Pulmonary and pancreatic response to cystic fibrosis modulator therapy in young children

Active Project Grant Awarded Last action

Award ID
R01HL173340
Recipient
Awarding agency
Obligated
$4,993,617

Award facts

Assistance listing
93.837 Cardiovascular Diseases Research
Completion date 50% complete
Place of performance
Atlanta, GA 30322, USA

Award details come from USAspending.gov.

Description

As the agency reported it.

Abstract
Highly effective modulator therapy (HEMT) which dramatically improves pulmonary function has been developed for people with cystic fibrosis (CF). In 2019, the HEMT elexacaftor, tezacaftor, ivacaftor (ETI) became available for the 90% of CF teens and adults with at least one F508DEL mutation. Use of ETI is now widespread in the US and is standard of care. With chronic use of ETI, some people with CF (PWCF) have resolution of pulmonary symptoms and successfully wean burdensome daily pulmonary treatments. Unfortunately, the pulmonary response is variable and there are some PWCF who have no, or only mild, pulmonary improvement with ETI. Furthermore, there is no evidence of improvement in either exocrine or endocrine pancreatic function in CF teens and adults with ETI. This lack of a pancreatic response most likely is related to the fact that in CF the pancreas is largely replaced by lipofibrotic tissue by the teenage years, leaving little to no pancreatic tissue for ETI- mediated restoration of function. However, recent data show that younger CF children may have a significant pancreatic response to ETI. In april 2023, the FDA approved ETI for CF children aged 2 to 5 years. Our overall hypothesis is that in this young age group, chronic ETI will prevent progression of airway disease to bronchiectasis, prevent further destruction of pancreatic exocrine tissue, possibly permit repair/regeneration of exocrine function, and prevent CF related diabetes (CFRD), the most serious complication of pancreatic disease. In this project, we propose to study a cohort of 30 CF children aged 2- to 5-years old before and four years after starting ETI for clinical indications. We aim to: 1. Determine the impact of ETI-mediated cftr restoration on progression of airway disease in these young CF children; 2. Determine the impact of ETI-mediated cftr restoration on pancreatic exocrine and endocrine function in this cohort; and 3. Elucidate computational phenotypes of therapeutic responsiveness in the lungs and the pancreas and their trajectories using machine learning. This project is important as IT has the potential to usher in the next generation of therapeutics for CF which will be the initiation of HEMT in infancy to prevent the many later manifestations of CF including bronchiectasis, pancreatic exocrine insufficiency, and CFRD.

Transactions

Action Obligated Date
Amendment $0
Amendment $686,183
Amendment $686,183
Amendment $69,015
Amendment $69,015
Amendment $621,136
Amendment $621,136
Amendment $0
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Amendment $0
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Amendment $0
Amendment $746,983
Amendment $746,983
Original award $746,983
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Subawards

Subrecipient Amount Awarded
Duke University Pulmonary and pancreatic response to cystic fibrosis modulator therapy in young ch $133,409
Childrens Healthcare Of Atlanta, Inc. $1,210
Duke University $75,995
Duke University $72,743
Arthur M. Blank Hospital, Inc. $27,601

Quick answers

Who received award R01HL173340?

R01HL173340 was awarded to Emory University by National Heart Lung and Blood Institute (National Institutes of Health) on May 15, 2024.

How much is award R01HL173340 worth?

$4,993,617 has been obligated on R01HL173340.

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