Laurie,
I hope I'm not being called one of the 'questionable participants' in this forum
All,
I think this was one of the first forums I contacted when we first knew my wife showed first symptoms and boy I've learnt a lot in the last two and a half years about the disease and the pharma / medical industry.
There are quite a few points / questions raised on here about RCH4 and I wanted to answer some of them from what I know about it.
Also I want to point out that I have noting to do with the suppliers of RCH4. My wife is just a user. It may sound like I am but that is because I also get quite defensive about the suppliers of RCH4 as I've got to know them over the last 2.5 years and have found them to be very compassionate and very helpful and I believe my wife is only here because of the drug RCH4.
RCH4 has got orphan drug status and I know exactly which one it is. It is FDA listed under a different name and that is why it cannot be found.
The drug has registered worldwide IP protection but I believe that the suppliers don't really want its identity and composition out there until enough money can be secured to go through phase 2 trials. There is not enough info on the IP / orphan drug details to copy the drug but there is enough to try counterfeiting it. We have already seen the company ALS Worldwide offered a patient RCH4 for sale which just goes to prove this fact.
There is no real secrecy about the drug apart from the ingredients and how to make it which I would not expect a inventor to release anyway. If a serious investor was found, there would be no 'secret' as you call them. It would be open book.
The suppliers or 'RC Charity' cannot afford to move to phase 2 trials and so instead are supplying a small group of PALs hoping that the results will interest investors. Large companies would normally develop a drug, get some efficiency measurement on mice and then take the gamble of pumping in loads of money to go to phase 2 trials. 'RC charity' cannot afford this.
Loads of people keep getting hung up on 'The Charity' word. The RC Charity is not a registered charity as it does not collect or receive any donated monies. It just gives the drug away. That sounds pretty charitable to me and my wife and we are very grateful for it.
When it comes to questioning how we can inject something that we don't know what it is, I do know what it is. I have seen the labs results from a batch....but I still don't know what all the number and letters mean
To all the people who keep saying get it copied. 18 months ago I thought my supply was going to be finished and that scared the hell out of us both, so as a contingency I asked several labs would they be able to copy it and I was told 'no' wherever I went, as the molecule has not previously existed (they need the order or something like that to reverse engineer) and once they found out it had worldwide IP protect, they said they would not even try.
The one thing that really does frustrate me is that my wife is one of 3 medically identical triplets and the 2 sisters have already died and her mother has died, all from the Lie114thr mutation of SOD1 and no-one she has seen medically has gone 'what is going on here?'. Her sisters and mother all died within 18 months of on-set. My wife declined rapidly for 2 months, started RCH4 and stopped progress. We are now 18 months on with no progress. That is not placebo. The geneticist claimed my wife would go exactly the same way as they were medically identical. Now if I was her Neuro, I would be interested to know what is going on. Our Neuro is supposed to be the best in Australia. He had her gene tested to be sure of her diagnosis, which backed up all the previous tests he had done. This is almost the reaction I see everywhere, where all the 'professionals' have been used to the fact there is no cure, and so how can this small 'secret' society come up with a treatment that the mainstream Neuros and Dr's paid loads of money can't come up with.
On the same tune, it amazes me that ALSUntangled still has not contacted the RC charity to do a proper review. RCH4 is the top of the chart, there is loads of interest. I think if Bedlack was to appoint a reviewer to conduct a proper review, the RC Charity would work with them.
So how is RCH4 going to move forward. Easy you might say. Just find someone loaded to put in the money to go to Phase 2 trials. Easier said than done. I've tried writing to people but usually don't even get replies. Perhaps some of you may know someone that would take it to trials? It is harder than you think.
Here is a brief of what process needs doing for RCH4 to go to Phase 2 and this is why it is hard and why someone needs to come in with money to progress. It shocked me just how many steps.
Design the trial and write the protocol
Find a neurologist willing to act as the Principal Investigator (the PI)
Find and recruit clinics or hospitals who are capable of recruiting patients (about 10 clinics - depending on the number of subjects in your trial. An active, good, ALS clinic typically is able to recruit 2 PALS per month. If you have 100 subjects, then you need 50 clinics to get started in one month, or one clinic to achieve it in 50 months. Take your pick - but the more clinics, the cost of the trial explodes as contracting with clinics needs travel and (usually) numerous meetings with each clinic management team.
Then before the trial start, every clinic must be given a training session for their staff so they fully understand the trial, its objectives and understand the protocol. More clinics = more time and travel = more cost.
The monitor must travel and visit each clinic every few months to check the security of paperwork, that the protocol is being followed, etc. and check the blinded doses which must be distributed to each clinic`s designated pharmacist who controls the handing out of which blinded code dose to which patient.
More clinics, faster recruitment, faster trial and higher cost.
Fewer clinics, longer time to do the trial but at lower cost.
Get the trial protocol approved by an ethics committee
Appoint a data safety monitoring board (DSMB)
Get Regulatory Authority approval for the trial
Negotiate a contract with a CRO (Clinical Research Organisation) to run the trial.
Appoint a trial monitor
Negotiate a contract with a secondary manufacturer to process, code the doses (remember, a double blind trial requires two different doses, placebo water and the drug itself in identical packaging so that neither the clinic doctors nor the patient know what they are giving / getting).
Retain the services of an approved entity to break the coding when the trial is finished so then who got what is revealed.
Appoint an approved medical statistician
Retain the services of a Pharmaceutical distributor to deliver the doses to the clinics
Place the contract for manufacture of the IND (Investigative New Drug) for the clinical trial. Lead time 4 months, so it is a calculated risk / guess when to do it - your trial may be refused. Or one can wait until approval is granted - but then 4 more months is added to the start date for the trial.
Contract with medical writers to tabulate the trial results and write the report in compliance with the format and layout requirements
See how hard it is
Anyway, I don't have any secrets about the use of RCH4 and I'm quite happy for anyone on the forum to PM me for my details and you can come visit my wife, Facetime her or just give us a ring.
Hope this helps with some questions
By the way, my wife is still doing great with no progress. We even went to Sydney a few weeks ago for our wedding anniversary and she walked miles.
Thanks
Marlon