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. 2024;11(5):1085-1093.
doi: 10.3233/JND-240033.

Clinician Perspectives of Gene Therapy as a Treatment Option for Duchenne Muscular Dystrophy

Heidi Cope  1 , Ryan Fischer  2 , Emma Heslop  3 , Megan McNiff  3 , Alexandra Johnson  4 , Eric Camino  2 , Brian Denger  2 , Niki Armstrong  2 , Sejal Thakrar  4   5 , Alison Bateman-House  6 , Katherine L Beaverson  7 , Ione O C Woollacott  8 , Dawn Phillips  9 , Vivian Fernandez  9 , Annie Ganot  10 , Roxana Donisa-Dreghici  10 , Carol Mansfield  1 , Holly Peay  1
Affiliations

Clinician Perspectives of Gene Therapy as a Treatment Option for Duchenne Muscular Dystrophy

Heidi Cope et al. J Neuromuscul Dis. 2024.

Abstract

Background: Duchenne muscular dystrophy (DMD) is a progressive, life-limiting, neuromuscular disorder. Clinicians play an important role in informing families about therapy options, including approved gene therapies and clinical trials of unapproved therapies.

Objective: This study aimed to understand the perspectives of clinicians about gene therapy for DMD, which has not previously been studied.

Methods: We conducted interviews with specialist clinicians treating patients with DMD in the United States (n = 8) and United Kingdom (n = 8). Interviews were completed in 2022, before any approved gene therapies, to gain insight into barriers and facilitators to implementing gene therapy and educational needs of clinicians.

Results: Most respondents expressed cautious optimism about gene therapy. Responses varied regarding potential benefits with most expecting delayed progression and duration of benefit (1 year to lifelong). Concern about anticipated risks also varied; types of anticipated risks included immunological reactions, liver toxicity, and cardiac or renal dysfunction. Clinicians generally, but not uniformly, understood that gene therapy for DMD would not be curative. Most reported needing demonstrable clinical benefit to justify treatment-related risks.

Conclusions: Our data demonstrate variability in knowledge and attitudes about gene therapy among clinicians who follow patients with DMD. As our knowledge base about DMD gene therapy grows, clinician education is vital to ensuring that accurate information is communicated to patients and families.

Keywords: Patient preference; clinical trials; clinician perspectives; duchene muscular dystrophy; gene therapy.

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Conflict of interest statement

KB is an employee of Pfizer Inc. IOCW is an employee of Pfizer Ltd. KB and IOCW were representatives of Pfizer on the Study Advisory Board and provided input into the design of the study, development of study materials and review of the manuscript. DP and VF are employees of Regenxbio Ltd. DP and VF were representatives of Regenxbio on the Study Advisory Board and provided input into the design of the study, development of study materials and review of the manuscript. AG and RDD are employees of Solid Biosciences Inc. AG and RDD were representatives of Solid Biosciences Inc. on the Study Advisory Board and provided input into the design of the study, development of study materials and review of the manuscript. There are no other conflicts of interest reported by authors.

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