One problem I see with this proposal is that the premise underlying it is based on a sample size of only two. I think the current trial needs to be completed before a new trial such as the one proposed could commence.

As you said recently in another thread, Nan, the main reason the current trial will take so long is a lack of resources, not a lack of volunteers. If there was some way to increase those resources, the end date could surely be brought forward considerably.

As I see it, there is my PD (which may be different to yours) and my response to it (which almost certainly is different to yours). Given this, why focus on increasing n, the sample size? If X works for me, the fact that it doesn't work for you doesn't stop it working for me.

As I see it, what we really need is evidence that the therapy (including any placebo effect) is really working for Nan (taking into account the risks). If it is working, try to continue with the therapy, monitoring your progress, good and bad, as you go. If it stops working for you, then (possibly over a period of time) stop using it.

John

If it only works for a few PwPs, then I would think that it would be too expensive to provide the service. I think that it needs to work for a large number of PwPs. To determine this you would surely need something similar to the current trial(s).

Similar to the current trials, but not the same. Instead of having such a broad range of patients, narrow it down as I suggested in my long post. People who cycle, people who are fit, people whose symptoms are a, b and c. Additionally, I think there should be a compassionate response that would allow someone who experiences relief to continue the treatment as John suggests.

Nan, I don't think it can be narrowed down yet. N equals 2 at present. I think that N needs to equal at least 18 (i.e. the end of the current trial) before any narrowing down takes place.

Regarding the possibility of a compassionate response, I see loads of obstacles to overcome:
- determining who qualifies;
- setting the length of the response;
- getting permission from donors for their plasma to be used in this way;
- setting up the infrastructure, and processes, to support this response;
- securing the funding.

I'm not sure that a young-plasma service for PwPs is even the goal of this research. Maybe if the current trials are successful, the next project will be to find out exactly which component of young plasma is responsible for the improvement(s). Maybe it will only be made available to PwPs if/when it reaches the form of a medication/supplement.

How should we interpret Nan's apparent good response and the other lady's apparent non-response to the treatment?

I don't see either response as conclusive for either party. I see the situation probabilistically. Based on prior experience the two ladies went into the trial with a probability of success. On the basis of the results we have seen, we need to adjust the probability for both. For instance, we may of thought that Nan went into the trial with a probability of success of 0.2, and on the basis of the response so far we need to increase this to 0.5, say. (This approach is called Bayesian.)

We can add to our knowledge by increasing the number of new patients or by increasing the number of data points for existing patients.

There are all sorts of confounding events that could have affected the responses: the type of PD; the placebo effect; and the weather being obvious ones.

I agree with jeffreyn that there's a long list of difficulties that need to be overcome before someone can be allowed to continue with the treatment. But, I don't think that these are insurmountable.

If we want more people to volunteer for trials then we need to make their experience better. A compassionate response should be the least that we can expect.

John

Clearly my response to all of this is the skewed by the fact that it's my body we're talking about. Thanks for bringing more rational perspectives to the table.

I had my last infusion on Valentine's Day and three months later, nearly all the positive benefits are retained. This week I bumped into the Movement Disorder Specialist who diagnosed me nearly 10 years ago and has seen me seldom since. She was flabbergasted at how little I've changed from when she knew me before any PD.

More Than Placebo May 26, 2017

Background In December 2016 I started the Stanford Young Plasma Study. I had eight infusions of blood plasma taken from 18-25 year old males as part of the Phase I study to see if this procedure was safe for humans. As I was the first Parkinson’s patient in the world to have such infusions, no one really knew what to expect. The hope, of course, is that young plasma will somehow be able to mitigate the course of the disease, or perhaps even reverse it. I had three extensive on and off medication batteries of tests, including UPDRS part 3, 1) in January prior to the infusions, 2) after Valentine’s Day at the end of the infusions and then 3) 5.5 weeks later. The PI performed the first round of the off medication testing and then other staff (4 or 5 in all) performed the other tests. We were thrilled that my test scores went from 28 off meds and 13 on meds prior to infusions to 14 off meds and 10 on meds at the end of the infusions and 14 and 7 after 5.5 weeks with no infusions. Infusions? Placebo? Who knows what is going on? I can report that I feel much better in nearly all aspects of my Parkinson’s and the effects continue.

Update This week I traveled to Baltimore for my third year of participation in another Biomarkers research trial, this one at Johns Hopkins. I was more than a little eager to see how I would score on the same UPDRS test at JH. The PI tested me a year ago on the UPDRS and tested me again May 25th. She self-reports as a “hard scorer”. I refused to tell her about Stanford until after she had completed the UPDRS test, now 3.5 months after the infusions. I scored 19. More significantly, she told me that last year I scored 28 using the same test and the same person giving the test, a drop of 9 points! Later she told my husband that when I walked into the office she was startled at how much better I looked from last year.

I think we can safely say that this is more than placebo. So far we have N=1. What is there about me that I respond so strongly to the infusions? Is this a combination of cycling, dance, medicine and infusions or is something else going on here? Questions I can’t answer. I can only be happy and grateful and hope the trial will become available to others soon.

That's a wonderful update, Nan! Thanks for all you do for our community and for all of the trials that you participate in. Let's hope that they move this study along into a larger phase 2 as quick as possible.

Gary