Forum for Science, Industry and Business

Sponsored by:     3M 
Search our Site:

 

Discovery leads to effective treatment of painful skin condition

26.11.2009
Drug has broad implications for a wide range of genetic diseases

Researchers at Huntsman Cancer Institute, in collaboration with a worldwide group of physicians and scientists, have discovered a remarkable treatment for a rare, yet debilitating, skin condition.

The study, published online November 24 in Molecular Therapy describes a new treatment for pachyonychia congenita, an ultra-rare genetic skin condition caused by mutations in a gene called keratin. The disorder is characterized by painful, blistering calluses on the feet and limbs that limit a patient's ability to walk. Other skin and nail problems also occur.

The new treatment involves a relatively new class of drug called siRNA, and works by preventing the gene with the mutation from being expressed but permitting the healthy keratin genes to function normally. The study marked the first time that the skin of a human subject was treated with this type of drug. Researchers say that in this single patient trial the drug worked, had no serious side effects, and has vast potential because of its ability to specifically and potently target single molecules, making it an option for many other genetic diseases, including cancer.

"The result is exceptionally promising since it suggests that siRNAs can be used safely on the skin and also possibly to treat genetic disorders that would otherwise have few therapeutic options," says Sancy Leachman, M.D., Ph.D., the study's lead author. "The patient was treated with siRNA on her right foot and with placebo on the left foot. The callus on the right foot that received the siRNA fell off at the site of injection, but this did not happen on the left foot."

Since the injections of the drug are particularly painful, the next step in the research will be to develop a more patient friendly, perhaps topical delivery of the medication.

This study marks a major collaborative accomplishment among international physicians, scientists, pharmaceutical companies, and charitable foundation members.

Leachman is an investigator at Huntsman Cancer Institute, a dermatologist, and an associate professor in the Department of Dermatology at the University of Utah.

The study's principal author, Sancy Leachman, MD is available for interviews. Photo and video opportunities are available, including slides of the skin condition and visuals of siRNA. To schedule an interview, contact the office of public affairs at 801-587-7639.

The mission of Huntsman Cancer Institute (HCI) at The University of Utah is to understand cancer from its beginnings, to use that knowledge in the creation and improvement of cancer treatments, to relieve the suffering of cancer patients, and to provide education about cancer risk, prevention, and care. HCI is a National Cancer Institute-designated cancer center, which means that it meets the highest national standards for cancer care and research and receives support for its scientific endeavors. HCI is also a member of the National Comprehensive Cancer Network (NCCN) a not-for-profit alliance of the world's leading cancer centers, which is dedicated to improving the quality and effectiveness of care provided to patients with cancer.

Linda Aagard | EurekAlert!
Further information:
http://www.huntsmancancer.org

More articles from Health and Medicine:

nachricht Satellites, airport visibility readings shed light on troops' exposure to air pollution
09.12.2016 | Veterans Affairs Research Communications

nachricht Oxygen can wake up dormant bacteria for antibiotic attacks
08.12.2016 | Penn State

All articles from Health and Medicine >>>

The most recent press releases about innovation >>>

Die letzten 5 Focus-News des innovations-reports im Überblick:

Im Focus: Electron highway inside crystal

Physicists of the University of Würzburg have made an astonishing discovery in a specific type of topological insulators. The effect is due to the structure of the materials used. The researchers have now published their work in the journal Science.

Topological insulators are currently the hot topic in physics according to the newspaper Neue Zürcher Zeitung. Only a few weeks ago, their importance was...

Im Focus: Significantly more productivity in USP lasers

In recent years, lasers with ultrashort pulses (USP) down to the femtosecond range have become established on an industrial scale. They could advance some applications with the much-lauded “cold ablation” – if that meant they would then achieve more throughput. A new generation of process engineering that will address this issue in particular will be discussed at the “4th UKP Workshop – Ultrafast Laser Technology” in April 2017.

Even back in the 1990s, scientists were comparing materials processing with nanosecond, picosecond and femtosesecond pulses. The result was surprising:...

Im Focus: Shape matters when light meets atom

Mapping the interaction of a single atom with a single photon may inform design of quantum devices

Have you ever wondered how you see the world? Vision is about photons of light, which are packets of energy, interacting with the atoms or molecules in what...

Im Focus: Novel silicon etching technique crafts 3-D gradient refractive index micro-optics

A multi-institutional research collaboration has created a novel approach for fabricating three-dimensional micro-optics through the shape-defined formation of porous silicon (PSi), with broad impacts in integrated optoelectronics, imaging, and photovoltaics.

Working with colleagues at Stanford and The Dow Chemical Company, researchers at the University of Illinois at Urbana-Champaign fabricated 3-D birefringent...

Im Focus: Quantum Particles Form Droplets

In experiments with magnetic atoms conducted at extremely low temperatures, scientists have demonstrated a unique phase of matter: The atoms form a new type of quantum liquid or quantum droplet state. These so called quantum droplets may preserve their form in absence of external confinement because of quantum effects. The joint team of experimental physicists from Innsbruck and theoretical physicists from Hannover report on their findings in the journal Physical Review X.

“Our Quantum droplets are in the gas phase but they still drop like a rock,” explains experimental physicist Francesca Ferlaino when talking about the...

All Focus news of the innovation-report >>>

Anzeige

Anzeige

Event News

ICTM Conference 2017: Production technology for turbomachine manufacturing of the future

16.11.2016 | Event News

Innovation Day Laser Technology – Laser Additive Manufacturing

01.11.2016 | Event News

#IC2S2: When Social Science meets Computer Science - GESIS will host the IC2S2 conference 2017

14.10.2016 | Event News

 
Latest News

Researchers identify potentially druggable mutant p53 proteins that promote cancer growth

09.12.2016 | Life Sciences

Scientists produce a new roadmap for guiding development & conservation in the Amazon

09.12.2016 | Ecology, The Environment and Conservation

Satellites, airport visibility readings shed light on troops' exposure to air pollution

09.12.2016 | Health and Medicine

VideoLinks
B2B-VideoLinks
More VideoLinks >>>