In massive news for neurology, The New England Journal of Medicine has published three important studies reporting that two new drugs for multiple sclerosis are more effective than existing treatments and can be taken in pill form.
Multiple sclerosis is a bitch. It’s a neurological disorder where the immune system starts attacking myelin – the protective covering of nerves in the brain and spinal cord – leading to unpredictable attacks that typically leave the person a little more disabled each time.
Problems can include movement difficulties, chronic pain, fatigue, cognitive problems, mood instability and impairments to the body’s automatic processes like digestion, bladder and bowel control.
One problem with the the current treatments that try and slow down the disease itself, rather than just manage the effects, is that they all require regular injections or infusions via a drip.
These new studies report on two drugs: one is cladribine which is already widely used in leukaemia, and the other is fingolimod, which is not yet available commercially. Crucially, both can be taken as pills without the need for injections.
The studies that investigated these drugs were very impressive. They had large numbers of patients in many countries; they were conducted with the co-operation of drug companies but were led by independent researchers; they continued for about two years; they were compared against placebo and, in the case of fingolimod, against the current best available treatment – beta interferon; and they looked at both chances of relapse and at changes in brain structure.
The studies did not include the most disabled people with MS are all were able to walk, although patients with mild and moderate disability were included.
The results suggest that the drugs are not only easier to take but are better than the current best available treatments and reduced the chances of the patient having a relapse of MS as well as the damage to the white matter in the brain.
The drugs work quite differently from current treatments – which largely reduce inflammation directly – by changing the balance of how the immune system releases T cells so more antiinflammatory ‘helper’ T cells are available.
Unfortunately, the drugs are not without side effects, and although these effects were rare, altering the immune system led to more herpes infections and an increase in the development of cancer.
Herpes infections can take the form of the annoying but relatively benign, like in local infections such as cold sores, shingles and genital herpes, but when it gets into the whole body or brain it can cause serious damage or even lead to death, which happen to two patients in the trial, although in this case it was out of more than 1,100 people in total. The people with cancer generally recovered well – there was one death but it isn’t entirely clear it was linked to the treatment.
Although these drugs are not cures, they only slow the disease down, this is still massive news and a major development in neurology.
One of the practical big issues will be how the drugs are priced by the pharmaceutical companies and you can be sure they’re not going to be cheap.
However, one small hope is that the two compounds are owned by rival companies and as they seem to have broadly equivalent effects it will be hoped that competition will drive the price down.
Link to good write-up from The Times.
Link to good technical summary in the NEJM, sadly paywalled.