The Parkinson's Drug Trial: Hi all This... - Parkinson's Movement

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The Parkinson's Drug Trial

Hi all

This is a link to my vlog about the recent BBC documentary about the Parkinson’s Drug Trial.

Cheers,

Ian

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Ian, thanks again for another good vlog. Too bad the people who run the governments of the UK and the US are not able to see the simple math that it would be cheaper to fund research if only to slow down the progression than it is to provide care for 10 to 20 years for all those who will need it. "If the onset of therapy could be delayed by only 5 years, it would cut the cost of long-term care in half." -- Dr. Laura Mischley.

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I know, it's so short-sighted...

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I’m glad they are doing another trial. The two part video you mentioned was heartbreaking. It seemed to work very well for some of the participants.

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It is worth reiterating that 1 of the problems with for-profit drug research is that many, many trials are very beneficial to some of the people, but not beneficial to a large enough percent for the drug company to make is much as they want going to market. If the pharmaceutical industry were taxed more, it would allow the government to follow through with research that would be beneficial to some, even if not all. For every 1,000,000 people who have PD, isn't restoring normalcy to 200,000 worth it?

I'm getting tired of the whole "don't raise my taxes" mantra/crowd. How boorish.

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The Phase II clinical trial recently completed for GDNF was a total bust. Do yourself a huge favor and actually read the clinical trial results instead of getting something 3rd or 4th hand like from the BBC whose expertise in pharmacology and chemistry is almost non-existent. Miracle Cure? Good grief. Seriously?

>>>"The results of the Phase II clinical trial suggest that the treatment was having an effect in the brain (based on imaging data), but the clinic-based methods of assessment indicated no significant effect between the treatment and placebo groups."<<<

MBA: try to understand the issues in bringing a drug to market via the FDA lengthy approval process. You make it sound like it is no big deal and cost pennies. Wrong.

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Sharon,

Are you a PWP? You are obviously very intelligent and a good researcher with a lot of good information to share, but your tone is sometimes condescending and rude. Everyone here is doing the best they can while struggling with a debilitating disease or caring for someone struggling with a debilitating disease. Having patience and being kind is possible even while disagreeing with someone.

Julie

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I have no affiliation with the Parkinsons Wellness Project (PWP) or nor am I someone with PD (the cute PwP abbreviation is humorous, but who cares), but I work for free with 2 local PD support groups since a good friend was diagnosed a couple of years ago. I would like to let some things pass, and probably should but frankly, I have little patience with things that are published on this forum (and others) which are ridiculous if not downright erroneous, or dangerous. Sorry, but sometimes I just can't let some things pass because somebody took the time to post.

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The acronym PWP has been in use for many years, well before any organisation used it, it simply means person /people with Parkinsons. It is not a 'cute little abbreviation' it is the shorthand by which we refer to ourselves. It is not just used in one country to mean that, but globally when English is the language of communication. If an organisation has now somehow used it, perhaps inadvertently it does not take its hiatory away. I am 17 years on from diagnosis and there was an established communities of people using this acronym then, as far as I know they had been using it way back when the internet was list serves and chatrooms, in the 1990's. Many of them had been lobbying Congress in the US when I first engaged with them around 2003. In the UK a dialogue was underway by 2004 and the term was being used here too. We understand who we are, and understand a lot of the research that has been going on. More importantly we have an intimate understanding of how the disease actually plays out in our lives and what losses we have as a result.

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And the BBC documentary is first hand, filmed over a period of years with the patients and doctors directly involved.

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The Phase II trial was a complete failure. The concept is a complete failure. So why even do a documentary extolling the possibility of GDNF being a possible miracle cure? I can tell you why but you might consider it rude.

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I don't think you understand the significance of this trial at all. Firstly as the film documents the trial was held in two parts, of which only the first part was used to determine the outcome. The data collection was predicated on a rating scale called the UPDRS which is to say the least controversial in its own right because it does not really reflect things well and is highly subjective. The endpoint of the trial was set before it commenced at 30% improvement over standard treatment as measured by UPDRS. However it only managed 6% in that first phase. That is 6% improvement so it does nor represent as you claim a total failure at alk, it represents the trial not meeting preset endpoints. A total failure would have been no improvement at all or worse than that, a deterioration. But that did not happen. Furthermore, in the second part of the trial nearly everyone had a very good improvement, which was clearly demostrated in the second part of the film. Out of a total of 42 participants only six had no improvement, there could have been various reasons for this, and the data shows some huge improvements. I personally know four participants three of whom were in the film, and can attest that there is visible and tangible improvement that happened to them.

I have no personal axe to grind on this, my parkinsons is atypical and it is unlikely I will benefit from any of this research as I am now 70. It takes around 17 years to get a drug from research bench to market.

