HSP Research Program update March 2017

Planning for Clinical Trials


. . Frank McKeown . . Foundation President

Assessment of the factors relevant to the design of clinical trials has been the major focus of recent planning. Noscapine has now been identified as the preferred drug candidate for the clinical trials based on its effectiveness on HSP stem cells, safety data, toxicology data, patent status, availability, and future affordability to HSPers.


Opinion has been sought from consultants specialising in clinical trial design regarding how much of a ‘jump start’ in the process might be possible based on the long-established good safety and toxicology record with the candidate drug. This is an important and critical process as the decision that will be made will significantly influence how long clinical trials will take and how much they will cost. It will also have bearing on the likelihood of acceptability and approval from ethics and regulatory authorities.


Preliminary discussions have also been held with contract research organisations regarding implementation of early stage trials. Several levels of highly specialised, technical documentation are required for submission to one or more ethics committees, and to regulatory bodies, for approval. 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.




. . .

2016 was by far the best year yet for fundraising with a grand total of $139,196 being raised over the 12 months, close to a whopping 40% increase over 2015 and a very healthy 17% above target.


This reflects the high levels of both generosity and commitment from around 100 members of the HSP community. We know that many in the HSP community have very limited means and therefore capacity to give, and on behalf of all, our sincere thanks goes to those who have given so generously on a continuing basis.


No part of clinical trials has yet been costed as planning is not sufficiently advanced, nor decisions made, on which costing would be based. Those figures will be forthcoming in time. Let’s hope that accumulated funds are adequate to at least get the process started.


The next three months


Working to come up with an optimal trial design with an advantageous starting point, good collaborators, good partners and further clarity on the path forward into trials is the objective for the next quarter with the next report due at the start of June.


  1. This is great news, well done to you all for working so hard in trying to achieve a treatment for HSP. Please can you tell me if this is mainly aimed at SPG4 or could it possibly apply to other more complicated SPGs or is it too early to say yet?

    1. Editor’s Note: The focus of studies to date has been primarily on the SPAST/SPG4 HSP, however comparisons have been done with non-SPAST HSP genes, as well as with healthy controls throughout experimentation. There is overlap in the mechanism associated with/causing SPAST HSP and other HSP types, including some complicated forms of HSP. There is some chance that an effective treatment for SPAST HSP may also be effective with some other types of HSP. Planning for clinical trials is in progress, and while SPAST HSP is the focus, decisions on participant inclusion/exclusion criteria are still to be determined, so, yes, it is too early to say yet.

  2. Wonderful news to hear that this research has progressed so far and thank you to all who have contributed.

    Whilst I understand as a person with atypical complicated SPG 4, approximately 30 years ago I worked as a laboratory technician and for twelve years as a volunteer layperson on a Human Ethics Committee. The structure of these committees has changed significantly since then but if I could assist in a voluntary capacity I would do so.

    Thanks Jordana

  3. This is great news, well done to you all for working so hard in trying to achieve a treatment for HSP. Can you please tell me how much longer until there is an effective treatment for complicated SPGs?

  4. Many thanks to all those people for their great work in the search for a cure for our disease. I am Catalan from Barcelona. Here, some neurological doctors are also trying to find a way to cure HSP, but I think they are behind the work done in Australia. If in the end you can find a way to cure HSP, what will we have to do, we non-Australians?

    Now, through a web page, I will make a request to get more donations for you, so that they can continue with their work that I hope with all my heart that they achieve their objective, and thus, to be able to cure HSP and make it disappear so that nobody else can inherit it.
    Thank you very much again.


    1. Editor’s Note: The Foundation communicates progress every quarter to the AEPEF support group in Spain, as we do to all national support groups around the world. There will be plenty of communications about progress with clinical trials, and rest assured that if an effective treatment is found, you will hear about it. Thank you for raising funds for us to support HSP clinical trials.

  5. Hi. I am a 24 year old from Indiana living with HSP. When trials happen I really want to be a part of them. If this is possible please contact me.

  6. Hi, I live in Chicago, Illinois and living with HSP. I would like to be part of the trials, if possible. Please contact me.

  7. Am too diagnosed with spg4. Any guess on when cure will be available? Thnx. Clinical trials-i would like to be able as well

    1. Editor’s Note: There are far too many time dependent variables in the clinical trials process to make an educated guess as to when trials will be finished – including the assumption that the drug treatment is successful.

      It appears that you are from the USA. Make sure that you are a member of the SP Foundation there, as they will be providing their members with relevant, up-to-date information on clinical trials as it becomes available.

  8. I have a variant of spg4 that they have not seen before. It is in 3 known generations of my family. I would be more than happy to help out in ANY way that I can!
    I am in Canada.

    1. Editor’s note: There is no way of knowing at this stage. It might work. Whilst there are 70+ HSP types described to date, there are only a handful of causal mechanisms https://hspersunite.org.au/about-hsp/understanding-hsp-mechanism/. Theoretically, an effective treatment for any given type of HSP would likely mean restoration of the functions that were impaired or success in bypassing the particular HSP causing mechanism. The mechanism of SPG13 type HSP is not well-defined to this point. However some studies have found a connection with SPG4 HSP, while others reference mitochondria http://neuromuscular.wustl.edu/spinal/fsp.html#spg13. So this is the basis for saying that an effective treatment for SPG4 HSP ‘might’ also be effective with SPG13 HSP.

      First we need to test the effectiveness of the drug in SPG4/SPAST HSP and then find out what other forms it might work on. The theoretical hypotheses and assumptions need to be put to the test… then there could be an answer to your question.

  9. I am in the process of testing for HSP. . Very depressed on how my life has changed in just three years.
    I found this article by accident and found it promising

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