Today we were given some hope. I have never had the thought that there is a treatment for some of the symptoms that I have, let alone one of the two syndromes with which I have been diagnosed. We were visiting my neurologist, who is a Movement Disorder Specialist (a neurologist who specializes in degenerative brain diseases that cause movement abnormalities, such as Parkinson's, MSA, PSP, CBD, etc.), and she explained that there is a new treatment in the final stages of approval and will soon be available for Multiple System Atrophy patients. She indicated that I am going to be eligible for its use and I feel very excited.
I will do my best to give an idea of what it is, how it was developed and the process that is being utilized to bring it to fruition. At the conclusion of this brief summary, I will provide a link so that you may read about it further. In the studies utilizing animals, the agent PBT434 was used to target accumulations of iron found in the brains of animals. This iron builds up and contributes to the clumping of alpha synuclein, the substance that misfolds and destroys brain cells. This process of misfolding proteins causes clumps known as Lewy Bodies that bring on oxidative stress in Multiple System Atrophy, leading to movement issues that are disabling and contribute to severe disease progression.
The substance has shown the ability to stop neuron loss and improve movement disorder symptoms. As a result of the studies that have taken place, human trials ensued and are leading toward the potential release of the drug for the treatment of MSA. As you know I have MSA and Corticobasal Syndrome, two forms of Atypical Parkinsonian disorders that are brain diseases leading to severe disability.
If you would like more information, I have the following links that I have referenced in making this post:
https://www.parkinsonsmovement.com/pbt434-iron/
https://www.empr.com/home/news/drugs-in-the-pipeline/investigational-treatment-for-multiple-system-atrophy-gets-orphan-drug-status/
This is new hope that I have seldom experienced the 13 years I have had the MSA and CBS diagnosis. Thanks for visiting and for reading. -- Patient-Online
I have done some more looking, and a friend of mine let me know that the above drug may be a little further away from realization that I thought. The one my physician was referring to may have been a different treatment. I am going to be following up on this blog with more information. In the meantime, the above looks like it holds promise. Another being studied and that is further along in the pipeline is found here:
https://www.biohavenpharma.com/investors/news-events/press-releases/02-19-2019
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