MedCity Influencers

A patient’s case for human experimentation

I received a heart breaking email recently. Frankly it made me cry. There are a lot of patient confidentiality details I must withhold but it is worth expressing this story. A care giver emailed me concerning a loved one he/she is taking care of (for ease of reading, this person will be female and the […]

I received a heart breaking email recently. Frankly it made me cry. There are a lot of patient confidentiality details I must withhold but it is worth expressing this story.

A care giver emailed me concerning a loved one he/she is taking care of (for ease of reading, this person will be female and the patient; her daughter). She had read about some research I published several years ago that might be used to treat her daughter. The clinical case she presented was in line with the research I am doing and that research had progressed a long way in the last few years. The key question this mother had was did we have a treatment yet.

We the answer is difficult. I have a compound that I know (am positive) will work but it is not ready for patients. I dare not give false hope to this woman but there are absolutely no treatments. She knows her daughter is not going to recover without treatment so there is desperation for her. The compound I have is experimental and not approved for humans with this condition. We do not know what dose will work or what side effects to watch out for. So we are not ready to go to clinical trials.

This is very sad.

Please remember I’ve blogged about some people experimenting on patients too soon, and I’m opposed to doing that here, but what do I tell this woman? By the time my research is ready for her daughter; it will be much too late. Doing nothing to help her is tearing me apart too. But if I were to say treat with compound X there are many questions I could not answer like: how much, for how long, what do we watch for, when will we know if it will work, how much will it cost and all the things people expect from a drug; but we do not know yet. Answering those questions is the life of a researcher like me but it does not help this woman.

I know this is hurting her and it is affecting me too. But I’m stuck. I can’t tell her about what we are studying but I also do not want to ignore her. I’m left with telling her that there is hope on the horizon, but it is not ready yet. I really do not know when it will be ready, but it will not likely be ready for her daughter. That is going to be hard to say.

Oh and did I mention that I have applied to the NIH 3 times to develop this treatment for patients and it has been rejected every time? I’m trying to get companies to work with us on this too, but right now we are funding this on our own. This woman’s email convinces me even more to push forward with this work if not for her for the next passionate mother.

Dr. Joseph Clark is a professor of neurology at the University of Cincinnati and the author of My Ambulance Education. He writes regularly at Josephfclark.com.

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