Novartis AG this week won approval for a revolutionary new class of drugs in the battle against cancer, shortly after Gilead Sciences Inc. spent $11.9 billion on a biotech company working in the field.
Neither can rest on their laurels. Research in the field is moving so fast that today's breakthroughs can easily become tomorrow's has-beens.
"I can't think of any other therapies in oncology that have seen such rapid development," said Rachel Webster, senior director of oncology at Decision Resources Group, a health care research and consulting firm in London. "In the coming months and years, with the new therapies that are brought to the market, the best will be the ones that win, not necessarily the first."
The novel cell therapies that reprogram the body's immune system to attack tumors are just now coming of age, but their developers are already bracing for challengers. Unlike many traditional drugs, being first isn't enough. Rival products could eliminate the laborious, expensive process needed to make each treatment from scratch. And equipment manufacturers are developing technology to let hospitals and researchers produce the therapies called CAR-T themselves, sidestepping the drug companies' laboratories.
"CAR-T therapy is in open water, vastly uncharted," said Jay Bradner, president of Novartis Institutes for BioMedical Research, which is leading cell-therapy development at the Swiss drugmaker. "We have to make bold bets on where we can make the biggest impact."
Part of the reason why the first CAR-T products will be so vulnerable to newcomers comes from the complex, boutique process behind the treatments. Unlike mass-produced pills, the one-time treatments are tailored for each patient through an intricate, weeks-long process that involves extracting infection-fighting cells from blood; sending them to a centralized manufacturing plant for modification; and shipping them back to be re-infused into the patient at medical centers.
Competition will come from a wide array of innovations that could simplify the process: from ready-to-use vials of donated cells that could be stored in hospitals to equipment the size of a large espresso machine that could create CAR-T therapies on-site. Companies developing technologies that could disrupt the CAR-T model range from biotech startups to giants such as Johnson & Johnson and General Electric Co.'s health care unit.
Among the most promising products on the horizon are off-the-shelf versions of CAR-T, which stands for chimeric antigen receptors T-cell. The off-the-shelf versions use infection-fighting T-cells from healthy donors, rather than the patient's own, meaning they could be made in large batches and be readily available. The current leader, Paris-based Cellectis SA, hopes to have a product in the next two to three years, and companies including Johnson & Johnson are looking into such therapies, known as allogeneic transplants.
Cellectis' product is licensed to New York-based Pfizer Inc. and privately held Servier Laboratories from France. The companies have presented data on their first seven patients, including two patients dubbed the "London babies" who responded to treatment under a compassionate use program.
"You can give these immediately to the patients, without having to wait for manufacturing," said Andre Choulika, chief executive officer and co-founder of Cellectis. "This is the way to go."
Novartis is also experimenting with allogeneic therapies, and Kite Pharma Inc., the California company being acquired by Gilead for $11.9 billion, is watching the space. Gilead Chief Executive Officer John Milligan said Kite has what he called a "pathway" to allogeneic, but pointed to the risks inherent to grafts, including infections carried by donors and rejection.
Also emerging are manufacturing advances that would allow hospitals and researchers to produce CAR-T therapies themselves, bypassing drugmakers. Existing technology requires specialized clinicians and scientists to re-engineer infection-fighting cells, a costly, complex process. That won't be sustainable as the drugs get approved for more types of cancer and they're needed in far greater quantities, said Ger Brophy, general manager of GE Healthcare's cell therapy business.
"We have to make sure hospitals globally have access so the potential of the therapy can be realized," Brophy said. "This is a space that's crying out for operational efficiency."
Information for this article was contributed by Caroline Chen of Bloomberg News.
Business on 09/02/2017