Monoclonal antibodies and funmmarines

There cannot be modern medicine where there is no modern economy

At his annual press conference, Vladimir Putin said that Russian medicines are “no worse than imported”. When you hear this, I want to ask: and you yourself in the Kremlin, what are you treating? Really domestic?

Let's just say: in the 60s, there was a good medicine for its time in the scoop.

But now, after 60 years, Russia and the developed world are on different planets.

The reason for this is very simple. Modern medicine is a business. This is a huge factory that produces health for a lot of money. If the medicine is state, but the patient does not have money, then there is no business. From development to the counter, the medicine on average costs $ 1 billion. Therefore, 95% of new drugs are the United States.

Of course, I am not a doctor. But in order not to be unfounded.

In August of this year, Science said that in Dallas, scientists first used CRISPR/CAS9 technology, i.e. Gene Editing technology, gene editing - to cure muscle dystrophy of Duchenne in dogs. This is one of the most common genetic dystrophy that is found in people. It affects one boy out of 5 thousand. The muscles are gradually weakening, and by the age of 13-15, the boy in a wheelchair just refuses to beat in a wheelchair. Or he cannot breathe.

In the same August, two American companies used CRISPR at once to treat blood disease-beta-Talassemia. The mechanism is the same. They take blood from the patient, edit a faulty gene, as the corrector replaces the typo in the text, the gene begins to produce hemoglobin. And blood is administered back. The drug entered the phase of clinical trials, and on living patients.

One of the most promising technologies is the use of CRISPR to treat lung cancer - one of the worst types of cancer. Clinical trials began in 2016. Cells were extracted from a person with a cancer, edited them to erase the expressed RD1 gene, and introduced it back. Interestingly, most of the tests are conducted by Chinese scientists.

In total, CRISPR/CAS9 research is carried out for cancer, sickle anemia, hemophilia, heart disease, Huntington diseases, etc.

To make it clear: CRISPR, how the technology began to be developed in 2013-2014. Jennifer Dudna, 54-year-old American from UC Berkeley, and Feng Zhang from Broad Institute 2015 received a prize for her as a medical breakthrough of the year.

While we arranged Donbass and planted scrapers, in the USA, like mushrooms, new medical companies that CRISPR are engaged -

Millipore Sigma, Toolgen, Caribou Biosciences, ERS Genomics, etc. It would be interesting to ask President Putin what Russian companies are engaged in CRISPR? Who is no worse for us than Dow AGROSCIENCES? Who is in the place of Crispr Therapeutic? Who instead of Jennifer Dudna and Feng Zhang?

Apart from universities, almost all the companies engaged in CRISPR are startups. These are future medical Amazon and Google. Who heard about PayPal twenty years ago? Who heard about Airbnb ten years ago? And now we buy a plane ticket on the Internet, pay hotels, make purchases.

The same will be with CRISPR. Because the point is not only in terrible diseases like cancer or sickle anemia. The fact is that with the help of a technology invented by Jennifer Duda and Feng Zhang, you can edit the genome so that you never get AIDS. Which was just done by the Chinese scientist Jiankui He.

You can increase the effectiveness of antibiotics. You can edit the gene so that we never have herpes, and 95% of the population has herpes. Herpes is a rubbish, a trifle seems to be, but the trouble is: herpes virus usually has to jump in a DNA chain from place to place. And where he tears the chain, there is a high probability of damage and cancer.

Twenty years later, one of these CRISPR startups will occupy the first lines of capitalization, squeezing Amazon. Do the Kremlin really believe that there will be at least one Russian company among them?

If the Russian physician is embarrassed by a crazy idea to engage in these technologies in Moscow - where will he find equipment? Colleagues? Investments? How soon will the vigilant Feesbashniks will put him for writing a letter to Harvard?

I say another planet.

CRISPR is not all. Another fresh technology that continues the triumphal procession is monoclonal antibodies.

