Module 9
9.1: Introduction to Specialty Medications
In general, specialty medications are high cost medications used for treating complex disease
... [Show More] states. They can be
challenging to both manufacture and administer, and they often require significant patient education and close
monitoring to ensure their safe and appropriate use. Although specialty medications were once a very small piece of the
pharmaceutical industry, there has been a significant change over the last 20 years and especially within the last decade.
Specialty medications are now the fastest growing segment of the pharmaceutical industry. Common characteristics
associated with specialty medications are listed in Table 9.1.
Table 9.1 Specialty Medication Characteristics
High Cost Potential for limited or exclusive availability for
distribution
Complex treatment regimen that require ongoing
monitoring and patient education
Treat rare diseases
Special handling, storage, or delivery requirements Treat diseases known to have long term or severe side
effects or increased fatality
Biologically derived and available in injection, infusion, or
oral form
Payers may define what they consider to be a specialty
medication for reimbursement and contracting purposes.
With the increase in specialty medications over the last two decades, a whole new approach to dispensing these
medications emerged called specialty pharmacies. Generally, specialty medications are not available at typical
community pharmacies, so patients must obtain them through specialty pharmacies. There are many reasons for this:
some practical, some financial, and some clinical. Traditional retail pharmacy is not designed to handle these complex,
costly medications. First, the high cost alone, in many cases, would prohibit retail pharmacies from stocking the
medication. Second, the often-busy nature of a retail pharmacy does not align itself well with being able to appropriately
manage and support the needs of patients with complex disease states. For these reasons, the first specialty pharmacies
began in the early 2000s. Specialty pharmacies are typically required, through their contract with health insurance
companies, to meet unique requirements that are not part of their contracts with retail pharmacies. Examples of these
requirements are listing in Table 9.2.
Table 9.2 Examples of Specialty Pharmacy Services
Coordinating care and facilitating the drug access Case management- disease state management
Facilitating mail order delivery logistics Product device training when applicable
Working with health insurance to determine coverage
and help coordinate any requirements of the insurance
company
Data management of technical and clinical patient care
services.
Investigating patient assistance programs for patients
without insurance or lack of coverage
Call center development
Patient Experience
A patient receiving a prescription for a specialty medication should be aware that the process for getting the medication
is going to look different than the normal retail pharmacy experience. However, the system is designed so that the
patient has the best chance of successfully obtaining and using the medication safely and appropriately. The prescription
would first be sent to the specialty pharmacy. The specialty pharmacy then takes responsibility for making sure the
patient gets the medication, understands the risks and benefits, is able to afford it, able to take it appropriately, and will
follow through with any necessary monitoring. The patient may be connected with a case manager that will call them on
a monthly basis to make sure there are no issues with the medications, check to see if they had any required monitoring
completed, provide any necessary education, and/or answer patient questions. Patient follow-up often happens every
month prior to sending out the next months’ worth of medications in order to minimize waste, ensure safety, and assess
for efficacy. As specialty pharmacies are generally not local to the patient they are caring for, care and coordination are
often provided telephonically. The process is centralized, and the medications are shipped to the patient’s house or, in
some cases, to the facility that will be administering the medications.
Brief Overview of Common Disease States Treated with Specialty
Medications
Common disease states managed by specialty pharmacies include (1) oncology, (2) multiple sclerosis, (3) rheumatoid
arthritis, (4) Crohn’s disease, (5) hepatitis C, and (6) HIV/AIDs. This module with introduce some of the most commonly
used specialty medications that most health care professionals would come into contact with, regardless of their
specialty. Many of these conditions represent an entire specialty in medicine and are very complex. Therefore, for the
purposes of this module, these conditions will be introduced briefly.
Oncology is the study and treatment of cancer. Cancer is a disease process that involves the development and
proliferation of abnormal calls. Cancer cells are marked by both a structural change and a loss of function from the
original healthy cell. They are often characterized by multiplying at a faster than normal rate. As this collection of
abnormal cells, called a tumor, grows it can become life threatening as it deprives normal body cells of the nutrients they
need to function.
