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.

 

Work Experience 101: Hospital Work Experience

From Tuesday 28th of February until Thursday 2nd March I was given the amazing opportunity to do 3 days of work experience at Derby Royal Hospital on the ‘Hospital Experience Program for Potential Medical Applicants’ AKA HOSPEX.

This work experience was very challenging for me. Although I was only observing, finding my way around the hospital and being thrown into an unfamiliar environment with people I have never met before was amazing growth for me. Overall I gained so much confidence from the experience as I had to be completely independent.

On the first morning, I was thrown straight in to shadowing a consultant in the renal department. I found it so interesting looking at all the dialysis machines and then doing ward rounds. The Doctors that I was following were all very nice, and they asked me some medical questions, to help me engage into the rounds. For example I got asked ‘How do we know this patient is internally bleeding?’. Honestly I had no idea. I then realized the patient had a really high heart rate and extremely low blood pressure, which to my surprise was actually one of the indicators. This was my first time being surrounded by ALOT of blood. I had no clue how I was going to react. I didn’t think I was squeamish at all, however after about 15 minutes of observing bloods being taken, I felt slightly weird. So I went for a walk outside and then I was completely fine. The consultant told me the first time that he was surrounded by blood he felt extremely squeamish but now as a consultant, it doesn’t bother him at all. From this, I learnt that it is completely fine to not be ‘ok’ with the practicality of being a Doctor at first. The sense of normality comes over time,  also surgery and other things involving blood seem less strange as Doctors are in absolute love with what they’re doing.

I was involved in two theatre sessions whilst I was there. One was removing calcification from the femoral artery, and the other was a fistula repair. Getting into the scrubs was so exciting, and I realized whilst watching the surgery that medicine is right for me and I am completely determined to do it. I was intrigued by all the different roles in the surgical team, and also completely shocked at how calm and relaxed the atmosphere was. Obviously I acknowledge that not all surgeries are like this- however the surgeons were calm and chatting to me as they operated.

I also spent time on a cardiac care unit. I learnt there that fluidity is so important in a medical team; as well as good teamwork. Every role is vital, from the consultant for medical care to the lady who does the tea and coffee rounds for wellbeing and comfort of the patients. Fluidity is important as it prevents patients waiting too long for treatment. If it wasn’t fluid, nothing would get done. Patients would be waiting hours to be taken to operations after they have been prepped, and even the well needed cups of tea would come at the wrong times. In metaphorical terms, a medical team must work as a well oiled machine.

Throughout the experience I learnt a lot about myself. The key thing I learnt is that, I am independent. I loved finding my way around the hospital and making sure I was on time (and time management is tricky for me, as I always find myself late) , confident when speaking to the consultants and making sure I was in the right place at the right time to see all sorts of medical procedures. Also I learnt that my imperfections as an aspiring medic are common and absolutely fine. For example, being squeamish when seeing blood for the first time. Imperfections exist for us to either sort them out, or accept them. I learnt that we do not progress in our skill unless we have imperfections to be fixed. However that one is to be taken with a pinch of salt, as if you have more serious imperfections and make big mistakes, that is not good.

Thank you for reading.

Research Shows ‘One Too Many’ Can Cause Fatal Heart Risk

Alcoholism. How common is it? It is extremely hard to answer that question. As many alcoholics live through their drinking abuse in silence. However just take this statistic: on average, it is estimated that the lives of five other people will be harmed when one individual becomes addicted to alcohol.

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According to Government statistics, more than 1.4 million people are dependent on alcohol in the UK. 33,000 people die each year due to alcohol-related incidents or associated health problems. Alcohol is involved in 15% of road accidents, 26% of drownings, and 36% of deaths in fires. A quarter of accidents at work are drink-related.

Realistically, every body loves and enjoys a drink from time to time, however when this becomes excessive, it can cause problems.

By definition cardiovascular disease  generally refers to conditions that involve narrowed or blocked blood vessels that can lead to a heart attack, chest pain (angina) or stroke. Thus possibly leading to death. In the UK it is estimated that 2.3 million people suffer from heart disease. New research examines the link between arterial stiffness – a significant indicator of cardiovascular health – and alcohol consumption over time.

