This article below from the NYT lays out many of the challenges that plague drug testing. I remain convinced (as many of you are) that we need more patients contributing more data --- it is likely the only way to better overcome persistent failure.

Many here are already engaged/active...but what does it take to increase engagement by the multiples needed? How can we do better? MJFF is working on new web-based tools for patients to provide more data (hopefully to debut this Fall/Winter). Peer-to-peer discussion/encouragement is our best hope I believe.

http://www.nytimes.com/2013/07/14/op...nted=1&_r=0&hp

Debi

Debi, many of us responded to your previous post related to this subject. I agree that we need much larger sample sizes so we can get to homogenous sub-samples, which are needed. Peer-to-peer discussion will certainly help and, as patients, we need to improve on how we can accomplish this. However, I strongly believe that the best hope is more involvement from our primary PD doctors. I think that most doctors are not aware of the current studies and are not doing enough to get their patients to participate. Maybe it's an issue of liability. But, they are the primary link to the PD patient, more so than these forums. The docs need to advise their patients on the importance of participation and guide them to the appropriate research study.

Just my opinion. Your work is greatly appreciated.

Gary

Debbie, one of the problems is that by the time people become proactive there are many things that exclude them from studies. i.e. they by that time are not de novo. Other factors, the exclusion factors actually are the exact thing that you would find in pwp, so the trial is designed for an almost unfindable minority of people.

My idea on this, as someone who has looked at the trialfinder in my own country, and found not a single one that I would be eligible for, is that if they want participants they are going to have to widen the criteria. Patients could help with this. But it needs having patients there in the planning stages of trials, to say, well, if you do that, you are setting up barriers to participation that will be very hard to overcome. This is the message that needs to go to trial designers.

In my opinion, clinical trials need to have some hope of an immediate benefit to the participants. Add this and you will get a lot more volunteers.

RudeI have contacted every research trial sent to me by Foxfinder. I have 2 mutations of my Parkin gene, I have a positive FDOPA Scan, and use sinemet for livable symptoms. I do not qualify for any tests Foxfinder has sent to me because of my DBS. Most researchers insist on having the ability to perform a MRI scan or DAT scan.

Vicky

I am very excited by the proposed MJFF data collection system. The power of the MJFF brand name is such that I expect that within a few months 10000 people will have enrolled, enough to give some serious data mining opportunities.

I hope the data will be open to everyone to analyse.

My experiences with PDMeasure, which covers similar ground, but with far smaller numbers of participants (96), may be of interest.

What have we learnt so far:

1. The learning effect of repeatedly taking a symptom measurement test, in our case the side to side tap test, is more powerful and goes on longer than expected.

2. Only 9 healthy people have enrolled, clearly an insufficient number of controls. Effort needs to be put into attracting them.

3. A distinction must be drawn between the reporting of people's condition and even minor interventions to change it. The latter requires a stronger ethical framework. I've been unable to get an ethics committee formed. The moral of the story is: get the necessary organizational structures in place before releasing.

PDMeasure can be found at:

http://www.parkinsonsmeasurement.org/PDMeasure/

John

Another thing that will not be probed and discussed and brain-stormed at the World Parkinson’s Congress in Montreal.

“You must not take any Parkinson’s drugs, or any other drugs, or ingest any food or any liquid other than water, for 12 hours before your appointment. You must be able to remain perfectly still during the scans. Any movement will result in the scans being unusable.”

What is wrong with this picture?

“You are responsible for your own transportation to and from your residence and the 3 test sites.”

(Volunteer lives 50 miles away and his driver’s license was revoked, and he is unfamiliar with the city).

He managed to get a ride to the city and was in time for the 8:00 a.m. appointment, having taken no pills for 12 hours, but then spent four hours in the waiting room. No pills for 16 hours.

At noon, two bored and blasé college students arrived and gave him the childish cognition and balance tests. Shown pictures of animals and asked to name them. Recited imbecilic stories and asked to repeat them. Pulled backwards to see if he could regain balance. And of course, lots of finger tapping.

An hour later (17 hours without pills), a lab technician came and said get undressed, put on this gown, remove your wedding ring; lie down, stay perfectly still. And admonished him aggressively when he could not stay still for the scans. “It’s a complete waste of time if you don’t stay still.”

Two college kids and a grumpy lab tech. At no time was there a doctor or a scientist present

The volunteer specifically asked to know the results of the testing, at least his own results. This was refused. They said it was never done. They would not even release the results to his doctor. Proprietary information.

His father had volunteered for World War II. His grandfather had volunteered for World War I. He thought, this was his war.

He thought he was joining something. He thought he had volunteered for a fight to the finish. He thought they would come out of the trenches and make a frontal assault. A band of brothers. Willing to sacrifice so that future generations would be free.

That was four years ago. He never heard from them again.

He never knew their names. He met no other volunteers.

As far as he knows, the study was never published.

“I volunteered because I wanted to be part of something bigger than myself. But they showed me how small I am.”

