Promising new treatment for respiratory syndrome

14 February 2014

Image of a person's hand with a drip feed

A new treatment for patients with acute respiratory distress syndrome has seen mortality rates plummet by about 80 per cent.

The results come from a phase II trial led by Dr Geoff Bellingan at UCL Clinical Physiology and medical director at University College London Hospitals.

Acute lung injury (ALI) and its more severe form, acute respiratory distress syndrome (ARDS), are major causes of death in intensive care units.

It is estimated that approximately 300,000 to 400,000 people suffer from ARDS in Western countries each year and the mortality rate remains high, around 35-45 per cent, despite modern day care.

At the moment there is no effective drug treatment for the syndrome, so patients tend to be treated with mechanical ventilation and optimisation and support of vital functions.

Major causes of ARDS include direct lung injury such as lung infection (pneumonia), aspiration pneumonia or indirect injuries such as severe sepsis, major multiple trauma or pancreatitis.

But a clinical study to test the new drug developed by Faron Pharmaceuticals Limited, demonstrated an astonishing 81 per cent reduction in mortality in ARDS patients, with a mortality of only 8.1 per cent in patients treated with Traumakine, compared to the 32.2 per cent mortality seen in the control group of ARDS patients.

All other measured parameters, including length of mechanical ventilation needed, length of ICU stay and support of vital functions also clearly benefited from the treatment.

The trial was conducted in eight intensive care units around the UK and was led by a team of scientists and clinicians which included UCL professor of intensive care medicine Hugh Montgomery and UCL/UCLH intensive care clinical lead David Howell.

The results are published together with the molecular studies of Professor Sirpa Jalkanen from Finland's University of Turku.

"Pulmonary vascular leakage occurs early in ALI/ARDS, and mortality remains high. An effective pharmacotherapy is desperately needed," said Dr Bellingan.

"Traumakine has been shown to reduce capillary leak and we were very happy to see this translate into a benefit in ARDS patients as predicted. The drug promotes the formation of a local anti-inflammatory molecule and improves the amount of oxygen entering the blood stream from the air," he said.

Meanwhile Dr Bellingan said he and his team were looking forward to starting further clinical investigations.

Faron has been granted orphan drug status for the treatment of ALI/ARDS with FP-1201-lyo (TraumakineĀ®) by the European Commission and European Medicines Agency (EMA) and is waiting for EMA advice to start phase III studies.

"We are very pleased to receive the FPCLI001 trial results and related information published in this prestigious journal", said Faron's CEO Markku Jalkanen.

"We hope that this publication promotes our efforts to attract active intensive care sites and clinicians to join our next step, the pan-European phase III pivotal trial starting in 2014," said Professor Jalkanen.

In addition to its high rate of mortality, ARDS is also a major economic burden to hospitals and health care budgets.

It is estimated that due to a long ICU and hospital stay, the cost of every saved life from ARDS is approximately Ā£43,000. With a new efficient pharmacotherapy, hospital stays are likely to become shorter and the costs will likely diminish significantly.

The paper,  The effect of intravenous interferon-beta-1a (FP-1201) on lung CD73 expression and on acute respiratory distress syndrome mortality: an open-label study, is published in The Lancet Respiratory Medicine.

 

  • Results from a phase II clinical trial show that a new drug to treat acute respiratory distress syndrome (ARDS) could reduce mortality by as much as 80 per cent. 
  • ARDS and its less severe form, acute lung injury (ALI), are major causes of death in intensive care units. ARDS is caused by lung injuries and infections such as trauma or pneumonia - and there is currently no effective drug treatment. 
  • The drug, called Traumakine, has been granted orphan drug status for the treatment of ALI/ARDS by the European Commission and European Medicines Agency (EMA). The developer, Faron Pharmaceuticals Limited, is awaiting EMA advice to start phase III studies.