Young patient among first to try 'cancer pill'

- Today marks another milestone for 3-year-old Sofia Anderson.  She's the second patient at John's Hopkins All Children's Hospital to take experimental pills that contain DFMO, the same drug used to treat African sleeping sickness.

"The medicine is amazing," her father Patrick offered.  "I keep on telling people it's kind of like science fiction but it's happening now."

We first met Sofia, dad Patrick, mom Catalina and brother Aiden back in April 2015, two months after Sofia was diagnosed with neuroblastoma -- a serious childhood cancer.

Doctors found tumors near Sofia's spine, around her eye, and in her bone marrow.

"I just remember dropping down to the ground and just saying, 'it can't be,'" her mom recalled.

Since then, Sofia's endured 12 rounds of radiation and six of chemotherapy.  One chemo dose was so strong, she spent a month in the hospital and required a stem cell transfusion.

"I remember that being a very difficult time just because she did well with most of the chemo but that one really -- the combination of the drugs they used -- really put her down," Patrick continued.

But 2016 began with a celebration.  Sofia rang the bell, letting everyone know she was cancer-free.

But for almost half the children with neuroblastoma, that celebration is cut short when the cancer comes back.  Dr. Gregory Hale says, when it does, most kids don't survive.

"In Sofia's case, her cancer is actually even more difficult to treat than usual.  And her cancer was actually detectable for more rounds of chemotherapy than we typically see in our patients," he said.

He hopes DFMO will boost her odds.

"Hopefully it's the first day of the rest of her life."

A life that 2-year-old Ezra Matthews never realized.  Ezra died in 2010 after completing his therapy.

"It was absolutely horrible.  It was the day before his second birthday and we he had had stem cell transplant and we were expecting a clean bill of health," Ezra's dad Kyle said.  "It was very quick for Ezra after that. He relapsed the last day in August and he died November."

He and wife Robyn head up the Tampa-based non-profit Because of Ezra.  Their efforts helped bring DFMO to the Bay Area.

"Funding is really important," Matthews continued.  "That's what pays for the trial, that's what pays for use of the drug."

It's a drug that could help Sofia survive.

"We're so excited," Patrick added.  "It's a dream for us to have her take a couple of pills in the morning and a couple of pills at night and for her to remain cancer-free.  It's perfect."

Up Next:


Up Next

  • Young patient among first to try 'cancer pill'
  • Shoe offers stroke recovery revolution
  • Expert: This year's allergy season 'catastrophically bad'
  • Wine extract 'could help fight tooth decay'
  • St. Pete free clinic offers healthcare, hope
  • Glassblowing offers PTSD relief for veterans
  • Treatment for peanut allergy passes first clinical trial
  • STUDY: Drinking alcohol may be better than exercise for living past 90
  • Women get clean to prevent opioid-addicted babies
  • Doctors turn to opioid alternatives to curb addiction