Type 1 diabetes mellitus

A major milestone in diabetes therapy: the first cellular treatment for type 1 diabetes approved by the FDA

In June 2023, the US Food and Drug Administration (FDA) approved donislecel, the first allogeneic (donor) pancreatic islet cellular therapy made from deceased donor pancreatic cells for the treatment of type 1 diabetes.

• Islet-cell transplantation has the potential to restore glucose levels in patients with type 1 diabetes.​​​[1]American Diabetes Association. Standards of care in diabetes - 2024. Diabetes Care. 2024 Jan;47(1):S1-321. https://diabetesjournals.org/care/issue/47/Supplement_1 ​

• Donor cells are injected into the portal vein and seed in the liver where they produce insulin.​​[134]Vantyghem MC, de Koning EJP, Pattou F, et al. Advances in β-cell replacement therapy for the treatment of type 1 diabetes. Lancet. 2019 Oct 5;394(10205):1274-85. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6951435 http://www.ncbi.nlm.nih.gov/pubmed/31533905?tool=bestpractice.com

• Donislecel is approved for the treatment of adults with type 1 diabetes who are unable to approach target HbA1c because of current repeated episodes of severe hypoglycaemia despite intensive diabetes management and education. There is no evidence to show a benefit of administration of donislecel in patients whose diabetes is well-controlled with insulin therapy.

• Donislecel is not currently approved for use in Europe.

Two pivotal clinical trials showed that 21 out of 30 participants with type 1 diabetes did not need to take insulin for a year or more after donislecel. However, some participants did not achieve any days of insulin independence, and the majority experienced at least one serious adverse reaction related to the infusion procedure or the use of immunosuppressive medications, so treatment benefits need to be carefully weighed against risk.

Despite the potential freedoms that this cellular treatment may bring some type 1 diabetes patients, it remains controversial:

• Questions have been raised over the ethics of commercializing the use of deceased donor islet cells.

• While the FDA has designated this treatment as a biologic drug, some experts believe that it should be reclassified as a form of organ transplant.[135]Witkowski P, Philipson LH, Buse JB, et al. Islets transplantation at a crossroads - need for urgent regulatory update in the United States: perspective presented during the scientific sessions 2021 at the American Diabetes Association Congress. Front Endocrinol (Lausanne). 2022 Jan 6;12:789526. https://www.frontiersin.org/articles/10.3389/fendo.2021.789526/full http://www.ncbi.nlm.nih.gov/pubmed/35069442?tool=bestpractice.com ​​​​

• The requirement for donor cells limits its supply.

• There are also concerns about the need for long-term immunosuppressive therapy, lack of easy availability, and the high cost of this therapy.

See Management: emerging

Original source of update