Cleveland Clinic Children’s Brings Novel Therapies to Patients with Pediatric Cancers

New research initiative aims to increase the number of clinical trials and therapies

Although rare, pediatric cancers are the leading cause of death among American children past infancy. And even though substantial progress has been made in the recent years in the treatment of leukemias and lymphomas, effective therapeutic options for a significant number of childhood cancers are still lacking. In order to bring novel, more effective therapies to the youngest patients, Cleveland Clinic launched a new, early phase clinical trial program called Cleveland Clinic Children’s Cancer Innovative Therapy Program.

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In November 2019, pediatric oncologist Matteo Trucco, MD, joined Cleveland Clinic Children’s. In his new position, Dr. Trucco will be responsible for the development, expansion and implementation of this new research initiative. In this Consult QD interview, he reflected on the main goals and future directions of the program under his leadership.

Q: What are the main goals of the program?

A: Our main goal is the development of more effective, less toxic therapies for children with all cancers.

Q: How was it developed and who are the participants in this research initiative?

A: We are partnering with various consortia throughout the country, as well as the neighboring University Hospitals and Case Western University, to bring innovative therapies to the Cleveland area. We are also partnering with pharmaceutical companies to bring exciting new drugs, which were developed for adult tumors, to pediatric oncology where there is a huge need for new therapies. Currently, we have 11 clinicians from the Departments of Pediatric Hematology and Medical Oncology and Blood and Marrow Transplant participating in the program.

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Q: What are the innovative aspects of the program?

A: Our main goal is to make new therapies available to our patients. Typical chemotherapy has worked well for many years, curing up to 85% of pediatric cancers…but it comes with a lot of toxicity and we haven’t made much progress for some cancers in 40 years. We also learned that more chemotherapy is not the answer.

So, we are now trying to develop new drugs and repurpose the old ones to see if we can improve cancer therapies. Some of these drugs are designed to overcome the resistance to chemotherapy and make cancer cells more sensitive to this type of treatment, allowing us to decrease chemotherapy doses. We are also developing drugs that attack tumors in completely different ways, by blocking blood vessel formation. With novel immunotherapy techniques, we’re using the body’s own defenses to fight cancer. New drugs are also being developed that target the metabolism of cancer cells as well as lysosomes, effectively “starving” the cancer cells.

Q: Which clinical trials are already part of the program?

A: There are several clinical trials for bone and muscle tumors that have been recruiting patients, as well as a few open trials that were originally developed by Peter Anderson, MD, at Cleveland Clinic Children’s. My goal is to help Dr. Anderson expand these trials and develop new ones. Our initial focus will be on osteosarcoma, Ewing sarcoma and rhabdomyosarcoma. On the side of rare tumors, we’re working to develop novel treatments for clear cell sarcoma, paraganglioma and medullary thyroid carcinoma. Eventually we also plan to develop clinical trials for brain tumors and leukemias.

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Q: How do you expect this new incentive to impact pediatric cancer management over the long term?

A: I think that it will make the most cutting-edge therapies available to the patients at Cleveland Clinic Children’s, so that they will not need to go anywhere else to receive treatment. I also think that it has the potential to attract the patients from around the country and around the world to Cleveland Clinic Children’s to receive some of these exciting new therapies. What we learn [in the scope of our program], we will share and expand with the other pediatric cancer groups and centers, so that the therapies developed at Cleveland Clinic Children’s can become the new standard-of-care treatments throughout the country and the world.

Q: What advice can you offer to other medical centers interested in starting similar programs? What basic requirements need to be in place for successful program implementation?

A: You need a strong clinical research team — doctors, nurses, pharmacists and regulatory personnel, who know how to open and conduct clinical trials safely and effectively. Having expertise on the specific cancers being treated and drugs being used in the trials are also key to be able to develop trials that have the most potential to benefit the patients. Another important aspect is knowing that you don’t have to do it alone. Pediatric cancers are relatively rare, which makes it very difficult to treat enough patients to know if a new treatment is effective. So we partner with other institutions and work together to develop these trials. Having a strong team, internally or across institutions, not only helps the trials to develop faster, but also makes the treatments available to a greater number of children and facilitates fundraising efforts for the trials.