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Scientists have ideas to spend Trump's money for childhood cancer

any other cancer where more than three-quarters of patients are cured as low-priority, compared to, say, cancers with an approximately 0 percent cure rate, such as adult glioblastoma. But childhood cancers are, well, childhood cancers.

"Today's overall cure rate of 80 percent means than 1 in 5 children will die of their disease," said Dr. James Downing, president and CEO of St. Jude Children's Research Hospital in Memphis, Tenn. "So there is still a lot of work to be done."

The pediatric cancer community applauded President Trump's State of the Union promise of an additional $ 500 million over the next 10 years to fund research into cures for more childhood cancers. It was not just because the doctors generally did not turn extra money, and it was not because of the emotional tug of dying children. Instead, experts say there are specific, actionable research questions that could help answer them, including in two areas that the "cancer moonshot," developed by the Obama administration, identified.

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That's because the overall high cure rate is largely driven by leukemia, but in other pediatric cancers, the cure rates have not changed in 20 years, "said Downing, who served on the moonshot's blue-ribbon panel.


The Trump administration is far from alone in believing that an additional research effort could pay off. On Thursday, the American Cancer Society and the St. Baldrick's Foundation, the largest charitable funder of childhood cancer research, announced that they were joining forces to raise $ 11 million to fund studies. kids with cancer, "the groups said in a statement. The money would support, among other things, expanded clinical trials (just about half a year) and efforts to discover novel treatments.

Of every 100 cancers diagnosed in the U.S., one is in a child. About 11,060 children younger than 15 will be diagnosed with cancer in 2019, the American Cancer Society projects, and 1,190 will die (as will 606,880 adults). The 80 percent cure rate compares to just under 60 percent in the mid-1970s. "Before, there were so few cures that doctors would let children go," Downing said.

But the success rates for different childhood cancers, measured by five-year survival, vary significantly. It is 98 percent for Hodgkin's lymphoma, 95 percent for retinoblastoma (an eye cancer), 91 percent for acute lymphocytic leukemia (the most common pediatric cancer), and 90 percent for non-Hodgkin's lymphoma. It is just 80 percent for the brain tumor neuroblastoma, though, 70 percent for both osteosarcoma and rhabdomyosarcoma, and 66 percent for acute myeloid leukemia.

The National Institutes of Health is projected to spend $ 462 million in 2019 on childhood cancer research, compared to $ 433 million in 2017 and $ 486 million in 2018. (Not all of 2019's grants have been included in the recording system) compared to last year.) An infusion of $ 50 million a year would therefore represent a roughly 10 percent increase, something Downing calls "a welcome supplement."

If nothing else, it would send cancer cancer biologists that their chances of winning National Cancer Institute funding has ticked up. Scientists are sometimes reluctant to jump into pediatric cancer research because the high cure rates, "they're worried that they will not get a high enough priority score to get funded," believing that adult cancers with abysmal survival rates will prevail in the grants competition, said Dr. ir. Lee Helman, a former NCI official and pediatric cancer specialist who is now at Children's Hospital Los Angeles. "A little extra money for research could bring scientists into the field."

Scientists do not have countless ideas of how they would spend it, which is actually an encouraging sign.

One is the remarkable progress that immunotherapies have made in some adults. Unleashing the immune system on cancer, as checkpoint inhibitor drugs such as Keytruda and Opdivo do, works most effectively when tumors are treated with antigens, which are produced by mutated genes. (Checkpoints are off-switches that keep cells from devouring tumor cells, and the drugs block the switches, unleashing the immune system on tumors.) Immune cells recognize these antigens as foreign and hence attack.

But "pediatric cancers have shown less responsiveness to these drugs because they have less of a canceling cancer," Downing said.

Also on the to-do list is identifying childhood-cancer-specific targets for CAR-T therapies, T cells that are genetically engineered to find and destroy particular antigens: The first approved CAR-T, Novartis's Kymriah, is for childhood acute lymphoblastic leukemia, and the hope is to expand CAR-T's into solid cancers.

Pediatric cancers have also suffered the "targeted therapy" revolution in cancer. Though it has not been successful as immunotherapies, this approach has yielded significant, and sometimes lifesaving, drugs such as Gleevec and Herceptin, which cause the cancer-causing effects of a mutation.

A thorough analysis of the genetics of childhood cancers, led by St. Jude Scientists, was published only last year, however, and that is what everyone said about half of the "driver mutations" – ones responsible for fueling a tumor's uncontrolled growth – are not seen in adult cancers. "The genetic landscape of pediatric tumors is just different from adults," said Helman, who oversees the pediatric cancer research funded by Stand Up to Cancer.

As a result, Downing said, "The whole idea is that we can wait for molecularly targeted adult drugs to trickle down [and work in children] is thrown out the window. "

Instead, drugs that target the pediatric-specific driver genes will have to be discovered from scratch. "That's hard, because pediatric cancers are relatively rare," said Dr. Scott Armstrong or Boston Children's Hospital and the Dana-Farber Cancer Institute. "Companies are interested, but they are not the same as a lung cancer.

Academic researchers, whom Trump's additional money would probably fund, therefore "have to do more work [on a potential drug] before pharmaceutical companies will pick it up, "Armstrong said. "You have to de-risk it for them, making it clear that drug X has a high likelihood of success." That, too, obviously takes money.

The one piece of good news from the 2018 study: Nearly half or childhood cancers harbor a "potentially druggable" genetic glitch, the researchers found. That offers hope that molecularly targeted therapies for childhood cancers could be in the cards. But finding them will not be easy. Many of the aberrant proteins in pediatric cancers are different from the other proteins in adult cancers, many of which belong to a family called kinases. (There are 50 approved cancer drugs that target kinases, including Pfizer's Xalkori and Janssen's Imbruvica.) In childhood cancers, Armstrong said, the proteins-causing genes "are of a type that has historically been undruggable," meaning scientists haven the found any molecule capable of defeat them.

Trump said the new money would be proposed fund research, but other uses for the $ 500 million could actually speed the discovery of new therapies even more.

"I do not think another $ 50 million a year for more studies," said John Parker, managing director of Springhood, an early-stage investor focused on children's health. But putting that money into tax incentives to induce pharmaceutical companies to accelerate drug discovery, he said, as might "angel tax credits" that let investors write off any losing bets they make on startups with this focus. Both could draw much-needed investment, Parker said, possibly a multiple of Trump's $ 500 million.

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