Synergistic Co-Treatment with anti-HDAC6 for Adult Onset Polycystic Kidney Disease

Case ID:
C14531
Disclosure Date:
1/6/2017
Description:
UNMET NEED: Adult-onset polycystic kidney disease (ADPKD) is one of the most prevalent, potentially lethal, monogenic human disorders. Disease progression causes fluid filled cysts to grow in the kidneys, which can lead to declining kidney function and eventually kidney failure. More than 50% of patients afflicted with ADPKD eventually develop end stage kidney disease and require dialysis or kidney transplantation. Here, inventors identified a novel target when modulated in combination with inhibition of histone deacetylase 6 (HDAC6) shows potent activity to treat renal diseases, such as ADPKD. Advantages include:
  • Synergistic effect of co-inhibition of HDAC6 and a newly-discovered target
  • Possible combination strategy to treat renal diseases, including renal cystic diseases and polycystic renal diseases such as ADPKD 
TECHNICAL OVERVIEW: ADPKD causes cellular abnormalities such as increased proliferation, fluid secretion, and changes in cyclic AMP (cAMP) concentrations and signaling pathways. Johns Hopkins researchers were able to demonstrate that co-inhibition of HDAC6 and a herein-disclosed new target synergistically influence cAMP and Calcium levels in Madin-Darby canine kidney (MDCK) cells. Because cAMP and Ca2+ affect cyst growth, this combination strategy would result in improved efficacy compared to HDAC6 inhibition alone.
 
STAGE OF DEVELOPMENT: In Vitro Data
 
DISEASE INDICATION: Polycystic Kidney Disease
 
INVENTORS: Liudmila Cebotaru, William Guggino
 
PATENT STATUS: Provisional pending
Patent Information:
Title App Type Country Serial No. Patent No. File Date Issued Date Expire Date Patent Status
INHIBITION OF STROMAL INTERACTION MOLECULE 1 (STIM1) AS A COTREATMENT FOR ADULT ONSET POLYCYSTIC KIDNEY DISEASE (ADPKD) PCT: Patent Cooperation Treaty PCT PCT/US2018/014844   1/23/2018     Pending
Inventors:
Category(s):
For Information, Contact:
Vera Sampels
vsampel2@jhu.edu
410-614-0300
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