There are increasing numbers of young, somwtimes very young people being diagnosed with parkinsons, the current estimate is 150,000 in the UK and 10 million world wide. Frankly it is not whether GDNF works that is most in question, it is what can be done to get it to the brain in a simpler and less invasive way. It will not treat all cases of Parkinsons, because there are multiple causes, but it is likely to help many. The participants were courageous and selfless in their approach, personally I am very heartened by this and how their experience will improve a lot of things for others, hopefully including the UPDRS which is in drastic need of yet another oveehaul, it is 20th century science in a twenty-first century world.

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Thank you for responding in a way that as a YO PwP, 11 years in, I am no longer capable of doing.

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You could argue that GDNF was unsuccessful, but the participants and clinicians believe that the trial needs extending to test dosage and time period variations. What was a total success was the new delivery system which has potential for other diseases and other drugs.

On the costs point, there is massive research in repurposing existing drugs for PD. The cost and time is much reduced because the Licencing has already been done.

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Sharon, I'll try harder to understand the issues of bringing a drug to market.

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Sharon,

Focusing attention on how much a pharmaceutical company spends bringing a drug to market is meaningless except in the context of how much they make.

It is judging the profit and loss statement without the balance sheet. Pointless.

If a drug company spends $500,000,000 to bring a drug market, some are tempted to sympathize with them, but if they make $2,000,000,000 in profit per year for the next 17 years, should we really feel sorry for them?

True, a lot of drugs don't make it the market, but that doesn't count for anything either. The only thing that counts is how profitable they are and last I checked they're doing pretty well.

Marc

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MBA: What socialized government has ever developed a meaningful drug? None. What meaningful, significant drugs are developed in socialist countries (countries that offer free (crap) medical care and drugs)? Very, very, very few if any. IOW, regardless of your political beliefs about profit, without the profit incentive the drugs you are on, such as Sinemet by Merck, would never have come to market, nor would their copy-cat generics. The cheap generics exist only(!) because of the money the big pharmas spend to bring a drug to market.

Your idealism is fine the the abstract, but totally irrelevant in reality. If you don't believe me, try bringing a drug to market. Don't believe me, Go read the article in Scientific American about the cost to bring a new drug online. Roughly $2.5-$3.0 billion. So, your figure of $.5B is way, way, way too low.

As to these companies profitability, if they weren't profitable they wouldn't exist and you would nothing in the way of the meds you are on. It is a high risk business regardless of what some would have you believe.

P.S. I'm not a "rah-rah" cheerleader for big pharma.

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I doubt that this is the only possible truth: the really useful drugs are only about fifty and were almost all discovered by independent people, often randomly and accidentally (see A. Fleming or discovery of helicobacter pylori for example, ah also the components of simenet). This is the big problem of the big pharmaceutical companies (and of all NASDAQ companies, including Apple), i.e how to maintain the commercial monopoly of research and discovery if an independent researcher can get a better result?

Solution: a very expensive patent system that is not affordable for everyone.

(reference: history of medicine)

nature.com/articles/466S6a

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"What socialized government has ever developed a meaningful drug. None."

Swiss companies;

Hoffmann-La Roche: Basel.

Novartis: Basel.

Alcon: Geneva.

Bayer: Basel (Consumer Health Division)

Lonza Group: Basel.

Abbott: Basel (Established Pharmaceuticals Division)

Galenica: Bern.

Actelion (Johnson & Johnson): Basel.

Upjohn - Sweden

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"... My political beliefs about profit..." Nothing I said reveals my political beliefs about profit. I've started and built a couple companies for the purpose of making profit. I was trying to explain the expense side of the ledger only has meaning if it's in relation to the revenue side of the ledger.

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"Cheap generics exist only because of the money big Pharma spends to bring drug to market." Cheap generics exist because unlimited patents stifle competition/capitalism/profits.

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Typical time from origination of a drug compound to FDA final approval (if any)? 10-15 years with 13 about average.

Historical success rate for bring a new drug to market? About 1 in 12 and will probably decline even further in the future if previous historical norms are any indication (success rates are declining, not improving).

Average cost to bring a new drug to market? generally about $2.5-$3 billion although with the newer immunology drugs it can run higher towards $5 billion.

Number of top selling drugs still on patent protection? Last I looked very few as in the example of Humira, which is still probably the #1 selling drug but does not have patent protection.

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I know all that, but you still have not responded to my point.

Looking at how much they spend per trial or how many drugs don't make it to market is the same exact thing and is still looking at half the ledger. It is not in context. It has no meaning. The pharmaceutical industry is among the most profitable of all.

What concerns me is not how much they make, but the character and morality of the executives who run these companies and the record is there for all to see -- in court documents where they are found to be in violation on a routine basis.

And, the difference between their violations and that of other industries is that when they withhold and distort data, people die.

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Couldn’t agree more. I also would like to see vit b1 part of a formal trial. It has done wonders for me

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