CF Foundation awards $900K to accelerate genetic therapy research
University of Michigan aiming to aid those for whom current meds don't work
Written by |
The Cystic Fibrosis Foundation has awarded University of Michigan researchers $900,000 over two years to advance new treatments for cystic fibrosis (CF) — including genetic therapies that could offer novel options for people who do not benefit from existing medicines.
“There are many different mutations that cause CF and several types of genetic therapies are being developed to address them,” Alex Piotrowski-Daspit, PhD, the program’s principal investigator and an assistant professor of biomedical engineering at U-M, said in a university press release announcing the grant. “There is still a great deal to do, particularly in delivering those therapies to the right tissues and cells.”
The new funding will support the university’s Cystic Fibrosis Research Development Program, or CF@UM, an effort bringing together researchers in engineering, medicine, pharmacology, infectious diseases, and RNA therapies. The monies will, in part, go toward expanding research infrastructure and supporting trainees as the school works toward creating a nationally recognized CF research center.
The award follows a $500,000 Program Development Award from the CF Foundation granted in 2024. That earlier funding helped lay scientific and other groundwork that has been key to bringing CF researchers together at U-M.
“I believe the broader CF research community and the CF Foundation are excited by the work we’re doing here and the momentum we have generated,” Piotrowski-Daspit said. “We are grateful for the continued support from the CF Foundation as we continue to develop infrastructure and build a center focused on therapeutic development, delivery, and translational impact.”
Also supported by the program will be pilot research projects, a series of seminars, a yearly retreat, and workshops to help researchers form interdisciplinary teams. Six travel awards each year are also part of the package and are meant to enable trainees to attend the North American Cystic Fibrosis Conference, which is held in a different location each year. This year’s conference will be in Atlanta, running from Oct. 7-10, while next year’s event will be held in San Antonio, Texas, in November.
“Our goal is to stimulate the community in multiple ways,” Piotrowski-Daspit said. “The seminar series, project development workshops, annual retreat, and pilot projects all create opportunities for people to find collaborators on campus and turn promising ideas into CF research programs.”
U-M’s Bold Challenges initiative has separately provided funding to help the group prepare a proposal for a larger research center.
“It is immensely meaningful to have additional support from the University of Michigan,” Piotrowski-Daspit said.
Award creates a ‘full-circle moment’ for University of Michigan
The effort comes on the heels of a long track record of CF research at U-M. In 1989, a team co-led by Francis Collins, MD, PhD, then at U-M, helped identify the gene responsible for CF. Researchers at the university now study the biology of the disease, as well as respiratory infections and potential treatments — including approaches for rare forms of CF.
“It is a full-circle moment to bring an emphasis on basic CF research back to Michigan,” Piotrowski-Daspit said. “We have made significant progress in bringing the community together and engaging new investigators in CF research.”
I am excited to be part of building this program. … Our goal is to create an enduring community that brings together engineering, medicine and basic science to develop better therapies for people with CF.
CF is caused by mutations in the CFTR gene, which provides instructions for making a protein that helps regulate the movement of chloride, or salt, and water in and out of cells. CFTR modulators are medications that improve the function of the defective CFTR protein and have transformed the outcomes for many people with CF. However, they do not work for every disease-causing mutation. Genetic therapies are therefore being developed as options that may suit people who are not eligible or do not tolerate CFTR modulators.
CF@UM works around three interconnected research areas: therapeutic development, therapeutic delivery of treatments to the cells where they are needed, and translational testing, which aims to find approaches that work in humans after showing promise in early testing.
The program will study the impact of CF throughout the body and across the lifespan, according to the university. It is also designed to connect laboratory research and patient care: Samples from people with CF can inform laboratory studies, while findings from those studies can progress toward clinical testing.
“The goal is to connect discoveries made in the laboratory with CF care teams and patients in Michigan Medicine’s adult and pediatric clinics,” Piotrowski-Daspit said.
“I am excited to be part of building this program at such an early stage in my career,” she added. “I have appreciated the opportunity to grow with this program. Our goal is to create an enduring community that brings together engineering, medicine and basic science to develop better therapies for people with CF.”
Leave a comment
Fill in the required fields to post. Your email address will not be published.