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Targeted Therapy for Cancer

Dr. Vallance, Cook Children's hematologist/oncologist, takes us through the most advanced and promising targeted therapy research and clinical trials available to pediatric cancer patients. She explores how this oncological research is leading to higher response rates, less toxicities, more cures and the hope for a long and happy future for babies, children and young adults with rare forms of cancer.

Meet the speakers

Dr. Kelly Vallance

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Host: Welcome to Cook Children's Perspectives podcast. We're here today with Dr. Kelly Vallance from our Cook Children's Oncology Program. Dr. Vallance joined the Cook Children's family in 2009 and specializes in treating childhood cancer, including solid tumor and genetic syndromes that predispose children to developing cancer. She has served as a young investigator on the Children's Oncology Group Renal Committee, and is the vice chair of the Children's Oncology Group, A R E N 1831 Wilms' tumor research trial. Dr. Vallance is currently active in research at Cook Children’s most recently she received the St Baldrick's infrastructure grant to help develop a targeted therapeutics program, allowing her to focus on targeted therapy for cancer patients. Welcome Dr. Vallance and thank you for being here today to talk about targeted therapies.


Dr. Vallance: Thank you. I'm very excited to be here and to share some of the future research trials and opportunities for our patients here at Cook Children’s.


Host: So to get started, can you give us a quick overview of what targeted therapy for cancer patients is?


Dr. Vallance: So targeted therapy is different from traditional cytotoxic or chemotherapy, and that targeted therapy is molecular medicine that specifically blocks the growth of cancer cells by interfering with targeted molecules or cellular processes that is needed for the tumor cells to grow and tumor cells to survive in the body. So targeted therapy acts specifically to kill tumor cells. Traditional cytotoxic chemotherapy, or the traditional chemotherapy that has been around for decades is not specific, it doesn't target certain cellular processes. It works by killing all cells that are growing rapidly. So traditional cytotoxic chemotherapy has a lot more side effects and toxicities associated with it.


Host: Why has Cook Children's chosen to participate in targeted trials?


Dr. Vallance: So Cook Children’s is participating in targeted therapy trials because it truly is the future of cancer treatment for children and for adults, but especially for types of cancer that are not easily curable with traditional therapies.


Host: What are the types of targeted therapies we offer at Cook Children’s?


Dr. Vallance: We have a large assortment of different targeted therapies that are currently available for our patients. We offer MIBG. And in addition, other targeted trials and immunotherapies that have monoclonal antibodies that we use in neuroblastoma and other solid tumors. We have opportunities for relapsed patients that have diseases that are difficult to cure and have multiple trials that will use tumor sequencing to identify mutations in the tumors that then we're able to pair targeted therapies that are specific for that patient and that patient's tumor mutations that will hopefully offer opportunities for responses to treatment and cures in children that otherwise have no options. One of the trials that I'm very excited about that we will be able to offer our patients is a collaboration with Texas Children's where a grant has been funded through the NIH. That is an opportunity for our patients here at ...


... Cook Children's called Kid Can Sequence and Kid Can Seq is a trial that all of our new diagnosis patients will be offered in eligible for, for solid tumors and lymphomas in which we have opportunities to sequence the patient's germline or their DNA to look to see if there are cancer predisposition syndromes, or a genetic reason that these kids developed cancer. And that would have implications for their family members and for siblings, but also some of these patients that have high risk disease, we'll be able to sequence their tumor through this trial. And that may provide additional treatment options or additional treatment therapies that these kids may be eligible for. And, um, this was a great opportunity through this NIH grant for the children of our North Texas and West Texas and surrounding areas.


Host: So who is a candidate for targeted therapy? And when is it an option?


Dr. Vallance: When traditional standard treatments don't work for children then targeted therapy is certainly an option that we want to provide to all, all of our patients. Childhood cancer and research in childhood cancer has made tremendous strides over the last four decades. There are many cancers that are now curable in children. Unfortunately, there are high-risk cancers, um, ones that are aggressively spread at diagnosis and also cancers, solid tumors and brain tumor cancers and relapsed cancers that are still very difficult to treat and cure. These patients are the ones that we think targeted therapy will offer more cures, more chances for prolonged life and maybe cures.


Host: Do you see a time when targeted therapy might become the protocol, I guess, for, for treating children?


