Current Appeal

Research Project 2010/11 - Towards a Cure for HSP Research Program

Identifying Therapeutic Drug Candidates for treating HSP. A two-year research project to commence September 2010

The research provider for the HSP Research Foundation (HSPRF), the National Centre for Adult Stem Cell Research(NCASCR) located at Griffith University in Queensland, has just successfully completed a pilot study, the first part of a research program Towards a Cure for HSP to discover and develop drugs for treating HSP.

In the Pilot Study, olfactory stem cells from HSP patients and controls have been successfully differentiated into neurons. Gene expression profiling was done and altered cell functions identified and validated.

This new Project over 2 years is the next step in the program. The aim of the project is to identify therapeutic drug candidates for treating HSP using olfactory (nasal) stem cells from patients as cellular models of HSP.

The first year of research aims to:

  1. Increase understanding of the consequences of changes caused by mutations in the SPG4 gene, the most common HSP related gene, responsible for 40% of all HSP occurrence
  2. Leading to an understanding of the major cellular dysfunctions: detect the dysfunctions/abnormal behavior in cellular pathways; what cell functions are changed/how do cells work in a disease situation?
  3. Define and describe specific cell functions as candidate targets for therapeutic drugs.

The second year of research will aim to:

  1. Devise assays for subsequent screening of therapeutic drugs against the target cell functions.
  2. Validate the assays.
  3. Identify any prospective drugs that emerge through the validation process.
  4. Screen a compound library against targets to identify potential therapeutic drug candidates that compensate for cell functions altered by HSP.

Because the NCASCR research directly targets the disease in human tissue as opposed to lab animals, confidence in positive results for the subsequent stage of drug development is warranted. This project’s outcomes will be the basis of an NH&MRC proposal for the drug development stage.

The 2 year Research Project requires the HSPRF to find $100,000 per year for a post-doctoral scientist at an annual cost of $80,000, plus consumables of $20,000 per year.