Biotechnologies and medicine: waiting for miracles and real benefits

Biotechnologies: real benefits or "soap bubbles"?Vladimir Sychev, STRF.ru
The XXI century is often called the century of biotechnology.

They are expected to improve people's quality of life. First of all, we are talking about medical applications of biotech. But doubts often arise — how great is the return on all those developments that were declared breakthrough? This question is asked by people who are not aware of the complexity of the tasks solved by scientists, and therefore unwittingly under the influence of inflated expectations. At the same time, drugs created with the help of biotechnologies have long been used in clinical practice for the treatment of serious diseases. The effectiveness of their use is confirmed by statistics.

In Russia, where, for obvious reasons, the authority of scientists has fallen very low, disappointment in the possibilities of science has naturally led to an increase in the popularity of crooks of various stripes. It is enough to mention the numerous advertisements for the treatment of various ailments with the help of stem cells, creating the impression that this field of science and technology is developing at a global level.

In the rest of the world, a negative attitude towards new biotechnologies is also expressed in attempts to limit, or even stop scientific research in some areas of the life sciences. For example, the fear of cloning, which has practically no rational basis, has led to the adoption of legislation prohibiting it in many countries, which has seriously complicated research related to stem cells, one of the most promising areas of modern medicine. In Europe, due to unfounded consumer concerns, as well as the EU's protectionist policy in the field of agriculture, genetically modified products, which are very popular in the United States, are practically not sold. A number of African countries refused to receive humanitarian food aid due to the far-fetched fears of EU politicians that genetically modified grain will be used for crops and will harm products imported into EU countries.

It is impossible to start a scientific process only along the path of obtaining "good" knowledge. And it is unclear what this is: hardly anyone will challenge the thesis that the same knowledge and technology can be used both for good and for evil. And the rejection of new scientific developments in general deprives humanity of the chance to provide itself with a decent standard of living. Therefore, concerns about the risks posed by new technologies should be treated with understanding — such is the nature of a person who perceives the realities of the next technological order with distrust.

But at the same time, questions are being asked: how big is the return on all those developments that were once presented as breakthrough? Are taxpayers' money going into the sand? Since biotechnologies are called an important element of the innovative development of the leading countries of the world, is there no danger of inflating new financial "soap bubbles", this time in the high-tech sector of the economy?

The complexity of the tasks and the complexity of their understandingIn fact, it may seem that the effect of introducing innovations in biotech is not so great that people feel their real impact on their lives.

Therefore, a counter question is appropriate: what should be compared with? Where are the criteria by which to evaluate the effectiveness of the achievements of the world biotech?

The most frequent reproaches from ordinary people relate to medical applications of biotechnologies: they say that scientists have not yet cured people of cancer, AIDS remains invincible! The human genome has been deciphered — but where are the practical results of this work? The reproaches can be understood, because it is medicine that is the main application of the results of the activities of biotechnologists.

Unfortunately, such questions arise due to two things: people's lack of understanding of the complexity of the tasks that scientists solve, and the desire of the researchers themselves to tell about something unusual that was created in their laboratory. But there is nothing to reproach the scientists themselves here: it is necessary, after all, to demonstrate the goods in person in order to receive funding for the work. As a result, the real results of scientists' activities are replaced by a virtual picture, people have inflated expectations — they say, a little more, and serious illnesses will be defeated, it will be possible to prolong human life, etc.

In reality, everything turns out to be wrong. First of all, this concerns cancer and AIDS. The existing ideas of scientists about the mechanism of the occurrence of cancerous tumors turned out to be far from reality. Over the past four decades, a lot of factors affecting the growth and development of malignant tumors have been discovered, textbooks on molecular biology have been rewritten, but most importantly, it has become clear that it is impossible to find a single method (or combination of methods) for the treatment of cancer. Simply put, there can be no panacea for cancer — each type of tumor requires its own approach to treatment; moreover, a lot depends on the specific organism in which tumor growth occurs. Naturally, the conditions in which the search and testing of new chemical compounds — candidates for a drug are conducted also have their effect on the final effect.

