Fighting to Cure Cystic Fibrosis

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This campaign ends on January 1 at 12:59AM EST
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About

Morris and Lana Knopf of Minneapolis, Minnesota initially supported clinical research efforts undertaken by the Cystic Fibrosis Foundation promptly after the birth of their grandson, Sam Akerman-Knopf, in 2002 and his diagnosis of Cystic Fibrosis. After discovering that critical CF research, leading to positive treatments for this mutation, was being done at the University of Iowa Hospitals & Clinic (“UIHC”) funded by the CF Foundation,  Morris and Lana, graduates of the University of Iowa, established the Sam Akerman-Knopf Cystic Fibrosis Research Fund (the “Fund”) in 2015 to provide money directly to support the research efforts at UIHC and initiated an annual campaign to raise moneys for this Fund which will enable the University of Iowa to bring remarkable discoveries to fruition for the CF community.

Since the Fall of 2018, due to attentive and persistent medical care and advances in treatment, Sam’s life has been dramatically altered resulting in a significant increase in pulmonary function and lung capacity and thus vastly improving his life style.  Fundamental research from research institutions at UIHC has led to developments in CF medication by pharmaceutical companies.  Dr. Michael Welsh is the research mentor at UIHC.  His work was so critically important for the development of drugs that he and a key scientist at one of the drug producing companies received the Shaw Prize in Medicine for their discoveries.   This medication, Trikafta, has dramatically impacted Sam’s life leading to an improved life style.

Sam just completed his second year in the College of Engineering at the University of Iowa.  In addition to his rigorous academic regimen and some necessary college fun, Sam has continued to spend one afternoon a week at the CF research lab under the mentorship of Alejandro A. Pezzulo, M.D.  Sam’s life style is now pretty similar to any other college student.  During his first and second years, Sam lived in University student housing and enjoyed the proximate location to his various classes.  He found time to cheer on the Hawkeye football team and both basketball teams and participated in the Robotics program at the College of Engineering.  For the coming school year, Sam will live in an off-campus apartment with two college friends.  Sam spent the past summer in Iowa City working for ProtoStudios.  He also was a member of the University of Iowa Robotics team that competed in competition in Calgary, Canada in mid-August, 2023.

According to Dr. Pezzulo, support from the Fund has helped the Pezzulo Lab and the CF and Lung Biology Center to continue to advance work by the Lab and Center with human airway cells to understand chronic lung disease.  That continued work relates to the following matters:

  • Understanding mechanisms dictating disease severity.  The inquiry focuses on why some people with CF or other lung diseases do well whereas others have severe progressive disease. In most cases the answer is not known.  The Lab and Center are approaching this question by comparing the cellular responses to multiple stimuli in cells from people that develop chronic severe disease against cells from healthy controls.  Progress has been enabled by the Eppendorf automatic pipettor provided by the Fund.  The first publication showing the development of this method is now publicly available in the journal “Scientific Reports”.  For the next step, the Lab and Center are currently applying the method to cells from people with CF and another rare lung disease called sarcoidosis which, like CF, is driven by chronic inflammation and fibrosis.
  • “Theratyping” for personalized therapy in cystic fibrosis.  Many people with CF have rare mutations that could potentially respond to Trikafta triple therapy but are ineligible for a trial of the drug due to their type of mutation.  The Fund provided support for the Oxford Nanopore Technologies MiniON sequencer and for reagent costs which allowed the Lab and Center to obtain and grow nasal cells from people with rare CFTR mutations and test them in the Lab for response to Trikafta.  More people with CF including pediatric patients have been tested.  This approach directly impacted the lives of these patients as it facilitated insurance approval for Trikafta use.  A report is currently being written describing the response to treatment in patients.
  • Epigenetic memory in airway cells.  Past infections and exposure to allergens may affect how lung airway cells function and respond to future exposures in CF and other lung diseases (asthma, pulmonary fibrosis).  The mechanism may involve modifications to a cell’s DNA and RNA without affecting genetic sequence (“epigenetic”).  The Lab and Center are developing a sample bank and techniques to study how this “epigenetic memory” is established, propagates to the whole lung and affects human health.  This project will be a large area of focus for the Lab in the upcoming years.

For this fund raising year, our goal is to raise $12,000 for this Fund as we did, with your help, in 2022.  Since the initiation of the Fund, we have raised $102,666.11.  We are hopeful that you will support this critical, ongoing research at UIHC.


NOTICE: The University of Iowa Center for Advancement is an operational name for the State University of Iowa Foundation, an independent, Iowa nonprofit corporation organized as a 501(c)(3) tax-exempt, publicly supported charitable entity working to advance the University of Iowa. Please review its full disclosure statement.


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