Multiple Sclerosis (MS) is an often unpredictable, disabling disease of the central nervous system. It disrupts the flow of
information both within the brain as well as between the brain and the body. Damage to the myelin coating around the
nerve fibers in the CNS causes the nerve signals to be disrupted. This damage ultimately leads to the symptoms of MS
that can vary between individuals with the disease. Some of the more common symptoms include fatigue, difficulty
walking, numbness or tingling, stiffness in the limbs, weakness, vision problems, cognitive changes, pain, depression, and
emotional changes.
Rheumatoid Arthritis (RA) is a chronic autoimmune disorder that causes inflammation and tissue damage in the joints. It
is a very painful and (often) disabling disease. The common symptoms of RA include pain, stiffness, and reduced range of
motion. Generally, the treatment would not start with a specialty medication. The initial medications fall under the class
disease modifying antirheumatic drug (DMARD). However, these are not always effective or in some instances, patients
may no longer respond as they once did. In either case, a patient would be then switched to one of the specialty
medications reviewed below.
Crohn’s Disease is an inflammatory bowel disease caused by inflammation in the digestive tract which can lead to
symptoms such as abdominal pain, severe diarrhea, fatigue, weight loss, and malnutrition. The inflammation can spread
deep into the layers of the affected bowel tissues making the symptoms extremely painful and often hard to control.
Hepatitis C is a viral infection that causes liver inflammation which can sometimes lead to serious liver damage. The virus
is spread through infected blood. There are often no symptoms associated with chronic hepatitis C until the virus
damages the liver significantly enough. Symptoms of hepatitis C then include bleeding and bruising easily, fatigue, poor
appetite, dark urine, swelling, weight loss, and confusion. Interestingly, acute hepatitis C often goes undiagnosed
because of the lack of or self-limiting nature of the symptoms. Additionally, some people that contract hepatitis C never
go on to develop chronic hepatitis C because their body is able to clear the virus on its own. However, those that do
progress to chronic hepatitis C, will likely develop liver failure and need a liver transplant should they live long enough if
the disease is left untreated. Recent treatments have made the possibility of a cure very likely with only three months of
treatment.
HIV/AIDS is a notorious virus with incredible advancements in treatment over the last 30 years. HIV stands for human
immunodeficiency virus and is the causative agent of acquired immunodeficiency syndrome (AIDS). The virus is spread
through sexual contact, perinatally from an infected mother, or by injection into the blood. The typical course of the
infection is characterized by an acute clinical illness that varies in presentation followed by a longer clinical latency.
People may go years without any symptoms. However, during this asymptomatic time, the virus is working in the body to
destroy the immune system, specifically the CD4 Cells (T-cells). With the destruction of immune cells, the person is
eventually no longer able to fight off pathogens (disease causing agents), a state referred to as being
immunocompromised. When a pathogen is able to take advantage of the lowered immunity, it is called an opportunistic
infection. At this point, the patient is generally considered to have AIDS. HIV/AIDS cannot be cured, but it can be
controlled with antiretroviral therapy (ART).
9.2: Introduction to Immunomodulating Drugs
Over the last twenty years, medical technology has developed a new group of drugs that affect the immune system.
Many of these drugs are synthesized through recombinant DNA technology, which is the process of joining DNA
molecules from two different sources and inserting them into a host organism which then generates specific products for
human use. These drugs are often referred to as biologics and are almost always considered specialty medications. They
are in a large part responsible for the growth of the specialty pharmacy industry. Such drugs work by altering the body’s
response to diseases such as cancer, autoimmune, inflammatory and infectious diseases. Biologics can work either by
enhancing or restricting the patient’s natural immune response. To better understand how such medications work within
the body, we will briefly cover the physiology of the immune system.
Immune Response Overview
One of the main functions of the immune system is to identify substances as being either foreign or of self. When
bacteria or viruses enter the body, the immune system should recognize both as foreign and initiate an immune response
to eliminate it from the body.
