On April 8, The Dutch BRAINmodel project organized a parent panel meeting on potential future gene therapies for STXBP1-related disorders (STXBP1-RD). The aim of this meeting was to explore the perspectives of European parents of children with STXBP1-RD on potential future gene therapies. Parents from seven European countries joined this online dialogue.
The meeting was organized by researchers from the Erasmus Medical Center in Rotterdam, within the context of the BRAINmodel project. At present, treatment options for children with STXBP1-RD are limited. However, several research groups worldwide are investigating potential future therapies, such as RNA and DNA therapies, that target the underlying causes of the disorder. Some of these therapies may be tested in clinical trials in the upcoming years, but most are still in very early investigational stages. At this stage, insight into the ethical perspectives of families with a child with STXBP1-RD is important to help guide research in directions that align with the needs, hopes, and concerns of these families.
A wide range of topics was discussed. Participants in our panel meeting hoped for several benefits from potential future therapies for both their child’s well-being and the quality of life for the family as a whole. They felt that there is especially a significant need for therapies that support children’s cognitive development and communication skills. Furthermore, the pace of drug development was discussed. Participants noted that ‘slow’ progress in drug development may lead to demotivation and skepticism in the STXBP1 community and some participants worried that therapies might be introduced ‘too late’ for their child to be able to benefit.
Participants in our panel meeting identified several challenges regarding potential future gene therapies. They were concerned about equal access to potential future – likely expensive – therapies, as these therapies, once they are approved, may not be automatically reimbursed in every country and thus not become available for all families. Furthermore, involving the child in decision-making about gene therapies was deemed important, yet challenging when children cannot verbally express their own preferences. Participants stressed the need for clear information about the safety risks and efficacy of potential future therapies to support them in making well-informed decisions about enrolling their children in clinical trials. Lastly, the diversity of parent perspectives on potential future therapies was highlighted, for instance, as parental preferences may differ depending on their children’s ages and symptoms.
The meeting will be analyzed, and results will be shared via the ESCO newsletter once they are available. We thank all participants for joining this meeting and for sharing their perspectives.
The BRAINmodel project is responsible for the article, “‘You constantly have to be switched on’: A qualitative interview study of parents of children with STXBP1-related disorders in the Netherlands.” You can read the full article by clicking the button below.