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 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.
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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."
(Editing by Alexander Smith)
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