Sarcoma: Continuing the Search for Treatment Alternatives

Current trials study novel therapies for rhabdomyosarcoma and clear cell sarcoma

Treatment options for many sarcomas haven’t moved forward in decades. In many cases, clinicians are still using the same chemotherapies first developed a generation ago. But that may be starting to change.

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Several new clinical trials at Cleveland Clinic are part of an effort to find better treatment options for a variety of sarcomas.

Current therapies tend to be highly toxic and not universally effective says Cleveland Clinic pediatric oncologist Matteo Trucco, MD. For decades, treatment focused on simply pushing chemotherapy doses higher and higher. But those strategies eventually plateaued.

“For the last 10 years or so, we’ve been trying to find therapeutic alternatives,” he says. “A lot of that started in the lab and is finally reaching maturation. Now we’re finally bringing these treatments to patients.”

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Different treatments for different sarcomas

Historically, sarcomas have been understudied compared to other tumors, notes Cleveland Clinic medical oncologist Dale Shepard, MD, PhD. Trials often grouped together many different sarcomas, taking a one-size-fits-all approach to treatment, often with poor results.

“Both researchers and clinicians are getting away from lumping sarcomas together and instead are treating them as the individual diseases they are,” he says.

A sarcoma dream team

Cleveland Clinic’s multidisciplinary team of specialists has made it a leader in sarcoma treatment and research, adds Dr. Shepard. Those specialists include radiologists, medical oncologists, radiation oncologists, pediatric oncologists, orthopaedic surgeons and a nationally recognized pathology lab, all focused on sarcoma treatment.

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“It’s a dream team of people who specialize in these tumor types,” says Dr. Trucco.

Current trials

Current sarcoma research at Cleveland Clinic includes these two trials being led or co-led by Dr. Trucco:

  1. Evolutionary inspired therapy for newly diagnosed metastatic fusion-positive rhabdomyosarcoma
    Sponsor/Collaborator: H. Lee Moffitt Cancer Center & Research Institute; National Pediatric Cancer Foundation

    Summary: Metastatic fusion-positive rhabdomyosarcoma responds well to initial treatment, but 94% of patients relapse within three years, after which the disease becomes much more difficult to treat. This study will look at whether attacking the cancer with different schedules and combinations of chemotherapy is more effective than repeatedly using the same drugs at high doses, applying principles of evolution to attempt to outsmart the cancer and make it “extinct.”

    Eligibility: Any newly diagnosed patient with metastatic fusion-positive rhabdomyosarcoma

  2. Devimistat and hydroxychloroquine for clear cell sarcoma
    Sponsor: Rafael Pharmaceuticals

    Summary: Clear cell sarcoma is extremely rare and has an extremely poor prognosis. A small paper in Japan showed that in a mouse model, the drugs devimistat and chloroquine in combination dramatically blocked the sarcoma growth — even though each drug alone had little effect. This trial was inspired by the patient community, after the family of a patient found the paper and asked that their son receive the treatment via compassionate use. While this patient received the treatment when he already had end-stage disease, it inspired support for a clinical trial of the combination of devimistat and the more tolerable hydroxychloroquine for patients earlier in their disease course, when it is more likely to be beneficial.

    Eligibility: Patients age 11 to 75 with relapsed clear cell sarcoma or other relapsed fusion-positive sarcomas