More specifically, there is humoral immunity and cell-mediated immunity.
Humoral immunity is defined as the immune response mediated by B-cells and the production of antibodies targeted
against specific antigens. B-cells are leukocytes that develop into plasma cells and then produce antibodies that bind to
and inactivate antigens. Antibodies are molecules that have the ability to bind to and inactivate antigen molecules
through the formation of an antigen-antibody complex.
Cell-mediated immunity which works in collaboration with humoral immunity, is the immune response mediated by Tcells.
T-cells (T lymphocytes) are not involved in the production of antibodies but instead act through either direct cellto-
cell contact or through the production of cytokines. Cytokines are a generic term for non-antibody proteins released
by specific cell populations (activated T-cells) upon contact with antigens. They act as intercellular mediators of an
immune response. Of note, there are various subtypes of T-cells: (1) helper, (2) suppressor and (3) cytotoxic.
T helper cells are cells that promote the direct actions of numerous other cells associated with the immune system.
T suppressor cells regulate and limit the immune response, balancing the effect of T helper cells.
Cytotoxic T cells (natural killer cells) are differentiated T-cells that can recognize foreign antigens being presented on the
surface of another cell. Once recognized, the cytotoxic T-cell then attacks and destroys the particular target cell.
The main types of biologics covered in this module are classified as immunomodulating drugs.
Immunomodulating drugs are defined as a subclass of biologics that specifically or nonspecifically enhance or reduce
the immune response. The subclasses of immunomodulating drugs include (1) interferons, (2) monoclonal antibodies, (3)
interleukin receptor antagonists and agonists and (4) other miscellaneous drugs. Since these drugs alter a patient’s
immune response, they are often used in cancer treatments because they are able to specifically target the cancer cells
while leaving healthy cells alone. They are also commonly used to treat autoimmune and inflammatory conditions by
interfering with a patient’s overactive immune response in diseases like rheumatoid arthritis.
Select Immunomodulating Drugs
Interferons are proteins that have antitumor, antiviral, and immunomodulating properties. They are most commonly
used in the treatment of certain cancers and viral infections. Interferons have three different effects on the immune
system. (1) They can restore function if it is not working properly, (2) they can augment its function, or (3) they can
inhibit its function. Inhibiting its function becomes important in autoimmune diseases because the immune system is not
working properly.
Note: Due to the fact that most of these drugs are only available in a brand name option, clinicians generally refer to
them by their brand name. It is recommended that the student be familiar with both the brand and generic name of the
drugs covered in this module.
Example Interferon
Interferon beta-1a (Avonex or Rebif) is indicated to treat relapsing multiple sclerosis. It interacts with the specific cell
receptors found on the surface of human cells. They have been shown to slow the progression of physical disability and
decrease the frequency of clinical exacerbations.
Avonex is a once weekly intramuscular injection while Rebif is a three times per week subcutaneous injection. The most
common adverse effects with interferons are flu-like symptoms such as fever, chills, malaise, myalgia, and fatigue.
Monoclonal Antibodies are becoming the drugs of choice for many diseases such as cancer, rheumatoid arthritis, Crohn’s
disease, multiple sclerosis, and organ transplant. In the treatment of cancer, they have an advantage over traditional
chemotherapy medications in that they can specifically target cancer cells and leave healthy cells alone. These drugs are
made using recombinant DNA technology making them extremely costly. Despite being more targeted than traditional
cancer therapies, severe allergic inflammatory type infusion reactions can occur. Patients often need to be pre-medicated
to reduce the incidence.
Example Monoclonal Antibodies
Adalimumab (Humira) acts on tumor necrosis factor (TNF), which is a naturally occurring cytokine involved in the normal
inflammatory and immune response. Adalimumab works by preventing TNF molecules from binding to the TNF cell
surface. It also works by impacting the typical inflammatory responses regulated by TNF. Although originally indicated for
rheumatoid arthritis, it can also be used in Crohn’s disease, ulcerative colitis, plaque psoriasis, and psoriatic arthritis. The
most common adverse reactions are infections, injection site reactions, headache, and rash. Infliximab (Remicade)
works very similarly to adalimumab, although it does carry a unique contraindication. Due to being shown to worsen
heart failure, it should not be used in patients with class III or IV on the New York Heart Association Scale.
