Videogame-based coordinative training can improve advanced, multisystemic early-onset ataxia

Dear Sirs,Treatment options are rare in neurodegenerative childhood-onset ataxias, especially if presenting in advanced diseasestages and with multisystemic disease load. Moreover,wheelchair-bound children and young adults with ataxiaare commonly excluded from current drug treatment trials[1, 2], thus leaving them without prospects of access tonovel treatments. Using a rater-blinded intraindividualcontrol design, we here provide first proof-of-principleevidence that videogame-based coordinative training mightserve as an effective treatment even for advanced, multi-systemic degenerative ataxia.A 10-year-old boy suffering from genetically confirmedAtaxia Telangiectasia (compound heterozygous ATMp.Glu2596Aspfs*4 and p.Asp1625_Ala2626delinsGluPro)since 3 years of age and wheelchair-bound with onlyresidual standing and walking capacities since 7 years ofage (clinical details, Supplement 1) was recruited for asequentially structured 12-week coordinative training pro-gram based on specifically selected, commercially avail-able Nintendo Wii games (for details, see Fig. 1a). Thesubject was examined four times: 5 weeks before theintervention (E1), immediately before the first trainingphase (E2), after the first home training phase (E3) andafter the second home training phase (E4). Delta E1–E2was taken as an intraindividual control period to control forchanges associated with a monthly intravenous applicationof 750 mg methylprednisolone per day for 3 days whichhad been initiated by the patient’s local physicians [8 months before the study. Dosis and interval were keptconstant before and throughout the study. The outcomemeasures were: (1) clinical degree of clinical ataxiaseverity, rated by means of videotaped Scale for theAssessment and Rating of Ataxia (SARA) [3] scores pre-sented in a random order to an ataxia specialist (J.W.)blinded to the number of the specific examination (E1–E4);(2) quantitative movement analysis of balance capacities insitting (methodological details, [4, 5] and Supplement 3);and (3) achievements in patient self-selected individuallyimportant goals with respect to standing and sitting,assessed by the goal attainment score (GAS) [6] (Supple-ment 4). This study has been approved by the appropriateethics committee and has been performed in accordancewith the ethical standards laid down in the 1964 Declara-tion of Helsinki and its later amendments.The SARA score of 26.5/40 points, indicating anadvanced disease stage, remained unchanged before train-ing (E1–E2), thus demonstrating no major effect of corti-costeroids or substantial fluctuations in the natural history