Realizing Futures

Behind the Science: Q & A with Dr. Michael Ortiz

Funding Lifesaving Research with Dr. Michael Ortiz

We’d like you to meet Dr. Michael Ortiz, Emerging Investigator Grant recipient from . This type of grant is designed for emerging pediatric cancer researchers to pursue exciting research ideas and encourages and cultivates the best and brightest researchers of the future. Dr. Ortiz’s hope is to improve the way we care for patients with Wilms tumor, the most common childhood kidney cancer. Dr.Ortiz is making progress towards addressing both of these challenges through his research which is supported by the Pediatric Cancer Research Foundation.

He tells us more about his role in helping to ensure all children have a chance at a long, healthy future.

Why are you interested in finding better treatments for Wilms tumor? Although I spend most of my time studying Wilms tumor and other high risk solid tumors in the lab, one or two days per week I get to take care of kids with cancer in the clinic. The majority of these children have Wilms tumor. Most will be cured, however in order to achieve this cure we frequently need to remove one of their kidneys and administer chemotherapy and radiation which can damage their healthy cells. This can be significant; In fact, a quarter of the survivors of Wilms tumor will have severe long-term side effects related to our treatment by the time they’re in their 20s and 30s. So even for the kids who are cured, we need less toxic therapies.

Despite our best efforts and decades of research, still some of these children cannot be saved, even with maximal therapies. This is a very hard reality for me to accept. The best way that I know how to deal with the devastation from losing a child to cancer is to learn as much as we can about their cancer.

Every cancer is different so each patient’s tumor provides a unique opportunity to learn more and move us one step closer to finding a cure for all patients. I am genuinely optimistic that one day in my life, we will be able to cure all of these kids using therapies that do not come with such significant long-term toxicities.

Share your thoughts on why you think it’s important to fund pediatric cancer research. Cooperative research efforts have been quite successful at improving outcomes for children with cancer. Now 4 out of every 5 children diagnosed with cancer will be cured. Unfortunately, there is still a significant number of children who need our help, as almost 2,000 children in the US die each year of cancer. Funding is essential to support researchers to identify new cures for those kids. Governmental funding for studying rare diseases has dwindled in recent years.

Childhood cancer researchers have relied on philanthropy to continue our efforts to identify new cures. More specifically, pediatric cancers receive only a small fraction of government funding and even less from pharmaceutical companies. As a result, foundations such as PCRF have become indispensable fo fill this funding void which allow physicians and scientists to continue efforts to identify life-saving cures.

What is your point of view on the climate of pediatric cancer research? We are at an inflection point in the fight against cancer. With the improvement in genomic technologies, it is becoming cost effective to sequence every patient’s tumor. And by doing this sequencing, we are discovering things. We are discovering that tumors are often heterogeneous, so one piece of the tumor may have different mutations than another part. We’re learning how the tumors evolve over time and become resistant to our therapies, so newer and more targeted therapies are being developed to address these resistance mechanisms. We are getting better at identifying new biomarkers to predict which patients need more or less therapy in order to achieve a cure. Finally, we are increasingly recognizing the role of epigenetics, or non-genetic changes, that are involved in driving certain cancers, which is particularly relevant in many Wilms tumors.

From a therapeutic standpoint, in the past five years there has been an amazing improvement in our ability to harness the immune system to identify and kill cancers. Checkpoint inhibitors, CAR-T cells, antibodies, vaccines, and other immunotherapies are providing a novel means to cure these tumors. In addition, we have many more precise drugs in our arsenal. This provides a variety of opportunities for cure whilst avoiding the longer-term toxicities of chemotherapy.

Now that newer drugs are being developed, we are increasingly treating tumors based on their target, mutation, or biomarker rather than by their location. We are even to the point of opening precision medicine based clinical trials. For example with the currently ongoing NCI-COG Pediatric MATCH study. I think this trend towards precision medicine is a very good thing because as we treat every patient’s tumor according to their specific aberrations. We will be able to maximize tumor-directed efficacy whilst minimizing toxicity to the rest of the body.

Learn more about Dr. Michael Ortiz, our impact and the future of curing childhood cancers.

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When you are young and healthy, it never occurs to you that in a single second your whole life could change. – Annette Funicello