Development of pharmaceuticals: new drugs and methods of treatment in the world
I. Oncology: Revolution in the treatment of cancer
1. Immunotherapy: freeing the power of the immune system
Immunotherapy, which is one of the most significant revolutions in the treatment of cancer over the past decades, uses the patient’s own immune system to combat the tumor. Instead of direct exposure to cancer cells, immunotherapy enhances or restores the ability of the immune system to recognize and destroy them.
- Control points inhibitors (Checkpoint Inhibitors):
These drugs block proteins known as control points on immune cells, such as T-lymphocytes. Control points, such as PD-1 (Programmed Cell Death Protein 1) and CTLA-4 (Cytotoxic T-Lymphocyte-Ssociated Protein 4), normally help prevent excessive activity of the immune system. However, cancer cells can use these control points to suppress the immune response and avoid destruction. Control points inhibitors, such as Pembroralizumab (Keytruda) and Nivolumab (Opdivo), block these proteins, allowing T-lymphocytes to attack cancer cells more efficiently.
Clinical tests showed the impressive results of the use of control points inhibitors in the treatment of various types of cancer, including melanoma, lung cancer, kidney cancer, bladder cancer and Hodgkin lymphoma. Some patients have a long remission and even a complete cure.
- CAR-T-cell therapy
CAR-T-cell therapy is a personalized approach to cancer treatment, in which the patient T-lymphocytes are modified in the laboratory for the expression of a chimeric antigenic receptor (CAR). This receptor allows T-lymphocytes to recognize specific antigens on the surface of cancer cells. Modified T-lymphocytes are then introduced back into the patient’s body, where they find and destroy cancer cells expressing the target antigen.
Car-T-cell therapy showed high efficiency in the treatment of certain types of blood cancer, such as B-cell lymphoma and acute lymphoblastic leukemia. Examples of Car-T-cell drugs are Tymriahlesel and Axycabtagen Siloleusel (Yescarta).
- Oncolytic viruses (Oncolytic Viruses):
Oncolytic viruses are genetically modified viruses that selectively infect and destroy cancer cells, without causing harm to healthy cells. When the virus infects the cancer cell, it multiplies inside the cell, causing its lysis (destruction). In addition, infected cancer cells emit signals that stimulate the immune system to attack on the tumor.
The tallimogen camp (Imlygic) is an oncolytic virus approved for the treatment of melanoma, which is not amenable to surgical treatment. Studies continue to assess the effectiveness of oncolytic viruses in the treatment of other types of cancer.
2. Targeted therapy: accurate aiming on cancer cells
Targeted therapy is an approach to cancer treatment, in which drugs are aimed at specific molecules involved in the growth, development and spread of cancer cells. Unlike traditional chemotherapy, which affects all rapidly dividing cells, targeted therapy is more selective and, as a rule, causes less side effects.
- Tyrosine Kinase Inhibitors – Tkis):
Tyrosinkinase is enzymes that play an important role in transmitting signals inside cells, regulating growth, differentiation and cell survival. Mutations in genes encoding tyrosinkinase can lead to uncontrolled growth of cancer cells. Tyrosinkinase inhibitors block the activity of these enzymes, suppressing the growth and spread of cancer.
Imatinib (GleEvec) is one of the first and most successful inhibitors of tyrosinkinase, which is used to treat chronic myeloidal leukemia (KML). Other Tyrosinkinase inhibitors are used to treat various types of cancer, including lung cancer, kidney cancer and breast cancer.
- Parp inhibitors (Parp Inhibitors):
PARP (Poly (Adp-Ribose) Polymerase) is an enzyme that is involved in DNA reparations. PARP inhibitors block the activity of this enzyme, which leads to the accumulation of DNA damage in cancer cells and their death. PARP inhibitors are especially effective in the treatment of ovarian cancer, breast cancer and prostate cancer in patients with mutations in BRCA1 and BRCA2 genes, which also participate in DNA reparations. Examples of PARP inhibitors are Olanparza and Rubraca.
- Inhibitor mTOR (mTOR Inhibitors):
Mtor (Mammalian Target of Rapamycin) is a protein that regulates the growth, proliferation and metabolism of cells. MTOR inhibitors block the activity of this protein, suppressing the growth and spread of cancer. Everolimus (Afinitor) is an inhibitor of MTOR, which is used to treat kidney cancer, breast cancer and neuroendocrine tumors.
3. Liquid biopsy: non -invasive cancer monitoring
Liquid biopsy is a non -invasive method of diagnosis and monitoring of cancer, which is based on the analysis of blood samples or other biological fluids to detect circulating tumor cells (CEC), tumor DNA (Central Military Code) or other cancer biomarkers.
