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What are the immediate challenges to break through in seeking a cure for mad cow disease?

I know that mad cow disease has no treatment as of yet.

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up vote 4 down vote accepted

The main problem is that Mad Cow disease is not caused by a "normal" pathogen but by a prion, a protein.

Traditionally, disease causing agents can be classified into viruses, bacteria, fungi, and parasites. Bacteria, fungi and parasites are all living organisms, alive in the traditional sense. It is, therefore, possible to design drugs that kill them.

Viruses are trickier, while not really alive in the traditional sense, they still have to make copies of their genetic material (DNA, or RNA in the case of many viruses) in order to cause infection. Therefore, drugs like ganciclovir that stop the formation of viral DNA can be an effective treatment.

Prion based diseases such as Mad Cow disease, Creutzfeldt–Jakob disease and Kuru, are not caused by a classical infectious agent but by a simple protein, a prion. Prions are misfolded versions of proteins already present in the host's body. When a prion interacts with the host protein, it causes this protein to adopt the same, misfolded, state as the prion. Protein function depends directly on the protein's structure, the way it is folded in three dimensional space. Therefore, the misfolded protein can no longer carry out its physiological role and disease symptoms occur.

Now, since the prion is just a protein, one single molecule, there is no essential life process that we can disrupt. We have to attack them using chemical agents that target the prion while not affecting the healthy, correctly folded, host protein. This is very hard to do.

The blood-brain barrier mentioned by Larry_Parnell is another problem. Briefly, the BBB, is a kind of fence that only allows certain of the various items circulating in the blood stream to enter the organism's brain. This is a wonderful defensive feature but it makes it much harder to design drugs that can enter the brain and target pathogens found there.

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Thanks for the wonderfully clear explanation! – Oreotrephes Dec 21 '13 at 17:04

A big challenge is obstacles presented by the blood-brain barrier.

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Could you please expand on that a bit? The question is posed from a laymen's perspective and might need more explanation to be understandable by the asker. – Mad Scientist Sep 27 '12 at 5:48

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