HSP clinical trial program update June 2020

Progress in pre-clinical studies

Portrait of Professor Alan Mackay
Alan Mackay-Sim

Dose range finding study (Brisbane)


The final reports on the second mouse study were received in mid-April from TetraQ and QIMR confirming that Noscapine is indeed reaching the brain and spinal cord of mice in amounts that are dependent on the quantity of the drug administered by oral dose. It has also been confirmed that levels of a potential biomarker are elevated in the spinal cord of the mice in direct relationship to the drug dose given.

Following the inconclusive results from the first dose range finding study in mice that was reported in June 2019, the findings of this second study are much more in line what was hoped for and expected.

The clinical trial research team has recently met to review the study results and discuss the translation to dose levels to test in humans. The implications for the design of clinical trials in people with HSP were also discussed and detailed design of the initial trial is now underway.

The animal experiments and analysis of drug levels in the blood, brain and spinal cord of the mice were carried out at TetraQ  http://www.tetraq.com.au/ Research Infrastructure Centre at the University of Queensland. Analysis of the biomarker of microtubule stability in brain and spinal cord was done at the Queensland Institute of Medical Research (QIMR) https://www.qimrberghofer.edu.au/ also a facility of the University of Queensland.


Gautam Wali
Gautam Wali

Biomarker study (Sydney)


Progress in this investigation was not possible during the quarter. Access to the lab was limited to just basic care and maintenance functions for the living HSP cell cultures. On top of limited access for the researchers, it has not been possible to plan for the participation in the study of a larger number of people with HSP due to unknown factors regarding lifting of restrictions and minimising the risk to health of all concerned.


However, the circumstances have allowed time for further data analysis of the multiple parameters that are now being measured in the biomarker study. The opportunity has also been taken to complete a scientific paper for publication, and expand grant writing efforts and applications for research funding for the continuance and development of the biomarker study.


Sue-Faye Siow
Sue-Faye Siow

Developing a Quality-of-Life survey for HSP (Sydney)


Dr Sue-Faye Siow is working with a research team at Royal North Shore Hospital in Sydney to develop an HSP-specific quality of life survey (HSPQoL). To date, there are no disease-specific QoL surveys for HSP, whilst generic surveys don’t adequately cover important issues specific to HSP that include the genetic nature of the condition, increased frequency of falls, the challenges of access to care and the coordination of complex care needs.


The survey will have application in both clinical practice and research, as well as potentially providing an important outcome measure for the ultimate effectiveness of any treatment tested in a clinical trial. The aim is to create a validated, standardised, HSP-specific QoL survey by modifying an already established generic QoL survey with HSP-specific questions. It is beyond question that HSP has a significant impact on quality of life. Research studies into quality-of-life for people with HSP show reduced life satisfaction, reduce mental well-being, inadequate social support, poorer memory and reduced quality of sleep compared to people without HSP.


Blood biomarker study (Tübingen, Germany)


As reported in the March update, the blood biomarker study by Dr Rebecca Schüle at the University of Tübingen in Germany was scheduled to be completed this month but has been delayed due to the shift in priorities necessitated by the coronavirus pandemic.


Smart phone app study (Brisbane)


Another round of testing of participants in the smartphone app study at Griffith University scheduled for April was indefinitely postponed. This will be rescheduled as soon as it is safe for all concerned to reconvene.





  1. What does it means dear researchers
    That noscapine is indeed reaching brain and spinal cord
    I am confused ….please explain in detail

    1. Editor’s Note: “Noscapine is indeed reaching the brain and spinal cord of mice in amounts that are dependent on the quantity of the drug administered by oral dose”. The results of the first mouse study from March 2019 were inconclusive. It was not clearly established that the drug was reaching the brain and spinal cord of individual mice in the experiment. Where the drug was found in the brain and spinal cord, there was no clear relationship with the dose given.

      In the second study that has recently been completed, the evidence is now there that the drug reaches the brain and spinal cord of all the mice. Further, the amount of the drug reaching the brain and spinal cord is clearly related to the dose given.

    1. Editor’s Note: The research team is working on planning and design of an initial trial based on the results of the mouse study. No timeline or start date for a human trial is available as yet.

  2. First a HUGE thank you to everyone working on this!!! HUGE!!!
    For the trials of noscapine, I would love to participate, does location affect ability to participate in this trial? I am in Canada, but would love to participate in this trial and any other relating to HSP.

    1. Editor’s Note: Because of the need for multiple visits to the clinic in Sydney over an extended period, location of participants within reasonable travel time of the clinic is desirable. However, if the results of early clinical trials warrant further investigation, Canada may well be a highly suitable location for a subsequent trial given the excellent work on HSP at McGill University in Montréal, including the impressive CANHSP database.

  3. Hey. Great work! Also willing to participate from Norway if ever needed. Would you guys recommend to buy noscapine from the pharmacy and try it, or is this non productive against HSP? Keep up the good work. 🙂

    1. Editor’s Note: Thank you! It is probably unlikely that a trial would be held in Norway due to small population and low numbers of people with HSP. However we have had regular communications with naspa https://www.naspa.no/ over several years now and they would inform you of any initiatives that might be relevant locally. Regarding the candidate drug under investigation, there is still quite a way to go to determine if the drug is effective… and other questions such as – in what form? with what types of HSP? what dose? how often? If you are thinking of trying the drug, talk with your neurologist for guidance.

  4. Are there any studies in America? I would like to be apart of finding a cure. I have HSP and it’s such a struggle. I went to a Dr visit and he took a look at me and said I have HSP. I gave no type of blood test or anything else he just looked at said hey you have HSP 20 plus years ago.

    1. Editor’s Note: To the best of our knowledge there are no clinical trials at present in the USA. Do you know about the SP Foundation in the USA sp-foundation.org/? They are highly active as a community of people with HSP and PLS. Their website is a huge resource of relevant information about the two conditions. They raise a lot of money and fund a lot of important research every year. If you are not a member already, we highly recommend that you join up.

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