Bevacizumab (Avastin) works by binding to and inhibiting vascular endothelial growth factor, which is a protein that
promotes the development of new blood vessels in both tumor and normal body tissues. It is indicated to treat
metastatic colon cancer, rectal cancer, non-small-cell lung cancer, and malignant glioblastoma (type of brain cancer).
Adverse effects include blood clots, GI issues, headache, dizziness, and weight loss.
Natalizumab (Tysabri) is a humanized monoclonal antibody derived from murine myeloma cells. It works by binding to
the alpha4 subunit of integrins, which are proteins found on the surface of leukocytes. These proteins have been
associated with the disease process of multiple sclerosis, although the exact mechanism is not completely understood. It
is known that natalizumab inhibits the leukocyte adhesion that the alpha4 protein subunits are involved. It is indicated to
be used to treat multiple sclerosis (MS). There is a risk of a rare viral infection in the brain for patients on this medication.
Therefore, in 2006 the FDA limited its distribution and patients must enroll in a specific program prior to being able to
receive the drug. Less severe adverse effects include depression, fatigue, headache, GI issues, and lower respiratory tract
infections.
Interleukins are a natural part of the immune system. They are actually classified as lymphokines because they are
cytokines produced at least in part by lymphocytes. They are soluble proteins released from activated lymphocytes such
as natural killer cells. There are several different interleukins that have been identified, and each has different specific
actions within the body. For example, interleukin-2 (IL-2) specifically is known to have anti-tumor actions.
Example Interleukins
Aldesleukin (IL-2) (Proleukin) is an interleukin-2 derivative and works indirectly to restore the immune response by
binding to receptor sites on T-cells, stimulating them to multiply. One type of cell that results is called the lymphokineactivated
killer (LAK) cell. LAK cells can recognize and destroy cancer cells while leaving other healthy cells alone. For this
reason, Aldesleukin is specifically indicated for metastatic renal cell carcinoma (kidney cancer that has spread) and
melanoma (skin cancer). Adverse events include severe toxicities, specifically capillary leak syndrome—the body’s
capillaries are no longer able to retain the substances that make up blood causing them to “leak” into the surrounding
tissue. This results in severe fluid retention that can be life-threatening if not treated. The syndrome is reversible with the
discontinuation of the drug. Other more typical adverse events include fever, chills, rash, fatigue, liver toxicity, muscle
pain, and headache.
Anakinra (Kineret) is a recombinant form of human interleukin -1 (IL-1) receptor antagonist. It acts by inhibiting the
binding of IL-1 to its many receptor sites throughout the body. Because this drug serves to block an immune process, it is
effective in treating rheumatoid arthritis, which is a condition where the immune system is not functioning properly.
Adverse events most commonly include injection site reactions, respiratory tract infections, and headache.
Miscellaneous
There are several immunomodulating drugs that work by various mechanisms but do not fall into one of the previous
categories. Two of the more commonly used miscellaneous immunomodulators are covered below.
Example Drugs
Abatacept (Orencia) is a selective co-stimulation modulator indicated for the treatment of rheumatoid arthritis. It works
by inhibiting T-cell activation. Common adverse effects include headaches, upper respiratory tract infections, and
hypertension. It should not be combined with TNF blocking drugs like adalimumab and infliximab or with anakinra due to
the increased risk of serious infection.
Etanercept (Enbrel) is a recombinant DNA-derived TNF-blocking drug. It works by binding to TNF and blocking its ability
to bind to its target receptors on the cell surface. It is indicated to treat rheumatoid arthritis and plaque psoriasis.
