From the South Wales Echo, 10 June 2013. Story by health correspondent Julia McWatt and Cathy Owen.
Although research is ongoing into nearly all conditions, there are a number which remain more under the radar than others. Health correspondent Julia McWatt and Cathy Owen look at areas which need further investigation.
Myalgic encephalomyelitis, or ME, is a potentially chronic illness experienced by approximately 200,000 people in the UK, making the disease twice as prevalent as multiple sclerosis, systemic lupus and HIV infection. This means that people with severe illness can be counted in thousands – though their voices are rarely heard.
Dr Neil Abbot, research and operations director of ME Research UK, points out that in 2002, a report to the Chief Medical Officer, pointed out that the “severely ill are severely overlooked; just ignored and invisible.”
“That’s still true 11 years later.” he said.
“Funding smallish pilot studies is one thing, but real breakthroughs come at the end of a programme of painstaking work by a specialist group of researchers. Big money will be needed to unravel the causes and find cures for ME.
“Some medical research funding in the UK comes from larger national agencies such as the Medical Research Council and the NHS Research and Development Forum, which allocate funds to established research groups with a track record of success in a certain area, on the basis of a reasonable scientific hypothesis. But getting monies from these larger funders is very difficult.”
Ongoing research has been undertaken into treatments and cures into cystic fibrosis but it’s on a much lower scale compared to many other conditions.
In April, the Cystic Fibrosis Trust published a new five-year research strategy, setting out how it will ensure that its investments in cutting-edge research will benefit people with cystic fibrosis.
The trust says the ultimate goal for research into cystic fibrosis is to understand the basic fundamental defect of CFTR. Investments in this transformational research are “long-term and high risk: results do not appear overnight”, it adds.
But the future looks bright for those with the condition. The trust says the cystic fibrosis community may be on the cusp of a significant change towards personalised medicines to treat the condition.
Meanwhile, a life-changing drug will now be available on the Welsh NHS after Health Minister Mark Drakeford went against the recommendation of the All Wales Medicines Strategy Group, which had initially rejected the drug on cost.
Kalydeco is the first licensed drug that treats the fundamental defect in people with the G551D mutation form of cystic fibrosis – a life-threatening genetic disorder which damages the lungs and digestive system.
By correcting the genetic disorder, the drug, also known as Ivacaftor, can extend the lifespan of cystic fibrosis patients from an average of 27 years to an average life length.