Question

Describe the mechanism of action of the drug ZAVESCA

Briefly describe the normal cell biology, the pathogenic state and how the drug alters the pathogenic state.

Come up with an experimental plan by which you can prove where in the cell the drug acts; what cellular functions it modulates, and the mechanism by which it works.

Answer 1

ZAVESCA is a medicine which is used to treat
mild to moderate Type 1 Gaucher disease in
adults. It is also used to treat Niemann-
Pick type C disease in children, adolescents
and adults.
Type 1 Gaucher disease is an inherited disease
that one can get from both of their parents. People with Type 1 Gaucher disease are partially missing an enzyme that breaks down a chemical in the body. It is called glucosylceramide.
Too much glucosylceramide causes an
increase in the size of the liver and spleen.It
also causes bone disease and changes in the
blood.
ZAVESCA reduces the formation of
glucosylceramide to a level the partially
missing enzyme can cope with.
Niemann-Pick disease is an inherited disease which one can get from both parents. If someone have
NP-C, fats build up in the
cells of brain. This can result in
disturbances in neurological functions such as
eye movements, balance, swallowing, and
memory, and in seizures.
ZAVESCA works by inhibiting the enzyme
called 'glucosylceramide synthase' which is
responsible for the first step in the synthesis of
most glycosphingolipids.

The pathogenesis of a disease is the biological mechanism. It leads to a diseased state. The term can also describe the origin and development of the disease, and whether it is acute, chronic, or recurrent. The pathogenic mechanisms of a disease that are set in motion by the underlying causes, which if controlled would allow the disease to be prevented. Often, a potential cause is identified by epidemiological observations before a pathological link can be drawn between the cause and the disease. The pathological perspective can be directly integrated into an epidemiological approach in the interdisciplinary field of molecular pathological epidemiology. Molecular pathological epidemiology can help to assess pathogenesis and causality by means of linking a potential risk factor to molecular pathologic signatures of a disease. Thus, the molecular pathological epidemiology paradigm can advance the area of causal inference.

How the drug alters the pathogenic state.
Infection is the invasion of the host by microorganisms, which then multiply in close association with the host's tissues. Infection is distinguished from disease, a morbid process that does not necessarily involve infection . Bacteria can cause a multitude of different infections, ranging in severity from inapparent to fulminating. The capacity of a bacterium to cause disease reflects its relative pathogenicity. On this basis, bacteria can be organized into three major groups. When isolated from a patient, frank or primary pathogens are considered to be probable agents of disease. Opportunistic pathogens are those isolated from patients whose host defense mechanisms have been compromised. They may be the agents of disease. Finally, some bacteria, such as Lactobacillus acidophilus, are considered to be nonpathogens, because they rarely or never cause human disease. Their categorization as nonpathogens may change, however, because of the adaptability of bacteria and the detrimental effect of modern radiation therapy, chemotherapy, and immunotherapy on resistance mechanisms. In fact, some bacteria previously considered to be nonpathogens are now known to cause disease. Serratia marcescens, for example, is a common soil bacterium that causes pneumonia, urinary tract infections, and bacteremia in compromised hosts.
Virulence is the measure of the pathogenicity of an organism. The degree of virulence is related directly to the ability of the organism to cause disease despite host resistance mechanisms; it is affected by numerous variables such as the number of infecting bacteria, route of entry into the body, specific and nonspecific host defense mechanisms, and virulence factors of the bacterium. Virulence can be measured experimentally by determining the number of bacteria required to cause animal death, illness, or lesions in a defined period after the bacteria are administered by a designated route. Consequently, calculations of a lethal dose affecting 50 percent of a population of animals or an effective dose causing a disease symptom in 50 percent of a population of animals are useful in comparing the relative virulence of different bacteria.
Pathogenesis refers both to the mechanism of infection and to the mechanism by which disease develops. The purpose of this chapter is to provide an overview of the many bacterial virulence factors and, where possible, to indicate how they interact with host defense mechanisms and to describe their role in the pathogenesis of disease. It should be understood that the pathogenic mechanisms of many bacterial diseases are poorly understood, while those of others have been probed at the molecular level. The relative importance of an infectious disease to the health of humans and animals does not always coincide with the depth of our understanding of its pathogenesis. This information is best acquired by reading each of the ensuing chapters on specific bacterial diseases, infectious disease texts, and public health bulletins.
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