‘Thaw-and-inject’ cells given to new groups in Parkinson’s trial

Aspen Neuroscience has expanded its ongoing Phase 1/2a clinical trial by dosing two new groups of Parkinson’s disease patients with sasineprocel (ANPD001). For the first time in the study, these participants received a new, commercial-ready formulation of the cell therapy designed to make future manufacturing and treatment delivery much easier to scale.

This formulation utilizes cryopreserved, “thaw-and-inject” cells that are ready for dosing at the clinical site.

The open-label ASPIRO study (NCT06344026) is testing the safety and tolerability of sasineprocel over one year in adults with moderate-to-severe Parkinson’s.

“We are proud to reach this important milestone, which reflects meaningful progress in the sasineprocel program and underscores the growing clinical experience with our personalized, autologous approach,” Damien McDevitt, PhD, Aspen’s president and CEO, said in a company press release. “Importantly, the use of our commercial formulation in these cohorts represents a critical step toward scalable manufacturing and commercial readiness as we prepare for Phase 3 initiation.”

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Replacing lost dopamine neurons

Parkinson’s is caused by the loss of dopaminergic neurons, which are the nerve cells that produce dopamine, a brain signaling molecule involved in motor control. Impaired dopamine signaling in the brain leads to Parkinson’s symptoms, including slowed movements, rigidity, tremors, and balance issues.

Sasineprocel is an autologous cell therapy designed to replace these gradually lost dopaminergic neurons. The process begins by collecting skin cells from the patient, which are then reprogrammed in the lab into induced pluripotent stem cells.

These cells are then guided to develop into dopaminergic neuronal precursors, which are delivered to the patient via MRI-guided injection into the putamen, a brain region involved in Parkinson’s disease, where they’re expected to mature into functional dopaminergic neurons.

The ASPIRO study includes patients 40-75 years old who have uncontrolled motor symptoms despite responding to levodopa, a mainstay of Parkinson’s treatment. The main goal is to test sasineprocel’s safety over one year, with patients followed for up to 14 more years.

Secondary goals include measuring the treatment’s efficacy at increasing on time, or periods when symptoms are well controlled with medication, reducing motor symptoms, and improving quality of life. Cell survival after the transplant will also be assessed.

To date, the treatment has been found to be generally safe and well tolerated, with no serious side effects in the first two groups of patients, who were all discharged 48 hours after surgery. Data from the first three patients treated also demonstrated that the cell therapy eased motor symptoms and improved daily functioning over a six-month period.

Subsequent results at the one-year mark showed sustained safety, tolerability, and clinical benefits, including improvements in daily functioning, physician- and patient-reported outcomes, and quality of life. This data represented the first eight patients who received the therapy at a low or high dose.

The two new groups received the commercial formulation, which, in preclinical studies, was comparable to the early formulation tested in the first two groups of patients. The therapy is the same; what changes is how the cells are manufactured and delivered.