Cell therapy for type-1 diabetes in new-onset children and adolescents

Type 1 diabetes is a disease that affects many people worldwide, both children and adolescents as well as adults. The disease develops as a result of the body's own immune system attacking the cells in the pancreas that produce insulin. Today, the standard treatment is injections of insulin into the subcutaneous fat 4–5 times a day.

To participate in the study, send an email to: diabetesforskningtyp1@akademiska.se

The cell therapy company NextCell Pharma AB has conducted a clinical study that has shown that treatment with a drug of cells is safe and can preserve insulin production in adults with new-onset type 1 diabetes. The study consisted of two parts, in the first part the safety was evaluated. The second part of the study included 15 adult patients with newly diagnosed type 1 diabetes who received either a dose of cell therapy or placebo. The aim was to study whether cell therapy could protect the remaining insulin-producing cells and thus preserve their own insulin production. The study was conducted at Karolinska University Hospital and the principal investigator was Per-Ola Carlsson, senior physician and professor at Uppsala University Hospital.

"The results of cell therapy are very promising and it has now shown efficacy in type 1 diabetes in a randomized, placebo-controlled study in adults. We hope to see the same positive effect on children," says Per-Ola Carlsson, chief physician and professor at Uppsala University Hospital, who is responsible for the current drug studies.

Scientists working in lab

In a follow-up study, NextCell Pharma AB together with Principal Investigator Per-Ola Carlsson evaluates the safety and efficacy of repeated treatment with the cell therapy drug in adults with type 1 diabetes. At the same time, a study is being conducted in which the long-term outcome in adult patients with type 1 diabetes treated with one dose of the cell therapy drug is evaluated with respect to residual endogenous insulin production and metabolic control. These patients will be followed semi-annually to five years after treatment.

The cell therapy drug consists of selected so-called mesenchymal stem cells from the umbilical cord. Mesenchymal stem cells are cells that can form several different types of connective tissue cells, but which have also been shown to have a unique ability to suppress immune attacks. They have therefore previously been tested in a number of clinical studies also on children to, for example, prevent organ rejection after transplantation. NextCell's drug candidate, ProTrans, for the treatment of type 1 diabetes is a mesenchymal stem cell treatment that counteracts the loss of the insulin-producing beta cells. The company has developed a method for selecting optimal cells for the manufacture of ProTrans (more information can be found on www.nextcellpharma.com).

"In type 1 diabetes, the insulin-producing cells are destroyed in an immune attack, which has made stem cell treatment interesting to test on this disease as well," explains Per-Ola Carlsson.

A new study is now underway in Uppsala with newly debuted children and adolescents.

Study design

The clinical trial is divided into three parts:

Part 1: Safety is evaluated in three patients aged 12-18 years and three patients aged 7-11 years. All patients receive the same treatment and the dose is adjusted according to weight. The patients in this part were treated in the spring of 2022 and are now being followed in the study.

According to plan, a safety committee has in the autumn of 2022 reviewed all data from the six patients collected during 3 months after treatment. The Safety Committee did not identify any safety issues and recommended that the study be continued with Part 2.

Part 2: Efficacy and safety are evaluated in 30 patients aged 12–21 years. Patients are randomized (randomized) to cell therapy treatment or placebo (same infusion but without cells). Neither patient, doctor nor nurse will know who gets what.

A safety committee then reviews all the data from the thirty patients collected during 6 months after treatment. If the safety committee recommends that the study should continue, part 3 starts.

Part 3: Efficacy and safety are evaluated in 30 patients aged 7–11 years. Patients are randomized (randomized) to cell therapy treatment or placebo (same infusion but without cells). Neither patient, doctor nor nurse will know who gets what.

The effect of the treatment is evaluated by measuring the patient's own insulin production before and after treatment.


From August 2024, patients aged 7-11 years with type 1 diabetes diagnosed within the last 6 months and with continued self-sufficient insulin production can participate in the study.

It is no longer possible for patients aged 12-21 years to participate as parts 1 and 2 have been completed.

The principal investigator for the study on children and adolescents with newly diagnosed type 1 diabetes is Per-Ola Carlsson. If you are interested in more information about the study and possible participation, the contact address is: diabetesforskningtyp1@akademiska.se

More information about the study is available in Swedish on Uppsala University's website:


and more detailed about the study in English: