ELISEO JOJI SEKIYA
Projetos de Pesquisa
Unidades Organizacionais
LIM/31 - Laboratório de Genética e Hematologia Molecular, Hospital das Clínicas, Faculdade de Medicina
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conferenceObject MESENCHYMAL STEM CELLS FROM ADIPOSE TISSUE IN THE TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS - A CASE REPORT(2013) SEKIYA, E. J.; JORDY, S. S.; KUHN, T. I.; FORTE, A.; BRUNIERA, G.; ALVES, A.; BYDLOWSKI, S. P.Amyotrophic Lateral Sclerosis (ALS) is a fatal neurodegenerative disease that selectively affects motor neurons in the brain and spinal cord leading to bulbar and respiratory muscle weakness. Currently there is no treatment proven effective and the disease is considered incurable. Mesenchymal Stem Cells (MSCs) derived from adipose tissue have immunomodulatory properties, inducing suppression and differentiation into neural cell types in vitro. Here we describe a compassionate intervention in a 66 years old male patient with Amyotrophic Lateral Sclerosis. The patient is suffering from Amyotrophic Lateral Sclerosis diagnosed 4 years ago, unsuccessfully treated with Riluzole. It was observed atrophy of the shoulder girdle, upper limbs and thenar and hypothenar regions, fasciculations, tetra-pyramidal release (Hoffmann and Babinsky bilateral and global hyperre flexia) and inexhaustible clonus in the lower limbs. ALSFRS (ALS Functional Rating Scale) and its revised version ALSFRS-R were ALSFRS18 and ALSFRS-R26, respectively. After approval by the Ethics Committee, adipose tissue was collected from abdominal region by liposuction. Adipose tissue-derived MSCs were expanded under GMP conditions and administered intrathecaly. Five days after the first infusion of 1 10 7 cells, patient showed improvement of overall muscle strength and speech, exhaustible clonus in limbs, absence of Babinsky in lower left limb, and ALSFRS25 and ALSFRS-R33. One month later clinical condition worsened, with ALSFRS18 and ALSFRS-R26. The patient received other two higher dose infusions (5 10 7 and 10 10 7 cells) during the next 6 months and clinical data improved (ALSFRS25 and ALSFRS-R33) for a longer period. Moreover, no adverse effects were observed by MSC administration. In conclusion, although the study was performed in a single patient, intra-thecal administration of autologous adipose tissue-derived MSC is potentially safe. It is tempting to speculate that administration of these cells could be beneficial for ALS patients, mainly using higher doses.