CHC Newsletter Eigth Issue, July 2018

Welcome to the 8^th issue of the Clinicians Health Channel Newsletter.

In this issue:

New Uses for Old Drugs

British Medical Journal (BMJ) has recently published an interesting editorial on drug repurposing, this is a promising field in drug discovery that identifies new therapeutic opportunities for existing drugs. Pharmaceutical companies pursue this strategy to increase their productivity (new drugs to market) by reducing the discovery and development timeline. This decreases the overall cost of bringing the drug to market because the safety and pharmacokinetic profiles of the drugs are already established.

One such example is the ReDO project (http://www.redo-project.org) which has compiled a list of more than 250 non-cancer drugs for which there is pre-clinical and clinical evidence of anti-cancer action.

Pharmaceutical Benefits Scheme (PBS) listing to help kids with Spinal Muscular Atrophy

Spinal Muscular Atrophy (SMA) is a genetic disease caused by a shortage of a protein called survival motor neuron (SMN). This results in the loss of nerve cells in the spine, leading to weakness of the muscles in the shoulders, hips, thighs and upper back.

SMA occurs when a faulty SMN1 gene is passed on to a child by both parents. The SMN1 gene is responsible for the body’s production of SMN protein, which is vital to the function of muscles that enable movement, speech, breathing and swallowing. Without ample production of SMN protein, the nerves of the spinal cord deteriorate and muscle wasting gradually occurs. 5q SMA is the most common form of the disease and represents approximately 95% of all SMA cases.

There are four known forms of SMA – Type I, Type II, Type III and Type IV – each determined by the age of onset (from those diagnosed before six months of age to those diagnosed in adulthood) and the physical milestones achieved.

In the past the treatment of SMA has been symptomatic, based on comfort measures and management of complications of weakness, feeding and breathing difficulties. However, the landscape for SMA patients is radically changing with the identification of a novel therapeutic agent, Nusinersen, which has been shown to improve motor development in babies and children with SMA types 1 and 2. Nusinersen marketed as Spinraza, was registered on 3 November 2017 with the Therapeutic Goods Administration (TGA) as a treatment for SMA, is marketed by the pharmaceutical sponsor Biogen Australia Pty Ltd (Biogen). The drug has not previously been available in Australia, other than to babies with type one SMA receiving it in clinical trials or as part of an extended access program sponsored by the drug manufacturer.

From 1 June 2018 SPINRAZA ^® (Nusinersen) is available on the PBS for the treatment of paediatric patients (18 years and younger) with infantile-onset or childhood-onset of Spinal Muscular Atrophy with onset of symptoms prior to three years of age.

It is hoped the drug, administered by spinal infusion, will halt the progression of the disease or even reverse muscle loss to improve patients’ motor function.

The PBS listing has reduced the cost for patients to a maximum of $39.50 per script for general patient, and $6.40 for concessional patients. Otherwise patients would have to pay $367,850 a year for this medicine.

Additional research information on single-dose gene-replacement therapy for Spinal Muscular Atrophy was published in the New England Journal of Medicine.

Fluids in Surgery: Good or Bad?

A global study led by Alfred Health has found giving patients more intravenous fluids during surgery can reduce the risk of kidney damage and wound infection post-surgery.

While small studies had previously indicated that limiting IV fluids was beneficial for patients undergoing abdominal surgery, the restrictive versus liberal fluid therapy in major abdominal surgery (RELIEF) trial – conducted across 47 hospitals in seven countries – proved otherwise.

The RELIEF trial enrolled 3000 people having all types of major abdominal surgery over the past six years. Half of the patients were given limited IV fluids and the other half were given liberal fluids. All study participants were followed up to a year after their operation.

A more liberal amount of IV fluids protects against kidney damage and reduces the risk of wound infection after surgery. The implications for patients are significant – the study has proved that administering more fluids will lead to substantially better outcomes for patients in the long term.

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Resources Updates:

New therapeutic group in the Women’s Pregnancy and Breastfeeding Medicines Guide

A new therapeutic group Anthelmintics has been added in the Women’s Pregnancy and Breastfeeding Medicines Guide. It includes general advice about treatment of worms during pregnancy and breastfeeding. Several types of worms that can cause problems in pregnant women, including threadworm (or pinworm), whipworm, tapeworm, roundworm and hookworm. Of these, threadworms are the most common in Australia. More information on drugs, treatment for threadworm, hookworm and roundworm infestations in pregnant women can be found in this section.

Updated cardiovascular guidelines in Therapeutics Guidelines (eTG Complete)

The eTG Complete has recently updated Cardiovascular guidelines. It includes – patient-centred therapy to optimise treatment and prevention of common cardiovascular conditions. The topic on – lipid modification advises on the use of lipid-modifying drugs for the primary and secondary prevention of cardiovascular disease, including new information about high-intensity statin therapy. Practical advice on managing hypertensive urgency and emergency, a discussion of high-potency statin therapy, and an overview of pulmonary hypertension.