Natalizumab, a humanized monoclonal antibody against the 4 integrin did not help in treating a patient suffering from chronic inflammatory demyelinating polyradiculoneuropathy (CIDP). Although experimental evidence in an animal model pointed out that targeting 4 integrins in the inflamed peripheral nervous system may have clinical relevant effects, this was not the case in a patient.

Natalizumab, 4 integrin binding and absence of clinical response in CIDP 

T cells expressing the 4 integrin are found in inflamed peripheral nerve. Natalizumab binds with high affinity to the 4 integrin on T lymphocytes, and this was proven to be the case in a patient suffering from CIDP. Although this biomarker for efficacy was positive, the patient's clinical condition deteriorated and as seen on magnetic resonance imaging without any measurable effect after treatment with this natalizumab. A nice and crisp n=1 study showing that biomarkers do not always predict clinical efficacy, even when these markers seem to be highly logical to select as markers!

Natalizumab Treatment in a Patient With Chronic Inflammatory Demyelinating Polyneuropathy. Christian Wolf, MD; Til Menge, MD; Max-Philipp Stenner, MSc; Gerd Meyer zu Hörste, MD; Andreas Saleh, MD; Hans-Peter Hartung, MD; Heinz Wiendl, MD; Bernd C. Kieseier, MD Arch Neurol. 2010;67(7):881-883. 

Jan M. Keppel Hesselink, MD, PhD, august 2010