The potential for personalised medicine is being realised and your students could be the next generation of researchers that make it accessible.

This article in the NY Times really brings home what we as secondary instructors teach our students and the very real implications it has for improving global health and well-being through the cultivation of young curious minds.

Eight year old Mila Makovec suffers from Batten disease. This disease consists of a rare group of inherited conditions that affect the nervous system causing seizures, blindness and eventually death. Batten Disease is a recessive condition, but Mila's disease was progressing despite only having inherited one of the disease causing alleles. Doctors identified an extra piece of mRNA being transcribed that was causing a key neurological protein to be produced incorrectly. Their solution? To produce a custom piece of RNA to block the extra DNA.

Since receiving the drug, Mila has experienced relief from the frequency and intensity of her seizures and has regained some control over her ability to swallow and hold herself upright. The research, which is spearheaded by Dr Timothy Yu and his colleagues of Boston Children's Hospital, continue to investigate Mila’s illness and offers a new avenue for treatment possibilities for others like Mila.

What are the ethical implications?

Information and treatment: a question of access.

As the field of personalised medicine grows, numerous scholars and scientists have considered its social, legal, and ethical implications. A central concern is access. Who gets access to this kind of treatment, what that means on a wider social scale with regards to disparity and discrimination, and who gets access to genetic data given that genetic data and other sensitive health information is required for this technology to work?

Access to private health information raises concerns about stigma, discrimination, and personal autonomy and the objections are not dissimilar to those surrounding the My Health Record distrust. Brothers and Rothstein (2015) suggest that for personalised healthcare to be beneficial, the inclusion of a wider range of genetic information-including that of previously under-researched populations is necessary, yet, the reasons for certain populations being underrepresented are complex. Ensuring that personalised medicine is available for all is part of the motivation and ethical agenda of leaders in the field (Colins et al 2003), however the future has yet to be determined around this specific issue of access.

The Technology

RNA Interference

“RNA interference (RNAi) is a biological process in which RNA molecules inhibit gene expression or translation, by neutralizing targeted mRNA molecules…Since the discovery of RNAi and its regulatory potentials, it has become evident that RNAi has immense potential in suppression of desired genes. RNAi is now known as precise, efficient, stable and better than antisense technology for gene suppression. However, antisense RNA produced intracellularly by an expression vector may be developed and find utility as novel therapeutic agents.” (Information accessed from Wikipedia Jan 2020).

Personalised Cancer Treatments In Australia

Ben Tran of the Walter Eliza Hall Institute and Jamie Fletcher at the Children’s Cancer Institute in Australia are working toward personalising cancer treatments based on the genetic profile of a cancer. Rather than just looking at genetics these researchers grow cancer cells from specific cancers in culture and if they can’t grow them in culture, they graft them to a mouse so the cells will proliferate. They then test a range of chemotherapy treatments to find the most effective for each specific disease. Not only does this enable them find the most effective therapies it gives them greater insight into the process of proliferation and treatment so they can better understand cancer on a larger scale.

RNA interference (RNAi) is an important process, used by many different organisms to regulate the activity of genes. This animation explains how RNAi works a...