scholarly journals Safety, Tolerability, and Pharmacodynamics of Intrathecal Injection of Recombinant Human HGF (KP‐100) in Subjects With Amyotrophic Lateral Sclerosis: A Phase I Trial

2018 ◽  
Vol 59 (5) ◽  
pp. 677-687 ◽  
Author(s):  
Hitoshi Warita ◽  
Masaaki Kato ◽  
Ryuta Asada ◽  
Atsuko Yamashita ◽  
Daichika Hayata ◽  
...  
Keyword(s):  
Amyotrophic Lateral Sclerosis ◽  
Phase I ◽  
Phase I Trial ◽  
Intrathecal Injection ◽  
Lateral Sclerosis

scholarly journals Lumbar Intraspinal Injection of Neural Stem Cells in Patients with Amyotrophic Lateral Sclerosis: Results of a Phase I Trial in 12 Patients

Stem Cells ◽  
2012 ◽  
Vol 30 (6) ◽  
pp. 1144-1151 ◽  
Author(s):  
Jonathan D. Glass ◽  
Nicholas M. Boulis ◽  
Karl Johe ◽  
Seward B. Rutkove ◽  
Thais Federici ◽  
...  
Keyword(s):  
Stem Cells ◽  
Amyotrophic Lateral Sclerosis ◽  
Neural Stem Cells ◽  
Phase I ◽  
Phase I Trial ◽  
Lateral Sclerosis ◽  
Intraspinal Injection

Intrathecal Ciliary Neurotrophic Factor Delivery for Treatment of Amyotrophic Lateral Sclerosis (Phase I Trial)

Neurosurgery ◽  
1997 ◽  
Vol 40 (1) ◽  
pp. 94-100 ◽  
Author(s):  
Richard D. Penn ◽  
Jeffrey S. Kroin ◽  
Michelle M. York ◽  
Jesse M. Cedarbaum
Keyword(s):  
Amyotrophic Lateral Sclerosis ◽  
Phase I ◽  
Neurotrophic Factor ◽  
Phase I Trial ◽  
Ciliary Neurotrophic Factor ◽  
Lateral Sclerosis

scholarly journals Phase I Trial of Repeated Intrathecal Autologous Bone Marrow-Derived Mesenchymal Stromal Cells in Amyotrophic Lateral Sclerosis

2015 ◽  
Vol 4 (6) ◽  
pp. 590-597 ◽  
Author(s):  
Ki-Wook Oh ◽  
Chanil Moon ◽  
Hyun Young Kim ◽  
Sung-il Oh ◽  
Jinseok Park ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Amyotrophic Lateral Sclerosis ◽  
Phase I ◽  
Mesenchymal Stromal Cells ◽  
Stromal Cells ◽  
Phase I Trial ◽  
Autologous Bone Marrow ◽  
Autologous Bone ◽  
Lateral Sclerosis

Intrathecal Ciliary Neurotrophic Factor Delivery for Treatment of Amyotrophic Lateral Sclerosis (Phase I Trial)

Neurosurgery ◽  
1997 ◽  
Vol 40 (1) ◽  
pp. 94-100 ◽  
Author(s):  
Richard D. Penn ◽  
Jeffrey S. Kroin ◽  
Michelle M. York ◽  
Jesse M. Cedarbaum
Keyword(s):  
Amyotrophic Lateral Sclerosis ◽  
Phase I ◽  
Neurotrophic Factor ◽  
Phase I Trial ◽  
Ciliary Neurotrophic Factor ◽  
Lateral Sclerosis

Therapeutic benefit of intrathecal injection of insulin-like growth factor-1 in a mouse model of Amyotrophic Lateral Sclerosis

2005 ◽  
Vol 235 (1-2) ◽  
pp. 61-68 ◽  
Author(s):  
Isao Nagano ◽  
Hristelina Ilieva ◽  
Mito Shiote ◽  
Tetsuro Murakami ◽  
Masataka Yokoyama ◽  
...  
Keyword(s):  
Amyotrophic Lateral Sclerosis ◽  
Growth Factor ◽  
Mouse Model ◽  
Intrathecal Injection ◽  
Therapeutic Benefit ◽  
Insulin Like Growth Factor ◽  
Lateral Sclerosis

scholarly journals Sequencing of neurofilament genes identified NEFH Ser787Arg as a novel risk variant of sporadic amyotrophic lateral sclerosis in Chinese subjects

