Gene-modified mesenchymal stem cells as a potential treatment for Huntington’s disease: preparing for a planned Phase I clinical trial

Available to view on demand. The UC Davis research team present an update on preclinical development of the cell-gene therapy product and the planned Phase I trial.

Huntington’s disease (HD) is an autosomal dominant neurodegenerative disease caused by a CAG triplet expansion in the Huntingtin gene, which causes progressive neuropsychiatric and motor dysfunction and leads to death. The HD mutation causes selective loss of striatal neurons, leading to more widespread brain degeneration. No current disease-modifying therapies exist, and treatment is strictly palliative. An important mechanism postulated to lead to neuronal death in HD is impaired expression of brain-derived neurotrophic factor (BDNF), and multiple studies in animal models support increased BDNF as a potential disease-modifying therapy for HD. Mesenchymal stem/stromal cells (MSCs) have innate neurorestorative properties that promote endogenous neuronal growth, and we have engineered donor MSCs to express high levels of BDNF for neurotransplantation in a planned Phase I safety and tolerability trial for patients with early-stage HD.

What you will learn?

  • Proposed mechanism of engineered MSCs as a potential treatment for Huntington’s disease
  • Progress made to date in developing and manufacturing MSC/BDNF in
    readiness for regulatory approval for a first-in-human Phase I trial
  • Results of preclinical studies in transgenic HD mouse models in support of the application for regulatory approval
  • Design of the lead-in observational study and the future planned Phase I clinical trial
  • Potential future applications of this approach in other neurological disorders

Who may this interest?

  • Basic science researchers in stem cell research, genetics, neurodegeneration and gene therapy
  • Clinicians (neurology, psychiatry and primary care) involved in the care of patient with neurological and psychiatric diseases
  • Patients and families affected by neurodegenerative diseases


  • Vicki Wheelock, Health Sciences Clinical Professor, Department of Neurology, UC Davis
  • Jan Nolta, Professor, Director of the Institute for Regenerative Cures and Stem Cell Program, UC Davis
  • Kyle Fink, Ruth L Kirschstein NRSA Postdoctoral Fellow, UC Davis

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