An 18-year-old suffering with congenital myasthenia, an inherited neuromuscular disorder, took salbutamol and after a week the teen stunned doctors by being able to walk unaided after being wheelchair-bound for seven years.
Jimmy Webster, from Cardiff, had been suffering to such an extent that he had required an oxygen mask to breathe on occasions.
On taking salbutamol, Mr Webster commented: “Within three days I could stand and within a week I could walk.” Next on the agenda for Jimmy is a camping trip. He said: “I wouldn’t have contemplated this last year.”
Salbutamol is a fast-acting medicine used in the treatment of asthma, bronchospasm and is also sometimes used for delaying the delivery in women going through a premature labour. The drug is a beta-2-agonist, and works by relaxing the muscles within air passages of the lungs. This means the airways are kept open and it is much easier to breathe.
However, Jimmy’s experience could pave the way for salbutamol being a main treatment option for more of the estimated 12,000 Brit’s with myasthenia.
Myasthenia can either be inherited, or caused by an autoimmune disorder. This means that the immune system – usually working to protect the body from infections – mistakenly attacks its own tissues.
Those with the autoimmune type of myasthenia have a fault in the transmission of nerve messages from the nerves to the muscles. This results in the muscles not being stimulated correctly and therefore do not tighten (contract) sufficiently, becoming easily tired and weak.
Muscle weakness is a particularly common symptom of the disease, but there are a range of symptoms – which may appear suddenly. Weakness in the eye muscles is often the first symptom, with one or both of the eyelids drooping, and there may also be blurred or double vision.
For some, the muscles around the mouth are affected first, resulting in problems with swallowing (dysphagia), chewing and even talking. Impaired speech and a nasal-sounding voice then usually follow.
Like in Jimmy’s case, some people with the condition can have breathing difficulties, especially during exercise or lying flat, and in some extreme scenarios, myasthenia can cause total paralysis.
Previous studies that shown a similar drug – ephedrine – contained in cold and flu remedies, could occasionally be effective for treating myasthenia. For example, there was another teenage patient who ended up being able to go out jogging and do sit-ups after previously having to rely on crutches to walk.
Therefore, Professor David Beeson at the Weatherall Institute, of Molecular Medicine, Oxford University, wanted to find other drugs that could be just as effective, if not better. Salbutamol was one of the medicines analysed.
Salbutamol may be used in conjunction with other drugs or standalone. Professor Beeson commented: “It is not yet licensed for use in the condition, but clinicians are happy to prescribe it.”
He advised Jimmy to try salbutamol together with pyridostigmine, a treatment he had been on since the age of four.
“I expected I’d get stronger, but had no idea I’d be able to walk again,” says Jimmy. “I still use my wheelchair out and about, but at home I walk around – just getting dressed, washing and cooking, it’s amazing to be able to normal things.”
Professor Beeson is delighted with Jimmy’s progress, adding: “It is incredibly rewarding to see results like this. Over the last couple of years we have seen patients rise from their wheelchairs within months, but Jimmy’s quite exceptional.”
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