The research was conducted by Darragh O’Neill, Ph.D., who is an epidemiological researcher at University College London in the United Kingdom. He found a link between heart disease and alchol. He found that overconsumption of alcohol can lead to stiffness of the arteries occurs when the walls of the blood vessels lack elasticity, which can negatively impact the artery’s response to variation in blood pressure.

Consistent moderate drinkers at risk were defined as those who consumed between 1-112 grams of ethanol per week, so less than the 14 units. One U.K. alcohol unit is roughly the equivalent to a shot of whiskey or half a pint of beer. So when thinking about it in terms of a night out, that is 7 pints of beer or about 11-14 shots.

Interestingly, after the longditudinal study it was found that after adjusting for several factors including BMI, heart rate, and arterial pressure, the associations were not significant for women, even though 73 percent of the study participants were men. By no means does this mean women are free to drink as much as they like, as there are many other health risks alcohol can lead to.

After vigorous research, they also found that alcoholism could lead to aging of the arteries.

The researchers finally concluded that excessive drinking can increase risk of cardiovascular disease, especially in men. They also found that constant heavy drinking can affect the arteries, thus interfering with blood flow. So I can conclude from this, that maybe its about time people really started looking into how MUCH they are actually drinking.

Thankyou.

 

Work Experience 101: Residential Care Home

In January, I began my work experience in a local care home. Since then for a few hours each week I have been volunteering at the care home, and joining the care assistants with their day to day jobs.

Before this I had never worked in a care home before. I had no clue what to expect, as the grandparents I have left aren’t in care homes, but in residential villages, which means they have a lot more independence that a care home resident. At first I was nervous, as the care home is small and there are not many care assistants and above all things I was scared the residents wouldn’t like me! However that was completely the opposite of the actual truth; the residents were all so lovely and just accepted me as a normal care assistant.

As I am only 17 and not qualified I am not allowed to take part in any personal care activities, however my activities include, helping to make tea, talking to and interacting with the residents, helping them move around the care home safely (giving assistance when getting up and down into chairs) and observing drugs rounds.

So far I have learnt a lot at the care home. Something, really special I learnt was that even in old age, despite many health problems and lists of medications that some of the residents have, it is still possible for happiness to exist. I learnt this through my various conversations with the residents. For example some of the residents like to know about what I do at school, how my friends are and comically the question I get asked most often is if I have a boyfriend or if any boys are catching my eye, which always brings a smile to my face. These may feel like simple things to converse about, but the engagement in the conversation and the smile on the resident’s face when we talk about these things really is special. I have learnt that the elderly find happiness in the simplest of things which is truly inspiring. Consequently, I believe that is an attribute that we all should aspire to have.

Even though I thoroughly enjoy my work, the experience has taught me what it is like to interact with someone that has dementia first hand. It is a harsh truth that dementia can reduce a person’s quality of life, thus it has taught me that awareness must be raised for types of dementia in the elderly. Also, in care homes, it is not uncommon for residents to come and go quickly. It is a sad thing to acknowledge however it is one of the realities of being human, that one day we will eventually die, whether we want to or not. When a member of the elderly is put into a care home, or a hospice, my experience has taught me that it is important to favor increasing the quality of life not the quantity of it. I learnt this through seeing a resident who had severe dementia, diabetes, and was very immobile due to other health problems. When her time came it was sad however the reassurance that I (and I’m sure the family) found was that the resident had a poor quality of life when they were alive, and maybe death was not such a bad thing for them.

Something I learnt about myself on this care home experience is that I love communicating with the residents which could be applied to real life patients. I was really interested in getting insight into the daily struggles and stresses that some people face with dementia, and the resilient approach to life, which was displayed by some of the residents. This has further inspired my pursuit of a medical career.

I absolutely love working in the residential home, and I am finding it so valuable, and it spurs on my inspiration and dream to study medicine.

Thank you.