STEM-PD Phase 1 trial advances with positive early safety findings
4 patients will receive higher dose and be monitored for 3 years
A Phase 1 clinical trial testing the investigational stem cell-based therapy STEM-PD in people with Parkinson’s disease has advanced to a higher dose following positive findings about the safety of the lower dose, according to a press release from Lund University.
Four patients will receive the high dose and will be monitored for three years. The four patients already given the lower dose will also continue to be monitored for clinical outcomes.
Initially, the first in-human trial (NCT05635409) only recruited participants at a single hospital in Sweden, but U.K. regulators approved the trial last October so patients in the U.K. may be included in the second dosing group.
Patients recruited by invitation only
Investigators have indicated that eligible patients will be recruited by invitation.
“Participation is not an open recruitment process, and the clinical trial team will not be accepting any requests from individuals volunteering to take part in the trial,” states the trial’s webpage.
Parkinson’s patients experience the progressive loss of nerve cells that produce the signaling chemical dopamine, or dopaminergic neurons, and this drives the motor symptoms of the neurodegenerative disease.
There’s been increasing interest in developing cell-based therapies to replace these lost cells, addressing the disease’s underlying cause to slow or stop its progression.
Developed at Lund University in Sweden, STEM-PD is an investigational therapy that leverages human embryonic stem cells to achieve that goal. These cells have the ability to develop into virtually any mature cell type in the body under the right conditions.
With STEM-PD, the stem cells are isolated and programmed in the lab to become dopaminergic precursor cells. They’re then surgically transplanted directly into the putamen, a brain region in which dopaminergic cells are particularly lost in Parkinson’s, where they’re expected to mature into healthy dopaminergic neurons.
Preclinical studies found STEM-PD to be safe
Preclinical studies have found the therapy to be safe, with the ability to produce dopaminergic cells in the brain and reverse motor problems in rats.
The STEM-PD trial was cleared by Swedish regulators in 2022, with the first patient dosed early last year.
The trial mainly seeks to assess the safety and tolerability of STEM-PD in the year after transplant among a group of eight adults with moderate Parkinson’s disease, ages 50-75, who have been living with the condition for more than 10 years and have symptoms not appropriately controlled by existing oral therapies.
All participants will receive a single dose of STEM-PD and be followed for up to three years.
The first four patients were given a dose of 3.5 million cells per putamen. Investigators now report that no concerning side effects have been reported and that all patients are doing well.
Based on these findings, they have initiated dosing of the next four patients with 7.1 million cells per putamen.
Secondary trial goals include imaging to determine the survival of the transplanted cells as well as clinical efficacy for three years after transplant.
While it is too soon to evaluate clinical efficacy, the investigators note that PET imaging scans from patients treated at the lower dose show signs of dopamine cell survival at 6-12 months post-transplant.
STEM-PD’s development and the trial are funded by national and European Union funding agencies, as well as Novo Nordisk.
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