Are steroids detrimental to survival in brain tumours?

As I update neurochecklists I come across some papers which make me go, ‘really!’ Such studies challenge established theories and threaten conventional practice. Such is the case with a recent paper in Brain titled, unequivocally, Corticosteroids compromise survival in glioblastoma.

By Christaras A - Created myself from anonymized patient MR, CC BY 2.5,
By Christaras A – Created myself from anonymized patient MR, CC BY 2.5,

Glioblastoma is the worst form of primary brain tumour, and survival is already poor. Treatment is usually palliative with debulking surgery and radiotherapy. Dexamethasone, a corticosteroid, effectively reduces the swelling or oedema that the tumour evokes around it. Corticosteroids are therefore often the first treatment for glioblastoma because they  almost immediately improve symptoms such as reduced consciousness, headache, and visual blurring.

By LHcheM - Own work, CC BY-SA 3.0,
By LHcheMOwn work, CC BY-SA 3.0,

It is, therefore, surprising when a study suggests that corticosteroids cause harm. But this is no ordinary study; it is a classic bench-to-bedside research which looked at patients with glioblastoma, and then devised a mouse model to study the real impact of steroids on the tumour.

By Jensflorian - Own work, CC BY-SA 3.0,
By JensflorianOwn work, CC BY-SA 3.0,


The authors show that a ‘ dexamethasone-associated gene expression signature correlated with shorter survival’. They pass the verdict that corticosteroids are detrimental to survival and urge caution when prescribing dexamethasone.


You may be feeling a bit low after reading. You may, however, lift your spirits by reading my previous posts titled maggots, viruses and lasers: some innovations for brain tumours and calming the rage of brain tumours.



How is neurology stamping out the anguish of Duchenne?

Duchenne muscular dystrophy (DMD) is the most familiar of the inherited muscle diseases called muscular dystrophies. DMD is life limiting, but advances in care are enabling children born with this disease to survive well into adulthood. The disease is named after the French neurologist Guillaume Duchenne.

See page for author [CC BY 4.0], via Wikimedia Commons
See page for author [CC BY 4.0], via Wikimedia Commons

The foundation of long survival in DMD is close supervision of breathing and heart functions. DMD however affects much more than these vital functions, and it remains a challenging disease for families and management teams. Thankfully researchers are not resting on their laurels, working ever hard on heart-warming advances. Here are three.


By Ring0 (Own work) [Public domain], via Wikimedia Commons
By Ring0 (Own work) [Public domain], via Wikimedia Commons
Steroids are now well-established in the treatment of Duchenne muscular dystrophy. What is new however is a better understanding of their benefits in DMD, together with clearer guidance on their use. This is contained in the recent practice guideline update summary: Corticosteroid treatment of Duchenne muscular dystrophy.

Published in the journal Neurology, this document shows how steroids help to improve muscle strength, maintain breathing functions, stabilise ambulation, prevent spinal deterioration (scoliosis), and delay onset of heart disease.

Is there more one could hope for? Yes, a lot more when it comes to genetic diseases.


Mitokondria. 140264jd on Flikr.
Mitokondria. 140264jd on Flikr.


Idebenone is not new to neurologists. Researchers at Newcastle have been investigating its vision-preserving effect in the mitochondrial disease called Leber’s hereditary optic neuropathy (LHON). Idebenone is thought to improve the activity of mitochondria, the energy-producing component of all cells. Idebenone has also been investigated in other neurological disorders such as Friedreich’s ataxia.

Perhaps as an indication of its growing importance, researchers have now looked at the effect of Idebenone in people with DMD, and they did this in two separate trials. DELPHI is published in the journal Neuromuscular Disorders as Idebenone as a novel, therapeutic approach for Duchenne muscular dystrophy. The authors reported benefit in both cardiac and respiratory function.

DELOS, the second trial, is published in Lancet Neurology and titled Efficacy of idebenone on respiratory function in patients with Duchenne muscular dystrophy not using glucocorticoids. The authors again reported similar benefits. For a synthesised take, see this useful review in Touch Neurology.

But is this enough for ambitious researchers? Of course not…not when you see the promise of gene editing.


Jazz Mouse. Richard Scott on Flikr.
Jazz Mouse. Richard Scott on Flikr.


I first came across this in Eureka Alert which proclaimed: Gene-editing technique successfully stops progression of Duchenne muscular dystrophy. The gene editing, or gene splicing, technique is called CRISPR. The research itself is published in the journal Science as In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy.

The researchers used CRISPR technology to delete exon 23 from the Duchenne gene on the X chromosome. Exon 23 is the site of the fault that makes DMD patients unable to produce the muscle protein called dystrophin. By splicing this exon out, the researchers demonstrated an increase in the production of dystrophin. And this increase was significant enough to lead to an improvement in muscle strength.

OK, its only the humble mouse at the moment, but exon skipping therapy is clearly beckoning.

B0007267 Muscle Fibers. Welcome Images on Flikr.
B0007267 Muscle Fibers. Welcome Images on Flikr.