In class we talked about the formation of VLDL lipoproteins in the liver. The as

Question

In class we talked about the formation of VLDL lipoproteins in the liver.   The assembly of VLDL particles within the liver involves a protein called microsomal triglyceride transfer protein (MTP), which is essential for the assembly of apolipoprotein B (apo-B) containing lipoproteins. Known inhibition of MTP prevents hepatic VLDL secretion by preventing VLDL production. Given this effect, drugs targeting MTP are of interest as potential drugs for hyperlipidemia (a condition with elevated levels of lipid and/or lipoproteins).

In class we also talked about familial hypercholesterolemia (FH). Patients who are homozygous for FH (i.e., both copies of the LDL receptor gene are defective) have complete loss of LDL receptors whereas patients who are heterozygous (i.e., only one copy of the gene is defective) have an approximate 50% loss of LDL receptors. Would drugs targeting MTP be beneficial to homozygous familial hypercholesterolemia (FH) patients? Please explain your answer, including their effect on blood levels of cholesterol.

Explanation / Answer

Yes, drugs targeting MTP be beneficial to homozygous familial hypercholesterolemia (FH) patients because it has recently proved an approved by FDA that drug "lomitapide" can inhibit the microsomal triglyceride transfer protein (MTP) even though it is tough to treat with "statins & lipid lowering drugs". This drug specifically inhibits synthesis of cholesterol through inhibition of microsomal triglyceride transfer protein (MTP), which is essential for the assembly of apolipoprotein B (apo-B) containing lipoproteins. There is another drug approved by the US FDA in December 2012 to treat homozygous familial hypercholesterolem is "mipomersen" to abolish the activity of apolipoprotein B result in no synthesis of VLDL or cholesterol

Related Questions

Navigate

• Browse (All)
• Browse I
• Subjects

Integrity-first tutoring: explanations and feedback only — we do not complete graded work. Learn more.