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"The method does not provide progress and will never form the basis for a therapy." With these words, the renowned journal Nature Medicine rejected in 2005 a manuscript of Don Cleveland. On paper, the teacher described medicine and Neuroscience at the University of California at San Diego A New Way to Treat Degenerative Nerve Disease Amyotrophic Lateral Sclerosis (ALS) – the disease suffered by world-famous physicist Stephen Hawking until his death. Cleveland and his team were able to show in experiments with mice that a DNA drug, called a designer, significantly decreases the course of the disease.

Today, 13 years later, the new therapy developed by Don Cleveland is at the threshold of use in patients. Several clinical trials are already underway with designed DNA drugs. And this not only in an inherited form of ALS, but also in other neurodegenerative diseases, such as genetic disease, Huntington's disease, Alzheimer's disease or frontotemporal dementia.

The principle is always the same: designed DNA drugs, the so-called antisense oligonucleotides (ASO), ensure that the production of a disease-causing protein is strangled. In Alzheimer's dementia or frontotemporal, for example, this is the "tau" protein, which can aggregate into bundles in nerve cells and thus lead to cell death.

DNA drug for the way of muscle loss is on the market

In another nerve disease, spinal muscular atrophy (AME), the new drug for DNA has already reached patients. In this inherited disease, so-called motor neurons do not function properly, it is these nerve cells that drive the muscles of the spinal cord. The reason for this is a faulty protein. As a result, muscles throughout the body degenerate due to lack of stimulation by motor neurons.

Babies born with the heavier form of SMA can not sit, hold their heads or spin, they also struggle to breathe and swallow; usually they do not survive the second birthday. Last year, a first drug was approved for the deadly hereditary disease. The drug Spinraza (manufacturer Biogen), which is based on the principle developed by Cleveland, decelerates a bit of muscle massively, some of the treated children have developed almost normally.

ALS, the disease that Stephen Hawking suffered, may soon become treatable. Photo: Getty

It will take a few years to see if the success of the new DNA drugs in ALS, Alzheimer's and other neurodegenerative diseases will be so resounding. They always give cause for hope. For example, in Huntington. A first clinical trial was so promising that pharmaceutical company Roche, in Basel, bought development and marketing rights for the therapy of biotechnology company Ionis, California, in April. By the end of 2018 or early 2019, Roche plans to launch a major clinical trial. "We know the drug is safe," says Cleveland, "and I hope it will benefit patients."

Cleveland's tenacity seems to be paying off. A few years ago, he was mocked at the idea of ​​paralyzing hyperactive or incorrectly active genes with stretches of DNA and thereby reducing protein production, he tells us at our meeting in the race for the "Distinguished Scientist Award" by the Swiss private Nomis Foundation. October. In the cell biology books was finally confessed that does not work. "It seems, however, that nerve cells did not read the textbooks," he adds with a smile, and immediately recounts the anecdote mentioned earlier with the rejection of his manuscript by "Nature Medicine."

In his dissertation he isolated the protein "tau"

Cleveland is laughing at the moment as he and his team at the Louis Institute of Cancer Research in San Diego have developed a method that will allow them to treat many other diseases in the future – and that's what the list says. Glioblastoma, an incurable brain tumor to date. For this breakthrough, Cleveland received the $ 3 million Breakthrough Award last year. And now the price of the Nomis Foundation. At the awards ceremony, Cleveland was praised accordingly. "You will be the first scientist to bring therapies against the destructive neurodegenerative diseases in the clinic," said Alzheimer's researcher Christian Haass of the University of Munich in praise.

Research has always been more than a profession for Don Cleveland. "I've always wanted to become a scientist," he says. "I can not remember anything else." Cleveland grew up with two brothers in the state of New Mexico, not far from the Mexican border. Her father taught physics at a local college and one of his sisters does the same in chemistry today. Cleveland first studied physics, but later moved to biochemistry during his doctoral thesis at Princeton. During this time he made his first discovery: He isolated and described in 1977 the "tau" protein, the protein that forms in Alzheimer's disease, but also in boxer and Footballer's disease "Chronic Traumatic Encephalopathy" (CTE) and ball Nerve cells destroyed inside.

Later he was able to elucidate the complicated mechanism of cell division, a fundamental process of biology.

Your career was so well launched. And it continued at a similar pace. He was also the first to isolate and describe genes for proteins known as keratin (hair), actin (muscle), or tubulin (cytoskeleton). Later he was able to elucidate the complicated mechanism of cell division, a fundamental process of biology. And then only the development of DNA drugs designed against a variety of neurodegenerative diseases.

The latest idea of ​​the 68-year-old man is already underway: Cleveland wants to regenerate new nerve cells in the brain. Dementia diseases like Alzheimer's, Parkinson's or even CTE kill countless nerve cells. As a rule, it still has enough support cells, called astrocytes, which, in contrast to nerve cells, regress easily. His team has now been able to use a DNA drug to turn the astrocytes into nerve cells. And they would have intertwined properly with mice, says Cleveland. "I never expected this." Presumably, this is also different in textbooks.

(Editors Tamedia)

Created: 16/11/2018, 19:16 clock


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