What would it cost to synthesize 5’-CTCAGtaacattgacACCAC-3’ reported in Sustained Therapeutic Reversal of Huntington's Disease by Transient Repression of Huntingtin Synthesis?
A confluence of events leads me to ask this question:
- A few weeks ago the human safety trial reported "no surprises". As expected from the extensive animal trials, there are no deleterious effects at therapeutic doses. (Caveat: For any allele non-specific gene silencing, it is possible to over-dose and so-reduce the wild-type expression to the point that you get pathology and this is no exception. However, contrary to what many "experts" will tell you, there _is_ an acceptable level of wild-type silencing to achieve net therapeutic benefit.)
- A couple of months ago I received a response from the clinical trial authorities to my question, "How much money would it take to service an FDA compassionate use waiver without interfering with the clinical trial pipeline?" The answer was "no amount of money", which is not very credible.
- My wife did not qualify for the clinical trial at the time it started accepting applicants last year because she was already too far gone. She will not qualify for any further stages of clinical trial as a result. They do _not_ want subjects that are in the advanced, life threatening, stages of HD for these clinical trials.
- My wife has, over the last year, gone from being intelligible verbally and able to walk, dress and eat unassisted, to losing all of those capacities, along with her higher mental faculties. This is an accelerating decline. She will very likely not live long enough to benefit from the drug when it is released and even if she did, the damage would be so great by that time that death may be welcome.
- She has already suggested that she may start refusing food at some point and asked me about funeral arrangements.
- My US Senator (from a town 15 minutes away) has given me the cell phone number for her staffer that is liaison with the FDA to obtain a compassionate use waiver in the event that my wife stops eating or otherwise faces imminent hospice.
If I had a dollar amount for the synthesis, I might have some ammunition to fight for my wife's life in response to the rather questionable response from the clinical trial.
