News & Events

Personalized cancer therapy for children a reality at Floating Hospital


Children whose cancers have recurred can now seek the very latest in targeted treatments at Floating Hospital for Children.

Under the direction of Giannoula Klement, MD, a pediatric hematologist/oncologist who specializes in rare tumors, vascular anomalies and novel drugs, this initiative offers young patients and their families new hope for improved quality of life and, ultimately, survival.

While many pediatric cancer hospitals claim to offer personalized therapy, what sets Floating Hospital’s program apart is its use of individual tumor markers for the selection of the most appropriate treatment, development of rigorous computer-based algorithms for choosing therapeutic agents as well as the advancement of biomathematical tools for evaluating outcomes by comparing each patient’s predicted response to treatment to the observed response. Data from each treated patient will be used to provide invaluable feedback to help guide treatment decisions for subsequent patients.

“Traditionally, oncology diagnoses have been made histologically; tumors are lumped together by the site where they occur,” explains Klement. “But through molecular biology research, in recent years we’ve learned that not all same-site histologically similar tumors have the same characteristics. By analyzing the tumor at the gene, protein and metabolic level, we can identify specific tumor targets that make that tumor grow and modify those targets.”

This type of gene-sequencing analysis that cost thousands of dollars several years ago now costs less than $100, and can be done in less than one-tenth of the time. It gives us a wealth of information for targeting treatment and doing so quickly, when time is so critical.

A concurrent development fueling the advancement of Floating’s personalized pediatric cancer therapy is the large and growing number of novel agents currently available.

These drugs have been rigorously tested in large adult patient populations, targeted to inhibit certain molecules we can now detect with new diagnostic tools. We are using these drugs in children who have run out of other treatment options. 

“This is different from the past paradigm in that we’re not defining our therapy by where the cell came from,” he says. “Every patient is unique; every tumor is unique. So if a patient’s cancer is
c-KIT-positive, regardless of whether it’s leukemia or a brain tumor, we look at what drug will target c-KIT.”

“With Ewing’s sarcoma, for example, we know from molecular studies that IGF-1 (insulin-like growth factor) is frequently activated,” Klement says. “There is an agent approved for treating multiple myeloma that targets IGF-1, so we’ll use it for Ewing’s sarcoma on an individualized basis.”

Doctors at Floating Hospital have developed a computer-based algorithm that analyzes each tumor’s markers and matches it with an appropriate novel drug. But we don’t use a drug just because the computer says so. We take our information to the tumor board and discuss it among experienced colleagues, gaining consensus for what is really best for the patient and family.

Once treatment is administered, it’s necessary to analyze the outcome. This is where Floating Hospital is truly on the leading edge.

Medical science has moved forward for decades by using biomathematical tools to analyze groups of patients treated the same way. The queen of these tools is the Phase III study in which a large number of patients, say a hundred, are treated the standard way and another hundred are treated a new way, and five years later survival is compared.

But we can’t use that approach anymore because we don’t have 100 patients treated the same way. The ‘old’ math is useless for us. Our solution – published within the last year – changes what is called an ‘ordinary scale’ in math terms into a quantitative scale that provides more detailed information about each tumor’s response to treatment.

“We are creating a database that will include patients treated here as well as at MD Anderson (where our previous Chief, Johannes Wolff worked before joining Floating Hospital) which we will use to predict the outcome for each individual. Then, at various time points, we carefully monitor multiple measures of the tumor’s response to treatment, including how fast the tumor is growing or shrinking, or first growing then shrinking.

Bottom line, we get quantitative measures about the tumor biology of the individual. When we want to know what worked, we need fewer patients to answer the question.

“With each subsequent treatment, we improve the accuracy of our choices,” Klement says. “And when we find a successful treatment for a particular genetic mutation, we will publish and make the knowledge widely known so others can benefit from it.”

We have to be cautious and not raise expectations too high. The reality of testing new things in medicine is that, at least in the beginning, you don’t cure. But having said that, we do have something new here. There is hope. And we have the science and innovative biomathematics to learn from those in whom it does work to teach the future.

“This is an exciting time in oncology, and it’s been a long time since we could say that,” Klement adds. “When a child has exhausted all standard treatments, I hope referring physicians will give us the option of analyzing the tumor to see if there’s a mutation we can target.”