Stem Cell Therapy for Basement Membrane Diseases (BIDMC 905)

Category:    Therapeutics - Methods

KeyWords:  Epithelial;  Transplantation;  Inflammation;  

BIDMC ID:    905

Abstract:

A novel therapy to prevent or repair the damaged GBM by administering stem cells which then become kidney epithelial cells and provide normal GBM components, such as collagen. Certain kidney diseases result from defects in the glomerular basement membranes (GBM), crucial to proper kidney function. Alport’s Syndrome, for example, is an inherited disease of kidney inflammation (nephritis) resulting from a mutation in collagen, a key GBM component. Treatment options for diseases with impaired GBM are dialysis or renal transplantation. While these treatments may increase a patient’s survival, quality of life remains poor.

Inventor:   Lead Investigator: R. Kalluri, Ph.D.

Commercial Opportunity:

This technology is broadly applicable to diseases which exhibit other basement membrane defects, beyond GBM, including: Knoblach Syndrome Hematuria Epidermolysis bullosa Diabetic Nephropathy Opportunities for Licensing Available

Competitive Advantages:

Stem cells can be administered into the blood stream or by bone marrow transplant Eliminate the need for dialysis Only a small percentage of cells are needed to provide the proper GBM components

Related Publications:

“Bone-marrow-derived stem cells repair
  basement membrane collagen defects and  
  reverse genetic kidney disease”
PNAS May 9, 2006 (103), 19, pgs 7321-7326

State of Development:

Pre-clinical studies with stem cells transplanted into a mouse model of Alport Syndrome:

• Restored kidney function as a measure of
    urine albumin excretion
• Restored kidney cell morphology
• Inhibited progression of tubular damage &
    fibrosis
• Attenuated kidney disease & decreased
    proteinuria
• Stem cells differentiated into kidney cells &
    expressed GBM proteins

Related Technology URL:

http://kalluri.med.harvard.edu

Patent Status:

National Phase pending

TVO Contact Info:
     Christine Jost
     Associate Director TVO
     cjost@bidmc.harvard.edu
     Phone: 617-667-4239   Fax: 617-667-0646

     Beth Israel Deaconess Medical Center
     Technology Ventures Office  Room: BR-0200
     330 Brookline Avenue
     Boston, MA 02215