Antiprion drug
Antiprion drugs are a class of pharmaceutical drugs that are used in the treatment of prion diseases, a group of rare, fatal brain diseases that affect both humans and animals. These diseases are caused by misfolded forms of the prion protein, which can induce normal, healthy proteins to also misfold and accumulate in the brain, leading to neurodegeneration.
Mechanism of Action[edit | edit source]
Antiprion drugs work by targeting the abnormal prion proteins that cause the disease. They aim to prevent the conversion of normal prion proteins into the disease-causing form, halt the progression of the disease, and reverse the damage already done to the brain. The exact mechanism of action varies between different antiprion drugs, but they all aim to reduce the level of abnormal prion protein in the brain.
Types of Antiprion Drugs[edit | edit source]
There are several types of antiprion drugs currently in use or under investigation. These include:
- Quinacrine: This is an antimalarial drug that has been found to have antiprion properties. It is thought to work by binding to the prion protein and preventing it from misfolding.
- Pentosan polysulfate: This is a drug that is used to treat interstitial cystitis, a painful bladder condition. It has been found to have antiprion properties and is currently the only drug that has been shown to prolong survival in patients with prion diseases.
- Amphotericin B: This is an antifungal drug that has been found to have antiprion properties. It is thought to work by binding to the prion protein and preventing it from misfolding.
Side Effects[edit | edit source]
Like all drugs, antiprion drugs can have side effects. These can vary depending on the specific drug used, but may include nausea, vomiting, diarrhea, and changes in liver function. It is important for patients to discuss these potential side effects with their healthcare provider before starting treatment.
Future Research[edit | edit source]
Research into antiprion drugs is ongoing, with the aim of finding more effective treatments for prion diseases. This includes research into new drugs, as well as research into how existing drugs can be used more effectively.
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Contributors: Prab R. Tumpati, MD