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Neurodegeneration Biomarkers in Adult Spinal Muscular Atrophy (SMA) Patients Treated with Nusinersen

Fecha de publicación:

Autores de IIS La Fe

Participantes ajenos a IIS La Fe

  • Andrés-Benito, P
  • Colomina, MJ
  • Marco, C
  • González, L
  • Terrafeta, C
  • Domínguez, R
  • Ferrer, I
  • Povedano, M

Grupos

Abstract

The objective of this study is to evaluate biomarkers for neurodegenerative disorders in adult SMA patients and their potential for monitoring the response to nusinersen. Biomarkers for neurodegenerative disorders were assessed in plasma and CSF samples obtained from a total of 30 healthy older adult controls and 31 patients with adult SMA type 2 and 3. The samples were collected before and during nusinersen treatment at various time points, approximately at 2, 6, 10, and 22 months. Using ELISA technology, the levels of total tau, pNF-H, NF-L, sAPP beta, A beta 40, A beta 42, and YKL-40 were evaluated in CSF samples. Additionally, plasma samples were used to measure NF-L and total tau levels using SIMOA technology. SMA patients showed improvements in clinical outcomes after nusinersen treatment, which were statistically significant only in walkers, in RULM (p = 0.04) and HFMSE (p = 0.05) at 24 months. A reduction in sAPP beta levels was found after nusinersen treatment, but these levels did not correlate with clinical outcomes. Other neurodegeneration biomarkers (NF-L, pNF-H, total tau, YKL-40, A beta 40, and A beta 42) were not found consistently changed with nusinersen treatment. The slow progression rate and mild treatment response of adult SMA types 2 and 3 may not lead to detectable changes in common markers of axonal degradation, inflammation, or neurodegeneration, since it does not involve large pools of damaged neurons as observed in pediatric forms. However, changes in biomarkers associated with the APP processing pathway might be linked to treatment administration. Further studies are warranted to better understand these findings.

Datos de la publicación

ISSN/ISSNe:
1661-6596, 1422-0067

INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES  MDPI

Tipo:
Article
Páginas:
-
PubMed:
38612621
Factor de Impacto:
1,176 SCImago ℠
Cuartil:
Q1 SCImago ℠

Citas Recibidas en Web of Science: 12

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Keywords

  • adult spinal muscular atrophy; nusinersen treatment; biomarkers

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