Study used blood test to identify distinct molecular signatures of disease; paves the way to precision medicine for cystic fibrosis
Researchers at Stanley Manne Children’s Research Institute at Ann & Robert H. Lurie Children’s Hospital of Chicago, and colleagues, used a blood test and microarray technology to identify distinct molecular signatures in children with cystic fibrosis. These patterns of gene expression ultimately could help predict disease severity and treatment response, and lead to therapies tailored to each patient’s precise biology. Findings were published in Physiological Genomics.
“Our findings pave the way to precision medicine for cystic fibrosis patients, eventually helping us match treatment to each patient’s unique genomic pattern of disease,” says lead author Hara Levy, MD, MMSc, from Manne Research Institute at Lurie Children’s, who is an Associate Professor of Pediatrics at Northwestern University Feinberg School of Medicine. “Our study was the first to identify molecular signatures of cystic fibrosis from a blood test taken during a routine clinic visit, giving us a baseline. Greater understanding of these molecular signatures may lead to unique molecular markers that could help us intervene earlier to changes in a patient’s inflammatory response to airway infection or pancreatic function, allowing us to provide more focused treatment. It would be a huge improvement over the one-size-fits-all treatment approach we currently have for patients with cystic fibrosis.”
To identify baseline molecular signatures in cystic fibrosis, Dr. Levy’s lab obtained genomic information from patients’ blood samples using cutting-edge technologies such as Affymetric array and Illumina MiSeq. The team then merged this genomic information with each individual’s clinical history gathered from electronic medical records. They compared this snapshot of patient-specific data with healthy controls. Their study provides strong evidence for distinct molecular signatures in cystic fibrosis patients that correlate with clinical outcomes.
Cystic fibrosis is a progressive genetic disease that damages multiple organs, including the lungs and pancreas. Currently, the average predicted survival is 47 years. Although cystic fibrosis is caused by dysfunction of a single gene (CFTR) and treatment that targets CFTR mutations is available, the relationships between the abnormal gene product, development of inflammation and disease progression are not fully understood. This limits the ability to predict a patient’s clinical course, provide individualized treatment and rapidly monitor treatment response.
For example, it is not clear why patients with cystic fibrosis are susceptible to chronic lung infections, since they are considered to have a functional immune system.
“We are now trying to discover why patients with cystic fibrosis become infected so easily,” says Dr. Levy. “We are taking a closer look at the immune cells that make up many of the molecular signatures we found in cystic fibrosis.”
More study is needed before precision medicine for cystic fibrosis reaches the clinic.
“With more research, a blood test to gather genomic specifics of each patient’s disease might be available in the clinic within the next five years,” says Dr. Levy. “Precision medicine will revolutionize care for cystic fibrosis patients.”
The Latest on: Cystic fibrosis
via Google News
The Latest on: Cystic fibrosis
- Medicare Coverage for Cystic Fibrosis: Testing, Management, and Treatmentson January 12, 2021 at 1:31 pm
Medicare covers the testing, treatment, and care you need for cystic fibrosis. Find out exactly what's covered by the different parts of Medicare.
- Vertex CEO says it has the ‘financial firepower’ to make more dealson January 11, 2021 at 3:58 pm
Vertex Pharmaceuticals is looking to acquire “mid- and late-stage assets” to bolster its current research pipeline, CEO Reshma Kewalramani said Monday at the virtual J.P. Morgan Healthcare Conference.
- Vertex Pharma taking more aggressive M&A stance, flush with cash from cystic fibrosis drugson January 11, 2021 at 8:40 am
With its balance sheet growing with cash from its cystic fibrosis drugs, Vertex has the “financial firepower” to consider deals, CEO says.
- Benefits of 'transformational' cystic fibrosis drug highlighted in new reviewon January 11, 2021 at 6:22 am
A new systematic review by University of Liverpool researchers highlights the positive impact that triple-combination therapy has on the wellbeing of people with Cystic Fibrosis (CF).
- Machine learning comes of age in cystic fibrosison January 8, 2021 at 7:18 am
World-leading AI technology developed by the Cambridge Centre for AI in Medicine and their colleagues offers a glimpse of the future of precision medicine, and unprecedented predictive power to ...
- Translate Bio Announces Timing of Interim Results from mRNA Therapeutic Clinical Trial in ...on January 7, 2021 at 10:45 pm
MRT5005 is the first clinical-stage mRNA therapeutic with nebulized delivery to the lung and first to be administered in multiple doses for the treatment of a chronic genetic disease -- -- Completed ...
- Felix Biotechnology announces initiation of the CYPHY Phase 1/2 study at Yale for its lead asset targeting P. aeruginosa infectionson January 7, 2021 at 1:04 pm
Felix Biotechnology today announced the initiation of CYPHY, a Phase 1/2 investigator-initiated single center trial at Yale University ...
- Supermarket worker awarded compensation after foot injury led to aggravation of cystic fibrosison January 4, 2021 at 9:41 pm
A worker who suffers cystic fibrosis has been awarded compensation for a lung transplant he needed as a result of a foot injury while at work, despite not telling his employer of his long term illness ...
- Dallas respiratory therapist commits to helping patients, even as cystic fibrosis puts her at riskon January 3, 2021 at 10:00 am
When the coronavirus pandemic began, everyone told Breanna Pierce what she should do. Her doctors suggested and her family urged: Take a break ...
- Global Cystic Fibrosis Therapeutics Market (2020 to 2025) - Industry Trends, Share, Size, Growth, Opportunity and Forecaston December 31, 2020 at 12:53 pm
The "Cystic Fibrosis Therapeutics Market: Global Industry Trends, Share, Size, Growth, Opportunity and Forecast 2020-2025" report has been added to ResearchAndMarkets.com's offering. The global cystic ...
via Bing News