A Stitch in Time; Why too much research is never enough.

Thirty years ago yesterday, I married Mark Parrilla, with much hope that he and I could beat his recent diagnosis of Chronic Myeloid Leukemia (CML).?While he had a roughly 20% chance of surviving, we believed with 4 degrees and 2 university medals between us, Mark would surely be one of the lucky ones! We were disciplined, capable of sticking out tough treatment regimens and new treatments with interferon were promising. Early results of treatment showed he had a positive response. His haematologist thought a cure would be available in the next decade.

Just four years later, Mark was dead, aged 32. The following year, final-stage CML patients were given a new breakthrough targeted treatment, Gleevic, with miraculous results. By 2001 Gleevic was approved by the FDA, and CML patients went from having a life expectancy of 3 to 5 years to effectively being cured. Initial results showed 95% of sufferers achieving a five-year survival rate!

While I was happy to see there was now an effective ‘cure’, I felt deflated that it had come just a couple of years too late for Mark. While finding this ‘silver bullet’ in fact was based on decades of research into CML, Mark’s experience shows the time-sensitive nature of research, development and translation of treatments for patients and their families.

Of course, history is littered with both miraculous and incremental improvements in treatments for a huge array of ailments and diseases, especially over the last century.

For example, one hundred years ago, on 11 January 1922, a 14-year-old boy called Leonard Thompson received the first insulin injection to treat Type 1 diabetes (www.diabetes.org.uk). Prior to the discovery of insulin in 1921, diabetes sufferers had no effective treatment, other than a strict diet, which usually only extended their lives by a few extra years! The 3,100 Australians that were newly diagnosed with Type 1 diabetes in 2020 (AIHW), would have life expectancies of 3 years if insulin treatment was not available!

?Continued medical research, development and translation is the great hope for those suffering from poor health. In Australia, Government is by far the main funder of such research which is why the establishment of the Medical Research Future Fund (MRFF) in 2015 was so important. Now $20 billion in size, it effectively doubled the Federal Government's annual contribution to health and medical research.

?Funding from the MRFF has been used in many areas of R&D, including funding the establishment of a registry of patients with congenital heart disease, under the auspices of HeartKids. HeartKids, which I have the privilege to chair, provides support to people with childhood-acquired heart disease, and their families, to help them live their best lives. This community thrives on peer-to-peer and professional support, information, and on the hope that future research and development.

#medicalresearch #MRFF #cancertreatment #HeartKids #diabetescare #insulin #leukemia