Editor’s Note: Shannon Maude, M.D., Ph.D. is a physician scientist at Children’s Hospital of Philadelphia (CHOP), where she has specialized in treating children with acute lymphocytic leukemia, or ALL. A member of the St. Baldrick’s Foundation — Stand Up To Cancer Pediatric Cancer Dream Team, she got her start in pediatric cancer research in part thanks to a St. Baldrick’s Scholar grant.
Two years of her scholar grant were funded by the Rally for Ryan Hero Fund. Ryan’s mom reports that he is “doing great” after his latest checkup in August 2019.
We recently discussed her career and her work with children impacted by ALL. An edited transcript of that discussion is below.
(December 20, 2019) – Washington, DC – St. Baldrick’s Foundation, the nation’s largest charitable funder of childhood cancer research grants, released the following statement from Kathleen Ruddy, Chief Executive Officer, St. Baldrick’s Foundation, regarding the approval of increased funding for childhood cancer research and support for the implementation of the Childhood Cancer Survivorship, Treatment, Access and Research (STAR) Act in the final Fiscal 2020 spending package.
Congress’s Focus on Palliative and Hospice Care Shines New Light on a Variety of Issues for Pediatric Cancer Patients and Families
About 10 years ago, the St. Baldrick’s Foundation expanded its research portfolio to include funding for supportive care grants. Since then, it has funded more than 40 innovative studies in this area. Supportive care, according to the National Cancer Institute, refers to care given to improve the quality of life of patients who have a serious or life-threatening disease.
Our supportive care grants have encompassed a wide variety of topics, including studies focused on psychosocial screening, survivors’ nutrition and physical activity, chemotherapy-induced neuropathy, and mindfulness training — just to name a few. These grants are in sync with needs across the spectrum of cancer in children and adolescents/young adults (AYA) from diagnosis through active treatment to post-treatment survivorship and life-long health surveillance. As many have said, the cancer experience doesn’t end when treatment ends. Supportive care can improve the quality of life for its entire duration.
For this National Hospice and Palliative Care Awareness Month, we’ve decided to look at some of the reasons why this category of care is so very important to pediatric cancer.
The St. Baldrick’s Foundation has always been committed to innovation. As the largest private funder of childhood cancer research grants in the world, it will be no surprise that we fund innovative science, but our commitment extends beyond that.
As part of our strategic plan, we challenged ourselves to find new innovative ways that we could partner with donors in order to help them realize their goals and get more dollars working to find better treatments and cures. We developed our own “venture philanthropy” program, one that allows a donor, or a group working together, to target in what they want to fund with more specificity and involvement than we have ever been able to do before.
The first group to join us in this new effort was the Osteosarcoma Collaborative. Led by Michael Egge, father to Olivia (who was diagnosed with osteosarcoma in February 2017), and current St. Baldrick’s Foundation board chair, Katherine Lugar, the Osteosarcoma Collaborative had a clear vision about what they wanted to accomplish through their fundraising and partnership with St. Baldrick’s. They wanted to fund research that needed support to push it over the finishing line – something that was very close to being ready to be put in a clinical trial and was specific to osteosarcoma research.
St. Baldrick’s infrastructure grants are designed for one reason: treat more children on clinical trials, often their best hope for a cure. Thanks to the support of St. Baldrick’s Foundation donors, these grants, totaling nearly $1.5 million this cycle, will help treat more children on clinical trials. In this spirit, these grants primarily provide support for Clinical Research Associates.
The grand total of grants made by the foundation since 2005: $282 million. That’s an outstanding number – and we could not have done it without the support of you, our donors. Thank you!
Below, we’ve listed 25 institutions that are receiving infrastructure grants in this cycle. In this blog post, we highlight four of these grants – to help paint a fuller picture of what these grants make possible.
In August of 2019, we blogged about several research outcomes that were fueled by St. Baldrick’s donations and published in scientific journals so that other researchers can build upon them. Research publications are a major way that science moves forward.
With more than 200 new publications a year resulting from research supported by St. Baldrick’s, we’ve decided to make this an occasional series, to highlight some of the most interesting outcomes you’ve made possible.
Today on the blog, we’re talking with long-time St. Baldrick’s supporter Dr. Len Mattano, whose career in the field has included pediatric oncology and pharmaceutical research. His novel is called Celtic Crossing, and he recently discussed his writing and his life’s work in pediatric cancer with St. Baldrick’s Foundation CEO Kathleen Ruddy.
Art + Science = Innovation
“I have always been artistic in my life and creativity is very important to me,” says Dr. Anne Rios, recipient of the prestigious St. Baldrick’s Robert J. Arceci Innovation Award, who leads a research team and runs the Imaging Center at the Princess Maxima Center of pediatric oncology in Utrecht, the Netherlands. “So, when I started my scientific career, I wanted to combine both art and science to visualise the behaviour of cells in physiological condition but also during cancer.”
Kids with cancer and their families are in a constant state of worry. While we can’t always ease those burdens, we can prevent them from worrying about whether their lifesaving medication will be available. We must do more to prevent drug shortages.
Over the past decade, pediatric cancer drug shortages have become more and more common. The most recent is a widely used chemotherapy drug called Vincristine. Vincristine is a sterile generic injectable that has been approved to treat children with cancer for over 5 decades. It is utilized by nearly every child with cancer and is a critical component of treatment regimens for children with leukemias, lymphoma, brain tumors, bone tumors, neuroblastoma, Wilms tumor, and rhabdomyosarcoma.
Doctors and families are particularly concerned about the Vincristine shortage because there is no alternative or recommended substitute for the drug. Given the lack of a comparable replacement, doctors will be forced to ration the drug by either skipping or lowering doses. The shortage is already causing pediatric oncologists to alter clinical trial treatment protocols and even delay trial enrollments.
For years, Vincristine was manufactured by both Teva Pharmaceuticals and Pfizer. In July, Teva notified the Food and Drug Administration (FDA) that they planned to discontinue the drug leaving a single manufacturer responsible for the entire U.S. supply. Pfizer has encountered a manufacturing delay but is working to produce additional shipments to try to address the shortfall. Pfizer recently notified the FDA that they hope to rectify the problem by the end of October, but that there may be significant supply problems that could last until the end of the year.
St. Baldrick’s is committed to helping solve the childhood cancer drug shortage crisis. We are already working with our champions on Capitol Hill on longer-term solutions and continue to work with the broader childhood cancer community on advocacy solutions to encourage the government to do more to guarantee safe, uninterrupted and sufficient cancer drug supplies for children in the United States.
For any family that is currently having difficulty obtaining vincristine for your child, the FDA recommends that you contact them at firstname.lastname@example.org.
Read the letter from Dr. Peter Adamson, Chair of the Children’s Oncology Group, to the childhood cancer community on steps to resolve the shortage.
“I’m an accidental tourist in the childhood cancer world,” says Patrick Sullivan, who chairs the Patient Advocacy committee for the St. Baldrick’s Foundation — Stand Up 2 Cancer Pediatric Cancer Dream Team. “It’s not a place I’d ever thought I’d be in, until I heard that my son had rhabdomyosarcoma in 2007.”
Patrick’s role is more than just tourist, though. When the Pediatric Cancer Dream Team was created, its goal was to push the envelope, to marry the emerging fields of genomics and immunotherapy to create targeted therapies for cancers. It was also important to the Dream Team to involve patient advocates, to inspire and work alongside the researchers, to maximize the Dream Team’s success. Along the way, each researcher was paired with a young investigator on the team, to help the advocates better understand the science and to help the young investigators better communicate their work to families and the lay public.
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