Common adverse effects include headache, injection site reactions, upper respiratory tract infections, dizziness, and
weakness.
In general, these drugs are administered by injection although the exact route varies. See the summary chart (Table 9.3)
of the immunomodulating drugs covered in this module for specifics on the route of administration.
9.3: Antivirals & Antiretroviral Therapy
Antivirals for the Treatment of Hepatitis C
There have been major advancements in the treatment of hepatitis C in the last 5 years. Prior to these new medications,
the treatment for hepatitis C included interferon and a drug called ribavirin. Ribavirin did not target the virus itself but
rather helped to enhance the immune system with the hope that the body would then be able to fight off the virus. The
cure rates were low while the side effects were high; so many people did not even attempt treatment. Over time, the
likely outcome for these patients (if they lived long enough) was the need for a liver transplant. With the development of
a new class of antiviral medication that targets the hepatitis C virus specifically, the likelihood of a cure is now close to
96%. One of the best aspects of the new treatments is the short duration. In fact, patients may only need to be treated
for 8-12 weeks. This represents a significant improvement from traditional treatment that lasted anywhere from 24-48
weeks and had a 50% success rate at best. The new treatments available are considered “direct acting” antiviral
medications. In simple terms, they interfere with the proteins that help the virus grow and spread. Two of the more
common options are introduced below.
Ledipasvir/sofosbuvir (Harvoni) was the pill that launched this radical change in the treatment of Hepatitis C. In general,
the side effects are mild, and the cure rate is high. The price tag is also high at about $95,000 for a 12-week treatment
course.
Ombitasvir/paritaprevir/ritonavir with dasabuvir (Viekira Pak) has two separate pills. In general, the side effects are
mild with the exception of some cases of serious liver injury, mostly seen in patients with underlying advanced liver
disease. Of note, ritonavir is a protease inhibitor (see section below on HIV) with no activity against hepatitis C. It is a
potent inhibitor of CYP 3A4 enzymes within the liver and is used to increase the concentrations of paritaprevir. The cost
is also high at about $84,000 for a 12-week treatment course.
Antiretroviral Therapy (ART)
A full review of ART is beyond the scope of this course. This module will simply introduce the main classes of
antiretrovirals and provide a basic understanding of the pharmacologic treatment of HIV. There have been many great
advancements in the treatment of HIV since it was first identified in the early 1980s. Due to the high cost of most HIV
medications and the importance of compliance, HIV medications are almost always considered specialty medications.
HIV is unique from other viruses because it mutates rapidly which leads to drug resistance. When this happens, the drugs
are no longer effective against the virus. In order to better understand the different classes of antiretrovirals, HIV as a
virus needs to be reviewed in a bit more detail.
HIV
HIV is a retrovirus. As such, HIV contains the enzyme reverse transcriptase which coverts its RNA genome into DNA. The
viral DNA can then be integrated into the host cell’s DNA where it will be replicated alongside the host DNA. Thus, the
virus ‘tricks’ the host into replicating its viral genome which then leads to the production of the viral proteins required to
assemble new viruses. HIV specifically targets macrophages and helper T-cells, effectively compromising the immune
system of the host. Once HIV has control of the cell, it often begins producing more HIV that can go out and infect more
host cells. Over time, the patient becomes more and more immunosuppressed, meaning that the body’s ability to fight
infection has been compromised. Once the patient begins developing multiple opportunistic infections because of the
depletion in lymphocytes (T-cells), they are said to have AIDS. Importantly, once a patient is infected with HIV, they have
it for life. There is no cure. The medications are simply used to help control the virus and prevent it from propagating
further to hopefully delay the onset of AIDS.
HIV treatment should always include more than one mechanism of action. Monotherapy cannot counter the
development of drug resistance, and a missed dose can quickly lead to a poor outcome. The current treatment strategy
is referred to as HAART (highly active antiretroviral therapy). HAART consists of three antiretrovirals taken in
combination. This increases the effectiveness of the regimen by presenting the virus with multiple obstacles to try and
overcome.
