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Stem Cell Therapy for Severe Forms of Epidermolysis Bullosa

 

CLINICAL TRIAL

This is a single institution trial to determine the effectiveness of stem cells from closely matched related and unrelated donors to replace missing or defective proteins responsible for severe forms of epidermolysis bullosa.

Eligibility Criteria

  1. Patients aged < 25 years of age
  2. Diagnosis of a severe form of EB characterized by collagen, laminin, integrin or plakin deficiency
  3. Adequate organ function
  4. HLA matched related or unrelated marrow donor or partially HLA matched unrelated umbilical cord blood donor

Ineligibility Criteria

  1. Active infection
  2. History of HIV infection
  3. Evidence of squamous cell carcinoma

If you or your child may meet the above criteria, you may be eligible to come to the University of Minnesota for the proposed treatment. Prior to evaluation and screening to confirm eligibility, the following information is requested:

  1. Clinical summary from a treating physician, including history of prior surgeries, transfusions, skin grafts, medications, allergies, infections
  2. DNA mutation report if available
  3. Skin biopsy report (including immunofluorescent staining)
  4. HLA typing on child or adult with severe EB and unaffected siblings (instructions will be provided by our team)

SCREENING VISIT

The screening visit will involve skin biopsies, blood tests, photographs, assessment of skin fragility, and genetic testing. In addition, the visit will entail review of the clinical history and detailed to review of what is known and unknown regarding the proposed treatment procedure using stem cell populations from an allogeneic donor.

Based on this information, you or your child may need additional evaluations (e.g., dermatology, surgery, gastroenterology).
If you would like more information or have questions regarding the study, please contact John E. Wagner, M.D. at 612-626-2961.

How your support for EB Research at the University of Minnesota will be used?

With additional funds, we will search for more effective, safer cures. For example, our research team will search for better ways of isolating the cells responsible for the correction of EB and enhancing the rate of recovery.

Funds will also be used to expand the breadth of clinical activities to meet all the needs of this unique patient population and support the associated clinical trials.

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