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BMJ 2011; 342 doi: https://doi.org/10.1136/bmj.c7452 (Published 06 January 2011) Cite this as: BMJ 2011;342:c7452

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New unorthodox approach to treat inflammatory bowel disease in autism gets approval from the FDA

New unorthodox approach to treat inflammatory bowel disease in autism
gets approval from the FDA

I read a remarkable report published recently in The Scientist (Vol
25, Issue 2, Page 42)that shows how effective treatment of gut
inflammation can reverse the most severe symptoms in autistic individuals.
What is perhaps even more remarkable is that this new highly unorthodox
treatment now has the stump of approval from the FDA. Why unorthodox?
Because it involves feeding autistic patients with live ova of the
nematode Trichuris suis, pig whipworm !

The idea for this came from Stewart Johnson (father of a child with
autism) who discovered the work of researchers at the University of Iowa
who had previously successfully treated patients with Crohn's disease and
ulcerative colitis with Trichuris suis ova (TSO) [1-2]. Both Chron's and
ulcerative colitis are autoimmune disorders in which the immune system
attacks the intestinal walls. Stewart's hypothesis was that parasitic worm
infection would modulate his son's immune system and calm the gut
inflammation that was causing his disruptive behaviors. He came to this
conclusion after reviewing the landmark study by Vargas et al. [3] which
showed extensive glial activation and neuroinflammation in the brain of
patients with autism. This led Stewart to conclude that indeed, there was
a gut-brain connection in autism and that by fixing the gut problem the
latter would be fixed as well.

He proposed this treatment to Dr. Eric Hollander from the Montefiore
Medical Center University Hospital of the Albert Einstein College of
Medicine.

After obtaining permission to administer the treatment from the US
Food and Drug Administration (FDA) under "compassionate use" rules,
Stewart and Hollander initiated the treatment.

Within 10 weeks of treatment (with 2,500 eggs given every two weeks),
the boy's symptoms did no just improve. They vanished. He stopped
smashing his head against walls and gouging at his eyes. His psychotic
behaviours stopped and the family was finally allowed to have a normal
life.

The big question is: what is the mechanism by which TSO modulates the
immune system to calm down autoimmune attacks and inflammation in autism ?
It was initially assumed that intestinal worms could activate a Th2-type
response and as a consequence, suppress the Th1 response, which is usually
implicated in autoimmune diseases. However, this hypothesis was later
dismissed in favour of more recent research which showed that the
mechanism involves direct modulation of regulatory T-cells (T-reg) by the
worms. T-reg cells function to suppress activation of the immune system
thereby preventing it from attacking the body's own tissues. Research
indicates that intestinal worms can induce T-reg cells initially in the
gut and that this changes the inflammatory setting which finally results
in less inflammation in the brain.

This is good news! Even better is that the FDA finally started
thinking outside of the box!

References:

1. R.W. Summers et al., "Trichuris suis therapy in Crohn's disease,"
Gut, 54:87-90, 2005.
2. R.W. Summers et al., "Trichuris suis therapy for active ulcerative
colitis: a randomized controlled trial," Gastroenterology, 128:825-832,
2005.
3. D.L. Vargas et al., "Neuroglial activation and neuroinflammation in the
brain of patients with autism," Annal Neurol, 57:67-81, 2005.

Competing interests: No competing interests

14 February 2011
Lucija Tomljenovic
Postdoctoral Fellow
University of British Columbia