Cystic Fibrosis: Antioxidant-Enriched MVI May Decrease Respiratory Illnesses

MedicalResearch.com Interview with:

Scott D Sagel MD PhD Professor of Pediatrics University of Colorado School of Medicine Aurora, Colorado

Dr. Sagel

Scott D Sagel MD PhD
Professor of Pediatrics
University of Colorado School of Medicine
Aurora, Colorado

MedicalResearch.com: What is the background for this study?

Response: Inflammation is an important feature of cystic fibrosis (CF) lung disease and contributes to lung damage and lung function decline in CF. We need safe and effective anti-inflammatory treatments in CF. Anti-oxidant therapy has been an area of promise, but with mixed results in CF.

This clinical trial, conducted at 15 CF centers affiliated with the cystic fibrosis Foundation Therapeutics Development Network, enrolled 73 patients who were 10 years and older (average age 22 years), with pancreatic insufficiency, which causes malabsorption of antioxidants. Subjects were randomized to either a multivitamin containing multiple antioxidants including carotenoids such as beta(β)-carotene, tocopherols (vitamin E), coenzyme Q10 (CoQ10), and selenium or to a control multivitamin without antioxidant enrichment. The antioxidants used in the study were delivered in a capsule specifically designed for individuals with difficulties absorbing fats and proteins, including those with cystic fibrosis.

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Widely Use Antibacterial in Hand Sanitizers and Toothpaste Can Attack Biofilms

MedicalResearch.com Interview with:

Christopher M. Waters PhD Departments of Microbiology and Molecular Genetics BEACON Center for The Study of Evolution in Actio Michigan State University East Lansing, MI 

Dr. Waters

Christopher M. Waters PhD
Departments of Microbiology and Molecular Genetics
BEACON Center for The Study of Evolution in Actio
Michigan State University
East Lansing, MI 

MedicalResearch.com: What is the background for this study? What are the main findings?

Response: Our research really centers on understanding and targeting bacterial biofilms. These are multicellular communities of bacteria encased in a slimy matrix that protects them from the immune system and antibiotic treatment during infections. One of the most common types of biofilm infections is in the lungs of cystic fibrosis by the bacterium Pseudomonas aeruginosa. CF patients can become chronically colonized by P. aeruginosa, and antibiotics are not able to clear these infections.

Our idea was can we find other molecules that make antibiotics more effective at killing biofilms? To this end, we screened about 6,000 compounds for those that would make tobramycin more effective at killing P. aeruginosa biofilms, and one of the best hits we found was the antimicrobial triclosan that has been widely used for decades in hand sanitizers, soaps, and tooth paste. Although neither triclosan nor tobramycin can kill biofilms alone, the combination is 100X more effective against virtually every P. aeruginosa strain tested. It also worked against other bacteria that commonly infect cystic fibrosis lungs such as Staphylococcus aureus and Burkholderia cenocepacia.

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Cystic Fibrosis Patients Survive Ten Years Longer in Canada than in US

MedicalResearch.com Interview with:
Dr. Anne L. Stephenson, MD, PhD

St. Michael’s Hospital
Toronto Canada

MedicalResearch.com: What is the background for this study? What are the main findings?

Response: Both Canada and the US have maintained national registries on individuals with cystic fibrosis (CF) dating back to the 1970s. Previous reports suggested that survival differed between the two countries however direct comparisons of survival estimates between national registry reports were limited because of differences in methodologies used, data processing techniques and possible differences in the patients captured within each registry.

We aimed to compare survival in  cystic fibrosis between Canada and the US to determine if differences existed when we applied a systematic and standardized approach.

Our analysis showed that between 1990 and 2013, survival for individuals with CF increased in both countries, however, the rate of increase was faster in Canada compared to the USA. The survival gap widened at two distinct time points: 1995 and 2005.