So, if some chemical compound destroys cancer cells in a test tube, this does not mean that it will work as effectively in the body, for example, of an experimental mouse. And if the rodent's condition improves, then tumors in the human body may well be resistant to the new drug. That is why periodic bursts and fading of hopes occur: it is worth getting some strong effect in vitro — in a test tube, for example, the destruction of cancer cells with a new chemical compound, as a breakthrough in cancer treatment is announced. But as time passes, this news is forgotten — because in the body of experimental animals, the new drug may not work at all.

The same applies to AIDS. Since the discovery of the human immunodeficiency virus in 1983, it has become the most studied virus in the history of molecular biology. Again, it may seem to an uninitiated person that a huge amount of accumulated information should lead to victory over this virus. But alas. The immunodeficiency virus has truly diabolical properties that have arisen in the course of evolution, which do not allow us to hope for the complete removal of the virus from the patient's body and even for the creation of an HIV vaccine in the near future. It would not be an exaggeration to say that humanity is faced with a completely unique mechanism of viral infection. Accordingly, fundamentally new approaches are needed for its treatment. At the same time, failures in testing innovative medicines and vaccines, conducted, in particular, by large pharmaceutical companies, often lead to claims that multinational corporations are not interested in curing AIDS. At first glance, these are phrases from the arsenal of conspiracy theory lovers, but in most cases they reflect a misunderstanding of the essence of HIV infection.
Statistics — for scientists

"If death has found one gate closed, it will surely find others" — this phrase is the best way to describe the problems associated with finding remedies for cancer and AIDS-associated diseases. However, this does not mean that scientists are only accumulating information and that there are no fairly effective drugs against these diseases. Existing methods (the so—called antiretroviral therapy) can prolong the life of patients, and statistics confirm this.

For example, with the implementation of an antiretroviral therapy program (based almost entirely on the achievements of biotechnologists) for HIV-infected people in Ethiopia, the number of deaths from AIDS decreased by 50 percent over five years — from 2004 to 2009. And when Brazil introduced the universal free HIV treatment program, the number of deaths from AIDS decreased by 65 percent from 1995 to 2002. Finally, in New York, with the advent of antiretroviral therapy, the number of AIDS deaths has decreased by 63 percent in two years. It is no coincidence that AIDS is already considered a chronic disease in a number of developed countries.

And for the treatment of some forms of cancer, approaches are used to prolong the life of patients. Moreover, we are talking about the use of point effects on cancer cells — this opportunity appeared only thanks to the success of biotechnologists. Of particular note is the technology of creating anti-cancer drugs based on the so-called monoclonal antibodies. The idea of using antibodies as anti-cancer agents has long been very tempting to scientists since their discovery. After all, figuratively speaking, an antibody can be likened to a homing missile, which either neutralizes the intruder — a foreign agent, or, if equipped with a "warhead", destroys a specific target cell, including a tumor one. A radioactive isotope or a powerful toxin can be used as such a "warhead". And it would be absolutely wonderful to learn how to get so—called monoclonal antibodies in large quantities - only one type, against a single target.

But it was only in 1975 that the Americans G. Koehler and S. Milstein managed to create the foundations of such a technology: they merged together the cells of mouse lymphocytes that produce antibodies and tumor cells of myeloma. The resulting hybrid cell could only produce a certain type of antibodies. It seemed that this was the solution to the problem: develop antibodies against the right targets and use them for treatment. However, reality has cooled the initial enthusiasm. It quickly became clear that mouse antibodies have a number of significant drawbacks: they bind poorly to targets, are unable to trigger an immune response, and are quickly excreted from the body without having time to show a therapeutic effect. But the most dangerous thing is that they caused a strong immune response from the patient's body. The search began by trial and error. As a result, with the help of genetic engineering, such monoclonal antibodies were created in which some "mouse" sites were replaced with fragments of human antibodies. And this approach has significantly reduced the side effects of the use of these drugs. Moreover, over time it became clear that specific cases of clinical use require modification of monoclonal antibody molecules. This stimulated the work of scientists, and now they can create antibodies from different molecular "blocks" like a LEGO constructor. Advances in bioinformatics make it possible to model antibodies with predetermined properties, and advances in genetic and cellular engineering technologies allow us to create new methods for developing therapeutic drugs.

