Ziad Khatib, MD, Medical Director – Division of Hematology/Oncology, Nicklaus Children’s Hospital

Invest: sat down with Dr. Ziad Khatib, director of the Division of Hematology and Oncology at Nicklaus Children’s Hospital, to discuss the expansion of the hospital’s cancer and blood disorders programs, the role of research in improving access to advanced care, and the future of more precise pediatric treatments. “The future is going to be more effective treatments, what we call more targeted treatments, with fewer side effects,” Dr. Khatib said.

What sets your division apart in the care of pediatric cancer and blood disorders?

What sets us apart is the breadth of what we offer and the level of specialization we have built over time. Nicklaus has designated four institutes, including cancer, heart, neuro, and orthopedics, which reflect a significant investment in key areas of pediatric healthcare. The Division of Hematology/Oncology is part of the Helen & Jacob Shaham Cancer and Blood Disorders Institute. 

I have been here for more than 30 years, so I have seen the hospital evolve from Miami Children’s to Nicklaus Children’s Hospital. Today, Nicklaus Children’s is home to one of the two largest pediatric cancer programs in the state of Florida. We cover the full spectrum of pediatric hematology and oncology, including leukemia, brain tumors, solid tumors, sarcomas, anemias, sickle cell disease, bleeding disorders, and routine blood-related concerns. We also provide bone marrow transplantation, and we performed the first bone marrow transplant in South Florida in 1991.

Another important differentiator is that Nicklaus Children’s combines an academic and teaching mission with a strong community presence. We are one of the largest teaching hospitals in the Southeastern United States. We have a pediatric residency program, fellowship programs, and a close relationship with Florida International University’s Herbert Wertheim College of Medicine. That means we are caring for patients while also helping train future pediatric specialists.

We want families in South Florida to understand that high-level, state-of-the-art pediatric cancer care is being delivered here. People may be familiar with national institutions, but the treatment of these patients is happening here in our community.

What advancements in treatment have you seen firsthand, and how is your division incorporating them?

Pediatric hematology and oncology has always been at the cutting edge of research and treatment. One of the biggest changes has been in diagnosis and precision. Today, when we diagnose cancer, we rely heavily on genetic testing and molecular classification. In the past, diagnosis depended much more on what a pathologist saw under the microscope. Now, we can classify tumors with far greater precision by analyzing their molecular and DNA characteristics.

That has made personalized medicine much more real. We can design treatments based on the specific genetic changes in a tumor, which allows us to use targeted therapies in a more effective way. At this point, molecular classification and targeted therapy are becoming standard for many of our cancers.

We have also seen major advances in imaging and diagnostics. For example, in brain tumors, we have access to intraoperative MRI technology. That allows surgeons to perform MRI imaging during or immediately after surgery, without having to wake the patient and wait until the next day. We are one of the only hospitals in South Florida with that capability.

On the research side, we are able to offer studies that are not available elsewhere in Florida. In brain tumors, for example, we are part of the CONNECT Consortium, one of a small number of hospitals in the country offering certain early-phase treatments. We are also working with Magnetic Resonance Focused Ultrasound, which helps improve drug delivery to the brain. We are one of only three hospitals in North America to have that technology.

Immunotherapy is another major advancement. For childhood leukemia, we are using CAR T-cell therapy for patients with recurrence or relapse. That involves engineering a patient’s T cells to attack the cancer. We are also preparing for more gene therapy applications in conditions such as hemophilia and sickle cell disease.

What role does research play at Nicklaus Children’s, and how do you make clinical trials accessible to patients?

Research is central to what we do. It is one of the main factors that distinguishes us from other pediatric hospitals in South Florida. Our responsibility is to offer treatments and opportunities that are not available elsewhere.

We have a research institute that supports the regulatory and operational work needed to conduct clinical studies, while our physicians focus on the clinical side. We participate in national and specialty networks, such as the Children’s Oncology Group, the CONNECT Consortium, and bone marrow transplant research protocols. We also have physician-led investigations.

One example is a collaboration with Florida International University for a study called the ex vivo trial. In that study, we can take a fresh tumor sample from a patient during surgery or biopsy and send it to a specialized lab, where it is tested against a large panel of drugs to determine which may be most effective for that specific patient. That is a practical example of personalized treatment driven by research.

For pediatric oncology, research is a top priority. Our goal is to have more than 50% of our oncology patients enrolled in some type of clinical trial. We are also accredited through the American College of Surgeons for cancer, and research plays an important role in maintaining those standards and advancing care.

What are some of the biggest challenges your team faces today?

The encouraging part of pediatric oncology is that outcomes are often better than in adult cancer. Overall, we cure about 80% to 85% of children with certain types of cancers, and in some diseases such as leukemia, cure rates are now around 95%. Those advances are the result of years of research, better diagnostics, and newer therapies, including immunotherapy.

The hardest challenges are the diseases for which we still do not have a cure. Some brain tumors remain especially difficult to treat, including DIPG. We also continue to offer hope through clinical trials and new treatment approaches, because that is how progress happens.

Another challenge is that pediatric cancer is relatively uncommon, which means it does not always receive the same level of attention as adult cancers. There is less financial incentive for drug companies, so we continue advocating for more support from the FDA, local agencies, and philanthropic organizations. Local foundations also play an important role in helping fund research and support families.

Access is another major issue. In some cases, treatments exist, but the difficulty lies in getting them covered and delivered. Gene therapies and other advanced treatments can be transformative, but they can cost millions of dollars for a single patient. That makes insurance coverage a serious barrier. Science is moving quickly, but the healthcare system can still be difficult for families to navigate.

What developments are you most excited about for the future of pediatric hematology and oncology?

We are excited because the pace of discovery continues to accelerate. Molecular classification and targeted therapy are helping us move away from older, more toxic approaches and toward treatments that are both more effective and less burdensome for patients.

We are increasingly treating cancers with therapies that are highly specific to the biology of the disease, whether that is a pill, an injection, or an immunotherapy that harnesses the patient’s own immune system. CAR T-cell therapy is one example, and we expect that type of approach to expand into more cancer types over time.

The future is going to be more effective treatments, what we call more targeted treatments, with less side effects. We want children not only to survive cancer, but to go on to live healthy adult lives without long-term complications from treatment. That is where the field is heading, and that is what makes this an exciting time in pediatric hematology and oncology.

Are there any additional areas of your program that are worth highlighting?

In addition to cancer, we are treating vascular malformations with a multidisciplinary approach and using targeted therapies even though those conditions are benign. We are also treating neurofibromatosis with newer targeted options.

In bleeding disorders such as hemophilia, we are seeing major change. Instead of frequent injections, we now have treatments that can be given once a month to prevent bleeding, and gene therapy has the potential to provide a lifetime cure with a single treatment. Similar promise exists in sickle cell disease.

Those advances show how much the field is changing. The challenge now is not only continuing to innovate, but making sure patients can access the innovations that already exist.