A recently approved Health Canada drug that has been shown to delay the clinical onset of type 1 diabetes has been provided to two children at BC Children’s Hospital, in what is a first-in-Canada treatment for pediatric patients.
The drug is not yet publicly funded, and the treatment option is currently restricted to patients identified as part of a research-clinical collaboration which aims to advance both the screening and treatment options for pediatric diabetes in the years ahead.
The medication, teplizumab, is a treatment that targets the immune system in patients with early-stage type 1 diabetes, slowing the process that destroys insulin-producing cells in the pancreas. Although not a cure for diabetes, this treatment allows patients and families to prepare for the next phase of the disease, postponing the need for insulin and lowering the risk of severe complications.
“In about 40-60% of pediatric cases, type 1 diabetes can present with a life-threatening condition called diabetic ketoacidosis (DKA). With DKA, the severely high blood sugars and insulin deficiency lead to dehydration, breathing difficulties, a reduced level of consciousness and an increased risk of brain and kidney injury requiring urgent hospitalization,” says Dr. Dina Panagiotopoulos, pediatric endocrinologist at BC Children’s Hospital.
“Delaying the progression of the disease can significantly reduce acute complications like DKA and other long-term complications to the eyes, kidneys, nerves and heart. It also allows time for people to educate themselves on day-to-day management and adapt to the physical and emotional challenges of diabetes.”
About the treatment:
- In recent years, scientists have determined that type 1 diabetes progresses through three stages, and that autoantibodies – which are markers of immune destruction and can be measured in the blood – help predict the progression of the disease.
- Teplizumab works to delay the advancement from stage 2 to stage 3 diabetes by a median of two years, with stage 3 being described as the typical overt presentation of the disease: symptoms such as excessive thirst, frequent urination, fatigue, weight loss.
How the treatment is administered:
- Teplizumab was administered daily via intravenous (IV) infusions at BC Children’s over a two-week period.
- Carefully monitored by a team of physicians and clinical staff, this was the first time in Canada this medication has been able to be administered continually in a child-friendly hospital location, administered daily via intravenous (IV) infusions.
- The two children offered teplizumab were identified through a research screening program led by Dr. Panagiotopoulos, who is also a clinician scientist at BC Children’s Hospital Research Institute.
Dr. Dina Panagiotopoulos, pediatric endocrinologist at BC Children’s Hospital, says: “We screen for autoantibodies in first- and second-degree relatives of people with type 1 diabetes. For those who have positive autoantibodies, we also monitor their blood sugars on a research basis. The testing of all four autoantibodies is not clinically available in BC, so research like this is pivotal to help us catch people at earlier stages and further life-altering treatment.”
Colin Nash, parent of one of the participants receiving the teplizumab treatment, says: “When our son Lucas was diagnosed with stage 1 type 1 diabetes seven years ago, it was a devastating day for our family. His older sister received a type 1 diabetes diagnosis several years earlier, so we fully understood what this meant for him. It can be a helpless feeling a as a parent, knowing there is nothing we can do to help. The Teplizumab medication feels like a ray of hope, and we’re very hopeful that it will delay the onset of stage 3 of type 1 diabetes for several more years for him.”
Dr. Panagiotopoulos is supported by an Investigator Award from the BC Children’s Hospital Research Institute and the BC Children’s Hospital Foundation. The medication teplizumab is being provided on a compassionate basis by the pharmaceutical company Sanofi.
