I hate it when someone treats me
Living conditions around are constantly changing, and not everyone can adapt to them. For this, perhaps, came up with the science of healing. It is aimed at maintaining and strengthening the health and working ability of people: diagnosing, treating and preventing diseases of various organs, prolonging life and alleviating suffering from physical and mental ailments. Medicine has come a long way from sorcery and shamanism to personalized medicine. In 2017, cell therapy is allowed for use in Russia and access to high-tech medical care is facilitated, but the main recommendations for maintaining a healthy lifestyle are drinking enough water and a regular daily regimen with adequate sleep duration. It becomes more difficult when you need medication. Read about the failures of ambitious clinical trials and the difficulties of creating new drugs in the latest Genotek material.
The first and foremost condition for the existence of medical technology is an understandable mechanism of action. Otherwise we are talking about medicine. Earlier, when a person had a headache, he went to the shaman, he gave him a magic ointment and told him to rub it at a certain point on the body clockwise a week before the waning moon. Now a lot has changed (the “SARKAZM” sign is shown after this phrase at the request of the reader). In fact, the choice of a drug by a doctor, the diagnosis of the disease and the treatment regimen is a complicated procedure that has reached its gold standard in recent years. However, the real process of interaction with a doctor - if the possibility of such interaction exists at all - is often far from ideal.
If the visit did take place and the specialist gave a list of valuable instructions, it is important to get down to business in a timely manner. For a long time to be treated, and then no one wants to suffer from side effects. The main advantage of modern drugs is to fine-tune the functions, which contrasts sharply with the deadly power of drugs of past generations. Developing a good medicine is difficult. For example, in 2016, the launch of mass production of 19 medicines was approved in the USA. In 7 of them, the mechanism of work is fundamentally new . For example, one of the drugs is aimed at the treatment of dry eye syndrome. It reduces inflammation by binding the active substance with protein on the surface of leukocytes, which prevents their penetration into the cornea. At the same time, “the discovery of new drug properties ” is more likely a disadvantage of the research and the developed drug than an advantage. In order to prevent the repetition of the shameful story with Theranos , which everyone is trying to forget, innovations are now required to be transparent in describing the details of the technology.
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The key is the fact that in order to launch drugs into mass production, it is necessary to demonstrate a well-conducted experiment with statistically convincing results. Richard Peto, a professor at Oxford University, formulated the main idea of ​​clinical research as early as 1980: “you need to ask an important question and find the appropriate answer to it”. Today, one of the criteria for the adequacy of clinical studies is considered to be a rich statistical sample. And this makes sense: if a publicly available medicine is produced, it is worth checking how it works on a large number of people.
One of the largest clinical studies was the study of the effect of vitamin A on reducing mortality in children under 6 years of age. At the beginning of the two thousandth, 720,000 children of pre-school age from Indian villages took part in it. For 5 years, they ate 4 tons of vitamin A, but not all survived. Statistical analysis of the results did not allow us to conclude that vitamin A helps Indian children to survive. The classic clinical study of the drug's properties with colossal funding did not give a “beautiful” result, which made scientists pull with the publication. The data was contradictory and generated more questions than answers.
A method for testing gene therapy drugs reverses ideas about clinical research. Hemophilia is a hereditary incoagulability disease of the blood of the most august persons who are also affected by mortals. Last year, BioMarin conducted a study of a gene therapy drug for hemophilia A. This disease is associated with an insufficient amount of protein in the blood plasma (clotting factor VIII), which is especially dangerous for injuries and internal bleeding. The study participants (there were only 9 people) were injected with a viral vector, which delivered the coagulation factor VIII gene to the cell. In 6 of 7 patients with high-dose therapy, the protein content increased by 50% and the frequency of bleeding decreased, which indicates the success of therapy. The remaining two received medication in smaller doses - their dynamics were positive, but less pronounced. However, the medicines of the new generation and their testing are now ambiguous. In 1999, during the experiment on gene therapy, Jesse Jelsinger was killed. It was a sudden death: a young man died 4 days after drug administration. Doctors fear repetition of this tragedy. Therefore, it is especially scary to miss something during the tests. At a minimum, a large sample is required to find out about possible side effects or the dynamics of the drug's action. There are several problems here. On the one hand, it is difficult to look for suitable patients: hemophilia is a rare disease. On the other hand, the cost of the drug is very high, which also makes it difficult to conduct clinical studies. Whether it's vitamins.
