Stories about: everolimus

Finally in the game: Patient in drug trial for PTEN mutation seems to benefit

The first patient to receive everolimus for PTEN hamartoma tumor syndrome
Preston Hall is the first Boston Children’s Hospital patient with PTEN hamartoma tumor syndrome to be treated with everolimus. At left, Siddharth Srivastava, MD. (PHOTO: SEBASTIAN STANKIEWICZ/BOSTON CHILDREN’S HOSPITAL)

From the time of Preston Hall’s birth at 30 weeks, his parents navigated multiple diagnoses, surgeries and sometimes life-threatening medical issues. At 11 months, Preston underwent skull revision surgery for trigonocephaly (a fusion of the skull bones causing a triangular-shaped forehead). After surgery, his doctors discovered serious airway and gastrointestinal issues that led to his failure to thrive. Preston eventually bounced back, but the underlying cause of his complex medical problems remained a mystery. All the while, his fraternal twin Luke overcame more typical preemie issues by age 3.

“At one point Preston had 20 different diagnoses,” his mother, Jennifer Hall, says. “It wasn’t until he was about 4 years old that we started to think his delays were not due to prematurity alone.”

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Taking a targeted approach when leukemia comes back

Lewis Silverman, MD, thinks he may have a powerful new tool for treating children with relapsed acute lymphoblastic leukemia. (VashiDonsk/Wikimedia Commons)

The news that your child has cancer always comes as a shock, but for one cancer, acute lymphoblastic leukemia (ALL), parents can take comfort in the fact that doctors are really good at treating it. The cure rate for ALL has, over the last 40 years, climbed to nearly 90 percent.

Less comforting is the fact that some 10 to 20 percent of children who initially respond well to treatment suffer a relapse within five years. And right now, the drugs at our disposal aren’t very good at turning a relapse back into a remission.

“We have standard treatment regimens for newly diagnosed and relapsed ALL, both of which rely heavily on corticosteroids like prednisone and dexamethasone,” says Lewis Silverman, MD, director of the Pediatric Hematologic Malignancy Service at Dana-Farber/Children’s Hospital Cancer Center (DF/CHCC). “But we know that leukemias with any level of steroid resistance are more likely to relapse. Anything we can do to overcome that resistance would let us help many children.”

Silverman has launched a clinical trial that will try a new strategy for tearing down ALL cells’ barriers against corticosteroids.

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