Experimental Therapeutics Program

Experimental Therapeutics Program

For Pediatric Cancer

The only program in the region providing emerging therapies for a range of childhood cancers that resist treatment or have returned.

When front-line treatments for pediatric cancer are not responsive, the Experimental Therapeutics Program at Children's Health offers new, promising medicines and treatment methods before they are more widely available. 

The specialists and sub-specialists in our program are recognized across the country and the world for their outstanding care and leading research. They provide the treatments and extensive support your family needs for cases of:

  • Refractory cancer: The cancer does not respond to established therapies, either showing resistance right away or developing it during the course of treatment.
  • Relapsed cancer: Treatment initially improves your child’s condition, but then the disease returns

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An Innovative Approach to Fighting Cancer

During standard chemotherapy, much of the drug remains in the bloodstream, failing to reach the tumor but still causing side effects. We are exploring an alternative called MR-HIFU, or magnetic resonance-guided high intensity focused ultrasound. A combined MRI scanner and ultrasound device heats the tumor, causing its blood vessels to dilate and become leaky. That opens the way for the chemotherapy to destroy the tumor, with the potential to improve therapy and reduce side effects. We are aiming to open the first clinical trial in the country focusing on using MR-HIFU to improve chemotherapy delivery to relapsed and recurrent pediatric cancer patients, including those with neuroblastoma and sarcoma.

Access to the Latest Treatments

The Experimental Therapeutics Program is one of the few cancer programs nationally that enrolls patients in early Phase I and II clinical trials. These clinical trials are often the first time a therapy is given to pediatric patients and examined for effectiveness and dose. That means our patients with the most challenging cancers receive the earliest opportunity to receive up-and-coming medicines and treatment methods.

While the prospect of brand-new treatments might worry you at first, it’s important to know we only recommend therapies backed by solid research, showing the greatest potential for your child. Treatment is delivered by an experienced team with specialized skills, following strict safety standards. Learn more about current clinical trials.

A Center for Innovation

We work closely with your family to identify when a newly emerging therapy might benefit your child and to choose the best one. We accomplish this through collaboration with other cancer centers as well as developing our own innovative research.

  • Consortiums: These clinical trials are conducted at a select number of centers nationally, with limited openings for patients. Only top programs are invited to participate.
  • Homegrown research: Our experts also create new therapies and the initial clinical trials to study them. These trials are held first at our center before spreading more broadly. This gives our families even earlier access to the very latest treatments. Many of these trials are based on promising research done at our local partner, UT Southwestern Medical Center, which includes the NCI-designated Simmons Comprehensive Cancer Center.
  • Sequencing tumors: As part of a partnership with other centers, we sequence the genes of certain tumors that represent an especially high risk to the patient or that have returned. Our goal is to tailor therapy to particular genetic mutations to potentially improve outcomes.
  • Developing new treatments: Our researchers have developed new ways of fighting pediatric cancer, including targeting pathways to halt tumor growth and heating tumors to aid chemotherapy treatment (see MR-HIFU above).

Meet the Care Team