Experimental drug shows signs of slowing motor neurone disease

There are signs of slowing the progress of motor neurone disease in an experimental drug.

A phase three trial of the same drug had failed to make a difference to patient outcomes after six months of treatment.

The latest results show that patients who continued taking Tofersen for another six months had better mobility and lung function.

One man, who was in a wheelchair when he joined the trial, was able to walk without a stick, and another patient said they were able to write their own Christmas cards after receiving the treatment.

Dame Pamela Shaw is a co-author of the study. I have done more than 25 trials and never have I heard patients say they have improved.

The drug works by targeting a genetic abnormality, SOD1, which is the cause of 2% of patients, but experts said the results could pave the way for a new class of gene-targeting treatments for a much wider group of patients.

One of the first people to enroll on the trial was Les Wood, who was diagnosed with the disease ten years ago. Wood returned to Spain with his wife, Val, after the first year of taking Tofersen.

He said that he was able to come off some of his painkillers and that he felt better in himself. Although my symptoms have continued to get worse, I would not be without the drug and the change it has made to my quality of life.

Until now, the outlook has been bleak for people with the disease. The nerve cells in the brain and spine are destroyed by the disease, causing weakness, stiffening and waste.

Current treatments only marginally increase life expectancy and don't delay the loss of the patient's ability to walk, talk, eat and breathe.

In the initial phase, a group of patients were recruited and two-thirds of them were given monthly doses of the drug.

Tofersen is a drug that works by blocking the production of a toxic version of the SOD1Protein that is caused by a genetic defect.

There was no significant difference in the patient's mobility or lung function after six months after the drug was used. The trial was extended because the results were encouraging and people who had been on placebo switched to the drug.

According to the results published in the New England Journal of Medicine, there was a significant difference in lung function between the early and late start on the drug. The researchers believe the lag is just a reflection of how long it takes for motor neurons to heal and for the drug to make a difference.

Although it might have a more powerful effect if given earlier in the course of the disease, the drug is not seen as a cure and even delaying the progress of the disease is seen as a major advancement.

The FDA is considering an application to license the drug and it is being offered to patients in the UK.

The speed of change is what makes it so frightening. Patients can't get their heads around one aspect of disability before something new has to be faced If we can slow it down, it will be a lot less frightening.

The latest results show that Tofersen is having a beneficial effect on people living with the disease.

Similar gene therapy-based approaches may be helpful for other forms of the disease.