To speed up research and trials, we requi... - Cure Parkinson's

Cure Parkinson's

19,280 members20,119 posts

To speed up research and trials, we require financial support

RoyProp profile image

A great deal of money will be necessary to fund: research, study, testing. It may be possible that B1 could evolve into a more efficient treatment.

Here is information that I have on file.

“As you all know we submitted to the Michael J Fox Foundation a project proposal for a clinical trial involving the High Dose Thiamine treatment. Although the pre-proposal was invited for further development (stage 1) unfortunately the proposal has been rejected by the reviewers at stage 2.

The reviewers expressed various concerns and a general skepticism about the impact that thiamine with the protocol developed by Dr. Costantini can actually have on PD. The three reviewers although contradicting each other on specific sections of the review, generally implied that the research is not groundbreaking and novel enough, not very trustworthy, same goes for our design (based on dose titration aka giving each patient its efficient dosage or else no improvements can be seen whereas they would have preferred a one size fits all type of dosage) and the lack of experience and reputation of us as Investigators. Reviewers also pointed out that our estimates concerning the recruitment of patients were far too optimistic (200 patients from two of the largest hospitals in Italy in 12 months), one highlighted that the budget we estimated for the clinical trial was too low to be true and in general said that our proposal is unrealistic and the expected results would not be very relevant to the PD community.

I am very sorry but this is their verdict and we can only accept it even though I personally feel this is a missed opportunity for many PD patients.” End Item

17 Replies

Roy,

Some of us contributed $$ to this proposed trial using "Go fund me." Can't we continue doing that?

RoyProp profile image
RoyProp in reply to Despe

I don't remember were I read, said the find hasn't been drawing any funds. The intention is to keep the go fundme going.

Despe profile image
Despe in reply to RoyProp

Well, I am willing to contribute again, but we need more info.

RoyProp profile image
RoyProp in reply to Despe

CopyTalked about this with Dr. Fancellu last month. They will apply again with new data and a new approach. Almost 1M $ is needed if anyone is a billionaire ... 😅🤞

WinnieThePoo profile image
WinnieThePoo in reply to Despe

gofundme.com/f/high-dose-th...

The funding site is still there.

It's some way short of it's E1,000,000 target with E5245

It is shambolic - as MJFF observed. It doesn't appear to know what it is trying to achieve

It says "In order to be accessible to every patient in the world, the High Dose Thiamine protocol requires approval from the various international Drug Administrations."

That is not true. Thiamine no more requires Drug administration approval to be accessible to every patient worldwide than do frozen peas or yoghurt. And so no trial is going to enable it to get DA approval - because it doesn't need it.

"Approval" in this context means "permitted to use". It doesn't mean recommended or suggested. Does it's lack of FDA approval in any way make it inaccessible to you?

The further mis-apprehension is that DA approval would direct doctors to prescribe. This is also not true. Sinemet is approved & authorised for treatment of Parkinsons disease and my doctor has not chosen to prescribe it for me .

And then the proposed trial structure is jumping the gun. Before you can organise a randomised blinded placebo controlled trial - you need to have decided dosing

So what dose of B1 are you going to trial? Roy's 6g? his former 4g? What sort of dropout rate would you anticipate? And if you are going to do it by injection instead - 100mg twice a week? Is that what your husband is using? Has it always been?

If you want to adjust the dose until you get efficacy or avoid side effects what are you going to do with the placebo?

Even Art couldn't produce data for a dose per Kg bodyweight. That would probably have to be adjusted for gender, ethnicity, and time since diagnosis.

But I think Roy has 3000 subscribers to his Facebook page. If they each fund $350 - you can fund your trial. And then...

kevowpd profile image
kevowpd in reply to WinnieThePoo

The other risk here is that it comes back as no better than placebo. I'm not suggesting that that outcome is a foregone conclusion. At all. But it is worth considering. How would those that have a firm belief in its efficacy react to that news, if they were to receive it?

Make very good points, WinnieThePoo.

Despe profile image
Despe in reply to WinnieThePoo

WTP,

I am aware of all you wrote. I know we are on our own and B1 is an individual trial/error process. However, it doesn't mean that B1 doesn't warrant trials. Always $$ that gets in the way!

