Realizing Futures

Behind the Science Q & A with Dr. Eugenie Kleinerman

We’d like you to meet Dr. Eugenie Kleinerman, Basic Science Grant Recipient from The University of Texas MD Anderson Cancer Center. These types of grants fund work that is the basis for childhood cancer research, helping to move science in the direction of improved treatments and eventually finding a cure. Osteosarcoma (OS) is the most common primary malignant bone tumor in children, adolescents, and young adults. The five-year survival rate for patients with localized osteosarcoma hovers around 65 percent. Once the original tumor metastasizes, that rate drops to 30-40 percent. Dr. Kleinerman and her team are studying the potential to identify new therapeutic approaches for treating children and adolescent and young adults (AYAs) with relapsed/metastatic and primary OS. If efficacy is demonstrated, this approach can be translated into a clinical trial for children and AYAs with OS lung metastases.

The Pediatric Cancer Research Foundation is looking forward to the new hope offered by the work of Dr. Kleinerman.

What interested you in studying sarcomas, particularly osteosarcoma? 

I was headed for a career in allergy/immunology, working with children with immune deficiencies such as Wiscott-Aldrich Syndrome. Mid-way through my fellowship at NIH, my husband took me to see a movie called “Promises in the Dark” starring Marsha Mason. It was about a women physician who moves to a small town to join a general practice.  She is called to the ER to see a high school girl who kicked a soccer ball and broke her leg.  She does not like what she sees on the X-Ray. She requested more films which revealed that this young girl had osteosarcoma.  The story is about the relationship that develops between her and this young osteosarcoma patient as she goes through her initial chemotherapy treatment and surgery, and then her relapse and death. What moved me was the scene in the movie where the doctor is on the phone with the NCI trying to find a therapy for her relapsed patient. She was told there is nothing.  Although you are unable to hear the words coming from the other end of the phone, you get the message…….”no one cares about this disease, it is too rare.” I walked out of the theater numb, not knowing what to do. I was forever changed by this movie.  Over the next 2 years, I learned all that I could about osteosarcoma and switched my research focus to investigating and developing an immunotherapy (Mepact) for children with metastatic osteosarcoma in the lung.  I wanted to change that scene. Two years later I left my permanent position at NIH as there was no avenue for me to translate my discoveries into the clinic. I was recruited to MD Anderson in 1984 where my dream became a reality. I initiated the early phase clinical trials for Mepact, which led to a randomized trial within COG which showed improved survival. Mepact was approved by the European Medicine Agency in 2010 and is now used in 45 countries around the world to treat children and adolescents with osteosarcoma.

Share your thoughts on why you think it’s important to fund pediatric cancer research.

Initiating new innovative research that is not in the mainstream is extremely challenging, particularly when it involves a rare cancer such as osteosarcoma in children and adolescents. One needs extensive preliminary data to convince NIH panels of the validity of the hypothesis. New collaborations, even between established investigators, must show not only productivity but a track-record of joint publications on the topic. Receiving support from the PCRF will allow me to build my collaboration with Dr. Watowich, an expert in dendritic cell vaccine development and tumor immunity, and expand our exciting preliminary findings to produce and publish the robust data required to support the development of a dendritic cell vaccine for the treatment of patients with osteosarcoma. Without such support, the project could not proceed.

What is your proudest accomplishment so far in regards to your research?

My proudest accomplishment is translating my idea that the immune system could be harnessed to eradicate osteosarcoma lung metastases. This necessitated a unique phase I trial design for Mepact where the “optimal biologic dose” (based immune activation), rather than the “maximum tolerated dose” (based on toxic side effects), was the goal; and a phase II trial design that used “disease-free interval” rather than “tumor shrinkage” as the endpoint to judge efficacy. These trials were done in the 1980s, well before the explosion and universal interest in immunotherapy for cancer treatment. I am one of the rare translational investigators that has had the opportunity and ability to turn my dream into a reality.

Can you tell us something about you not many people know?

I was an aerobics instructor and I also taught “BODY PUMP”, which is a syndicated weight-training program from New Zealand that is offered in numerous health clubs in the US.

What do you enjoy doing in your spare time?

In my spare time, I play golf with my husband and son. I do Pilates, lift weights, and fit in some kind of aerobic exercise (except running……hate to run!).

Learn more about Dr. Eugenie Kleinerman, 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