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Understanding disease progression: PSPA researcher publishes results

We are delighted that our Sara Koe Clinical Research Fellow, Dr Edwin Jabbari, has published the results of some his work in the journal Annals of Neurology. Ed originally set out to improve understanding of why some people with PSP experience faster progression than others, and his new paper provides part of the answer. Ultimately, this area of work will help neurologists give families a more accurate prognosis, as well as making clinical trial results easier to interpret.

Ed’s project wouldn’t have been possible without your support, and he has written a blog post for us to explain his findings in a little more detail and tell us what else he’s been up to.

Dear PSPA supporters,

It has been a long time since my last update – rest assured I have been very busy with a number of different research projects!

I’m delighted to say that we have completed a research study which will be published in the journal, Annals of Neurology. We have looked at the genetic (inherited material) differences between patients with typical PSP compared to patients with slower progressing forms of PSP. In total we studied 600 PSP patients and found that variation in a gene called TRIM11 was an important determinant of the phenotype (characteristics) of PSP.  The interesting thing about this gene is that it may play an important part in nerve cells’ waste disposal system called the ubiquitin proteasome system (UPS), involved in the breakdown of misfolded proteins, including tau.

Whilst follow up work needs to be done, including looking at the impact of this gene in other neurodegenerative diseases such as CBD, it is possible that the UPS could be a target for future drug development.

Importantly, this study wouldn’t have been possible without the PSPA and all of its supporters. A significant portion of the 600 PSP patients came from our PROSPECT-UK study with the clinical data and DNA samples that patients donated being central to the success of the study. So I’d like to say a massive thank you to the PSPA and our PROSPECT-UK patients and their families!

In the coming weeks we’re also hoping to publish our biomarker data that was generated using blood and spinal fluid samples from PROSPECT-UK patients, looking at variation in a number of proteins related to PSP. Recruitment to the PROSPECT-UK study is currently ongoing and the total number of participants is now over 700.

At The National Hospital for Neurology and Neurosurgery, we have also had the opportunity to be one of the UK sites participating in the Biogen tau antibody clinical trial. Recruitment to the study has now closed and we hope to see preliminary results of the trial in the next 6-12 months. This study has recruited more than 400 PSP patients across the world. We believe that it is likely that further therapy studies will develop over the next year.

Lastly, I’m really looking forward to presenting both the genetic and biomarker findings at the PSPA/CurePSP research symposium in London in October of this year. It’s great that the international research community will be meeting to share ideas and forge new collaborations.

Have a great summer,

Ed

You can read Ed’s paper in full at https://onlinelibrary.wiley.com/doi/full/10.1002/ana.25308

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