Corporate Banner
Satellite Banner
Technology
Networks
Scientific Communities
 
Become a Member | Sign in
Home>News>This Article
  News
Return

First Gene Therapy Study in Human Salivary Gland Shows Promise

Published: Tuesday, November 06, 2012
Last Updated: Tuesday, November 06, 2012
Bookmark and Share
Treatment proves safe and effective, helps cancer survivors with chronic dry mouth.

Gene therapy can be performed safely in the human salivary gland, according to scientists at the National Institute of Dental and Craniofacial Research (NIDCR), part of the National Institutes of Health.

This finding comes from the first-ever safety, or Phase I, clinical study of gene therapy in a human salivary gland. Its results, published this week in the Proceedings of the National Academy of Sciences, also show that the transferred gene, Aquaporin-1, has great potential to help head and neck cancer survivors who battle with chronic dry mouth. Aquaporin-1 encodes a protein that naturally forms pore-like water channels in the membranes of cells to help move fluid, such as occurs when salivary gland cells secrete saliva into the mouth.

These initial results clear the way for additional gene therapy studies in the salivary glands. Although sometimes overlooked, salivary glands present an ideal target for gene therapy. They are easily accessible and, once a gene is introduced, it has no obvious escape route into the bloodstream, where it can have unintended consequences.

“You cannot imagine how fulfilling it is to jot down an idea on a napkin in 1991 and then see it enter a clinical trial and help people.,” said Bruce Baum, D.M.D., Ph.D., lead author on the study and recently retired NIDCR scientist who spent the last 21 years moving gene therapy in the salivary glands from the research bench to the clinic. “Can a scientist ask for anything better?”

Baum's interest in helping head and neck cancer survivors dates to the early 1980s. While attending to patients in the NIDCR's Dry Mouth Clinic, Baum encountered numerous people with head and neck cancer who had received radiation therapy to shrink their tumors. The radiation, while effective in treating cancer, had inadvertently damaged nearby salivary glands, compromising their ability to secrete saliva into the mouth.

Baum said he was thoroughly frustrated at the time because he had no effective moisture-restoring treatments to offer most patients. They had beaten cancer, but the radiation had left them with a permanent parched sensation in their mouths that diminished their quality of life and often led to chronic oral problems, such as difficulty swallowing, inflammation, infection, bad breath, and pain.

In the early 1990s, as the first gene-therapy studies entered research clinics, Baum saw an opportunity to make a difference. An initial napkin sketch of the procedure and 15 years of research later, Baum and his colleagues had assembled a compelling scientific case in animal studies that the transferred Aquaporin-1 gene, once expressed, will create new water channels in the impermeable salivary gland cells and allow water to flow through them. After rigorous reviews by NIH and the U. S. Food and Drug Administration, the Phase I protocol was launched and the first patients treated in 2008.

The scientists gave 11 head and neck cancer survivors a single-dose injection of the Aquaporin-1 gene directly into one of their two parotid salivary glands, the largest of the major salivary glands. The gene was packaged in a disabled, non-replicating adenovirus, the cause of the common cold when intact but incapable of causing a cold in this case. As is standard in gene therapy studies, the virus served as the vector, or Trojan horse, to deliver the gene into the cells lining the salivary gland.

The scientists found that five participants had increased levels of saliva secretion, as well as a renewed sense of moisture and lubrication in their mouths, within the study's first 42 days, the period covered in this report. Of the six who didn't benefit from gene therapy, none had serious side effects. The most common side effect was a transient and relatively minor immune response against the disabled adenovirus.

“It is time to evaluate a different vector to deliver the Aquaporin-1 gene, one that will cause only a minimal immune response,” said Baum. “But these data will serve as stepping stones for other scientists to improve on this first attempt in the years ahead. The future for applications of gene therapy in the salivary gland is bright.”

The National Institute of Dental and Craniofacial Research is the Nation's leading funder of research on oral, dental, and craniofacial health. For more information about NIDCR and its programs, visit http://www.nidcr.nih.gov/.


Further Information

Join For Free

Access to this exclusive content is for Technology Networks Premium members only.

Join Technology Networks Premium for free access to:

  • Exclusive articles
  • Presentations from international conferences
  • Over 4,000+ scientific posters on ePosters
  • More Than 5,300+ scientific videos on LabTube
  • 35 community eNewsletters


Sign In



Forgotten your details? Click Here
If you are not a member you can join here

*Please note: By logging into TechnologyNetworks.com you agree to accept the use of cookies. To find out more about the cookies we use and how to delete them, see our privacy policy.

