This is a significant advance for people with Charcot-Marie-Tooth disease, which currently has no treatment. The FDA recently gave PXT3003 its “fast track” designation, which speeds the development of drugs for which there is an unmet medical need.

PXT3003 is a combination of three familiar drugs, naltrexone (an opioid receptor blocker), baclofen (a muscle relaxant), and sorbitol (an alcohol sugar). The how and why of this combination of drugs is not well-understood at present. The manufacturer Pharnext says there are “multiple main mechanisms of action” that improve nerve, muscle and immune cells.



In other words, research on existing drugs with known risk profiles has led to a novel treatment. Ordinarily, the use of an opioid and a muscle relaxant is regarded as clinically inadvisable and is actively counseled against in many prescribing guidelines.

Benzodiazepine Research

A similar outcome is occurring with long-term benzodiazepine therapy in congestive heart failure (CHF). An editorial in Psychotherapy and Psychosomatics reported that low-to-moderate doses of benzodiazepines “seem to be helpful in silent myocardial ischemia, angina, essential hypertension, and CHF, especially in patients with comorbid anxiety.”



This builds on research from Taiwan in 2014 showing that anti-anxiety medications are “associated with a decreased risk of cardiac mortality and heart failure hospitalization in patients after a new myocardial infarction.”



Long-term benzodiazepine therapy is already seen as important in the treatment of rapid eye movement sleep behavior disorder, a condition in which causes people to act out vivid and violent dreams, often injuring themselves or bed partners. Low-dose clonazepam therapy for months or even years turns out to be a highly effective treatment.



In the same fashion, benzodiazepines are used to treat stiff-person syndrome, a rare neurological disorder that causes extreme muscle rigidly and spasms that can make walking impossible. According to the National Institutes of Health, therapy to treat stiff-person syndrome includes “anti-anxiety drugs, muscle relaxants, anti-convulsants, and pain relievers.”

‘Political Interference’ in Medicine