9DS) in other related substances has been shown to reduce lethality by 100x. Our goal is to explore this alteration to see if we can address the lethality of the compound while preserving its anti-cancer properties.
Give people the references, and some laymen will dig all day long. Motivated people are really smart.
Not trying to be a hard-ass, just stating what I'd look for in either a paper up for funding review or for making my own crowdfunded investment.
You guys are doing something awesome.
https://drive.google.com/file/d/0By7AdX1eMEY2ZXZ5RzZ0Vl9QZG8...
I apologize, I had accidentally put in a wrong link there previously, it's been fixed, but the file has been publically available and visible for a while. Some non-scientific aspects (like our launch date) have obviously been changed at the last minute, but the proposal stands.
This is research funding. He's trying to raise money to see if it's something or not. If he can raise the money this way, then he can release it without patent encumbrance, and it's for everyone - including the world's poor.
Most drugs fail. This one probably will too.
I gave because I want to see intellectual property not be the only viable funding path anymore.
1) it's a "natural product derivative". These compounds do way way better than most other drug classes.
2) it has single-digit nanomolar activity in the dish. This is on-par and competitive with currently used chemotherapeutics, a prerequisite for any sort of chemotherapy.
3) there is another compound in the same family called SJG-136 which has passed phase II.
4) phase I trials for SJG-136 apparently showed extremely mild side effects. I still need to double-check clinicaltrials.gov on this, this was a personal communication from the developer of SJG-136. Because SJG-136 and 9DS broadly share the same mechanism it's very likely that 9DS will also have mild side effects.
To answer ThomPete's question, I'm not sure how I can verify this besides "trust me" to the layperson (especially #4). However, there is literature on #1 and #2, although, again I'm not sure how useful this is to the lay person.
#1: http://onlinelibrary.wiley.com/doi/10.1111/j.1751-7915.2010....
I have been lucky enough to be accepted at Sloan Memorial en New York given I have more than a thousand moles.
I wonder if I could ask my dermatologist there.
For example, the trend now is toward maintenance drugs and biologicals, because that's what the execs want. Whether or not this has anything to do with efficacy is questionable. During my biophysics days, I saw so many drugs enter the clinical pipeline even purporting to treat alzheimers, that were basically following up on "popular memes in the field" that from first principles I would look at and say, there's no way this will work.
I've spoken to one pharma consultant (to get the chat, I didn't tell him I was making an unpatented drug) who basically lamented what had happened to the cancer field as the big companies lost their R&D and turned to acquiring biotechs for their blue sky stuff - and he characterized biotechs as being on to "fad-dey" stuff. Now, it's alos possible that he's wrong too, and that I'm on the wrong track. So you are correct to worry about the likelihood of success.
I think the concept is incredibly radical. If this were a business idea, it would be functionally illegal: you could only solicit funding from wealthy, accredited investors under highly regulated procedures. Because people are easily mislead. And that's for stupid things like selling sporks. But here we are, pretending to scrutinize a million-dollar medical research grant, as if we were PhDs at the NIH office or something. Where do you want to go with this?
However, you are right, there could be unforseen toxicity issues.
9DS is probably synthetically tractable, but attachment of the sugar typically uses chemistry that destroys the imine on the other side, and vice versa. So that kicks any industrial processes in the knees.
Curious about it, because I work on this stuff from a structural perspective as my profession.