The first patient in the world has received an experimental stem cell therapy as part of a clinical trial for Huntington’s disease.

The phase 1b/2a trial, led by the University of California, Irvine, will evaluate the safety of hNSC-01 neural stem cells, which are delivered to the brain by a specialised neurological mapping and targeting stereotactic system and performed in an MRI suite.

The trial is the result of several years of basic and translational science carried out at UC Irvine by a research team led by Leslie M. Thompson (pictured) and funded by the California Institute of Regenerative Medicine.

In animal studies, the hNSC-01 therapy was shown to protect existing brain cells, replace lost cells, rebuild impaired brain circuits and reduce harmful protein accumulations that damage brain cells.

The first patient received the intervention at UCI Health, home to Orange County’s first adult bone marrow/stem cell transplant and cellular therapy program, in May.

The stem cells were implanted during a roughly six-hour surgical procedure under general anaesthesia. While lying face down in an MRI scanner, the patient received stem cells implanted directly into the striatum deep in the brain.

A second patient is scheduled to receive the intervention next month.

“The first patient intervention went very well. To date, they haven’t reported any serious adverse events,” said UCI Health neurologist Dr. Ravi Rajmohan, the clinical trial’s principal investigator.

“This trial may help us move one step closer to a future with available treatments that could potentially slow the progression of Huntington’s disease.”

Huntington’s disease is a fatal, progressive genetic disorder that gradually destroys brain cells. It usually begins between the ages of 35 and 50, worsening over 10 to 20 years.

Researchers believe that, if successful, the therapy could prolong independent living among patients with Huntington’s Disease and significantly reduce long-term care costs, easing the strain on families.

The clinical trial will enrol 21 people aged 18 to 65 with early-stage Huntington’s disease. Twelve participants will be enrolled into a phase 1b dose-escalation group and nine in a phase 2a expansion group.