Navigation Links
U of M performs first systemic therapy for fatal childhood disease
Date:11/2/2007

University of Minnesota Childrens Hospital, Fairview physicians have performed the first bone marrow and cord blood transplant to treat recessive dystrophic epidermolysis bullosa (RDEB).

Children with RDEB lack a protein that anchors skin to the body, resulting in fragile skin that sloughs off with little movement or friction. They suffer painful wounds and must be bandaged at all times to protect their skin from further damage and infection. The 18-month-old boy who was transplanted has the most severe form of RDEB, which also causes skin to slough off on the inside of the body, affecting the mouth, esophagus, and gastrointestinal tract. EB is genetic and severe forms are always fatal. Those who live to be young adults get an aggressive form of skin cancer called squamous cell carcinoma.

With the help of an EB mouse model and in collaboration with investigators at Columbia University, University of Minnesota researchers were able to correct the disease in mice using bone marrow. They tested various types of adult stem cells to determine which would give rise to the development of type VII collagen the protein people with RDEB lack. One type of immature cells from bone marrow proved to be the best at producing anchoring fibrils that bind the skin to the body.

This is the first time physicians have approached EB from a systemic perspective, using transplant as a means to rid the body of the defective blood system and replace it with a healthy blood system that produces type VII collagen.

Our goal is to determine the usefulness of stem cells, whether from the umbilical cord blood or adult tissues like bone marrow, in the treatment of human disease, said John E. Wagner, M.D., professor of Pediatrics and director of the Division of Hematology, Oncology, and Blood and Marrow Transplantation and director of clinical research of the Stem Cell Institute at the University of Minnesota. There are hundreds of thousands of children and adults waiting for new breakthroughs in stem cell research, and time is never enough. In two years, the team was able to move this project forward remarkably fastfrom testing in animal models to treating patients. Time will tell whether this risky treatment will work as effectively in humans. But, RDEB is a horribly debilitating, life-threatening disease with no existing curative therapy.

The boy received both umbilical cord blood and bone marrow from a perfectly matched sibling. If the results mimic the animal model, doctors anticipate the healthy blood system will aid in the skins ability to produce type VII collagen necessary to anchor the skin and lining cells of the gastrointestinal tract to the body. Doctors anticipate in early 2008approximately 100 days after transplantthey will be able to judge whether this the treatment helped.

This represents a real change in thinking within the dermatological community. The possibility of this approach compels us to explore more broadly the way some skin diseases are typically treated, said Maria Hordinsky, M.D., head of the Department of Dermatology at the University of Minnesota and member of the care team.


'/>"/>

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

Related medicine news :

1. Doctors At The TMCH Performs Their First Open-heart Surgery
2. West Bengal Performs Yagna to Ward off Dengue, Malaria
3. Vanderbilt Performs States First Stem Cell Heart Regeneration Therapy
4. Spanish Doctors Performs Worlds First Hand Transplantation Successfully
5. UK Hospital Performs Heart Surgery Using Robotic Arm
6. First Vaccine Designed for Africa Cleared for Testing in Humans
7. Infant receives first bloodless liver transplant
8. Oracle Corp. to help build worlds first "Digital Hospital"
9. Ajanta Launches Worlds First Once-A-Day Nimesulide Oral Formulation
10. First human clone is near
11. First Artificial Heart patient has Major setback
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:10/13/2017)... New York, NY (PRWEB) , ... October 13, 2017 , ... ... most influential people in business to advocate for action towards gender equality at their ... 18,000 views from around the globe, and reached a social audience of over 3 ...
(Date:10/13/2017)... (PRWEB) , ... October 13, 2017 , ... ... will be giving viewers the lowdown on sciatica in a new episode of ... that focuses on current events and innovation and investigates each subject in-depth with ...
(Date:10/13/2017)... ... , ... “America On The Brink”: the Christian history of the United States ... creation of published author, William Nowers. Captain Nowers and his wife, Millie, have ... thirty years in the Navy. Following his career as a naval aviator and ...
(Date:10/12/2017)... ... October 12, 2017 , ... Planet Fitness, one of the largest and fastest ... to open a flagship location in Covington, LA at 401 N. U.S. Highway 190, ... next to Office Depot in the Holiday Square shopping center. Its location allows it ...
(Date:10/12/2017)... ... October 12, 2017 , ... The American College of Medical Informatics (ACMI) will ... during the Opening Session of AMIA’s Annual Symposium in Washington, D.C. AMIA’s Annual ... F. Collen, a pioneer in the field of medical informatics, this prestigious award is ...
Breaking Medicine News(10 mins):
(Date:10/2/2017)... -- Diplomat Pharmacy, Inc. (NYSE: DPLO ) ... , and named its founder as Diplomat,s chief information ... Tennessee , will operate under Diplomat subsidiary Envoy ... for health care partners to include IT outsourcing, consulting, ... "In an interoperable world, technology delivers comprehensive insight and ...
(Date:9/28/2017)... -- Hill-Rom Holdings, Inc. (NYSE: HRC), will host its ... on Friday, November 3, 2017, beginning at 7:00 a.m. ... 8:30 a.m. (CDT) / 9:30 a.m. (EDT). ... and guidance for 2018, Hill-Rom executives will also highlight ... and long-range financial outlook through 2020. ...
(Date:9/25/2017)... 25, 2017  EpiVax, Inc., a leader in ... immune-engineering today announced the launch of EpiVax Oncology ... personalized therapeutic cancer vaccines. EpiVax has provided $500,000 ... to enabling technologies to the new precision immunotherapy ... EpiVax Oncology as Chief Executive Officer. Gad brings ...
Breaking Medicine Technology: