Clinical trial in advanced planning stage
SPG4 Clinical drug trial
COVID restrictions have hampered some research, with the biomarker studies being most affected. However, planning for clinical trials has been able to continue and is well advanced. The planning currently underway includes:
- Grant funding applications – identifying and assessing grant funding opportunities; developing and submitting applications.
- Finalising the documentation for the trial with an initial two-part plan:
- Phase 1 determines safety and tolerability of the drug at the doses being tested.
- Phase 2a determines if the drug works in a 6-month trial with SPG4 HSP participants.
- When finalised, the documentation has to be submitted for ethics, regulatory and governance approvals.
- Getting quotes for implementing Phase 1 from organisations that specialise in clinical trials.
- Assess options for the manufacture of the drug treatments for Phases 1 & 2a.
- Site Feasibility Assessment for Phase 2a to ensure that the right people with the right resources, technologies and funding are available for the successful implementation of the 6-month trial with SPG4 HSP participants.
SPG7 research (Sydney)
Meanwhile, important research on the SPG7 form of HSP has recently been published by members of the HSP Research Program team, from a study funded by the Spastic Paraplegia Foundation (USA) and this Foundation. The published paper reports that mitochondria are dysfunctional in SPG7 but not in SPG4 cells.
Whilst the Foundation focus has been pushing ahead towards clinical trials with a potential treatment for SPG4 HSP, we never lose sight of the need for treatments for all forms of HSP. The HSP Research Program team is actively engaged in research on SPG7, following a similar pathway to the SPG4 research. They are now evaluating olfactory stem cells developed from tissue samples taken from people with SPG7 mutations to understand cell functions and mechanisms that are impaired in this HSP type. This understanding will be used to screen for potential drug treatment candidates in the future. The team has received new grant research funding from the Spastic Paraplegia Foundation (USA) to further pursue this work over the next two years.
Blood biomarker (Sydney)
The blood biomarker assay was initially developed to measure the differences between biomarker levels in people with HSP and non-HSP individuals. We now intend to use this assay to measure the biomarker levels in people with HSP pre and post treatment in the Phase 1/2a clinical trials being planned. However, this requires significant redesign of the assaying process with the time between sampling, processing and analysis now narrow, critical and requiring precision, where previously samples could be stored for weeks and analysed when logistically convenient. The assay redesign is presently underway and planned for completion in the September quarter. (Dr. Wali also leads this study)
Skin fibroblast biomarker (Sydney)
Using skin fibroblasts (skin cells grown from a skin tissue sample), the aim is to develop a biomarker to aid in HSP diagnosis and drug testing. For this, we are applying a novel machine-learning-guided, high-content microscopy image analysis approach to HSP and control skin fibroblast samples. The experimental phase has been completed and data is now being analysed. (Dr. Wali also leads this study)
Smartphone app (Brisbane)
The 4th and final round of testing in the smartphone app study at Griffith University in Brisbane took place in mid-August, having been rescheduled from March due to COVID restrictions. 6 of the 9 regular HSP participants in the study once again took part, with 3 unavailable.
The study seeks to establish the smartphone app as a reliable and valid source of regular data on changes in the mobility of HSP participants that might occur over time as a response to drug treatment in the clinical trial.
Now that all the data is collected from the study, the challenge is to analyse and make sense of it. A new, additional member of the Griffith University Electronic Engineering team that has been working on this study will take on the huge task of data analysis as the topic of a Masters degree. Our sincere thanks to the whole engineering team who have generously volunteered both their time and expertise to this initiative.