Eurostars project E!10838, DOPALAM, BioLamina: Production of GMP laminin-111 for establishing clinical grade dopamine cells

Purpose and goal

With this project, we aimed to facilitate the development of cell therapy to treat patients with Parkinson´s disease. We planned to: i) develop MX111, a regulatory compatible substrate ii) produce dopaminergic (DA) cells on the MX111 for clinical use iii) perform preclinical safety tests of the DA cell product iv) make a strategic plan for commercializing the DA cells v) preparation for clinical phase 1/2 study Fulfillment: 80% - i and ii still do not have final parts, which will be done within 6 months of the end of the project; others completely satisfactory.

Expected results and effects

The expected main results of the project were: 1) A scale-up method for laminin-111 (DONE) 2) Commercially available MX111 (STILL ONGOING) 3) Manufacture of DA cells from ES cells with authentic properties (DONE) 4) Preclinical validation of DA cell product in PD disease models (DONE) 5) Plan phase 1/2 clinical trial and investment strategy (DONE) Fulfillment: 80%. We need still to commercialise the developed MX111. See annex with a summary of some results.

Planned approach and implementation

The sub-goals were: i) developing, manufacturing and commercializing MX111 ii) use MX111 to differentiate therapeutic DA cells iii) perform preclinical validation of new stem cell regenerative therapy for PD Sub-goal i) took longer time than anticipated due to technical complications and resource conflicts. Therefore we replaced MX111 with RUO LN111 to progress the project. Looking in the rear view, we made a good risk assessment initially and had good backup plans, but we could also had built in some more flexibility in the time frame in order to avoid side stepping the original plan.