Prior research has shown that a class of medications called microtubule inhibitors may be effective at controlling the growth of EHE tumors. Recently, researchers conducted a phase 2 study to test whether the microtubule inhibitor eribulin is a safe and effective way to treat patients whose tumors continued to grow after prior treatment.

Six (6) people with EHE enrolled in the study, and two (2) of them experienced meaningful tumor shrinkage while enrolled. Although most patients had a brief period of stable disease before their tumors began to grow again, averaging 2.6 months, some patients had stable disease for as long as 13.5 months. The researchers also reported that side effects, like anemia and lung infections, were generally manageable by reducing the dose of the drug or increasing the time between doses.

Although this small study enrolled only people with progressive EHE, it is possible that some patients could benefit from treatment with eribulin as first-line therapy, or in combination with other kinds of treatment, such as immunotherapy. The authors suggest further study of eribulin for people with EHE is warranted.

Why this matters:

While this is a very small study, a promising treatment response is always encouraging. While researchers don’t yet understand which patients might benefit, future studies could test whether the drug works for patients who haven’t been treated with other therapies, or whether it works better when combined with other therapies.

Clinical studies in EHE like this one move research forward step by step. Progress in rare diseases requires collaboration among researchers across institutions and countries, as well as the willingness of patients and families to participate in research. EHE Foundation thanks the study authors for continuing to explore new treatment options and extends sincere gratitude to the people living with EHE who took part in this trial, whose participation helps build the knowledge needed to develop safer, more effective treatments for everyone affected by EHE.