Adv lec2


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  • Proteins can be classified according to a wide range of functions in the cell. For examples, antibodies act in defense of the cell. They bind to specific foreign molecules, such as viruses and bacteria, to help protect the cell. Enzymes perform thousands of chemical reactions. Regulator proteins control biological processes within cells, tissues, and organs. Many proteins, like keratin and actin, provide structure, support, and range of motion. Transport and storage proteins, like hemoglobin, bind and carry atoms and small molecules within cells and throughout the body .
  • Erythropoietin , also known as erythropoetin or erthropoyetin or EPO , is a glycoprotein hormone that controls erythropoiesis, or red blood cell production. It is a cytokine (protein signaling molecule) for erythrocyte (red blood cell) precursors in the bone marrow.
  • التصلب المتعدد هو مرض التهابي مزمن , يصيب الجهاز العصبي المركزي (المخ والحبل الشوكي) وذلك بسبب قيام الجهاز المناعي للجسم بمهاجمة أنسجته الخاصة فيما يعرف بالمناعة الذاتيه ( autoimmunity ) , وهنا يقوم بتدمير غشاء الميلين ( myelin sheath ) الذي يغلف معظم الخلايا العصيبة في المخ,مما يسبب تكون بقع(لويحات)من المناطق الخالية من الميلين ( plaques of demyelination ) مما يتسبب بظهور أعراض المرض والاعاقة فيما بعد.
  • RRMS is characterised by relapses (also known as exacerbations) during which time new symptoms can appear and old ones resurface or worsen. relapsing-remitting MS: The relapses are followed by periods of remission, during which time the person fully or partially recovers from the deficits acquired during the relapse.
  • Metastatic cancer is a cancer that has spread from the part of the body where it started (the primary site) to other parts of the body. a lymphokine-activated killer cell (also known as a LAK cell ) is a white blood cell that has been stimulated to kill tumour cells. If lymphocytes are cultured in the presence of Interleukin 2, it results in the development of effector cells which are cytotoxic to tumour cells The NK cells targets "non-self", i.e. cells that are not an integral part of you (or your "self").  The mechanism of LAK cells is distinctive from that of natural killer cells because they can lyse cells that NK cells cannot. [4] LAK cells are also capable of acting against cells that do not display the major histocompatibility complex, as has been shown by the ability to cause lysis in non-immunogenic, allogeneic and syngeneic tumors. [4] LAK cells are specific to tumor cells and to not display activity against norma
  • . Turner syndrome: A chromosome disorder in females that is characterized by the absence of all or part of a second sex chromosome in some or all cells  webbing of the neck (due to congenital lymphedema), short stature, delayed growth of the skeleton, shortened fourth and fifth fingers, broad chest, cardivascular abnormalities, and gonadal dysgeneesis. Women with Turner syndrome are usually infertile due to ovarian failure.
  • Sepsis is a potentially deadly medical condition characterized by a whole-body inflammatory state (called a systemic inflammatory response syndrome or SIRS) that is triggered by an infection. The body may develop this inflammatory response by the immune system to microbes in the blood, urine, lungs, skin, or other tissues. A lay term for sepsis is blood poisoning , also used to describe septicaemia. Severe sepsis is the systemic inflammatory response, infection and the presence of organ dysfunction.
  • Anabolic therapy: which bone formation is directly stimulated.
  • Incretins are a group of gastrointestinal hormones that cause an increase in the amount of insulin released from the beta cells of the islets of Langerhans after eating, even before blood glucose levels become elevated.
  • Cervical dysplasia refers to abnormal changes in the cells on the surface of the cervix that are seen underneath a microscope.
  • Adv lec2

    1. 1. Advanced Pharmaceutical Biotechnology Phar571 Lec. 2 Dr. Yara Al Tall JUST / Faculty of Pharmacy
    2. 2. Pharmaceuticals and Biotechnology- Benefits of CombinationWhen the two disciplines- pharmaceuticals and biotechnology- come together, they result in many advantages for humankind in terms of healthcare.This is possible through Pharmacogenomics (derived from pharmacology and genomics) which refers to the study of how the genetic inheritance affects individual human bodys response to drugs.
    3. 3. Biopharmaceutical drugs aim at designing and producing drugs that are adapted to each person’s genetic makeup.Thus pharmaceutical biotechnology companies may develop tailor-made medicines for maximum therapeutic effects.
