Oncothon: Sarcoma Program

Oncothon is a telethon spanning 24 hours, dedicated to gathering donations for childhood cancer research. Professor Mascarenhas, who holds positions as the Director of Pediatric Hematology and Oncology at Cedars-Sinai Guerin Children’s and the Medical Director of the Sarcoma Program at Cedars-Sinai Medical Center, will outline the aims and targets of the sarcoma program at Cedars-Sinai Medical Center. 

Speakers

Leo Mascarenhas, MD, MS, is a globally renowned pediatric hematologist-oncologist specializing in sarcomas. He holds the position of Director of Pediatric Hematology and Oncology at Cedars-Sinai Guerin Children’s and serves as the Medical Director of the Sarcoma Program at Cedars-Sinai Cancer Center.

Additionally, Mascarenhas chairs the voting body of the Children’s Oncology Group (COG). He has previously served on the board of the American Society of Pediatric Hematology and Oncology and contributes as a reviewer for numerous esteemed medical journals.

Additionally, he provides medical consultation for the Oncologic Drugs Advisory Committee of the U.S. Food and Drug Administration. His research is focused on mainly solid tumors, particularly rare tumors.

Moderator

Ricardo García is the Co-Founder, Chief Executive Officer, and a Member of the Board of Directors at Oncoheroes Biosciences. Ricardo has a diverse background in computer engineering, management, and business administration. His journey into entrepreneurship began early, leading to the successful sale of his first company when he was just 22 years old. This led Ricardo to start and grow multiple tech-focused companies, all driven by a passion for innovation.

In 2011, his son Richi was diagnosed with a brain tumor. This challenging experience prompted Ricardo to uproot his life and relocate to Boston to support his son’s treatment journey. During this period Ricardo discovered significant gaps and challenges in the support system for childhood cancer patients and their families.

Motivated by his journey, Ricardo founded the Richi Childhood Cancer Foundation, a non-profit organization with a global outreach. The foundation aims to address the unmet needs in childhood cancer care and support, leveraging Ricardo’s passion for making a meaningful difference in the lives of children and families affected by cancer.

0:15 Introduction

0:46 Leo Mascarenhas

Follow for transcript bellow

Ricardo Garcia: We had a third speaker that is not able to join the call, but we’re gonna play a video. His name is Leo Mascarenhas, the division chief of the pediatric hematology oncology and director of the sarcoma program from within the oncology group.

So, because we have so much time, I really just want to thank you again, both of you, for joining the call today and I want to ask my colleagues to play the video so we can finish the session on time. Thank you so much.

Leo Mascarenhas: Hi, everyone. My name is Leo Mascarenhas, and I’m the division chief and director of pediatric hematology and oncology at Cedars-Sinai Guérin Children’s, and the medical director of the Sarcoma program at Cedars-Sinai Cancer in Los Angeles, California.

I would like to thank Ankur Daily for the invitation and the opportunity to speak on International Childhood Cancer Day about the benefits of a sarcoma program that is not restricted by age limits.

As you may know, sarcomas are cancers that arise in the bones and soft tissues of the body and are rare in both the pediatric and adult populations.

In pediatrics, they comprise less than 10% of all cancers diagnosed, compared to less than 1% of all cancers in adults. Some sarcomas are more common in children, while others are more common in adults, with a considerable overlap, especially in the adolescent and young adult age groups.

There are over 100 sarcoma subtypes that can have varied presentations, but more importantly behave very differently clinically based on biology.

Further, many sarcomas have known molecular oncologic fusions or drivers, some of which that can be targeted therapeutically. Therefore, it is important that sarcoma treatment be dictated by biology or subtype, rather than whether the patient is being treated by a pediatric oncologist at a children’s hospital or a medical oncologist at a center that primarily cares for adults. Sarcoma care is truly the model of multidisciplinary care in oncology.

It is critical to have an expert multidisciplinary team comprised of pediatric and medical oncologists, surgical and orthopedic oncologists with expertise in both adults and children, reconstruction surgeons, radiation oncologists with experience treating adults and children, radiologists with expertise in musculoskeletal and solid organ radiology, pathologists with expertise in sarcoma, molecular and cancer geneticists, genetic counselors, expert nurses with experience in complex care management, physical and occupational therapists, and clinical social workers and psychologists.

It is very important for sarcoma patients to have access to fertility preservation. It is essential that this expert multidisciplinary team meet regularly to discuss the management and progress of their sarcoma patients for them to have the best possible outcomes. The approach to diagnosis and management of sarcoma patients is similar in both children and adults.

The supportive care and survivorship needs of pediatric and adult patients are often similar, particularly for specific needs that bridge the age gap, as seen in the adolescent and young adult (AYA) population. Evidence suggests that young adult patients treated with pediatric-type regimens for certain sarcomas, such as Ewing’s sarcoma and rhabdomyosarcoma, experience improved survival outcomes compared to historical controls.

This mirrors the outcomes reported in young adults diagnosed with acute lymphoblastic leukemia, who also have better survival rates when treated with pediatric regimens. Additionally, patients with fusion-driven sarcomas, such as TRK fusion sarcomas, benefit more from treatment with selective TRK inhibitors rather than traditional chemotherapy.

These findings apply to both children and adults, indicating that treatment strategies from pediatric oncology may offer advantages for young adults and that targeted therapies can improve outcomes for certain sarcoma patients across age groups.

Patients with sarcoma, regardless of age, are likely to benefit significantly from being treated within a single program. Such a program involves a team of dedicated expert physicians and healthcare providers who understand the patient’s needs from diagnosis through treatment, supportive care, and survivorship, including research efforts aimed at improving outcomes.

Clinical trials should incorporate children early in development, considering possible differences in pharmacokinetics and potential toxicities related to growth and development. Additionally, attention should be given to drug formulation needs specific to very young children to ensure safe and effective treatment.

Recently, the lower age limit for participation in many adult sarcoma trials has been reduced to include teenagers, while the upper age limit for certain pediatric sarcoma trials has been extended to include adults up to age 50. This expansion allows for broader access to new drugs and may potentially improve accrual times, enabling faster acquisition of clinical trial results.

Efforts are ongoing within certain research organizations such as the Sarcoma Alliance for Research Collaboration, the Arkansas Sherm, to include children in their clinical trials. Meanwhile, pediatric clinical trial cooperative groups like the Children’s Oncology Group and the European Pediatric Sarcoma Study Group are working to include young adults in their sarcoma trials at Cedars-Sinai Cancer and Guérin Children’s.

By bridging age groups in these trials, researchers can advance treatment options and outcomes for a wider range of sarcoma patients.

We have established a sarcoma program that cares for sarcoma patients of all ages, with the required expertise to offer and deliver the best multidisciplinary care, provide patients with access to clinical trials, and opportunities to participate in research with the goal of achieving the best possible outcomes for our patients.

We live in exciting times where there are new therapies being developed every day, including new drugs that specifically target cancer cells or harness the immune system to fight cancer, bringing hope to our patients.

While outcomes for certain sarcomas have improved in recent years, there is still much work to be done, especially to improve the outcomes for patients with metastatic sarcomas and relapsed sarcomas, as well as for survivors who suffer from long term treatment effects.

This can only be done through providing access to expert care, improving participation in clinical trials, and supporting research at all levels. This is a good reminder for us all on International Childhood Cancer Day and every day of each other.

Thank you and have a good day.