DOPAMINE
MOLECULE
BACKGROUND
Just a few of the treatments scientists and physicians hope to use in order to help those afflicted with neurodegenerative diseases.
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CURRENT THERAPIES FOR
NEURODEGENERATIVE DISEASE
There are currently many ongoing experiments that seek to find the ideal cure for one or many of the known neurodegenerative diseases. Drug, stem cell, and gene therapies are three of the most widely tested methods of treatment, and have managed to yield quite promising results so far.
EXAMPLES OF DRUG THERAPIES FOR
NEURODEGENERATIVE DISEASE
Until recently, medication available for people suffering from neurodegenerative diseases were only symptomatic treatments, which could relieve the symptoms of a neurodegenerative disease, but could not reverse the damage done to the patient's nervous system. Recently however, trials are being conducted with the ultimate goal of discovering a way to prevent or reverse the effects of nervous degeneration. [S2.4]
In 2015, a group of researchers at the University of Liverpool published their findings regarding a potential drug treatment for neurodegenerative disease. In fact, their research did not involve the creation of a completely new compound, but simply managed to repurpose the anti-epileptic medication known as ethosuximide to prolong the life of mammalian nervous cells. [S2.5]
Ethosuximide
The French biopharmaceutical company, PharNext, has been working on developing a drug that will improve the nervous function of patients affected by Charcot-Marie-Tooth (CMT) disease, a congenital disease that causes nervous degeneration. The researchers created a compound that was successfully able improve the state of tested mice models, and proceeded to test the product, called the PLEODRUG, on 80 patients with CMT type 1A. The results were favorable, revealing better Charcot-Marie-Tooth Neuropathy scores, and significantly improved function in the limbs of tested patients. [S2.6]
PLEODRUG
Chromosome of CMT Victim
New studies have shown that the nanoparticles known as C60 (Carbon-60) fullerenes may be the key to developing a drug therapy for Alzheimer’s, Parkinson’s, and more. In Alzheimer's patients, for instance, the administration of a treatment containing the C60 fullerenes was able to successfully inhibit the aggregation of amyloid plaques, which are known to cause the debilitating symptoms of Alzheimer’s Disease. Similarly positive results were observed when the nanoparticle was given to patients with Parkinson’s, ALS, and MS. [S2.7]
Carbon-60 Fullerenes
EXAMPLES OF STEM CELL THERAPIES FOR
NEURODEGENERATIVE DISEASE
The well-studied concept of using stem cells as a means of treating neurodegenerative disease shows promise, both in preclinical and clinical trials. The versatile cells could potentially be used to cure nervous deterioration, from common dementia to Parkinson's, to much rarer conditions, such as Alper's Disease. [S2.8]
One group of researchers found that adult olfactory cells could possibly be used to treat Parkinson’s. The olfactory stem cells could be differentiated into neural cells that mimic the dopaminergic neurons that Parkinson’s patients often lack. As dopaminergic neurons are the brain’s primary source of dopamine, the introduction of the cells mentioned above could replenish the brain’s dopamine supply without the side effects that often accompany Parkinson’s current treatment, L-Dopa. [S2.9, S3.1]
Adult Olfactory Stem Cells
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In one instance, a group of researchers used mesenchymal stem cells, taken from the patient’s own bone marrow, to treat ALS. The cells were injected into the spinal cord, and seemed to effectively slow the progression of nervous deterioration and loss of motor functions. The study was deemed safe for potential treatment of ALS in the future, as no major side effects were observed. [S3.2]
MSCs for ALS
MSCs from Bone Marrow
Research done on monkey models of Huntington’s Disease revealed the possibility of using dental pulp stem cells. These stem cells were able to differentiate into various cell types, and could be taken with ease from a patient at almost any age. The usage of dental pulp cells has a great advantage, because, as the cells can be taken easily from the patient, it will eradicate the need for the patient to take immunosuppressants. [S3.3]
Dental Pulp Stem Cells
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A study was conducted on patients with multiple sclerosis, who had been unresponsive to the standard interferon beta treatment. Non-myeloablative stem cells from the red bone marrow were transplanted into the patients, and the patients were then studied for around 24-48 months depending on the rate of recovery. The results were encouraging, as the majority of patients showed some signs of improvement, despite the appearance of some negative symptoms that accompanied the treatment. By the end of the study, each of the treated patients showed no sign of further nervous deterioration, and additionally, as determined by a questionnaire, experienced a better quality of life post-treatment. [S3.4]
Haemopoietic Stem Cells
One group’s research found that the treatment of mouse models of Alzheimer’s with neural stem cells was able to reverse the memory-loss symptoms that are famously known to accompany Alzheimer’s Disease. The study revealed that the administration of neural stem cells into the hippocampus was able to improve cognitive function and, in doing so, was able to restore the memory capacity in the brains of the tested mice. [S3.5]
NCSs to Treat Alzheimer's
Neural Stem Cells
EXAMPLES OF GENE THERAPIES FOR
NEURODEGENERATIVE DISEASE
Approximately 5% of neurodegenerative cases can be attributed to genetic mutations, and many belonging to that 5% suffer from early-onset neurodegenerative disease, as seen in early-onset familial Alzheimer’s Disease or early-onset Parkinson’s Disease. Ranging from the results of a Mendelian gene-type mutation that can be directly linked to neurodegeneration, to seemingly unrelated genetic factors that may simply increase an individual’s susceptibility to neuron deterioration, there is a wide spectrum of genetic factors that may encourage the degeneration of the nervous system. For these patients, however, there may exist the possibility of new genetic therapies, which could alter and prevent the symptoms of their neurodegenerative disease from as early as childhood. [S1.2]
Studies conducted by BrainCAV uses an unlikely viral vector to transport a “good” copy of a damaged gene into animal models of Sly Disease. The idea behind the use of canine adenovirus-2 as the mode of transportation for the gene, is that the virus, which typically affects the canine respiratory system, is not attacked by the cells of the human immune system. Additionally, the canine adenovirus-2 preferentially affects only neurons, and leaves the remainder of the brain’s cells unharmed. In mice models of Sly disease, researchers noted the inhibition of nervous degeneration, or the partial restoration of cognitive function. [S3.6]
CAV-2 Vectors
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Various studies were conducted with the purpose of figuring out a way to use lentiviral vectors to provide genetic therapy for patients with nervous degeneration. Two studies found that the administration of a protein that encourages the growth of new blood vessels in the body along with its many other functions, could also prevent or slow the rate of ALS progression when injected into the brain of mice and rat models of ALS. When injected into the muscles of the rodent models, the protein, known as the vascular endothelial growth factor had an even more drastic effect on the subjects, by inhibiting cell death and extending the lifespan of the rodents. [S3.8]
VEGF to Cure ALS
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