Why Take Part in a Drug Trial?
Without clinical trials, many of the medicines we rely on today would not exist. Clinical trials ensure that medicines are generally well tolerated and effective, and that new, innovative treatments are continuously being developed for people with different illnesses.
As a scientist and medical nerd, I have offered my blood, DNA and other data to be used in research for years. I feel that as a person with many different chronic disorders and health issues, it is important to help, (even in a small way), those who will be diagnosed in the future, as there is very little research and knowledge about many of the things I suffer with.
"Clinical trials are at the foundation of some of the biggest medical advances seen over the last four to five decades. Novartis is committed to using science-based innovation to improve research and develop innovative medicines for some of the most difficult-to-treat diseases, with many studies being conducted in the UK."
My particular type of sickle cell (HbSC) is very often NOT included in the little research that there is out there. Until recently. Those leading the trial believe that the severity of sickle cell disease and various other factors make it important to understand differences in response of various subgroups of patients in order to increase understanding.
So in January 2020, I became only the second person in the whole of the UK to take part in a phase III clinical trial (multicentre, multinational, randomised, placebo-controlled, double-blind). This means I have a number of blood test, ECGs, and infusions (of the drug or placebo), urine analysis and have to keep a daily questionnaire diary.
The drug has already received approval from the US Food and Drug Administration (FDA). This means it is commercially available in the US (priced between $84,852 and $113,136 per year for most patients). In Europe we have different standards for approving drugs hence this addition phase in the trial. The cohort of the trial are split into 3 groups; placebo, low dose and high dose.
What does the drug do?
In sickle cell, a lot of the pain experienced is caused when the sickle-shaped red blood cells block blood flow through the blood vessels. P-selectin is a protein that is found on the surface of endothelial cells (the cells that line the inner walls of blood vessels) and in platelets (blood cells that are involved in clotting). P-selectin normally works to control the flow of white blood cells through blood vessels and how they adhere to blood vessel walls during periods of inflammation and tissue repair, such as after an injury.
In sickle cell disease, P-selectin contributes to the adhesion of sickle-shaped red blood cells (cells with an abnormal crescent shape) to blood vessels, preventing blood flow through smaller vessels. This causes inflammation and vaso-occlusive, pain crises.
The drug being trialled uses a monoclonal antibody (an antibody produced by a single clone of cells consisting of identical antibody molecules) to bind P-selectin on to the surface of endothelial cells and platelets in the blood vessels, causing a blockade of P-selectin. By blocking or inhibiting P-selectin, the drug prevents this adhesion molecule from starting the process that leads to blood vessel occlusion, inflammation, and pain, and helps to maintain normal blood flow.
This would mean that people with sickle cell would experience less pain crises (vaso-occlusions which usually lead to hospital admission because they can cause severe pain, strokes and acute chest syndrome). In addition this may also mean that those with sickle cell and chronic pain disorders could experience less pain overall.
After the minimum cohort number has been achieved and have completed the first section of the trial, we find out if we have been on the low/high dose or the placebo. Eventually all participants will be given access to the drug.
It is an exciting prospect but a long process. It is time consuming but worth it. I have been know to be weighed, have blood tests and cannulas fitted in my lunch breaks, return to work for a few hours and then go and have my infusion : D
Further information for those who may want to consider taking part in the trial...