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Chief Scientific Officer of the CMT Research Foundation talks about being cognizant of the blood-nerve barrier when treating patients and developing therapies.
Charcot-Marie-Tooth (CMT) may be the most common condition involving the peripheral nervous system. However, according to the CMT Research Foundation, it's estimated that 150,000 Americans and 3 million people across the globe live with it, making it a rare disease.
In an interview with HCPLive®, Keith Fargo, PhD, Chief Scientific Officer, CMT Research Foundation, described CMT as an umbrella term for a number of different diseases that would possibly be considered separate diseases if they were just discovered today.
"So, one way to think about Charcot-Marie-Tooth disease, is that it encompasses any hereditary peripheral neuropathy," he said, "that affects both the motor axons, and the sensory axons in the peripheral nerves."
As muscle weakness progresses in patients with CMT, symptoms typically become apparent in adolescence or early adulthood. Although, it can present in men and women of all ages, races, and ethnicities.
Fargo addressed some of the obstacles related to the field of CMT. One of the main challenges, that is also being investigated in fields like oncology, is developing therapeutics that can "sneak" past the blood-nerve barrier without flooding the entire body and potentially causing toxicity.
The neurons that give rise to long axons are the targets, and trying to target the Schwann cells that myelinate those axons is even more difficult, he said.
"A lot of this work is being done actually, in the oncology field, where people are trying to target very specific kinds of cancerous cells to kill while leaving the rest of the cells alone," Fargo explained. "And so we're trying to take some of the technologies that have been developed there and potentially apply those to the problem of drug delivery and CMT."
According to Fargo, that's the number one challenge for CMT right now, but when looking ahead, gene editing is a realistic future for CMT.
Currently, there are no cures for CMT, but in terms of treatments that would slow down, stop, or reverse the course of the disease itself–or prevent the disease from developing in people who have these genetic differences–right now, progress is being made, Fargo said.
He believes that gene therapy could change the treatment landscape for CMT. The CMT Research Foundation focuses a great deal of funds and research on genetic therapies while still deliberating some programs to look at traditional small molecules as well.
"While we wait for the cure, we want to make sure that we're also doing things that we can do to help people have a better quality of life through therapeutics as well," he said.
Another great barrier is just spreading awareness and gaining attention for this rare disease. Fargo emphasized the amount of activity happening in the biotech field, but there's not enough awareness of CMT among the people in biotech.
Developing a therapeutic for a rare disease does have the tremendous benefit. He explained that there's potential for biotech or pharmaceuticals companies to receive a priority review voucher from the US Food and Drug Administration (FDA), which has a market rate of around $100 million.
"But of the rare diseases, it's one of the most prevalent, and it's monogenic, so we know what it is we have to overcome," Fargo said. "So, that's part of the role of the CMT Research Foundation, it's kind of serving as an evangelist, if you will, for the disease."
"It's our job to educate them and help them understand why it would be beneficial for them, as well as for CMT patients, for them to work in CMT."