A unique combination therapy that refashions #rapamycin into a polymeric nanoparticle delivery vehicle for ponatinib can release both drugs to shrink venous malformations in mice with a single dose.
Polymeric rapamycin nanoparticles encapsulating ponatinib cause regression of venous malformations in mice
Venous malformations are treated with oral rapamycin, but this therapeutic approach requires daily oral dosing and is associated with systemic side effects that reduce adherence and limit therapeutic effectiveness. To address this issue, Tang et al. developed nanoparticles made of the mTOR inhibitor rapamycin as a polymeric drug. To add further therapeutic benefit, the authors encapsulated the c-ABL inhibitor ponatinib in the nanoparticles. A single dose of these nanoparticles in a murine model reduced the sizes of venous malformations without evidence of systemic toxicity. These findings both offer a candidate therapeutic approach for venous malformations and demonstrate that rapamycin itself may be an effective drug delivery platform. —Courtney Malo