Targeted Therapy for Ewing Sarcoma

Ewing sarcoma is a rare and aggressive cancer that primarily affects bones and the surrounding soft tissues. The cancer results from a genetic mutation. Specifically, a translocation between chromosomes 11 and 22 leads to the abnormal fusion of the EWSR1 gene and FLI1 gene, resulting in uncontrolled cell growth and tumor formation.

Although Ewing sarcoma can occur at any age, it is most commonly diagnosed in children and young adults. Symptoms can include pain, swelling and possibly a lump at the tumor site.

Treatment for Ewing sarcoma typically involves a combination of surgery, chemotherapy and radiation therapy. Targeted therapy may be considered if the tumor does not respond well to traditional treatments or has metastasized or recurred. A form of precision medicine, targeted therapy focuses only on cells with specific molecular characteristics. Unlike other treatments, such as chemotherapy and radiation therapy, it does not affect healthy cells.

How does targeted therapy for Ewing sarcoma work?

Targeted therapy works by identifying and homing in on specific molecules or pathways involved in the growth and survival of a tumor, such as the abnormal fusion protein EWSR1/FLI1, the defining genetic mutation of Ewing sarcoma. By inhibiting the function of this protein—which is only found in Ewing sarcoma cells—targeted therapy can block tumor growth and spread while leaving healthy cells unaffected.

What does targeted therapy for Ewing sarcoma involve?

Targeted therapy for Ewing sarcoma is highly personalized. In general, the drugs may be:

• Administered via a needle inserted in a vein (intravenously)
• Injected under the skin (subcutaneously)
• Swallowed in pill or capsule form

The treatment may be given daily, several times a week, weekly, monthly or less frequently. In some cases, targeted therapy is a standalone treatment; in others, it is used along with chemotherapy or radiation therapy for heightened effectiveness.

Sometimes, cancer becomes resistant to targeted therapy, reducing its effectiveness over time. The patient’s progress will be closely monitored by their physician. In addition to regular medical checkups, this may involve blood work and imaging scans. If necessary, the physician will adjust the treatment regimen and help the patient manage any side effects.

What are the risks and possible complications of targeted therapy for Ewing sarcoma?

Targeted therapy for Ewing sarcoma carries several risks and possible complications, which can vary widely based on the drug used. Some examples of possible side effects include:

• Nausea, vomiting and diarrhea
• Dry skin, rash and nail changes
• Sensitivity to ultraviolet (UV) light (photosensitivity)
• High blood pressure (hypertension)
• Elevated liver enzymes
• Slow wound healing

What to expect during recovery from targeted therapy for Ewing sarcoma

While recovering from targeted therapy for Ewing sarcoma, the patient can expect a period of adjustment as their body responds to the treatment. Mild side effects, such as fatigue, gastrointestinal issues and skin rashes, may linger for some time after the therapy ends. More serious side effects, such as high blood pressure and changes in liver function, may require ongoing monitoring by the healthcare team.

How effective is targeted therapy for Ewing sarcoma?

Targeted therapy has shown promise in certain cases of Ewing sarcoma, particularly when the specific molecular targets are well-defined. However, its effectiveness can vary based on several factors, including:

• The stage of the tumor
• The complexity of the cancer’s genetic profile
• How the tumor responds to the treatment
• The development of drug resistance

Usually, the goals of targeted therapy are to slow the growth of the tumor, manage its symptoms and improve the patient’s quality of life, particularly when conventional treatments such as chemotherapy and surgery prove ineffective.

Benefit from world-class care at Moffitt Cancer Center

Through ongoing studies and clinical trials, the dedicated research team in Moffitt’s Sarcoma Program is continually evaluating and refining targeted therapies to improve outcomes and quality of life for patients with Ewing sarcoma. If you would like to learn more, you can request an appointment with a specialist at Moffitt by calling 1-888-663-3488 or submitting a new patient registration form online. We do not require referrals.