Preclinical and clinical evaluation of forodesine in patients with leukaemia

Forodesine is a drug that has been used in clinical trials to treat patients with chronic lymphocytic leukaemia (CLL) but has shown limited efficacy in humans despite promising in-vitro studies.

Scientists at the University of Oxford have demonstrated greater efficacy of the drug in patients with CLL that have a specific genetic mutation compared to those who do not. It is expected that CLL patients with this mutation will have a higher response rate to the drug which will ultimately improve treatment efficacy and increase life expectancy.

Chronic lymphocytic leukaemia (CLL) is a type of cancer that affects the white blood cells and tends to progress slowly over many years. In patients with CLL, the bone marrow makes too many lymphocytes. Symptoms include higher levels than unusual of bruising and bleeding, night sweats, high temperatures and swollen glands. It is one of the most common types of leukaemia in adults and usually occurs around or after middle age with about 90% of chronic lymphocytic leukaemia diagnosed in middle age.

The incidence rate of CLL is higher in men and women over 50 years of age. It is rarely seen in people under age 40 and is extremely rare in children Over 3000 people are diagnosed with CLL every year in the UK alone and in 2016 there were over 18,000 new cases of CLL in the US.

The CLL market is expected to grow from $7.7bn in 2017 to $9.2bn by 2027 at a compound annual growth rate (CAGR) of 1.8%. Treatment for CLL largely depends on what stage the condition is at when it’s diagnosed. The increasing elderly population worldwide is driving market growth and there is a strong need for more effective treatments.

New treatment in CLL

Researchers at Oxford University have identified a specific use of the drug forodesine to treat CLL in patients with a specific genetic mutation.

Scientists have demonstrated greater efficacy of the drug in patients with CLL that have a specific genetic mutation compared to those who do not. It is expected that CLL patients with this mutation will have a higher response rate to the drug which will ultimately improve treatment efficacy and increase life expectancy.

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