HSP clinical trial program update September 2018

Posted - September 2018 in HSPRF News

Progress in clinical trial planning and preclinical studies

 

Prof Carolyn Sue

Clinical Trial

A detailed plan and budget for an HSP clinical drug trial has now been developed by members of the clinical trial team over the past quarter. It was a substantial undertaking with multiple sources of input, review and revision, with 10 drafts in all being produced along the way.

 

Thanks is due to the dedicated team of investigators, especially Prof Carolyn Sue who made an enormous contribution while shouldering a staggering workload.

 

When funded, the trial will span three years and has a budget of $1.5 million. The challenge now is to explore additional avenues for funding to make this happen.

 

 

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 reports that collecting has now begun with an intake of four HSPers having gone through the process and given blood, and with one of their number so far being assessed on the Spastic Paraplegia Rating Scale (SPRS). The bulk of the past three months has been spent operationalising the plan for this study, which required significant amounts of planning and organising between the three units involved – the HSP clinic, the pathology services and the research laboratory.

 

A strict protocol had to be developed that effectively integrated these three separate functions and insured interchangeability and repeatability with possible changes in personnel. Standard forms had to be modified and customised; personnel to manage and implement the different parts of the study had to be selected and communication and coordination activities developed and refined.

 

Dr Wali has also approached the University of Technology Sydney to explore and negotiate accessing a high throughput microscope with which to help perform the laboratory analyses. This has been a multistep process and appears set for a positive resolution in the near future. It is expected that the first round of sample collections from HSPers will be completed on schedule by the end of the calendar year.

 

Associate Prof Michelle Hill

Drug dose modelling study

This study will measure drug concentrations in the blood, brain and spinal cord of mice at different drug dosage levels. 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.

 

Associate Professor Michelle Hill of the Precision & Systems Biomedicine laboratory at QIMR Berghofer Medical Research Institute in Brisbane heads up this study. She recently reported that the study is on track with progress being made in developing the test and measures to be used in the tissue analysis. Completion date for the development and validation of the test is October, and for the analyses, December.

 

Comments on this story

  1. Nasser posted at 8:45 pm on 2 September 2018Reply

    Hello

    I am an HSPer for about 4 years now. According to my own experience, mental issues such as positive thinking, happiness and good mood have a direct effect on HSP severity. Also crying has the same effect (recently I lost my grandpa and my limping got so much better, but it is temporary).

    I think this information may lead to a new idea or something 😉

    thank you all doctors who put their time and knowledge to solve our problem.

    Nasser

  2. Jay posted at 1:44 am on 3 September 2018Reply

    Hello, I’m an HSP patient from China. I’m concerned that the drug has already started clinical trials. Would you like to consult if there is any chance for the experiment to be carried out in China?

    China has a large population, so rare diseases are not uncommon. There are a lot of HSP patients around, who are eager to be treated. How can I keep in touch with you at any time? 😛 😛 😛

  3. claire posted at 5:50 am on 3 September 2018Reply

    I just don’t know what to say … Im over the moon!! This is fantastic news and the progress you have made is such good news … Our family has HSP (male side) and I hope this will bring positive results for those who have this nasty diagnosis. Such great news for my 11 year old son – your updates are something new I can relay back to our UK Consultants.
    As you know – Clinical Trials.. we are more than happy to help at that particular phase as Im sure lots of other people will.
    Best of luck for the future and cannot wait to read the next stage to the progress that has been made.

  4. Milind posted at 4:02 pm on 3 September 2018Reply

    Dear editor, is the drug used in clinical trial noscapine? Or will there be other brain chemicals with it?
    And when the drug will be given to people to try, that is, when will the clinical trials start?

    • Editor posted at 4:00 pm on 5 September 2018Reply

      Editor’s Note: The drug candidate to be tested in the clinical trial is Noscapine. It will not be combined with any other active compounds. The clinical trials can start as soon as there is firstly funding and secondly regulatory approval.

  5. Jeannette posted at 1:33 am on 5 September 2018Reply

    Could I be a part of the clinical trials? Would they take info from Canada? There are Canadian universities in Australia could we communicate through them?

    • Editor posted at 4:06 pm on 5 September 2018Reply

      Editor’s Note: The initial clinical trial is planned for Australia only. Multiple clinical visits are necessary for participants and it is therefore impractical to have people in the trial not within reasonable ground transport distance of the trial site. It is possible that a subsequent Phase II clinical trial may be held in Canada, especially as there is already a large register of people with HSP through the CANHSP national program based at McGill University in Montréal.

  6. Denis posted at 5:34 pm on 19 September 2018Reply

    and the epothilone will no longer be used?

    • Editor posted at 10:01 pm on 19 September 2018Reply

      Editor’s Note: Only one drug can be tested in the clinical trial because HSP is a rare disease and it will be challenging enough to scientifically prove or disprove the effectiveness of one drug with the limited number of eligible participants. Noscapine was chosen because it is no longer covered by patent and therefore readily available, relatively cheap and equally effective.

  7. Chris posted at 11:58 pm on 19 September 2018Reply

    I am a 45-year-old male living with spg4 late onset around 40. I started taking Noscapine a total of 90mg per day, in 3 batches of 30mg, spaced out around 4 hours apart (Half-Life). I have been taking now for 15 days. This pill and the dosage is making a big change in the way I walk and tension and pain in my feet and legs. This may be the answer to all our prayers. I was wondering the dosage that was going to use in the study? I had heard of people taking 400mg and 600mg per day as well.

    • Editor posted at 11:27 am on 20 September 2018Reply

      Editor’s Note: The question of dosage/dose range to be used in the clinical trial is being decided in a year-long study using mice. This dose modelling study is due for completion by the end of calendar 2018. Until the data from that study is available, the required dosage and dose frequency is just guesswork.

    • Denis posted at 2:38 pm on 20 September 2018Reply

      Hello Chris. where do you buy noscapine and in what form? is this cough remedy? I’m from Russia and found so far only the Japanese cough remedy, which includes noscapine. I ordered and wait. I do not know how legal noscapine is in Russia.

  8. Denis posted at 3:04 am on 20 September 2018Reply

    both of these substances have been known for a long time. and about their medicinal properties. but we still do not accept it. this is somewhat absurd. but then there are some problems associated with this. This is money? or nothing can be done about this and it remains only to wait? I just want to be sure that I did my best. it is necessary to begin the clinical trials.

  9. Tara posted at 12:42 pm on 20 September 2018Reply

    I am also from Canada and would love to participate in this trial!

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