- Detection and monitoring of the Central Administration:
Analysis of the Central Administration allows you to identify genetic mutations in cancer cells, monitor the effectiveness of treatment and identify signs of cancer relapse in the early stages. Liquid biopsy can be used to select the most effective targeted therapy based on the genetic profile of the tumor.
- Identification of TsOC:
Tsok is cancer cells that separated from the primary tumor and circulate in the blood. The amount of TsOC can be used to predict the outcome of the disease and evaluate the effectiveness of treatment.
II. Neurology: Progress in the treatment of neurological diseases
1. Modifier drugs with multiple sclerosis (RS):
Scattered sclerosis (RS) is a chronic autoimmune disease that affects the central nervous system. Modifying drugs (MBP) are drugs that slow down the progression of RS and reduce the frequency and severity of exacerbations.
- Interferon Beta:
Beta interferons (for example, betferon, rebif, avonex) are immunomodulating drugs that reduce inflammation in the central nervous system.
- Headmeter acetate (Copaxon):
Acetate glatirameromer is a synthetic polypeptide that imitates the main protein of Myelin, a component of the myelin shell of nerve fibers. It is assumed that Acetate Glatyrameroma reduces an autoimmune attack on Myelin.
- Fingolimod (Gilenya):
Fingolimod is a spingozin-1-phosphate receptor modulator (S1P), which holds lymphocytes in the lymph nodes, preventing their penetration into the central nervous system.
- Natalizumab (Tysabri):
Natalizumab is a monoclonal antibody that blocks the adhesion of lymphocytes to the vascular endothelium, preventing their penetration and spinal cord.
- Ocrelizumab (Ocrevus):
Ockrelizumab is a monoclonal antibody, which is aimed at B-lymphocytes that play an important role in the development of RS.
2. New methods of treating Alzheimer’s disease:
Alzheimer’s disease is a progressive neurodegenerative disease, which is characterized by a decrease in cognitive functions, such as memory, thinking and speech.
- Anti -amyloid drugs:
Anti -amyloid drugs, such as adukanumab (aduhelm) and LEQEMBI, are aimed at removing amyloid plaques from the brain. Amyloid plaques are deposits of protein beta amyloid, which are considered one of the main pathological signs of Alzheimer’s disease. Clinical tests have shown that these drugs can slow down the progression of the disease in the early stages.
- Tau-controlled therapy:
Tau-controlled therapy is aimed at preventing the formation and spread of neurofibrillar balls consisting of Tau protein. Neurofibrillar balls are another pathological sign of Alzheimer’s disease, which is associated with the death of neurons. Several drugs aimed at Tau are at different stages of clinical trials.
3. Gene therapy for spinal muscle atrophy (SMA):
Spinal muscle atrophy (SMA) is a genetic disease that causes progressive muscle weakness and atrophy. Gene therapy is a promising approach to the treatment of SM, which is aimed at restoring the function of the SMN1 gene, the deficiency of which is the cause of the disease.
- Onasemlogen Abekewovek (Zolgensma):
The Semnogen Abarrings is a genetotherapeutic drug that delivers a functional copy of the SMN1 gene to the patient’s cells using an adenoassed virus (AAV). The drug is administered once intravenously and can significantly improve motor functions and survival in children with SMA.
III. Cardiology: Innovation in the treatment of cardiovascular diseases
1. PCSK9 inhibitors: decrease in LDL cholesterol level
PCSK9 inhibitors (Proprotein Convertase Subtilisin/Kexin Type 9) are drugs that reduce the level of low -density lipoproteins (LDL), also known as “bad” cholesterol. PCSK9 is a protein that regulates the number of LDL receptors on the surface of the liver cells. PCSK9 inhibitors block the activity of this protein, increasing the number of LDL receptors and thereby reducing the level of LDL cholesterol in the blood.
Ailirocumab (Praluent) and REPATHA are PCSK9 inhibitors, which are introduced subcutaneously every two weeks or once a month. Clinical tests showed that these drugs effectively reduce LDL cholesterol and reduce the risk of cardiovascular events, such as myocardial infarction and stroke.
2. New anticoagulants: direct oral anticoagulants (SOA)
Direct oral anticoagulants (PAK), also known as new oral anticoagulants (PLA), are drugs that prevent blood clots. Unlike warfarin, which requires regular blood coagulation monitoring, do not require regular monitoring and have less drug interactions.
- Dabigatran (Pradaxa):
Dabigatran is a direct thrombin inhibitor.
