Drug Re purposing for Rare Diseases

Medicine is always changing, reforming and altering. In order to provide the best patient care there needs to be new innovation every day, or else not everybody’s needs will be satisfied.

You may wonder what I mean by ‘needs be satisfied’. Well withing British medicine the word ‘needs’ can be extremely broad. However in relation to today’s topic it means from the normal, common diseases to the rare uncommon diseases.

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In the EU if 1 in 2000 people have a particular disease, in order for it to be classified as rare. Also in the UK 3.5 million people live with rare diseases, and due to their rarity, can be chronic and life threatening. Sadly, at the current rate of development, it would take 500 years to formulate new drugs for all of these diseases. However there is something else we can do to speed up this process.

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Drug re purposing is discovering new uses for approved drugs to provide the quickest possible transition from bench to bedside. It works in two ways:  identification of candidate drugs and testing of their effect.  Prior knowledge about drugs and re purposing studies has helped us to follow these two steps.

Re purposing studies have allowed us to form intelligent drug screens, which specifically target a small set of drugs thought to act on the relevant pathway.

For example, the re purposing of one of the first monoclonal antibodies which has been re purposed from cancer into multiple sclerosis, acting to effectively boost the immune system to protect against the disease’s degenerative effects.

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After a re purposing opportunity is identified, the second major component of a re purposing study is the proof of concept. This is the completion of rigorous preclinical and clinical trials to test the effect of the candidate drug in the patient population. This rigorous testing process will hopefully help us cure more diseases than we could have ever dreamed of.

A massive advantage of using re purposed drugs is that its using known substances, that has been shown to be absorbed by the body and also more importantly, safe for human consumption. This means it saves time and money dramatically.

Thankyou.

 

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