Patient Screening Study for Potential Cell Therapy to Open Soon in US

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by Marta Figueiredo, PhD |

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During Parkinson’s Awareness Month, Aspen Neuroscience will launch a patient screening study to potentially advance ANPD001, its experimental, personalized neuron replacement therapy for Parkinson’s disease, into clinical trials.

Considered to be the first of its kind, the Trial-Ready Cohort Study will involve several screening sites in the U.S. They are expected to be announced throughout the spring.

It will serve as a preliminary step to support Aspen’s filing of a new investigational drug (IND) application with the U.S. Food and Drug Administration seeking clearance to test ANPD001 in people, starting with a planned Phase 1/2a trial in Parkinson’s patients.

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The Trial-Ready Cohort Study is expected to provide information necessary to screen patients as potential candidates for the trial.

“This is an historic moment for patients and for the Aspen Neuroscience team, as we open our first screening study to expedite our investigation of iPSC-derived cell replacement therapies for Parkinson’s disease,” Damien McDevitt, PhD, Aspen’s president and CEO, said in a press release.

“We are excited and very humbled to begin this next phase during Parkinson’s Awareness Month,” McDevitt said, adding that “this is a significant step forward for the patient community, for health care providers and the neuroscience field.”

Parkinson’s is characterized by the progressive loss of dopamine-producing, or dopaminergic, neurons in brain regions involved in voluntary movement. Dopamine is a major brain signaling molecule, and its deficiency leads to the disease’s hallmark motor symptoms.

By combining its expertise in stem cell biology, genetics, and neurology, Aspen developed ANPD001, the first potential, patient-derived neuron replacement therapy for Parkinson’s.

The therapy involves collecting a patient’s own skin cells and reprogramming them back to a stem cell-like state — then called induced pluripotent stem cells, or iPSCs — that can give rise to almost every type of human cell, including dopamine-producing neurons.

iPSC-derived dopaminergic neurons are then re-implanted in the patient, which is expected to ease or reverse Parkinson’s motor symptoms. Before being transplanted, each patient’s cells will be analyzed using artificial intelligence-based genetic tools.

Because these cells are derived from the patient, they can be re-implanted without the risk of being rejected, eliminating the need for immunosuppressive treatment, which can increase the risk of infections.

ANPD001 is being tested in IND-enabling preclinical studies, supported by the $70 million Aspen raised in 2020 to advance both of its Parkinson’s therapy candidates.

In addition to ANPD001, the company is developing ANPD002, a gene-editing therapy for Parkinson’s associated with the most common mutation in the GBA gene — a major genetic risk factor for the disease.

This treatment differs from ANPD001 in that it involves correcting the GBA mutation in patient-derived cells before their re-implantation. This approach is in its early stages of development, and proof-of-concept preclinical studies have yet to be completed.