Funding preclinical studies
Developing clinical trial plans
To test the effectiveness in HSPers of drugs that have proved successful with human HSP stem cells in the laboratory requires a clinical trial. To get approval for, and conduct a clinical trial, preclinical investigations are needed to come up with answers to questions such as ‘how much of the drug needs to be taken and how often to achieve the target concentration in the neurons?’; and ‘what markers of HSP status are sufficiently sensitive and reliable to be suitable for the clinical trial?’
Preclinical investigations to answer these questions commenced in late 2016 and are continuing:
Over 2017 and 2018, two dose modelling studies have been carried out – a computer-based modelling initially, followed by a study in mice that is scheduled for completion by March 2019.
A blood based biomarker study this commenced in Germany in 2017 and is progressing. A second blood based biomarker study was initiated in Sydney in mid-2018, looking at a different compound as a potential marker of HSP status.
A study of individual mobility of HSPers using a custom smartphone application began in February 2018 with the aim of determining its suitability as a marker of changes in HSP status.
$350,000 given by people just like you in the HSP community has funded the preclinical studies over the past 18 months, including $50,000 since June …YOU really do make a difference! And WE are working hard and not wasting a cent … literally! 99c out of every dollar you give goes directly to fund HSP research.
Planning is sufficiently developed for the clinical trial to allow costing and budgeting estimates of $1.5 million total. Suitable grants are being sought in an ongoing process, with one major grant application decision pending currently (December 2018). Funding of the overall process through government, institutional and philanthropic grants, private equity investors and commercial partnership with a pharmaceutical company has also been discussed and now requires concerted investigation and consideration to ensure the best decisions are made over time in the interests of establishing an effective treatment that is widely available and affordable to HSPers globally.
Read the latest HSP research program update.
Your generous and continuing support helps make this possible. 99 cents in every dollar you give goes directly to fund the HSP research program. Gifts of $2 or more are tax-deductible.
An effective treatment for HSP is now within reach