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]
Donor cells are injected into the portal vein and seed in the liver where they produce insulin.[134]
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]
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.
Summary
Definition
History and exam
Key diagnostic factors
- polyuria
- polydipsia
- diabetic ketoacidosis
Other diagnostic factors
- young age
- weight loss
- blurred vision
- nausea and vomiting
- abdominal pain
- tachypnea
- lethargy
- altered mental status
Risk factors
- genetic predisposition
- geographic region
- infectious agents
- dietary factors
- drug-induced
Diagnostic tests
1st tests to order
- HbA1c
- fasting plasma glucose
- 2-hour post-glucose load plasma glucose
- random plasma glucose
Tests to consider
- plasma or urine ketones
- fasting C-peptide
- autoimmune markers
Treatment algorithm
nonpregnant
pregnant
Contributors
Authors
Rajesh K. Garg, MD
![Rajesh K. Garg](/api/image/25/en-us/normal/25-auth-2_default.jpg)
Professor of Medicine
David Geffen School of Medicine at UCLA
Harbor-UCLA Medical Center
Torrance
CA
Disclosures
RKG is an author of a number of references cited in this topic.
Acknowledgements
Dr Rajesh K. Garg would like to gratefully acknowledge Dr Varsha Vimalananda, previous contributor to this topic.
Disclosures
VV declares that she has no competing interests.
Peer reviewers
Zachary Bloomgarden, MD
Clinical Professor
Medicine/Endocrinology
Diabetes and Bone Disease
Mount Sinai School of Medicine
New York
NY
Disclosures
ZB declares that he has no competing interests.
Alicia Jenkins, MB, BS, MD, FRACP, FRCP
Associate Professor
Department of Medicine
University of Melbourne
Melbourne
Australia
Professor
Harold Hamm Oklahoma Diabetes Center
University of Oklahoma Health Sciences Center
Oklahoma City
OK
Disclosures
AJ has been a (non-salaried) co-investigator on multi-center clinical trials supported by Novo, Eli Lilly, Sanofi-Aventis, and Medtronic. She does not hold any stocks or shares in these companies. She has received a speaker's honorarium from Novo Nordisk.
Differentials
- Monogenic diabetes: maturity onset diabetes of the young
- Monogenic diabetes: neonatal diabetes
- Latent autoimmune diabetes in adults (LADA)
More DifferentialsGuidelines
- Standards of care in diabetes - 2024
- Management of individuals with diabetes at high risk for hypoglycemia
More GuidelinesPatient information
Diabetes type 1: what is it?
Diabetes type 1: what treatments work?
More Patient informationVideos
Subcutaneous injection animation demonstration
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