2021 ◽  
Vol 14 (1) ◽  
Author(s):  
Feng Lin ◽  
Wanhui Lin ◽  
Chaofeng Zhu ◽  
Jilan Lin ◽  
Junge Zhu ◽  
...  
Keyword(s):  
Amyotrophic Lateral Sclerosis ◽  
Phase I ◽  
Phase Ii ◽  
Multiple Testing ◽  
Statistical Power ◽  
Neuronal Cell ◽  
Sporadic Amyotrophic Lateral Sclerosis ◽  
Risk Variant ◽  
Chinese Subjects ◽  
Lateral Sclerosis

Abstract Background Amyotrophic lateral sclerosis (ALS) is a devastating neurodegenerative disease with neuronal cell inclusions composed of neurofilaments and other abnormal aggregative proteins as pathological hallmarks. Approximately 90% of patients have sporadic cases (sALS), and at least 4 genes, i.e. C9orf72, SOD1, FUS and TARDBP, have been identified as the main causative genes, while many others have been proposed as potential risk genes. However, these mutations could explain only ~ 10% of sALS cases. The neurofilament polypeptides encoded by NEFH, NEFM, and NEFL are promising protein biomarkers for ALS and other degenerative diseases. However, whether the genetic variants of these genes were associated with ALS remain ambiguous. Methods Here, we used PCR-Sanger to sequence the exons of these three genes in a cohort of 371 sALS patients and 711 healthy controls (Phase I) and validated the risk variant in another 300 sALS patients and 1076 controls (Phase II). Results A total of 92 variants were identified, including 36 rare heterozygous variants in NEFH, 27 in NEFM, and 16 in NEFL, and only rs568759161 (p.Ser787Arg) in NEFH reached nominal statistical power (P = 0.02 at Phase I, P = 0.009 at Phase II) in the case–control comparison. Together, the Phase I and II studies showed the significantly higher frequency of the variant in cases (9/1342, 0.67%) than in controls (2/3574, 0.07%) (OR 12.06; 95% CI 2.60–55.88; P = 0.0003). No variants passed multiple testing in the discovery cohort, but rs568759161 was associated with ALS in a replication cohort. Conclusions Our results confirmed that NEFH Ser787Arg is a novel sALS risk variant in Chinese subjects, but NEFM and NEFL were not associated with sALS. These data may have implications for genetic counselling and for understanding the pathogenesis of sALS.

Abstract #5: A Phase I, Dosage-Escalation Study of Creatine Monohydrate in Subjects with Amyotrophic Lateral Sclerosis

2010 ◽  
Vol 7 (3) ◽  
pp. 329-329 ◽  
Author(s):  
Nazem Atassi ◽  
Eva Maria Ratai ◽  
David Greenblatt ◽  
Merit Cudkowicz ◽  
Allitia DiBernardo
Keyword(s):  
Amyotrophic Lateral Sclerosis ◽  
Phase I ◽  
Creatine Monohydrate ◽  
Dosage Escalation ◽  
Lateral Sclerosis

Mesenchymal stem cell transplantation in amyotrophic lateral sclerosis: A Phase I clinical trial

2010 ◽  
Vol 223 (1) ◽  
pp. 229-237 ◽  
Author(s):  
L. Mazzini ◽  
I. Ferrero ◽  
V. Luparello ◽  
D. Rustichelli ◽  
M. Gunetti ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Amyotrophic Lateral Sclerosis ◽  
Stem Cell ◽  
Mesenchymal Stem Cell ◽  
Stem Cell Transplantation ◽  
Phase I ◽  
Cell Transplantation ◽  
Phase I Clinical Trial ◽  
Mesenchymal Stem Cell Transplantation ◽  
Lateral Sclerosis
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