A Phase 1/2 clinical trial dubbed ASPIRO thats testing Aspen Neurosciences ANPD001 a stem cell therapy candidate designed to replace the nerve cells that are lost in Parkinsons disease has dosed its first patient.

A first transplant was conducted at the Banner-University Medical Center Tucson by neurosurgeon Paul Larson, MD, the trials lead investigator, Aspen announced in a company press release

ASPIRO (NCT06344026), which was cleared by the U.S. Food and Drug Administration (FDA) last year, is testing the long-term safety and tolerability of the ANPD001 stem cell therapy when transplanted at two escalating doses in people with moderate to severe Parkinsons. The eligible study participants range in age from 50 to 70.

The initiation of this clinical trial is a major milestone in Aspens mission to develop and deliver personalized, regenerative neurologic therapies for people with unmet medical needs, starting with Parkinsons disease, said Damien McDevitt, PhD, president and CEO of Aspen Neuroscience.

To date, there is no disease-modifying therapy that can stop, replace or prevent the loss of dopamine neurons or slow the progression of Parkinsons, McDevitt said.

Additional trial goals will include assessments of the therapys early efficacy by measuring so-called on time, or periods when a patients symptoms are controlled by medications and the easing of motor symptoms. The trial is running at five clinical sites in the U.S.

Parkinsons is caused by the death of dopamine-producing neurons in the brains nigrostriatal dopaminergic pathway. That pathway includes the substantia nigra and the dorsal striatum, both involved in motor control. Dopamine is a major brain chemical messenger.

ANPD001 aims to replace the dopaminergic neurons that are lost in Parkinsons.

This experimental therapy for Parkinsons uses a type of stem cell called an induced pluripotent stem cell, or iPSC, which is able to generate nearly any type of cell in the body including dopamine-producing neurons. Its manufacturing is a three-step process.

The first step involves collecting skin cells from a patient, which are then modified in the lab and reprogrammed into iPSCs. The iPSCs are then provided with biochemical cues that guide them into transforming into dopamine neuronal precursor cells. These cells will eventually mature into dopamine-producing neurons once transplanted into patients.

The procedure is known as autologous because it uses a patients own cells.

This is the first use of the autologous approach in a formal clinical trial, saidLarson, also a professor of neurosurgery at the University of Arizona College of Medicine-Tucson.

Larson called it an honor to take part in ASPIRO, saying its an important study.

Parkinsons disease is the most common neurodegenerative movement disorder, primarily affecting the depletion of dopamine neurons in the midbrain, Larson said.

By the time of diagnosis, it is common for people with Parkinsons to have lost the majority of dopaminergic (DA) neurons in the nigrostriatal pathway, which leads to progressive loss of motor and neurological function, Larson said.

The enrolled participants expected to be nine in total were first remotely monitored via a digital health platform, by Rune Labs, as part of a Trial-Ready Cohort Screening study. The goal was to have a comprehensive view of the disease ahead of patient recruitment.

Also included in the trials additional goals, along with increased on time and reduced motor symptoms, is improvement in patients quality of life.

This first-in-human trial holds significant promise to investigate the ability of ANPD001 to improve the lives of people with moderate to advanced Parkinsons disease.

The trials primary phase is expected to be completed by next year, and patients will be assessed for five years post-transplant, with the use of imaging scans of the brain.

This first-in-human trial holds significant promise to investigate the ability of ANPD001 to improve the lives of people with moderate to advanced Parkinsons disease, said Edward Wirth III, MD, PhD, Aspens chief medical officer.

Our 2022 Trial Ready Cohort Screening Study has completed enrollment, and we plan to dose patients in the ASPIRO Phase 1/2a study this year, Wirth said.

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Trial testing Parkinson's cell therapy ANPD001 treats 1st patient - Parkinson's News Today