When Connecticut Children’s patient Ian Brackett was 19, he began having severe headaches, intense and persistent enough that his doctor sent him to get a CT scan. The results of that scan were any parent’s nightmare: “They saw three black masses in my skull,” Ian says.
It turned out that those masses were germ-cell tumors in his brain. These tumors derive from stem cells that, in men, are supposed to turn into male reproductive cells but sometimes turn malignant and form tumors in other parts of the body.
“I was young and invincible,” Ian says, “yet, suddenly, I was faced with the reality of brain cancer and my own mortality. I remember the devastated look on my family’s faces, and I realized: I might fall victim to cancer, but they’d have to keep on living.”
Building a New Life
Happily for Ian and his parents, the treatments provided by Connecticut Children’s oncologist Eileen Gillan, MD, proved effective, and Ian’s tumors have disappeared.
“This experience has opened up a whole new doorway for me and helped me create this business that I’m starting up,” he says. “It showed me who I am as a person. It was the worst thing and the best thing that could have happened to me. It showed me how powerful a smile can be. After you realize you can smile at such a devastating time, it’s impossible not to smile and laugh at all things.”
Ian says that realization gave him the encouragement he needed to make a dramatic change. He moved to Hawaii, where today, at age 22, he is building a new life.
In Search of Happy Endings
Ian’s story has the best possible outcome, but for too many young patients, happy endings are not guaranteed. And that is why Ching Lau, MD, PhD, Division Head of Connecticut Children’s Center for Cancer & Blood Disorders and the holder of the Martin J. Gavin Endowed Chair in Hematology/Oncology, is conducting cutting-edge research into new methods of treating cancer.
He’s studying a given cancer’s genetic alterations, which can be used as a therapeutic target and then finding drugs that are effective against that target. Dr. Lau is using this approach to address three kinds of brain tumors, and he is making impressive progress on all three.
Medulloblastoma is the most common form of malignant pediatric brain tumor, and scientists have identified its genetic target. But no one has found a drug that is effective against that target—until now. Dr. Lau sorted through 1,300 existing drugs that were developed for other conditions to see if any of them had the potential to work against those targets.
And indeed one of them did: A class of drugs approved to treat heart conditions appeared to have the right characteristics based on computer analysis, so Dr. Lau tested it on mice that were specially bred to carry actual patient tumor tissue. The drug, called digoxin, shrank the tumors in the mice, clearing it for the next level of testing. Now, because the drugs have already been approved for other uses, Dr. Lau is designing human trials to be conducted at Connecticut Children’s.
“We are on the threshold of a new, effective treatment for the most common kind of malignant brain tumor in children,” Dr. Lau says.
The germ-cell tumors that Ian suffered from are one step removed from medulloblastoma in that scientists have not known the genetic target for it. But that, too, is now changing. Working with colleagues in Japan and Thailand (where this cancer is most prevalent), Dr. Lau discovered one of the genes that appears to predispose children to develop germ-cell tumors in the brain.
Dr. Lau’s discovery is so significant that it was published by Nature, one of the world’s most prestigious scientific journals. In addition, the National Institutes of Health provided a multimillion-dollar grant to fund an expansion of this promising work, looking for other genes that may play a role.
In his third significant project, Dr. Lau is studying a form of intracranial ependymoma, a particularly dangerous form of brain tumor that claims the lives of half the children who have it, especially the younger ones. This cancer appears to be caused by a fusion of two genes, and Dr. Lau used CRISPR gene-editing techniques to prompt the natural development of these tumors in mice. In addition, his team created a three-dimensional model of these human tumor cells growing in the lab. Now, Dr. Lau is using a worldwide network of computers to screen five million chemical structures, looking for one that might be effective against this gene.
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