Patient with rare bleeding disorder becomes first to test new treatment

Each year, thousands of participants come to Richmond Pharmacology’s London Bridge clinic to take part in drug trials. With their help, since its foundation in 2001, Richmond has undertaken some 500 early-phase studies and contributed to delivering 30 medicines to market - including drugs for heart failure, hypertension and cancer. But what is it like to take part in a trial? And how does it feel to be the first to test what could be a ground-breaking new treatment?  

Rizwana was diagnosed with Glanzmann Thrombasthenia(GT) when she was a baby. GT is a rare but severe bleeding disorder caused by platelets in the blood not working properly to form clots. Approximately 50% of the patients report one bleed every day and 13% report more than 500 bleeds per year. Currently, there is no cure and sometimes the only way to stop a bleed is to go to hospital for treatment.

Rizwana’s childhood was overshadowed by a fear of getting hurt and she suffered frequent bleeds. As an adult, she is constantly having to explain her condition, even to doctors, most of whom are not familiar with GT. When she was involved in a bad car accident, it took the person who was with her hours to persuade the medics that she needed to be taken to the one hospital where she was known and where she would get the appropriate care.

But in November 2023, Richmond Pharmacology embarked on a first-in-human trial of a new therapy aiming to prevent and reduce bleeds and invited Rizwana to be a participant. The treatment involves a simple injection of a particular type of antibody intended to help bind platelets together and enable blood to clot normally. If the treatment is licensed, patients will receive regular injections.

Rizwana now finds herself the first person in the world to have been tested with a potential new treatment for GT. Richmond continues to monitor her progress and is focused on including more patients in the trial to explore the potential of this new treatment and ultimately to make successful treatments widely available to patients fast.

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