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Updated: Jun 19, 2026

Development of Recombinant Proteins to Treat Chronic Pain
Published on: April 11, 2018
A clinical trial of gene therapy for chronic pain
Darren Wolfe1, James Wechuck, David Krisky
1Diamyd Inc, Pittsburgh, PA, USA.
Abstract:
The first human trial of gene therapy for chronic pain, a phase 1 study of a nonreplicating herpes simplex virus (HSV)-based vector engineered to express preproenkephalin in patients with intractable pain from cancer, began enrolling subjects in December 2008. In this article, we describe the rationale underlying this potential approach to treatment of pain, the preclinical animal data in support of this approach, the design of the study, and studies with additional HSV-based vectors that may be used to develop treatment for other types of pain.
Insights
Gene therapy using a herpes simplex virus (HSV) vector to deliver pain-reducing genes is being tested in humans. This first trial offers a new approach for managing intractable cancer pain.
Area of Science:
- Neuroscience
- Gene Therapy
- Pain Management
Background:
- Intractable cancer pain presents a significant clinical challenge.
- Current pain management strategies have limitations.
- Gene therapy offers a novel therapeutic avenue for pain control.
Purpose of the Study:
- To describe the rationale and preclinical data for a gene therapy approach to chronic pain.
- To detail the design of the first human trial using a herpes simplex virus (HSV)-based vector.
- To explore the potential of HSV-based vectors for treating various pain conditions.
Main Methods:
- Phase 1 clinical trial enrollment began in December 2008.
- Utilized a nonreplicating herpes simplex virus (HSV)-based vector.
- Vector engineered to express preproenkephalin for pain relief.
Main Results:
- The study focuses on the initial human trial design and rationale.
- Preclinical animal data supports the efficacy of this gene therapy approach.
- The trial investigates gene therapy for intractable cancer pain.
Conclusions:
- The first human trial of gene therapy for chronic pain has commenced.
- HSV-based vectors show promise for developing new pain treatments.
- This approach may offer a new strategy for managing intractable pain.
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