Nobody wins a war that way. Just ask Alice, when she is two feet tall.

are you trying to start a discussion bob?

Move along. Nothing to see here. These are not the droids you are looking for.

I know of a small town, where there is one big factory, one motel, one bar, 5 hamburger joints, and 6 churches.

In that town, there is a General Practitioner, in his 70’s, who has been the only doctor in town for the past 45 years. He knows everybody, and he knows all about everybody. He writes up the birth certificates; he is there when they are born. He writes up the death certificates; he is there when they die.

He is not just a doctor, he is a DOCTOR. A Country Doctor. Old school. He still sometimes makes house calls. At his own cost.

Call him in the night, and the conversation goes:

Are you bleeding?
No.
Do you have fever?
No.
Are you getting crazy?
No.
Well go to sleep. You’re okay.

Because he knows everything about everybody, he knows that there are 18 people in town, and on the surrounding farms, who have Parkinson’s.

One is a teacher, one is a truck driver.

About half of the rest had worked at the factory. Rubber door mats, the rubber blades of windshield wipers, the rubber rims around car windows. We’re talkin’ black rubber here, moulded when steaming hot. Before pollution controls, you could smell the rubber all over town. Black soot darkened the factory windows. The factory dumped pollutants straight into the little river that ran beside the railway tracks.

The other half were farmers; or more precisely, they had big apple orchards. In spring, you could smell the pesticides and fungicides floating in the air on the days when they sprayed the trees. And then herbicide between the trees.

And they all drank artesian well water.

Parkinson’s was not the only disease in town. Somebody at a big city university pin-pointed it as a “hot spot” for a lot of bad things.

Volunteers were called for. A longitudinal study that is still on-going.

Of the 18 People With Parkinson’s, 15 joined the medical trial. En bloc. As a group. All at the same time.

On their own terms.

With their questions answered.

With their concerns addressed.

With their pride and dignity intact.

For a hoped-for victory that they could call their own.

Feeling in charge, not pawns in a game.

Moving as a group, not solitary and isolated.

Being told the truth, the whole truth, and nothing but the truth.

Coming back home like war heroes, not like beaten dogs.

The pride of a proud community.

It started about ten years ago, with the Buddy System, launched at the demand of the Country Doc. Every PWP gets a call, at least once a week, from another PWP. Or from 10 other PWP’s. All the buddies you want, and it plugs you into the local gossip network. They start off talking Parkinson’s; then they find other affinities; they visit each other; they watch hockey games together; they visit you in hospital; the mobile ones come and paint your kitchen or shovel your driveway.

This is not healthy people taking care of and watching over PWP. This is PWP helping and watching over each other.

It becomes a fierce little group. Some die, some, newly diagnosed, join. The Country Doc said “I can’t watch out for every kook in town, so they have to take care of each other.”

They don’t collect money. They don’t have an organization or an address. Just folks watching out for each other.

It is Parkies looking out for Parkies. Carry your grocery bags to your door for you. Help you do your taxes. Bake apple pies for all. Be there in your dying days. Over time, they develop a fierce group solidarity, which people outside cannot see, and may never experience.

Nothing brings people together more than a deadly catastrophe, and Parkinson’s is a never-ending deadly catastrophe in the lives of those afflicted by it

The Buddy System had been building the solidarity for about 5 years when an epidemiologist in a big city university decided to compare them to half-a-dozen other mill towns and mining towns and heavy pesticide orchards.

They called for volunteers. The Buddy Network went in to overdrive, and they all agreed that NONE OF THEM would volunteer, unless:

The scientist came to their town and met with all 18 PWP at the Legion Hall; (very important – he went to them)

The scientist explained to them what questions they were seeking answers to; what the scientists already know and what they don’t know;

The risks involved;

What contribution this research would make in the bigger picture;

Guarantee that they would have access to all the results;

Guarantee that the Country Doc would have access to all the results;

Guarantee that the Country Doc would have veto power over any medical process that he deemed to be dangerous or useless or wrong;

Guarantee that all data from the trial would be made public;

The Country Doc would take the blood samples and do the routine tests at his office in town – the PWP would not go to the city for this;

The only time they would go to the city would be for X-rays, MRI’s or similar scans;

To get to and from the city each time for scans, the research project would pay for a mini-bus to pick them all up at their own homes, take them 100 miles to the city, and bring them back;

All 15 would travel to the city together as a group;

They would be greeted at the door of the university by an experienced nurse who would guide them around and hold their hands and explain everything to them throughout the process.

The informed consent contract was re-written in language that did not require ten lawyers to understand.

All of the above points were granted, to the Parkinson’s patients, who made it clear that otherwise, they would refuse to participate. THEY made the rules.

The Buddy network got back on the phone, and they all agreed together that they would ALL volunteer, except three who were too sick to leave their homes.

There are three keys to this story:

The Buddy Network.

The Country Doctor.

Feeling that you are part of something bigger than yourself.