Novel Topical Formulation for Prevention and Treatment of Lymphedema


Novel Topical Formulation for Prevention and Treatment of Lymphedema



Lymphedema, which causes chronic swelling, recurrent infections and decreased quality of life, is the most common long-term complication of cancer treatment. No curative treatment options exist, with current therapies limited to palliative measures.

There is accumulating evidence that T cells play a key role in the pathology of lymphedema by promoting tissue fibrosis, a hallmark of lymphedema, and inhibiting lymphangiogenesis, which plays a key physiological role in homeostasis, metabolism, and immunity. Using mouse models, MSK investigators have screened topical formulations of FDA-approved anti-T cell (tacrolimus, teriflunomide) and anti-fibrotic (pirfenidone, captopril) therapies for their effectiveness at blocking the pathology of lymphedema in a preclinical model. Investigators found that all topical treatments were highly effective at both preventing lymphedema development and treating established lymphedema. Moreover, minimal systemic absorption as well as no obvious toxicity was observed in treated animals.

MSK investigators are currently developing a proprietary topical formulation of one of these drugs for the treatment of lymphedema that is suitable for both long-term use and use on large surface areas of the body. This novel formulation is expected to enter clinical trials at MSK in 2020.


  • Lead investigator is a pioneer in preclinical and clinical studies of lymphedema; his approach represents a paradigm shift in treating lymphedema
  • MSK research demonstrates that anti-T cell and anti-fibrotic topicals are effective in both prevention and treatment of lymphedema, with potential for synergy and enhanced outcomes by combining these two classes of drugs
  • IP protection for a novel method that addresses an area of high unmet need
  • Established safety and tolerability record for these FDA-approved drugs
  • Topical formulations offer low systemic absorption


It is estimated that more than 6M patients in the U.S. alone are afflicted with lymphedema, with as many as 50,000 new cases diagnosed annually. Given that lymphedema is a lifelong disease with no cure, the number of patients with this condition is expected to increase each year. Potential expansion to the treatment of other chronic swelling conditions (e.g. after joint replacement) also exists.


U.S. National issued. Nationals issued in Europe, Australia, Japan, Hong Kong, and China. Canada nationals pending.


Gardenier et al. (2016) Topical tacrolimus for the treatment of secondary lymphedema. Nature Communications (PubMed link)


Babak Mehrara, MD, Laboratory Head, Memorial Hospital Research Laboratories, Memorial Sloan Kettering; and Chief, Plastic and Reconstructive Surgical Service, Memorial Hospital, MSK


Imke Ehlers, PhD, Associate Director, Technology Development
E-mail: [email protected]

Stage of Development

Animal studies