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?"

My brother and I have been very fortunate over the past couple of years to be study participants in the medical trial of the newest CF modulator drugs. This medicine rescues CFTR protein function and makes the mucus in the lungs more fluid, allowing for far easier breathing. This medicine has been truly life-changing for both of us. Currently, about ninety percent of the CF Community benefits from one of these drugs, but that leaves ten percent of patients without an effective treatment that hits at the cause of CF vs. just treating the symptoms. These people have the greatest unaddressed need in the CF community today. I am aware of one approach to help this group, namely to develop new modulator drugs that are more effective and extend existing drugs to cover people with rare genetic mutations in this ten percent group. However, these drugs may still not be effective because some of these patients do not produce the CFTR protein at all. For these people, one option that looks promising would be to stimulate other proteins that work like CFTR. A second option would be gene therapy, which would rewrite mutant genes, like CFTR in CF patients, so that their sequence is corrected. To help the cause of aiding these patients, I would partner with the CF Foundation in order to get the funding and advocacy to continue this research. I would also partner with pharmaceutical companies and colleges/universities that are currently doing this research.