"Is a cure in my sight?" Multi-stakeholder perspectives on phase I choroideremia gene transfer clinical trials.

ABSTRACT:

Purpose

Ocular gene transfer clinical trials are raising patient hopes for the treatment of choroideremia--a blinding degenerative retinopathy. Phase I choroideremia gene transfer trials necessitate communicating about the risks of harm and potential benefits with patients while avoiding the sensationalism that has historically undermined this field of translational medicine.

Methods

We conducted interviews between June 2011 and June 2012 with 6 choroideremia patient advocates, 20 patients, and 15 clinicians about their hopes for benefits, perceived risks of harm, and hopes for the time frame of clinical implementation of choroideremia gene transfer.

Results

Despite the safety focus of phase I trials, participants hoped for direct visual benefits with evident discrepa

SUBMITTER: Benjaminy S 

PROVIDER: S-EPMC4018498 | biostudies-literature | 2014 May

REPOSITORIES: biostudies-literature