The main strategic approaches of Huntington's disease research can be summarized as follows:
1 – Reducing the production of the pathologically mutated protein (mHTT).. Since the disease is caused by a single dominant gene that contains the code of the toxic protein, attempts are made to SILENCE this gene by inhibiting its activity, through sophisticated technologies and the use of drugs called Anti Sense Oligonucleotides (ASO's), aimed at blocking the production of messenger RNA that, produced on the mold of the mutated gene, orders the cells to produce the mHTT. The experimentation is currently limited to the animal model, but the collaboration between ISIS (owner of the ASO proprietary technology) and the pharmaceutical giant Roche gives hope for an early start of experimentation in humans;
2 – Improving the ability of cells to survive the harmful effects of mHTT, or performing a dopaminergic stabilization to mitigate symptoms. The most interesting molecule for this purpose is - to date - pridopidine, on which a phase II trial is underway involving 29 centers in Europe and 22 in the United States;
3 – Implementing other 'neuronal regeneration' strategies through methods of cellular and molecular biology able to exert neuroprotection interfering with the basic mechanisms of the disease.
It is clear that a fundamental role is played by TRASLATIONAL RESEARCH, that is, the research capable to transfer discoveries from the laboratory to the clinic.