Note: The effects of the HIV virus and the counter effects of the antiretrovirals can get very technical very quickly and
again is really beyond the scope of this course.
Basic concepts of virus propagation will be introduced in order to have a working understanding of these drugs. As
mentioned above, in order to multiply, viruses must be able to enter the cell nucleus and integrate with the host DNA.
They first enter the cell by attaching to the outer cell membrane. Thus, this initial attachment is a potential site of action
for drug therapy. The second thing that must happen is the virus needs to be able to take over the process of DNA
transcription so that the host cell is now ultimately producing progeny virus to be sent out to infect more cells. This is
another site of action for drug therapy—the drugs target the process of transcription. When the host cell is successfully
producing the virus, it also must be able to expel the virus out of the cell (egress) and into circulation in order to find
another host cell to infect. Inhibiting egress is another potential site of action for drug therapy.
The most common combination therapy to start with is a triple cocktail that combines two nucleoside-analogue reverse
transcriptase (NRTIs) and one nonnucleoside reverse transcriptase inhibitor (NNRTI), or one protease inhibitor.
Fortunately, there are now combination tablets that have all three of these medications in a single dose. New
advancements are continually being made in the area of HIV research. This module will introduce five primary drug
classes used in the treatment of HIV.
Fusion Inhibitors work by preventing the complete fusion of HIV to the host cell. Therefore, penetration of the virus into
the host cell is blocked as are any subsequent steps. However, fusion inhibitors are not 100% effective. For this reason,
additional strategies are used to combat the virus.
Nucleoside-analogue reverse transcriptase inhibitors (NRTIs) are part of the recommended HAART regimen.
Nucleosides are the fundamental building blocks of RNA and DNA. The NRTIs work by incorporating themselves into the
DNA and inhibiting the reverse transcriptase and synthesis of new viruses. This, in turn, decreases viral replication and
the infection is reduced.
Nonnucleoside reverse transcriptase inhibitors (NNRTIs) are also part of the recommended HAART regimen. NNRTIs
work by directly binding to the enzyme reverse transcriptase found within HIV, thus blocking its ability to take over the
transcription process of the host cell.
Integrase Inhibitors work by blocking the enzyme integrase which is essential for the virus to be able to enter the host
cell nucleus and begin incorporating its viral DNA into the host cells’ DNA.
Protease Inhibitors are the third option for the recommended HAART regimen. As their name indicates they work by
inhibiting protease, an enzyme essential for the final assembly of the new virus. The new virus is thus unable to be
released into circulation to find a new host cell.
Table 9.4 below provides an overview of specific select antiretroviral medications.
Table 9.4 Examples of Antiretrovirals
Drug Class
Example Drug(s)
NRTI Zidovudine (Retrovir)
Tenofovir (Viread)
Emtricitibine (Emtriva)
Lamivudine (Epivir)
NNRTI Efavirenz (Sustiva)
Rilpivirine (Edurant)
Nevirapine (Viramune)
Protease Inhibitor Atazanavir (Reyataz)
Darunavir (Prezista)
Ritonavir (Norvir)
Integrase Inhibitor Dolutegravir (Tivicay)
Raltegravir (Isentress)
Fusion Inhibitors Enfuvirtide (Fuzeon)
HAART requires multiple medications in order for the best chance of controlling the virus. Many of the above
medications are available in combination pills in order to make the dosing regimen simpler. Adherence to these
medications is critical to increase effectiveness and avoid or at least delay the development of drug resistance. Some of
the common combination tablets are listed below in Table 9.5.
Table 9.5 Examples of Combination Tablets for the Treatment of HIV
Drug name and components Drug classes
Atripla (efavirenz/lamivudine/tenofovir) NNRTI/NRTI/NRTI
Truvada (emtricitabine and tenofovir) NRTI/NRTI
Complera (emtricitabine/rilpivirine/tenofovir) NRTI/NNRTI/NRTI
The adverse effects associated with HIV medications are generally very drug-specific and beyond the scope of this
module. However, most drugs tend to cause nausea and vomiting. More serious adverse events that need to be
monitored for include renal toxicity and neutropenia. Neutropenia is a decrease in white blood cells, specifically
neutrophils. This is especially concerning in patients that may already be immunocompromised. Patients will often
require frequent blood counts to monitor for these more serious adverse events.
