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WE WANT TO HEAR FROM YOU…

Calling all UCL graduates! We hope you enjoy this issue of Portico and that it sparks ideas, conversations and debate. Read it, share it with your friends and family and then tell us what you think. Are facts dead (page 14) or will expertise win in the end? Do you agree with Professor Dittmer that Donald Trump’s presidency can be understood in the context of Captain America (page 26)? What are your experiences of the front line of justice (page 30)? We look forward to hearing your thoughts on all of these and your memories of your time at UCL. So wherever are you in the world, and whether you graduated last September or last century, join us in print, by email and on social media and share your thoughts.

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Illustration Hanna Melin

HUNTINGTON’S TRIAL HAILED A “BREAKTHROUGH” MOMENT

Ground-breaking results in fight against brain disease

A world without degenerative brain diseases has become a small step closer to reality, with news of a major breakthrough of “ground-breaking importance”.

A landmark trial for Huntington’s disease, led by UCL scientists, has announced positive results, suggesting that an experimental drug could become the first to slow the progression of the devastating genetic illness.

The results have been hailed as “enormously significant” because it is the first time any drug has been shown to suppress the effects of the Huntington’s mutation that causes irreversible damage to the brain. Current treatments only help with symptoms, rather than slowing the disease’s progression.

After more than a decade in pre-clinical development, this first human trial of the IONIS-HTTRx drug began in late 2015, led by Professor Sarah Tabrizi (UCL Institute of Neurology) and sponsored by Ionis Pharmaceuticals.

The trial involved enrolling 46 patients with early Huntington’s disease at nine study centres in the UK, Germany and Canada, and giving some of them the IONIS-HTTRx drug and some others a placebo. As hoped, IONIS-HTTRx produced significant, dose-dependent lowering of the level of mutant huntingtin – the first time the protein known to cause Huntington’s has been lowered in the nervous system of patients.

Professor Tabrizi, Director of the UCL Huntington’s Disease Centre and IONIS-HTTRx Global Chief Investigator, says: “The results of this trial are of ground-breaking importance for Huntington’s disease patients and families. For the first time a drug has lowered the level of the toxic disease-causing protein in the nervous system, and the drug was safe and well tolerated. The key now is to move quickly to a larger trial to test whether the drug slows disease progression.”

According to Professor Michael Hanna, Director of the UCL Institute of Neurology: “These findings open a new chapter in therapy development for Huntington’s disease. In addition, it has clear relevance for other devastating neurodegenerative disorders including dementia, Parkinson’s disease and neuromuscular diseases such as motor neurone diseases that are caused by single gene mutations. The unique environment at the UCL Queen Square Institute of Neurology is perfect to lead the world in this next exciting phase of antisense therapy development for patients.”

For more information on the UCL Huntington’s Disease Centre, visit www.hdresearch.ucl.ac.uk

  • Contents 2017/18Contents 2017/18
  • DeconstructedDeconstructed
  • We Want to Hear From You...We Want to Hear From You...
  • UCL Leads on DementiaUCL Leads on Dementia
  • Free RadicalFree Radical
  • Festival A HitFestival A Hit
  • Jeremy Bentham SpeaksJeremy Bentham Speaks
  • Extra CurricularExtra Curricular
  • Experts And FactsExperts And Facts
  • For Facts’ SakeFor Facts’ Sake
  • A Different Type of Capitalist MarketA Different Type of Capitalist Market
  • The Big CThe Big C
  • Trump In The Age Of Captain AmericaTrump In The Age Of Captain America
  • The Front Line Of JusticeThe Front Line Of Justice
  • Calling All UCL GraduatesCalling All UCL Graduates
  • CloisteredCloistered
  • The Strong RoomThe Strong Room
  • This idea must dieThis idea must die
  • London  vs  WorldLondon vs World
Portico Issue 4. 2017/18
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