MSCs have properties that contribute to preventing further destruction of so-called islet cells, which is helpful in case of type 1 diabetes. The effect has been proven and a couple of challenges remain, besides proving the effect in larger patient studies: to cultivate cells in sufficient quantities, and to meet the regulatory requirements that are considerably more stringent now that cell therapies are considered as pharmaceuticals instead of transplants.
“We are practically at the finish line now with regard to transferring production from transplant to pharmaceutical regulations. We will have optimised and validated the production process during spring 2018”, says Ida Duprez.
Duprez has a PhD in cell biology and is the project manager of the clinical development of MSCs for Professor Katarina Le Blanc at Karolinska Institutet. Katarina has used MSCs clinically since 2002, mainly to treat serious complications after blood stem cell transplants. The project on diabetes has been a successful collaboration with professors Olle Korsgren and Per-Ola Carlsson at Uppsala University.
Mass production of MSCs
The breakdown of the islet cells starts long before diabetes symptoms appear.
“The islet cells that remain once the disease has been detected are very valuable and it is important to protect them; clinical studies have shown that MSCs do this. But large quantities of MSCs are required, so we are now working on developing the production of the cells and being able to produce cells for several patients simultaneously”, says Ida Duprez.
Extracting bone marrow from the patient enables the small proportion of MSCs present therein to be cultivated in bioreactors and returned to the patient in the form of an injection.
New classification is a challenge
“Previously, we cultivated the cells in cell culture flasks but the reactors have revolutionised the cell culture process”, says Ida Duprez:
“They look like cassettes and are full of fibres, which provides the cells with an incredibly large surface on which to grow. The previous cell culture method required two to three people per patient, whereas now one person can take care of six reactors, with one patient per reactor.”
Duprez visualises a new, large clinical trial – preferably a collaboration between different centres in the world – within the next five years.
“The challenge is mainly regulatory. Previously, the treatment was considered a transplant, but the EU has decided that it is to be classified as advanced medical therapy, which entails more numerous and stringent requirements in many respects”.
Lack of treatment
There is currently no treatment that can slow the development of recent onset type 1 diabetes. The figures from the Swedish Diabetes Association indicate that around 50 000 people in Sweden have type 1 diabetes, of whom around 7000 are children. The number of children who develop the disease has almost doubled since the 80s.
“Producing MSCs in bioreactors would enable treatment of many more patients than with traditionally cultivated cells. This makes it possible to scale up production and meet the needs of more patients, if the funding is in place.”
High effect and safety
In Sweden there are several companies using MSCs for various indications, including diabetes. However, they are using different starting material, such as umbilical cord blood and amniotic fluid, and are thereby required to draw up safety data for their products that cannot be compared to MSCs from bone marrow.
“Our competitiveness lies in experience, proven safety and a broad expertise. A lot depends on the skill of those handling the cells. We have very expert staff and have worked together for a long time, so knowledge has stayed within the group. Since our MSC product has been used for many years, very good safety and effect data are available for a number of different indications. We possess expertise at all stages, from obtaining the bone marrow through the production process and on to the patients.”
Text: Jörgen Olsson
Updated: 20 February 2018