Currently, several anti-cancer drugs based on monoclonal antibodies are sold in the world: Herceptin is for the treatment of one of the most aggressive forms of breast cancer, Rituxan and Bexar are for the treatment of non—Hodgkin's lymphoma, Avastin is a remedy for colon cancer. To date, nine-tenths of all drugs based on monoclonal antibodies are intended for oncological use, but in addition to oncology, antibodies are also used in transplantology, for the treatment of cardiovascular, autoimmune and infectious diseases. In the global pharmaceutical market, antibodies occupy the second place in terms of production after vaccines, and sales of therapeutic antibody drugs are projected to increase to $21 billion by 2011.

Biotech targets: not only cancer and AIDSGenetic engineering, being the basis of modern biotechnologies, of course, allows you to create new drugs for other areas of medicine.

Here are some examples as a comment given by one of the leading Russian biotechnologists, Corresponding Member of the Russian Academy of Sciences, head of the Biocatalysis laboratory of the Institute of Bioorganic Chemistry. Academicians M. M. Shemyakin and Yu . A . Ovchinnikov RAS Alexander Gabibov:

"Of course, both oncological diseases and AIDS are hot spots of modern medical biotech. But biotechnological developments are in great demand, for example, in hematology, in which drugs are needed to combat blood loss — both in injuries and in bleeding, for example, after surgical operations. We are talking about drugs that promote blood clotting. In recent years, the foreign recombinant drug NovoSeven, which is an activated factor VII of blood clotting, has been successfully used in the treatment of severe conditions. It is a protein that activates the blood clotting system directly at the site of bleeding, which reduces the risk of thrombosis. Blood clotting factor VII, generally speaking, is a strategic product, because rapid blood clotting, stopping bleeding is critically important for injuries in conditions of man-made disasters or military operations. Emergency measures cannot be carried out without this drug.

The second example of the application of biotechnological developments in hematology is the treatment of hemophilia. Now there are several hundred thousand patients with this disease in the world. For them, any injuries that result in bleeding, as well as medical procedures, such as dental interventions, are dangerous. In the treatment of hemophilia and the prevention of bleeding, another protein is used — coagulation factor VIII. It has been successfully used for the treatment of hemophilia for 20 years.

The advantages of the genetically engineered way of developing the necessary therapeutic proteins are obvious. After all, what is the main drawback of the traditional way of obtaining coagulation factors from blood plasma? If they are insufficiently purified, they may contain human immunodeficiency viruses, as well as hepatitis B and C. And existing biotechnological methods for obtaining recombinant blood clotting factors in principle exclude this possibility. In addition, with the help of genetic engineering methods, it is possible to purposefully improve the properties of proteins, and not only blood clotting factors. I will also say about the need for new drugs for the treatment of autoimmune pathologies and neurodegenerative diseases. It is also a hot spot of modern biopharma and similar developments are in the portfolio of Russian researchers.

Modern biotechnology applied to biopharma cannot exist without advanced industrial technology for the production of recombinant proteins. Those countries in which such technologies are not available are doomed to a progressive lag behind the modern main road of industrial development."

There are other examples of real achievements of biotechnologists in the world. They work, no matter what the skeptics say. New drugs are entering the market. Of course, they are not cheap — but it's not the scientists' fault. It is clear that people want to get an effective pill against some ailment faster and cheaper. But there are no miracles. Therefore, it will be much better if the scientists themselves do not give rise to false hopes, creating unnecessary noise around the next discoveries, which in fact will not be fruitful. Instead, let them try to explain what they are working on and what problems they are facing — then there will be less misunderstanding.

Portal "Eternal youth" http://vechnayamolodost.ru24.08.2009