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:6/27/2016)... ... June 27, 2016 , ... "FCPX editors can now reveal their ... Cut Pro X," said Christina Austin - CEO of Pixel Film Studios. , ... Pro X users can now reveal the media of their split screens with ...
(Date:6/26/2016)... PLAINSBORO, N.J. (PRWEB) , ... June 27, 2016 , ... ... same sources, yet in many ways they remain in the eye of the beholder, ... Oncology (EBO), a publication of The American Journal of Managed Care. For the full ...
(Date:6/26/2016)... , ... June 26, 2016 , ... ... once they have been diagnosed with endometriosis. These women need a treatment plan ... require a comprehensive approach that can help for preservation of fertility and ultimately ...
(Date:6/25/2016)... Lewisville, TX (PRWEB) , ... June 25, 2016 , ... ... in the United States, named Dr. Sesan Ogunleye, as the Medical Director of its ... be the facility Medical Director of our new Mesquite location,” said Dr. James M. ...
(Date:6/24/2016)... ... June 24, 2016 , ... A recent article published ... unfamiliar with. The article goes on to state that individuals are now more comfortable ... less common operations such as calf and cheek reduction. The Los Angeles area medical ...
Breaking Medicine News(10 mins):
(Date:6/23/2016)... Bracket , a leading clinical trial technology ... outcomes platform, Bracket eCOA (SM) 6.0, at the 52 ... 30, 2016 in Philadelphia , Pennsylvania.  A ... product of its kind to fully integrate with RTSM, will ... 6.0 is a flexible platform for electronic clinical outcomes assessments ...
(Date:6/23/2016)... , June 23, 2016 Revolutionary ... Oticon , industry leaders in advanced audiology ... of Oticon Opn ™, the world,s first internet ... possibilities for IoT devices.      (Photo: ... introduces a number of ,world firsts,: , ...
(Date:6/23/2016)... , June 23, 2016  Experian ... integrating and transforming the patient payment and ... several innovative new products and services that ... its revenue cycle offerings. These award-winning solutions ... efficient workflows, remain compliant in an ever-changing ...
Breaking Medicine Technology: