HSP clinical trial program update December 2018

Progress in clinical trial preparation and preclinical studies


Clinical Trial


A strategy and plan for securing approval from the regulatory authorities for clinical trials and, if successful, the candidate drug, has progressed over the quarter. Consultants specialising in working with the regulators to get such approvals have now been contractually retained and initial discussions had.

To assist with management and administration of the whole initiative, a hierarchical map has been created defining both the major and the sub-components of the plan. Nine major streams have been identified with 52 sub-components, each sub- component representing a significant project, or set of projects. This map constitutes the basis for detailed planning, which is well underway in some parts of some of the nine streams. The nine major streams are:

  • biomarker investigations
  • ethics
  • regulatory approvals
  • trial participants
  • clinical trial
  • dose modelling
  • candidate drug
  • market development
  • program management

Opportunities for applying for grant funding for the clinical trial are being regularly reviewed and the most suitable ones further investigated. Currently one application is pending decision and announcement by the Federal Minister for Health, which is expected in the near future.


HSP Clinical Trial team member Dr. Sue-Faye Siow

Blood based biomarker study


The process of collecting patient blood samples and Spastic Paraplegia Rating Scale scores is ongoing, with ten patient samples having been collected.

Ethics protocol amendments have now been approved. HSP patients from the Royal North Shore Hospital Neurogenetics clinic, who have previously consented to being contacted regarding relevant research will be invited to participate via phone call. This approval will expedite the sample collection process.

In the mean time, the biomarker is being tested in patient fibroblast cells, a resource available in Professor Carolyn Sue’s laboratory at the Kolling Institute, Royal North Shore Hospital. Patient fibroblast cells, like blood, are a source of easily accessible non-neuronal patient samples.

The work is currently in progress and is being carried out by clinical trial team member Dr. Sue-Faye Siow as part of her PhD research.

An update on the second blood biomarker study in Germany that has been underway for more than 12 months was not available at time of publication.


Smartphone app study


Prof Alan Mackay-Sim joins HSPers at the testing

Six HSPers from the south-east Queensland area participated in the second round of testing in mid-November for a smartphone app designed to quantify changes in movement and mobility, potentially constituting an important measure of drug effectiveness in a clinical trial.

The engineering team at Griffith University, Nathan, will now analyse the data, including a comparison with the the data collected in the first round in February on the four HSPers who participated in both rounds.


Associate Prof Michelle Hill

Dose modelling study


Associate Prof Michelle Hill recently advised that the development and validation of a reliable, repeatable test or assay to measure the minute concentration of the target compound is proving challenging and needs more time than originally scheduled.

Once the assay is validated, the measurement of target compound concentrations in the brain and spinal cord tissue of the experimental mice can go ahead.

Following that, the data will be analysed and interpreted with the desired result being the knowledge needed to determine what oral drug dose and formulation will achieve the target concentration in the upper motor neurons of people with HSP.

The projected date for completion of the study is now mid-February.





  1. Best Christmas pressie ever!!! – you are doing so so well. I just love hearing such good news… At last we are so close to reaching this phase everyone has been waiting for. Well done to such a clever team in helping to find medication that can help us. ..

  2. Thats the great, but how long will it take to finally declare the cure for hsp as I have been waiting how much a drug can be taken in a day ?

    1. Editor’s Note: How long it will be until an effective treatment is available for HSP is something no one knows. The HSP clinical trial may not be successful, although there may be some indications 9 – 12 months after it begins if it is successful. To get regulatory approval will take at least three years. Yes, you are waiting… And another half a million people worldwide are also waiting… We are all waiting.

      Again, no one knows how much medication needs to be taken. The dose modelling study currently underway is needed to determine a ‘dose range’ for the clinical trial, and the results of the clinical trial, if successful, will provide the answer to your question.

  3. Dear Editor. I taking cough dose and feel better. I hope you will tell us the modeled dose or dose range for clinical trials in February, when will you recognize them?

    1. Editor’s Note: It will be up to the clinical trial team to decide how much information on the trial is publicly available when it starts. Remember that the clinical trial’s purpose is to determine effectiveness – a main component of which is what drug dose is most effective.

  4. First a great big THANK YOU for working so hard on our behalf. I am very hopeful that this will help with HSP. I have an unknown variant of type 4, wondering if it would still work for me or other types of HSP? Also is it aimed only at uncomplicated HSP or complicated as well.
    Again thank you SO MUCH for your efforts! ❤

    1. Editor’s Note: it is expected that, if effective, the drug will work on any HSP types that have the same mechanism of disease causation. SPG4 type HSP, and the more than 150 identified variants of it, share the same mechanism, as do some other HSP types.

  5. Hi sir! This is Dr. P.K. a Pharmacologist from India. First I would like to thank you from the core of my Heart for your hard work. My son (10 years old) has been diagnosed with AP4M1-RELATED HSP (Spastic paraplegia 50)for over 5 years. Sir, i Would like to ask you some questions and will be obliged if I get your answers back.

    If you will be successful on your drug discovery and development project then will it cure AP4M1-RELATED HSP (Spastic paraplegia 50)?.

    And as a worried Father “how much maximum possible time it may need to complete your project/develop the discovered drug into a formulated drug for HSP”?

    will it available in india (if no) how we indian people can get the medicine?

    Sir, my last question is, can my son participate in clinical trials as a human model?

    Thanking you
    Dr. P.K.

    1. Editor: To answer your questions –
      * At this stage we don’t know if, or how much, the candidate drug may be effective with SPG50 or any of the adaptor protein related HSPs. There may be some overlap in causal mechanism https://hspersunite.org.au/about-hsp/understanding-hsp-mechanism/ between AP-4 related HSPs and SPG4 HSP, for which the candidate drug was screened and chosen.
      * Even if there is adequate funding for the currently proposed clinical trial, it could be up to 4 years from now before a treatment is available to people with HSP. This assumes no major obstacles to progress are encountered either in the current preclinical investigations or in the clinical trials themselves.
      * The vision of the Foundation is to “have a treatment for the HSPs that is highly effective, widely available and readily affordable”. The drug development plan will be created to respond to this vision, including drug availability in different parts of the world. This question however is well into the future.
      * Trial sites planned for currently include Sydney and Melbourne, with the aim of one or two additional sites in other countries over time. However there are no plans for a site in India and therefore it would be impractical for your son to participate.

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