With absolutely no available biomarker that accurately measures PD progression, and the clear lack of even anecdotal evidence demonstrating a robust neuroprotective action of isradipine for PD, is this study money well spent ?

John, I certainly hear you and understand that sense of urgency. Frankly, I really do agree with most of your points. In particular, I have been an advocate and am in full agreement that new research is needed at the PD subtype level. You certainly may be correct in suggesting that we are averaging out study results across subtypes and may well be missing out on possible drug efficacy in many situations. And, no, I don't necessarily think that the Isradipine trial will find THE answer.

However, where we disagree is that I believe that a properly designed and controlled cllinical trial is more likely to provide us with useable information about the efficacy of a drug than your suggestion of three month uncontrolled individual studies. In addition, even if you were able to find interesting information as a result of your study, it would never move the science forward because it would only be considered anecdotal, at best. Like it or not, we live in the world of the FDA and EMA. If we want treatments that will benefit all of us now and our children down the road, we have no choice but to follow the rules and get the proper government agency approvals. I am all for finding every possible way to speed this process up, but to avoid it will not help in providing long term treatments for all of us.

So, as my original posts pertained more toward this specific drug and trial, I guess we disagree as to the best course of action. Keeping in mind that the hypothesis behind Isradipine and PD is that it may be neuroprotective and not provide symptomatic relief, I don’t see where a three month, uncontrolled, individual study will give any of us much useful information. Is three years too long, I don’t know. But I certainly believe three months is way to short to test out any neuroprotective drug.

I would like to add that I appreciate all of your posts. Although I may not always agree, I think they are thought provoking and provide for interesting debates. In many other situations I think that your shorter term individual study suggestions would be quite useful. I'm just not sure it will help in this specific case.


Thanks,

Gary

Tupelo3,

You make a good point "we live in the world of the FDA and EMA". You go on to argue that "we have no choice but to follow the rules". You may be right. My wish is to change the rules.

In my opinion, PD research and treatment can be done more efficiently and more effectively by making better use of technology. As I see it, if there was the will, all PwP could, using electronics, have their symptoms monitored continuously, the data sent to their doctors and an anonymized version placed on a database open to all to research. In such a world, much of the distinction between treatment and research breaks down.

On a specific point, you write:

"However, where we disagree is that I believe that a properly designed and controlled cllinical trial is more likely to provide us with useable information about the efficacy of a drug than your suggestion of three month uncontrolled individual studies."

I actually wrote:

"I find it difficult to understand why a trial should take 3 years. What extra information will the last two years give? I've posted previously on high frequency testing - measuring 24/7 rather than every 3 months, say."

The 3 month figure is used as an example of bad practice, not good. To some extent, there is an equivalence in the power of a trial between the frequency of the observations and/or the number of participants and/or the duration of the trial.

The individual studies should last as long as they are benefitting the patient: constantly collecting data and using it to assess whether the treatment is working and should be continued. To enable the sharing of data, I see the need for each individual study to follow a common protocol. This provides control, but it should only be followed to the extent that it is in the interests of the patient.

John

5 years later ...

My thanks go to Iqbaliqbal at HU for alerting me to this.

The STEADY-PD III trial:

Blood pressure drug shows no benefit in Parkinson's disease -- ScienceDaily

"The phase 3 study involved 336 people with early Parkinson's disease at 54 sites in the US and Canada as part of the Parkinson Study Group. Half of the participants received 10 milligrams daily of isradipine for three years, while the other half received a placebo."

Calcium Channel Blocker Flops in Parkinson's Disease Trial | Medpage Today

"No significant difference was seen after 36 months in average Unified Parkinson Disease Rating Scale (UPDRS) Part I-III scores measured in the ON state for patients receiving isradipine (DynaCirc) at 10 mg daily versus placebo (treatment effect 0.27 points, 95% CL -2.5 to 3.0, P=0.85)"

John

Another one bites the dust....

Thanks for ALL your informaion! One and all.