Address for children with rare diseases. Report from the Center for Drug Discovery and Research

Imagine a future in which drugs are not so expensive that governments stop choosing which drugs they can subsidize, and new, more effective drugs hit the market more often and faster.

A future in which children suffering from orphan diseases (“orphan diseases”) will have a real chance of salvation without having to look for funds that are too heavy for an ordinary family. The first steps of this future are now taking place in several research centers, one of which is the Blavatnik Center for Drug Discovery and Research at Tel Aviv University under the academic leadership of Professor Ehud Gazit.

The modern pharmaceutical industry has created only about 1,200 drugs. About 400 more drugs were taken off the market due to problems that were revealed after they entered the market. And that’s all. Despite trillions of dollars and thousands of man-years.

If you want to develop a new drug, any representative of the so-called Big Pharma (large pharmaceutical companies) will tell you that just for the initial development of a new drug, even before the stage of clinical trials, it takes several billion dollars and ten years of work.

For a lone researcher or small laboratory, the chance of achieving such a result is negligible. Simply due to lack of time, equipment and funds.

In recent years, the scientific world has begun to come to the conclusion that something needs to be changed in the current situation. At the same time, as often happens, this conclusion was made almost simultaneously in different parts of the world, including the USA, EU countries and Israel.

According to the director of the Blavatnik Center, Dr. Lyudmila Buzhanskaya, the main problem of the modern scheme for the development of new drugs is the lack of orderly information and poorly established contacts between the academy and industry and between representatives of various disciplines directly at the academy.

The Blavatnik Center is intended to become the very link and information and analytical center of the pharmaceutical industry of the future.

The staff of the center is only 12 people, representing three main groups – chemists, biologists and computer specialists. A small in area, but carefully thought-out territory is also inhabited by a large number of the most modern robots that allow manipulating cells and solutions of potentially pharmacologically active substances with a speed, accuracy and accuracy inaccessible to humans.

“They don’t want to believe it, but what concerns need billions of dollars and years, we are ready to do for one hundred thousand dollars and a few weeks,” says Dr. Buzhanskaya.

“An incredible amount of effort, money and time is spent on repeating other people’s mistakes. Each scientist conducts many experiments, but the results of the overwhelming majority of them are not recorded anywhere. As a result, each next scientist or pharmaceutical company starts everything from scratch, instead of gaining access to the database and save significant resources. One of our goals is to create such a database, “she explains.

“Another example – to defend a doctoral dissertation, a chemist must create a certain amount of new, previously nonexistent, substances. Often he works in tandem with a biologist. However, he can test the biological activity of these substances on a very limited number of individual pure cell cultures. The vast majority of these substances in the end after the defense of the thesis are simply thrown away as unnecessary.Our robots and computer models can test on thousands of different tissues, determining whether the active substance can work with certain proteins and, if so, in what form. center in tandem with similar centers in other countries is total sifting – testing of all known chemical compounds for all possible types of pharmacological activity.This will make it possible to be sure that all the possibilities for detecting potential drug candidates are used, and the automatic screening system, together with the centralization and systematization of the data obtained, will reduce by an order of magnitude the time and costs of creating new drugs, “says Lyudmila Buzhanskaya.

“We are not engaged in clinical research and drug launching. Our task is to conduct preliminary analysis and preparatory work, which reduces the time and increases the chances of developing new drugs,” our interlocutor explains. trying to minimize risks. Our belonging to the world of science, to the academy, allows us to use a creative approach, not to limit ourselves for fear of failure. “

“We are creating a” library “of formulas, substances, experiments. The same unified standardized” libraries “are now beginning to be created in American and European centers. Due to the very large size of information arrays, we are forced to resort to the services of specialists working with Big Data, “says Buzhanskaya.

The head of the high-throughput screening and biological analysis department of the center, Dr. Eduard Pichinyuk, defines the center’s task as bringing the results of academic research to an industrial level.

“Currently, the laboratory is carrying out 26 different projects developed by scientists at Tel Aviv University. Among them are the development of drugs to fight cancer, neurodegenerative diseases such as Alzheimer’s, Parkinson’s, Huntington’s syndromes. Several projects are related to verification the influence of natural substances on psychiatric diseases. We carry out the isolation and screening of active substances from the recipes of Chinese medicine. Two more projects are related to testing new antifungal and anti-yeast substances, “he says.

However, the development of new drugs is still the future, albeit much closer than before. For some families, the center can help in the present.

“Our laboratory can take tissue from a sick person and test its response to all 1,600 existing drugs. Sometimes, in cases of rare diseases and diseases for which no treatment has been developed, this can save lives. rare diagnoses for which there is no medicine now, “says Dr. Buzhanskaya.

No pharmaceutical company will invest in the development of a cure for a disease that affects dozens of people around the world.

According to Dr. Pichinyuk, the center has established links with charities that support families with children suffering from rare diseases such as Duchenne muscular dystrophy or ataxia. These organizations refer such families to the center, where they try to help them, checking if one of the existing drugs is not suitable for this particular patient.

One of the initiators of this project is another member of the Blavatnik Center team, Professor Miguel Vile, who has devoted his career to the treatment of orphan diseases, one of which suffers from his son.

Currently, information about the laboratory’s capabilities is passed from hand to hand, but the center is working to obtain the official status of a national center for the development of drugs for rare diseases.

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