Pharmaceutical News: New Medications and Development

Pharmaceutical News: New Medications and Development

I. Oncology: revolutionary therapy and personalized approach

1. Targeted therapy: advanced line of defense against cancer. Targeted therapy is a fundamentally new approach to cancer treatment aimed at blocking specific molecules involved in the growth and spread of the tumor. Unlike traditional chemotherapy, which affects all rapidly dividing cells, targeted drugs have high selectivity, which can minimize side effects and increase the effectiveness of treatment.

   • Tyrosinkinaz inhibitors (TKI): These drugs block the activity of tyrosinkinase, enzymes that play a key role in transmitting growth signals and survival in cancer cells. For example, Imatinib (Glute) made a revolution in the treatment of chronic myelolecosis (HML), and Gephitinib (Isses) and Erlotinib (Tartseva) are successfully used with non -cellular lung cancer (NMRL) with mutations in the EGFR gene.
   • BRAF and MEK inhibitors: These drugs are used to treat melanoma with the Braf V600E mutation, which occurs in about half of the patients. BRAF inhibitors (for example, vemorafenib, dabrafenib) block the activity of the BRAF mutant protein, and MEK inhibitors (for example, trametinib, Kobimetinib) block the activity of the MEK protein, which is below BRAF in the signaling pathway. The combination of BRAF and MEK inhibitors showed higher efficiency and lower toxicity compared to monotherapy.
   • PARP inhibitors: These drugs block the activity of the PARP enzyme, which is involved in the restoration of DNA damage in cancer cells. PARP inhibitors (for example, Olaparib, Rhparib, Talazoparib) are especially effective in the treatment of ovarian cancer, breast cancer and prostate cancer with mutations in BRCA1 and BRCA2 genes.
   • CDK4/6 inhibitors: These drugs block the activity of cyclin-dependent kinase 4 and 6 (CDK4/6), which play a key role in the regulation of the cell cycle. CDK4/6 inhibitors (for example, Palbocyclib, Ribocyclib, Abemaclicclib) are used in combination with hormonal therapy for the treatment of hormone-positive, Her2-Negative cancer of the mammary gland.

2. Immunotherapy: activation of the immune system to combat cancer. Immunotherapy is a promising direction in the treatment of cancer aimed at activating the patient’s immune system to combat the tumor. Immunotherapeutic drugs do not attack cancer cells directly, but help the immune system recognize and destroy them.

   • Immune checkpoint incibitors): These drugs block proteins (for example, PD-1, PD-L1, CTLA-4), which inhibit the activity of immune cells. Blocking these proteins allows immune cells to more effectively attack cancer cells. Control points inhibitors (for example, pembrolizumab, nivolumab, Ipilimumab) showed high efficiency in the treatment of various types of cancer, including melanoma, lung cancer, kidney cancer, bladder cancer and Hodgkin lymphoma.
   • Car-T-cell therapy: This type of immunotherapy involves the genetic modification of the patient’s T-lymphocytes for recognition and destruction of cancer cells. T-lymphocytes are taken from the patient, modified in the laboratory for the expression of the chime-dimensional antigenic receptor (CAR), which specifically binds to protein on the surface of cancer cells, and then returned to the patient. Car-T-cell therapy showed high efficiency in the treatment of some types of leukemia and lymphoma.
   • Oncolytic viruses: These viruses are genetically modified for selective infection and destruction of cancer cells, without affecting healthy cells. In addition, oncolytic viruses can stimulate the patient’s immune system to combat the tumor.

3. Personalized medicine: an individual approach to cancer treatment. Personalized medicine involves the choice of the most effective treatment for each patient based on the genetic, molecular and clinical characteristics of his tumor.

   • Genomatic tumor profiling: This method allows you to determine the presence of mutations, amplification and delections in the genes of cancer cells. Information obtained as a result of genomic profiling can be used to select targeted therapy, immunotherapy or other treatment methods that are most effective for a particular patient.
   • Biomarkers: Biomarkers are measurable parameters that may indicate the presence of cancer, stage of the disease, forecast and response to treatment. Examples of biomarkers include the level of certain proteins in the blood, the presence of mutations in the genes of cancer cells and the expression of certain genes.
   • Liquid biopsy: This method allows you to obtain the genetic material of cancer cells from the patient’s blood. Liquid biopsy can be used to monitor the response to treatment, detect resistance to therapy and early detection of the relapse of the disease.

II. Neurology: Breakthrough in the treatment of neurodegenerative diseases and pain syndromes

1. Alzheimer’s disease: new hopes for a slowdown in the progression of the disease. Alzheimer’s disease (BA) is the most common form of dementia, characterized by a progressive loss of memory and cognitive functions. Despite numerous studies, until recently, there were no drugs that could slow down the progression of BA.

