Recent scientific advances into our knowledge of genetics have led to the development of targeted, personalised medicines for chronic illnesses which are creating excitement in the medical community.
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Personalised Health
Here at Amchara we champion the concept of personalised health.
At its heart, this approach believes we are all different, so no person’s journey to wellness will be the same.
We are all different because of our inherited genetic makeup, but added to this our life experiences, the food we eat and the lifestyle we live determine the extent to which we are influenced by our genetic inheritance.
By their nature medical drugs are ‘one size fits all’.
It’s never been practical to construct a totally individual drug for each person, but medicine is moving towards a more personalised approach than has traditionally been adopted in the past.
New research into the effects of our individual genetic influences is beginning to enable tailored treatments for certain health issues.
How Does Our Genome Affect Our Health?
Our genome is the complete set of our DNA and works a little like the instruction manual for our body.
Tiny differences in DNA can affect how susceptible we are to develop certain health issues; how severe a disease becomes and how effective specific treatments are.
Learning more about these differences can help scientists predict the best way to treat different diseases.
Even if we have inherited a specific tendency to develop a disease, nutritional and lifestyle factors can determine if these predispositions are expressed into the eventual development of the disease. This is known as epigenetics.
Research is now concentrating on splitting patients suffering from specific diseases into small groups depending on their genetic makeup, in order to learn more about how genes behave in a particular disease state.
Targeted Medicine for Cancer
Research in this field is tending to focus on cancer and has had some success in the prevention and prediction of the likelihood of developing the disease.
For example, mutations of the BRCA genes can increase a women’s risk of developing breast and ovarian cancer by between four and eight times.
Around one in 400 women in the UK are thought to carry this genetic mutation (1).
BRCA genes are responsible for repairing DNA damage to cells (2).
Measuring genes is now becoming commonplace in cancer care.
Mapping a patient’s genetic makeup can not only guide doctors in choosing the most appropriate treatment but it can also help to predict the likely side effects of the treatment in that person.
Looking at genes can also detect whether the cancer is becoming resistant to the treatment.
This way, scientists can not only see what is making the cancer develop but also how the body’s immune system is reacting to it.
When cancer develops, patients effectively have two genomes – their inherited DNA and the mutated genetic code in their cancer cells.
Research aims to switch genes off in cancer tumours in the lab, in order to highlight specific weaknesses which can guide subsequent treatment targeted at those errant cells.
This has great potential in cancer treatment, because current chemotherapy drugs affect the patient’s entire body, not just the cancer, which is why they produce so many side effects.
Personalised Medicine
Looking at the genome with the aim of targeting medical treatment is a huge step forward, but once we know exactly which mutations are causing a specific disease, this could enable a completely personalised drug to be administered.
If this seems years away, such a drug was recently approved for use in the NHS in England for lymphoma, a type of cancer in the infection-fighting cells of the immune system.
Patients with this type of cancer may in future receive a new treatment designed to programme their own immune system to fight the disease.
The treatment is tailor made for each patient using some of the body’s own white blood cells, known as T cells.
These are types of immune cells which travel around the body seeking out and destroying defective cells.
In the therapy, the patient’s T cells are removed from their bloodstream and genetically reprogrammed using a special type of harmless virus to encourage them to find and destroy cancer cells.
Usually, T cells are not good at recognising cancer cells so they don’t attack them.
Because the T cells are living, they remain in the bloodstream far longer than a traditional drug.
Although the treatment has not yet been used long term, clinical trials showed 40% of patients still had no signs of their terminal lymphoma over 15 months after the treatment (3).
The treatment has also been used for leukaemia, and in trials resulted in 76% survival rates after one year, with 50% of those being in complete remission (4).
Unfortunately side effects immediately following the treatment are severe, such as difficulty breathing, fever and low blood pressure.
In addition, in both these treatments, the modified T cells work by targeting a specific protein on the surface of a cancerous cells.
This protein is also found on healthy immune B cells, so the therapy does not distinguish between these cells and cancer cells.
Unfortunately this means healthy immune cells will also be destroyed, meaning it’s difficult for the immune system to fight infections.
However, since these cells make up only part of the immune system, it’s hoped it can recover over time.
The situation would not be the same with cancers in other organs which would require more precise targets to be identified.
Targeted Medicine for Other Diseases
It’s not only cancer which is being investigated in terms of developing targeted treatments.
One example is sepsis, which occurs when the immune system fights an infection, but its activity spirals out of control and attacks the body’s own organs. Sepsis is estimated to kill over 50,000 people each year in the UK.
One third of patents worldwide who develop sepsis will die from the disease.
Sepsis is generally treated with antibiotics targeted to the specific bacteria which is causing the sepsis.
However the bacteria can’t always be identified.
Instead, if research can look at gene activity in the immune systems of sepsis patients, specific aspects of the immune system which are not functioning as they should can be identified.
This would allow treatment to be targeted.
Gene Silencing
Looking at a person’s genes is becoming more and more commonplace in medicine.
Once genetic aberrations causing diseases can be identified, medicines can be developed to ‘switch off’ the expression of the gene and so halt the progression of the disease.
This form of medicine is called gene silencing and it’s now available on the NHS.
The technique is initially available for a previously untreatable disease called amyloidosis.
This affects the functioning of the autonomic nervous system causing progressive nerve and organ damage.
It leads to paralysis and can eventually be fatal. The symptoms are caused by toxic proteins which build up in the body.
Experts believe there is a 50% chance of developing the disease if a person inherits a particular genetic mutation.
Gene silencing drugs target messenger RNA (MRNA), a strand of genetic code which carries instructions to the DNA in the nucleus of our cells. The DNA then directs the functioning of the cell, like a tiny factory manager.
If the MRNA is destroyed, this effectively stops the mutated gene from influencing cells.
Gene silencing has also been shown to work for other genetic diseases in which the body erroneously makes toxic proteins, such as the neurodegenerative condition Huntington’s Disease.
Future research may concentrate on Parkinson’s and Alzheimer’s disease.
Takeaway
Development of treatments such as these are exciting for the future of personalised medicine and bring mainstream medical treatments more in line with the concept of personalised health.
We believe nutritional and lifestyle factors play a large part in determining whether our genetic tendencies are expressed and have an important role in helping our bodies to maintain optimum health.
This doesn’t need to be the end of the article. With your help let’s continue the conversation.
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