This, roughly speaking, is a protein complex that is associated only with another specific protein complex. Imagine a cage in the form of a door that has a block with a well. And a monoclonal antibody is a key that can be turned in this well and, say, cause apoptosis - programmed by nature the death of this particular cell, with such a protein. Now imagine that this cage is cancer.

There are many monoclonal antibodies in Russia in Russia?

The third largest technology, which is also rapidly developing, is the largest breakthrough in the treatment of cancer after the invention of chemotherapy: Genomic Profiling. When you are not just treated with cancer, but first they watch your DNA profile. And they give you medicines when they see specific biomarkers. It is again the same difference that breaking the door or find a key to the well. If earlier they looked at which organ is amazed, now they look at what broken gene is responsible for the disease. And, of course, medicines created taking into account Genomic Profiling are very expensive. Because they act, for example, on 4% of the sick. But they give 95% of efficiency. And on others this medicine does not act at all - not the genetic profile.

You can ask what Russian companies produce similar medicines?

Alzheimer's disease is now one of the worst diseases. It costs Americans 259 billion a year, and by 2050 there will be a trillion. In July of this year, Biogen announced: he has a medicine that slows down the development of the disease. BAN2401. Biogen shares have taken off. Question: How are we in Russia with drugs from Alzheimer? Are there also domestic analogues? What exactly - tea with raspberries? Scrapers in tablets?

Spark Therapeutics, Phfizer, Biomarin Pharmaceuticals and Unique all launch genetic therapy for hemophilia - and can you find out what Russian companies are working on this?

Hepatitis C. 150 million infected in the world. More recently, it was a fatal disease. Now it is completely cured in 99% of the thoroughs. Reason: all the same monoclonal antibodies, Sofosbuvir and Velpatasvir drugs. The question is closed. The price of the exchange rate is 80 thousand dollars. Normal for market medicine, unattainable for Russia.

Gout is a disease in which the patient suffers from monstrous pain caused by excess uric acid and its deposition in the joints. The drug Uloric, which appeared on the market since 2008, closed the issue with gout. Uloric simply blocks the overproduction of uric acid. And what has already been postponed is washing. Can you find out where the Russian counterpart is Uloric? In Russia, by the way, a little bit costs $ 300.

Another revolutionary medicine of this year: Aimovig and Ajovy for the treatment of migraine. Of course, on the basis of monoclonal antibodies. Actually, these antibodies turn off CGRP (Calcitonin Gene-Reled Peptide), which launches migraine.

And also on the basis of monoclonal antibodies - Praluen and Repatha. Scientists watched several thousand people with different blood cholesterol . They chose those who had a populist cholesterol. Cleaned their DNA. Determined which mutations reduce cholesterol. We looked at what kind of enzyme these mutations turn off. It turned out that Enzym PCSK9. They turned it off using monoclonal antibodies. So far, FDA approved these drugs only for patients with hypercholesterolelets. But in principle, these medicines are statins successors. And the solution to the issue with cholesterol once and for all. It is necessary to explain how the cholesterol, heart attack and stroke are connected?

And what do we have in Russia against this background?

The only thing we succeed in is in the production of numerous bullomicins, such as arbidol or kagocel, the clinical efficiency of which, to put it mildly, has not been proven, which does not prevent them from being included in the list of vital drugs. So they have monoclonal antibodies, and we have funmmarines.

What are all CRISPR technologies based on? What are all these new companies based on - Caribou Biosciences, Ers Genomics, Editas Medicine, Intellia Therapeutics, etc.?

• On a huge scientific baggage and scientific culture

• And on the fact that they offer new technology for patients and earn a lot of money on it.

And what are our funmicics based on? The fact that the medicine that has not yet passed the double blind randomized test is included in the vital list and the budget purchases it.

In one case, the final buyer is a patient. In another - the state.

Moreover, Russian patients still do not have that kind of money: Aimovig, for example, costs about 120 thousand a year, despite the fact that traditional statins cost $ 250. There can be no modern medicine where there is no modern economy.