   • Anti-amyloid antibodies: These drugs (for example, adukanumab, healer) are designed to remove amyloid plaques from the brain, which are considered one of the key pathological signs of BA. Aduumab was approved by FDA in 2021, and Lekanemab was approved in 2023, both drugs showed the ability to slow down a decrease in cognitive functions in patients with an early stage of BA. However, anti-amyloid antibodies can cause side effects, such as ARIA (Amyloid-RELEETED IMAGING ABNORMALITIS), which require careful monitoring.
   • Bace inhibitors: These drugs block the Bace enzyme, which is involved in the formation of amyloid. Several Bace inhibitors are at the clinical test stage.
   • Microglia modulators: Microglia is the immune cells of the brain that can play both protective and harmful role in BA. Microglia modulators are developed to stimulate the protective function of microglia and suppress inflammation in the brain.

2. Parkinson’s disease: improving symptoms control and slowing down the progression of the disease. Parkinson’s disease (PSU) is a neurodegenerative disease characterized by tremor, rigidity, slowdown in movements and postural instability. The main cause of BP is the death of dopamine neurons in the black substance of the brain.

   • Gene therapy: Gene therapy involves the introduction of genes that can compensate for the deficiency of dopamine in the brain. For example, the drug of the glial cellular line of the neurotrophic factor (GDNF) is at the stage of clinical tests and showed the potential in improving motor functions in patients with PSU.
   • Immunotherapy: Immunotherapy is developed to remove alpha synuclein units from the brain, which are considered one of the key pathological signs of BP.
   • Alfa Sinuclein inhibitors: These drugs block the formation of alpha-synuclein aggregates.

3. Migraine: new methods of preventing and stopping attacks. Migraine is a common neurological disease characterized by severe headache, often accompanied by nausea, vomiting and photophobia.

   • Anti-CGRP antibodies: These preparations (for example, Erenumab, Fremanesumab, Galkanzumab) block calcitonin-gene-related peptide (CGRP) or its receptor. CGRP plays a key role in the development of migraine. Anti-CGRP antibodies showed high efficiency in migraine prevention.
   • Hever: These drugs (for example, Riemegepant, Arrogupant) are CGRP receptor antagonists and are used to stop migraine attacks.
   • Ditan: This drug is a selective agonist of the 5-HT1F receptor and is used to stop migraine attacks.

4. Chronic pain: development of non-opioid analgesics. Chronic pain is a serious problem that can significantly worsen the quality of the patient’s life. Opioid analgesics are widely used to treat chronic pain, but they are associated with the risk of dependence and side effects. The development of non-opioid analgesics is a priority task.

   • Inhibiters NGF: Nerves growth factor (NGF) plays a key role in the development of chronic pain. NGF inhibitors (for example, Tanezumab) are at the stage of clinical trials and showed the potential in the treatment of chronic pain.
   • Nav1.7 inhibitors: NAV1.7 is a voltage-dependent sodium channel that plays a key role in the transmission of pain signals. Nav1.7 inhibitors are developed for the treatment of chronic pain.

III. Cardiology: new drugs for the treatment of heart failure and atherosclerosis

1. Heart failure: improving the survival and quality of life of patients. Cardiac failure (SN) is a condition in which the heart cannot effectively pump blood to satisfy the needs of the body.

   • SGLT2 inhibitors: These drugs (for example, dapagliflosin, empagliflosine) were originally developed for the treatment of type 2 diabetes, but later it was established that they are also effective in the treatment of SH. SGLT2 inhibitors reduce the risk of hospitalization for SN and improve the survival of patients with SN.
   • Soluble guanilateziclase stimulants (SGC): These drugs (for example, Vericiguat) stimulate the SGC enzyme, which plays a key role in the regulation of vascular tone. SGC stimulants reduce the risk of hospitalization for SN and improve the survival of patients with CH.

2. Atherosclerosis: reducing the risk of cardiovascular events. Atherosclerosis is a disease in which cholesterol and other substances accumulate on the walls of arteries, forming plaques. Atherosclerosis is the main cause of cardiovascular disease, such as myocardial infarction and stroke.

   • PCSK9 inhibitors: These drugs (for example, Evolocumab, Ailirocumab) block the PCSK9 protein, which is involved in the regulation of LDL cholesterol (low density lipoproteins) in the blood. PCSK9 inhibitors reduce LDL cholesterol and the risk of cardiovascular events.
   • Incalisiran: This drug is a small interpherging RNA (Sirna), which blocks the synthesis of PCSK9 protein in the liver. Incalisiran reduces the level of LDL cholesterol and the risk of cardiovascular events.
   • Bempedoic acid: This drug blocks the enzyme involved in the synthesis of cholesterol in the liver. Bempedoic acid reduces the level of LDL cholesterol and the risk of cardiovascular events.

