About Us Therapy Development Database
Clinical Trials
GDNF History
Advocacy & Education Sham Surgery Books Glossary Links News &
Op Eds

Hedgehog Pathway Agonist

Also known as:
  • Small molecule Hedgehog pathway agonist

  • Agonists of Hedgehog signaling

  • Hh pathway agonist

  • Shh pathway agonist

Other Hedgehog terms:
  • Sonic Hedgehog (Shh)

  • Hedgehog (Hh)

  • Sonic Hedgehog protein

  • Hedgehog signaling pathway

  • Hedgehog signal transduction

  • Hedgehog signaling


Curis Inc., has several small molecule agonists that may be able to treat diseases including Alzheimerís disease, Parkinsonís disease, and amyotrophic lateral sclerosis (ALS).[1] These agonists, called small molecule Hedgehog pathway agonists, would be used in activating the Hedgehog signaling pathway. Scientists at Curis believe that when the hedgehog agonists activate the Hedgehog signaling pathway, the result is neuroprotection.[2]

The Hedgehog signaling pathway regulates the normal development of the brain and spinal cord. The small molecule Hedgehog pathway agonists can activate the Hedgehog signaling pathway and promote nervous system repair. These Hedgehog pathway agonists can be taken orally and can enter into the brain and spinal cord.[3]

Potential benefits:

  • Promotes repair of nervous system

  • Restorative

  • Neuroprotective

  • Orally available


  • Not yet in clinical trials for humans

Research involving animals:

Animals treated with Sonic Hedgehog protein (Shh) showed significant improvement in tests of movement, and preservation of brain tissue normally lost with progression of Parkinsonís disease. Additionally, the animals that had been previously treated with Shh and later received L-Dopa showed significantly better improvement in movement than did non-Shh-treated controls.[4]

[1] BiotechTracker: Curis Approaches the Starting Gate; BioPortfolio, September 02, 2003 

[2] Hedgehog Small Molecule Agonist Neurological Disorders Programs, Curis, Inc.

[3] Press Release: Report of Hedgehog Pathway Efficacy in Preclinical Model of Parkinson's Disease; Source: Curis, Inc.; Friday December 17, 8:30 am ET

[4] Sonic Hedgehog Reduces Behavioral Impairment in a Primate Model of Parkinson's Disease, September 18, 2002 - Curis, Inc.


Copyright© 2012 Pipeline Project

All rights reserved. Revised: 01/26/12.