Study: Myeloma drug shows efficacy in treatment‑resistant neuropathies

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Researchers at Friedrich Schiller University Jena have reported the successful use of the bispecific monoclonal antibody teclistamab in two patients with severe, treatment‑resistant chronic autoimmune polyneuropathy. The drug, originally approved for multiple myeloma, showed marked therapeutic benefit in these rare neuroimmune disorders. The findings were published in Nature Communications.

Chronic immune‑mediated peripheral neuropathies are progressive diseases in which the immune system attacks the myelin sheaths of nerves, causing movement, balance and sensory disorders. Standard treatments – glucocorticoids, immunoglobulins, plasmapheresis and rituximab – are not always effective, and many patients continue to deteriorate. In the cases described, both women had failed to respond to years of therapy.

Teclistamab is a bispecific antibody that simultaneously binds to the CD3 T‑cell receptor and B‑cell maturation antigen. This directs T‑cells to attack mature B‑cells and plasma cells that produce pathogenic autoantibodies, thereby precisely eliminating the source of autoimmune damage.

In the first patient, a 65‑year‑old woman, walking distance increased significantly after four teclistamab injections, grip strength improved, and pain and fatigue decreased. Imaging showed reduced nerve swelling and improved nerve conduction. The disease‑associated paraprotein disappeared completely and remained undetectable for nine months of follow‑up.

In the second patient, aged 73, antibodies against myelin‑associated glycoprotein became undetectable within six weeks of the first injection and remained negative throughout follow‑up. Her walking distance increased more than six‑fold over the same period. Side effects in both patients were moderate and manageable, and clinical improvements remained stable with no signs of relapse.

The findings point to the significant therapeutic potential of T‑cell‑engaging therapy in treatment‑resistant chronic autoimmune neuropathies and possibly other neuroimmunological disorders.

The authors stressed that the findings need to be confirmed in larger clinical trials. For the pharmaceutical market, the results open up the prospect of expanding the label for an already approved drug, potentially increasing teclistamab’s commercial potential and encouraging manufacturers to develop similar bispecific antibodies for neuroimmune diseases.

Earlier, a separate study published in NEJM Evidence tested the decades‑old cancer drug rituximab in 10 women with autoimmune premature ovarian insufficiency. None of them had responded to ovarian stimulation before treatment; after therapy, six developed follicles that allowed egg retrieval, and three gave birth to healthy children.