Bellicum Announces Clinical Study Results Published in BLOOD and Featured in Late-Breaking Session at ASGCT Annual Meeting
Study shows T cells engineered with CaspaCIDe improved immune reconstitution
Safety switch activation rapidly resolved GvHD symptoms
The DOTTI study assessed immune reconstitution, safety and anti-viral response in patients undergoing T cell-depleted, haploidentical hematopoietic stem cell transplant (haplo-HSCT), followed by administration of donor T cells that were engineered with CaspaCIDe.
- All 12 study patients showed more rapid immune reconstitution and reduced infections, compared with reported results in T cell-depleted, haplo-HSCT procedures.
- Four cases of Graft-versus-host disease (GvHD) were reported; all were resolved with no recurrence following administration of rimiducid.
- One GvHD case analogous to cytokine release syndrome (CRS) or systemic inflammatory response syndrome (SIRS) associated with the cell transplant was reported; it was resolved within 2 hours following a single infusion of rimiducid, with no recurrence.
- Engraftment and anti-viral immunity were preserved in patients even after eliminating T cells causing GvHD, showing rimiducid (AP1903) is selective in eliminating alloreactive T cells.
“Removal of T cells is recommended in the haploidentical transplant
setting to avoid GvHD, but their removal can increase the risk of graft
rejection, relapse and viral and other opportunistic infections,” said
Twelve patients undergoing haplo-HSCT to treat blood cancers were treated in dose escalating cohorts with donor T cells engineered with the CaspaCIDe safety switch. The engineered T cells were infused 30 to 90 days following stem cell transplant. Patients were monitored for safety, immune reconstitution and ability of transplanted T cells to fight viral infections. Rimiducid was infused in patients presenting with confirmed Grade I or II GvHD.
Four of the 12 patients developed acute GvHD, and all were successfully treated with rimiducid with no GvHD recurrence. Results also showed that the transplanted engineered T cells provided rapid protection from the viruses EBV, CMV, VZV, HHV6, and BKV. Although administration of rimiducid resulted in a temporary reduction in circulating virus-specific T cells, those cells subsequently recovered and anti-viral activity was maintained.
One patient developed the signs and symptoms of cytokine release syndrome or systemic inflammatory response syndrome associated with the cell transplant. On day 18 following infusion of the engineered T cells, the patient experienced a fever of 105 degrees Fahrenheit, developed associated skin rash and diarrhea, and had high levels of circulating cytokines, including IL-6. Within 2 hours of infusion with rimiducid, the patient’s temperature normalized, skin rash improved and elevated plasma cytokine levels declined without further treatment.
“CaspaCIDe may improve haploidentical stem cell transplant outcomes,
while giving doctors a potential means for rapidly controlling and
eliminating GvHD,” said
Bellicum is a clinical stage biopharmaceutical company focused on discovering and developing novel cellular immunotherapies for various forms of cancer, including hematological cancers and solid tumors, as well as orphan inherited blood disorders. The Company is using its proprietary Chemical Induction of Dimerization, or CID, technology platform to engineer and control components of the immune system in real time. Bellicum is developing next-generation product candidates in some of the most important areas of cellular immunotherapy, including hematopoietic stem cell transplantation, or HSCT, CAR T cell therapy, and dendritic cell vaccines.
*CaspaCIDe® is a trademark registered with the U.S. Patent and Trademark Office.
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