Progress in pre-clinical studies
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.
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.
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.