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REVIEW article

Front. Physiol.
Sec. Striated Muscle Physiology
Volume 15 - 2024 | doi: 10.3389/fphys.2024.1496569
This article is part of the Research Topic State-of-the-art muscle physiology research: from single molecules and cells to living organisms View all 3 articles

From amoeboid myosin to unique targeted medicines for a genetic cardiac disease

Provisionally accepted
  • 1 Stanford University, Stanford, United States
  • 2 Stanford Bio-X, Stanford University, Stanford, California, United States
  • 3 School of Medicine, Stanford University, Stanford, California, United States
  • 4 Stanford Cardiovascular Institute, School of Medicine, Stanford University, Stanfordd, California, United States

The final, formatted version of the article will be published soon.

    The importance of fundamental basic research in the quest for much needed clinical treatments is a story that constantly must be retold. Funding of basic science in the USA by the National Institutes of Health and other agencies is provided under the assumption that fundamental research eventually will lead to improvements in health care worldwide. Understanding how basic research is connected to clinical developments is important, but just part of the story. Many basic science discoveries never see the light of day in a clinical setting because academic scientists are not interested in or do not have the inclination and/or support for entering the world of biotechnology. Even if the interest and inclination are there, often the unknowns about how to enter that world inhibit taking the initial step. Young investigators often ask me how I incorporated biotech opportunities into my otherwise purely academic research endeavors. Here I tell the story of the foundational basic science and early events of my career that led to forming the biotech companies responsible for the development of unique cardiac drugs, including mavacamten, a first in class human β-cardiac myosin inhibitor that is changing the lives of hypertrophic cardiomyopathy patients.

    Keywords: Myosin, Translational research, Hypertrophic Cardiomyopathy, Mavacamten, Aficamten

    Received: 14 Sep 2024; Accepted: 14 Oct 2024.

    Copyright: © 2024 Spudich. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.

    * Correspondence: James A. Spudich, Stanford University, Stanford, United States

    Disclaimer: All claims expressed in this article are solely those of the authors and do not necessarily represent those of their affiliated organizations, or those of the publisher, the editors and the reviewers. Any product that may be evaluated in this article or claim that may be made by its manufacturer is not guaranteed or endorsed by the publisher.