3. Atrial fibrillation: new anticoagulants for the prevention of stroke. Atrial fibrillation (FP) is a violation of the heart rhythm that increases the risk of blood clots in the heart. Cloths can come off and get into the brain, causing a stroke.

   • Direct oral anticoagulants (SOA): These drugs (for example, Dabigatran, Rivaroxaban, Apixban, Edoxaban) are an alternative to warfarin for the prevention of a stroke at FP. They have a more predictable pharmacokinetic profile and do not require regular monitoring of many (international normalized attitude).

IV. Infectious diseases: the fight against antibiotic resistance and the development of vaccines

1. Antibiotic resistance: development of new antibiotics and alternative treatment methods. Antibiotic resistance is a serious problem that threatens global healthcare. Many bacteria have become resistant to most available antibiotics.

   • New antibiotics: New antibiotics are being developed that are effective against stable bacteria. Examples of new antibiotics include ceftolosan/pelvis, ceftasim/avibactam and meropenem/vibobact.
   • Phagic therapy: Fagas are viruses that infect and kill bacteria. Phagic therapy is the use of phages to treat bacterial infections. Phagic therapy can be effective against stable bacteria.
   • Antimicrobial peptides: Antimicrobial peptides are small proteins that have antibacterial activity. Antimicrobial peptides are developed for the treatment of bacterial infections.

2. HIV/AIDS: Development of new drugs and treatment strategies. HIV/AIDS is an infectious disease caused by human immunodeficiency virus (HIV). HIV affects the immune system, making a person susceptible to various infections and cancer.

   • Long -acting drugs: Long -acting drugs for HIV treatment are developed, which can be administered less often than ordinary drugs. Examples of long -acting drugs include Cabotegvir and Rilpivirin.
   • HIV vaccine: The development of an effective HIV vaccine is a priority. Several HIV vaccines are at the clinical testing stage.
   • HIV cure: Researchers are actively working on the development of HIV cure strategies.

3. Hepatitis C: Development of highly effective drugs for treatment. Hepatitis C is an infectious disease caused by hepatitis C (HCC) virus. Higher School of Economics affects the liver and can lead to cirrhosis and liver cancer.

   • Direct antiviral drugs (PPPD): PPPD (for example, Sophosbuvir, Ladypasvir, Velpatasvir, Gleps, Pibrentasvir) showed high efficiency in the treatment of hepatitis C. PPPD can cure hepatitis C in most patients.

4. COVID-19: Development of vaccines and drugs for treatment. Covid-19 is an infectious disease caused by the SARS-COV-2 virus.

   • Vaccines against Covid-19: Vaccines against Covid-19 (for example, MRNA-based vaccines, vector vaccines, inactivated vaccines) have been developed and widely used. Covid-19 vaccines effectively prevent a severe course of the disease, hospitalization and death.
   • Antiviral drugs against Covid-19: Antiviral drugs for the treatment of COVID-19 have been developed (for example, Molnupiravir, Nirmatrelevir/Ritonavir). These drugs can reduce the risk of hospitalization and death in patients with COVID-19.

V. Diabetes: new approaches to controlling blood sugar and preventing complications

1. Type 1 diabetes: development of artificial pancreas. Type 1 diabetes (CD1) is an autoimmune disease in which the immune system destroys the insulin-producing pancreatic cells. People with SD1 need lifelong insulin therapy.

   • Artificial pancreas (Artificial Pancreas): Artificial pancreas is a device that automatically measures blood sugar and delivers insulin when necessary. Artificial pancreas can help people with SD1 better control the blood sugar level and prevent diabetes.

2. Type 2 diabetes: Development of new drugs to reduce blood sugar and organs protection. Type 2 diabetes (CD2) is a disease in which the body cannot effectively use insulin. SD2 is the most common form of diabetes.

   • GLP-1 agonists: These drugs (for example, Semaglutide, Tyrzepatide) stimulate the release of insulin from the pancreas and reduce blood sugar. GLP-1 agonists can also help reduce weight and protect the heart and kidneys.
   • SGLT2 inhibitors: These drugs (for example, dapagliflosin, empagliflosine) block the reabsorption of glucose in the kidneys, which leads to a decrease in blood sugar. SGLT2 inhibitors can also protect the heart and kidneys.

3. Prevention of diabetes complications: Development of drugs for protecting kidneys, eyes and nerves. Diabetes can lead to various complications, including kidney damage (diabetic nephropathy), eye damage (diabetic retinopathy) and nerves damage (diabetic neuropathy).

   • FinenoN: This drug is a selective non -steroidal antagonist of a mineralocorticoid receptor and is used to treat diabetic nephropathy. Finenerenon reduces the risk of progression of diabetic nephropathy and cardiovascular events.

