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Some medical research aimed at curing ALS is centered around what they believe to be the cause, a specific gene mutation. There are several investigational therapies such as gene therapy that are now in the clinical trials phase. The study I have been participating in is doing exactly that and it is very exciting. They are targeting the SOD1 mutation and I believe the C9 mutation (I’ll know more specifics when I go in December). Dr. Benatar explained that gene therapy is the use of genetic instructions to produce a protein to treat a disorder caused by the mutated SOD1 gene. The first clinical trial (currently underway) is targeting people who have been diagnosed with ALS. There are several possibilities being discussed for how the second phase will look.
What do you think about gene therapy? Do think this research is where funding should be focused? What other investigational therapies do you think should be high priorities? Is anyone involved in gene therapy and if so, would you please share your experience?
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