Texas Children's Cancer Center Alex's Lemonade Stand Center of Excellence
The Developmental Therapeutics Program at Texas Children's Cancer Center, Baylor College of Medicine is one of the country's leading centers for the development and conduct of early phase studies for childhood cancer The Alex's Lemonade Stand Foundation Centers of Excellence (ALSF COE) award addresses two of the major challenges in pediatric cancer drug development: the inadequacy of infrastructure support for early phase clinical trials, and the very small cadre of individuals optimally trained to implement and conduct such studies. ALSF COE funding will enable us to expeditiously open Phase I and 2 trials and to enroll more patients on such trials. Furthermore, it will provide outstanding Research Scholars in Developmental Therapeutics with durable and sustained support, helping to ensure their future success as independent investigators. Thus, the ALSF COE award will be transformational, increasing the number and quality of Phase I and 2 studies available for children with refractory childhood cancer while training the next generation of leaders in the field of developmental therapeutics.
Project Update
We spoke with May 2014 Featured Researcher Stacey Berg, MD about the Center of Excellence at Baylor College of Medicine:
Why did you choose to create a Center of Excellence at Texas Children’s Cancer Center, Baylor College of Medicine?
We were fortunate to have the opportunity to apply for a Center of Excellence in Developmental Therapeutics based on our longstanding interest in this area. We have had a very active Developmental Therapeutics Program since 1994, and the ALSF grant is enabling us to expand these efforts.
How long does it take to study a new therapeutic intervention?
It can take 10 years (and hundreds of millions of dollars) for a new anticancer drug to go from early phase testing to FDA approval. Even after that, it can be another 3-5 years before we fully understand the drug’s place in the treatment of childhood cancer.
How many studies do you run concurrently for childhood cancer?
At any given time we have about 30-40 Phase 1-2 studies open for new childhood cancer treatments. However, because early phase studies only enroll a few patients at a time, then observe them carefully for any untoward effects before the next children can enroll, the number of studies with open spots for patients at any given time is much smaller.
What obstacles/challenges do you face in bringing new treatments to children with cancer?
The advances that have occurred in treating childhood cancer over the years mean that more children are successfully treated for cancer. Paradoxically, this has made it harder to develop new treatments. Fewer children suffer from relapsed or refractory disease, meaning that clinical trials to test therapy for them are more difficult to conduct. In addition, access to new drugs for testing is still sometimes slower than we’d like.
What has this grant from ALSF allowed you to do that you wouldn’t be able to do otherwise?
The ALSF COE grant has filled a critical need for us. As many people know, the ongoing issues with the federal budget mean that funding for biomedical research has shrunk drastically. One of the hardest things to fund is infrastructure—meaning the people and processes to conduct clinical trials, as opposed to the supplies and lab equipment needed to conduct other kinds of research. By funding research nurses, study coordinators, and data managers, the COE has permitted us to maintain and expand our clinical research infrastructure in extremely challenging times.
What are Developmental Therapeutic Scholars? How are the scholars being trained and what are the long term expectations for them?
The Developmental Therapeutics Scholars are young scientists being trained and supported to become the next generation of leaders in developing new treatments for childhood cancer. Our center is one of the few in the nation that includes a Clinical Pharmacology Fellowship led by a pediatric oncologist, Dr. Lisa Bomgaars. This enables us to provide the Scholars with advanced training in translational research and new drug development.
Have you achieved effectiveness for any of the therapeutics you are studying? What does this mean for children with cancer and their families?
We are involved in a number of studies of new treatments for which we have great optimism. This includes several studies of new molecularly targeted drugs as well as innovative agents like chimeric antigen receptor (CAR) T-cells for both leukemias and solid tumors. Our ultimate goal is to move forward towards the day when no child or family has to hear that there is no treatment for their disease, and instead a cure is available for all forms of childhood cancer.
Developmental Therapeutics Scholars Spotlight
Jennifer Foster, MD, MPH
Dr. Foster is a COE Scholar who is receiving advanced training in translational research and drug development to help bring new treatments for childhood cancer to patients.
"I am very grateful to have the opportunity to be a Centers of Excellence Scholar. This grant has had a huge impact on my career, as it has given me the protected time necessary to both continue my research on novel cancer therapeutics, as well as complete an additional fellowship in Clinical Pharmacology. Furthering my research and receiving this extra training in pharmacology are invaluable in establishing my career as a junior investigator in novel cancer therapeutics."
What attracted you to pediatric cancer research and developmental therapeutics in particular?
I have been interested in pediatric cancer from the time I decided to pursue a career in medicine. Throughout my medical training and prior volunteer experiences, I have gained an enormous respect for both our patients and their families. Their daily struggles to battle cancer continuously leave me in awe of their strength and accomplishments. Cure rates for pediatric cancers have made marked advancements over the decades and continue to improve due to the efforts of patients, families, and researchers. Seeing the toll that our current treatments take on patients, and knowing that our current therapies are not enough to cure all of our patients motivate me to research ways to minimize toxic effects of therapies while discovering new cures.
Describe your role as a Developmental Therapeutics Scholar. How are you being trained? What are your long term professional goals?