Related Content

Exploring the Genome of the River Blindness Parasite
Researchers have decoded the genome of the parasite that causes the skin and eye infection known as river blindness.
Wednesday, December 07, 2016
Gene-Editing Improves Vision in Blind Rats
Scientists developed a targeted gene-replacement technique that can modify genes in both dividing and non-dividing cells in living animals.
Wednesday, December 07, 2016
Uncovering Cerebral Malaria’s Deadly Agents
NIH scientists film inside mouse brains to uncover biology behind the disease.
Wednesday, December 07, 2016
Study to Assess Shorter-Duration Antibiotics in Children
Physicians plan a clinical trial to evaluate whether short course anti-biotics are effective at treating CAP in children.
Wednesday, November 30, 2016
First New HIV Vaccine Study for Seven Years Begins
South Africa hosts historic clinical trial of experimental HIV vaccine aiming to safely prevent HIV infection.
Wednesday, November 30, 2016
Antibody Protects Mice from Zika Infection
Researchers develop human-derived antibody protected pregnant mice and their developing fetuses from Zika infection.
Wednesday, November 23, 2016
Food Additives Promote Inflammation, Colon Cancer
Dietary emulsifiers promoted colon cancer in a mouse model by altering gut microbes and increasing gut inflammation.
Wednesday, November 23, 2016
Protein-Folding Gene Helps Heal Wounds
Researchers identified a protein that dramatically accelerates wound healing in animal models.
Wednesday, November 23, 2016
More Immunotherapy Options Approved for Lung Cancer
The FDA has approved a new immunotherapy drug for certain patients with non-small cell lung cancer.
Monday, November 21, 2016
Big Data for Infectious Disease Surveillance
NIH-led effort examines use of big data from health records and other digital sources for uses in infectious disease surveillance.
Tuesday, November 15, 2016
Potential Therapies Against Drug-Resistant Bacteria Identified
Researchers create new identification method for drug and drug combinations that may combat resistant infections.
Thursday, November 10, 2016
Testing Zika Vaccine in Humans Begins
The first of five planned clinical trials to test ZPIV vaccine in humans has begun.
Tuesday, November 08, 2016
Genetic Markers Predict Malaria Treatment Failure
By comparing 297 parasite genomes to a reference malaria parasite genome, researchers have identified two genetic markers that are strongly associated with the parasites’ ability to resist piperaquine.
Monday, November 07, 2016
Cannabinoid Receptor Structure Revealed
Scientists provided a detailed view of the primary molecule through which cannabinoids exert their effects on the brain. The findings might help guide the design of more targeted medicines with fewer side effects.
Wednesday, November 02, 2016
NIH Researchers Unveil New Wound-Healing Role for Protein-Folding Gene in Mice
The study found that topical treatment of an Hsp60-containing gel dramatically accelerates wound closure in a diabetic mouse model.
Friday, October 28, 2016
Scientific News
Big Genetics in BC: The American Society for Human Genetics 2016 Meeting
Themes at this year's meeting ranged from the verification, validation, and sharing of data, to the translation of laboratory findings into actionable clinical results.
Stem Cells in Drug Discovery
Potential Source of Unlimited Human Test Cells, but Roadblocks Remain.
Automated Low Volume Dispensing Trends
Gain a better understanding of the current and future market requirements for fully automated LVD systems.
Personality Traits, Psychiatric Disorders Linked to Specific Genomic Locations
Researchers have unearthed genetic correlations between personality traits and psychiatric disorders.
Forensic 3D Documentation of Skin Injuries
In this study, the validity of using photogrammetry for documenting injuries in a pathological context was demonstrated.
3-D Printed Dog’s Nose Improves Vapor Detection
By mimicking how dogs get their whiffs, a team of government and university researchers have demonstrated that “active sniffing” can improve by more than 10 times the performance of current technologies that rely on continuous suction to detect trace amounts of explosives and other contraband.
New Markers for Forensic Body-fluid Identification
University of Bonn researchers have successfully identified specific Micro-RNA signatures to help forensically identify body fluids.
Genetics Control Regenerative Properties Of Stem Cells
Researchers define how genetic factors control regenerative properties of blood-forming stem cells.
Major Neuroscience Initiative Launched
Tianqiao and Chrissy Chen Institute invest $115 million to further expand neuroscience research, while Caltech construct $200 million biosciences complex.
Making It Personal
Cancer vaccine linked to increased immune response against leukemia cells.
Scroll Up
Scroll Down
Skyscraper Banner

SELECTBIO Market Reports
Go to LabTube
Go to eposters
 
Access to the latest scientific news
Exclusive articles
Upload and share your posters on ePosters
Latest presentations and webinars
View a library of 1,800+ scientific and medical posters
4,000+ scientific and medical posters
A library of 2,500+ scientific videos on LabTube
5,300+ scientific videos
Close
Premium CrownJOIN TECHNOLOGY NETWORKS PREMIUM FOR FREE!