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Advanced Pharmaceutical
 Biotechnology Phar571
         Lec. 2
          Dr. Yara Al Tall
    JUST / Faculty of Pharmacy
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 body's
 response to drugs.
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.
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.
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.
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.
Proteins do most of the work in
                      the cell
            Enzyme
            Protease
                                                 Transport
            Degrades Protein
                                                 Hemoglobin
 Motion                                          Carries O2
  Actin
Contracts Muscles
                        Regulation
                        Insulin
                        Controls Blood Glucose
                                                      Support
                                                      Keratin
                            Defense               Forms Hair and
                           Antibody                   Nails
                          Fights Viruses
   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.
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 Vaccines
B. Recombinant DNA Drugs
C. Recombinant DNA Enzymes
D. Recombinant DNA Growth Hormone
E. Recombinant DNA Insulin
F. Recombinant DNA Proteins
G. Recombinant DNA Yeast
Biotech's 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:
The best rDNA drugs on the market today

1. 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.
Figure 1. Erythropoietin (Epo)
binds its receptor (Epo R) on the
surface of red blood cell
progenitors in the bone marrow.
It       causes      proliferation,
maturation, and differentiation,
thereby preventing or correcting
anemia.
Epo may also bind Epo R
expressed on the surface of
cancer cells and may elicit tumor
growth via cell proliferation,
protection from apoptosis, and/or
angiogenesis.     [Note:     figure
adapted from Brower, V. (2003)
Nat. Med. 9:1439.]
 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.
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.
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).
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.
 "Noone knows exactly how interferons
 work”!

 Uses
Infergen is used for patients with Chronic,
 non-responding, or relapsing hepatitis C
 viral (HCV) infection.
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.
 Uses:
Treatment    of relapsing forms of Multiple
 Sclerosis (MS). Slows the progression of MS by
 regulating the body's immune response against
 myelin.

Given    as an IM injection once per week.
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.
For  more information on Multiple Sclerosis,
 please have a look at the following website:

http://www.youtube.com/watch?v=qgySDmRRzxY
B.     Intron A
         It is rInterferon alpha-2b

      Uses:
       Over 20 indications!
i.     Malignant melanoma
                                         Sales
ii.    Non-Hodgkin's lymphoma          exceeding
iii.   Hairy cell leukemia                $440
                                       million in !
iv.    Kaposi's sarcoma               three years

v.     Chronic hepatitis B (HBV)
vi.    Chronic hepatitis C (HCV)
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. "
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
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
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
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
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
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
4. Genentech
A. 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
Converts  plasminogen to plasmin, which
 activates fibrin, breaking down blood
 clot.




                Clot dissolution
     ***FDP: FIBRIN DEGRADATION PRODUCTS
                                           32
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
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
 MOA:


  In CF patients, lungs and airways become
 clogged with mucous containing high
 concentrations of extracellular DNA
 released by degenerating leukocytes
 (neutrophils).

* Watch this!
http://www.pulmozyme.com/how-pz-
 works.jsp
                                             36
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
5.   The 5th company is: Eli Lilly:
                                      Worldwide
A.   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
1: Humulin pens

2: 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
Lispro (Humalog ®)      Eli lilly        Start < 15 min.
Rapid-acting                                                       Peak 30-60min
                         Aspart (Novorapid®)     Novo Nordisk



Short-acting             Humulin ® R             Eli lilly        Start 30-60
(Regular: identical to                                             min.
human insulin=                                                     Peak 4 hr
                         Novolin® R              Novo Nordisk
unmodified)


Intermediate-acting      Humulin ® N             Eli lilly        Start 1.5 hrs
                                                                   Peak 7 hr
(Isophane insulin:
                         Novolin® N              Novo Nordisk
insulin+protamine+zinc


                         Humulin® U: withdrawn   Eli lilly        Start 1 hr.
                         from market                               Peakless
                         Glargine (Lantus®)      Sanofi-Aventis
                         cannot be combined
Long-acting              with other insulins
                         Detemir (Levemir®)      Novo Nordisk
41
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
C.Xigris
It 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
 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
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
E.   Byetta
     It is rGLP-1 (glucagon-like peptide-1)

    Uses:
          Type II Diabetes: regulates glucose
     homeostasis     and    enhances glucose-
     dependent insulin secretion.




