American and European researchers publish study in Nature Medicine reporting a new, single treatment option for cystic fibrosis patients
Researchers from the George Washington University (GW), the University of Perugia, and the University of Rome have discovered a potential new drug to treat and stop the progression of cystic fibrosis. Thymosin ?1 (T?1) is a novel therapeutic single molecule-based therapy that not only corrects genetic and tissue defects, but also significantly reduces inflammation seen in cystic fibrosis patients.
“Right now there are multiple treatments for cystic fibrosis, and while these have improved life expectancy dramatically, there is still only a lifespan of about 40 years for patients. No one treatment can stand alone,” said Allan L. Goldstein, Ph.D., co-author of the paper and Professor Emeritus in Residence of Biochemistry and Molecular Medicine at the GW School of Medicine and Health Sciences. “We developed a single treatment that can potentially correct the genetic defect that causes cystic fibrosis and decrease the inflammation that happens as a result.”
Cystic fibrosis is a genetic disease that causes persistent lung infections and limits the ability to breathe over time, and it affects approximately 70,000 worldwide and 30,000 in the U.S. alone. Cystic fibrosis is the result of mutations in the gene encoding the protein called the cystic fibrosis transmembrane conductance regulator (CFTR), which is important to maintain chloride-channel activity affecting the salt and water balance in the lungs. This mutation results in a misfolded CFTR protein, and its premature degradation leads to impaired chloride permeability and persistent lung inflammation.
Goldstein and co-authors Luigina Romani, Ph.D., M.D., at the University of Perugia and Enrico Garaci, M.D., at the University of Rome “San Raffaele,” both in Italy, published their results in Nature Medicine. They report that T?1, a synthetic version of a naturally occurring peptide first isolated from the thymus, corrects the multiple tissue defects found in the lungs and small intestines in a mouse model of cystic fibrosis, as well as the defects in the CFTR seen in cells isolated from cystic fibrosis patients. T?1 not only significantly reduces the inflammation seen in cystic fibrosis, but also increases CFTR maturation, stability, and activity. Due to this two-pronged action, T?1 offers a strong potential to be a single-molecule therapeutic agent to treat and stop the progression of cystic fibrosis.
Goldstein and colleagues first isolated and characterized T?1 as a biological response modifier with potent immune therapeutic activity in 1979. This research was done in large part at GW. Although the peptide is produced in small amounts in several peripheral lymphoid and non-lymphoid tissues, the highest concentrations of T?1 are found in the thymus. T?1, whose commercial name is Zadaxin, has been approved for clinical use for over 15 years in 35 countries in the treatment of patients with viral infections, immunodeficiency diseases, malignancies, and HIV/AIDS. Though not currently available in the U.S., it has an excellent safety profile and does not induce the side effects and toxicities commonly associated with most immunomodulatory agents.
The Latest on: Cystic fibrosis
via Google News
The Latest on: Cystic fibrosis
- Cystic Fibrosis Can Be Diagnosed Using New Intra-nasal Imaging Technologyon August 17, 2019 at 3:45 am
Cystic fibrosis patients can be diagnosed using a minimally invasive new tool for viewing differences in the nasal airways at a cellular level. A paper published today in Science Translational ...
- I have Cystic Fibrosis and I climbed Kilimanjaro - here's howon August 15, 2019 at 11:01 pm
Cystic fibrosis (CF) is a pretty rare, genetic condition affecting more than 10,500 people in the UK. It's something you're born with, as a result of both of your parents carrying the genes that code ...
- Orkamb hailed little 'magic' pink pill and changing lives for children with cystic fibrosison August 15, 2019 at 1:10 pm
Days spent at home in pyjamas is what many young people do on school holidays, but for one North Queensland teenager it's something she's just experienced for the first time. After taking the cystic ...
- Melissa Chambers - Cystic Fibrosis Foundationon August 15, 2019 at 10:57 am
The Kentucky Attorney General's Office has confirmed it has secured restitution for 1,400 Auto Plaza USA customers who were overcharged.
- Parents of boy with cystic fibrosis call for paid care assistance from Governmenton August 15, 2019 at 10:25 am
The parents of a toddler with cystic fibrosis want paid care to be extended to help families like theirs to help cope with the financial burden the condition brings. Two-and-a-half-year-old Tailen ...
- Hero dogs can sniff out cystic fibrosis before it takes holdon August 14, 2019 at 11:22 pm
The team of dogs, Lizzie, Flint and Oakley, who can sniff out the bacteria that is a major cause of lung damage for cystic fibrosis sufferers (Picture: PA) Dogs can sniff out bacteria that is a ...
- Lung damage bacteria sniffed out by dogs in cystic fibrosis researchon August 14, 2019 at 4:21 pm
A new study found the animals can detect ultra-low concentrations of pseudomonas, the commonest cause of lung infection in the disease. Dogs can sniff out bacteria that is a major of cause of lung ...
- Scotland tells Vertex its cystic fibrosis drugs are not cost-effectiveon August 12, 2019 at 3:32 pm
In a setback to Vertex (VRTX) Pharmaceuticals, a Scottish agency determined the government should not cover a pair of cystic fibrosis medicines over their cost, posing another challenge to the drug ...
- Families 'devastated' as Scotland opts to not fund cystic fibrosis drugson August 12, 2019 at 10:15 am
People with cystic fibrosis in Scotland will continue to be denied potentially life-extending drugs after they were rejected for use by the NHS, prompting an outpouring of dismay and anger. The ...
via Bing News