Problem Set
Question 1
List characteristics that typically describe a specialty drug.
High Cost Potential for limited or exclusive availability for
distribution
Complex treatment regimen that require ongoing
monitoring and patient education
Treat rare diseases
Special handling, storage, or delivery requirements Treat diseases known t have long term or severe side
effects or increased fatality
Biologically derived and available in injection, infusion,
or oral form
Payers may define what they consider to be a specialty
medication for reimbursement and contracting
purposes.
Question 2
List some common additional requirements that the specialty pharmacy must offer to patients in order to ensure that
these high cost, potentially high adverse events medications are used safely and appropriately.
Coordinating care and facilitating the drug access Case management- disease state management
Facilitating mail order delivery logistics Product device training when applicable
Working with health insurance to determine coverage
and help coordinate any requirements of the insurance
company
Data management of technical and clinical patient care
services.
Investigating patient assistance programs for patients
without insurance or lack of coverage
Call center development
Question 3
Explain the process that a patient would need to follow in order to obtain one of these medications.
The prescription would first be sent to the specialty pharmacy. The specialty pharmacy then takes responsibility for
making sure the patient gets the medication, understands the risks and benefits, is able to afford it, take it
appropriately and follows through with any necessary monitoring. The patient may be connected with a case manager
that will call them on a monthly basis to make sure there are no issues with the medications, check to see if they are
had any required monitoring, provide any necessary education or answer patient questions. This often happens every
month prior to sending out the next months' worth of medications to minimize waste, ensure safety, and assess for
efficacy.
Question 4
List the most chronic disease states that are treated with specialty medications.
oncology, multiple sclerosis, rheumatoid arthritis, Crohn’s disease, hepatitis C, and HIV/AIDs
Question 5
List a description of the basic disease process including common symptoms of following disease states: Rheumatoid
arthritis, Multiple Sclerosis, Crohn’s disease, Hepatitis C, and HIV
RA: is a chronic autoimmune disorder that causes inflammation and tissue damage in the joints. The common
symptoms of RA include pain, stiffness, and reduced range of motion.
MS: It disrupts the flow of information within the brain and between the brain and the body. There is damage to the
myelin coating around the nerve fibers in the CNS that cause the nerve signals to be disrupted. Some of the more
common symptoms include Fatigue, difficulty walking, numbness or tingling, stiffness in the limbs, weakness, vision
problems, cognitive changes, pain, depression and emotional changes.
Crohn’s disease is an inflammatory bowel disease caused by inflammation in the digestive tract which can lead to
symptoms such as abdominal pain, severe diarrhea, fatigue, weight loss, and malnutrition. The inflammation can
spread deep into the layers of the affected bowel tissues making the symptoms extremely painful and often hard to
control.
Hepatitis C: a viral infection that caused liver inflammation and sometimes leads to serious liver damage. The virus is
spread through infected blood. There are often no symptoms with Chronic hepatitis C until the virus damages the liver
significantly enough. Symptoms of hepatitis C then include bleeding and bruising easily, fatigue, poor appetite, dark
urine, swelling, weight loss, confusion.
HIV is s retrovirus that attacks the body’s immune system. The typical course of the infection is characterized by an
acute clinical illness that varies in presentation followed by a longer clinical latency. People may go years without any
symptoms. However, during this asymptomatic time the virus is working in the body to destroy the immune system,
specifically the CD-4 Cells (T-cells). Over time the person is no longer able to fight off bacteria that generally do not
cause infection called opportunistic infections.
Question 6
Define the following terms related to the immune system: Humoral immunity, cell mediated immunity, Antibodies, T-cells
and cytokines.
