Background: Neuropathic pain (NP) is an important public health problem and despite
recent progress in the understanding, diagnosis, pathophysiological mechanisms and the treatment
of NP, many patients remain refractory to pharmacotherapy.
Objective: Currently used drugs have limited efficacy and dose-limiting adverse effects, and thus
there is a substantial need for further development of novel medications for its treatment. Alternatively,
drugs approved for use in diseases other than NP can be applied as experimental for NP conditions.
This paper covers advances in the field of NP treatment.
Results: The prime focus of this paper is on drugs with well-established pharmacological activity
whose current therapeutic applications are distinct from NP. These drugs could be a potential novel
treatment of NP. Data from preclinical studies and clinical trials on these experimental drugs are
presented. The development of advanced methods of genomics enabled to propose new targets for
drugs which could be effective in the NP treatment.
Conclusion: Experimental drugs for NP can be a treatment option which should be tailor-made for
each individual on the basis of pain features, previous therapies, associated clinical conditions, recurrence
of pain, adverse effects, contraindications and patients’ preferences. At present, there are
only some agents which may have potential as novel treatments. Increasing knowledge about
mechanisms underlying NP, mechanisms of drug action, as well as available data from preclinical
and clinical studies make botulinum toxin A, minocycline, ambroxol, statins and PPAR agonists
(ATx086001) promising potential future treatment options.