We are very grateful to Dr Carmel Nanthakumar, Scientific Director and Science Fellow at GlaxoSmithKline, for coming to give a talk to Habs Girls and Boys in Years 11-13. Dr Nanthakumar spoke to us about her work leading a team of cell and molecular biologists to identify the next generation of medicines. In particular, we heard about her research into idiopathic pulmonary fibrosis, a fibroproliferative disease that causes scarring of the lungs. Because this is a rare condition, it is classed as an orphan disease, which means that researchers are given incentives to find new treatments, such as the protection of any new drugs they develop from market competition. In addition, patients often present late with the condition, meaning their remaining life expectancy may only be a few years. Therefore, scientists are trying to find a particular molecule involved in the disease that they could target with a new drug. Transforming growth factor beta (TGF-β) is a pro-fibrotic cytokine that has been identified to promote excessive extracellular matrix deposition, leading to scar tissue. Dr Nanthakumar hopes that by developing a drug that prevents TGF-β binding to receptors, the team can improve the prognosis of patients with idiopathic pulmonary fibrosis. Ideally any drugs would be taken orally to minimise systemic effects by only interacting with target cells of the lungs.

Dr Nanthakumar also gave us insights into how the team can discover whether or not a drug is having the desired effect. By studying detailed signalling pathways, scientists can monitor levels of proteins downstream of a substance binding to receptors. For example, the enzyme p13 kinase initiates signalling after TGF-β binds, so lower levels of this protein or others it activates would indicate a drug was successfully blocking TGF-β from binding. Another method used is autofluoresence, which uses the natural emission of light from structures in cells to determine how much collagen is present in a deep tissue slice. This gives an indication of the extent of scarring because collagen is deposited by myofibroblasts as scar tissue is formed.

Dr Nanthakumar clearly explained the processes involved in bringing a drug to market – through pre-clinical and clinical trials, including the importance of precise dosage so that a drug is effective and not poisonous. She answered a wide-range of questions from students following her presentation and motivated us to take our scientific studies further.

by Fleur U6