Feasibility study for new drug

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jethro

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let's say there are 5 [email protected] statistic data say less ([email protected]), but there are many poor, old, sick from other diseases etc.
it means that there are on earth max 500000 pals (according to official data 100-200000). 90% cannot afford themselves drug which is permanent and cost (lets say) 1000 usd/month (radicava), espetially people from asia, india, china etc... clinical cost for a new drug in phase 2 are app 20mil usd, and phase 3 is 30-50mil usd (prices are for ALS). cheapest are phases 1 and 4. how many people have 2500 usd/55days for radicava? rilutek is useless and costs in europe app 200 usd/month, and many eastern europeans have no even such a money. is it daemon who can heal tennis players, rugby, basketball and other players? what to expect from nurown, which is, as far as i know, only drug in phase III? i understand why gm604 wont enter phase III, pay i.e. 25mil usd being refused by fda. it needs time, pharmaceutical companies, certain people and some things to bring up drug which maybe already exists.
 

KimT

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Hi Jethro,

I suspect the number of people with ALS is higher than we think. Many people who are poor, even in the US, do not have access to a doctor who can diagnose and treat ALS. They just die without ever getting diagnosed. Add to that ALL the poor nations that have no access to anything but clinics run by nurses and the clinics might be miles away. They just die without any kind of help.

Our Country is very political when it comes to approving meds. If money were taken out of the equation, I believe there would be more progress. There are very few studies done on supplements and medical cannabis even though some PALS report much relief and even slowing of progression.

The FDA does not have to approve natural substances but drug companies pour a lot of money into blocking medical cannabis from Federal legalization and they would love it if the FDA made supplements go through a long approval process. I'm not saying the process is unnecessary or that drug companies are not risking their shareholders' money by this long process but time is of the essence for terminally ill people.

Our politicians get huge contributions from drug companies so the process from birth to approval remains long. They have made some gains but it still is way too slow for terminally ill people.

ALS is a world problem just like AIDS. I really don't know what it's going to take to ramp up the war to find a cure.
 

Gorby

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I don't understand why drug trials are so expensive. Look at Radicava. There were 140 people in the 6 month trial, half on placebo. So, if the figure of 50 million USD is correct, then that is $350K per patient. That seems like a lot. I wonder if the 50 mil figure comes from more typical drug development and so accounts for failures? Say you are working on drugs for cancer. Then maybe it costs 5 mil per trial but takes 10 trials to come up with a winner?

I like the idea of re-purposing old drugs to save money. Radicava was developed to treat stroke, so they already had a fair idea of its' safety and manufacturing.
 

whereswaldo

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The dollars that you are talking about, is not just for the trial. you need to spend time developing the drug before it can think about a trial.
So a repurpose drug will save money developing new and helping the expense of the original.
 

KimT

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Most of the dollars go into recouping R&D costs. I'm ok with that but what bothers me is a dying person should have the right to try drugs as long as they hold harmless the drug companies.
 

jethro

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amounts are for ALS trials. of course, trials like ALCAR, creatine monohydrate, L-serin etc actually are not trials, but experiments. no doubt that ALS is oxidative stress consequence. it narrows a problem. i told to docs in clinic where i am diagnosed that it is inner problem, inside us, 99% it is oxidative stress, so we have to prepair/change our organism to defend itself. drug is just like you trying to kill fly with rifle.
they laughed at me saying: stick with your profession, neurology is very complex science. they know how to interpret emng, they pass all exams on university, they know pharmacology, but actually, what do they know about als? never heard of badlack, alsuntangled, pubmed, patientlikeus, stem cells... all they know is to write rx for riluzole. no enthusiasm.
great, i'm on my own.
i still dont believe in healing drugs, but i do believe in organism restore. hence, stem cells can be the only cure, BUT not the way like most clinics do, even mayo. they should be processed and prepaired. this is theory. still dont know what to do with sod1 mutated which is actually sod1 +o2 (that's why we need antioxidants), which affects mitochondria. therefore, CarboH. act like fittness to mitochondria, and generally phisical activity. destroyed neurons are irrepairable, but there are 2 great things: plateau and supercompensation. both things are inside us, but stem cells, processed and prepaired for injection in medula spinalis under certain pressure and volume can bring healing prerequisite. i believe in nurown, but first i need to survive its approval and maybe price. many drugs stopped and didnt want to go to phase III trials. it seems that it is another story.many people trying to participate in trials, they got placebo, dye, and they are just number without name and surname. if drug emerges, those people wont be able to apply new drug which will be very expensive. their only hope is to get real cure, not placebo (50%), and if it works (0,x%). how much worths ALShuman life, and who can get benefit of it?
 
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