A novel diabetes treatment option
This week, we reported that the FDA approved the first ever cellular therapy to treat patients with type 1 diabetes. The go-ahead was granted to a University of Illinois faculty startup called CellTrans, launched out of a scientific research group focused on islet transplantation.
Allogeneic islet transplantation offers a potential alternative to whole-organ pancreas transplantation. Cells are harvested from a deceased donor’s pancreas — typically a pancreas that was rejected for whole-organ transplant — processed and infused into the recipient’s liver portal vein.
The approval for CellTrans' donislecel, now branded Lantidra, was a hard-fought victory. CellTrans first filed its BLA back in 2017, but the FDA responded with multiple concerns. CellTrans withdrew the application and spent three years revising it and working with the FDA in order to resubmit it in 2020.
The therapy got the green light from FDA's Cellular, Tissue and Gene Therapies Advisory Committee back in April 2021. But the 14-4 vote did not come without debate either.
During the AdComm meeting, reviewers had concerns about quality controls when it came to ensuring the potency and purity of each specific lot. Because each lot is derived from a different donor pancreas, the committee discussed the difficulty of consistency. The four AdComm reviewers who voted ‘no’ had cited the possibility of side effects and the design of the trials, including the lack of a control group. The BLA was based on two non-randomized, single-arm studies in which a total of 30 participants with type 1 diabetes and hypoglycemic unawareness.
Following the AdComm nod, consumer advocacy organization Public Citizen sent a letter to the FDA, asking the agency not to approve donislecel, echoing those same concerns.
The BLA itself has also been a source of controversy among industry leaders in the diabetes and cell therapy space. Many take issue with the FDA’s regulation of donor islet cells as a biologic, rather than an organ for transplant, as is the case in other countries such as Europe and Canada. (The FDA made the decision to regulate allogeneic islet cells as a biologic back in 1993.)
While not everyone may agree that Lantidra's approval is a watershed moment for CGT, it is definitely a milestone for islet cell transplantation and diabetes care.
—Karen Langhauser
Parkinson's cell therapy shows promise
As Parkinson's remains challenging to treat, any promising developments in the field deserve careful attention. This week, we reported how BlueRock Therapeutics cell therapy may offer a new glimmer of hope.BlueRock Therapeutics, a subsidiary of Bayer, announced promising results from a phase 1 clinical trial of their cell therapy for Parkinson's disease. The experimental therapy, called bemdaneprocel, involves surgically implanting dopamine-producing neural progenitor cells into the putamen, a brain region responsible for movement control. This treatment aims to address the gradual loss of dopamine, a key neurotransmitter in Parkinson's disease.
The primary objective of the phase 1 trial was to evaluate the safety and tolerance of bemdaneprocel transplantation one year after the procedure. The trial also assessed cell survival, motor effects at one- and two-year intervals, ongoing safety and tolerance at the two-year mark, and the feasibility of the transplantation process. The data from the study indicated that all 12 patients tolerated the therapy well without significant safety concerns and the secondary objectives showed that transplantation was feasible, with evidence of cell survival and engraftment in the brain up to one year.
BlueRock Therapeutics already announced plans for a phase 2 study, expected to begin enrolling patients in the first half of 2024.
Currently, there are no FDA-approved stem cell therapies for Parkinson's disease. Although bemdaneprocel is made with neural progenitor cells and not stem cells, the two are similar. Unlike stem cells, they are already more specialized and can only be directed to differentiate into their intended target cell.
Although still in the earlier stages, bemdaneprocel holds promise.
—Andrea Corona