In the contemporary period between 2009 and 2013, the median age of survival for individuals with cystic fibrosis in Canada was found to be 50.9 years compared to 40.6 years in the US. Overall, the risk of death was 34% lower for Canadians compared Americans with CF after adjusting for markers of disease severity. When US CF subjects were classified by insurance status, we found a 77% lower risk of death among Canadians with CF compared to Americans who indicated unknown or no insurance, a 44% lower risk compared to Americans receiving continuous Medicare/Medicaid, and a 36% lower risk in Canadians compared to Americans receiving intermittent Medicare/Medicaid. The risk of death for Americans with private insurance was not statistically different from that of Canadians with cystic fibrosis .

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New Step Toward Gene Cure For Cystic Fibrosis

MedicalResearch.com Interview with:
Dragana Vidovic and Marianne Carlon

Laboratory for Molecular Virology and Gene Therapy
KU Leuven, Belgium

Medical Research: What is the background for this study? What are the main findings?

Response: Cystic fibrosis (CF) or mucoviscidosis is a genetic disorder caused by mutations in the CFTR gene which codes for a chloride/bicarbonate channel that regulates fluid secretion across the epithelium in different organs, for instance, the airways and the gastrointestinal tract. In the cells of CF patients, these anion channels are dysfunctional or even absent leading to the formation of sticky mucus. Persistent airway infection is the major clinical manifestation. The symptoms can be treated, but there is no cure for the disorder. Gene therapy holds promise to cure the disease. Previous studies suggested that the treatment is safe, but largely ineffective for Cystic fibrosis patients. However, as gene therapy has recently proven successful for inherited disorders such as haemophilia and congenital blindness, we wanted to re-examine its potential for CF.

Here we developed an improved gene therapy treatment based on inserting the CFTR gene into the genome of a recombinant AAV viral vector (rAAV), which is derived from the relatively innocent AAV virus. We used this vector to “smuggle” a healthy copy of the CFTR gene into the affected cells. We administered rAAV to CF mice via their airways. Most of the mice recovered. In patient-derived intestinal cell cultures or organoids, chloride and fluid transport was restored.

Medical Research: What does the study add to the field?

Response: Development of Cystic fibrosis gene therapy requires a thorough preclinical examination of a candidate vector in relevant cell and animal models before being administered to humans. Here, both in mice with Cystic fibrosis and in mini-guts or intestinal organoids derived from Cystic fibrosis patients, this approach yielded positive results setting the stage for further validation in large animal models which mimic the CF patient situation more faithfully. We believe that our study will revive the interest in CF gene therapy as a promising, mutation-independent approach to ultimately cure Cystic fibrosis.

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Cystic Fibrosis Survival Improved By Decades

Anne Stephenson MD, PhD Division of Respirology The Toronto Adult Cystic Fibrosis Centre St. Michael's Hospital Toronto, ON MedicalResearch.com Interview with:
Anne Stephenson MD, PhD
Division of Respirology
The Toronto Adult Cystic Fibrosis Centre
St. Michael’s Hospital Toronto, ON

Medical Research: What is the background for this study? What are the main findings?

Dr. Stephenson: Cystic Fibrosis is progressive genetic disease that results in very thick secretions in various organs such as the lungs, pancreas, and digestive tract. Over time, these thick secretions damage organs in particular, the lungs, which results in respiratory failure due to recurrent chest infections. Cystic Fibrosis patients are also at high risk for malnutrition due to the inability to absorb food which is associated with poor survival. In the 1960s, people with Cystic Fibrosis died at a very young age and in fact, parents who had a child born with Cystic Fibrosis at that time were told that there was a 50% chance their child would not live to attend kindergarten. Over the last several decades, we have seen a significant increase in the survival of individuals with CF. Individuals born with Cystic Fibrosis today can expect to not only attend kindergarten, but complete high school, perhaps attend college or university, have a career, get married or have children as people are living well into adulthood with this disease. The median age of survival in Cystic Fibrosis today is approximately 50 years of age in Canada which is quite remarkable.
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