Previous studies have suggested that targeting intravitreal vascular endothelial growth factor A (VEGF-A) counters choroidal neovascularization and hence age-related macular degeneration. A double-blind, placebo-controlled trial of ranibizumab, which neutralizes all isoforms of VEGF-A, for treatment of minimally classic and occult age-related macular degeneration retarded the progression of the disease and improved visual acuity in some patients.

Previous studies have implicated intravitreal vascular endothelial growth factor A (VEGF-A) as a target for countering neovascularization and, therefore, age-related macular degeneration. This double-blind, controlled trial comparing ranibizumab, which neutralizes all isoforms of VEGF-A, with photodynamic therapy with verteporfin showed that ranibizumab was better able to retard the progression of predominantly classic neovascular age-related macular degeneration.

In standard immunotherapy, small amounts of allergen are injected to induce a state of clinical tolerance of allergen reexposure. In this trial, the investigators studied immunotherapy in which a ragweed allergen was conjugated to an oligonucleotide. The vaccine did not reduce the albumin level in nasal-lavage fluid (the primary end point) but did have a positive effect on an array of secondary end points, suggesting that this approach merits further study in larger, structured trials.

Anemia is a principal feature of the myelodysplastic syndrome with deletion of chromosome 5q; most patients require transfusions, and iron overload develops as a consequence. In this phase 2 trial, lenalidomide reduced dependence on transfusions in most patients, and two thirds of them could stop transfusions altogether. The drug, an analogue of thalidomide, also reversed chromosomal and cytologic abnormalities in many patients.