New Brain Cancer Treatment May be More Effective, Less Toxic

Results of UCSF Clinical Trial May Change Standard of Care for Primary CNS Lymphoma, Researchers Say

By Jason Bardi    | | April 8, 2013

New Brain Cancer Treatment May be More Effective, Less Toxic

The figure shows the before (A) and after (B) brain scan of a primary CNS lymphoma patient treated with a combination of high-dose chemotherapy with immune therapy, avoiding the need for whole-brain radiotherapy.
Image courtesy of James Rubenstein.

A Phase 2 clinical trial testing a new protocol for treating a relatively rare form of brain cancer, primary CNS lymphoma, may change the standard of care for this disease, according to doctors at UC San Francisco who led the research.

Described this week in the Journal of Clinical Oncology, the trial involved 44 patients who were given a combination of high-dose chemotherapy with immune therapy, rather than the standard combination of chemotherapy with a technique known as whole-brain radiotherapy.

The new treatment approach was significantly less toxic because it avoided whole-brain radiotherapy, which at high doses can kill brain cells and lead to a progressive deterioration of the function of the nervous system in patients. Many patients die from the toxicity of the radiation as opposed to the cancer itself.

The new treatment also seemed to work better, with the majority of patients on the trial still alive with a follow-up of nearly five years, researchers found.

The lymphoma-free survival of patients with this form of brain cancer was doubled compared to the lymphoma-free survival in previous multicenter U.S. cooperative-group-sponsored clinical trials involving brain radiotherapy, said UCSF oncologist James Rubenstein, MD, PhD, associate professor of medicine, who led the study.

In addition, unlike previous treatments for primary CNS lymphoma, the new approach was equally effective in older patients – those over 60 – as it was in younger patients. This is particularly significant given that the incidence of this type of brain tumor appears to be increasing in patients 65 and older.

Rubenstein is a member of the UCSF Helen Diller Family Comprehensive Cancer Center, which is one of the country’s leading research and clinical care centers, and is the only comprehensive cancer center in the San Francisco Bay Area.

The work raises the possibility of taking a “personalized medicine” approach to guiding treatment for this form of cancer because the researchers identified a biomarker – a gene called BCL6 – which could predict the outcome of treatment depending on how much of the gene was present in the tumor.

A randomized clinical trial, which will test the effectiveness of the new therapeutic approach in a larger patient population, is now enrolling at UCSF and at other medical centers in the United States.