Improved preservation of insulin production over 12 months in newly diagnosed adult type-1 diabetes patients
NOTE: Find at the end of this article a webcast by Professor Carlsson and the NextCell team presenting the results of this trial and the roadmap forward for ProTrans in type-1 diabetes.
NextCell Pharma announced on the 8th of September that their ProTrans-2 clinical trial for the treatment of type I diabetes mellitus (T1DM), successfully met its primary endpoint. Patients treated with a single dose of ProTrans demonstrated a statistically significant improved preservation of insulin production over 12 months, compared to patients treated with placebo control (p-value < 0.05). To our knowledge this is the first randomized, double-blinded placebo controlled clinical trial evaluating the effect of mesenchymal stromal cell (MSC) therapy in the treatment of T1DM.
“These are really exciting results. Two out of 9 patients treated with ProTrans had increased insulin production after 1 year, and in total 4 of 10 receiving active treatment had a decrease of 5 % or less, as compared to none of the patients receiving placebo”, says Mathias Svahn, CEO NextCell Pharma, AB.
The trial was performed by Principle Investigator Per-Ola Carlsson, Uppsala University and Uppsala University Hospital, and included a total of 15 patients, 10 of which received the ProTrans stromal cell therapy. Patients were diagnosed as Type I diabetics within the last 2 years, but otherwise were healthy, had a C-peptide level of > 0.12nM, were between 18-40 years and 50-100kg body weight. The principle aim of the treatment is to prevent disease progression, rather than its reversal. It is expected that by reducing the severity of disease it will be easier for patients to manage their symptoms, and thus hopefully experience less complications associated with the disease long term.
“Allogeneic cell therapy has again shown an effect in Type-1 diabetes. This time in a randomized controlled trial”, says the principal investigator Professor Per-Ola Carlsson.
To show efficacy in a small trial is of course exciting and the results from the continuing phase III trial are awaited with anticipation. However, patients should not take this as general evidence that ’stem/stromal cell’ treatments work. NextCell’s ProTrans MSC product is derived from allogeneic umbilical cord stromal cells, and with the final drug product produced by selecting and pooling the highest quality MSC donors using NextCell’s proprietary selection algorithm. The cells are processed/manufactured under Good Manufacturing Procedure (GMP) conditions, ensuring the highest level of safety standards for patients, and rigorously tested for both safety and functionality, prior to patient delivery. This is in stark contrast to commercially provided, unproven treatments provided in unregulated clinical settings, many that use liposuction based, fat stromal cell autologous treatments, with no quality, sterility or activity testing prior to delivery to the patient.
Randomized placebo controlled double blinded trials are the most robust and stringent in their conduct. Neither the patient or the patient’s physicians are aware of whether the individual is receiving active treatment or placebo at any point during the trial, thereby ensuring no bias. Of course, for safety purposes, should any adverse reaction to the therapy be detected, this code can be broken in order for the patient to receive appropriate medical care.
While clinical trial registers are full of trials using MSCs for the treatment of a plethora of conditions, in most trials there is no placebo group to show that any benefit is actually coming from the addition of the cell product and/or the treating physician is aware of who has received what treatments. Both aspects make it difficult to not bias data in favour of benefits of the treatment. Often MSC treatments are provided at substantial cost to the patient both within and outside clinical trial settings, adding commercial complications around the rigour of data collection, follow up and dissemination. While not so prevalent in regulated countries like Sweden, this ’stem cell tourism’ is a burden for serious actors like NextCell and the cell therapy brand.