Gene Modification Therapies: Views of Parents of People with Down syndrome
This paper was co-authored by PaSAGE team members Michie and Allyse.
The goal of this study was to find out how parents of people with Down syndrome (DS) feel about different treatments. The team asked parents about treatment situations. Two of the scenarios described treatments that are currently available. The other three were about hypothetical future treatments. The first situation was surgery on the baby during pregnancy to fix a block in its bladder. The second situation was a shot during pregnancy to silence the extra copy of the 21st chromosome. The third situation was surgery on a new baby with DS to repair a heart flaw. The fourth situation was a daily pill to improve memory and attention in kids with DS. However, the pill might alter the child’s personality. The last situation was a vaccine that could decrease risk of DS-related Alzheimer’s disease. However, the vaccine could cause seizures. Situations 1 and 3 are currently available. Situations 2, 4, and 5 are not.
Over half of the parents responded positively to all five situations. Parents had different views on how DS would impact their children and families. These different opinions influenced how much they supported two types of treatments. These treatments were those done before birth and ones done during childhood to improve child cognition. Scenarios where the treatment was for an adult with DS were not affected by these parental views.
This study showed that is important to talk to patient communities. Future treatments cannot succeed without their support. Many parents of people with DS showed support for future genetic therapies. However, more research into how these parents feel about potential risks and benefits of these treatments.