Phase 2b Trial Completed for Potential Disease-Modifying Parkinson’s Treatment

Living Cell Technologies (LCT) has completed treatment in the six patients enrolled in the third and final cohort of the Phase 2b clinical trial evaluating NTCELL in the treatment of Parkinson’s disease (PD) at Auckland City Hospital in New Zealand.

LCT’s lead product, NTCELL, is an alginate-coated capsule containing choroid plexus cells sourced from a unique herd of pathogen-free pigs first discovered in the sub-Antarctic Auckland Islands.

Choroid plexus cells are naturally occurring “support” cells for the brain and secrete cerebrospinal fluid (CSF), which supports correct nerve cell functioning and protects enzymes critical to healthy nerve growth.

After transplant, NTCELL promotes new central nervous system growth and repairs disease-induced nerve degeneration. NTCELL’s encapsulation technology, IMMUPEL, allows the cell therapy to be used without the need for co-treatment with immune-suppressors as it protects the cells from the immune system’s attacks.

Current treatments available for Parkinson’s tend to focus on the symptoms and do not slow or reverse neuron degeneration. Most treatment options restore the balance of dopamine or other neurotransmitters, but their effectiveness declines as the disease progresses.

This Phase 2b trial was initiated in March last year to confirm the most effective NTCELL dose, define any placebo component of the response, and further identify the initial target Parkinson’s disease patient sub-group.

In the Phase 2b trial, four patients had 120 NTCELL microcapsules implanted into the putamen on each side of their brain and two patients had sham surgery with no NTCELL implanted. No safety issues were recorded with any of the six patients.

The company is blind to the results until 26 weeks after the completion of the final group in the trial. The results are expected to be analyzed in November. Patients given placebo will receive the optimal treatment after this analysis.

“The completion of treatment for the patients in group 3 brings us a step closer to our goals of obtaining provisional consent and launching NTCELL as the first disease-modifying treatment for Parkinson’s disease,” Ken Taylor, chief executive officer of LCT, said in a press release.

Due to the fact that choroid plexus cells help produce CSF as well as a range of neurotrophins (nerve growth factors) that have been shown to protect against neuron (nerve) cell death in animal models of disease, NTCELL has the potential to be used in a number of other central nervous system indications, including Alzheimer’s disease, Huntington’s disease, and ALS.

If the trial is successful, LCT plans to apply for provisional consent later this year to treat paying patients in New Zealand and launch NTCELL as the first disease-modifying treatment for Parkinson’s.