The pharmas making pills tailored to your genes

Reading your genome is becoming ever easier and cheaper. That makes it possible to figure out which medicines are more likely to work for you. Dr Mike Tubbs looks at the companies best-placed to profit.

Californian twins Alexis and Noah Beery were first diagnosed with cerebral palsy in 1998. By the age of two, the pair had failed to meet various developmental milestones and their parents, Joe and Retta, were understandably very worried. After treatment started, the twins appeared to improve, but by the time they were five, Alexis in particular started to regress, and her parents noted that the symptoms didn’t match up to those of the typical cerebral-palsy sufferer.

Dogged research by Retta eventually led to a new diagnosis of a rare genetic disorder called DRD, which resulted in low levels of dopamine. Yet while treatment with an artificial dopamine drug (L-dopa) helped, other symptoms persisted and worsened. By this point, Joe had taken a job with a company that made genome-sequencing machines and, in 2010, the parents arranged to have the twins’ genomes sequenced.

The results showed that the twins had both inherited a gene variant from each parent that, together, caused them to have low levels of not just dopamine but also two other neurotransmitters – serotonin and noradrenalin. As a result, the twins were treated with both L-dopa and a supplement called 5-HTP, a precursor for serotonin. This has enabled the twins to live normal lives and take part in sports – something that was inconceivable after the original diagnosis of cerebral palsy, and before the correct treatment was started. The Beerys provide a striking example of the power of personalised medicine – the use of advanced diagnostic techniques to produce far more precise and effective treatments tailored to an individual’s specific circumstances. And the good news for both patients and investors is that the field is coming on in leaps and bounds.

New diagnostics from smaller companies

Several smaller companies are also developing new diagnostic techniques and collaborating with smaller drug companies on new treatments. An example is Aim-listed company Oncimmune Holdings PLC (LON:ONC) with its EarlyCDT liquid biopsy platform technology (which uses a blood test to detect cancer, rather than a more invasive surgical biopsy). It is commercialising its EarlyCDT-Lung test, which can detect lung cancer up to four years earlier than other tests. It hopes to commercialise its liver test later this year. Oncimmune is cooperating with Scancell on the latter’s melanoma (skin cancer) immunotherapy, SCIB-1. A trial of 16 patients with melanoma showed that the Oncimmune test predicted, with above-80% accuracy, those patients that would respond well to Scancell’s melanoma treatment. Two-thirds of the patients immunised with this vaccine are “alive and well” five years later. A Phase II trial is expected to start in the latter half of this year.

Another Aim-listed company, Angle, is developing its Parsortix liquid biopsy technology for the early detection of ovarian, breast and prostate cancer. And Oxford Biodynamics PLC (LON:OBD) is developing its EpiSwitch product platform both for early diagnosis and for distinguishing between responders and non-responders to a particular drug. Recent publications concern the application of this technique to rheumatoid arthritis and neuro-degenerative diseases.

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