Skip to Content


Lower-Risk Myelodysplastic Syndrome Studied

CancerWise - September 2007

By Renee Twombly

While many oncologists have taken a watch-and-wait attitude toward low-risk and intermediate-1 stages of myelodysplastic syndrome (MDS), a precancerous leukemia condition, some researchers believe that treatment will improve poor survival rates seen in the majority of patients.

To that end, researchers in M. D. Anderson’s Department of Leukemia are set to begin a series of clinical trials, the first of which will test the targeted agent vorinostat (Zolinza®).

Goal of the study

The protocol is designed to assess two different dosages of oral vorinostat in patients with low-risk and intermediate-1 stages of MDS.

These risk categories are used to predict age-related survival rates and progression to acute myelogenous leukemia.

Researchers will document:

  • Time to response
  • Response duration
  • Progression-free survival

Significance of the study

The M. D. Anderson study is the first phase IIa clinical trial to test vorinostat in patients with lower-risk MDS. The drug was approved in 2006 for use in progressive cutaneous T cell lymphoma (CTCL), a cancer of white blood cells.

Many blood disorders are caused by the “silencing” of beneficial genes, and vorinostat works by reactivating these genes.

Side effects

The primary side effects of vorinostat in preclinical studies have been nausea, vomiting and diarrhea, all of which are controllable with medication, says the study’s principal investigator Guillermo Garcia-Manero, M.D., chief of the Section of Myelodysplastic Syndromes in the Department of Leukemia.

Study description

Patients will be randomly assigned to receive vorinostat in daily doses of 400 and 800 milligrams.

Trial participants will be required to:

  • Take vorinostat for 14 days
  • Rest one week
  • Take vorinostat for another 14 days

Patients will complete eight of these cycles. Those who demonstrate a continuing response will receive treatment indefinitely or until their disease progresses.

Participants must be able to visit Houston for an initial 30-day testing period. After that, M. D. Anderson oncologists can work with a patient’s personal physician to ensure study medications are being administered wherever the patient lives, Garcia-Manero says.


To be considered for this study, patients must:

  • Have not been treated or had only one prior treatment
  • Be dependent on red blood cell transfusion


Although not a cancer, MDS is difficult to treat successfully. It usually strikes the elderly, and patients are typically given blood transfusions and supportive care.

More aggressive treatments, such as use of stem cell transplants, are not for every patient, and most oncologists believe that toxic therapy should not be used at lower stages of the disease.

However, Garcia-Manero recently conducted a study that found that up to 80% of lower-risk MDS patients have a relatively poor median survival rate of two years.

“That has prompted us to change the treatment paradigm,” he says. “We are very excited about the prospect of helping our patients with some of the newest, less toxic treatments.”

For more information, call askMDAnderson at 1-877-632-6789 and ask about protocol 2007-0201.


© 2015 The University of Texas MD Anderson Cancer Center