March 11, 2009
New hope for patients with rare brain tumors

Susan Chi, MD, administers an intense treatment regimen that is producing much-improved outcomes for children like Avalanna Routh, 3, who have a rare type of brain tumor. 

A few young children may be cured and others given a better shot at long-term survival thanks to a new intense, multi-pronged therapy regimen administered to young patients with a rare, aggressive type of brain tumor, say Dana-Farber Cancer Institute researchers who directed the first study of its kind.

Previously, children with the extremely rare brain cancer called atypical teratoid/rhabdoid tumor (AT/RT) typically lived less than a year. Results of a multi-center trial recently published in the Journal of Clinical Oncology showed that more than 70 percent of 20 such patients – four of them treated at Dana-Farber – have survived more than two to three years with the new treatment regimen. Most relapses occurred within months, and so "once the children are past the two-year mark, we're pretty hopeful they are cured," says Susan Chi, MD, who led the Dana-Farber study along with Mark Kieran, MD, PhD, and Mary Ann Zimmerman, CPNP.

Key to the improved outcomes was "a lot of treatment over a short period of time, early on," says Chi. The regimen included a combination of surgery, high-dose radiation, and chemotherapy. A portion of the latter was delivered directly to the brain and spinal canal.

The researchers say the study is the first to prospectively evaluate the new treatment regimen, which they developed and first administered in a small pilot study with encouraging results.

The study authors note that the regimen succeeded in extending "progression-free survival" - the period before the cancer began growing again - to a median of 27 months. With previous treatments, that period averaged six to 11 months, Chi says.

Some children who underwent the powerful treatments had complications such as infections and long hospitalizations for cycles of chemotherapy, says Chi. In addition, the high doses of radiation may have long-term cognitive effects, and those patients will be carefully monitored.

Despite these issues, Chi says, parents of the surviving patients are "very, very happy" with the outcomes. Other families have brought children with AT/RT to Dana-Farber because of the successes, and five of them are currently undergoing treatment.

"We're really pleased," Chi says. "We're not curing everybody yet, but it's a great place to start."

– Richard Saltus
Richard_Saltus@dfci.harvard.edu