    4. 4. Also, biotechnology drugs can be given to the patients in appropriate dosages as the doctor would know the patient’s genetics and how the body processes and metabolizes a medicine.One more benefit of pharmaceutical biotechnology is in the form of better vaccines. Biotech companies design and produce safer vaccines by organisms that are transformed through genetic engineering. These biotech vaccines minimize the risks of infection.
    5. 5. Pharmaceutical Biotechnology ProductsThe common pharmaceutical biotechnology products:1. Antibodies- Antibodies are proteins that are produced by white blood cells and are used by the immune system to identify bacteria, viruses, and other foreign substances and to fight them off.  In the recent years, monoclonal antibodies are one of the most exciting developments in biotechnology pharmaceuticals.
    6. 6. 2. Proteins - Proteins made of amino acids are large, complex molecules that do most of the work in cells and are required for the structure, function, and regulation of the body’s tissues and organs.
    7. 7. Proteins do most of the work in the cell Enzyme Protease Transport Degrades Protein Hemoglobin Motion Carries O2 ActinContracts Muscles Regulation Insulin Controls Blood Glucose Support Keratin Defense Forms Hair and Antibody Nails Fights Viruses
    8. 8.  Protein biotechnology is emerging as one of the key technologies of the future for understanding the development of many diseases like cancer or sickle cell anemia.
    9. 9. 3. Recombinant DNA Products - Recombinant Deoxyribonucleic Acid is the genetically engineered DNA created by recombining fragments of DNA from different organisms. Some of the Recombinant DNA Products include:A. Recombinant DNA VaccinesB. Recombinant DNA DrugsC. Recombinant DNA EnzymesD. Recombinant DNA Growth HormoneE. Recombinant DNA InsulinF. Recombinant DNA ProteinsG. Recombinant DNA Yeast
    10. 10. Biotechs First Wave: Recombinant DNA DrugsIn this section we will focus mainly on Drug Biotech companies (started as biotech firms) rather than the "Big Pharma" companies (Pfizer, J&J, Merck, Abbott, Bayer, Roche, etc) - many of which license biotech drugs and may even have developed a few of their own (Lilly is an exception).The following is a list for many of the best rDNA drugs on the market today classified according to their companies:
    11. 11. The best rDNA drugs on the market today1. Amgen:A. Epogen/Procrit: This drug produces rErythropoetin.   Epo binds the Epo receptor (Epo R) on bone marrow erythroid progenitors, inducing proliferation, maturation, and differentiation of red blood cells.
    12. 12. Figure 1. Erythropoietin (Epo)binds its receptor (Epo R) on thesurface of red blood cellprogenitors in the bone marrow.It causes proliferation,maturation, and differentiation,thereby preventing or correctinganemia.Epo may also bind Epo Rexpressed on the surface ofcancer cells and may elicit tumorgrowth via cell proliferation,protection from apoptosis, and/orangiogenesis. [Note: figureadapted from Brower, V. (2003)Nat. Med. 9:1439.]
    13. 13.  why Epogen/Procrit are prescribed and what they do in the body? For patients with anemia due to Dialysis/ Chronic Kidney Disease / Renal Failure / Chemo / HIV Stimulates production of RBCs. Truly revolutionized treatment of anemia! Epo: For patients on dialysis with anemia. Procrit: For non-dialysis use only = Cancer, Chronic Kidney Disease, HIV (anemia due to Zidovudine treatment AZT); some blood transfusions.
    14. 14. B. Aranesp This drug also produces rErythropoetin. Uses: For patients with anemia due to Dialysis/ Chronic Kidney Disease / Renal Failure / Chemo It has a longer lasting effect (3X greater that Epo), requires fewer injections - 1 shot every 3 weeks, rather than once/week.
    15. 15. C. Neulasta rGranulocyte colony-stimulating factor (G-CSF) G-CSFs are glycoprotein cytokine hormones that stimulate proliferation and growth of granulocytes (A type of WBC filled with microscopic granules that are little sacs containing enzymes) Uses: For Neutropenia: low WBC count febrile neutropenia (low WBC count with fever/ infection) due to chemo, bone marrow transplant (BMT), Acute myeloid leukemia (AML).
    16. 16. D. Infergen: rInterferon alpha. Question: What are interferons? Interferons (IFNs) are proteins made and released by host cells in response to the presence of pathogens—such as viruses, bacteria, or parasites—or tumor cells. They allow communication between cells to trigger the protective defenses of the immune system that eradicate pathogens or tumors.
