We are waiting, sir…

Kirill Kayem: Medicine is waiting for genetic drugs

At the end of last year, a drug from a previously incurable disease, hepatitis B, received the status of "breakthrough therapy" in the United States. The developer of the innovative drug was the Russian biotechnological company "Hepatera", a resident of the Skolkovo Foundation. In addition to this innovative company, the Skolkovo biomedical cluster today has 460 resident startups, half of which are engaged in the development of innovative medicines. Kirill Kayem, a hereditary doctor, Senior Vice President for Innovation of the Skolkovo Foundation, told Invest-Foresight about the medicines of the future, the significance of the "figure" for healthcare as an industry and, of course, what medical innovations are being born today in Skolkovo .

Figure for medicine

– Kirill Vladimirovich, what technologies are changing medicine as an industry today?

– This is, of course, a wide stack of technologies, which is commonly referred to as a "digit". In fact, medicine includes many directions. There is medicine related to diagnostics, medicine related to medicines, there is MedTech, which also accounts for a lot of innovations, but it is the "figure" that permeates, unites them all together. And it is the "figure" that brings two fundamentally important innovations to medicine. On the one hand, these are opportunities for the development of new drugs. We are talking about both a serious reduction in the cost of the process and a reduction in risks. On the other hand, the "figure" opens up large–scale predictive possibilities from the point of view of human health.

It will allow you to combine all clinical data, diagnostic signs, both laboratory and behavioral, and predict what will happen to a person, what he should do to live a long, happy and productive life. And this is the main hype that will happen now, because it will really change our paradigm of what a healthy person is. Platformization will have a very serious impact on the field of diagnostics, medicines, and MedTech. Therefore, globally medicine today is changing, of course, the "figure". Other extremely important trends are the development of cellular, regenerative medicine and the development of medicines that affect the genetics of the patient.

– We will change the idea that there is a healthy person. And what will a healthy person mean in the future? 

– This will be considered a person for whom the system will give a good prognosis from the point of view of health. Health will be considered as a system of weighted risks of a particular pathology, which dictates the adoption of a certain set of preventive measures. If a person has a clear predisposition, genetically confirmed, for example, to a stroke or heart attack, this will mean a certain lifestyle, nutrition, a set of examinations that must be regularly passed. And predictive therapy, for example, to reduce cholesterol levels.

– Will human responsibility for health decrease due to technology? 

– A person's personal responsibility for the state of their own health, in my opinion, should become higher. This means that if a person fulfills all the recommendations, medical insurance for him should cost less than for the same person with the same predispositions, but who abuses alcohol, does not exercise, does not monitor cholesterol. Today, people who take care of their health, reducing risks, pay the same insurance premiums as those who lead the wrong lifestyle.

– Will there be fewer mistakes in medicine with the development of technology? The same antibiotic resistance is associated with the incorrect use of drugs…

– Will there be mistakes in the framework of preventive therapy? Probably, just like now there are errors in treatment. Even medicines that enter the market are sometimes recalled. This is the inevitable price for progress. Humanity makes mistakes both at the level of tactical decisions and at the level of global trends. And mistakes with antibiotics are inevitable. But antibiotic resistance is not only a factor in the use of antibiotics in medicine. This is the industrialization of agriculture, the turbulent stage of which humanity has been going through for the last 40 years, including largely the result of the use of antibiotics in animal husbandry. The amount of antibiotic residues in the meat we consume is one of the reasons. But this does not mean that we should take a step back and return to the pre-antibiotic era. It's the same with predictive medicine. Of course, there will be some mistakes, but it's still better than not doing it.

Medicines of the future

– Will medicines change in the future?

– From the point of view of medicines, there is a gradual movement towards genetic drugs. Medicine developed in such a way that at first we treated the external manifestation of the symptoms of the disease, then we tried to treat the cause of the disease. Then the drugs became more and more narrowly acting in terms of symptoms. Then there was targeted therapy, when medicine understands which specific receptor acts to turn off a particular cause of the disease. This has all been happening at the cellular level so far. The next generation of drugs will "work" at the subcellular level. We will introduce into the body no longer substances that affect the disease, but agents that affect the cause of cell malfunction. After all, how do genetic drugs work? They affect the expression of a particular protein that causes the disease, or causes symptoms of the disease, or, conversely, they are not produced when the body needs them. By influencing these mechanisms, we will influence the underlying causes of pathology.

– Will it be possible to turn off some, roughly speaking, the wrong gene if necessary? 

– Yes, including locally. There are already the first drugs that have now entered clinical trials. The Russian venture fund RBV invested in one of them. We are talking about a drug for the treatment of retinopathy, which causes blindness. This is a medicine that "turns off" a certain gene, stops the development of the disease, moreover, allows you to restore vision.