WinnieThePoo profile image
WinnieThePoo in reply to Despe

My point was its not so much dollars as amateurism. Why does the fund raising page say it wants drug administration authorisation for a substance which requires no authorisation?And why does it say that lack of authorisation interferes with accessibility?

B1 therapy is accessible to anyone worldwide who can afford it's modest cost.

Still, if you want to give them money for pointless research I've given you the link to their go fund me page, so make as large a donation as you care to

WinnieThePoo profile image
WinnieThePoo in reply to Despe

The following quote is from your NAC post

Interestingly, with COVID-19 treatment as a new indication, the U.S. Food and Drug Administration suddenly cracked down on NAC, claiming it is excluded from the definition of a dietary supplement, as it was approved as a new drug in 1985.26

As such, NAC cannot technically be marketed as a supplement, even though there are no fewer than 1,170 NAC-containing products in the National Institutes of Health's Dietary Supplement Label Database.27

So if the team COULD get B1 approved as a drug it would cease to be a dietary supplement, and become less accessible than it is today. Be careful what you wish for.

Despe profile image
Despe in reply to WinnieThePoo

Basically, Gio expressed my opinion on B1 and L-dopa (velvet beans). Aren't they in the same category as B1, both natural substances?

GioCas profile image
GioCas in reply to Despe

So according to the WTP rules if levodopa were discovered today would it not be available because you couldn't do a study for the same dose reasons? it is known, we use different doses, sometimes very different.

In fact, at the beginning no double blind was done on levodopa, indeed it was much contested by the scientific world, see "history of levodopa".

(Link at the end).

So the problem is the dose of thiamine or are there predetermined contrary intentions?

It is true , thiamine does not need a study because as long as it gives results, word of mouth (which is the most effective form of disclosure I know) will be enough to make it known, otherwise in the absence of these, it will end in nothing.

I don't ask anyone's permission to survive so I don't need authoritative approval as long as thiamine is available in my country.

I tell critics and supporters to put their hearts in peace, cheer for or against it will not change the substance of what is true. The principle is: if it works it's true.

This is the story of thiamine: a doctor discovers gains with its use, the test gets results with other people who are happy to talk about it to others and so on.

But not taking into account the countless success stories I consider it at least disrespectful and superficial by anyone.

We also have another aspect here that WTP pretends not to know, but that is enough to read the documents and that is that HDT is based on thiamine injections and in most of the world the prescription is required.

No doctor will prescribe a PROTOCOL that is not approved by government agencies and therefore this possibility is precluded without a double blind and right or wrong these are the rules.

I am confident.

Times must change!

soon the PD will have his care and we will all win.

Here is the link by Nature "Levodopa: the story so far" , you will find it technically interesting ...

nature.com/articles/466S6a

WinnieThePoo profile image
WinnieThePoo in reply to GioCas

It was quite long. I may have misunderstood you.I think you are saying that

you don't need a trial to allow you to use B1

You don't need a trial to tell you that B1 works for you

And therefore a trial is unnecessary and you don't support the project on the go fund me page I linked to.

Is that correct?

GioCas profile image
GioCas in reply to WinnieThePoo

Obviously no WTP,but if my 338-word post is long, your 318-word post is too, and you've written four here. I have read your posts trying to understand without making an altered and useless summary.

I will be short colleague Winnie because I understand you. :-)

If you had deepened the subject in the correct context, you would have found all the clarifications you are asking me now and you would understand my point of view right there on Gofundme where in addition to donations you will find thanks from those who have benefited from HDT.

In my opinion, ignoring them is disrespectful by anyone.

Buona Pasqua! Gio :-)

C
RoyProp profile image
RoyProp in reply to Despe

MemberLaura Dell’Erba

Talked about this with Dr. Fancellu last month. They will apply again with new data and a new approach. Almost 1M $ is needed if anyone is a billionaire ... 😅🤞

@royprop, I just won the Timbuktu lottery. Where should I send the 100 million dollars that is burning a hole in my pocket?🤔🤔😊

RKM

For the people who want the trial, what is your reasoning? B1 is freely available. It seems to have very few side effects. It's pretty cheap. What benefit would it be to spend 5million on a trial for a vitamin that doesn't need fda or ema approval and can be freely tried by anyone? The results on this forum have been hit and miss. Works for some. Doesn't work for others. A trial would probably replicate that and the trial would fail. So what's the point?

You may also like...