    17. 17.  "Noone knows exactly how interferons work”! UsesInfergen is used for patients with Chronic, non-responding, or relapsing hepatitis C viral (HCV) infection.
    18. 18. 2. Biogen This is another biotech company which has released the following drugs:A. Avonex It is rInterferon beta-1a: it "Calms" or down- regulates the immune system Made in Chinese hamster ovary (CHO) cells.
    19. 19.  Uses:Treatment of relapsing forms of Multiple Sclerosis (MS). Slows the progression of MS by regulating the bodys immune response against myelin.Given as an IM injection once per week.
    20. 20. Q.Anyone knows Multiple Sclerosis ‫بلصتلالتصلب‬ ‫?بلصتلالمتعدد‬ Multiple Sclerosis (MS) is an inflammatory disease of the Central Nervous System (CNS) -brain and spinal cord. It is a disease of the "white matter" tissue. The white matter is made up of nerve fibers which are responsible for transmitting communication signals both internally within the CNS and between the CNS and the nerves supplying rest of the body.
    21. 21. For more information on Multiple Sclerosis, please have a look at the following website:
    22. 22. B. Intron A It is rInterferon alpha-2b Uses: Over 20 indications!i. Malignant melanoma Salesii. Non-Hodgkins lymphoma exceedingiii. Hairy cell leukemia $440 million in !iv. Kaposis sarcoma three yearsv. Chronic hepatitis B (HBV)vi. Chronic hepatitis C (HCV)
    23. 23. 3. Engerix-B/Recombivax: They are a rHepatitis B vaccine Uses: Infection with HBV prevention of 1° liver cancer. The global HBV market exceeds $1 billion dollars annually. It will grow as more countries adopt WHO recommendations for the vaccination of newborns, teenagers, healthcare workers and other at-risk populations. "
    24. 24. 4. Amevive: It is a protein which blocks the growth of T-cells, and acts as an immunosuppressant. The exact mechanism of action is complicated, however, the T-cell activity is weakened, which reduces the symptoms of psoriasis and the inflammation that is involved in it. Uses: Moderate-to-severe chronic plaque psoriasis Suppresses overactive T lymphocytes found in autoimmune diseases
    25. 25. 3. Chiron ( a Novartis partnership)A. Betaseron: It is rInterferon beta-1b. Uses: Multiple Sclerosis: Significantly delays the progression of secondary MS, including relapsing-remitting MS. 25
    26. 26. B. Proleukin It is Interleukin-2 - IL-2 Uses: Cancer: Metastatic renal cell carcinoma, and metastatic melanoma. IL-2 activates lymphokine-activated killer (LAK) cells, Natural killer cells (NK) that normally destroy tumor cells. 26
    27. 27. MOA: IL-2 activates the immune system in several ways, but the major one is to stimulate T cell and natural killer (NK) cell proliferation, increasing and activating these immune system cells to find and destroy cancer cells. 27
    28. 28. 28
    29. 29. C. Regranex: It is rPlatelet-derived growth factor (PDGF). Uses: Treatment of diabetic foot ulcers. PDGF is a cytokine (growth factor) that stimulates skin cell and blood vessel production 29
    30. 30. PDGF is involved in developing protective tissue and skin after a wound or ulcer (a process called granulation) >>>>>However, FDA has concluded that patients who use three or more tubes of Regranex experience a five-fold increase in the risk of cancer death compared to patients who do not use the drug. 30
    31. 31. 4. GenentechA. CathFlo Activase: It is rTPA, tissue-plasminogen activator. Uses: Thrombolytic: Approved for treatment of acute myocardial infarction (AMI), cardiac ischemia, acute massive pulmonary embolism, and management of stroke. 31
    32. 32. Converts plasminogen to plasmin, which activates fibrin, breaking down blood clot. Clot dissolution ***FDP: FIBRIN DEGRADATION PRODUCTS 32
    33. 33. B. TNKase rTPA, tissue-plasminogen activator TNKase can be administered over five seconds in a single dose, fastest administration of any thrombolytic Uses: Thrombolytic: TNKase is bioengineered with 3 amino acid substitutions from natural TPA: T, N and K (Thr, Asp, Lys).=Increased Fibrin Specificity=Long Plasma Half-Life=Greater Resistance to Plasminogen Activator Inhibitor-1 33
