Thursday | February 17, 2022 | 8:00 - 10:00 PM EST
Dopamine Gene Therapy: An Update w/ Matthew Klein, MD, MS, FACS ; Chief Operating Officer, PTC Therapeutics Inc.
A constant refrain is "we're short of dopamine" (the brain-chemical messenger).
It's a mantra -- it helps us feel situated -- and yes: it's simplistic. But, in all this, maybe there's a kernel of truth.
What makes this important? An effort, elsewhere, to boost the impact of this crucial neurotransmitter.
How? Through gene therapy. Specifically, the infusion of a piece of DNA that tells the brain to make an enzyme -- aromatic amino-acid decarboxylase, or AADC -- that controls the synthesis of dopamine itself.
On February 17, we'll learn about AADC deficiency: a rare, inherited condition caused by the absence of this particular enzyme. One that destroys motor function, making it impossible to sit or stand or walk; and that can shorten the lifespan of the children it afflicts.
We'll learn, too, of the task of easing this with a quantity of the AADC gene sent to the middle of the brain. It's a treatment that may have some adaptability and some staying power.
And -- while the headline is the astounding motor progress seen in certain cases -- there's a hint that something equally rare has happened on another front. Since the procedure, some of these kids have become more successful learners; with cognitive windfalls reported in at least two countries.
At the moment, three research teams are pursuing the matter, including a New Jersey-based biotech company, PTC Therapeutics Inc. And it is PTC's chief operating officer, surgeon Matthew Klein, who will join us on the 17th.
Dr. Klein earned a medical degree from Yale. Later, he taught restorative burn surgery at the University of Washington.
Still later, he led the BioElectron Technology Corporation, which focused on diseases of mitochondria and oxidative stress. In 2019, BioElectron was absorbed by PTC, which continues its mission.
Dr. Klein notes: "It's challenging, but we're excited and embrace that we're a company that's pioneered new therapies, new techniques and new technologies. We're really excited to be where we're at with this therapy and really look forward to bringing it to as many patients that can benefit as possible."
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