HSP clinical trial program update March 2019

Progress in preclinical studies and clinical trial preparation


Dr. Sue-Faye Siow
Dr Gautam Wali

Blood based biomarker study


16 HSPers with SPG4 type HSP have now given blood samples at the Royal North Shore Hospital Neurogenetics clinic in Sydney in this biomarker study, up from 10 over the last three months.

They have also been assessed on the Spastic Paraplegia Rating Scale that covers walking stamina, speed and quality; agility and speed going up and down stairs; ability in standing from a sitting position; measures of spasticity and muscle weakness, pain, bladder and bowel function; and recording of any other HSP related symptoms.

For comparison, blood samples have also been collected from 6 people who don’t have HSP.

The aim of the current part of the study is to develop a test (assay) for the blood samples that is relatively easy to use and generates reproducible results.

The team is evaluating multiple techniques for measuring levels of the blood biomarker that meet these requirements.

Over the next three months an initial batch of samples comprising 15 from HSPers and 15 from non-HSPers will be evaluated.


Prof Alan Mackay-Sim

Smartphone app biomarker study


Following the analysis of data from the second round of testing in November 2018, the research engineering team at Griffith University met with HSP research program principal investigator Prof Alan Mackay-Sim in February to discuss the results and assess the prospects of continuing the study.

The consensus was that promising differences were being seen in the data and that a third round of testing was warranted.

This is expected to happen towards the end of March when the volunteer HSPers for this study will reconvene at Griffith University in Brisbane.


Associate Prof Michelle Hill

Dose modelling study

The Precision & Systems Biomedicine Laboratory at QIMR Berghofer Medical Research Institute, Brisbane, is in the process of measuring minute changes in a target protein in mouse tissue samples.

The mice were given various doses of the candidate drug for clinical trials, noscapine, in a dose-escalation study.

Changes in levels of the target protein were found to reflect effectiveness of the drug in earlier HSP stem cell studies.

A method had to be created and developed to measure levels of the target protein extracted from spinal cord and brain samples of the mice.

The test (assay) uses advanced mass spectrometers to measure the minute changes at a particular region of the target protein that is affected by noscapine.

The aim of the dose escalation study is to determine the effectiveness of various noscapine dosing regimes. This information will guide decision-making on a regime to be taken into the proposed clinical trial.

The results of the study are expected to be available in mid-March.


Clinical Trial preparation

Grant funding: the pending application for $1.5 million funding for the clinical trial from the Medical Research Future Fund over three years was unsuccessful.

New opportunities to apply for funding are continually being sought and assessed.

Planning: A major planning activity was conducted in late December in conjunction with our external clinical trials consultant.

Once a drug formulation and dosing regime have been determined for the clinical trial, the search for a suitable manufacturer of the oral medication and lookalike placebo begins.

The requirements of the regulatory authorities regarding specifications for each and every ingredient in the formulation and the attendant documentation are exacting.

The medication must undergo various stages of monitoring and approval including the manufacturing process and shelf-life stability testing. This will involve a significant amount of time and significant expense.



  1. Thank you so much for the update. Really appreciate the information. Fingers crossed for next stage.

    1. Editor: Whilst participant inclusion and exclusion criteria for the clinical trials have not yet been finalised, the current thinking is for participants to be aged 18 and over.

  2. Thank you, thank you, thank you. I wait impatiently every 3 months for your updates. I try to provide all the financial help I can and I also cross my fingers for the next steps. Good luck!

    (Original: Gracias, gracias, gracias. Espero impaciente cada 3 meses sus actualizaciones. Intento aportar toda la ayuda económica que puedo y también cruzo los dedos para los siguientes pasos. Mucha suerte!)

  3. Hello HSP Research Foundation
    I wanted to thank you for all the progress made for HSP4. I was hoping to find out a little more about the study to determine the effectiveness of various noscapine dosing regimes. I assume the clinical trial can’t begin until the dosage is determined.

    1. Editor: yes, that is so. Both the scientists and the regulators need evidence, normally from animal studies, to support a proposed drug dosing regime to take into a clinical trial. The dose modelling study has been completed and the report submitted just this week to the principal investigator, so we expect to learn about those results in the coming weeks.

  4. Greetings from Barcelona. How are the trials going with the Noscapine? The results were for the end of March and we are already in the middle of May, without knowing the results? Are they positive?

  5. My feelings are in keeping with those of Matthias. I’m really grateful for all your work and live in anticipation.

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