Excerpts: Seven patients along with Eric Jonasch, MD shared their VHL stories to educate 45 representatives of the FDA… [VHL Alliance participated in a virtual FDA (US Food and Drug Administration) Patient Listening Session, June 11th, 2020] *

Questions and Answers by FDA

What has been the experience with clinical trials?

-        The criticality of the role of doctors as Principle Investigators and site staff who oversee the conduct at the site level was noted.

-        Michaela Thorne, a participant in a current clinical trial, shared that her experience was extremely smooth; while anemia developed, all else has gone well with being fully accommodated for visits and regular scans.

-        It was noted that this specific study was the fasted [fastest] known enrolling trial; partially contributing was word of mouth amongst patients and ultimately met enrollment in approximately 2 months, truly demonstrating an unmet need.

How much outreach exists internationally?

-        There are VHL Alliance affiliates [or close associates] throughout the world including Europe, Russia, and Asia. The U.S. VHL Alliance is considered the global patient organization since it is the only one with paid staff. Thus the materials that are developed at VHLA are shared with affiliates. An example of the interaction collaboration is the biennial International VHL Medical/Research Symposium. This year it was supposed to take place in Amsterdam, but will be held virtually due to the pandemic.

-        Dr. Eric Jonasch added that the VHL research community is extremely small thus organizations are not wide spread globally as one sees with other diseases.


*Please note: the official summary is on vhl.org, but this part was from a slightly different version of a much earlier one that I had downloaded.

Eric Jonasch, MD is Professor, Department of Genitourinary Medical Oncology, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center (US) and VHLA councils


US centers…

·         University of Alabama at Birmingham

·         Mayo Clinic, Scottsdale, Arizona Website

·         University of California, Los Angeles (UCLA)

·         Stanford University Hospital and Clinics* Website

·         University of Colorado, Denver

·         University of Miami

·         Moffitt Cancer Center, Tampa

·         Northwestern Memorial Hospital, Chicago

·         University of Chicago*

·         Indiana University Medical Center, Indianapolis

·         University of Iowa Hospitals & Clinics, Iowa City

·         University of Kansas Medical Center, Kansas City

·         The Johns Hopkins Hospital, Baltimore, Website

·         Lahey Medical Center, Burlington Website

·         Massachusetts General Hospital, Boston* Website

·         Henry Ford Hospital, Detroit Website

·         University of Michigan Medical Center, Ann Arbor* Website

·         Mayo Clinic, Rochester Website

·         University of Minnesota, Minneapolis

·         WashU Barnes/Siteman Cancer Center, St. Louis

·         Columbia University Medical Center, New York Presbyterian Hospital, New York City* Website

·         Memorial Sloan Kettering Cancer Center, New York City Website

·         Roswell Park Comprehensive Cancer Center, Buffalo

·         SUNY Upstate Medical University, Syracuse

·         Levine Cancer Institute, Charlotte

·         University of North Carolina, Lineberger Cancer Center, Chapel Hill* Website

·         Cleveland Clinic* Website

·         Ohio State University, Wexner Medical Center, Columbus

·         Hospital of the University of Pennsylvania, Philadelphia* Website

·         University of Pittsburgh Medical Center

·         St. Jude Children’s Research Hospital, Memphis**

·         Vanderbilt University Medical Center, Nashville*

·         MD Anderson Cancer Center, Houston* Website

·         University of Texas – Health San Antonio

·         University of Texas – Southwestern, Dallas

·         Huntsman Cancer Institute, Salt Lake City*Website

·         University of Virginia, Charlottesville* Website