A single dose of the investigational oral therapy PLN-74809 for idiopathic pulmonary fibrosis (IPF) achieved therapeutic target engagement of up to 98% in the lungs of patients, according to interim results of a Phase 2a trial. At all doses tested, the anti-fibrotic, or anti-scarring, therapy engaged its intended target…
Potential IPF Gene Therapy Engages Target in 98% of Patients in Trial
Photo courtesy of Jerry Barnum Day 13 of 30 This is Jerry Barnum’s story: My name is Jerry Barnum. I am 70 years old and live in Minnesota. In 2017, I was diagnosed with interstitial pulmonary fibrosis with autoimmune features. As one who learned I have a terminal…
Blocking the activity of a cell death-suppressing protein called Bcl-2 promoted the death of macrophages — a type of immune cell involved in pulmonary fibrosis (PF) — and reversed established lung scarring in a mouse model, a study showed. Notably, Bcl-2’s anchoring at the surface of macrophage mitochondria was…
Photo courtesy of Terri Dominick Day 12 of 30 This is Terri Dominick’s story: The IPF roller coaster has many riders, not just that main person in the front car (aka the one with the diagnosis). I was chosen to ride this roller coaster with my father,…
Photo courtesy of Steve Dragoo Day 11 of 30 This is Steve Dragoo’s story: I was initially diagnosed with interstitial lung disease in Manila in October 2016 and just thought I had walking pneumonia. I had never heard this term before and such cases are quite rare in…
Participation in clinical trials exposes rare disease patients to financial, physical, and emotional pressures, according to the results of a patient focus group series. “Rare disease trial participants are running an endurance race they are highly motivated to complete, but these incremental burdens negatively impact their ability or willingness to…
Photo courtesy of Nicholas Sloop Day 10 of 30 This is Nicholas Sloop’s story: I’ve always had a pretty active lifestyle, which I attribute to my dad. Growing up with my dad as an elementary school PE teacher — one who coached many different sports during his teaching career…
A trio of global companies is teaming up to develop an inhaled dry powder formulation of SF2523, an investigational small molecule that blocks the activity of two fibrosis-driving proteins that may help block tumor growth and ease lung scarring and inflammation. SF2523 is being developed as a treatment for…
Photo courtesy of Linda M. Day 9 of 30 This is Linda M.’s story: We have been watching our friends, Kevin and Dana Olson, go through their PF journey for the past five years. Kevin was diagnosed with PF in 2014 and received a double lung transplant…
Photo courtesy of Latoya Hawkins Day 8 of 30 This is Latoya Hawkins’ story: After years of a cough that just would not go away, I was diagnosed with pulmonary fibrosis in December 2019. I was prescribed oxygen at that time, which made my disability more visible. Although I…
