ANPD001 eases motor symptoms, early trial data show
First 3 patients treated saw improved daily functioning without side effects
The first three patients treated with ANPD001, a cell therapy for Parkinson’s disease, showed reduced motor symptoms and improved daily functioning over six months, without any serious side effects.
That’s according to early data from the Phase 1/2a ASPIRO (NCT06344026) study, an open-label clinical trial testing the safety and tolerability of ANPD001 over one year when injected at either of two doses in adults with moderate to advanced Parkinson’s. Researchers are also tracking changes in on time, or periods when motor symptoms are adequately controlled.
“In this first-of-its-kind study, we are seeing clinician-reported and patient-reported improvements as well as a strong safety and tolerability profile at six months,” Edward Wirth III, MD, PhD, chief medical officer at developer Aspen Neuroscience, said in a company press release.
The data were presented in a poster at the 30th World Congress on Parkinson’s Disease and Related Disorders, held May 7–10 in New York. An abstract for the poster, “Safety, tolerability, and efficacy of intracranial delivery of autologous iPSC-derived dopaminergic precursors in moderate to advanced Parkinson’s disease,” was published in Parkinsonism & Related Disorders.
Parkinson’s symptoms result from the loss of dopaminergic neurons, the nerve cells that produce dopamine in the brain. Dopamine is involved in motor control, and low levels of dopamine lead to slowed or stiff movements, tremors, and problems with balance.
Replacing lost neurons
ANPD001 is designed to replace the dopaminergic neurons that are gradually lost in Parkinson’s. As an autologous therapy, it uses a patient’s own cells, eliminating the need for immunosuppressive drugs to prevent rejection by the immune system. The process begins by collecting skin cells from the patient, which are then reprogrammed in the lab into induced pluripotent stem cells.
These stem cells are guided to develop into precursors of dopaminergic neurons and returned to the patient by MRI-guided injection into a brain region called the putamen, where they’re expected to develop into functional dopaminergic neurons.
The ASPIRO study involves patients ages 50–70 who respond to levodopa, a mainstay Parkinson’s treatment that provides the body with a molecule it can use to produce dopamine, but still have uncontrolled motor symptoms. Its main goal is to test ANPD001’s safety over one year, with patients continuing to be monitored for up to 15 years.
Since the study’s launch last year, ANPD001 has been found safe and well tolerated, with no serious side effects. All patients in the first two groups who received the investigational cell therapy were sent home within 48 hours as planned.
The recently presented data showed that the three patients who received a low dose of ANPD001 — 5 million precursors of dopaminergic neurons each, delivered in a surgical procedure under general anesthesia — experienced no bleeding or graft-induced dyskinesia (uncontrolled movements caused by the cell therapy). The most common side effects were pain at the surgical site and tongue swelling from lying face down during the procedure.
At six months, patients experienced a 45% improvement in MDS Unified Parkinson’s Disease Rating Scale (UPDRS) Part III scores, a measure of motor symptom severity, when they were off medication. They also reported a 71% improvement in MDS UPDRS Part II scores, which measures their ability to perform everyday activities such as eating, dressing, and walking.
On average, patients had two fewer hours each day of off time, periods when motor symptoms aren’t adequately controlled despite treatment with levodopa, and 1.5 more hours each day of good on time, when motor symptoms are adequately controlled without troublesome side effects, such as involuntary movements.
ANPD001 has received fast track designation from the U.S. Food and Drug Administration, which may help accelerate its development and regulatory review. Benefits of this status include frequent guidance, expedited approval processes, and eligibility for priority review and accelerated approval.
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