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Inhaled gene therapy for cystic fibrosis is being tested in the UK and Europe
An inhaled drug that could improve lung health in people with cystic fibrosis, regardless of mutation type, is being tested with people in the UK and Europe.
Cystic fibrosis (CF) is caused by defects in the CFTR gene, which leads to a build-up of thick, sticky mucus in the lungs and digestive system. This causes lung infections and gradually impairs the ability to breathe. A new gene therapy based on a lentiviral vector works by inserting a functioning copy of the CFTR gene into the DNA of epithelial cells in the patient’s airways.
Currently, some people with CF respond well to relatively new treatments known as CFTR modulator drugs, which act on a CFTR-related protein. However, for about 10-15% of patients, these drugs are not effective.
The aim of the new investigational drug, known as BI 3720931, is to improve lung function and reduce the number of exacerbations (flare-ups of the disease, often leading to hospitalisation) in people with CF regardless of the type of mutation, including those who are genetically unable to benefit from other CF treatments.
The LENTICLAIR 1 trial, which will evaluate the safety, tolerability and efficacy of the treatment, is being conducted by biopharmaceutical company Boehringer Ingelheim in partnership with the UK Respiratory Disease Gene Therapy Consortium (GTC) and OXB (formerly Oxford Biomedica). Approximately 36 men and women with cystic fibrosis will be treated at centres in the UK, France, Italy, the Netherlands and Spain.
Cystic fibrosis is an inherited, incurable disease that worsens over time. It is estimated to affect 105,000 people worldwide. There are more than 2,000 known mutations in the CFTR gene that result in varying degrees of disease severity.
Lentiviral vectors are a type of gene therapy that utilises the ability of lentiviruses to infect human cells. Lentiviruses are a family of viruses that infect an organism by inserting their genetic material into the genome of a host cell. By modifying lentiviruses, scientists have been able to use them as a means of inserting useful genes into cells.
The research is led by Eric Alton, professor of gene therapy and respiratory medicine at the National Heart and Lung Institute. He coordinates the UK CF Gene Therapy Consortium, which brings together three centres in the UK (Edinburgh and Oxford Universities and Imperial College London) dedicated to translational gene therapy for respiratory disease. He is also an Honorary Consultant Physician at the Royal Brompton Hospital, one of the UK’s research centres.
‘The UK CF Gene Therapy Consortium is delighted to have reached this milestone after 24 years of dedicated effort and close collaboration with our funding partners. Although the immediate target is patients who are unsuitable for CFTR modulators, this new therapy has the potential to achieve lasting improvements in CFTR function and disease modification in people with CF regardless of mutation type and, importantly, has the potential for reapplication if required.’
Eric Alton, Professor of Gene Therapy and Respiratory Medicine at the National Heart and Lung Institute.
The research scientists share that it has been incredibly exciting to see the health benefits of CFTR modulators, but upsetting that those people who cannot benefit from these drugs are in urgent need of alternative therapies.
In this trial, they broke new ground by developing a gene therapy that can deliver long-term CFTR expression. The scientists expressed their gratitude to people living with CF who are willing to give their time to trials of new drugs, and without whom the researchers could not have made such progress.
In the first phase of the study, different doses of the drug will be used to assess safety, tolerability and dose selection for the second phase of the study. In Phase 2, two selected doses or placebo will be administered in a randomised, double-blind, placebo-controlled dose escalation study to assess clinical efficacy and safety.
After completion of the 24-week study period, participants will participate in long-term follow-up in the LENTICLAIR-ON study.
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