Linus Pauling, a brilliant scientist of the 20th century, who left a mark in physics, chemistry and biology, in 1966 wanted to live at least another 20 or 25 years - he wondered what would happen next in this world. This request did not go unanswered. Biochemist Irwin Stone found that most mammals (with the exception of primates and guinea pigs) synthesize in the liver a volume of vitamin C proportional to body weight. He wrote a letter to Pauling, in which he recommended taking ascorbic acid 3 grams per day. Linus followed this advice and felt much better, which he did not fail to state in his book Vitamin C and the Common Cold. The book became a bestseller, and Pauling's fellow citizens dared vitamin C from pharmacy counters, completely ignoring the more expensive drugs than caused the outrage of pharmaceutical companies. Studies on the clinical action of antioxidants and vitamin C in particular are still ongoing, but its effect “against all ills” has not been proven. Someone is trying to discredit the great scientist on the basis of this. Be that as it may, Linus Pauling died of cancer only in 1994, retaining the vigor of his mind and spirit, having lived even longer than the cherished 25 years.
Innocent faith in the discovery of one authoritative person is not at all the same as betting on a dangerous substance for profit. The most famous pharmaceutical disaster - “thalidomide tragedy” , which occurred in the 60s. An effective, non-addictive sleeping pill has entered the world market, which has also been recommended for all women from toxemia in the first trimester of pregnancy. Due to the haste of the drug to the market, the manufacturer did not take into account the strong effect of the drug on embryonic development. Accidents began 9 months after taking the pills by the first patients: babies with underdeveloped limbs began to be born. Thalidomide was recalled, but it was too late. The pharmaceutical company still pays cash compensation to people who become disabled through its fault. It was a monstrous tragedy, but it was she who became the first step towards the creation of an institute to oversee the development and production of drugs.
The pharmaceutical industry is an extensive industry that is now actively developing. Surely there will be a lot of new drugs that can be chosen to suit your taste, genome and wealth. Experts agree: the future is still in preventive medicine, because it is cheaper, simpler and controlled by the person himself. A truth that does not age: "Disease is easier to prevent than to cure." Since ancient times, man has been studied using a variety of biology methods. It is known that the pathological state of the body is only one of the phases that can be aggravated if you do not monitor your health.
People used to go to the doctor when they got sick. In the modern health care system there is such an event as a clinical examination. In fact, it is the simplest tool of preventive medicine. The chief city specialist in preventive medicine reported that in the first 6 months of 2016, 400,000 Muscovites underwent medical examination. In 2016, the population of Moscow amounted to more than 12 million people. That is, about 3% of Muscovites are interested in their state of health and effectively spend the state budget for these purposes. Clinical examination saves time and treatment costs, but is not very popular and is not always effective. In the future, diagnostics will become more efficient, faster and “more preventive.” The future is here!
In the modern world, preventive medicine of the new generation is available in the form of DNA tests. There are several types of them, and they should be understood.
Someone is interested to pass a basic survey, just to see their weak points. To do this, you can pass a biological sample for analysis on microchips . Microchips, like punched cards, emit 650,000 positions of the genome, the most likely to find mutations. According to data from microchips, you can define a large set of mutations and learn a lot about yourself. However, sometimes curiosity and concern about our own health lead us on. In this case, it may be necessary to analyze more information.
One of the main challenges of humanity at the beginning of the XXI century was the complete sequencing of the genome. It is now available to all comers , but this procedure is quite expensive. Today, the sequencing of the complete genome is of great value for science, but not for health. The analysis is a complete transcript of the entire DNA molecule, which is packaged in human cells. In addition to detailed information about their health, as in the analysis on microchips, a person gets the opportunity to see not the individual letters (nucleotides), but the entire text. It is as if you, going to a certain site, consider not only the basic information, but also all the advertising banners and text from the developer who is written there. Manipulations on a full-genome scale are not available in mass medicine, because we still don’t know much, but by sequencing the genome, a person enters a special club.
Compromise in this case is sequencing of exome - all genes encoding proteins. In this type of analysis, 12,000,000 points (loci) are investigated, which is significantly smaller than the entire genome. This is the most detailed information about the carrier of genetic diseases. Suppose you have a family history and you want to know about specific risks in more detail, to protect yourself and your children. Personalized drug development is a matter of the future, but you can already consult a geneticist.
In special cases, a clinical analysis of exome is required, which is prescribed by the attending physician. This is the way to diagnose certain diseases, for example, neurological disorders in children.
Finally, the question of awareness arises. In essence, who is a person and what is important for him in life? The world is leaning in favor of individualism: people live for themselves and create integrity in themselves, to a lesser extent feeling themselves part of the whole. A course for 4P medicine is taken, the main principles of which are predictability, prevention, personalization and perseptivity (adopted, mutual aid). Science opens the door to a new generation of medicine, but the question is whether a person can enter them. How will it be: through evolution or with revolution? Wait and see.
