HSP clinical trial program update September 2019

Progress in preclinical studies and clinical trial preparation


Dr Gautam Wali

Blood biomarker study (Sydney, Australia)


We have now tested the target HSP blood biomarker in multiple sources of easily accessible SPAST patient sample types.

Sample type 1: The pilot test run on 10 samples from SPAST patients and 9 unaffected healthy controls showed that the biomarker was effective in identifying the patient samples. This is very promising and confirms that we are headed in the right direction to develop a biomarker suitable for use in clinical trials.

Sample type 2: The biomarker can now be identified in different fractions of blood samples. This is an important development as the expression of the biomarker varies within the different fractions. It is most advantageous to identify the blood fraction that has the highest expression of the biomarker, increasing the chances of finding differences between patient samples and control samples. This has now been achieved in 2 control samples.

Next steps include testing for differences in all control samples and then comparing them with the SPAST patient samples.



Alan Mackay-Sim

Dose range finding – new study (Brisbane, Australia)


Prof Alan Mackay-Sim reports that after careful consideration of the inconclusive results from the dose range finding study completed in April and a review of the design of that study, a new, small study has now been designed, planned and initiated.

The dose range now being investigated has been recalculated and testing will involve a smaller number of laboratory mice. The animal part of the study has again been contracted with TetraQ, who will analyse brain and spinal cord samples to detect and determine drug levels present.

Subsequently there will be testing of equivalent brain and spinal cord samples using different methodology to detect a potential biomarker associated with the effects of the drug. Once again this part of the study has been contracted with QIMR. Both contracted companies are associated with the University of Queensland and both enjoy our full confidence. A start date for the new study is expected in the near future.


David Thiel

Smartphone app biomarker study (Brisbane, Australia)


A fourth session of data collection took place recently at Griffith University in Brisbane under the capable guidance of the Engineering Research team led by Dr. David Thiel with colleagues Dr. David Rowlands and Dr. Hugo Espinosa.

In this session, a control group of 9 healthy volunteers with no blood relative family history of HSP undertook the identical testing protocol that has been used with the HSP group in the preceding three sessions. The control group data will provide a benchmark for comparison and analysis, with a view to attributing meaning to the changes in the ’signature’ individual mobility patterns generated by the app for HSP participants.

Sophisticated data analysis in both Australia and Europe is where the potential for this technology to be used as an HSP clinical trial biomarker will be evaluated.


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