HSP Research update June 2015

Progress report from the research team


Prof. Alan Mackay-Sim
Prof. Alan Mackay-Sim

This year has been productive so far in moving towards a clinical trial. Our immediate goal is to strengthen the “biological case” to take to the Therapeutic Goods Administration (TGA) for approval for a clinical trial. Before approval for a drug trial, the TGA requires evidence for how a drug candidate works (the “mechanism of action”) in target cells. So we are extending our experiments to show that our drug candidate, which works in HSP patient-derived olfactory stem cells, has similar actions in neurons (nerve cells).


Gautam Wali
Gautam Wali

In Carolyn Sue’s lab in Sydney, Gautam Wali and the team are making neurons from patient-derived “induced pluripotent stem cells” (iPS cells). These are like embryonic stem cells that are made by genetic engineering from patient’s skin cells. We are now well down this road. We have made the cells and are doing the important quality control experiments, to make sure we have got them right. iPS cell colonies are notoriously variable and it is essential for reliable results that quality control on the cells manufactured is precise. We have been optimising the method to make the iPS cells differentiate into neurons and have started this in iPS cells from an HSP patient with a SPAST mutation.


Dr. Yongjun Fan
Dr. Yongjun Fan

In Alan Mackay-Sim’s lab in Brisbane, Yongjun Fan has been further exploring how the biology of patient-derived olfactory stem cells from patients with and without SPAST (SPG4 HSP) mutations are the same or different. This will give us a better idea of the potential for a drug developed for cells with SPAST mutations to also work for cells with different HSP mutations.


Meanwhile Gautam submitted for publication the first manuscript that is part of his PhD project. This is a big step for him and we have our fingers crossed that it will move quickly through the review process. This new research paper has been submitted to a scientific journal and reports on studies into peroxisome trafficking and oxidative stress in HSP cells.






  1. The sooner the better for some cure or remedy. This is controlling my mind 24/7 causing stress and depression. The unknown is horrible.

  2. My two sons and I have HSP. I am 73 my sons in their early 50s. We walk with canes. I got a wheel chair last week. Do you have a guess when a cure or help will be available. I understand that a new drub CTP -354 that I have been following for 2-3 years is not going to be produced
    Is there any hope in the near future.? We are available for trials.

    1. Editor’s Note: In the very best case scenario with the research currently being done in Australia, an available treatment is at least 3 years away, i.e. late 2018 at the very earliest, and likely later even with continued progress and success. CTP-354, to the best of my understanding, is an anti-spasticity drug, and as such would not be considered as a potential treatment for HSP, but rather as a potential treatment for HSP symptoms (in this case, spasticity). From where did you obtain your news regarding CTP-354 “not going to be produced”?

      All HSP support groups around the world will widely publicise clinical trials at the appropriate time in the quest to secure suitably qualified candidates for the trials. It appears that you are from the US, so make sure that you are signed up with the SP Foundation in the USA to receive the relevant communications.

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