UC Irvine Receives Funding for Groundbreaking Clinical Trial of Neural Stem Cell Therapy for Huntington's Disease
The University of California, Irvine, has received a substantial $11,999,933 grant from the California Institute for Regenerative Medicine to fund a groundbreaking clinical trial. This trial will explore a novel neural stem cell therapy for Huntington's disease, a devastating genetic disorder that affects the brain. The study marks a significant advancement in the field of regenerative medicine, offering new hope to patients with limited treatment options.
The clinical trial, led by Leslie M. Thompson, Donald Bren Professor of Psychiatry and Human Behavior and Neurobiology and Behavior at UC Irvine, will focus on the safety and tolerability of an embryonic stem cell-derived neural stem cell product. This is a crucial step towards developing a therapy that can potentially protect brain cells, repair damaged circuits, and slow or prevent the progression of Huntington's disease. The trial will be the first of its kind, using embryonic stem cells, as opposed to fetal cells or tissue, in human patients.
Huntington's disease, which typically affects individuals between the ages of 35 and 50, is a progressive disorder that destroys brain cells over a period of 10 to 20 years. Symptoms include involuntary movements, cognitive decline, and mood changes such as depression. The therapy, known as hNSC-01, utilizes neural stem cells to protect existing brain cells, replace lost cells, and rebuild impaired circuits. These cells also release beneficial proteins like BDNF, which are often lacking in Huntington's disease patients, and reduce harmful protein accumulations that damage brain cells.
The clinical trial will enroll 21 individuals with early-stage Huntington's disease, divided into two groups: a Phase 1B dose-escalation group and a Phase 2A expansion group. The cells will be surgically administered into the brains of participants, and their safety and potential benefits will be closely monitored.
The impact of Huntington's disease is immense, affecting not only patients but also their families and caregivers. Hospital costs for HD patients in California alone can range from $3 million to $25 million annually. If this therapy proves successful, it could significantly improve the quality of life for individuals with Huntington's disease, allowing them to live more independently and reducing long-term care costs. This could alleviate the burden on families and healthcare systems.
The trial is a testament to the dedication of the research team, including Dr. Jack Reidling and Yuna Muyshondt at UC Irvine, as well as the UC Davis GMP facility, which produced the cell product. Thompson's extensive research on Huntington's disease, spanning over three decades, has been supported by $17.6 million in CIRM funding. In 2019, she received $6 million to develop stem cell-based therapies for Huntington's, and her efforts have led to the clearance of an investigational new drug application with the FDA.
Additionally, Thompson's recent $2 million CIRM grant aims to unravel the mystery of why Huntington's disease affects some brain cells while sparing others. Her laboratory employs a multidisciplinary approach, combining patient-derived stem cells, transcriptomics, proteomics, and bioinformatics to investigate disease mechanisms and understand the vulnerability of specific brain cells.
This award is part of CIRM's Clinical Trial Stage Projects, which aim to accelerate the development of promising stem cell therapies from late-stage preclinical research to clinical trials, addressing critical medical needs. The trial represents a significant step forward in the field of regenerative medicine, offering a glimmer of hope to patients and families affected by Huntington's disease.
