Progress in preclinical studies
Movement/mobility biomarker study
For the first time in the HSP clinical trial program, HSPers themselves have been involved in the trials.
Nine participants from Brisbane and surrounding areas, including four with SPG4 type HSP, came to the Griffith University Institute of Drug Discovery in the second week of February to take part in a movement/mobility biomarker study.
The effectiveness of any treatment for HSP must ultimately be in terms of improvement in movement and mobility functionality for HSPers, or at least in significantly slowing down or stopping the decline that happens with a progressive condition like HSP.
This is the first time the team has worked with a medical issue. Team leader and head of the School of Research in Engineering at Griffith University, Prof David Thiel said “Alan has been a colleague and friend for the best part of 30 years, so when he came to me with the idea, I was immediately interested. Now that the software has been developed and we are now testing the potential of the application with people who have HSP, the team is excited about being involved in this initiative and the bigger goal of finding an effective treatment for HSP”.
In this study, a smartphone app has been developed by the Engineering Research team at Griffith University, working with clinical trial program principal investigator Prof Alan Mackay-Sim. The team has broad experience in developing hardware and software for assessing and measuring sports performance by athletes. The smartphone application is employing a novel approach to quantifying and characterising an individual HSPer’s mobility, including its potential to be a good measure of improvement, of things staying the same, or of a decline.
The HSPers who volunteered for the study had the opportunity to socialise as, one-by-one, they walked about 20 m – 10 m out and back – carrying a smartphone in a pouch at the waist with the application, and separate video, recording every movement. To increase the amount of data generated, participants did this in three rounds. The data is now being analysed to assess, if possible, the potential for the smartphone application to be used as a biomarker of HSP status. HSPers who participated in the study were very positive about the opportunity and glad to finally be able to actively participate in the research program after several years of laboratory work with stem cells. Peter from the Sunshine Coast said “I’m happy to do whatever I can if it helps find a treatment for HSP”, which was echoed by the others in the group.
Drug dose modelling study
In the last update, we shared with you the need to undertake a further drug dose modelling study, this time a mouse study to measure drug concentrations in the blood, brain and spinal cord at different drug dosage levels.
The aim is to learn more about various factors that affect the calculation of dose needed to achieve drug active ingredient target levels in the neurons of HSPers in clinical trials.
This study is now fully planned, agreements have been entered into with the two contract research organisations who will be performing different parts of the work, it is scheduled for completion in May, and has been budgeted for from Foundation funds of around $100,000.
Two of the three biomarker studies have been approved for funding by the Foundation Committee in late January.
One of the studies looking for a blood biomarker for HSP status has been underway since the middle of last year, and can now be expanded to include more HSP participants with the extra funding.
The other blood biomarker study is for a brand-new test and so the test itself (known as an assay) needs to be proven and validated as a first step. This work is underway.
The final biomarker study using brain imaging needs further deliberation and planning to maximise the potential for definitive results to be delivered from the study. Due to resource constraints in availability of the relevant researchers, this further planning is scheduled for May.