– What is the role of residents in the work on innovative medicines Skolkovo?

– There are currently 460 projects in the biomedical cluster of the Skolkovo Foundation, of which about half are engaged in the development of early-stage medicines. Unfortunately, there are not very many institutions in Russia that support the development of medicines at an early stage. It is clear that under the department of the Ministry of Health and the Ministry of Education and Science, developments are being carried out within the framework of scientific work. And it is clear that there are good programs conducted by the Ministry of Industry and Trade. But in recent years, the Ministry of Industry and Trade has focused more on the later stages of development, when the risks are lower. Now there is an initiative of the Ministry of Industry and Trade to create a special venture fund within the framework of RVC to support projects of earlier stages. We are very supportive of this, but the venture fund has not been launched yet. It was the Skolkovo Foundation that for several years remained one of the rare development institutions that continued to do this at an early stage. Unfortunately, everything is cynical with medicine. As a rule, a private investor does not give money to early-stage drug development projects. And all over the world it is supported by the state.

– Are they working on genetic medicines at Skolkovo? 

– Yes, of course. For example, the biotech company AGCT is a startup from St. Petersburg, which is still at a fairly early stage. They are developing a genetic therapy for HIV patients. Roughly speaking, they want to "turn off" a certain gene in order to stop the development of complications. Mostly patients die from HIV-associated complications. Taking into account the specifics, they can start testing with humans early enough, it cannot be simulated on animals, and they will provide assistance to patients. I am proud that this is happening at Skolkovo, but not only high-tech startups are moving along this path. Russian pharmaceutical companies are also moving in this direction, conducting their innovative developments. Finally, Russian pharma does not copy, but makes its own developments.

– And what breakthrough drugs are already available?

– There are several drugs, cool and breakthrough, that have already received permission for the final clinical stage of trials. This is a development of the biotechnological company "Hepatera". Myrcludex B is the world's first drug for previously incurable hepatitis D. There is viral hepatitis B, which is very common, and it has a complication, viral hepatitis D is another type of virus, there was no cure for it. And colleagues made it. This is orphan medicine. In Russia, there are about 100 thousand people with such a complication, for them there was only symptomatic therapy. Myrcludex B is a "first–in-class" drug with an original mechanism of action, when the virus, in fact, is locked in a cell, which prevents it from multiplying and reduces the viral load on the patient. Patients are very much waiting for this medicine.

You can also name such a drug as "Alofanib". It was developed by Rusfarmtech, which successfully completed preclinical studies, and the Ministry of Health gave permission for clinical trials. The first indication of the drug is stomach cancer, targeted therapy. But it can also be used to treat, for example, triple negative breast cancer, when none of the three lines of the main therapy works. Clinical trials of the drug are now beginning.

– They will also produce in Russia?

– Yes, these medicines can be produced in Russia. By the way, this is one of the rare innovative drugs where the Ministry of Health has issued a permit to a Russian company for clinical trials of a drug of its own development for the treatment of oncopathology. And they are already now, within the framework of clinical research, will help people. Free use of the drug can be expected in a few years.

– New medicines are very expensive to develop, isn't it easier to land existing foreign ones?

– What our startups are developing is not available in foreign companies. The criteria for choosing a startup for Skolkovo are uniqueness, innovation and global scale. Perhaps there are some competitive developments, but Myrcludex B and Alofanib have no competitors. These are medicines that will be in demand both in Russia and in the world, for sure.

A cancer pill

– One of the hottest topics in medicine is the fight against oncopathology. Is it true that such a diagnosis has become more common today?

– People have begun to live longer, the number of mutagenic factors is growing. The percentage of oncological diseases is growing for these two reasons mainly.

– How can Skolkovo help here?

– I gave an example of an oncological drug. We even have a separate oncology manager who combines oncological diagnostics and oncological drugs. We work with the Russian Society of Clinical Oncology (RusCO). We hold separate sessions at the Oncological Congress to tell how we support oncological projects.

– Is there a chance that the long-awaited cancer pill will be invented in Russia? 

– No, because in principle there can't be one pill for cancer. There is no such disease "cancer" – these are groups of diseases that doctors have grouped according to some similarity of signs. It is wrong even to say "breast cancer" or "stomach cancer" – the genesis of the disease is very different, in many cases – a combination of several important factors, although we habitually call them the same. And the medications that are currently developing affect the cause of the disease. This means that for each of the reasons, you have to develop your own drug and use a combination of drugs and other means to combat the disease. But there won't be one pill in the foreseeable future.

I hope that Russia will develop and produce many effective drugs for specific nosologies, and we see that the number of such projects is growing. But the problem for humanity is so great that all countries will move in this direction and participate in this process. And everyone will invent something the best that will be used in all countries. Now innovative pharma is a global market.

Talking Olga Blinova

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