Stem cell research progress report, December 2011

Breakthrough progress, an outstanding award, additional funding, additional staff, eminent publication and drug screening to start early in 2012… all the news is good news!


Breakthrough Progress

The researchers have finalised validation of the assays (tests) for the drug screening. While this may not sound like much, it is an extremely important step and a significant breakthrough in the research project. What they have done is create a test where none previously existed. Imagine the time when people were aware of feeling hotter or colder but had no way to measure temperature as thermometers had not been invented. The stem cell research team have created a reliable ‘thermometer’ for measuring the effect of potential drug compounds on various functions altered in HSP affected stem cells – that is, they can detect and measure restoration of cell contents, distribution, processes and functions to control cell levels. This breakthrough is vital for the drug screening phase.

The findings will hopefully be published in an eminent journal before the middle of next year. Our heartiest congratulations to the whole team!


The principal investigator on the HSP stem cell research project, Prof Alan Mackay-Sim, has won the 2011 Australian Museum Eureka Prize People’s Choice Award for Australia’s most popular scientist.

There could be no more deserving recipient of this highest of accolades and the whole Australian HSP community adds our rousing congratulations and best wishes for continuing success.

Frank McKeown & Robin Bligh of the Foundation flank Prof Alan Mackay-Sim











Additional Funding… Additional Staff

As a reflection of, and a tribute to, the progress to date on the project… made possible largely by funding from HSP community members through the Foundation… the National Health & Medical Research Council of the Australian Government Department of Health and Ageing have made available funding for the HSP stem cell research project that has made it possible to expand staffing for the next 3 years. The funding is for work associated with “platform technologies” of stem cell banking and gene expression profiling, and also for consumables.

There are now effectively 3+ people dedicated to the HSP stem cell project, where 12 months ago there was just one. This is amazing progress!

Professor Denis Crane

A cell biologist and biochemist, and expert on neurodegenerative diseases, is an addition to the team and will assist Prof Alan Mackay-Sim as a principal investigator.

Dr Yongjun Fan

Fan will continue his excellent work this year and focus on the all-important drug screening in 2012. We eagerly await news of potential drug compounds being identified next year.

PhD student Simon Weyers

Simon is new to the team and will quantify the characteristics of stem cells from non-SPG4 HSP samples collected. This is a whole new area and it has already been demonstrated that there are significant differences from the SPG4 cells already studied.

PhD student Gautam Wali

Gautam is also new to the team and will work on the dynamics of organelle movement inside stem cells and neurons derived from SPG4 stem cells and healthy control stem cells.

Yongjun Fan, Gautam Wali, Frank McKeown, Alan Mackay-Sim, Robin Bligh, Simon Weyers



  1. My husband has HSP and I would be grateful if you could update us on stem-cell research and potential solutions to this condition. We live in Scotland.



  2. I am in the same boat as Claire as my wife (second marriage) has HSP also her Mother, brother and sister have it.The onset to all came on later in life, ages vary between 40-60. Mother was diagnosed as Multiple Sclerosis and treated for same. My wife’s second daughter has been diagnosed with HSP at age 40 (now 45). Also her youngest daughter was diagnosed at 2 years of age (now 22) and yes I/we would be really grateful for any solutions to the condition. We live in Australia.
    Kind Regards

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