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Experimental Therapeutics



Front-line treatments often work well for pediatric cancers, controlling and frequently curing the disease. Sometimes challenging cases do not respond as intended to the best of initial treatment or relapse after success seems within reach.

That is why our Experimental Therapeutics Program offers new, promising medicines and treatment methods before they are more widely available — a highly specialized service available at only a small number of the nation’s top academic medical centers.

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.

Why Children's Health℠?

We run the only experimental therapeutics program for pediatric cancer in Dallas and one of the few in Texas. That means our patients with the most challenging cancers receive the earliest opportunity to receive up-and-coming medicines and treatment methods.

We give these treatments through clinical trials. These are not the Phase III trials found at all pediatric cancer programs (and elsewhere in our center), which are years into the Food and Drug Administration (FDA) approval process. Instead, our Experimental Therapeutics Program enrolls patients in earlier Phase I and II trials, which are often the first time a therapy is given to pediatric patients and examined for effectiveness and dose. Most centers can’t do this.

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.

Conditions We Treat

Our program provides emerging therapies for a range of challenging cancers that resist treatment or have returned. Learn more about some of the conditions we treat most frequently:

Other solid tumors including hepatoblastoma

Expert Care

Every pediatric cancer center participates in well-established clinical trials, but we are among the select few offering new therapies when they first emerge.

That requires extensive commitment, since patients with the most challenging cases need even more individualized care. It also requires highly specialized expertise to treat patients safely, evaluate and relieve potential side effects, and evaluate tumor response. We are the only pediatric program equipped to do this in Dallas and offer:

  • Specialized Team: Providing access to clinical trials for the newest therapies requires a highly experienced and specially trained team. We are the only such team in Dallas, and one of the few in Texas. Members include:
    • Doctors: Our doctors are faculty members at UT Southwestern Medical Center. They identify patients who might benefit from a clinical trial, work with families to choose the best therapy and meet with patients regularly during treatment.
    • Investigational pharmacist: Our specialized pharmacist prepares the medications to ensure patients get the right dose and formulation.
    • Nurse practitioners (NPs) and physician assistants (PAs): NPs and PAs meet with patients regularly, work with doctors to ensure new medications are given safely and gather the information needed to gauge effectiveness.
    • Nurses: Nurses provide treatments, watch for side effects and record data.
    • Clinical research coordinator: Our coordinator monitors patient data to ensure patients are treated safely, previously unknown side effects are identified and effectiveness is determined.
  • Safety: We share the same goal as our families — finding an effective treatment while still protecting your child. We take numerous precautions, including:
    • Evidence: We only use new therapies that were extensively tested in the lab with promising results. Often, the therapies are also tested in adults first.
    • Observation: Our team is trained to watch for and recognize side effects from therapy, and every child is carefully monitored. When needed, treatment is quickly adjusted or stopped.
    • Oversight: Our trials receive thorough reviews by the FDA, the Simmons Comprehensive Cancer Center at UT Southwestern and our institutional review board (IRB).
  • Partnerships: We partner with the Simmons Comprehensive Cancer Center and UT Southwestern, giving us access to a wide range of experts and leading research. While some trials are designed by our own investigators, we also partner with top research groups, including:


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:

  • Clinical trials: Our program provides innovative, promising treatments through two different sources of early-stage clinical trials:
    • Consortiums: We partner with other cancer centers, providing wide access to promising approaches. These studies 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.
  • Innovations: Our researchers continue to shape the treatment of many tumors. Recent developments include:​
    • Heating tumors: 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. Later this year we plan 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.

      See how MR-HIFU works

    • Targeting pathways: Cells communicate through pathways. In preliminary laboratory studies, one of our researchers discovered two pathways that enable germ cell tumors to grow. He found that a novel combination of two drugs — sirolimus and erlotinib — blocks the signaling and appears to halt tumor growth. We now lead a study open at several institutions evaluating this treatment in patients, the first of its kind.
    • 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.