"The method does not provide any progress and will never form the basis for therapy." With these words, the famous journal "Nature Medicine" declined by Don Cleveland. In the paper, the teacher described about it medicine and Neuroscience at the University of California in San Diego New Road for the Use of Lateral Amyotrophic Graduated Nerve Disease (ALS) – the disease suffered by world-renowned physicist Stephen Hawking until his death. Cleveland and his team were able to show in experiments with mice that a DNA drug so-called substantially slows down the course of the disease.
Today, 13 years later, the novel therapy developed by Don Cleveland is about to use it in patients. Many clinical trials are already underway with DNA designer drugs. And this is not just in hereditary form of ALS, but also in other neurosurial diseases such as Huntington's disease, Alzheimer's or frontotemporal dementia.
The principle is always the same: DNA's designer, antisense oligonucleotides (SOO) of this name, ensures that the production of a protein causes ill-health to be photocopied. Alzheimer's or frontotemporal dementia, for example, is the "tau" protein, which can be combined into bundles in the nervous cells and thus lead to cell death.
DNA drugs are for muscle wastes on the market
In another nerve disease, the muscle respiratory (SMA), the new DNA medication has already reached patients. In this hereditarian disease, the motorized so-called neurons are not really working, these are those cells that drive the muscular & # 39; r back strand. The reason for this is a defective protein. As a result, muscles through the body are declining due to a lack of stimulus by the motor neurons.
Babies born with the hottest type of SMA can not sit, catch their heads or turn, they also have breathing and swallowing difficulties; they usually do not survive the second anniversary. Last year a first drug for hereditary hereditary disease was approved. The Spinraza drug (Biogen maker), based on the principle developed by Cleveland, slowing down some muscle is massive, and some of the children who have been usually treated almost.
ALS, the disease that Stephen Hawking is suffering from, could soon be treated. Photo: Getty
It will take a few years to see if the success of the new DNA drugs in ALS, Alzheimer's and other neurotic diseases will be so amazing. They always give a reason for hope. For example, in Huntington. A first clinical trial was so promising that Roche's pharmaceutical company in Basel had bought the development and marketing rights for the California Ionis biotechnology company company therapy in April. By the end of 2018 or early 2019, Roche intends to launch a large clinical trial. "We know the drug is safe," said Cleveland, "and I hope it will benefit patients."
Cleveland catchment seems to be paying. A few years ago, it was ridiculed by the idea of temporarily or incorrectly engaging active genes with DNA cuttings and thereby reducing protein production, it tells us at our meeting in the pre-"Diverse Scientist Award" period Private organization Nomis Swiss October. In the biology cell books, it was finally admitted that it does not work. "It seems, however, that the nerve cells have not read the textbooks," it adds a great, and immediately reports the anecdota mentioned earlier with the refusal of its manuscript by "Nature Medicine. "
In her dissertation isolated the «protein tau»
Cleveland is currently having a good laugh, since he and a team at the San Diego Institute for Cancer Research San Diego have developed a way that will allow them to treat many other diseases in the future – and that's what the list says Glioblastoma, a rigid tumor to the brain until today. For this success, 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 who will bring therapies against the destructive harmful diseases in the clinic," said Alzheimer's Christian Haass researcher at the University of Munich in the talks.
Research has always been more than a profession for Don Cleveland. "I've never wanted to become a scientist," he said. "I can not remember anything else." Cleveland grew up with two brothers and sisters in the state of New Mexico, not far from the Mexican border. His father taught physics at a local college, and one of the sisters is the same in chemistry today. Cleveland studied first physics, but then moved to biochemistry during his PhD thesis in Princeton. During this time, he made his first development: It is isolated and describes in 1977 the protein "tau", the protein that forms in Alzheimer's, but also in the box and the ball Driver "Ensephalopathy Traumatic Chemistry Soccer" (CTE) and Nerf cells having been destroyed from the inside.
Later, he succeeded in explaining the complex mechanism of cell division, a basic process of biology.
His career was well launched. And it continued at a similar pace. He was the first to insulate and describe genes for proteins known as keratin (hair), actin (muscle) or tuberculosis (cytoskeleton). Later, he succeeded in explaining the complex mechanism of cell division, a basic process of biology. And then only DNA drug development designer against a variety of neurosurial diseases.
The latest 68-year-old idea is already underway: Cleveland wants to regenerate new nerve cells in the brain. Dementia diseases such as Alzheimer's, Parkinson's or even CTE kill unnecessary nerve cells. As a rule, she has enough support cells, known as astrocytes, which, unlike nerve cells, run easily. His team has now managed to use a DNA drug to make astrocytes turning nervous cells. And that they would have fallen really with mice, says Cleveland. "I've never expected that." Probably, that is also different in the textbooks.
Created: 16.11.2018, 19:16 cloc