The Alpha-1 Foundation hosted the virtual 22nd Gordon L. Snider Critical Issues Workshop entitled, CT Lung Density Estimates in Alpha-1 Antitrypsin Deficiency: State of the Art on Friday, April 9, 2022.
This workshop was laser-focused on the clinical endpoint CT densitometry and will be composed of several informative sessions on topics such as the regulatory approach to rare and orphan diseases, what CT densitometry means to clinical trials, the results of the RAPID trial, and more.
Scientific meetings and conference organized by the Alpha-1 Foundation are designed to answer important questions in Alpha-1 Antitrypsin Deficiency (Alpha-1), identify new approaches, and help to establish the Foundation’s credibility as a resource for serious research. The Gordon L. Snider Critical Issues Workshops are named after the late Dr. Gordon L. Snider one of the most influential researchers in Alpha-1 Antitrypsin Deficiency. Dr. Snider not only made seminal scientific contributions which have clarified the pathogenesis of Alpha-1, but also laid the groundwork for the Foundation’s research program. In particular, he stressed the importance of encouraging young investigators to pursue a career in Alpha-1 research. The Gordon L. Snider Critical Issues Workshops are single-topic workshops that facilitate exchange between experts and answer important questions or stimulate additional investigation.
“So much has been accomplished towards the development of CT lung densitometry since last conference hosted by the Alpha-1 Foundation twenty years ago on this biologically meaningful outcome measure. Listening to the overwhelming amount of data presented by the conference speakers, I am convinced that CT lung densitometry is ready to be used as a primary outcome for trials evaluating the efficacy of novel drugs to prevent progression of emphysema in alpha-1-antitrypsin deficient individuals,” said Dr. Mark Brantly.
The virtual event was made possible by the hard work and dedication of the workshop’s co-chairs, Dr. Kenneth Chapman and Dr. Mark Brantly. We are grateful for their dedication and input into the workshop program and for inviting and securing the participation of this esteemed group of experts. The Foundation was honored to have Dr. Peter Marks as the guest keynote speaker.
The agenda included presentations from experts in the field:
Mark L. Brantly, MD– University of Florida College of
Medicine
Peter Marks, MD, PhD– Center for Biologics Evaluation and Research, FDA
James K. Stoller, MD, MS– Cleveland Clinic
Berend C. Stoel, PhD– Leiden University Medical Center
Noel G. McElvaney, MD, BCh, BAO, FRCPI, FRCPC- Royal College of Surgeons in Ireland
Kenneth R. Chapman, MD, MSc, FRCPC, FACP, FERS- University of Toronto
A special thank you to our patient presenter:
Jon Hagstrom- Westchester County, New York
“The uncertainty about what is the best yardstick for alpha-1 research trials has stymied our best efforts to develop new treatments. Today’s meeting of international experts brought all the right people to the table – patients, regulators, clinicians, research scientists and innovative companies. There was great information sharing and real enthusiasm to find a way forward,” exclaimed Dr. Kenneth Chapman.
The outcome of this workshop will be a white paper and our proposed collaboration with the regulatory agencies on the issuance of guidance. In addition the co-chairs will work with all of the presenters to publish a journal article. By bringing together a group of leading experts, we will be able to impact the use of CT densitometry as a clinical endpoint.
“I had two major takeaways from the workshop. First of all, CT Densitometry as an endpoint in clinical trials could significantly reduce the development time of new lung therapies. Secondly, much research has already been done on its validity and the results presented seem convincing,” commented Jon Hagstrom.
Thank you to the generous sponsors for their support of the 22nd GLS Workshop: AlphaNet, CSL Behring, Grifols and Takeda.
