Novartis readies for FDA review of 1 billion dollar CAR-T child cancer hope

ZURICH: Two decades ago in a Memphis, Tenn., hospital lab, cancer scientist Dario Campana and his team were hunting for a new way to fight deadly acute lymphoblastic leukemia (ALL).

They began with several molecules, but finally settled on one, called 4-1BB, because it seemed to prime the human immune system to attack blood cancer more aggressively than others.

"Our first tests convinced us these T cells were special," Campana, now a scientist at the National University Cancer Institute in Singapore, told Reuters by email this week.

"I had never seen any drug killing leukemic cells so rapidly and specifically."

Come Wednesday, it will be showtime for his molecule, when the U.S. Food and Drug Administration publicly reviews Novartis' investigational drug, CTL019, for safety and effectiveness against ALL in children.

Campana's 4-1BB is the key ingredient of the Swiss drugmaker's therapy, which involves T cells being extracted from patients, genetically re-engineered, and then unleashed to kill leukemia cells growing out of control.

Of 68 desperately ill children who started Novartis's pediatric ALL trial 17 months ago when other treatments had failed, 57 are alive. Novartis' chief drug developer Vas Narasimhan thinks Campana's molecule is a big reason why.

"This might be part of the reason we see the profile that we do in terms of long-lasting remissions with CTL019," he said.

The chimeric antigen receptor therapy developed by Novartis, called CAR-T, is poised to become the first of its kind to secure FDA approval, ahead of Kite Pharma and Juno Therapeutics.

The Swiss drugmaker eventually expects more than $1 billion in annual sales, including from the treatment of other forms of cancer in adults.

Paternity Dispute

The discovery of 4-1BB by Campana, an Italian, and his Japanese colleague Chihaya Imai, during their tenure at St. Jude's Children's Research Hospital in Memphis, had gone relatively unheralded.

Instead, the University of Pennsylvania, including its cancer immunotherapy pioneer Carl June that borrowed the molecule from Campana in the early 2000s for his own work, has won most of the accolades.

Around the time Novartis struck a multi-million deal with Penn to commercialize CTL019 in 2012, St. Jude's sued the university, alleging patent violations.

Moreover, Campana's and Imai's names were left off the Penn team's publications.

But the patent dispute was finally settled in 2015, when Novartis agreed to pay $12.25 million, plus royalties.

The scientific record has been set straight too. Last year, June's team updated three New England Journal of Medicine publications that had previously ignored Campana's contribution.

Despite the initial snub, Campana is rooting for Novartis' drug.

"Many scientists have contributed in different ways to the development of this technology," said Campana, a founder of Boston-based cancer immunotherapy company Unum Therapeutics.

"It is wonderful that it works."