Emily’s Entourage convened a Key Opinion Leaders Meeting in Philadelphia on September 9, bringing together world-class scientists to brainstorm new, out-of-the-box approaches to treat Emily’s rare CF mutation and those like her.
The meeting focused specifically on therapies that could be developed quickly—within one to five years—that might offer real hope to patients with CF with a focus on those like Emily who have rare nonsense mutations, including W1282X.
Significantly, the summit included a number of experts from inside and outside the CF community to facilitate cross-pollination of ideas and technologies. In Emily’s words, the goal was to “force collisions” of leading minds from aligned but different disciplines to spur creative, innovative thinking.
Among those assembled were experts in nanotechnology, molecular biophysics, neuro-genetics, pediatric pulmonology and molecular pathology.
The impressive lineup of participants included:
Chris Ahern, PhD (University of Iowa)
David Bedwell, PhD (University of Alabama, Birmingham)
Beverly Davidson, PhD (University of Pennsylvania)
Kevin Foskett, PhD (University of Pennsylvania)
Justin Hanes, PhD (Johns Hopkins University)
Paul McCray, MD (University of Iowa)
Martin Mense, PhD (Cystic Fibrosis Foundation)
Ted Torphy, PhD (Cystic Fibrosis Foundation)
Alan Verkman, MD, PhD (University of California San Francisco)
Rob Wilson, PhD (University of Pennsylvania)
In her welcoming remarks, Emily urged the group to “dare to dream big and audaciously” and to “take on the orphans, the special snowflakes,” referring to the patients with rare and currently unaddressed CF mutations. “We’re in a fierce race against time and we need Key Opinion Leaders like you on our team,” she explained.
Presenters then shared relevant research—most of it “right off the test tubes” and not yet published—and hypothesized ways that their work might be harnessed to expeditiously treat CF. Especially promising was the discussion of novel techniques for delivering gene therapy to CF-compromised lungs, long a stumbling block for researchers.
The Game Plan
The afternoon was spent setting priorities and creating an action plan. Participants considered a number of factors as they decided which research should be fast-tracked: how quickly the study could begin clinical trials, the likelihood of funding, clinical efficacy, safety, dosing convenience and manufacturing feasibility. When the meeting adjourned, it was clear that EE had made new friends at the highest levels.
“This was a spectacular engagement of top thought leaders on how to address this disease for Emily and thousands like her looking for cures,” said Dr. Ted Torphy, board chair for Cystic Fibrosis Foundation Therapeutics. “Today has certainly made me much more aware of Emily’s W1282X mutation. Emily should leave here feeling that there are people committed to not stopping until everyone with CF leads a full and productive life.”
Dr. Rob Wilson, a molecular genetic pathologist at the University of Pennsylvania, called the day “groundbreaking.” He explained, “Scientists are extremely busy and often lack opportunities for cross-fertilization. Gathering together scientists with different areas of expertise allows us to think about applying science in new ways. Hearing Emily’s story creates a sense of urgency and purpose. Having her here reminds us that it’s not just about the science; it’s about saving lives.”
Emily left the meeting upbeat and energized. “This was awesome,” she said. “Some of these people had hardly worked in CF and we were able to pull them into our fight. We have a clear plan of action. It’s time to put the pedal to the metal. EE can be the fire that propels it forward.”
For more photos from the meeting: http://bit.ly/EEKOLMtg