John Pollman
Member
- Messages
- 1,293
- Location
- Rochester Hills, MI
Last week was a good but tiring week. The conclusion of the first stage of my clinical trial for a new drug, and the start of stage two (another two years), at the National Institutes of Health in Bethesda, MD. Then four days with my NTSAD family in Reston, VA. for our annual family conference. It was wonderful!
There was some exciting news at the conference. With that, I have a HUGE decision to make sometime in the next couple of years. ( I'll have to finish my current clinical trial first ) We are getting close to human trials of gene therapy for Late Onset Tay Sachs and Sandhoff diseases. I have been participating in assessments and tests that lead to this for over eight years. We have worked hard for this and have been looking forward to it for a long time.
They are currently preparing the AAVs to start the non-human trials. ( monkey ) If those go well, it's on to human trials. The thing about it is that I really never considered HOW they do gene therapy. It's a one-time treatment, but it involves two injections directly into the center of the brain. They are saying it's safe and fairly routine. But for me, that's scary as hell! I am 59 years old and have spent the last ten years dreaming and hoping for this. I feel that I've had a good life and owe it to the younger generation to try this in hopes that it will help them down the road. But I also feel that although my quality of life has been severely diminished because of this disease, I am happy and feel that I can continue to live with it and deal with it for the time I have left.
As I said, this is a HUGE decision, and I'm glad that I don't have to make it now. This isn't a cry for pity. I just need to put this down "on paper" so I can go back later and read through it again to help me make the decision. I guess it all comes down to a risk/reward assessment. It will be one of the toughest decisions of my life! I'm gonna have to work on putting as much as I can into the "courage bank" over the next two years.
There was some exciting news at the conference. With that, I have a HUGE decision to make sometime in the next couple of years. ( I'll have to finish my current clinical trial first ) We are getting close to human trials of gene therapy for Late Onset Tay Sachs and Sandhoff diseases. I have been participating in assessments and tests that lead to this for over eight years. We have worked hard for this and have been looking forward to it for a long time.
They are currently preparing the AAVs to start the non-human trials. ( monkey ) If those go well, it's on to human trials. The thing about it is that I really never considered HOW they do gene therapy. It's a one-time treatment, but it involves two injections directly into the center of the brain. They are saying it's safe and fairly routine. But for me, that's scary as hell! I am 59 years old and have spent the last ten years dreaming and hoping for this. I feel that I've had a good life and owe it to the younger generation to try this in hopes that it will help them down the road. But I also feel that although my quality of life has been severely diminished because of this disease, I am happy and feel that I can continue to live with it and deal with it for the time I have left.
As I said, this is a HUGE decision, and I'm glad that I don't have to make it now. This isn't a cry for pity. I just need to put this down "on paper" so I can go back later and read through it again to help me make the decision. I guess it all comes down to a risk/reward assessment. It will be one of the toughest decisions of my life! I'm gonna have to work on putting as much as I can into the "courage bank" over the next two years.
