Two European Parkinson’s studies kick off 2018 with promising discoveries; proof to me, for example, that not all biotech worth noting come from the USA.

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The News:

A combination therapy made up of two neurotrophic factors has been shown in a report to reduce the degenerative effects of Parkinson’s disease, report researchers from the UPV/EHU-University of the Basque Country. The two factors; Vascular Endothelial Growth Factor (VEGF) and Glial Cell-derived Neurotrophic Factor (GDNF), are proteins that play an essential role in nerve cell function by inducing cell growth, plasticity and survival.

Parkinson’s disease is the second most common neurodegenerative disorder, affecting more than 1 million people in the US alone. The disease is characterized by the progressive loss of dopaminergic neurons, which produce dopamine–a neurotransmitter that is key in the control of involuntary movements. This causes motor symptoms including tremor, slow movement, limb rigidity, and problems with balance, which can have major implications on their quality of life.

Using an experimental model, the researchers reproduced the various stages of Parkinson’s disease and investigated the therapeutic effects of applying the two neurotrophic factors.

As reported in Molecular Neurobiology, VEGF and GDNF were delivered in microspheres or nanospheres made up of a biodegradable polymer called Poly Lactic-co-glycolic Acid, which enables the proteins to be released in a gradual and continuous manner. This combined administration meant the researchers could assess any synergistic effect the two factors may induce.

Dr. Catalina Requejo and colleagues reported encouraging findings for both early and late stages of Parkinson’s disease. Delivery of the VEGF/GDNF combination significantly reduced the degeneration of dopaminergic neurons in the black substance of the brain, as well as triggering new cell formation and cellular differentiation.

To confirm this synergistic effect, the team administered a molecule known to inhibit the cell receptors to which VEGF and GDNF bind.

“The [resulting] consequences for the dopaminergic system were even worse, which supports the beneficial synergistic effects exerted by the VEFG and the GDNF in Parkinson’s,” Dr. Requejo said.

The study also showed that the most effective results were observed when nanospheres were used as the delivery mode during early-stage disease, providing further evidence of the importance of early diagnosis and the therapeutic potential nanotechnology has to offer.

Steve’s Take:

Breakthroughs in many disease categories seem to be coming fast and furiously these days, and one of the leading fronts is Parkinson’s. What’s particularly striking to this admittedly geographically biased observer (increasingly unwarranted as the data pour in) is where some of the most advanced scientific thinking, testing and outright brilliant discovery is taking place: Europe.

Steve's Take: #Europe is where some of the most advanced scientific thinking, testing and outright brilliant #biotech discoveries are taking place. Click To Tweet

Study No. 1

Two very recent reports that kicked off the New Year begin with the Basque Country discovery (summarized above) that two proteins in the brain could protect against and repair the damage caused by Parkinson’s. I’d say that qualifies for one heck of an eye-watering discovery.

In a nutshell, researchers found two proteins that could be used to treat Parkinson’s disease patients by exerting neuroprotective and regenerative effects. This finding provides hope that an effective Parkinson’s treatment could be developed based on this particular mechanism.

Study No. 2

Then I stumbled upon another report of a pioneering study finding for the first time that patients with Parkinson’s have more errors in the mitochondrial DNA within the brainstem, leading to increased cell death in that area.

Experts at Newcastle and Sussex universities revealed that surviving brain cells in the brainstem have more copies of mitochondrial DNA and this has not been identified before.

The study’s deeper understanding into Parkinson’s disease suggests a new target for therapies.

Researchers say their findings, published in Annals of Neurology, are “surprising” as the results differ from what has been seen in studies of brain regions that harbor other brain cell-types.

Dr. Joanna Elson, a mitochondrial geneticist at Newcastle University, said: “Our study is a major step forwards in gaining an enhanced insight into the serious condition. Only by understanding the complexities of what happens in specific cell-types found in specific areas of the brain during this disease can targeted treatments for Parkinson’s disease be produced.”

Research shows that in Parkinson’s disease a brainstem region called the pedunculopontine nucleus (PPN) develops changes in DNA found in mitochondria–the batteries of the cell–as they produce and store energy that cells can use.

This study looked at cholinergic neurons that are responsible for producing the brain chemical acetylcholine, which is released by cholinergic nerve cells to send signals from one neuron to another.

Death of these cells in the PPN is believed to be the cause of some of the symptoms of Parkinson’s disease, such as problems with attention, walking and posture.

Identifying changes in the mitochondrial DNA in PPN cholinergic neurons has the potential to allow the development of more effective treatments targeted to specific cell-types. The PPN is an understudied part of the brain and researchers used post-mortem tissue from the Newcastle Brain Tissue Resource, based at Newcastle University, to isolate single neurons for in-depth analysis.

Dr. Ilse Pienaar, a neuroscientist at Sussex University, said: “At present, treatments are aimed at the whole brain of patients with Parkinson’s disease. We believe that not only would cell-specific targeted treatments be more effective, but they would also be associated with fewer side-effects.”

The PPN was of interest because, in previous studies, patients with Parkinson’s disease displayed lower levels of mitochondrial DNA (mtDNA) in remaining dopaminergic neurons.

This study showed that mtDNA levels are higher in the surviving cholinergic neurons of the brainstem, but with both cell-types that undergo profound degeneration during Parkinson’s disease.

The finding identifies how vulnerable cell groups react and respond differently to the accumulation of mitochondrial DNA damage seen in the disease, highlighting the need for cell-specific treatments.

Bottom Line:

Developing new therapies for diseases of the central nervous system is particularly difficult for biotech companies as regulatory bodies are exacting (to say the least) on drugs competing to enter the Parkinson’s market.

However, Newron Pharmaceuticals SpA, based in Milan, finally accomplished it last year with Xadago. Meanwhile, Swiss biotech Prexton Therapeutics SA started a Phase 2 trial to test its candidate for the motor symptoms of the condition. Labiotech.eu says one to watch out for is Edinburgh-based Synpromics Ltd. which, with help from researchers at University College London, is developing a gene therapy targeting specific types of neurons affected by Parkinson’s.

The Spanish and British researchers referenced herein have opened the door on new approaches to the treatment of Parkinson’s that, along with Alzheimer’s and ALS, is proving a massive challenge for biotech and pharma alike.

As we move into this new year with false nuclear-attack warnings in Hawaii right off the bat, at least on the scientific/medical front, the commitment to, and pace of, new discovery and much-needed disease treatment remains steadfast, not just here, but in many other parts of this scatter-brained world. Let’s hope that these reported findings ultimately help patients who are suffering and allay strained healthcare systems everywhere.

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