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Parkinson’s disease therapy pipeline offers promising new treatment hope

It was in 1817 that Parkinson’s disease was first described as a neurological disorder.

In a detailed medical essay written by the London-based doctor who was to go on and give his name to the progressive disease, James Parkinson, he talked about the ‘shaking palsy’.

More than 200 years on, and whilst scientists have made huge leaps in understanding the neurodegenerative disorder, there is currently no cure. Treatments hinge on relieving symptoms with the limited drugs currently available, and maintaining quality of life.

Those drugs have largely remained unchanged for decades, however, and usually involve taking one called levodopa alongside other medications.

Game-changing therapies could soon be on the horizon, though, as scientists push the boundaries in the hunt for treatments that slow – or ideally reverse – the progress of Parkinson’s disease, which is caused by a loss of cells that produce the neurotransmitter dopamine.

This causes neurons to fire out of control, leading to Parkinson’s hallmark symptoms of tremors in the limbs and head, impaired balance, muscle stiffness, slowness of movement, and possible mental confusion and speech problems.

But according to business consultant and market research firm, DelveInsight, the Parkinson’s disease clinical trial pipeline space is brimming with novel emerging therapies with over 120 active players globally working to develop 140-plus treatments.

Key Parkinson’s disease companies currently working to improve the treatment landscape include Cerevel Therapeutics, Neuraly, Peptron, Biogen, Roche, Brain Neurotherapy Bio, United Neuroscience Ltd, Luye Pharma Group, Takeda, Xoc Pharmaceuticals, AstraZeneca, MedImmune, and Kissei Pharmaceutical.

In its Parkinson’s Disease Pipeline Insight – 2023 report,  DelveInsight says there are promising pipeline therapies in various stages of development.

These encompass Tavapadon, IkT-148009, NLY01, PT320, BIIB122, Prasinezumab, KDT-3594, AAV2-GDN, AKST4290, anle138b, ITI-214, NTCELL, Buntanetap, ANAVEX2-73, ATH-1017, NE3107, MEDI1341, AZD0328, Liraglutide, UB-312, LY03003, FB-101, AV-101, ABBV-951, NYX-458, DSP-9632P, Valiloxybate, SER-214, UCB7853, TAK-071, XC130-A10H, K0706, Talineuren, Pirepemat, Lu AF28996, WD-1603, CVN424, CST-103, NBTX-001, MSK-DA01, adipose-derived mesenchymal stem cells, ND0612, P2B001, 1ST-103, 1ST-105, and others.

In January this year, Neurocrine Biosciences and Voyager Therapeutics announced the formation of a new strategic collaboration to advance multiple gene therapies for the treatment of neurological diseases. 

The collaboration includes Voyager’s preclinical, intravenously administered GBA1 gene therapy programme for Parkinson’s.

In addition, Neurocrine Biosciences and Voyager have agreed to collaborate on three new gene therapy programmes directed to rare central nervous system (CNS) targets.

In October 2022, Biogen and Denali Therapeutics announced that dosing had commenced in the global Phase 3 Lighthouse study to evaluate the efficacy and safety profile of BIIB122 (DNL151), as compared to placebo in approximately 400 participants with Parkinson’s disease and a confirmed pathogenic mutation in the leucine-rich repeat kinase 2 (LRRK2) gene.

And, in September 2022, Neuron23, an early-stage biotechnology company focused on developing precision medicines for genetically defined neurological and immunological diseases, and QIAGEN, a provider of sample and assay technologies for molecular diagnostics, applied testing and academic and pharmaceutical research, announced a collaboration.

They are looking to develop a companion diagnostic for Neuron23’s LRRK2 inhibitor for Parkinson’s disease.

Other recent advancements cover Neuroderm’s liquid levodopa/carbidopa known as ND0612, which it was announced earlier this year had shown positive phase three trial results, and adipose-derived mesenchymal stem cells.

Stem cell therapy may benefit Parkinson’s disease by replacing and repairing damaged nerve cells within the brain.

Meanwhile Tavapadon, being developed by Cerevel Therapeutics, has shown improvements in movement symptoms.

The cause of Parkinson’s disease is still largely unknown. But it is a progressive disorder caused by nerve cell degeneration in the substantia nigra, a part of the brain that controls movement.

These nerve cells die or become impaired, losing the ability to produce dopamine, an important chemical.

Theories as to the potential causes of the disease involve oxidative damage, environmental toxins, genetic factors, and accelerated aging.

The majority of Parkinson’s patients are given medications to alleviate their symptoms. These work by either stimulating the remaining cells in the substantia nigra to produce more dopamine (levodopa medications) or inhibiting some of the acetylcholine produced (anticholinergic medications), restoring the balance of chemicals in the brain.

Many celebrities have been diagnosed with Parkinson’s and have been vocal about the condition, helping to raise its profile globally.

They include the Scottish comedian Billy Connolly, the actors Ian Holm, Bob Hoskins, Michael J Fox and Alan Alda, former US President George HW Bush, boxer Muhammad Ali, the singers Neil Diamond and Ozzy Osbourne, TV presenter Jeremy Paxman, and Pope John Paul II.

 

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