HSP clinical trial program update June 2018

Posted - June 2018 in HSPRF News

Progress in preclinical studies


Associate Prof. Michelle Hill

Drug dose modelling study

The study to measure drug concentrations in the blood, brain and spinal cord of mice at different drug dosage levels is now well underway. The aim is to learn more about various factors that affect the calculation of the drug dose needed to achieve target levels of the active ingredient in the neurons of HSPers in clinical trials.


The drug dosage experiments with the animals were completed in early May with blood, brain and spinal cord tissues collected and preserved for analysis. Around the same time, work began in the laboratory that will perform the analyses, to develop and validate measurement techniques and methodologies.


The Precision & Systems Biomedicine laboratory at QIMR Berghofer Medical Research Institute in Brisbane will measure the effectiveness of the various drug dosing regimes used in the mouse study. Laboratory head, Associate Professor Michelle Hill said “These measurements have never been done before. There is no off-the-shelf procedure for doing them. It is an exacting and time-consuming task to develop and validate a new test to measure the tiny but functionally important modifications in the target protein, levels of which should theoretically be affected by the drug. We’re here to find out if that’s the case. Very sensitive instruments called mass spectrometers will be used for such precise measurements.”


The total cost of this study is over $140,000, more than half of which has already been paid for the animal experiments that are now completed. The projected completion date for the development and validation of the test is October, and for the analyses, December.


. . .

Movement/mobility biomarker study

The effectiveness of any treatment for HSP must ultimately be in terms of improvement in movement and mobility functionality for HSPers, or at least in significantly slowing down or stopping the decline that happens with a progressive condition like HSP.


In the last update, we reported that nine HSPers had taken part in testing a smartphone app at Griffith University in Brisbane, designed to capture and characterise their unique movement patterns when walking. The smartphone application is employing a novel approach to quantifying and characterising an individual HSPer’s mobility, including its potential to be a good measure of improvement, of things staying the same, or of a decline.


Data analysis is underway and ongoing by the Engineering Research team at Griffith University.


Dr. Gautam Wali

Blood based biomarker for HSP

The goal of this study headed up by long-term HSP Research Program and clinical trial team member, Dr. Gautam Wali, is to identify a blood based biomarker that:

* reflects disease-specific defects, and

* can be used to quantify the effects of drug treatment.


Dr. Wali said, “Our previous research using HSP stem cells and induced pluripotent stem cell derived cortical neurons has consistently shown imbalance in certain key proteins that can explain the axonal degeneration that occurs with HSP. In this biomarker study, we are aiming to identify these disease-specific changes in blood.


We have so far optimised the techniques, processes and methods that will be used in analysing the samples. We will now start collecting blood samples from HSP patients and unaffected control individuals. As we do this, we will also be working on some ethics approvals required for the study”.



The foundation has provided funding of around $220,000 during the quarter including $107,000 for parts of the dose modelling study and $112,000 for the other blood biomarker study being conducted in Germany by clinical trial team member Dr. Rebecca Schüle.




Comments on this story

  1. Darin posted at 11:35 am on 3 June 2018Reply

    How do I get this information to my neurologist ASAP?

  2. grant posted at 12:24 pm on 4 June 2018Reply

    This is great news you are getting some results slowly but surely we hope, I am sure there are a lot of us waiting to hear your next part of your trial – fantastic !

  3. Denis posted at 10:03 pm on 19 June 2018Reply

    let’s rather, I’m already walking badly 😛

  4. jayxie posted at 12:14 am on 6 August 2018Reply

    When is the human clinical trial

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