Thursday, 4 August 2016

Spatax Meeting - Papers, Day 2, part 2

This is the third instalment of the papers presented, this time the afternoon of the second day. As previously, the papers were a mix of HSP and Ataxia papers, and I focused on paying most attention to the HSP papers.

Full post index:
Papers Day 1:
Papers Day 2a:
Papers Day 2b:
Papers Day 3:
Posters 1:
Posters 2:

The first of the afternoon sessions focused on therapeutic approaches, although all of the papers had an Ataxia spin.

Helene Puccio ( described using computer gait analysis, and using it to investigate gene therapy in mice. One of the aspects they have been looking at is what happens if treatments are given after symptoms are showing - they conclude that if the treatment is given before the death of the neurons then it works.

Eleonora Di Gregorio ( talked about speech communication problems affecting quality of life - The symptom is dysarthria - and this occurs in some HSP's too.

The second of the afternoon sessions focused on therapeutic approaches with biomarkers, and again, most of the papers had an Ataxia spin.

Thorsten Schmidt ( talked about some work involving re-purposing existing drugs for SCA3, and gave 2 examples - Riluzole and Citalopram, both of which were tested on mice - Riluzole was not found to give any benefits and Citalopram was. They are trying to get Citalopram into pre-clinical trials.

Benjamin Cravatt ( similarly talked about drug re-purposing for HSP. He began by describing that some cell pathways for the spread of disease are known, but many are not. They are looking at potentially re purposing diabetes drugs for HSP.

In the discussions at the end of the session it was commented that patient numbers for studies are generally low, and there would be some benefit from regional/continental/worldwide patient registries. As there are overlaps between HSP and Ataxia it is good for the researchers to work together.

As before, I realise that it is on a very different level to my normal posts. I know I've just used a lot of terms that I dont normally use, and I've just kept this post to the points which I perceived to be most important.

Key takeaway points are:
  • Treatments are unlikely to be effective if given after the death of neurons.
  • Some are looking at drug re-purposing for HSP/Ataxia at the moment
  • Several Ataxia symptoms and the way they feature are similar to HSP
  • Researchers are looking for bigger/better patient registries

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