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 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.
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.
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 studies that show how “genomic risk” can be used for early detection and prevention programmes, 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.
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.
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.
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 more than $13 billion.
More than half a million Kiwis have been diagnosed with depression at some time in their lives. And for some, 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 were 579 deaths by suicide in New Zealand in the 2016 financial year – more than 11 per week – so the need for an effective treatment for severe depression is imperative.
Almost a decade ago, a new wave of diabetes drugs hit the pharmacies, with promises to lower blood-sugar levels. Yet they failed to alleviate one of the biggest concerns for Type 2 diabetes sufferers – their increased risk of cardiovascular disease (CVD).
Last year, two new drugs showed signs of slashing mortality rates from diabetes related CVD. Empagliflozin modifies the progression of heart disease by working with the kidney, while liraglutide has a comprehensive effect on many organs.
In light of these results, a distinguished panel of doctors and researchers, assembled by the Cleveland Clinic in the US, predicts that, this year, there will be a total shift in the line-up of drugs prescribed for diabetes – as well as a wave of research into new avenues targeting Type 2 diabetes and its related diseases.
In New Zealand, 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 programme has been in place since 2008 for schoolgirls and began this year for schoolboys. But the biggest prevention effort to date is about to take shape, through the deployment of self-administered HPV tests in Australia.
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 at home, rather than a doctor’s surgery. The tests could be later introduced in New Zealand.
More than 170,000 New Zealanders are living with diagnosed coronary heart disease. And many of those have metal stents inserted into their chests to treat coronary artery blockage. More often than not, the stent remains there for ever, 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.
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.
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.
There is nothing more eagerly awaited in cardiovascular medicine than the results from a large clinical trial of a new cholesterol-lowering treatment.
Called PCSK9 inhibitors, this class of drugs has been shown in earlier trials to silence the gene that creates LDL – or “bad” – cholesterol. The outcome of the large Phase III trial should be known soon.
Cholesterol-lowering statins, the current standard-bearer in the fight against heart disease, have had a bad rap in recent years. While still regarded as safe and effective, their adverse side effects (such as muscle pain and weakness) cause many patients to discontinue therapy or take less than the recommended dose.
Experts say PCSK19 inhibitors, while not without side effects, could be a viable option for statin-intolerant patients. The FDA last year approved PCSK19 for high-risk patients, saying the drugs will likely be used more widely, once the Phase III trial is completed.
This is good news for New Zealand, where someone dies from heart disease every 90 minutes.
Words: Ingrid Pyne
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