- Rivaroxaban (Xarelto):
Rivaroxaban is an inhibitor of the Ha factor.
- Апиксабан (eliquis):
Apixban is an inhibitor of the Ha factor.
- Edoxaban (Savaysa):
Edoxaban is an inhibitor of the Ha factor.
The CACs are used for the prevention and treatment of thromboembolic diseases, such as atrial fibrillation, deep vein thrombosis and pulmonary thromboembolism.
3. Transcatter implantation of the aortic valve (TAVI):
Transcate implantation of the aortic valve (TAVI) is a minimum invasive procedure in which the new aortic valve is implanted through a catheter introduced through the femoral artery or other blood vessel. Tavi is an alternative to surgical replacement of the aortic valve for patients with aortic stenosis, especially for those who have a high risk of complications during a traditional operation.
IV. Infectious diseases: combating resistance to antibiotics and new infections
1. New antibiotics for the treatment of infections resistant to antibiotics:
Antibiotic resistance is a serious global healthcare problem that complicates the treatment of many infectious diseases. The development of new antibiotics, active against bacteria resistant to antibiotics, is a priority.
- Nextolosan / Tazobettam (Zerbaxa):
Ceftolosan/pelvis is a combination of cephalosporin antibiotic ceftolosan and inhibitor of beta-lactamaz tazobactam. The drug is active against many gram-negative bacteria, including Pseudomonas Aeruginosa and Escherichia Coli.
- MONTA / Waborbakam (vabomere):
Meronema/Vaborbaktam is a combination of carbapenem events and inhibitor Karbapenemaz Vabarbaktam. The drug is active against many schematic bacteria producing carbapenemase, including Klebsiella pneumoniae.
- Octamicine (Orbactiv):
Oritamycin is a liplycopeptide antibiotic that is active against many gram-positive bacteria, including Staphylococcus aureus and Enterococcus Faecalis.
2. Antivirus drugs against hepatitis C:
Hepatitis C is a viral liver disease that can lead to cirrhosis and liver cancer. In recent years, new direct antivirus drugs (PPPD) have been developed, which allow you to cure hepatitis C in most patients.
- SOVALDI (SOVALDI):
Sophosbuvir – this inhibitor RNA polymerase NS5B virus hepatita S.
- Daklatasvir (Daklinza):
Daklaratasvir is an inhibitor of the protein NS5A Hepatitis Clus S.
- Ladypasvir (Harvoni):
Ladypasvir is an inhibitor of the protein NS5A Hepatitis Ciluity C.
- Velpatasvir (Epclusa):
Velpatasvir is an inhibitor of a protein NS5A virus hepatitis C.
Combinations of the PPPD, such as Sophosbuvir/Ledipasvir (Harvoni) and Sophosbuvir/Velpatasvir (Epclusa), allow us to achieve a high frequency of hepatitis C cure for most patients, regardless of the genotype of the virus.
3. Vaccines against Covid-19:
The Covid-19 pandemic has led to rapid development and introduction of vaccines against SARS-COV-2, a virus causing COVID-19. Covid-19 vaccines were highly effective in preventing the severe course of the disease, hospitalization and death.
- MRNC-vaccines (Pfizer-Biontech, Moderna):
MRNC-vaccines contain genetic information (MRNA), which encodes the SARS-COV-2 virus protein. After the introduction of the vaccine, the cells of the body begin to produce protein S, which stimulates the immune response.
- Vector vaccines (Astrazeneca, Johnson & Johnson):
Vector vaccines use a modified virus (for example, adenovirus) as a vector for the delivery of genetic information (DNA) of the SARS-COV-2 virus protein to the body cells.
V. General therapy: prospects and challenges
Gene therapy is a revolutionary approach to the treatment of genetic diseases, which is aimed at correcting or replacing defective genes. Gene therapy can be used to treat a wide range of diseases, including hereditary diseases, cancer and infectious diseases.
1. Types of genetic therapy:
- EC Vivo Gene therapy:
With vivo genetic therapy, the patient’s cells are extracted from the body, genetically modified in the laboratory and then introduced back into the body. Car-T-cell therapy is an example of vivo gene therapy.
- In vivo gene therapy:
In in vivo gene therapy, genetic material is introduced directly into the patient’s body, where it is delivered to target cells. The Semnogen Abeparuvets (Zolgensma) is an example of in vivo gene therapy.
2. Genes delivery methods:
- Viral vectors:
Viral vectors, such as adenoassed viruses (AAVs), retroviruses and lentiviruses, are used to deliver genetic material to target cells. Viruses are modified so that they do not cause a disease, but retain the ability to infect cells and deliver genetic material.