Take care of yourself. Happy New Year!
PS Until January 15, our contest of incredible stories about genetics continues - hurry to participate and vote!
Visit to the shaman
The first and foremost condition for the existence of medical technology is an understandable mechanism of action. Otherwise we are talking about medicine. Earlier, when a person had a headache, he went to the shaman, he gave him a magic ointment and told him to rub it at a certain point on the body clockwise a week before the waning moon. Now a lot has changed (the “SARKAZM” sign is shown after this phrase at the request of the reader). In fact, the choice of a drug by a doctor, the diagnosis of the disease and the treatment regimen is a complicated procedure that has reached its gold standard in recent years. However, the real process of interaction with a doctor - if the possibility of such interaction exists at all - is often far from ideal.
Subtle Matter Gene Therapy
If the visit did take place and the specialist gave a list of valuable instructions, it is important to get down to business in a timely manner. For a long time to be treated, and then no one wants to suffer from side effects. The main advantage of modern drugs is to fine-tune the functions, which contrasts sharply with the deadly power of drugs of past generations. Developing a good medicine is difficult. For example, in 2016, the launch of mass production of 19 medicines was approved in the USA. In 7 of them, the mechanism of work is fundamentally new . For example, one of the drugs is aimed at the treatment of dry eye syndrome. It reduces inflammation by binding the active substance with protein on the surface of leukocytes, which prevents their penetration into the cornea. At the same time, “the discovery of new drug properties ” is more likely a disadvantage of the research and the developed drug than an advantage. In order to prevent the repetition of the shameful story with Theranos , which everyone is trying to forget, innovations are now required to be transparent in describing the details of the technology.
')
Ask the right questions - get the right answers
The key is the fact that in order to launch drugs into mass production, it is necessary to demonstrate a well-conducted experiment with statistically convincing results. Richard Peto, a professor at Oxford University, formulated the main idea of ​​clinical research as early as 1980: “you need to ask an important question and find the appropriate answer to it”. Today, one of the criteria for the adequacy of clinical studies is considered to be a rich statistical sample. And this makes sense: if a publicly available medicine is produced, it is worth checking how it works on a large number of people.
Mass recovery
One of the largest clinical studies was the study of the effect of vitamin A on reducing mortality in children under 6 years of age. At the beginning of the two thousandth, 720,000 children of pre-school age from Indian villages took part in it. For 5 years, they ate 4 tons of vitamin A, but not all survived. Statistical analysis of the results did not allow us to conclude that vitamin A helps Indian children to survive. The classic clinical study of the drug's properties with colossal funding did not give a “beautiful” result, which made scientists pull with the publication. The data was contradictory and generated more questions than answers.
Unique finds
A method for testing gene therapy drugs reverses ideas about clinical research. Hemophilia is a hereditary incoagulability disease of the blood of the most august persons who are also affected by mortals. Last year, BioMarin conducted a study of a gene therapy drug for hemophilia A. This disease is associated with an insufficient amount of protein in the blood plasma (clotting factor VIII), which is especially dangerous for injuries and internal bleeding. The study participants (there were only 9 people) were injected with a viral vector, which delivered the coagulation factor VIII gene to the cell. In 6 of 7 patients with high-dose therapy, the protein content increased by 50% and the frequency of bleeding decreased, which indicates the success of therapy. The remaining two received medication in smaller doses - their dynamics were positive, but less pronounced. However, the medicines of the new generation and their testing are now ambiguous. In 1999, during the experiment on gene therapy, Jesse Jelsinger was killed. It was a sudden death: a young man died 4 days after drug administration. Doctors fear repetition of this tragedy. Therefore, it is especially scary to miss something during the tests. At a minimum, a large sample is required to find out about possible side effects or the dynamics of the drug's action. There are several problems here. On the one hand, it is difficult to look for suitable patients: hemophilia is a rare disease. On the other hand, the cost of the drug is very high, which also makes it difficult to conduct clinical studies. Whether it's vitamins.
Dreams of Vitamin
Linus Pauling, a brilliant scientist of the 20th century, who left a mark in physics, chemistry and biology, in 1966 wanted to live at least another 20 or 25 years - he wondered what would happen next in this world. This request did not go unanswered. Biochemist Irwin Stone found that most mammals (with the exception of primates and guinea pigs) synthesize in the liver a volume of vitamin C proportional to body weight. He wrote a letter to Pauling, in which he recommended taking ascorbic acid 3 grams per day. Linus followed this advice and felt much better, which he did not fail to state in his book Vitamin C and the Common Cold. The book became a bestseller, and Pauling's fellow citizens dared vitamin C from pharmacy counters, completely ignoring the more expensive drugs than caused the outrage of pharmaceutical companies. Studies on the clinical action of antioxidants and vitamin C in particular are still ongoing, but its effect “against all ills” has not been proven. Someone is trying to discredit the great scientist on the basis of this. Be that as it may, Linus Pauling died of cancer only in 1994, retaining the vigor of his mind and spirit, having lived even longer than the cherished 25 years.
