A new UCLA Health study will investigate whether a promising treatment called highly effective modulator therapy (HEMT) can help with a serious condition suffered by young children with cystic fibrosis (CF).
The study, led by Dr. Daniel Beswick, associate professor-in-residence of otolaryngology at the UCLA David Geffen School of Medicine and head and neck attending surgeon at UCLA Health, aims to characterize chronic rhinosinusitis (CRS), or sinus health, and olfactory dysfunction (OD), or sense of smell, in children with cystic fibrosis (CF).
It also tests the hypothesis that HEMT improves CRS and OD in CF. While researchers say that HEMT improves pulmonary and extra-pulmonary disease, its impact on sinusitis remains understudied. This research aims to understand the nature of CRS and OD in CF and to investigate whether HEMT can address these conditions.
“It was important for us to pursue this study, given the significant impact of these comorbidities on the quality of life in young children,” said Beswick. “While we believe it’s crucial to continue investigating HEMT’s effects in young children with CF, we believe this study will help develop targeted interventions in the future.”
The observational study will enroll 80 CF patients aged 2 to 8. Participants will be divided into two groups: Those receiving HEMT and those not on HEMT. The study will compare outcomes before and after the initiation of HEMT between these two groups.
Previous studies indicate that HEMT has significantly affected the lives of many individuals with CF. Researchers believe these medications can substantially enhance cystic fibrosis transmembrane conductance regulator (CFTR) protein function, body mass index, and quality of life, particularly for children two and younger.
Researchers hope the results of this study will improve prognostication and highlight the importance of the treatment of CRS and OD early in life, whether through HEMT or other interventions. The findings may also provide insights into the potential effects of future treatments, such as gene therapy, and encourage timely therapy initiation for certain pediatric patients.
The research underscores the need for continued investigation into the impact of HEMT and supports future efforts to develop tailored treatments.
The $2.3 million study, funded by the NIH, is active and will continue until June 30, 2029.
