Theranostic Innovation by Humane N-of-One Cancer Care in Real-World Patients.

Abstract

Patients with relapsed or refractory metastatic cancer unresponsive to standard therapies have motivated nuclear physicians to develop innovative radioligands, precisely targeted to tumor molecular receptors, for effective treatment of specific advanced malignancies. Individual practitioners in departments of nuclear medicine across the world have performed first-in-human studies on compassionate patient usage N-of-One protocols. These physician-sponsored studies then evolved into early-phase clinical trials and obtained real-world data to demonstrate real-world evidence of effectiveness in prolonging survival and enhancing quality of life of many so-called "End-Stage" cancer patients. Virtually all the therapeutic radiopharmaceuticals in current clinical oncology have been discovered and developed into effective specific treatments of targetable cancers by individual doctors in the course of their hospital practice. Pharma industry was not involved until many years later when performance of mandated Phase 3 randomized controlled trials became necessary to achieve regulatory agency approval. This article traces the history of several novel theranostic agents developed from compassionate N-of-One studies by hospital physicians over the past 36 years. It acknowledges the collegiality and collaboration of individual nuclear medicine specialists, worldwide, in pioneering effective humane therapy of particular advanced cancers unresponsive to conventional treatments.