New treatment for Duchenne muscular dystrophy.
New treatment for Duchenne muscular dystrophy.

An international team led by professor Gunnar Buyse of the University Hospitals Leuven has developed a medicine that – as shown in the trial – could put a stop to the decline in pulmonary and respiratory function in patients with Duchenne muscular dystrophy. Because the disorder cannot be cured and because pulmonary disease is the cause of early death, this new treatment is a big step forward.

Decline put to a halt

In an international trial, 64 European and North American Duchenne muscular dystrophy patients between 10 and 18 years were administered a daily dose of Raxone®/Catena®  or a placebo, and this for 52 consecutive weeks. After 52 weeks, there was a significant decline in the strength of the respiratory muscles (the so-called ‘peak expiratory flow’) in the patients that were administered a placebo. At the same time, in the patient group that was administered Raxone®/Catena®, the muscle strength stabilised. The same DELOS trial also revealed a similar effect in other pulmonary function parameters.     

No side effects

At the moment there is no adequate treatment for Duchene muscular dystrophy. Professor Buyse: “It is possible to slow down the muscle weakness with glucocorticosteroids, but the numerous side effects means that many patients can’t use the drug for a long time. Moreover, the benefit of glucocorticosteroids is limited. The new drug Raxone®/Catena® however, is well tolerated and no harmful side effects were recorded during the trial.”  

Longterm research

The development of the product was preceded by a long period of research. Professor Buyse: “Ten years we started research to test the effect of idebenone in mice, together with the Swiss company Santhera Pharmaceuticals. Idebenone is the active component in Raxone®/Catena®. After testing in a small group of patients with promising results, we initiated a larger scale trial. This wasn’t easy. In the medical world, it is often felt that this type of disorder is not well-suited for state-of-the-art research. But the combination of a good hypothesis and the unique collaboration between the various parties (pharmaceutical industry, academic and hospital environment, the patients and their parents) has proven to be a key to success.”    

Duchenne muscular dystrophy

Duchenne muscular dystrophy is a congenital neuromuscular disorder that affects 1 in 3,500 boys. The first symptoms (gait problems and muscular pain) occur during the first years of life. By the age of nine, patients are bound to their wheelchair. They die at an early age because of damage to respiratory and heart muscles.

The Lancet

The results of the trial have been published on 21 April in the prestigious medical journal The Lancet, and were presented on 24 April in Washington DC by professor Buyse during the annual meeting of the American Academy of Neurology.

More information about Duchenne muscular deystrophy (in Dutch).