Rare mutation prompts race for cholesterol drug

NY Times via Columbus Dispatch (OH) ^ | July 14, 2013 | Gina Kolata

[Rodamala]

I wonder if down the road there might be a gene therapy treatment.

I wonder if one day they find out that western medicine is bunk, and that you are best off eating bacon sammiches on butter-soaked toast while drinking a fifth of whiskey and chain-smoking 2 packs of Lucky Stikes every day.

Hey are eggs "OK" this week?

Fools.

[exDemMom]

So, due to the recent supreme court case, who owns the patent on the mutation?

If the drug company creates a process for advancing this gene, will the people they gleaned the gene mutation from own the mutation or does the drug company? Or nobody?

The drugs being tested here are not genes and do not alter genes. They are monoclonal antibodies.

If I am to guess at their mechanism, I would say that they bind to the normal form of the enzyme that is defective in these women. Their gene defect causes a defect in an enzyme involved in the formation of LDL. The monoclonal antibodies attach to this same enzyme in normal people, deactivating it. They won't be able to make LDL without it. I think people receiving this treatment will need constant monitoring to ensure that their LDL levels remain within the normal range.

I think the recent Supreme Court case is going to put a halt into development of diagnostics based on genetic mutations. It costs too much to push medical devices through FDA approval to make it worthwhile to base a diagnostic kit on an unpatentable marker.

[exDemMom]

The unintended consequences should be interesting.

Indeed. Given the quirks of human physiology, I wonder what problems might show up in Phase 4 (post-approval) testing or even further down the road?

I also keep going around in circles about the causes of heart disease. Are cholesterol levels the drivers of heart disease, or are they a marker of it? Could another problem that hasn't been discovered yet be responsible for high cholesterol and heart disease?