Does the pursuit of scientific excellence serve or hamper translational medical research: an historical perspective from hematological malignancies

Chris M Bunce*, Farhat L Khanim, Mark T Drayson

*Corresponding author for this work

Research output: Contribution to journalReview articlepeer-review

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Abstract

Despite enormous global investment, translational medical research faces considerable challenges and patients, and their doctors are frequently frustrated by the apparent lack of research activity or progress. Understanding the factors that prevent innovative research discoveries from making it to clinical trials is a multifaceted problem. However, one question that must be addressed is whether the nature of current research activity and the factors that influence the conduct of pre-clinical research, permit, or hamper the timely progression of laboratory-based observations to proof of concept (PoC) clinical trials. Inherent in this question is to what extent a deep mechanistic understanding of a potential new therapy is required before commencing PoC studies, and whether patients are better served when mechanistic and clinical studies progress side by side rather than in a more linear fashion. Here we address these questions by revisiting the historical development of hugely impactful and paradigm-changing innovations in the treatment of hematological cancers. First, we compare the history and route to clinical PoC, of two molecularly-targeted therapies that are BCR:ABL inhibitors in chronic myeloid leukaemia and all-trans retinoic acid (ATRA) in acute promyelocytic leukaemia (APL). We then discuss the history of arsenic trioxide as additional APL therapy, and the repurposing of thalidomide as effective multiple myeloma therapy. These stories have surprising elements of commonality that demand debate about the modern-day hard and soft governance of medical research and whether these processes appropriately align the priorities of advancing scientific knowledge and the need of patients.

Original languageEnglish
Article number142
JournalBlood Cancer Journal
Volume12
Issue number10
DOIs
Publication statusPublished - 7 Oct 2022

Bibliographical note

© 2022. The Author(s).

Keywords

  • Arsenic Trioxide/therapeutic use
  • Arsenicals/pharmacology
  • Hematologic Neoplasms/drug therapy
  • Humans
  • Leukemia, Promyelocytic, Acute/drug therapy
  • Oxides/pharmacology
  • Thalidomide/therapeutic use
  • Translational Research, Biomedical
  • Tretinoin

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