VI. Rare diseases: search for drugs for orphan diseases

1. Stimulating the development of drugs for rare diseases. Rare diseases (orphan diseases) are diseases that are rare in a population. The development of drugs for rare diseases is often disadvantageous for pharmaceutical companies, so governments and charitable organizations support the development of such drugs.
2. Gene therapy for rare diseases. Gene therapy is a promising method of treating rare diseases that are caused by mutations in one gene. Examples of genetic therapy for rare diseases include a seminogen Abparvent (Zolgensma) for the treatment of spinal muscle atrophy (SMA) and KOKSTORZA (Elevidys) for the treatment of Duchenne’s muscle dystrophy.

VII. Psychiatry: new drugs for the treatment of depression and schizophrenia

1. Depression: Development of antidepressants with a new mechanism of action. Depression is a common mental disorder, characterized by a decrease in mood, loss of interest in life and fatigue.

   • Equay: This drug is an antagonist of NMDA receptors and is used to treat depression resistant. Squetamine is administered intravenously or intranasally.
   • Aurora: This drug is an inhibitor of monoaminoxidase (MAO) type A and is used to treat depression.

2. Schizophrenia: development of antipsychotics with fewer side effects. Schizophrenia is a chronic mental disorder characterized by hallucinations, delirium, violation of thinking and social insulation.

   • Lumatepe: This drug is an atypical antipsychotic with a new action mechanism. Lumateperon has fewer side effects compared to other antipsychotics.
   • Kariprazin: This drug is an atypical antipsychotic, which has partial agonism to dopamine D3 and D2 receptors and antagonism to serotonin 5-HT1A receptors. Carprazine is effective in the treatment of negative symptoms of schizophrenia.

VIII. Pregnancy and childbirth: new drugs to prevent premature birth and treatment of postpartum depression

1. Premature birth: the development of drugs for extension of pregnancy. Premature birth is childbirth that occurs before 37 weeks of pregnancy. Premature birth can lead to various complications in a newborn.

2. Postpartum depression: development of drugs for treatment. Postpartum depression is a depression that develops after childbirth.

   • BREKSANOLON: This drug is neurosteroid and is used to treat postpartum depression. Brexanolon is administered intravenously.

IX. Dermatology: new drugs for the treatment of psoriasis and atopic dermatitis

1. Psoriasis: development of targeted drugs. Psoriasis is a chronic autoimmune skin disease characterized by the appearance of red, peeling plaques.

   • IL-17 inhibitors: These drugs (for example, secenumab, Xekyzumab, Brodalimab) are blocked by Interleukin-17 (IL-17), cytokine, which plays a key role in the development of psoriasis. IL-17 inhibitors are very effective in the treatment of psoriasis.
   • IL-23 inhibitors: These drugs (for example, Guselkumab, Tildrakizumab, Risaninkizumab) block Interleukin-23 (IL-23), cytokine, which plays a key role in the development of psoriasis. IL-23 inhibitors are very effective in the treatment of psoriasis.
   • TYK2 inhibitors: This drug (derakravACITINIB) blocks tyrosinkinase 2 (TYK2), which is involved in the signal routes IL-23, IL-12 and IFN type I. DelakeAcitinib is an oral drug for the treatment of psoriasis.

2. Atopic dermatitis: development of targeted drugs. Atopic dermatitis is a chronic inflammatory skin disease characterized by itching, redness and dry skin.

   • IL-4/IL-13 inhibitors: This drug (Dupilumab) blocks Interleukin-4 (IL-4) and Interlayykin-13 (IL-13), cytokines, which play a key role in the development of atopic dermatitis. Dupilumab is very effective in the treatment of atopic dermatitis.
   • JAK inhibitors: These drugs (for example, Topacitinib, Baricitinib, Padadacitinib) block Janus-kinase (JAK), enzymes that participate in the transmission of signals from various cytokines, including IL-4, IL-13, IL-31 and TSLP. JAK inhibitors are effective in the treatment of atopic dermatitis.

X. ophthalmology: new drugs for the treatment of age -related macular degeneration and diabetic retinopathy

1. Age macular degeneration (VMD): Development of drugs for inhibiting angiogenesis. Age macular degeneration (VMD) is a disease of the eye, which leads to the loss of central vision. The wet form of the VMD is characterized by an anomalous growth of blood vessels in the retina (angiogenesis).

   • VEGF inhibitors: These drugs (for example, Ranibizumab, Aflibert, Bevacizumab) block the vascular endothelium factor (VEGF), a protein that stimulates angiogenesis. VEGF inhibitors are introduced into the eye in the form of injections and are used to treat a wet form of VMD.

2. Diabetic retinopathy: development of drugs for inhibiting angiogenesis. Diabetic retinopathy is a disease of the eyes that develops in people with diabetes. Diabetic retinopathy can lead to blindness.

   • VEGF inhibitors: VEGF inhibitors (for example, Ranibizumab, Afliebercept, Bevacizumab) are also used to treat diabetic retinopathy.

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