"The method does not provide any progress and is never the basis of a therapy." In 2005, it was rejected by the manuscript of the famous Nature "Nature Medicine" by Don Cleveland. In the paper, professor medicine and New Pathway to Cure Neuroscience Degenerative Nervous Disease in the University of California at San Diego Amyotrophic Lateral Sclerosis (ALS) – a disease the world famous physicist Stephen Hawking lived to his death. Cleveland and his team showed that in experiments with mice, what is called designer DNA drug slows the course of the disease dramatically.
Today, 13 years later, the new therapy developed by Don Cleveland is on the threshold of use in patients. Various clinical trials with designer DNA drugs have already begun. And this is not only in an inherited form of ALS, but also in other neurodegenerative diseases such as Huntington's disease, Alzheimer's or genetic disease such as frontotemporal dementia.
The principle is always the same: designer DNA drugs, called antisense oligonucleotides (ASO), allow the production of a disease-causing protein to be reduced. In Alzheimer's or frontotemporal dementia, for example, the protein that can collect into bundles of nerve cells and thus cause cell death is "tau".
The DNA drug in the form of muscle wastage on the market
In another nervous disease, spinal muscular atrophy (SMA) has already reached patients with new DNA medicine. In this hereditary disease, so-called motor neurons do not function properly, they are nerve cells that move muscles from the spinal cord. This is due to a faulty protein. As a result, the muscles in the body are degenerate due to lack of stimulation of motor neurons.
Babies born with the most severe form of the SMA cannot sit, hold or turn, they also try to breathe and swallow; they usually do not survive the second birthday. Last year, the first drug was approved for fatal hereditary disease. Based on the principle developed by Cleveland, Spinraza (producer Biogen) slows down the massive mass of some muscles, and some of the children treated develop almost normally.
ALS, Stephen Hawking's illness could soon be treated. Photo: Getty
In ALS, Alzheimer's and other neurodegenerative diseases, it will take several years to see how much the success of new DNA drugs will be echoed. They always give a reason for hope. For example, in Huntington. The first clinical trial was so promising that the pharmaceutical company Roche in Basel purchased the development and marketing rights for treatment in April from the California biotech company Ionis. At the end of 2018 or early in 2019, Roche plans to launch a major clinical trial. Ar We know the drugs are safe, ”Cleveland says, or I hope it benefits patients.“
Looks like Cleveland's paying off. A few years ago, he was ridiculed for the idea of paralyzing overactive or false-active genes with DNA particles and thus reducing protein production, telling us about our participation in the "Distinguished Scientist Award" by the Swiss Nomis Foundation. October. In cell biology, it was admitted that textbooks did not work. Or It seems that the nerve cells have not read the textbooks, in he adds, with the rejection of the article “Natural Medicine,, he immediately explains the anecdote that was mentioned earlier.
Isolated "tau" protein in his thesis
Cleveland makes a good laugh as the team at the Cancer Research Institute in Cleveland and San Diego develops a method that will allow them to cure many other diseases in the future. Glioblastoma is an incurable brain tumor. For this breakthrough, Cleveland received a $ 3 million breakthrough last year. And now the price of the Nomis Foundation. Cleveland was praised accordingly at the award ceremony. A You'll be the first scientist to bring therapies against disruptive neurodegenerative diseases in the clinic, karşı said Christian Haass, researcher hero of Alzheimer's University of Munich.
Research has always been more than the profession of Don Cleveland. Dü I always wanted to be a scientist, sınır he says. Iyor I can't remember anything else. Iyor Cleveland grew up with two brothers in the state of New Mexico, not far from the Mexican border. His father taught physics at a local college, and one of his sisters is doing the same thing in chemistry nowadays. He studied physics before Cleveland but moved on to biochemistry during his doctoral thesis at Princeton. During this time he made his first breakthrough: In 1977, the protein "tau", which is the protein found in Alzheimer's, as well as the boxer and the Footballer's Disease "Chronic Traumatic Encephalopathy" (CTE) can destroy the nerve cells inside.
He then succeeded in clarifying the complex mechanism of cell division, the basic biology process.
His career was very well started. And continued at a similar speed. It was also the first to isolate and identify genes for proteins known as keratin (hair), actin (muscle) or tubulin (cytoskeleton). He then succeeded in clarifying the complex mechanism of cell division, the basic biology process. And then only the development of designer DNA drugs against various neurodegenerative diseases.
The 68-year-old latest idea is already in progress: Cleveland wants to revive new nerve cells in the brain. Dementia diseases such as Alzheimer's, Parkinson's and even CTE kill countless nerve cells. As a rule, it still has sufficient support cells called astrocytes, which, unlike nerve cells, grow easily. His team was now able to use a DNA drug to transform astrocytes into nerve cells. Cleveland says the mice were properly mice. ”I didn't expect that at all.” It's probably different in these textbooks.
Created: 16.11.2018, 19:16 hours