The medical breakthroughs that may save your life

Science may not have a cure for cancer – yet – but the likelihood of one being discovered may be just around the corner. Ingrid Pyne gives a rundown of the latest scientific breakthroughs changing and saving lives.

Groundbreaking 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 350,000-plus Australians who have the disease.

Compiling a definitive list of future medical game-changers is, therefore, impossible. So, The 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

Until AFL star Jarryd Roughead underwent immunotherapy to tackle recurring melanoma last year, most Australians had never heard of the cancer treatment, which uses the body’s own immune system to help fight the disease. Yet scientists at the Garvan Institute of Medical Research predict this revolutionary treatment 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.

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 courageous move, the film star splashed the subject of genetic testing for disease prevention across the 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

This approach is aimed at both preventing disease and tailoring treatments.”

studies that show how “genomic risk” can be used for early detection and prevention programs, such as in cancer. Beyond 2017, it will become more common for doctors to use genomic information as a first-line approach to the diagnosis of diseases.

Doctors should then be able to determine how best to treat patients. By reading the clues in individual genome sequences, they will be able to advise which drugs patients are most likely to respond to.

Big Data perspectives

The past decade has seen huge advances in the amount of data we routinely generate, as well as our ability to integrate, curate, analyse, understand, store and share it. The intersection of these trends is what we call “Big Data” and the healthcare sector (and so all of us) will be one of its main beneficiaries.

Until recently, the huge amount of data collected by the medical industry has been

siloed in archives controlled by different hospitals, surgeries, clinics and universities. Now, using advanced computing techniques, doctors can share all types of data – from symptoms and medications to test results and responses to medicine – to improve care.

Big Data approaches, such as computer algorithms, can also detect patterns and trends to predict epidemics, improve quality of life, avoid preventable deaths and even cure disease. Data-sharing arrangements between the pharmaceutical giants, for example, led to the discovery that a little used antidepressant might be able to cure some types of lung cancer. By using algorithms to analyse extremely large genetic and biological databases in this way, we should be able to find fresh uses for known drugs, accelerating the development of new treatments.

Experts say it usually takes a decade and about $1.3 billion to turn a laboratory finding into a successful drug treatment –

Big Data breakthroughs could cut this to two years and about $130,000.

Blood tests for cancer

Experts say it’s only a matter of time before diagnosing and treating cancer will be as routine as an annual check-up, thanks to so-called “liquid biopsies”. These simple blood tests look for cell-free circulating tumour DNA (ctDNA), which is shed from a tumour into the bloodstream and is more than 100 times more abundant in the blood than tumour cells.

Studies are still being conducted, but this technology may do away with invasive tissue biopsies. Several medical companies are developing test kits to hit the market next year and analysts expect huge demand, tipping annual sales of about $13.1 billion.

Hope for depression

In any year, one million Australians suffer from depression and for a third, traditional anti-depressants don’t really work. Their only option is intensive treatments, such as electroconvulsive therapy.

In 2013, a study to see if ketamine – which is commonly used for anaesthesia and, less commonly, as a party drug – could alleviate treatment-resistant depression (TRD) produced overwhelmingly positive results: 70 per cent of TRD patients reported improved symptoms within 24 hours of being injected with a low dose of ketamine. As a result, the FDA expedited the development of new medications based on the ketamine profile and some, such as esketamine, are expected to be available to US patients shortly. There are 3000 deaths by suicide in Australia each year – more than eight per day – so the need for an effective treatment for severe depression is critical.

Self-administered HPV test

In Australia, as in other developed countries, there have been huge strides in the treatment and prevention of the human papillomavirus (or HPV), the main cause of cervical cancer.

Women here are urged to see their doctor for regular Pap tests, while the free national HPV vaccination program has been in place since 2007 (for schoolgirls) and 2013 (for schoolboys). Yet experts claim that the biggest prevention effort to date is about to take shape, through the large-scale deployment of self-administered HPV tests.

These tests – which include a test tube, a swab and a mail-in box – would allow you to administer the test and process the results in the privacy of your own home, rather than the discomfiting atmosphere of a doctor’s surgery.

Bioabsorbable stents

There will be a total shift in the line-up of drugs for diabetes.

Every year, more than 20,000 Australians have metal stents inserted into their chests to treat coronary artery blockage. More often than not, the stent remains there forever, long after its mission has been accomplished. The stents can make some scans and future surgeries tricky and lead to blood clots.

Yet what if they could just vanish? The first bioabsorbable stent was approved in the US last July. Made of a naturally dissolving polymer, it widens the clogged artery for two years before it’s absorbed into the body in a manner similar to dissolvable stitches. Experts assembled by the Cleveland Clinic are hailing these stents as one of the top medical breakthroughs of 2017.

Mini pacemaker

It took Earl Bakken, founder of US medical devices company Medtronic, just four weeks in 1957 to craft the first battery-powered pacemaker. His design barely changed for 50 years, but last year, Medtronic’s new model, the Micra, which is one-tenth the size and so small it can rest inside the heart itself, became the first of the next generation of pacemakers to be approved by the FDA. Unlike traditional pacemakers, which are implanted under the skin using an invasive procedure, the Micra is slipped through the femoral artery via the groin using a catheter and docked inside the heart’s right ventricle. It operates without electrical wires that can break or get infected.

No more swallowing pills

Many people need to take medication every day, but find it difficult to swallow. Soon, this could be a thing of the past. In 2015, the FDA approved a new type of pill that is 3D-printed and dissolves into liquid as soon as you take a sip of water. The pill, epilepsy drug Spritam, hit the US market last March. Its creators are looking at other disease areas, so more rapidly disintegrating drugs could be coming to a 3D printer near you in 2017.

The Micra is so small it can rest inside the heart.