Chinese researchers have presented preclinical and early clinical data suggesting that thalidomide could become a new treatment option for sporadic arteriovenous malformations of the central nervous system – congenital vascular abnormalities that carry a risk of severe haemorrhage. Although the study was pilot‑scale and included a small number of patients, the findings point to the possibility of creating the first drug therapy for this condition. The results were published in the journal Science Bulletin.
Central nervous system arteriovenous malformations are abnormal vascular tangles in the brain and spinal cord that can cause haemorrhages, strokes, seizures and neurological deficits. Surgery, embolisation and radiosurgery remain the mainstay of treatment, but these are not suitable for all patients and carry a high risk of complications.
The researchers developed a mouse model of the disease that replicates key human pathology features, then used transcriptomic analysis to identify thalidomide as one of the most promising candidates for drug repurposing. In experiments, the drug improved survival, reduced malformation growth, decreased haemorrhages and promoted restoration of vascular wall structure.
The findings were further evaluated in a pilot clinical trial of 28 patients with sporadic brain or spinal cord malformations who were not candidates for invasive treatment. All patients completed therapy and follow‑up: 11 remained stable, while 17 showed a reduction in malformation size. No disease progression was recorded. Only mild to moderate adverse events were reported.
The authors stressed that larger randomised trials are needed to confirm thalidomide’s efficacy and safety. Nevertheless, the findings open the prospect of the first drug therapy for patients with sporadic CNS arteriovenous malformations who are not eligible for or have contraindications to invasive procedures.
Thalidomide was synthesised in 1954 by German company Chemie Grünenthal as an anticonvulsant. It was approved as a sedative and a treatment for morning sickness in pregnant women and was widely used from 1957. This led to the thalidomide disaster, also known as the “epidemic of deformities”, with around 10,000 children born with severe limb defects. The drug was subsequently withdrawn from the market.
However, it later made an official return to medicine due to its immunomodulatory and anti‑angiogenic properties, albeit under strict control. In 1998, the US Food and Drug Administration approved it for treating leprosy complications. It is also recognised as a vital drug for multiple myeloma. The drug is tightly controlled worldwide because of its teratogenic effects. Thalidomide is not registered in Russia, but its structural analogues lenalidomide and pomalidomide are legally used in oncology for multiple myeloma.