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Interviewer: Doctor Gitler, thank you for sparing the time today.
We're going to be talking about Amyotrophic Lateral Sclerosis, ALS.
I'm going to be asking your opinions on therapeutic interventions,
the conditions necessary to decide whether and
when to attempt the various approaches that can be considered,
and the likely timescale for commencing development of candidate interventions.
So, just to start,
what do you think of the opportunities presented by recent research?
Prof. Gitler: There have been many exciting new developments in ALS research,
and I think the most exciting new ones focus on human genetics.
So it's the discovery of mutations in genes that cause ALS.
Some of those discoveries have directly led to therapeutic strategies.
Two specific examples, the first one is the discovery in
1993 of mutations in the SOD1 gene as a cause of familial ALS,
and much research over the years has provided evidence that what those mutations
do is to cause some sort of toxicity to the protein that SOD1 gene encodes.
That realization has suggested that therapies that lower
the levels of the mutant SOD1 protein could have therapeutic efficacy,
and evidence for that was provided in animal models,
and that provided the basis for clinical trials using
antisense oligonucleotides to down-regulate the expression of mutant SOD1.