Her progress with a handful of children for whom standard care does
not work reveals the promise of modern cancer drugs, an increasingly
crowded pharmaceuticals field from which investors must try to
select future winners.
The new therapy using engineered white blood cells has shown
anti-tumor activity in the hardest to treat neuroblastoma patients.
"The beauty is that it is very specific in targeting the cancer
cells, while leaving healthy tissue unharmed," Straathof told
Reuters, after presenting her early findings at a science meeting in
Chicago in April. "It's an important step forward."
Autolus - the small British biotech company developing the chimeric
antigen receptor T-cell or CAR-T treatment - is equally excited, and
is planning a potential IPO on Nasdaq.
But Autolus is far from alone in pursuing CAR-T therapy. In fact,
CAR-T treatment - part of the wider field of cancer immunotherapy -
is one of the hottest areas of drug research today, with multiple
firms piling in.
The biotech dollars are flooding in not only in Europe and the
United States but also in China which, with 162 clinical trials, now
boasts more CAR-T studies than the United States, according to a
Reuters analysis of the latest data.
With over 2,000 drugs in the cancer immunotherapy space, the
competitive landscape has never been more crowded as each firm seeks
its own proprietary version of often similar drugs.
Overall, researchers are working on more than 5,200 cancer drugs, up
7.6 percent from a year ago, according to the Pharmaprojects
database. The sheer number is stretching the ability of scientists
to find enough patients to test them on.
Cancer now makes up 34.1 percent of the total drug industry
pipeline, up from 26.8 percent in 2010, as companies divert
resources into a promising sector where new treatments can often
fetch more than $100,000 a year.
'MORE CIRCUMSPECT'
With the first two CAR-T treatments from Novartis <NOVN.S> and
Gilead Sciences <GILD.O> winning U.S. approval last year for rare
blood cancers, the promise of such smart medicine is real and
life-changing - especially if it can be made to work in solid
tumors, as Straathof's work suggests is possible.
However, the wholesale rush by pharmaceutical and biotech companies
into the cancer area poses a dilemma for investors.
A flood of similar products makes it hard for investors to pick
those companies that will achieve commercial success.
"More competition means you should be more circumspect," said Nooman
Haque, head of life sciences at Silicon Valley Bank in London, which
provides financing for start-ups and venture capitalists.
"The traditional investment thesis in biotech is to have a
differentiated medicine with not many competitors, which helps drive
value. Here the problem is that even if there is a big patient
benefit, there are questions as to how long your advantage lasts and
what your commercial edge will be."
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Pharmaceutical executives are not blind to the issue, although each
hopes to find a winning formula in immunotherapy - the
fastest-growing part of the $100 billion-a-year cancer drug market,
with sales expected to top $25 billion by 2021, according to analyst
forecasts compiled by Thomson Reuters.
Roche <ROG.S> CEO Severin Schwan, head of the world's top cancer
company, says he expects "an enormous drop-out", while Sanofi's <SASY.PA>
outgoing research head Elias Zerhouni warned analysts last week that
duplication of effort would shrink the time available for drugmakers
to recoup their R&D investments.
"The cycle of innovation has been shortened significantly," agrees
Aiman Shalabi, chief medical officer at the non-profit Cancer
Research Institute. "There is no doubt we are seeing fast follow-on
and many identical agents hitting the same targets."
The good news for society is that patients will find out much faster
than in the past if new approaches work. But that means doctors can
rapidly switch to alternatives, leading to increased product churn
and uncertainty over future sales.
COMBINATION STUDIES
Twenty years ago, when Roche launched its state-of-the-art cancer
drugs Herceptin and Rituxan, it enjoyed years without rivals. Today,
there are multiple versions of new drugs targeting molecular
pathways with acronyms such as PD-1/L1, PARP and CDK, as well as
CAR-T.
"You're either first or you're best or you're nowhere because it has
become such a race," said Paul Major, an investment manager at BB
Healthcare Trust <BBH.L>, who is cautious about investing in cancer
immunotherapy.
Lydia Haueter at Pictet Asset Management is also wary, pointing out
there are already five PD-1/L1 drugs on the market - from Merck <MRK.N>,
Bristol-Myers Squibb <BMY.N>, Roche, AstraZeneca <AZN.L> and Pfizer
<PFE.N> - and more are coming.
"It seems everybody has a PD-1, so we especially don't go for those
kind of cancer companies," she said.
Some drugmakers like GlaxoSmithKline <GSK.L> and Novartis that
missed the initial PD-1/L1 wave are trying to make a virtue of
looking ahead to the next phase of cancer immunotherapy,
particularly drug combinations.
Yet last month's failure of a combination study using a
next-generation drug from Incyte <INCY.O> with Merck's PD-1 Keytruda
shows that adding a new agent is no slam dunk for expanding the
reach of immune-boosting medicine.
At Great Ormond Street, Straathof is less concerned about doubling
up on research and more focused on getting effective, affordable
cures - and she hopes automated processes will eventually bring down
today's sky-high drug prices.
"I'm not too worried about duplication. It's important to not ask
the same question in two trials but I think there are a lot of
questions to be addressed because there is a lot of nuance in the
system."
Cancer drug research and development - https://tmsnrt.rs/2I1zdkV
(Reporting by Ben Hirschler; Editing by Pravin Char)
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