Ossama Maher, MD, Medical Director – Blood and Marrow Transplant Program, Nicklaus Children’s Hospital

In an interview with Invest:, Dr. Ossama Maher, director of the Blood and Marrow Transplant Program and associate director of Pediatric Neuro Oncology at Nicklaus Children’s Hospital. Dr. Maher, who is also a clinical associate professor at Florida International University’s Herbert Wertheim College of Medicine, discussed the evolution of pediatric cancer treatment through multidisciplinary care, advanced surgical technologies, and innovative therapies. “The combination of multidisciplinary care, advanced technology, and ongoing research is reshaping Pediatric Oncology,” Dr. Maher said.

How does your multidisciplinary approach improve diagnosis and treatment for children with brain and spinal tumors?

Treating pediatric brain tumors remains one of the most challenging areas in oncology, as survival rates have historically lagged behind other childhood cancers. For many years, progress in diagnostics and treatment options was limited, which made outcomes difficult to improve.

Today, that is changing. Being part of a comprehensive brain and spine program allows the team to offer cutting-edge technologies, innovative therapies, and access to research and clinical trials. This multidisciplinary structure brings together specialists across fields to provide a broader range of options for patients and their families. Compared to a decade ago, there are now significantly more treatment pathways available, allowing for a shift from conventional approaches to more advanced, targeted therapies that aim to improve outcomes.

How does collaboration between neurosurgery, oncology, and advanced imaging enable more personalized treatment plans?

The collaboration with our Neurosurgery team plays a critical role as the first step in treatment. The goal is to remove as much of the tumor as possible while preserving the child’s neurological function. Achieving this balance requires advanced surgical precision.

The integration of technologies such as intraoperative MRI and ultrasound-guided imaging allows surgeons to assess tumor removal in real time. During surgery, imaging can confirm whether additional resection is needed or if the tumor has been fully removed. This minimizes the need for follow-up procedures and reduces risk to the patient.

These advances directly support oncology treatment by improving surgical outcomes, which in turn enhances the effectiveness of subsequent therapies, such as chemotherapy, radiation, and targeted treatments. The result is a more precise and individualized care plan for each patient.

How are innovations like CAR-T cell therapy and gene therapy changing outcomes for pediatric patients?

Cell therapy, including CAR-T cell therapy, represents a significant advancement in pediatric oncology. For patients with limited or no remaining treatment options, these therapies offer new hope.

The hospital’s CAR-T cell therapy program, in place since 2019, has demonstrated strong outcomes, particularly for patients with leukemia and lymphoma. Many patients who previously had few options are now achieving positive results, including remission and, in some cases, a cure.

In addition to oncology, the application of these therapies is expanding. New clinical trials are exploring the use of CAR-T cell therapy for autoimmune diseases, such as lupus, broadening the impact of this approach beyond cancer care. This expansion reflects the potential of immunotherapy to transform treatment across multiple conditions. Gene therapy, as a new innovative therapy in some hematological conditions, also provides great hope for children diagnosed with sickle cell disease and thalassemia when a bone marrow transplant carries high risk of complications.  

What distinguishes your bone marrow transplant program in terms of outcomes and patient care?

The blood and marrow transplant program at Nicklaus Children’s Hospital is one of the most established in South Florida, with a history dating back several decades. It specializes in treating highly complex cases, including high-risk malignancies and hematological and immune conditions.

The program consistently delivers strong outcomes, supported by a multidisciplinary team working closely together. Its reputation extends beyond the local community, attracting patients from across the region and internationally, particularly from Latin and South America.

Success is driven by a combination of experience, advanced therapies, and a commitment to treating even the most challenging cases. Many patients who arrive with limited options have been successfully treated through innovative approaches, including immunotherapy and transplant.

What factors are driving improved survival rates in transplant patients?

Improved outcomes are the result of several key factors, beginning with the expertise and dedication of the clinical team. Continuous education and the adoption of the latest treatment protocols ensure that care remains aligned with current best practices.

Patient selection and timing are also critical. Determining the optimal moment for transplant — neither too early nor too late — plays a significant role in success rates. In addition, treatment intensity is carefully calibrated for each patient. By tailoring therapy to the individual, the team can balance effectiveness with reduced toxicity, improving both survival and quality of life.

The introduction of newer therapies with lower toxicity has further enhanced outcomes, allowing for more effective treatment while minimizing adverse effects.

How is the hospital leveraging clinical trials and research collaborations to advance care?

Clinical trials are essential to progress in pediatric oncology. The hospital participates in multiple research consortia and collaborative studies, enabling access to the latest therapies and treatment protocols.

These collaborations span a wide range of specialties, including brain tumors, solid tumors, and bone marrow transplant. Participation in national and international research initiatives ensures that patients benefit from cutting-edge treatments while contributing to broader advancements in the field.

The program continues to expand its research efforts, including new trials focused on immunotherapy and other emerging treatments. This commitment to innovation allows the hospital to offer options that may not be available elsewhere, particularly for patients with rare or complex conditions.

How are advanced surgical technologies improving precision and safety in pediatric neuro-oncology?

Technological advancements have significantly transformed pediatric neuro-oncology over the past decade. Tools such as intraoperative MRI allow surgeons to visualize the tumor during the procedure, guiding decision-making in real time.

This approach enables more complete tumor removal while minimizing the risk of damaging critical areas of the brain. It also reduces the need for additional surgeries, as adjustments can be made immediately during the initial procedure.

Other techniques, including laser ablation, provide minimally invasive options for certain tumors. Together, these innovations enhance both safety and effectiveness, contributing to better overall outcomes.

How has expanded access to donor resources improved transplant opportunities for children?

Access to suitable donors has historically been a significant challenge, particularly in diverse populations. Today, multiple strategies have expanded the pool of available donors, making transplants more accessible.

In addition to traditional donor registries, cord blood has emerged as an important resource, providing an alternative source of stem cells. Advances in transplant techniques also allow for the use of partially matched donors, including family members, which was not previously possible.

These developments mean that nearly every patient can now find a viable donor option. As a result, more children can proceed with life-saving transplants, improving both access to care and overall outcomes.

Overall, the combination of multidisciplinary care, advanced technology, and ongoing research is reshaping pediatric oncology. With continued innovation and collaboration, the field is moving toward more effective, personalized treatments that offer greater hope for patients and their families.