Dr. Vallance: Absolutely. Right now, um, since they are newer medications, newer therapies and experimental medicines they’re used as second line therapies or salvage therapies, there are new medications, new targeted agents that are likely going to become the frontline therapies and may in the future be used with traditional chemotherapies, but they are able to target the tumor cells and may provide cures and may become the standard of therapy in the future.


Host: Sure. So is there anything different about the time it takes to open targeted therapy as compared to traditional treatments?


Dr. Vallance: Targeted therapies are very comprehensive and as they are experimental trials, often phase one or phase two trials, looking at dosing efficacy and have not been FDA approved yet, these trials take a incredible amount of time and resources and personnel from our research team and doctors, um, they investigators to open these trials and to conduct them safely and in standard with all IRB and research needs and regulations. So each targeted therapeutic trial is probably thousands of man hours and truly a multidisciplinary team effort with research to be able to open these trials. And that is certainly our goal to increase the amount of these therapies and trials that we have available for our patients.


Host: What kind of impact do these therapies have on pediatric patients?


Dr. Vallance: These trials absolutely can impact patient's survival in outcome and quality of life and can bring hope to patients and families for improved outcomes.


Host: Is there a specific case study or story from a patient who has received this type of treatment that you can share with us?


Dr. Vallance: I can think of several examples of kids that have benefited from targeted therapy and immunotherapy here at Cook Children's. One that is most striking is a case of infantile fibro sarcoma, a newborn infant that was born with infant fibro sarcoma of his lower leg. The entire leg, um, right at birth was found to have a tumor and was deformed. And this newborn underwent an amputation and several years of traditional chemotherapy, he for about a year and a half underwent chemotherapy, um, had relapsed later in his lungs. And then ultimately about six months after that when he was probably about 18, 20 months old developed metastatic lesions in his brain, these were inoperable though. He had had multiple surgeries throughout his, his short life, but, um, we obtain sample of one of the brain metastases and did perform genomic sequencing or tumor sequencing on his specific tumor.


Dr. Vallance: And in that we looked for any mutations that may have drugs that were available, that we could target, um, to try to combat his disease. Um, one of the mutations we found in his tumor was an entrek. So a tyrosine kinase receptor, uh, that had a mutation and there is a medication and in track inhibitor named entrectinib that has been shown to be effective with tumors that have that type of mutation. And at that time, we reached out to the pharmaceutical company and asked for the drug for, um, compassionate use. And, uh, we were able to provide this child toddler, the medication that he took once daily, he had significant response and his inoperable lesions and every metastasis that he had did shrink. And he had, um, had a very good, good response to this. Since that time we've actually partnered with the pharmaceutical company and now have the drug available through a phase two trial here at Cook Children’s. So we're excited to be able to offer that to other children and other infants that may have this terrible disease.


Host: What are the early outcomes for targeted therapies?


Dr. Vallance: So, there are certainly differences between all the targeted therapies, immunotherapies that use antibodies or other agents are now being considered, um, upfront therapy for neuroblastomas. So the outcomes for neuroblastoma is tremendous with great evidence of response. There are phase one phase, two trials that have showed significant response for many different types of cancer and malignancies. So immunotherapy is, and targeted therapy is very promising and has showed great early effects in early responses.


Host: So what does the future of cancer research look like here at Cook Children's?


Dr. Vallance: The future of targeted therapy and trials here at Cook Children’s is very promising. With the St. Baldrick's grant for infrastructure we have been able to create a targeted therapeutics program and through the, have created a targeted therapeutics program coordinator. And the goal of this is to increase the number and scope of phase one phase two, targeted clinical trials available to patients here at Cook Children’s. Our goal with this program is to increase the number and be able to offer a full complement of targeted therapy through consortium's that we already have. And in addition, new industry sponsors, we've already been able to increase the number of tumor sequencing and genomic profiling that we do for patients and with the targeted therapeutics program, we'll be able to exponentially bring more opportunities and more hope to the children of North Texas.

Host: Dr. Vallance, thank you so much for taking the time out of your very busy schedule to talk with us today and for all you do for the kids. From Cook Children’s Sparkle Fly studio, thanks for listening. And if you want to learn more about cancer treatment and research at Cook Children’s, please visit us at Cook Children’s dot org.