Contact: Dr. Marloes Tijssen, TroMegaTherapeutics (formerly at Haematology)
Mentor: Dr. Bill Matthews

Essential Thrombocythaemia is a chronic disease with increased blood platelet numbers and affects over 9000 patients in the UK, but has no specific treatment. Patients can survive up to 20 years post-diagnosis on current treatments, but these have many side effects and do not work consistently. Typically patients are diagnosed when they are over 55, but increasingly young women in their 20s are diagnosed with the same condition. The condition gives a ten-fold increase in the incidence of thrombotic events such as strokes and heart attacks.

The inventors have identified a protein that specifically regulates platelet formation, and have also identified a compound that can inhibit the function of this protein. This compound therefore represents a novel therapy that could reduce the platelet count in patients with Essential Thrombocythaemia without the side effects from the current broad acting drugs. The next stage is to raise funding to allow them to develop this compound further.

In addition, the inventors expect that the same treatment can be used for cardiovascular disease, since there is a proven link between the risk of stroke and genes that increase platelet count. This is a much larger market, with 100,000 patients a year suffering strokes in the UK, and typically 70% surviving the first acute event. These patients are then treated with aspirin to reduce platelet activity longer-term but, despite this, the risk of another stroke stands at 30% over the 5 years post initial event.

The role for the i-Team is to investigate the market need for a specific treatment of this sort, and to establish the constraints around its future adoption. The i-Team should also look at what experimental data is needed to prove sufficient efficacy for the next stage of the treatment’s development to be fundable.