Old 07-29-2018, 11:21 PM #256 (permalink)
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Default Re: Still here thanks to RCH4

TIANDB, I am not a PALS, to your comment. My husband was. But I'm confused -- you are recently diagnosed and "part of the 10%" [referring to survival or what?]?

My comment about agents was general, as there are various questionable participants in this thread.

Quoting any portion of a Web site unnecessarily will be snipped as per our ongoing policy.
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Old 07-30-2018, 03:46 AM #257 (permalink)
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Default Re: Still here thanks to RCH4

Hi lgelb. So Sorry, you have a diagnosis date by your Photo and sad about your loss. My 10% refers to the PALS outside of the generalized statistic that 90% will survive for 3-5 Years . I am on a very slippery slope and unless some miracle happens even one further one year is unreachable. I go by the dates on the user panel and understandably the portion of the 10% that survive longer than the 5 Years are the ones who post the most. Go ahead name your questionable participants ? Snipp away, The negativity displayed when anyone mentions RCH4 is clear. I started RCH4 very recently 27/7 as I need a lifeline chance. Even with all the removed posts it was clear to me when reading all the threads including closed ones where the light lay. We will see ? as you have not frightened me off. What is the title of the thread " Still here thanks to RCH4 " Peace.. TIANDB
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Old 07-30-2018, 04:07 AM #258 (permalink)
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Default Re: Still here thanks to RCH4

I may not understand the relationship between the ~10% that survive 10+ years (old, dubious data) and your being recently diagnosed. But thanks for clarifying that's what you meant.

Some of us CALS have a "diagnosis date" for context about the advice/experience we offer. My husband, as my bio states, died in 2014. So he was part of the ~20% that made it five years. Dismal stats, but I'm not seeing that most posters are super- long-term P/CALS. Remember, the bio panel does not show the death date.

I am not sure why you would make the adversarial statement that I am trying to scare you off. But shadowboxing is not a good use of anyone's time here. There is very little hard data in this thread, even allowing for "patient-reported data" [which is often collected and published as part of drug development]. So no, I'm not going to roll over and let people assert unquestioned that this is a miracle cure when there is a potential for harm. P/CALS have lost their money, time and lives chasing miracles.

I have said nothing about RCH4 that is not fact or clearly-labeled opinion, and this thread would be much more useful if everyone tried to follow suit. It is important not to confuse the two in a fatal disease, where P/CALS must make often-agonizing daily decisions about how to allocate their precious time, money, wishes and hopes.

I do feel a certain responsibility to point out that RCH4 is an unknown substance, not a religion. But as you see here, I am by no means the only member drawing that distinction.

Having faith and hope for the future doesn't mean that it follows that injecting yourself with a cipher that seems destined either for the supplement market or staying in the back alley is a good idea, when there are other modalities whose evidence base is much clearer, whose mechanism is much more transparent, as we often discuss in other threads, to feel better for longer.

Best,
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Old 07-30-2018, 06:05 AM #259 (permalink)
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Default Re: Still here thanks to RCH4

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
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Old 07-30-2018, 07:25 AM #260 (permalink)
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Default Re: Still here thanks to RCH4

Hi Marlon. I think we all believe in you. Your wife’s experience is certainly remarkable although the life we live changes gene expression ( expression not the genes themselves). This was demonstrated in identical brothers who were astronauts after one had an extended space stay. I am not saying this accounts for your wife’s course but it is a true statement.

One of the things I am surprised I can’t find on the website or elsewhere is a record of the phase 1/2 clinical trial that you and others have mentioned. The clinical trials site will show old trials if you search for them. A successful trial usually generates a paper. There are many many places you can publish so if I had had a successful trial I would have made sure to publish my findings. If the first journal refused me I would keep trying. Seeing that information would quiet most of the concern
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Old 07-30-2018, 10:38 AM #261 (permalink)
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Default Re: Still here thanks to RCH4

Marlon,

I know it's not your job to get funding to move RCH4 forward, but have you contacted the Gleason Foundation? They have a lot of respect in the ALS community and even some political clout.

Unlike some other PALS, your wife's case is clearly documented and there should be no doubt she indeed has ALS. She was declining, started RCH4, then stopped declining. No matter how you spin it or make excuses or explanations, the evidence that RCH4 worked on her is overwhelming. That and self-reported statistics from other confirmed PALS, should get positive attention.

I try to think of reasons why the entire ALS community is refusing to work with the RCH4 owners, but I can't come up with a reason.

Since Richard and the others cannot seem to find funding, perhaps the Charity could hire a good public relations/fundraising specialist to contact these groups that don't seem to be interested. That kind of money can easily be raised.

I'm glad you posted, Marlon. It's wonderful to hear your wife is doing so well.
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Old 07-31-2018, 01:42 AM #262 (permalink)
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Default Re: Still here thanks to RCH4

Glad to hear your wife is doing well, Marlon.

There are a number of steps to do a trial, indeed. I have participated professionally in many of the steps you listed, and there are many you didn't. Many are part of the infrastructure of a biopharmaceutical company, which is why partnerships are common when a small company comes up with something good.

So let me clarify one part of what you said. Once patents have been filed (it's the filing that confers protection, even before the patent is granted), it is very much the usual thing to start publishing papers about the mechanism, which encourages scientists working along similar lines to either compete or collaborate; contacting investigators to be part of human trials (which begin with healthy volunteers, not patients with the target disease), working with regulatory agencies, continuing animal trials, etc.

