Boston-based company uniQure is conducting the first HD gene therapy trial of their drug AMT-130. The Phase 1/2 trial is designed to test the safety of AMT-130, and uniQure shared more about the trial in a community statement during HDSA’s 2019 Convention. Further details, including site locations, have emerged this week and can be viewed at www.hdtrialfinder.org and at clinicaltrials.gov.
AMT-130 is a piece of genetic material that is packaged inside a type of harmless virus and delivered deep into the brain via a neurosurgery. Once the virus containing the drug enters a brain cell, it can find and chop up huntingtin RNA, so that the cell makes less toxic huntingtin protein. In theory, with only one surgical procedure, the drug will keep attacking huntingtin indefinitely. This experimental procedure has so far been successful at lowering huntingtin in animals like mice and minipigs.
During the trial, 26 patients with early HD symptoms will undergo an 8-10 hour surgical procedure. Microscopic catheters, the width of a human hair, will be guided to several locations within the striatum, the deep brain regions that are most affected by HD. 16 participants will receive AMT-130, and 10 will have an imitation surgery. For 18 months after the surgery, participants will have frequent follow-up visits, with imaging, neurological exams, blood draws, and other tests to determine the safety of the drug. Then they will have yearly visits until 5 years after the surgery.
This first HD gene therapy trial is an extraordinary milestone in HD clinical research, and HDSA will continue to work closely with uniQure to provide families with up-to-date information about the progress of the study.
Decision-Making Around HD in Romantic Relationships
Has Huntington’s disease affected the way you approach dating, relationships, and/or family planning? Researchers at the University of California, Irvine are conducting a study exploring the process of disclosing a diagnosis or family history to a romantic partner and which factors couples consider for reproductive decisions. The information gathered from this study can give healthcare professionals, such as genetic counselors, deeper insight into this important aspect of life for the Huntington’s disease community. Please visit this link to participate in a short confidential survey: https://is.gd/HDsurvey
To be eligible for this survey you must be at least 18 years old and:
- At risk for Huntington’s disease OR
- Diagnosed with Huntington’s disease OR
- The romantic partner of someone who is at risk for Huntington’s disease OR
- The romantic partner of someone with Huntington’s disease
Please feel free to contact researcher Marian Tsang with any questions or concerns at (714) 456-5837 or mjtsang@uci.edu
