ElevateBio, a richly funded biotechnology company, has formed a new startup with Boston Children’s Hospital that aims to develop more convenient alternatives to current cancer cell therapies.
The company, which doesn’t yet have a name, is the first to emerge from a partnership designed to advance gene and cell therapy research taking place at Boston Children’s. It’s based around a new method of turning induced pluripotent stem cells into CAR-T cells that are “universal and can be taken off the shelf,” said George Daley, a professor at Boston Children’s and Dana Farber Cancer Institute.
The research was published in the journal Cell Stem Cell on Thursday.
Personalized CAR-T cell therapies, like Gilead’s Yescarta and Bristol Myers Squibb’s Breyanzi, can drive certain blood cancers into remission, and their use is growing as their makers prove them in earlier treatment settings.
However, there remains room for better options. Administration of CAR-T treatment is usually limited to academic centers, as they require specialized resources and care. The therapies are complex and time-consuming to make, involving a multi-week process to collect and genetically modify a patient’s own T cells. In the meantime, patients may need another therapy or become too sick to receive CAR-T. Sometimes, there aren’t enough functional T cells collected from their blood to produce an effective treatment.
“For all those reasons, the opportunity to develop and manufacture a large batch of T cells that could be taken off-the-shelf and used in a wider range of patients just seems to me to have very significant appeal from both a clinical efficiency standpoint and the cost of treatment,” Daley said.
Aiming to address these problems, several biotechs are already trying to develop more convenient alternatives, either by collecting cells from donors rather than patients, or using different types of immune cells entirely.
One approach that’s gaining traction involves induced pluripotent stem cells, or IPSCs. These cells can reproduce indefinitely and be developed into any other cell type. Though researchers have struggled to transform them into the adult T cells capable of fighting cancers, recent progress has led biotech companies like Century Therapeutics and Fate Therapeutics to try.
The new startup from ElevateBio and Boston Children’s will be next in line. Daley says that, after about a decade of research, his lab “learned how to coax [IPSCs] into making more mature T cells that are comparable to the T cells that we would harvest from a patient's circulating blood.”
The research published Thursday describes a method by which scientists suppress an enzyme, dubbed EZH1, that hampers IPSCs’ ability to become mature T cells. Boston Children’s scientists also tried a different approach to growing the cells, leading to T cells with tumor-fighting power comparable to ones derived from currently used methods, the paper said. The cells were better able to clear cancer in mice than cells made with other IPSC approaches, researchers claimed.
The startup based on that research is the result of a five-year partnership between Boston Children’s and ElevateBio, a privately held company that helps others develop gene and cell therapies as well as makes its own. ElevateBio has raised nearly $1 billion from private investors, including $525 million last year, and is working with multiple research institutions to form new companies. Those startups get access to a suite of scientific tools as well as a manufacturing facility in Waltham, Massachusetts, that can help speed the advancement of their work.
“The partnership of our technology platform with some of the existing technologies within Elevate makes it a particularly potent combination,” Daley said.
While the company is still in its infancy, Daley has high hopes for its potential. For now, the new method of making cells could help improve access to CAR-T therapy. In the future, the technology might be used in treatments for immune and viral diseases, too.
“I'm very excited by the prospects that our basic work could ultimately translate into improved care for patients,” Daley said.