Terrible mistakes
Innocent faith in the discovery of one authoritative person is not at all the same as betting on a dangerous substance for profit. The most famous pharmaceutical disaster - “thalidomide tragedy” , which occurred in the 60s. An effective, non-addictive sleeping pill has entered the world market, which has also been recommended for all women from toxemia in the first trimester of pregnancy. Due to the haste of the drug to the market, the manufacturer did not take into account the strong effect of the drug on embryonic development. Accidents began 9 months after taking the pills by the first patients: babies with underdeveloped limbs began to be born. Thalidomide was recalled, but it was too late. The pharmaceutical company still pays cash compensation to people who become disabled through its fault. It was a monstrous tragedy, but it was she who became the first step towards the creation of an institute to oversee the development and production of drugs.
Explore people, not drugs
The pharmaceutical industry is an extensive industry that is now actively developing. Surely there will be a lot of new drugs that can be chosen to suit your taste, genome and wealth. Experts agree: the future is still in preventive medicine, because it is cheaper, simpler and controlled by the person himself. A truth that does not age: "Disease is easier to prevent than to cure." Since ancient times, man has been studied using a variety of biology methods. It is known that the pathological state of the body is only one of the phases that can be aggravated if you do not monitor your health.
3% of Muscovites - from the future
People used to go to the doctor when they got sick. In the modern health care system there is such an event as a clinical examination. In fact, it is the simplest tool of preventive medicine. The chief city specialist in preventive medicine reported that in the first 6 months of 2016, 400,000 Muscovites underwent medical examination. In 2016, the population of Moscow amounted to more than 12 million people. That is, about 3% of Muscovites are interested in their state of health and effectively spend the state budget for these purposes. Clinical examination saves time and treatment costs, but is not very popular and is not always effective. In the future, diagnostics will become more efficient, faster and “more preventive.” The future is here!
Not a prediction, but a warning
In the modern world, preventive medicine of the new generation is available in the form of DNA tests. There are several types of them, and they should be understood.
Introduction to your own health
Someone is interested to pass a basic survey, just to see their weak points. To do this, you can pass a biological sample for analysis on microchips . Microchips, like punched cards, emit 650,000 positions of the genome, the most likely to find mutations. According to data from microchips, you can define a large set of mutations and learn a lot about yourself. However, sometimes curiosity and concern about our own health lead us on. In this case, it may be necessary to analyze more information.
New level of self-knowledge
One of the main challenges of humanity at the beginning of the XXI century was the complete sequencing of the genome. It is now available to all comers , but this procedure is quite expensive. Today, the sequencing of the complete genome is of great value for science, but not for health. The analysis is a complete transcript of the entire DNA molecule, which is packaged in human cells. In addition to detailed information about their health, as in the analysis on microchips, a person gets the opportunity to see not the individual letters (nucleotides), but the entire text. It is as if you, going to a certain site, consider not only the basic information, but also all the advertising banners and text from the developer who is written there. Manipulations on a full-genome scale are not available in mass medicine, because we still don’t know much, but by sequencing the genome, a person enters a special club.
Golden mean
Compromise in this case is sequencing of exome - all genes encoding proteins. In this type of analysis, 12,000,000 points (loci) are investigated, which is significantly smaller than the entire genome. This is the most detailed information about the carrier of genetic diseases. Suppose you have a family history and you want to know about specific risks in more detail, to protect yourself and your children. Personalized drug development is a matter of the future, but you can already consult a geneticist.
Clinical indications
In special cases, a clinical analysis of exome is required, which is prescribed by the attending physician. This is the way to diagnose certain diseases, for example, neurological disorders in children.
Cog in the car
Finally, the question of awareness arises. In essence, who is a person and what is important for him in life? The world is leaning in favor of individualism: people live for themselves and create integrity in themselves, to a lesser extent feeling themselves part of the whole. A course for 4P medicine is taken, the main principles of which are predictability, prevention, personalization and perseptivity (adopted, mutual aid). Science opens the door to a new generation of medicine, but the question is whether a person can enter them. How will it be: through evolution or with revolution? Wait and see.
Take care of yourself. Happy New Year!
PS Until January 15, our contest of incredible stories about genetics continues - hurry to participate and vote!
Source: https://habr.com/ru/post/400491/
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