A pretty cool development here for fans of gene therapy. The essence of gene therapy is that if a person is born with a defective copy of some gene you can insert a good copy into a critical set of cells in that person and restore the broken functionality. In this case the researchers injected therapeutic viruses into 12 patients brains and saw therapeutic benefits for parkinsons sufferers.
The reason you use viruses for gene therapy is that a virus has evolved to attach to a cell and inject it’s own genes to force the cell to make copies of the virus. It’s possible to gut the virus and insert your own genes of interest, so when the virus infects a cell, all it does is inject your genes. There’s no replication, no spreading viruses, etc. Gene therapy has had a pretty rocky history though, and not a lot of medical success.
I’ll also point out that this is just a phase 1 clinical trial. Phase 1 is just about testing for safety, not testing for efficacy.
Like all phase 1 studies, this one was primarily focused on gauging the safety of the technique,” Dr. Kaplitt says. “And on that count it succeeded brilliantly: We saw no adverse events related to the treatment, no immunological changes or infections over the year of the study, no imaging evidence of toxicity whatsoever.
In this case they did see significant improvement in the patients, but that could break down in a larger study. Many, many new drugs die in phase 2 trials. But it’s still exciting news for parkinson’s patients and gene therapy hopefuls.
Another quote:
Will these remarkable improvements persist? Only longer follow-up can tell, but prior studies in animals, including primates, suggest that the transplanted gene does stay active for years, Dr. Kaplitt says.
how can i and what are the criteria for volounteering for clinical trials into gene therapy and PD
Hi Martin,
If you want to participate in a drug trial you should talk to your doctor. Let them know that you are interested in learning more about the opportunities and risks associated and they should be able to put you in touch with an appropriate group. You may have better luck if you see a physician at a university, but I suspect that any medical specialist (for example, a neurologist) would be able to get you into a trial if that was your desire. Taking part in a drug trial is something that you should bring up and explore with your doctor, who knows your particular case. For example, if you have one of the many “PD-like” disorders then a drug focused on PD would not help you, and would only expose you to danger. Your current doctor would be able to direct you to the right drug trial.
You might be interested in this short article I wrote earlier describing just what “clinical trials” actually means.
And I’ll also say that the point of my post was that gene therapy is unproven and risky. So have a serious conversation with your doctor about the risks associated with the trial compared to the risks and benefits of your current treatment.
Feel free to send me an email using the contact form on my site if you’d like to continue to discuss this privately.