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
- Translate Bio Announces Fourth Quarter and Full Year 2020 Financial Results and Highlights ...on February 25, 2021 at 1:31 pm
Translate Bio (Nasdaq: TBIO), a clinical-stage messenger RNA (mRNA) therapeutics company developing a new class of potentially transformative medicines to treat or prevent debilitating or ...
- Translate Bio Announces Fourth Quarter and Full Year 2020 Financial Results and Highlights Recent Progresson February 25, 2021 at 1:21 pm
Additional interim data expected in early Q2 from Phase 1/2 clinical trial of MRT5005 for the treatment of cystic fibrosis (CF) -- -- Two infectious disease mRNA vaccine programs expected to enter ...
- Insmed Reports Fourth Quarter and Full Year 2020 Financial Results and Provides Business Updateon February 25, 2021 at 4:34 am
Insmed Incorporated (Nasdaq:INSM), a global biopharmaceutical company on a mission to transform the lives of patients with serious and rare diseases, today reported financial results for the fourth ...
- Renowned Nutrition Expert Puts Rare Disease Day on the Radaron February 23, 2021 at 11:10 pm
To raise awareness of Rare Disease Day on February 28, Envara Health founder and nutrition expert Virginia A. Stallings, M.D., highlights the nutritional challenges and therapies for people living ...
- Global Cystic Fibrosis Market Size 2021 Top Manufacturers, Share, Growth Factor, Latest Trends, Type and Application Forecast 2026on February 23, 2021 at 1:06 am
Global “Cystic Fibrosis Market” report is a fact-based market research and important information to companies, ...
- How do patients with cystic fibrosis respond to COVID-19?on February 22, 2021 at 8:13 am
Based on clinical data, patients with cystic fibrosis (CF) don't appear to be especially susceptible to COVID-19, and when they do get infected, they don't seem to get sicker. But Ruobing (Ruby) Wang, ...
- Registration open for 30-day fitness challenge benefiting those with cystic fibrosison February 20, 2021 at 11:45 am
For most of us, taking a deep breath of fresh air is one of the simple pleasures in life. Fresh air fills our lungs as we inhale and leaves our ...
- Supplier of expensive cystic fibrosis medication yet to apply for Pharmac approvalon February 16, 2021 at 6:32 pm
Trikafta supplier yet to apply for Pharmac approval, but Parliament is warned the drug would be "extremely expensive" to fund.
- Cystic Fibrosis and COVID-19on February 16, 2021 at 1:31 pm
People with cystic fibrosis, or CF, don’t appear to be especially susceptible to COVID-19, and when they do get infected, they don’t seem to get sicker, based on clinical data so far. But Ruobing ...
via Bing News