Dilanubicel (NLA101) is a universal donor, off-the-shelf, ex vivo expanded hematopoietic stem and progenitor cell product that provides rapid, transient hematopoiesis with long-term benefits. Dilanubicel was intentionally developed to be a short-term therapy that provides temporary bone marrow function that persists 2-3 weeks until a patient’s immune system recovers while inducing long-term immunologic benefits with the potential for improved survival.
Dilanubicel is efficiently manufactured ahead of time, cryopreserved, and available for immediate use. NLA101 is differentiated from other autologous or patient-specific allogeneic cell therapies as it does not require tissue matching. It is currently the subject of two Phase 2 clinical trials to improve treatment outcomes in patients receiving cord blood transplants, and for patients receiving high-dose chemotherapy.
Dilanubicel for Cord Blood Transplants
Widespread adoption of cord blood transplants is currently limited by the low cell dose provided in a cord blood graft. On average, a cord blood graft contains one tenth the number of stem and progenitor cells compared to other stem cell sources, specifically hematopoietic stem cell grafts from bone marrow or peripheral blood donors. As a result, patients receiving a cord blood transplant are at increased risk for significantly-delayed engraftment or graft failure and experience higher early transplant-related morbidity and mortality compared with patients receiving bone marrow or peripheral blood stem cell transplants.
Dilanubicel has the potential to address this limitation by instantly providing significantly-increased numbers of stem and progenitor cells capable of rapid production of blood and immune cells. Clinical results to date demonstrate that dilanubicel may reduce morbidity and mortality associated with cord blood transplants and improve overall patient survival. In June 2018, the European Medicines Agency (EMA) granted dilanubicel PRIority MEdicines (PRIME) designation for the treatment of patients receiving an HSCT. Since its inception in 2016, only 21% of PRIME requests have been granted by the EMA. Nohla was also granted Orphan Drug designation for dilanubicel in HSCT by the European Commission in January 2018.
Dilanubicel following Intensive Chemotherapy
Dilanubicel is also in clinical development for patients receiving intensive chemotherapy. In these patients, life-threatening infections are very common, leading to lengthy hospitalization and increased reliance on supportive care. Despite the use of growth factors and antibiotics, nearly 40% of patients treated with high-dose chemotherapy still experience infections, which can be life threatening. In addition, patients who experience serious infections are often required to delay the start of additional chemotherapy cycles or have their chemotherapy dose reduced to avoid ongoing complications. These delays or reductions in treatment may lessen chemotherapy’s effectiveness and its ability to induce long-term disease response.
Dilanubicel provides these patients with additional treatment options by reducing toxicities associated with intensive chemotherapy, allowing for earlier administration of chemotherapy which could lead to improved patient outcomes.