Posted - September 2018 in HSPRF News
Progress in clinical trial planning and preclinical studies
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.
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.
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.