Ignacio Mata, PhD, on the Research Needed to Bring Gene Therapy to PD


The associate professor of neurology at the Cleveland Clinic Lerner Institute discussed the challenges that will need to be overcome to apply gene therapy to a complex neurological disorder like PD.

This is the second part of an interview with Ignacio Mata, PhD. For the first part, click here.

“I'm a big believer that there is a big chance that some of the things that have been tried in the past might actually not be completely wasteful... I think the problem that we've run into for the past maybe 10 years or so is that we are grouping patients with PD as 1 single group. We know that this is not true—biologically they're very different, even the way that they present the disease. I think that also has been a barrier perhaps in finding things that work for certain subgroups of people.”

Thus far, gene therapy approaches have been most successful in targeting disorders that are caused by mutations in a single gene. Despite this, even targeting well-understood monogenic neurological disorders with gene therapy has proven challenging. As such, using gene therapy to address a highly complex neurological disorder like Parkinson disease (PD), in which several thousand genes have been implicated across hundreds of disease variants, poses a far greater challenge. That said, the great unmet need for new treatment options in PD has compelled a number of undaunted companies and academic institutions to pursue the development of gene therapy approaches in the disease regardless.

In light of the ongoing interest in genomic medicines for PD, CGTLive® recently reached out to Ignacio Mata, PhD, an associate professor of neurology at the Cleveland Clinic Lerner Institute, to get his insight on the obstacles that will need to be overcome to make gene therapy successful in PD. Mata shared his view that trying to target a specific dysfunctional gene implicated in PD to be “fixed” or replaced is unlikely to be the best approach; instead early success in preclinical research has largely come from inserting genes that simply provide sustained expression of proteins, such as neurotrophic factors, that can help dopaminergic neurons stay alive. Mata also emphasized the need for innovative delivery technology to target gene therapies to the brain and the need to determine appropriate timing in terms of disease progression to deliver such treatments. He also noted that categorizing patients with PD into different subgroups based on the affected pathway behind their disease manifestation may help future research programs to find ideal gene therapy targets for each subgroup.

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