Stem Cell Pilot Study underway

Posted - December 2008 in HSPRF News

Research Agreement finalised

The Agreement with the National Centre for Adult Stem Cell Research at Griffith University in Brisbane was finalised on 26 September.

HSPRF President Robin Bligh and Professor Alan Mackay-Sim, Director of the National Centre for Adult Stem Cell Research (NCASCR) signed on the dotted line for the year-long pilot study.  There to witness this historic moment for the Foundation and the HSP community were Narelle and Ian Brock, and Kim Frost.

L to R: Ian Brock, Narelle Brock, Kim Frost (standing), Robin Bligh, Alan Mackay-Sim (seated)

L to R: Ian Brock, Narelle Brock, Kim Frost (standing), Robin Bligh, Alan Mackay-Sim (seated)

Prof. Mackay-Sim then took the group on a tour of the facility, where the cheque to allow work to commence was presented.

L to R: Narelle Brock, Robin Bligh, Alan-Mackay Sim, Ian Brock, Kim Frost

L to R: Narelle Brock, Robin Bligh, Alan-Mackay Sim, Ian Brock, Kim Frost

HSPers with known mutations in SPG4 or those with typical AD-HSP symptoms were invited to participate in the study by Co-principal Investigator, Associate Professor Carolyn Sue of the University of Sydney’s Kolling Institute of Medical Research. 

The 12-month pilot study has been funded by the HSP Foundation and will be initiated at the NCASCR in Sydney and Brisbane. This study will aim to identify a set of cellular targets for the future screening of prospective drug therapies. The pilot study will examine how mutations in the gene named Spastin (known as SPG4) disrupt cellular function and cause HSP.

Drug Discovery Quest

The research involves establishing and maintaining olfactory stem cell lines from patients with HSP. The HSP patient cell lines will be analysed by looking for gene expression and biochemical pathway differences using state-of-the- art equipment, at the recently founded NCASCR. These findings will be the basis for future submissions to the National Health and Medical Research Council with the overall aim being to discover drugs to treat this disease.