Amid a sea of data, the PF community is critical in the search for a cure
A pillar of the PFF's 5-year strategic plan is to use registry to 'accelerate research'
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I had the opportunity to get away for a couple of days recently and went to the shore. As I looked out at the vastness of the ocean in the late afternoon sunlight, it occurred to me that this is what pulmonary fibrosis (PF) researchers face every day — a sea of data right in front of them. They know the answer they are searching for is in that data. The real question is how to find it.
When I was diagnosed with idiopathic pulmonary fibrosis in early 2017, the realization that there was no cure came quickly. That year, there were only two approved anti-fibrotic therapies, Esbriet (pirfenidone) and Ofev (nintedanib). Today, there are three, following the approval of Jascayd (nerandomilast) this year.
I wrote this year about The PFF Is ME, the Pulmonary Fibrosis Foundation‘s (PFF) strategic plan for 2025-2030, which was presented at the organization’s summit in Chicago. One of the four pillars of the plan is to “accelerate research by advancing discovery through the PFF Registry, targeted funding, and partnerships that speed ideas from lab to clinic.”
Accelerating research into PF
I talked via Zoom with Amy Hajari Case, MD, the foundation’s chief medical officer, about the Accelerate Research pillar and asked her what makes this initiative different.
“This process was heavily informed by the community,” Case said. “We reached out to and heard back from more than 350 different individuals that represent all parts of the PFF community. These were patients, caregivers, family members, lay people, and there were people who work in industry who we have to partner with because they are such an important piece of progress. We heard from our academic partners, researchers, clinicians, and people who deliver the care.”
I asked Case how vital patient community engagement is in the research process.
A view of the ocean reminds Sam Kirton about the sea of data available to researchers exploring a cure for pulmonary fibrosis. (Photo by Sam Kirton)
Research is how new therapies become available and how therapies being investigated that don’t work are taken off the docket. One way the PFF hopes to accelerate research is by developing new clinical trial endpoints that can identify the success or failure of a new therapy earlier and more effectively. There is a need to “fish or cut bait” as quickly as possible if something does not work.
“That will let us move on to find the thing that does work,” Case said.
The key to finding what does work is for patients to participate in clinical trials.
Case noted, however, that the number of patients in the PF space is finite, and patient engagement in trials can be constrained by the fact that not all patients will meet the study criteria. Travel distance or time commitment may also prevent participation.
I asked Case what she would tell people who are unlikely to benefit from research in their lifetime, to encourage their participation. She said she believes “there is a lot of altruism inherent in clinical trial participation.”
Every role in any type of research is essential to the success of the trial, she said, noting that in most clinical trials, for example, there is a placebo arm. While every patient would want to receive the trial drug if it were successful, the role of those in the placebo arm is equally important.
“Research equals hope” is a phrase I use often when I talk with research teams. Making Accelerate Research one of the four pillars of the PFF’s new strategic plan brings more hope to the PF community.
The answer is out there. We, the PF community, are critical to finding it. If the PFF is to succeed in accelerating research, the PF community must be fully engaged. Success will allow everyone involved to make every breath count.
This is my final column of 2025. I will return on Jan. 6, recharged and ready to make every breath count.
Note: Pulmonary Fibrosis News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Pulmonary Fibrosis News or its parent company, Bionews, and are intended to spark discussion about issues pertaining to pulmonary fibrosis.
Byron Jeffrey
I have written a letter to the FDA with CC to my senator and. R. F. Kennedy Jr. I would like to have everyone read it and write their own letter to the FDA. It is important that we get involved.
Dan Prosperi
There are no clinical trials in Fresno the 4th largest city in California. That's a problem for a lot of us. A lot of lung issues in the valley.
Samuel Kirton
Hi Dan,
Not knowing your diagnosis or where you are being followed my suggestions are limited. I suggest discussing your desire to participate in clinical trials with your pulmonologist. While they may not be supervising a trial they may know of a trial at a Care Center Network site. My California geography knowledge is limited but Fresno is "roughly" equidistant to Sacramento, San Francisco and Standford. I understand traveling distance with IPF. I live ~2 hours from my care team. You might also consider making contact with the Research Coordinator at any of those three care center network sites.
Sam...
Daniel Nogueira
Hello Samuel,
Merry Christmas!! We are of course happy to live another one. Always fond to read your columns. I finished my participation in a phase II trial in October. I live in South America and had to remain in NYC for the first three months, then went home and returned to NYC every 3 months for the subsequent visits (April, July, October). I'm sure I got the placebo although no one yet confirmed. Having worked for a clinical trials company for almost 20 years, I always thought participation is the best option for a still incurable desease. But to feel in my own flesh that I got the placebo (the famous flip of a coin...) destroyed me. I lost a year of my life with not many to spare. The treatment by the doctors in Mount Sinai was, on the other hand, superb. Although it was out of their hands to cure me. Value added was their recommendation to pursue a bilateral transplant, probably coming my way in 2026 (will greatly appreciate your contact, eventually).
Anyway, there were so many clinical trials with a placebo arm, that pharma companies as a whole and the FDA in particular, already know how placebo behaves. The placebo arm in the A drug test will be the same as the placebo arm in the B drug test, assuming subjects are screened in the same conditions. The information is there already so that placebo arms can be eliminated and all subjects get the study drug. Study costs can be cut by 50% or more patients be included. IA will also accelerate results. All of this will stimulate more patients participation and a faster approach to the final cure. Also, why do all drugs have marvelous results in mice and disappoint in humans? That needs to be researched and answered fast. Many people are running out of time.
Best regards, Daniel
Susan Sorensen
Thank you Sam; I always enjoy your columns and the effort you continue to put into advocating for a cure of this dreadful disease.
Daniel Nogueira
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