It’s been five years since the first cell therapy was approved by the US Food and Drug Administration, and since then, they have been a critical way to treat aggressive blood cancers.
But the current method — called chimeric antigen receptor T cell therapy, or CAR T cell therapy — comes with challenges.
George Daley, a renowned stem cell researcher at Children’s Hospital Boston, believes his lab has discovered a new method to create cell therapies, which could potentially increase access for patients. The findings, which were published in the peer-reviewed journal Cell Stem Cell on Thursday, are also the catalyst for a new biotech company being launched in partnership with Waltham-based ElevateBio.
Today, CAR T cell therapies are made by harvesting cells from a patient’s blood, engineering them to produce cancer-fighting proteins, then infusing them back into the donor patient.
“Lots of patients benefit, and yet, it’s a highly individualized, patient-specific customized therapy,” Daley said. “These are among the most expensive drugs on the market.”
The hope is that with the new technique, scientists can produce CAR T cells at scale. If approved by regulators, the therapy would be a generic, or off-the-shelf, since it would not be made from individual patients’ cells.
It’s early, but David Hallal, chairman and chief executive of ElevateBio, said the approach could be “quite disruptive to the cancer cell therapy space.” He said it will take a “few years” to move the science into human trials.
The new firm, which does not yet have a name, is funded by ElevateBio. Daley, who is dean of Harvard Medical School, will serve as its scientific founder.
ElevateBio incubates, launches, and operates biotech companies focused on cell and gene therapy. Last year it inked a five-year partnership with Boston Children’s Hospital focused on starting companies based on the medical center’s research, which is how Daley’s lab got involved.
The scientific discovery at the heart of the new company involves a unique way to make cancer-fighting cells from induced pluripotent stem cells, or iPSCs.
The challenge with these types of cells is that they tend to be immature, or “not really good at fighting disease,” Daley said. His lab figured out how to grow mature, functioning T cells from iPSCs that resemble cells circulating in adult blood. They did this by identifying a gene that serves as a major regulator in immune system development.
“We recognized immediately when we made that observation, that this could be the linchpin for generating T cells and potentially cancer-fighting T cells [from iPSCs],” Daley said.
So far, ElevateBio has disclosed the creation of three companies: Cambridge-based AlloVir, HighPassBio, and the new one. Hallal said the firm plans to launch one or two biotech companies every one to two years.
ElevateBio’s main business involves licensing proprietary cell and gene therapy technology to other companies and academic researchers. Hallal declined to say how many companies ElevateBio works with but said it has “far more” external partners than internal companies.
The foundational science from Daley’s lab, which will fuel the new company, will not be available to its other customers, he said.
“When we see something like this … that we think can completely disrupt the space, we will look to potentially build a company around that technology,” he said.
The new company will be based off Route 128 in Waltham at 225 Wyman St., where ElevateBio recently signed a new lease to expand. To meet increasing demand, the company also is looking for more space in the New England area.
Hallal said ElevateBio is “well capitalized” with a multi-year runway, after raising $525 million from investors in March 2021. The company’s investors include Matrix Capital Management, SoftBank Vision Fund 2, and Fidelity Management & Research Co. ElevateBio has about 400 employees. It declined to say how many employees the new company has.