Question

Consider the following situation. Patients with Smith-Lemli-Opitz have an inborn error of cholesterol biosynthesis, which results in severe to mild cholesterol deficiency, and accumulation of cholesterol precursors. An investigator proposes to determine if pure crystalline cholesterol in an oil suspension will increase cholesterol levels and decrease cholesterol precursors. The investigator proposes to obtain cholesterol, mix it with soy oil, and administer it to subjects. The study then involves follow-up evaluations and assessments. What would be the best choice regarding an IND?

Answer 1

Cholesterol is essential for the body, but an excess can cause health issues. The proposed cholesterol oil suspension study for Smith-Lemli-Opitz patients should be preceded by an IND application to ensure safety and efficacy.

Step-by-step explanation:

High cholesterol levels in the bloodstream are a known risk factor for various health conditions, such as heart disease, but cholesterol itself is essential for numerous bodily functions. Cholesterol plays a vital role in cell membrane structure, the production of certain hormones, and as a precursor for vitamin D synthesis. While too much cholesterol can lead to problems such as atherosclerosis, the body needs a certain level of cholesterol to function properly. This is why eradicating all cholesterol from the body is not a desirable goal.

The study proposed to help patients with Smith-Lemli-Opitz syndrome by offering them an oil suspension of crystalline cholesterol might be able to increase their cholesterol levels and balance out the accumulation of cholesterol precursors. However, simply administering cholesterol without proper oversight could be risky. Instead, an Investigational New Drug (IND) application should generally be submitted and approved by regulatory authorities before proceeding with such clinical trials, as this would ensure safety and efficacy standards are maintained throughout the study.