MAY 2017 99
»Tailor-made medicine
Four years ago, Angelina Jolie announced to
the world she’d had a double mastectomy
to prevent the scourge of her family’s breast
cancer. In that courageousmove, the film star
splashed the subject of genetic testing for
disease prevention acrossthe front pages.
Single gene tests for disease risk – such as
the BRCA1 gene that Angelina carries – have
been around for some years. Yet, increasingly,
it is becoming possible to estimate people’s
individual risk of a whole range of diseases
by looking at patterns across their genome
sequence (the six billion base pairs of DNA
they carry in every cell). Call it what you will- personalised, genomic or precision medicine
- this approach is aimed at both preventing
disease and tailoring treatments.
The Garvan Institute predicts that, this year,
we are likely to see clinical proof-of-principle
studies that show how “genomic risk” can
be used for early detection and prevention
G
roundbreaking medical
innovations this year will
change our lives – and hopefully
even save them. Some of the
anticipated breakthroughs
seem to take us beyond the realm of science
and into science fiction territory (think
3D-printed pills or gene editing), while
others have less of a “wow” factor, but will
still offer much-needed relief to sufferers.
Yet medical innovation is a risky business.
For every success story – such as last year’s
stunning launch of a new hepatitis C cure –
there is a giant scrap-pile of shattered hopes.
Late last year, for example, pharmaceutical
giant Eli Lilly had to abandon a promising new
Alzheimer’s disease treatment at the 11th hour,
when it failed to meaningfully beat a placebo in
late-stage clinical trials, so dashing the hopes of
50,000 New Zealanders who have the disease.
Compiling a definitive list of future medical
game-changers is, therefore, impossible. So
The Australian Women’s Weekly spoke to
scientists, policy makers and pharmaceutical
industry insiders to bring you some of the
breakthroughs just over the horizon that may
have a profound impact on your health in the
year ahead.Immunotherapy for cancer
Scientists at the Garvan Institute of Medical
Research in Australia predict immunotherapy- a revolutionary treatment which uses the
body’s own immune system to help fight
cancer – will be the medical story of 2017.
Initially, immunotherapy was used to tackle
melanoma, then a type of lung cancer. Now, it
is being used (or tested in clinical trials) to treat
a broad range of malignancies, such as bowel,
pancreatic and bladder cancers. Scientists hope
that immunotherapies may one day prove to
be a “cure-all” for cancer, in much the same
way that penicillin is the panacea for infections.
This year, the US Food and Drug Administration
(FDA) is expected to approve the drug for the
treatment of acute lymphoblastic leukaemia,
triggering a wave of approvals for the treatment
of other blood cancers and lymphomas.
Australia’s Therapeutics Goods Administration
(TGA) tends to follow the FDA’s lead.
Scientists have long viewed immunotherapy
as the holy grail of cancer treatments, but it’s
proved incredibly difficult to make it work.
Now, many oncologists believe we may have
cracked it. While still used in conjunction
with chemotherapy, it’s hoped it will supplant
chemo, along with its horrific side effects.
Genetic microsurgery
Five years ago, researchers proved that they could perform microsurgery
on genes, using a protein called Cas9 as a “scalpel”. This gene editing
technique, known as CRISPR, set off an explosion of research, with
scientists using it to manipulate specific genes in mice, rats, bacteria,
yeast, zebra fish, fruit flies and plants.
Now, the first trials of gene editing in human embryos are underway in
China. Scientists there have reported manipulating the genes of embryos
to make them resistant to the HIV virus and also have edited genes
linked to the sickle cell blood disorder.
Other trials have been approved in the US, and the Garvan Institute in
Australia expects that these trials will really take off this year. This means
that, in the future, we could be deleting – rather than treating – diseases.“This approach
is aimed at both
preventing disease
and tailoring
treatments.”