                                              46
    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
 -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
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
 Uses:


     First Cancer Vaccine: Approved for the
    immunization of:
   children aged 9 to 15 years
   adult females aged 16 to 26 years

Used 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
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

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Adv lec2

  • 1. Advanced Pharmaceutical Biotechnology Phar571 Lec. 2 Dr. Yara Al Tall JUST / Faculty of Pharmacy
  • 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 body's response to drugs.
  • 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. 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. 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. 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. Proteins do most of the work in the cell Enzyme Protease Transport Degrades Protein Hemoglobin Motion Carries O2 Actin Contracts Muscles Regulation Insulin Controls Blood Glucose Support Keratin Defense Forms Hair and Antibody Nails Fights Viruses
  • 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. 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 Vaccines B. Recombinant DNA Drugs C. Recombinant DNA Enzymes D. Recombinant DNA Growth Hormone E. Recombinant DNA Insulin F. Recombinant DNA Proteins G. Recombinant DNA Yeast
  • 10. Biotech's 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. The best rDNA drugs on the market today 1. 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. Figure 1. Erythropoietin (Epo) binds its receptor (Epo R) on the surface of red blood cell progenitors in the bone marrow. It causes proliferation, maturation, and differentiation, thereby preventing or correcting anemia. Epo may also bind Epo R expressed on the surface of cancer cells and may elicit tumor growth via cell proliferation, protection from apoptosis, and/or angiogenesis. [Note: figure adapted from Brower, V. (2003) Nat. Med. 9:1439.]
  • 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. 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. 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. 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.  "Noone knows exactly how interferons work”!  Uses Infergen is used for patients with Chronic, non-responding, or relapsing hepatitis C viral (HCV) infection.
  • 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.  Uses: Treatment of relapsing forms of Multiple Sclerosis (MS). Slows the progression of MS by regulating the body's immune response against myelin. Given as an IM injection once per week.
  • 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. For more information on Multiple Sclerosis, please have a look at the following website: http://www.youtube.com/watch?v=qgySDmRRzxY
  • 22. B. Intron A It is rInterferon alpha-2b  Uses: Over 20 indications! i. Malignant melanoma Sales ii. Non-Hodgkin's lymphoma exceeding iii. Hairy cell leukemia $440 million in ! iv. Kaposi's sarcoma three years v. Chronic hepatitis B (HBV) vi. Chronic hepatitis C (HCV)
  • 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. 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. 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. 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. 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
  • 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. 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. 4. Genentech A. 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. Converts plasminogen to plasmin, which activates fibrin, breaking down blood clot. Clot dissolution ***FDP: FIBRIN DEGRADATION PRODUCTS 32
  • 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
  • 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.  MOA: In CF patients, lungs and airways become clogged with mucous containing high concentrations of extracellular DNA released by degenerating leukocytes (neutrophils). * Watch this! http://www.pulmozyme.com/how-pz- works.jsp 36
  • 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. 5. The 5th company is: Eli Lilly: Worldwide A. 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. 1: Humulin pens 2: 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. Lispro (Humalog ®) Eli lilly Start < 15 min. Rapid-acting Peak 30-60min Aspart (Novorapid®) Novo Nordisk Short-acting Humulin ® R Eli lilly Start 30-60 (Regular: identical to min. human insulin= Peak 4 hr Novolin® R Novo Nordisk unmodified) Intermediate-acting Humulin ® N Eli lilly Start 1.5 hrs Peak 7 hr (Isophane insulin: Novolin® N Novo Nordisk insulin+protamine+zinc Humulin® U: withdrawn Eli lilly Start 1 hr. from market Peakless Glargine (Lantus®) Sanofi-Aventis cannot be combined Long-acting with other insulins Detemir (Levemir®) Novo Nordisk
  • 41. 41
  • 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. C.Xigris It 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.  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. 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. 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. 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.  -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. 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.  Uses: First Cancer Vaccine: Approved for the immunization of:  children aged 9 to 15 years  adult females aged 16 to 26 years Used 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. 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

Hinweis der Redaktion

  1. 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 .
  2. 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.
  3. 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.
  4. 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.
  5. 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 &quot;non-self&quot;, i.e. cells that are not an integral part of you (or your &quot;self&quot;).  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
  6. FDP: FIBRIN DEGRADATION PRODUCTS
  7. . 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.
  8. 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.
  9. Anabolic therapy: which bone formation is directly stimulated.
  10. 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.
  11. Cervical dysplasia refers to abnormal changes in the cells on the surface of the cervix that are seen underneath a microscope.