About 4,400 researchers, doctors, patients and others from 46 countries are converging on Indianapolis for the 31st Annual North American Cystic Fibrosis Conference — the world’s largest gathering dedicated to cystic fibrosis research and care.
The Nov. 2-4 event at the Indiana Convention Center will feature more than 350 sessions and roundtable discussions, along with 50 exhibitors and more than 700 poster presentations. It’s being organized by the Cystic Fibrosis Foundation, or CFF.
“When the CFF was founded in 1955, parents were told to enjoy their children, because they often didn’t live past elementary school,” Anne Willis, vice president of the foundation’s patient access programs, told Cystic Fibrosis News Today at the organization’s headquarters in Bethesda, Maryland.
“But there have been some amazing improvements over the last few years in terms of healthcare and available treatments,” she said. “People are graduating, getting married, holding jobs — and reaching milestones they could have never imagined before.”
The conference, which began in 1986 with 300 participants, focuses on advances in CF care, breakthroughs in research, the use of patient data, and promising routes to a cure. This year’s agenda will include more than 60 sessions, with tracks ranging from faulty versions of the CFTR gene that causes the disease, to airways physiology, to nursing, nutrition and respiratory therapy.
Conference-wide presentations will be made on lung transplants, gene editing, next-generation therapies and personalized medicine.
There will also be a panel on pregnancy and CF. That’s because more women with CF are reaching adulthood, creating a new focus on reproductive health and family planning.
ProQR Therapeutics, a Dutch biotech company that develops RNA-based treatments for CF, epidermolysis bullosa and other rare genetic diseases, will be among the companies discussing a potential therapy it has developed — QR-010. The presentation will focus on the results of a Phase 1b trial of the mist-formulation treatment. The trial evaluated the treatment’s safety, and participants’ ability to tolerate it.
Preliminary indications are that QR-010 improves patients’ ability to deal with their symptoms, the company has reported.
QR-010 is designed to repair abnormal versions of the CFTR gene. The goal is to generate normal CFTR protein in people with one or two copies of the gene’s most widespread abnormality — the F508del mutation.
Stuart Elborn, immediate past president of the European Cystic Fibrosis Society, will make the QR-010 presentation, titled “A first-in-human, Phase 1B, dose-escalation study of QR-010, a novel antisense oligonucleotide administered in subjects with cystic fibrosis homozygous for the F508del CFTR mutation.”
Elborn, a consultant at London’s Royal Brompton Hospital who also is clinical chair of respiratory medicine at Imperial College, will discuss the therapy during a Nov. 4 workshop titled “Toward the Goal of a One-Time Cure: Challenges & Opportunities.”
ProQR will also conduct a poster session presentation on QR010 on Nov. 2. And at 8:30 p.m. Nov. 2, it will host an investor and analyst event. This will cover additional findings of the Phase 1b study of QR-010 and update participants on other therapy candidates in ProQR’s pipeline.
All the conference abstracts appear in the online edition of Pediatric Pulmonology.
CF, the most common fatal inherited disease in the Western world, has no cure. A defective CFTR gene causes a thick buildup of mucus in the lungs, pancreas and other organs in people with the disease. The mucus clogs the airways and traps bacteria, leading to infections, extensive lung damage and eventually respiratory failure.
Although scientists have identified more than 1,900 CF-causing genetic mutations, the F508del mutation affects about 85 percent of the world’s 75,000 or so CF patients.
ProQR created an aerosol device that patients can use to deliver QR-010 directly into their lungs. The mist format increases the therapy’s effectiveness.
The U.S. Food and Drug Administration and the European Union have granted QR-010 orphan drug designation, and the FDA fast-tracked the therapy in July 2016. The designations are aimed at speeding up the therapy’s approval process.
“We started this company five years ago to develop a therapy that would make CF patients like my son better,” ProQR’s CEO, Daniel A. de Boer, told CF News Today. “We are very pleased that QR-010 has demonstrated in this clinical trial to do exactly that. With these positive results in hand, we aim to advance QR-010 for the F508del mutation and expand to develop therapies for certain Class 1 stop-codon mutations that cause cystic fibrosis.”