Patients Self-Administer Protein in Nose Drops to Treat Kaposi's Sarcoma

University of Pennsylvania Cancer Center
Last Modified: May 16, 1999

An anti-angiogenesis and immune-stimulating protein, delivered via nose drops, has resulted in a complete or major response in more than one-third of patients with late-stage Kaposi's Sarcoma (KS), an AIDS-related skin and body cancer which originates from the cells that line the blood vessels, in a trial led by Dr. Parkash Gill of the University of Southern California School of Medicine. The small protein was used in 35 KS patients and showed no side-effects. Over half of the patients had more than 50 lesions upon entering the trial.

The naturally occurring protein, called IM862, increases production of interleukin-12, which is known to enhance the immune system. The protein also inhibits production of potent angiogenesis growth factors, VEGF and bFGF, to block blood vessel formation, thus depriving the cancer of the nourishment it needs to grow.

In light of the promising results of Dr. Gill's study and work of others, a larger, blinded, randomized and placebo-controlled Phase III trial of IM862 in Kaposi's sarcoma is underway. Additional trials in ovarian cancer and melanoma are in progress. If successful, Gill said the approach may be beneficial in the treatment of many cancers.

The advantages of this drug appear to be self-administration, direct absorption, and minimal side effects. It has been shown to have a 36% response rate in this phase I/II study which was durable for >1.5 years. Currently the main toxicity reported was headaches. It will soon be undergoing phase III trials in KS. This tumor was chosen because of its vascular nature and it is hoped that a significant response is seen in other less vascular tumors. It is also hoped that it can be used in combination with other more toxic conventional therapies, and thus enable a reduction of their dose and toxicities.

Slides from Dr. Gill's Presentation: