Array Technologies Inc (ARRY)

[XenaLives]

Apparently Zaida's cancer was extremely rare and unusually complex.

Archer found a unique gene fusion, which happens when one gene breaks and joins with another. This fusion contained the gene AKT1, known among oncologists as the wellspring of tumors. But this particular presentation was unique.

"Nobody's ever seen an AKT1 fusion before," Amsbaugh said. "No one's ever looked for it because it was not reported in scientific literature."

For science, it was a solid breakthrough. For Zaida, it was a miracle.

Her cancer had been notoriously difficult, not only to treat but even to name.

Since she first presented with a tumor at the age of three, various physicians have argued back and forth over whether it was a pediatric presentation of mesothelioma or of ovarian cancer.

Both are rare in people younger than 40, and even more so in pre-pubescent patients.

"The general consensus is mesothelioma," said Mattson. "I generally will call it a more generic term, peritoneal carcinomatosis" — a rare type of cancer that occurs in the thin layer of tissue that covers abdominal organs and surround the abdominal cavity.

After Archer's sequencing, it no longer mattered what to call the disease; the gene had been isolated. Like picking out the single off-tune violinist in the New York Philharmonic Orchestra, now the problem could, hopefully, be corrected.

'Out of hope'

Archer's team reached out to another Boulder company, Array BioPharma.

Array focuses on targeted treatments for cancer. It had a drug, previously developed, that would work at the AKT1 gene: ipatasertib. It was being tested in a handful of clinical trials; none of which were open to children.

By this point, Zaida had deteriorated further.

"You could actually see the (tumors) in the lungs protruding out on her right side," Ellefson said. "She couldn't walk for very long; she had to be pushed in stroller."

Mom Mattson started to put the pressure on teams at Archer and Array, willing them to find a solution, and fast.

"I was trying to rush things along. Surgery wasn't an option. We were running out of drugs to try. We needed this in a hurry."

Array's head of drug development scoured her network, connecting with doctors in Denver and New York. The Food and Drug Administration was brought in, mercifully granting what's called a compassionate use approval for the drug.

Now, Zaida was essentially in a one-person clinical trial for an unproven and unapproved drug. She flew with her mother to Memorial Sloan-Kettering Cancer Center in New York to begin the treatment.

"This was a long shot," said Ellefson. "But we were out of hope."

http://www.dailycamera.com/lafayette-news/ci_30604843/boulder-biotech-breakthrough-eases-lafayette-girls-long-cancer