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Jack Arbiser, M.D., Ph.D. |
The “strawberry marks” that show up on babies’ necks or faces usually go away on their own, without treatment. But sometimes the lesions, which are actually clusters of dilated blood vessels called hemangiomas, affect sensitive areas like the eyes, trachea, or lungs.
Although they do not become malignant, these lesions are the most common tumors of infancy and childhood and account for a disproportionate number of visits to pediatricians and dermatologists.
Dr. Jack Arbiser, Associate Professor of Dermatology at Emory’s School of Medicine, has discovered compounds that abolish the growth of these blood vessels, and show promise not only for the treatment of hemangiomas but also for more serious diseases such as cancer.
“The biology of hemangiomas has many similarities to the blood vessels seen in malignant tumors, inflammatory disorders, and macular degeneration,” he says. “Thus, by targeting angiopoietin-2, we can target vessels in these conditions, and compounds that target angiopoietin-2 might be of benefit for these conditions.”
Angiopoietin-2 is a peptide that stimulates blood vessels to grow and also causes blood vessels to leak. Whenever there is swelling and redness—such as at the sites of tumors, injuries, or infections—there are leaky blood vessels. If blood vessel leakiness can be stopped, delivery of chemotherapy or antibiotics is enhanced, Arbiser says.
Hemangiomas, which are present in about 5 percent of infants at one year of age, consist of clusters of endothelial cells, which exist in the lining of blood vessels. These clusters go through three phases: a proliferative phase, characterized by rapid growth of the tumor; an involuting stage, marked by decreasing tumor size; and finally an involuted stage, during which the original tumor is replaced by connective scar tissue.
Most hemangiomas are benign and disappear on their own. Traditional treatments of large hemangiomas, however, include lengthy courses of steroids or surgery, which can result in growth retardation, infection, neuropathy, and potentially death.
Arbiser saw the need for novel therapies for hemangiomas in humans, and several of the tested compounds have proven successful in mice models.
“We found that a group of compounds called triphenylmethanes prevents production of angiopoietin-2,” says Arbiser. “One compound that does this quite effectively is gentian violet, which is available over the counter in the U.S. Based upon our findings in the lab, we are using gentian violet in patients with psoriasis, eczema, warts, ulcers and other human skin conditions that are known to involve angiopoietin-2, and have seen benefits.”
A clinical trial is ongoing in Israel using another triphenylmethane for the treatment of hemangiomas.
Arbiser, who has previously developed beneficial therapies from such varied sources as ant venom, Turmeric, and the magnolia seed cone, has high hopes for these compounds.
“Ultimately, we plan to develop systemic forms for the treatment of cancer and inflammatory diseases, and we believe that this will be the ultimate market,” Arbiser says. “Leaky blood vessels are a major problem in cancer— such as ascites in ovarian cancer, and swelling in brain tumors—and these compounds are designed for these problems.”
Arbiser is working with Emory’s Office of Technology Transfer (OTT) on commercial development of the novel intellectual property related to this research.
“Dr. Arbiser’s research on angiogenesis is an example of great science yielding innovative therapeutic approaches that can directly benefit the public,” says Cale Lennon, who manages the technology for OTT. “His ongoing work to further develop this technology is quite exciting given the potential to treat patients affected by a wide variety of diseases.
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