Discovery of potential treatments
This publication is an elegant and comprehensive summary and review by the HSP research program team of the progress and discoveries over the past decade in studies supported by this Foundation. The findings and conclusions make a strong case for the clinical drug trials that we are currently pursuing.
This graphic neatly summarises the impairment that occurs in the axons of HSP neurons derived from stem cells in the lab and the restoration of those functions observed with the application of tubulin-binding drugs.
Here is a link to the full publication http://www.mdpi.com/2076-3425/8/8/142 for those with an appetite to understand more of the biology and the science that has brought us to this point.
Hereditary spastic paraplegia is an inherited, progressive paralysis of the lower limbs first described by Adolph Strümpell in 1883 with a further detailed description of the disease by Maurice Lorrain in 1888. Today, more than 100 years after the first case of HSP was described, we still do not know how mutations in HSP genes lead to degeneration of the corticospinal motor neurons. This review describes how patient-derived stem cells contribute to understanding the disease mechanism at the cellular level and use this for discovery of potential new therapeutics, focusing on SPAST mutations, the most common cause of HSP.
SOURCE: Brain Sci. 2018 Jul 31;8(8). pii: E142. doi: 10.3390/brainsci8080142. PMID: 30065201
Patient-Derived Stem Cell Models in SPAST HSP: Disease Modelling and Drug Discovery.
1 Department of Neurogenetics, Kolling Institute of Medical Research, The University of Sydney, Sydney, NSW 2065, Australia. [email protected].
2 Department of Neurogenetics, Kolling Institute of Medical Research, The University of Sydney, Sydney, NSW 2065, Australia. [email protected].
3 Griffith Institute for Drug Discovery, Griffith University, Brisbane, QLD 4111, Australia. [email protected].