Navigation Links
U of M sets course for cure of fatal childhood skin disease
Date:6/3/2008

Physicians at the University of Minnesota and University of Minnesota Children's Hospital, Fairview have set the path to a cure for a young boy's fatal genetic skin disease, recessive dystrophic epidermolysis bullosa (RDEB), by using a cord blood and bone marrow transplant. Nate Liao, a 25-month-old from Clarksburg, N.J., underwent the experimental therapy in October 2007.

"We have established a new standard of care for these EB patients, beginning with Nate," said John Wagner, M.D., the lead University of Minnesota Medical School physician who developed the clinical trial. "Nate's quality of life is forever changed."

Because they lack collagen type VII, children with RDEB have skin that is exquisitely delicate. The skin and lining of their gastrointestinal (GI) tract is fragile; tearing and blistering occur with minimal friction. Coughing and vomiting often result in tears in the lining of the esophagus and stomach. Those affected must have their entire body continuously wrapped in bandages. Those who do not succumb from malnutrition and infection in childhood will acquire a uniformly fatal, aggressive cancer of the skin in young adulthood.

In collaboration with Angela M. Christiano, Ph.D., professor of dermatology and genetics and development at Columbia University Medical Center (New York, N.Y.), and investigators at Asahikawa Medical College (Asahikawa, Japan), and Jefferson Medical College (Philadelphia, Penn.), University of Minnesota researchers, Jakub Tolar, M.D., Ph.D., and Bruce Blazar, M.D., discovered that certain stem cells found in bone marrow could correct the biochemical defect in RDEB in a mouse model of the disease. Marrow-derived stem cells greatly lengthened the life expectancy of the mice and healed existing blisters. Further testing by Columbia demonstrated that for the first time, these mice were producing collagen type VII and anchoring fibrils, the structures needed to bind skin to the body.

This is the first time physicians have approached the treatment of RDEB from a systemic perspective, using marrow-derived stem cells as a means to replace the missing protein, collagen type VII, throughout the body. Through the infusion of cells obtained from a healthy donor, the stem cells produce collagen type VII and correct the underlying genetic defect.

In October 2007, Nate Liao received marrow- and umbilical cord blood-derived stem cells and progenitor cells from his healthy, tissue-matched brother. Over the next six months, the skin and lining of his GI tract slowly improved, and skin biopsies on days 60, 130, and 200 documented increasing amounts of collagen type VII. By day 130, Nate's skin and the lining of his GI tract were beginning to show clinical signs that his skin was anchoring to his body.

Wagner and his team sought an external review of the skin biopsies. Photographs of the biopsies have been sent to dermatopathologists in London, England, and Portland, Ore., for independent analysis. Based on the success seen in Nate, Wagner will enroll additional RDEB patients into the clinical trial. Jacob Liao, Nate's brother who also has RDEB, received an unrelated donor cord blood transplant on May 30.


'/>"/>

Contact: Molly Portz
mportz@umn.edu
612-625-2640
University of Minnesota
Source:Eurekalert

Related medicine news :

1. World-Renowned Scientists to Lead Immunology Course
2. Gerresheimer Continues Expansion Course
3. Direct Response TV Course on Nutraceutical/Cosmeceutical Products to Premiere in New York City
4. Robotic Radiosurgery Course Yields Record Attendance
5. Genetic Changes May Dictate Course of Acute Myeloid Leukemia
6. Good sexual intercourse lasts minutes, not hours, therapists say
7. HCCS Partners With UHC to Deliver Cultural Competency Courses
8. Baxa Corporation Announces Safe Handling and Preparation of Hazardous Drugs Course Offering at its STAR Center Training Facility
9. HOYA ConBio Partners With Advanced Laser Training to Offer Complementary Laser Certification Course in Dentistry
10. Despite popularity, researcher finds not everyone can successfully learn through online courses
11. ESFs EMRC strengthens training course on clinical trials management
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:6/28/2017)... ... June 28, 2017 , ... ... recognized for excellence in employee engagement. Omaha-based C&A Industries, a national leader ... Most Engaged Workplaces™ in North America for 2017. The annual award, ...
(Date:6/28/2017)... ... 28, 2017 , ... Hayes, Inc., a leading provider of ... speaker lineup* for its second annual Client Symposium. Joining the symposium this year ... Fernandez of Privia Health; and Lisa Tourville of Anthem. , “We are ...
(Date:6/28/2017)... ... June 28, 2017 , ... ... corporate governance program for mid-market executives as a kick-off to the fifth annual ... Sept. 24, 2017 on the University of Pennsylvania campus, followed by the two-day ...
(Date:6/28/2017)... ... June 28, 2017 , ... Schneider Insurance ... assistance to clients in southern Montana, is announcing a focused charity effort to ... The outreach programs offered by Zoo Montana provide students with current knowledge on ...
(Date:6/27/2017)... ... 2017 , ... Excel Medical, the leader in Next-Generation Medical ... Executive Vice President, Sales and Marketing. Finnegan is a widely known industry leader ... leadership. He has received industry recognition for innovations and new approaches to sales ...
Breaking Medicine News(10 mins):
(Date:6/16/2017)... years ago today, the Monterey Pop Festival opened its gates ... Francisco "Summer of Love."  To celebrate the anniversary, Northern Light® ... market research portals that it will begin delivering to customers ... "Summer of Love (For Our Customers)."  ... Northern Light ...
(Date:6/13/2017)... Zimmer Biomet Holdings, Inc. (NYSE and SIX: ZBH), a global ... and Drug Administration (FDA) has notified the Company that the ... Zhejiang, China manufacturing facility has been closed ... of the Warning Letter related to our ... the progress we have made in our ongoing quality and ...
(Date:6/9/2017)... June 9, 2017 AirXpanders, Inc. (ASX: AXP) ... the design, manufacture, sale and distribution of the AeroForm® ... progress of its commercial roll-out in the ... more than one hundred (100) medical institutions and health ... offers a needle-free alternative for women who choose reconstructive ...
Breaking Medicine Technology: