Mothers Turn Grief into Action in the Fight against Childhood Cancer (CNN)

Jacob was treated with a 40-year-old chemotherapy protocol that Goodman says doctors knew wouldn’t cure him but that they continued anyway “because there were no other treatments at the time for his kind of brain cancer.” When Jacob died in 2009, he was 10 years old.

Three years later, Miller’s daughter was diagnosed with a brain tumor known as DIPG. Gabriella was 9 at the time of her diagnosis. She died nearly a year later in 2013 at age 10. Today the survival rate for DIPG remains near 0%.

There have been improvements in survival rates for some types of childhood cancers, like leukemia, but there are many others, like pediatric brain cancer, that still have no cure, no effective treatments and dismal survival rates. While medulloblastoma affects both children and adults, DIPG typically is a childhood cancer.

Both women, with no prior legislative experience, have been at the forefront of the childhood cancer movement drafting and lobbying Congress for impactful legislation aimed at increasing funds for research.

Ironically, Goodman, who practiced international trade law before Jacob’s diagnosis, never had her sights set on work on Capitol Hill.

She says she’s always had an aversion for the topic of medicine: “I think I stopped taking science in 10th grade. I just wanted to be exceedingly clear to the universe that I would not be a doctor, because I had zero interest in medicine.” However, after her son’s diagnosis, she said, “I started reading academic science pieces and teaching myself how to read them and teaching myself about cancer biology.”

The problem was that not even scientists or Jacob’s oncologists understood his disease. The fundamental reason for his death was the lack of research and effective therapies, Goodman said.

Miller stopped working when doctors diagnosed Gabriella with an inoperable tumor. Just as with Nancy’s son Jacob, a lack of research into Gabriella’s type of tumor meant she would die of her disease at age 10, Miller said.

When Gabriella was alive, she embraced the role of childhood cancer advocate. In 2013, two weeks before her death, a video of Gabriella went viral. In it she says to lawmakers, “Stop talking and start doing. … Talk is bulls**t! We need action.”

It caught the attention of then-House Majority Leader Eric Cantor. About two weeks after her daughter’s death, Miller found herself on Capitol Hill in a meeting with the Virginia Republican, who wanted to name a bill after Gabriella. The bill would call for diverting money earmarked for political conventions to pediatric cancer research. Miller, still grieving for her daughter, got to work trying to generate support for the bill among senators. Less than six months after Gabriella’s death, the Gabriella Miller Kids First Research Act became law.

“To this day I feel like her hands were on my back pushing me,” Miller said, “saying, ‘We are not done. We have work to do.’ ”

Both of these women are on a crusade to fix the injustice that they say many children with cancer face, a lack of adequate investment in studying cancer specific to them and a lack of development of effective and less toxic drugs and cures.

“Despite the huge public health impact of childhood cancer, the NCI [National Cancer Institute] devotes a small portion of its overall budget to pediatric oncology. In addition, pharmaceutical companies have limited incentives to study new treatments in children with cancer, given the relatively small potential market,” said Dr. Doug Hawkins, a clinician at Seattle Children’s Hospital who’s the chair of the Children’s Oncology Group, a National Cancer Institute-supported clinical trials group.

The day Jacob died, Goodman formed the non-profit Kids v Cancer, and she shifted gears from a crash course in cancer biology and chemotherapies to a crash course on understanding federal law and how to draft federal legislation.

She found a law firm that helped her pro bono, but she also needed to learn how to get around Capitol Hill and to advocate. So she found two lobbyists who took her under their wings.

Since then, Goodman has been credited for the Creating Hope Act, passed in 2012. The bipartisan law created significant incentives for the pharmaceutical industry to develop treatments and cures for children with rare life-threatening conditions, including cancer. Goodman can also be credited for the RACE for Children Act, passed in 2017, which requires companies that are developing cancer-targeted therapies to also develop their drugs for children.

Rep. Michael McCaul, a Texas Republican who has worked alongside Goodman, said, “Nancy Goodman has been and continues to be a trailblazer in the fight against childhood cancer. Her efforts past, present and future have been integral for the passage of many key childhood cancer bills. I cannot thank her enough for her endless work, and look forward to continuing our mission to end pediatric cancer.”

On Friday, McCaul and Democratic Rep. G.K. Butterfield of North Carolina unveiled the Give Kids a Chance Act, Goodman’s latest brainchild, and they’ll introduce it in the House next week. Under the bill, which builds on Goodman’s previous legislation, companies developing drugs for adult cancers would have requirements to test a combination of drugs for pediatric studies.

Miller’s priority is related to the Gabriella Miller Kids First Research Act, which needs a new funding source because it will sunset in 2023. In partnership with Democratic Rep. Jennifer Wexton of Virginia, Miller is advocating for the Gabriella Miller Kids First Research Act 2.0, which would redirect funds generated by big pharma penalties to childhood cancer research. This could add up to hundreds of millions of dollars. The bill already has bipartisan support in both the House and Senate.

“While tens of thousands of kids battle cancer in every community across the country, research for new treatments and cures for these diseases is woefully underfunded. That’s why we need the Gabriella Miller Kids First Research Act 2.0. This Act will deliver a long-overdue boost in resources in the hope that no other family has to know the heartbreak of losing a child to cancer,” Wexton told CNN in a statement.

The measures crafted through the pain of these two women enduring the unnatural phenomenon of losing a child to a terminal disease are seen as valuable assets in the community of pediatric oncology scientists, who want to cure these diseases but don’t have the monetary support to do so.

“The pediatric cancer research community depends upon the hard work of advocates, like Nancy Goodman and Ellyn Miller, to make sure the voice of children with cancer is represented in the federal budget and to increase the incentives for new drug development for pediatric oncology through regulatory requirements,” Hawkins said.

As impactful as their work has been, however, Goodman says it’s not enough. There’s still so much more to be done to transform the reality of pediatric cancer, she said: “Babies are still dying. Of course it’s not enough.”