Humoral immunity is defined as the immune response mediated by B cells and the production of antibodies targeted
against specific antigens.
Antibodies are molecules that have the ability to bind to and inactivate antigen molecules through the formation of an
antigen-antibody complex.
Cell-mediated immunity which is the immune response mediated by T-cells.
T-cells or T lymphocytes are not involved in the production of antibodies but instead occur in different types of
subtypes that act through direct cell to cell contact or through the production of cytokines.
Cytokines is a generic term for non-antibody proteins released by specific cell populations (activated T cells) on
contact with antigens. They act as intercellular mediators of an immune response.
Question 7
Describe the impact on the immune system of the more common immunomodulating drugs such as: interferons, anti-
TNF monoclonal antibodies, interleukins (specifically IL-2).
Interferons are proteins that have antitumor, antiviral, and immunomodulating properties. Interferons have three
different effects on the immune system. They can restore function if it’s not working properly, they can augment its
function, or inhibit its function.
Interleukins are a natural part of the immune system. They are actually classified as lymphokines because they are
cytokines produces at least in part by lymphocytes. Il-2 works by indirectly to restore immune response by binding to
receptor sites on T-cells stimulating them to multiply. One type of cell that results is called the lymphokine-activated
killer (LAK) cell. This LAK cell can recognize and destroy cancer cells while leaving other healthy cells alone
Anti-TNF Monoclonal antibodies acts on tumor necrosis factor (TNF), which is a naturally occurring cytokine that is
involved in the normal inflammatory and immune response. they essentially work by preventing TNF molecules from
binding to the TNF cell surface.
Question 8
List two of the new hepatitis C treatment options and describe how they improve previous treatment options.
Harvoni and Viekira Pak. With the development of a new class of antiviral medication that targets the hepatitis C virus
specifically, the chance for a cure, is now close to 96%. Possibly the best part of this is that the treatment is not
chronic. In fact, patients may only need to be treated for 8-12 weeks. Additionally, the side effects of these new drugs
are minimal in comparison to the old treatment regimen of interferon.
Question 9
Explain what the HIV virus does once it enters the body.
HIV is a retrovirus. This means that it contains the enzyme reverse transcriptase. Essentially, this allows it to take over
the process of DNA transcription (how cells multiply) once it enters the host cell. HIV specifically targets macrophages
and helper T-cells effectively compromising the immune system of the host. Instead of producing cells that help fight
infection, once the HIV has control of the cell, it actually begins producing more HIV that can go out and infect more
host cells.
Question 10
Explain why it is not recommended to treat HIV with only one medication.
Monotherapy cannot counter the development of drug resistance and a missed dose can quickly lead to a poor
outcome. The current treatment strategy is referred to as HAART (highly active antiretroviral therapy). HAART consists
of three antiretrovirals taken in combination. This increases the effectiveness of the regimen by presenting the virus
with multiple obstacle to try and overcome.
Question 11
List the primary antiretroviral classes covered in this module and provide a brief description at how they target the HIV
virus.
Nucleoside-analogue reverse transcriptase (NRTIs). The NRTIs work by incorporating themselves into the DNA and
inhibiting the reverse transcriptase and synthesis of new viruses. This in turn decreases viral replication and the
infection is reduced.
Nonnucleoside reverse transcriptase inhibitors (NNRTIs). NNRTIs work by directly binding to the enzyme reverse
transcriptase found within HIV virus thus blocking its ability to take over the transcription process of the host cell.
Protease Inhibitors. As their name indicates they work by inhibiting protease, an enzyme essential for the final
assembly of the new virus. The new virus is thus unable to be released into circulation to find a new host cell.
Integrase Inhibitors work by blocking the enzyme integrase which is essential for the virus to be able to enter the host
cell nucleus and begin incorporating into the transcription process.
Fusion Inhibitors work by preventing the complete fusion of HIV to the host cell. Therefore, the penetration into the
host cell is blocked and it cannot take over the transcription process. [Show Less]