Antisense therapy: the first swallow
Antisense therapy drug has been released to the American market
Meanwhile, as The New York Times notes, the drug Kynamro, addressed to several hundred American patients with SGLP, may become the first commercially successful case of the use of antisense therapy in pharmacology.
Antisense therapy is based on stopping the synthesis of a key protein responsible for the development of the disease by suppressing the expression of the corresponding gene. This is achieved by introducing specially designed short nucleotide sequences (antisense RNAs, ac-RNAs), complementary to the target protein matrix RNA.
SGLP is characterized by an extremely early development of atherosclerosis against the background of an unusually high (6-8 times higher than normal) level of "bad" cholesterol in the blood. Kynamro (mipomersen) manufactured by Isis Pharmaceuticals blocks the synthesis of apolipoprotein B (apoB-100), a protein that plays a key role in the transfer of lipids and cholesterol. It is an oligonucleotide of 20 nucleic bases that are anti-semantic with respect to the matrix RNA encoding apoB-100.
Clinical trials of the drug on 51 patients with SGLP have shown that when injected once a week, on average, the level of "bad" cholesterol in the blood drops by 25 percent over the first 26 weeks of use. At the same time, Kynamro has serious side effects, primarily related to its toxicity to the liver. On the market, Kynamro will have to compete with another drug for SGLP, Juxtapid, developed by Aegerion Pharmaceuticals and approved by the FDA in December 2012. The cost of the Kynamro course is expected to be from 235 to 295 thousand dollars per year.
The largest pharmaceutical companies in the world have been working on the introduction of "antisense technologies" into practice since the late 1980s, but the results of this work are only now appearing. Specialists have to solve problems related to the effectiveness of drug delivery to cells and their potential toxicity to the body as a whole. Over the years, there have been several precedents for the creation of drugs based on such technology, but they either did not enter the market, or were recalled for various reasons.
Isis Pharmaceuticals has been engaged in antisense technologies since its foundation in 1989, and Kynamro is its first project brought to the market, fully implemented on the basis of these technologies. "I have always said that it will take at least 20 years and two billion dollars for the technology to work," Stanley T. Crooke, founder and head of Isis Pharmaceuticals, said in connection with the release of Kynamro to the market. "I think this is a significant day for our company and for antisense therapy in general."
According to the forecasts of experts of the analytical company Cowen & Co, 2013 may become the year of "antisense technologies" due to the large number of promising experimental drugs at the stage of clinical trials. We are talking, for example, about a drug for the treatment of Duchenne muscular dystrophy – the brainchild of Sarepta Therapeutics and Prosensa, a drug for prostate cancer from TEVA, a drug for triglyceridemia and thromboembolism from the same Isis Pharmaceuticals and others.
Portal "Eternal youth" http://vechnayamolodost.ru31.01.2013