The 'gene silencing' trial for HD patients is about to start.
This trial represents a very preliminary stage of a very complex approach to cure the disease by reducing the toxic effects of the defective gene. How? By blocking the production of the ' sick ' protein huntingtin. We are not yet at a stage of proximity to a cure, but only in a very initial stage that will test the drug safety ant tolerability.
For this reason, only few patients from few centers (one in North America and others in Europe) will be involved.
We believe that it is appropriate to mantain a cautious attitude. In fact, the defective gene will be only partially turned off and it will not be turned off only the defective gene, but also the ' healthy ' one, whose functions are necessary for the proper human physiology. Although it is believed that the partial shutdown of the normal gene (along with that bad ) should not be a problem, there is the possibility that unpredictable side effects appear.
The distribution of the drug within the nervous system may not be sufficient to ensure future effectiveness, nor we can predict how long the drug will maintain its effect, once injected into the cerebrospinal fluid . Patients who undergo this trial will therefore accept an injection of the drug in the cerebrospinal fluid (the fluid that surrounds the brain and spinal cord ) and the risk related to the unknowns of this strategy .
To summarize: this is a Phase I Study, aimed at evaluating the safety and tolerability (not yet the effectiveness) of the drug in a small number of patients (not more than 36) at an early stage of the disease.
We do hope this first attempt will be successfull and will update you as soon as we will have updates.
The involved Centres are the following:
In Canada: University of British Columbia Vancouver
In Germania: Ruhr-University of Bochum; Ulm University Hospital
In Inghilterra: Cambridge University Hospital; University College London;University of Manchester, St. Mary's Hospital
At present, none of them has still started to recruit patients.