Aspen wins $8M to advance Parkinson’s stem cell therapy

Grant will support ongoing Phase 1/2a trial that's using patients' own cells

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by Andrea Lobo |

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Aspen Neuroscience has been awarded an $8 million grant to advance the clinical development of ANPD001, its investigational stem cell therapy that’s designed to replace dopamine-producing nerve cells lost in Parkinson’s disease.

The grant, funded by the California Institute for Regenerative Medicine (CIRM), will support the ongoing Phase 1/2a clinical trial that’s evaluating the treatment in people with Parkinson’s disease.  The trial is assessing the use of patients’ own cells.

“Providing patients in this study with dopamine neurons made from their own cells is a huge leap forward for personalized medicine, and has the potential to impact the entire field of neurodegenerative disorders,” Damien McDevitt, PhD, Aspen’s president and CEO,  said in a company press release.

“This clinical award represents a significant step forward in the treatment landscape of Parkinson’s disease by advancing individualized therapy, which has the potential to restore motor function in patients impacted by this devastating condition,” said Abla Creasey, PhD, vice president of therapeutics development at CIRM.

Parkinson’s is caused by the progressive dysfunction and death of neurons that produce dopamine, a major brain chemical messenger, in the nigrostriatal pathway, which includes the substantia nigra and the dorsal striatum, brain regions involved in motor control.

“By the time of diagnosis, it is common for people with Parkinson’s to have lost the majority of dopaminergic (DA) neurons, leading to progressive loss of motor and neurological function,” said Edward Wirth III, MD, PhD, chief medical officer at Aspen.

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What is ANPD001 expected to do in Parkinson’s disease?

ANPD001 uses induced pluripotent stem cells (iPSCs), a type of stem cell that can generate nearly all types of cells in the body, including dopamine-producing neurons. The process involves collecting skin cells from a patient and reprogramming them in the lab as iPSCs.

After iPSCs differentiate into dopamine neuronal precursor cells using specific chemical or biological molecules, they are transplanted back to the patient, where they eventually mature into dopamine-producing neurons.

The main goal of the Phase 1/2a ASPIRO trial (NCT06344026) is to evaluate the long-term safety and tolerability of ANPD001 when transplanted at two escalating doses in people with moderate to severe Parkinson’s, ages 50-70.

Secondary goals include increased on time, that is, periods when patients’ symptoms are well controlled by medication, reduced motor symptoms, and improvement in patients’ quality of life. Cell survival after the transplant will also be assessed, using imaging brain scans.

The study has completed enrollment, with the patients having been invited by the researchers in advance. The first patient was transplanted last month at the Banner-University Medical Center Tucson and patients will continue to be dosed this year, Wirth said.

The primary follow-up of the trial a year after the transplant should be completed next year and the treatment’s effects will be assessed for five years after the transplant. Long-term safety data will be evaluated annually for 10 more years via phone call.