Parkinson's Disease (PD). Alzheimer's Disease. Creutzfeldt-Jakob Disease (CJD). Transthyretin amyloidosis (TTR). These are diseases that are increasingly blighting our society. Almost everyone will have now heard of the first two as the misery of dementia slowly ebbs further into the public consciousness. CJD is the infamous mad cow disease that had the world panicking in the 1990s. As chance would have it I happened to be doing some work just today during my day job on TTR, a disease that can ravage the heart and liver.
Although these diseases can present in a variety of different ways they all have something in common, something that may play to our advantage. They are all caused by the misfolding of proteins. Yep, as simple as that. The abject horror of losing your mind to Alzheimer's is the result of a protein folding up slightly wrongly and producing a little kink that renders it insoluble in your cells. These misshapen chains of amino acids aggregate together into plaques that become toxic and kill your brain cells; we currently have no way of removing them. In PD we call them Lewy bodies, in CJD they're prions but the subtle differences between them all may be rendered irrelevant by a new drug that specifically targets the kink in the protein that unites them all.
Researchers at NeuroPhage Pharmaceuticals have released data from animal trials that shows their drug is capable of breaking up the plaques and subsequently improving their cognitive function. They have applied for a licence to do human trials and hope to start small scale trials in PD patients next year. The drug is derived from M13 bacteriophages, a very common organism in biomedical research. Bacteriophages are vanishingly small viruses that infect bacteria (a fact that makes them one of our best bets for the next generation of antibiotics), many thousands of them can fit inside a single bacterium. The drug comprises of two parts. The first is a compound from the M13 phage that can recognise and bind to the kink in the deformed proteins. The second is a human antibody that the first part is bound to. The antibody signals to the body's immune system that the plaque needs to be sequestered away and destroyed.
This compound is still a very, very long way away from being a useable drug, probably five years minimum, and there's probably an even chance that it won't come to anything; but it does show promise.
Although these diseases can present in a variety of different ways they all have something in common, something that may play to our advantage. They are all caused by the misfolding of proteins. Yep, as simple as that. The abject horror of losing your mind to Alzheimer's is the result of a protein folding up slightly wrongly and producing a little kink that renders it insoluble in your cells. These misshapen chains of amino acids aggregate together into plaques that become toxic and kill your brain cells; we currently have no way of removing them. In PD we call them Lewy bodies, in CJD they're prions but the subtle differences between them all may be rendered irrelevant by a new drug that specifically targets the kink in the protein that unites them all.
Researchers at NeuroPhage Pharmaceuticals have released data from animal trials that shows their drug is capable of breaking up the plaques and subsequently improving their cognitive function. They have applied for a licence to do human trials and hope to start small scale trials in PD patients next year. The drug is derived from M13 bacteriophages, a very common organism in biomedical research. Bacteriophages are vanishingly small viruses that infect bacteria (a fact that makes them one of our best bets for the next generation of antibiotics), many thousands of them can fit inside a single bacterium. The drug comprises of two parts. The first is a compound from the M13 phage that can recognise and bind to the kink in the deformed proteins. The second is a human antibody that the first part is bound to. The antibody signals to the body's immune system that the plaque needs to be sequestered away and destroyed.
This compound is still a very, very long way away from being a useable drug, probably five years minimum, and there's probably an even chance that it won't come to anything; but it does show promise.
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