    34. 34. 34
    35. 35. C. Pulmozyme It is a rDeoxyribonuclease I (rhDNase) Uses: For management of cystic fibrosis in children 3 months - 5 years old. Inhalation Solution – Pulmozyme hydrolyzes the DNA in sputum and airways of CF patients. 35
    36. 36.  MOA: In CF patients, lungs and airways become clogged with mucous containing high concentrations of extracellular DNA released by degenerating leukocytes (neutrophils).* Watch this! works.jsp 36
    37. 37. D. Neutropin It is a rHuman growth hormone (Somatropin; synthetic growth hormone) Uses: Treatment of GHD (growth hormone deficiency) in children and in adults Identical to pituitary-derived hGH. Also indicated for growth failure associated with chronic renal insufficiency (CRI) prior to kidney transplantation, and short stature associated with Turner syndrome. 37
    38. 38. 5. The 5th company is: Eli Lilly: WorldwideA. Humulin: revenues of $2.5 B It is rInsulin annually Uses: Diabetes: Used by over 3.5 million people in the U.S. every day 38
    39. 39. 1: Humulin pens2: Humalog: Humalog is the brand name of insulin lispro  which is a rapid-acting insulin.3: Humalog Mix (Biphasic insulin) Rapid acting + longer acting insulin : mixture of insulin lispro and insulin lispro protamine. 39
    40. 40. Lispro (Humalog ®) Eli lilly Start < 15 min.Rapid-acting Peak 30-60min Aspart (Novorapid®) Novo NordiskShort-acting Humulin ® R Eli lilly Start 30-60(Regular: identical to min.human insulin= Peak 4 hr Novolin® R Novo Nordiskunmodified)Intermediate-acting Humulin ® N Eli lilly Start 1.5 hrs Peak 7 hr(Isophane insulin: Novolin® N Novo Nordiskinsulin+protamine+zinc Humulin® U: withdrawn Eli lilly Start 1 hr. from market Peakless Glargine (Lantus®) Sanofi-Aventis cannot be combinedLong-acting with other insulins Detemir (Levemir®) Novo Nordisk
    41. 41. 41
    42. 42. B. Humatrope rHuman growth hormone (hGH) (Somatropin) Uses: For Somatropin Deficiency Syndrome (SDS) in adults and GHD in children due to hypopituitarism, a pituitary tumor or other pituitary disorder, or Turner Syndrome. 42
    43. 43. C.XigrisIt is rActivated Protein C. Activated Protein C acts inside the blood vessel as an anticoagulant/antithrombotic - reduces blood clots; controls inflammation. Activated by thrombin. 43
    44. 44.  Uses:Treatment of severe sepsis, a fast- moving, dramatic, and often fatal acute response to infection that claimed 215,000 lives each year (pre- Xigris) 44
    45. 45. D. Forteo It is rParathyroid hormone. Uses: Treatment of osteoporosis in women and men - Anabolic Therapy: stimulates new bone formation, osteoblasts, bone mineral density (BMD) and bone strength. 45
    46. 46. E. Byetta It is rGLP-1 (glucagon-like peptide-1) Uses: Type II Diabetes: regulates glucose homeostasis and enhances glucose- dependent insulin secretion. 46
    47. 47.  Byetta is an incretin mimetic similar to GLP-1, an incretin hormone that increases insulin release & decreases glucagon secretion from the pancreas; increases satiety/ fullness 47
    48. 48.  -ve:twice a day injection, but "an appropriate option to consider when Type II patients cannot control their blood sugar using oral medications." +ve: Steady, significant appetite suppression and weight loss in overweight diabetic patients 48
    49. 49. 6. Merck One drug from Merck : Gardasil: It is rQuadrivalent HPV (human papilloma virus) types 6, 11, 16, 18 L1 capsid protein of all 4 viruses made individually in yeast cells and combined into one vaccine. 49
    50. 50.  Uses: First Cancer Vaccine: Approved for the immunization of: children aged 9 to 15 years adult females aged 16 to 26 yearsUsed for the prevention of cervical cancer, high-grade cervical dysplasia (abnormality in development), and warts caused by HPVs types 6, 11, 16 and 18 50
    51. 51. Virtually 100% effective in protecting against the HPV-16 and HPV-18 strains.-ve: The wholesale price for Gardasil will be $120 per dose; $360 for all three doses. 51