As a Developmental Therapeutics Scholar, I spend the majority of my time researching both novel chemotherapy agents and the clinical pharmacologic effects of our current therapies. As such, I am taking formal pharmacology classes through the University of Houston School of Pharmacy, as well as participating in NIH lecture series on clinical pharmacology. My long term professional goal is to be a leading clinical investigator in the area of cancer pharmacology.
What therapy (in simple terms) are you working on? Has anything surprised you about your research?
I am currently studying a novel chemotherapy agent in the lab, and performing the pre-clinical work necessary to advance this drug into pediatric clinical trials.
Has effectiveness been achieved for any of the therapeutics studied by the COE? What does this mean for children with cancer and their families?
The COE provides a unique opportunity to form a small consortium of institutions across the nation who excel in advancing the field of developmental therapeutics. Being able to collaborate with other institutions, means that more children with cancer can benefit from research across the nation.
What obstacles/challenges do you face in bringing new treatments to children with cancer?
The process of developing a new cancer drug, conducting pre-clinical experiments, early phase clinical trials, and eventually being able to offer the drug to all children with cancer is a long and costly process. This process faces many obstacles and challenges, as funding for both the researcher’s time and cost of the experiments can be difficult to come by.
What has this grant from ALSF allowed you to do that you wouldn’t have been able to do otherwise?
This grant from ALSF has allowed me to overcome these challenges by both providing the means necessary to conduct the pre-clinical work necessary to advance novel therapeutics into early phase pediatric clinical trials, as well as allowing me to gain extra training in clinical pharmacology which will allow me to better understand the properties and effects of these novel therapeutics and thus lead to more effective treatments for patients.
Ronald Bernardi, MD, PhD
Dr. Bernardi is a COE Scholar who is receiving advanced training in translational research and drug development to help bring new treatments for childhood cancer to patients.
"The Alex’s Lemonade Stand Foundation Centers of Excellence Scholar grant has had a transformative impact on my career. At a time when research funding is exceedingly difficult to come by, this award has supported me during a critical period of my career as I develop as an independent investigator. It has allowed me to continue to pursue my basic, translational, and clinical research efforts, as well as obtain the high level training and mentorship that are required to become a leader in the field of pediatric oncology."
What attracted you to pediatric cancer research and developmental therapeutics in particular?
I have had a longstanding interest in developmental therapeutics, particularly as it pertains to pediatric oncology. Children who are stricken with a diagnosis of cancer have the potential of a lifetime of fulfillment that too often is a lost opportunity. Thus, the potential for impact of a cure is enormous for these children and their families. Nothing is more rewarding to me than the opportunity to make a positive change in the lives of these individuals by developing novel therapies that provide more hope.
Describe your role as a Developmental Therapeutics Scholar. How are you being trained? What are your long term professional goals?
As a Developmental Therapeutics Scholar, I am being trained by leaders in the fields of pediatric oncology, pharmacology, genetics, biochemistry, and ethics. I am learning not only from the faculty at my own institution, but I have also had the opportunity to meet with Scholars and faculty from other Centers of Excellence. In addition, I have received training in the regulatory process for drug development, which provides a framework for understanding the requirements for bringing a new therapy into the clinic.
What therapy (in simple terms) are you working on? Has anything surprised you about your research?
My personal interest is in finding treatments for what we currently call high-risk disease. In other words, these are the disease settings for which we do not presently have adequate treatments, and they include subsets of patients with neuroblastoma, sarcomas, and brain tumors. My long term goals include the use of functional genetic approaches to identify unique vulnerabilities of cancer based on the genetic alterations that drive the disease progression.
Frequently, such susceptibilities are most evident when combination therapies are used, and another goal of mine is to improve the way in which we combine therapeutic agents, including how we study potential combinations in the laboratory and early phase clinical trials. In this way, my goal is to increase the precision with which we are able to treat an individual patient. For example, I am currently working on two novel combinations of therapeutic agents, and we are working to identify the scope of patients for which these therapies are most likely to be beneficial. Thus, we intend to develop the means to identify the subset of patients who are likely to respond to a particular therapy. In that way, we can avoid exposing patients who are unlikely to respond to the risk of toxicities and allow other therapeutic options to be explored for them. Asking precise questions in clinical trials is particularly critical in pediatric oncology where the number of potential patients is relatively small.
Has effectiveness been achieved for any of the therapeutics studied by the COE? What does this mean for children with cancer and their families?
It is our obligation to our patients and their families to accomplish this goal. While my current efforts in the laboratory have not yet led directly to an improved treatment for pediatric oncology patients, we are encouraged by our findings that we will soon have some exciting new concepts to evaluate in the clinic. In addition, I continue to enroll patients on a variety of ongoing studies as a member of our Developmental Therapeutics Program at Texas Children’s Hospital. The Centers of Excellence grant has not only supported my career and our infrastructure to run such trials, but, in collaboration with the other Centers of Excellence, has brought together a team of expert and determined investigators at leading institutions working together to enable the rapid translation of new ideas into optimally designed and implemented clinical trials. Thus, we can speed the improvement of cancer therapy, as well as train the next generation of leaders in developmental therapeutics so that our progress will continue to come for years to come.
