Drug Discovery and Development

  • Home Drug Discovery and Development
  • Drug Discovery
  • Genomics/Proteomics
  • Oncology
  • Neurological Disease
  • Infectious Disease
  • R&D 100 Awards
  • Pharma 50
    • 2022 Pharma 50
    • 2021 Pharma 50

Scientists Develop Test to Measure Effectiveness of Treatments for Huntington’s Disease

By Drug Discovery Trends Editor | August 7, 2015

Blair Leavitt of UBC’s Centre for Molecular Medicine and Therapeutics (Credit: University of British Columbia)A new test developed by UBC researchers allows physicians to measure the effects of gene silencing therapy in Huntington’s disease and will support the first human clinical trial of a drug that targets the genetic cause of the disease.
 
The gene silencing therapy being tested by UBC researchers aims to reduce the levels of a toxic protein in the brain that causes Huntington’s disease.
 
The test was developed by Amber Southwell, Michael Hayden, and Blair Leavitt of UBC’s Centre for Molecular Medicine and Therapeutics and the Centre for Huntington Disease in collaboration with colleagues from Mayo Clinic. It was recently featured in a study published in Scientific Reports.
 
“This is an important breakthrough for several promising gene silencing therapies in Huntington’s disease that are now moving from the bench to the bedside,” said Leavitt. “We can move forward with these clinical trials and accurately measure whether our treatments are working.”
 
Huntington’s disease is a genetic disorder but symptoms generally don’t appear until later in life. It affects the brain and gradually worsens, causing problems with coordination and movement, mental decline and psychiatric issues.
 
The genetic mutation responsible for Huntington’s produces a toxic form of a protein called huntingtin, which progressively injures brain cells. Reducing brain levels of this toxic protein should prevent or delay the onset of symptoms. Several huntingtin-lowering therapies have already shown great promise in animal models of Huntington’s disease and are rapidly approaching trials in humans.
 
The UBC research team found that they could accurately measure the levels of mutant huntingtin protein in the brain by collecting cerebrospinal fluid from a spinal tap. The ultrasensitive test detects small amounts of the toxic protein and can be used to follow changes in brain levels of the protein over time in response to new therapies.
 
This study enables Leavitt to initiate a new clinical trial of a huntingtin gene-silencing therapy for patients at the Centre for Huntington Disease at the Djavad Mowafaghian Centre for Brain Health, a partnership between UBC and Vancouver Coastal Health. This trial will test the safety of a novel gene-silencing treatment in patients and is already in the process of screening patient candidates. The trial will be the first human study of a drug targeting mutant huntington.
 
Source: University of British Columbia

Filed Under: Drug Discovery

 

Related Articles Read More >

Takeda Pharmaceutical in the Drug Discovery & Development Pharma 50
Takeda’s Takhzyro fares well in pediatric hereditary angioedema study
Novartis in the Pharma 50
Novartis to cut up to 8,000 positions
Gilead Sciences in the Drug Discovery & Development Pharma 50
Gilead resubmits application to FDA for twice-yearly HIV drug lenacapavir
George Floyd mural
How the pandemic and George Floyd made clinical trial diversity a priority

MEDTECH 100 INDEX

Medtech 100 logo
Market Summary > Current Price
The MedTech 100 is a financial index calculated using the BIG100 companies covered in Medical Design and Outsourcing.

Need Drug Discovery news in a minute?

We Deliver!
Drug Discovery & Development Enewsletters get you caught up on all the mission critical news you need. Sign up today.
Enews Signup
Drug Discovery and Development
  • MASSDEVICE
  • DEVICETALKS
  • Medical Design & Outsourcing
  • MEDICAL TUBING + EXTRUSION
  • MEDTECH 100
  • Medical Design Sourcing
  • Subscribe to our Free E-Newsletter
  • Contact Us
  • About Us
  • Advertise With Us
  • R&D World
  • Drug Delivery Business News
  • Pharmaceutical Processing World

Copyright © 2022 WTWH Media LLC. All Rights Reserved. The material on this site may not be reproduced, distributed, transmitted, cached or otherwise used, except with the prior written permission of WTWH Media
Privacy Policy | Advertising | About Us

Search Drug Discovery & Development

  • Home Drug Discovery and Development
  • Drug Discovery
  • Genomics/Proteomics
  • Oncology
  • Neurological Disease
  • Infectious Disease
  • R&D 100 Awards
  • Pharma 50
    • 2022 Pharma 50
    • 2021 Pharma 50