It's worth noting that a fair number of CNS compounds fail safety trials at the animal stage, unexpectedly. We know of no approved even healthy animal safety data for RCH4 (the ALS mouse models are acknowledged as suboptimal, just as in MS and other disorders).

This is also the stage where you get an INN that serves as the going-forward generic compound name for development. You can't move forward without it. And yes, it does require "giving up" the formula, but who cares, because you've protected it, right?

So the part where you say they don't want to reveal anything until they get funding, even though they have "worldwide IP protection" seems very, very strange -- is not how science/clinical development is done -- and not so coincidentally, is a big part of our collective reservations about PALS' opting to inject this drug.

Best,
Laurie

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Old 07-31-2018, 04:46 AM #263 (permalink)
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Default Re: Still here thanks to RCH4

Laurie,

If they have gone through Phase 1 trials and received FDA Orphan status, does that not mean that mouse and safety testing would have been reported to the FDA to get that?

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Old 07-31-2018, 11:16 AM #264 (permalink)
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Default Re: Still here thanks to RCH4

There’s no proof of them going thru a phase one trial. If there is ,show it to us. I’d like to see it on a FDA list . Thanks.
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Old 07-31-2018, 01:39 PM #265 (permalink)
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Default Re: Still here thanks to RCH4

Hi guys.
This thread is filled with useful info and commentary, but still needs some attention from the mods.


I believe that the worldwide ALS community needs good communication.
Fortunately, there are forums out there (fora?) to serve us all.
Some sites focus on health stats. Some focus on emotional support.
Some are loosely moderated and encourage banter.

Many members come here looking for a conflict-free zone--a safe place.


This site is tightly modded and has many subfora to keep discussion focused: religion, CALS, PALS, veterans, and (importantly) DIHALS.


Our forum is tightly modded. For one thing, we don't let personal attacks or personal disagreements run freely. If a member wishes to report someone, please do so by clicking the "report" button under their post. If a member wants to discuss a mod, please contact the mod or another mod or just hit the "report" button.


But don't discuss other members or mods in the public space. That kind of “discussion” tends to devolve and get nasty. That's not what our forum is about.



Our mods strive--with the active participation of long-time contributors--to keep our forum a safe place with a friendly tone. (That's kind of rare on the Internet in general, isn't it?)


So if anyone wants to discuss another member or discuss a mod, report their post or write to a mod. Don't do it in public. That kind of discussion, although common elsewhere, is not what we do here.
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Old 07-31-2018, 06:03 PM #266 (permalink)
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Default Re: Still here thanks to RCH4

Thanks, Mike. Helpfulness and harmony are good things for both PALS and CALS.

As you pointed out, there are other forums that allow the opposite. If we disagree we should do so in the spirit of kindness.

I know I've said this before but I very much appreciate the moderators here. Their involvement and close attention to us is not met in any other forum I've seen.
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Old 07-31-2018, 06:38 PM #267 (permalink)
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Default Re: Still here thanks to RCH4

Marlon,

No, the FDA doesn't require specific development milestones to be met in submitting an orphan app, though it may refuse to grant one based on lack of demonstrated clinical merit. That is why usually some significant development establishing some face validity for the mechanism has usually taken place. It does require that development to date be documented, along with various aspects of the proposed use, forecasts, etc.
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Old 08-02-2018, 10:42 AM #268 (permalink)
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Default Re: Still here thanks to RCH4

Marlon, you said, "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."
Is there some reason you didn't go ahead and say what it's name IS in the FDA orphan drug list? This is public information after all, and all the interested participants in this thread would like to look it up and find out more about it.
I posted several days ago after searching the FDA orphan drug list and finding only 3 drugs with the ALS indication, edavarone, ibudilast, and riluzole, and we are all familiar with those. If you are going to say the RCH4 is listed under some other indication, you might as well provide all the information.
Giving you the benefit of the doubt--but there seems to be a divide between the people who would like to learn more, and the people who say they are RCH4 users. Were the RCH4 users sworn to secrecy?
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Old 08-03-2018, 09:34 AM #269 (permalink)
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Default Re: Still here thanks to RCH4

The RC Charity do not want that info out at the moment, I believe as they are worried about counterfeiting. Not sure. I found this a bit strange and somewhat hard to believe, until I heard ALS Worldwide were offering to to sell RCH4, which just goes to prove there are some sharks out there, as there is no-one else out there that knows the formula apart from the RC Charity. I know that this is frustrating as everyone wants to know the answer to the same question (what is the drug and where are the test results) but the wheels are in motion to secure funds to go to Phase 2 trials, so hopefully all this info will soon become common knowledge. Also, a bit of a shame, as the RC Charity applied to present at the upcoming Glasgow symposium which would have been an excellent forum to question the RC Charity on details, but for some reason were turned down again. Not sure what is going on out there in the world of ALS, as RCH4 is the top of ALSUntangled and is getting some excellent results in PALs around the world, so I would have thought it was a no brainer to let them present. I think they have been offered a 'consolation prize' of a poster presentation.
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Old 08-03-2018, 09:35 AM #270 (permalink)
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Default Re: Still here thanks to RCH4

How come I'm still a 'new member' is I've been on here since 2015?
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