Gamida, Editas Partner to Create Genetically Engineered NAM-NK Cells for Treating Blood, Solid Cancers

Gamida Cell and Editas Medicine have entered a partnership to explore the potential of using gene-editing technology to enhance the activity of certain immune cells against blood and solid cancers.

Under the terms of the agreement, the companies will test Editas’ CRISPR technology as a means to engineer Gamida’s NAM-NK cells and direct them toward specific cancer molecules.

“Natural killer cells are increasingly recognized as a potential breakthrough approach to treating various cancers,” Charles Albright, PhD, chief scientific officer of Editas Medicine, said in a press release. “This agreement with Gamida Cell enables us to combine our industry-leading genome editing platform with Gamida Cell’s proprietary NAM-NK cells in an effort to develop best-in-class cellular medicines.”

Natural killer cells are key players in the fight against tumors. But their insufficient numbers and short lifespan have limited their applicability as a treatment.

Using its NAM technology, Gamida increases the number of natural killer cells by exposing them to nicotinamide. The tweaked cells live longer, multiply faster, generate higher levels of inflammatory proteins, and are particularly good at recruiting other immune cells into tumors that boost their activity, according to the company’s website.

Because natural killer cells do not require genetic matching, these cells may come from healthy donors, allowing for a potential universal donor-based therapy. So, it follows that researchers would explore ways to improve the fighting ability of natural killer cells, like engineering the cells to recognize specific cancer targets.

NAM-NK cells are being studied in an ongoing Phase 1 clinical trial (NCT03019666) as a treatment approach for non-Hodgkin’s lymphoma and multiple myeloma patients who failed prior treatments.

The trial, which is still recruiting participants, is being conducted at the Masonic Cancer Center in Minnesota. To date, 14 patients have been screened for safety and 12 have been assessed for safety.

Participants in the trial first received a high-dose of chemotherapy before being given the NAM-NK cells. This chemotherapy regimen is expected to create room for the newly injected cells, while clearing residual cancer cells.

Also, lymphoma and myeloma patients received the antibodies Rituxan (rituximab) and Empliciti (elotuzumab), respectively, prior to and after the NAM-NK infusion to facilitate tumor targeting.

Among the six lymphoma patients, three achieved a complete cancer response, and one experienced a reduction in tumor burden (partial response). For those with myeloma, one patient achieved a complete response, two experienced stable disease, and three had progressive disease.

In general, NAM-NK cells were found to be well-tolerated, with no signs of graft versus host disease (GvHD) or neurotoxicity syndrome reported. One patient experienced cytokine release syndrome (a life-threatening overactivation of the immune response), and one death was reported due to sepsis.

These recent Phase 1 results were presented at the 2019 Transplantation & Cellular Therapy Meetings of the American Society for Blood and Marrow Transplantation and the Center for International Blood and Marrow Transplant Research in Houston, Texas.

The presentation was titled, “First-in-Human Phase I Study of Nicotinamide-Expanded Related Donor Natural Killer Cells for the Treatment of Relapsed/Refractory Non-Hodgkin Lymphoma and Multiple Myeloma.”

“We are encouraged by the early data generated in the Phase 1 study of NAM-NK as an investigational therapy for patients with non-Hodgkin lymphoma and multiple myeloma, and we are pleased to have the opportunity to accelerate and broaden our NAM-NK research efforts through this agreement,” said Julian Adams, PhD, CEO of Gamida Cell.

“By leveraging the collective expertise of the Gamida Cell and Editas Medicine teams, we hope to enhance the efficacy of NAM-NK cells through CRISPR editing and potentially bring life-changing immunotherapy treatments to patients,” he added.