Meet the 2020 Scholars

"What do you believe is the greatest unaddressed patient need in the CF community, and if you could, how would you try to fix it? Who would you partner with (e.g. public figure, organization) to help fix the issue and why?"

Despite great CF achievements, significant challenges remain because the complex etiology and pathogenesis of the disease aren’t fully understood. So, to me the greatest unaddressed need is for increased data-driven, computational-research that leverages innovations, like Artificial Intelligence (AI), to enhance our insights into CF, thus, enabling accelerated development of novel therapies and the dissemination of the results so patients can make risk-informed decisions about their treatment options. To address this issue and my reasoning are as follows: First, I’d develop public-private research partnerships (e.g., with AbbVie and the CF Foundation) that further the understanding of the complex CF etiology and pathogenesis, thus, enabling the replacement of unreliable conventional testing methods with more personalized, data-driven diagnostic indicators for enhancing treatment decision-making. For example, I’d leverage AI to analyze a vast amount of CF data to accurately predict the personalized lung transplant survival profile of patients with terminal respiratory illness, instead of using current practices which fail to account for heterogeneity in CF patient survivability. Second, I’d collaborate with gene therapy researchers (e.g., at Vertex Pharmaceuticals and the University of Iowa Gene Therapy Center) to implement innovations, like AI, to prevent off-target mutations, thus, providing safer CF reconstitution. Third, I’d seek collaborations to use efficient computational methods to investigate the efficacy of combining promising CFTR-modulators, to determine groupings that improve patient outcomes. Finally, I’d partner with CF advocates, pharmaceutical leaders, clinicians, and public figures, like the NFL’s CF celebrity Boomer Esiason, to disseminate the latest information so patients can make risk-informed decisions about their treatment options.