- Nevirus vectors:
Nevirus vectors, such as liposomes and plasmids, can also be used to deliver genetic material to target cells. Nevirus vectors, as a rule, are less effective than viral vectors, but they have less side effects.
3. Ethical and regulatory issues:
Gene therapy raises a number of ethical and regulatory issues that must be taken into account when developing and applying it. These include security issues, justice, accessibility and potential long -term consequences.
VI. Regenerative medicine: restoration of damaged tissues and organs
Regenerative medicine is a field of medicine that is engaged in the restoration of damaged tissues and organs. Regenerative medicine uses various approaches, including cell therapy, tissue engineering and stimulation of their own regenerative abilities of the body.
1. Cell therapy:
Cell therapy is a treatment method in which cells are used to restore damaged tissues and organs. Cells can be obtained from the patient’s own body (autologous cells) or from a donor source (allogeneous cells).
- Stem cell therapy:
Stem cells are non -specialized cells that can differentiate into various types of cells. Stem cell therapy can be used to restore damaged tissues, such as the heart muscle, nerve tissue and cartilage.
2. Fabric engineering:
Fabric engineering is a method of creating new tissues and organs in the laboratory. Fabric engineering uses a combination of cells, biomaterials and growth factors to create three -dimensional structures that mimic natural tissues and organs.
3. Stimulation of their own regenerative abilities of the body:
This approach is aimed at stimulating the body’s own regenerative abilities to restore damaged tissues and organs. For example, growth factors and other signal molecules can be used to stimulate tissue regeneration.
VII. Personalized medicine: an individual approach to treatment
Personalized medicine is an approach to treatment in which medical decisions are made on the basis of the individual characteristics of the patient, such as a genetic profile, lifestyle and environmental factors. Personalized medicine allows you to choose the most effective and safe treatment for each patient.
1. Pharmacogenomy:
Pharmacogenomy is a study of the influence of genetic variations on the response to drugs. Pharmacogenomic testing can be used to determine which drug and which dose will be the most effective and safe for a particular patient.
2. Target therapy:
Targeted therapy described in the oncology section is an example of personalized medicine, since the drugs are aimed at specific molecules involved in the growth, development and spread of cancer cells based on the genetic profile of the tumor.
3. Development of drugs based on biomarkers:
Biomarkers are measurable indicators that may indicate the presence of a disease or response to treatment. The development of drugs based on biomarkers allows you to develop drugs aimed at specific mechanisms of the disease and effective for patients with a specific biomarker profile.
VIII. Technologies in pharmaceuticals: artificial intelligence and automation
1. Artificial intelligence (AI) in the development of drugs:
AI is used to analyze large volumes of data, identify potential targets for drugs, predict the effectiveness of drugs and optimize clinical tests.
2. Automation in the production of drugs:
Automation is used to increase efficiency, reduce costs and improve the quality of drug production. Robotized systems can be used to perform various tasks, such as dosing, mixing and packaging drugs.
3. 3D printing of drugs:
3D printing allows you to create drugs with individual dosage and form. This approach can be especially useful for patients who need to take several drugs at the same time or for patients with difficulties when swallowing tablets.
IX. Regulation and accessibility of drugs: global calls
1. Harmonization of regulatory requirements:
Harmonization of regulatory requirements in different countries can simplify the process of developing and registering drugs, which will accelerate their access to the market.
2. Ensuring the availability of drugs:
Ensuring the availability of drugs, especially in countries with low and average income, is an important task. It is necessary to develop strategies that will reduce the cost of drugs and ensure their availability for all patients in need of treatment.
3. The fight against fake drugs:
The fight against fake drugs is an important task, since fake drugs can be ineffective and even dangerous to health. It is necessary to strengthen control over the production and distribution of drugs and take measures to prevent the spread of fake drugs.
X. Future of pharmaceuticals: new horizons
1. Nanomedicine:
Nanomedicine is the use of nanotechnologies in medicine. Nanoparticles can be used to deliver drugs directly to the target cells, for the diagnosis of diseases and to create new medical materials.
2. Biopeting of organs and tissues:
Biopeting is a technology that allows you to create three -dimensional biological structures, such as organs and tissues, using cells and biomaterials. Biopeting of organs and tissues can be used for transplantation, for testing drugs and to study biological processes.
3. Artificial intelligence in diagnosis and treatment:
AI will play an increasingly important role in the diagnosis and treatment of diseases. AI can be used to analyze medical images to predict the risk of developing diseases and to select the most effective treatment for each patient.