Perspectives of neuro–therapeutics : Investigator–guided clinical trial and the role of academism

Abstract

The Japanese POEMS syndrome with Thalidomide (J–POST) Trial is a phase II/III multicenter, double–blinded, randomized, controlled trial that aims to evaluate the efficacy and safety of a treatment with thalidomide in POEMS (crow–Fukase) syndrome. In this editorial, thoughts, opinions, and efforts to perform this investigator–lead clinical trial are described. A clinical trials are tough work, requiring a large amount of time, effort, and money, but it also give a great achievement for patients with intractable disease, and doctors and medical staffs involved in the trial. The advantage for the investigators includes expanding motivation for better and update understanding of the disease, as well as growing as a physician. The idea for novel treatments is more likely to be arising from young neurologists. Definitely this approach is necessary for rare diseases. The Japanese Society of Neurological Therapeutics should play an important role in the spread of clinical trial with novel treatments.