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Analysis And Comment Drugs

Lessons for clinical trials from natalizumab in multiple sclerosis

BMJ 2006; 332 doi: https://doi.org/10.1136/bmj.332.7538.416 (Published 16 February 2006) Cite this as: BMJ 2006;332:416

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A Patient's Perspective

A Patient’s Perspective

My friend’s father has multiple sclerosis (MS), so when he showed an
interest in medicine it was arranged for him to shadow the to-ing and fro-
ing in the neurology department from which his father was receiving care.
This proved to be enough to draw him into the profession. During his
surgical training, he too was diagnosed with MS. Aware that natalizumab
was awaiting licence it gave him some hope in the face of devastating
relapses despite beta interferon-1a treatment.

Whilst on beta interferon 1a he deteriorated from being a man who
once ran the trans-sahara marathon (150miles in 6 days) to developing an
unsteady gait and using crutches. Campath was suggested at this time and
he was referred to the only centre prescribing this for MS. The
assessment of this centre was that he was indeed appropriate for this
therapy however, due to concerns raised by the FDA at that time they
requested for it to not be prescribed until further notice, although it is
commonly used for haematological disorders.

During this delay he became unable to move his legs at all, making it
difficult to roll over in bed. He frequently self-catheterised and had
dreadfully slurred speech to the point of incomprehension and therefore
avoided talking to even those closest to him. His vision was so bad that
he could not read or distract himself for a few moments by watching
television. At this time, his own neurology centre after discussing with
him the pros and cons, prescribed the Campath after obtaining written
consent. The improvement has been dramatic. He can walk again with
crutches, is vociferous and completely clear when he verbally expresses
his opinion.

Having reached such depths of disability and unhappiness, a small
risk of developing progressive multifocal leucoencephalopathy (1 in 1000)
with natalizumab and autoimmune conditions with Campath, in his opinion
becomes a risk worth taking. So while you may argue that long-term
efficacy and safety data should be evaluated before new treatments are
offered, and that for most patients, MS is a relatively slowly progressive
disease, bear a thought for those for whom waiting is actually denying
them options and recognise that well–informed patients should have
autonomy and be able to consent to potentially useful therapies,
especially given that he himself routinely consented patients for
procedures with much greater mortality risks.

I agree that further studies are required to define the long term
risks associated with natalizumab but while promising short term data is
available this is adequate for some patients on which to base their
decision and it is therefore vital that clinicians be well informed.
Unfortunately employing caution through delay is a preserve of the
healthy.

Competing interests:
None declared

Competing interests: No competing interests

26 May 2006
Daisy R Sandhu
Senior House Officer
St George's Hospital SW17 0QT