Clinical trials of one person


#1

A number of clinical trials of potential PD therapies have ended unsuccessfully even though some of the participants claim that the impact of the therapies under test has been positive for them. It is easy to put this down to "noise" in the data, or to issues of statistical significance, or to the placebo effect, but it may be wrong to do so. We all come with different genetics, different environments, different medical histories, etc.  So we will not all react in the same way to a therapy. I am concerned that therapies which are good for some people with Parkinson's are not being identified because of this one size fits all strategy.

The correct level of running trials should be the individual. What I am interested in is whether a  therapy works for me. A therapy that cures me is still a cure even if it does not cure anybody else.

John


#2

Let's look at how this would work.

You would start with a baseline measurement of your condition and your rate of progression of the disease.

Working with your medical team, you would select from a menu of existing therapies and candidate therapies (that is therapies that have been tested as safe, but which haven't been proven as efficacious). You would continue to measure your condition, probably on at least a daily basis.

What outcomes might there be? Well, your condition might continue to deteriorate at least as fast as before. In this case, you would stop using the therapy, and go on to consider other therapies. (Note that in this case you are saying that the therapy hasn't worked for you, and not that the therapy doesn't work full stop.) Alternatively, your condition may improve. In this case you would continue with the therapy, but continue to take frequent measurements. (Note that in this case you are saying that the therapy is working for you, and not that the therapy works full stop.) If later on results got worse (perhaps it was a placebo), you would discontinue the therapy. 

All data would be open so other people can see whether the therapy has worked for you and others. They would use this information to help make their decision on whether to try this therapy.

Crucial to this approach is good measurement and open data. The reward is a system that learns how best to treat people in a personal way and that accelerates the introduction of focused therapies.

John