NTCELL

Overview

An alginate coated capsule containing clusters of neonatal porcine choroid plexus cells targeting Parkinson’s disease. 

About Parkinson’s disease 

Parkinson’s disease is a progressive neurological condition characterised by a loss of brain cells that produce dopamine (a neurotransmitter that conveys messages between brain cells to ensure effective movement and planning of movement) and many other types of neurons. People with Parkinson’s disease experience reduced and slow movement (hypokinesia and bradykinesia), rigidity and tremors.

Parkinson’s disease is the second most common neurodegenerative disorder after Alzheimer’s disease, affecting approximately 7 million people worldwide. The average age of onset is 60 years, and the incidence increases with age. Men are one and a half times more likely to have Parkinson's disease than women.

Current treatments for Parkinson’s disease are symptomatic and do not reverse or slow the degeneration of neurons in the brain. Most existing pharmaceutical treatment options focus on restoring the balance of dopamine and other neurotransmitters. The effectiveness of dopamine replacement therapy declines as the disease progresses. When dopamine treatments are no longer useful, some patients are treated with Deep Brain Stimulation (DBS), in which a medical device is surgically implanted in the brain in order to send electrical impulses to regions of the brain involved in the control of movement. While DBS leads to short-term symptomatic improvement, it does not impact disease progression and is not curative or neuroprotective.

NTCELL® is an alginate coated capsule containing clusters of neonatal porcine choroid plexus cells that are sourced from a unique herd of designated pathogen-free pigs bred from stock originally discovered in the remote sub-Antarctic Auckland Islands. Choroid plexus cells are naturally occurring “support” cells for the brain and secrete cerebrospinal fluid (CSF), which contains a range of factors that support nerve cell functions and protective enzymes that are crucial for nerve growth and healthy functioning. In NTCELL, the porcine choroid plexus cells are coated with LCT’s propriety technology IMMUPEL™ to protect them from attack by the immune system. Therefore, no immunosuppressive regimen is required for treatment.

Following implantation into a damaged site within the brain, NTCELL functions as a neurochemical factory producing CSF and secreting multiple nerve growth factors that promote new central nervous system (CNS) growth and repair disease-induced nerve degeneration while potentially removing waste products such as amyloids and proteins.

Scientific Advisory Board Members

Roger Barker

Roger Barker

Professor (Chairman)
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Patrik Brundin

Patrik Brundin

Dr.
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Thomas Foltynie

Thomas Foltynie

Professor
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Carolyn Sue

Carolyn Sue

Professor
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Barry Snow

Barry Snow

Dr
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Roger Barker

Roger Barker

Professor (Chairman)
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Patrik Brundin

Patrik Brundin

Dr.
Read Bio +
Thomas Foltynie

Thomas Foltynie

Professor
Read Bio +
Carolyn Sue

Carolyn Sue

Professor
Read Bio +
Barry Snow

Barry Snow

Dr
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Roger Barker

Roger Barker

Professor (Chairman)

Professor of Clinical Neuroscience and Honorary Consultant in Neurology at the University of Cambridge and at Addenbrooke's Hospital.

Professor Barker combines basic research looking at novel therapies to treat chronic neurodegenerative disorders of the brain with clinically-based work aimed at better defining such disorders. He is the co-ordinator of the TRANSEURO project looking at fetal cell grafting in patients with early Parkinson’s disease and is part of the GFORCE PD initiative as well as director of the ISSCR.

Patrik Brundin

Patrik Brundin

Dr.

Professor and Director of the Center for Neurodegenerative Science, VARI Associate Director of Research and Jay Van Andel Endowed Chair in Parkinson’s Research, Grand Rapids, Michigan.

Dr. Brundin has 35 years’ experience studying neurodegenerative diseases, Parkinson’s disease pathogenesis and therapeutic neural transplantation into people with Parkinson’s. His current research focuses on pathogenic mechanisms of Parkinson’s disease as well as the development of new therapies that slow or stop disease progression or that repair damaged brain circuits.

Thomas Foltynie

Thomas Foltynie

Professor

Professor of Neurology in the Department of Clinical and Movement Neurosciences, UCL Institute of Neurology and Consultant Neurologist at the National Hospital for Neurology and Neurosurgery, Queen Square, London.

 Professor Foltynie is responsible for movement disorder patients, particularly Parkinson’s disease patients undergoing advanced treatments such as Deep Brain Stimulation (DBS), Apomorphine and Duodopa.

Carolyn Sue

Carolyn Sue

Professor

Director of Neurogenetics at Royal North Shore Hospital and the Director of the Kolling Institute, University of Sydney. 

Professor Sue is an international expert in mitochondrial disease and movement disorders. Her research focuses on understanding the role of mitochondrial function in neurodegeneration, especially the disease processes involved in mitochondrial disorders, Parkinson’s disease and other movement disorders.

Barry Snow

Barry Snow

Dr

Director of Adult Medicine and Consultant Neurologist at Auckland District Health Board.

In addition to his general Neurology work, Dr Snow runs a Movement Disorder Clinic and research programme. He is the Principal Investigator in the clinical studies of NTCELL in Parkinson's disease.

Development to Date

After securing regulatory approval based on animal trials, LCT moved from an encouraging initial clinical trial in four people with end-stage Parkinson’s disease to a larger Phase IIb trail in people with mid-stage Parkinson’s disease that lasted two years. The aim was to secure provisional consent from regulatory authorities to treat paying patients in New Zealand.

The 18-patient, Phase IIb trial of NTCELL encapsulated porcine choroid brain cells studied three groups of six patients. The active cohorts received 40 microcapsules, 80 microcapsules and 120 microcapsules implanted on each side of the brain. Two patients from each group had sham surgery.

The key measure of efficacy was motor function in the off state, that is, when recipients were not taking anti-Parkinsonian medications. Patients were scored using the Unified Parkinson’s Disease Rating Scale standard (UPDRS).

At the initial study endpoint of 26 weeks post-implant there was not a statistically significant difference between the groups who received NTCELL and the placebo group. At this point the study was unblinded (patients then knew if they were treated with placebo) and it was agreed to continue to monitor patients out to two years post-implant as per an extended trial protocol.

In May 2019, at 24 months post-implant there was a clinically relevant effect observed (<-6.45 points from baseline) in both the 80- and 40-capsule group. The effect of 80 capsules was greater than that of 40 capsules.

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Future Development

A Phase III clinical study with a larger number of patients will be required to provide compelling efficacy data required by regulatory agencies for marketing approval.

LCT is examining the feasibility of carrying out such a further clinical trial with NTCELL by consulting with an expert neurology advisory board.

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