A pilot trial of RNS60 in amyotrophic lateral sclerosis
Corresponding Author
Sabrina Paganoni MD, PhD
Neurological Clinical Research Institute, Massachusetts General Hospital, 165 Cambridge Street, Suite 600, Boston, Massachusetts, 02114 USA
Spaulding Rehabilitation Hospital, Boston, Massachusetts, USA
Correspondence to: S. Paganoni; e-mail: [email protected]Search for more papers by this authorMohamad J. Alshikho MD
Neurological Clinical Research Institute, Massachusetts General Hospital, 165 Cambridge Street, Suite 600, Boston, Massachusetts, 02114 USA
Athinoula A. Martinos Center for Biomedical Imaging, Department of Radiology, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA
Search for more papers by this authorSarah Luppino RN
Neurological Clinical Research Institute, Massachusetts General Hospital, 165 Cambridge Street, Suite 600, Boston, Massachusetts, 02114 USA
Search for more papers by this authorJames Chan MA
Massachusetts General Hospital Biostatistics Center, Boston, Massachusetts, USA
Search for more papers by this authorLindsay Pothier BA
Neurological Clinical Research Institute, Massachusetts General Hospital, 165 Cambridge Street, Suite 600, Boston, Massachusetts, 02114 USA
Search for more papers by this authorDavid Schoenfeld PhD
Massachusetts General Hospital Biostatistics Center, Boston, Massachusetts, USA
Search for more papers by this authorPatricia L. Andres DPT
Neurological Clinical Research Institute, Massachusetts General Hospital, 165 Cambridge Street, Suite 600, Boston, Massachusetts, 02114 USA
Search for more papers by this authorSuma Babu MD
Neurological Clinical Research Institute, Massachusetts General Hospital, 165 Cambridge Street, Suite 600, Boston, Massachusetts, 02114 USA
Search for more papers by this authorNicole R. Zürcher PhD
Athinoula A. Martinos Center for Biomedical Imaging, Department of Radiology, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA
Search for more papers by this authorMarco L. Loggia PhD
Athinoula A. Martinos Center for Biomedical Imaging, Department of Radiology, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA
Search for more papers by this authorRobert L. Barry PhD
Athinoula A. Martinos Center for Biomedical Imaging, Department of Radiology, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA
Search for more papers by this authorSilvia Luotti MS
IRCCS Mario Negri Institute for Pharmacological Research, Milan, Italy
Search for more papers by this authorGiovanni Nardo PhD
IRCCS Mario Negri Institute for Pharmacological Research, Milan, Italy
Search for more papers by this authorMaria Chiara Trolese MS
IRCCS Mario Negri Institute for Pharmacological Research, Milan, Italy
Search for more papers by this authorSerena Pantalone MS
IRCCS Mario Negri Institute for Pharmacological Research, Milan, Italy
Search for more papers by this authorCaterina Bendotti PhD
IRCCS Mario Negri Institute for Pharmacological Research, Milan, Italy
Search for more papers by this authorValentina Bonetto PhD
IRCCS Mario Negri Institute for Pharmacological Research, Milan, Italy
Search for more papers by this authorFabiola De Marchi MD
Neurological Clinical Research Institute, Massachusetts General Hospital, 165 Cambridge Street, Suite 600, Boston, Massachusetts, 02114 USA
Search for more papers by this authorBruce Rosen MD, PhD
Athinoula A. Martinos Center for Biomedical Imaging, Department of Radiology, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA
Search for more papers by this authorJacob Hooker PhD
Athinoula A. Martinos Center for Biomedical Imaging, Department of Radiology, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA
Search for more papers by this authorMerit Cudkowicz MD
Neurological Clinical Research Institute, Massachusetts General Hospital, 165 Cambridge Street, Suite 600, Boston, Massachusetts, 02114 USA
Search for more papers by this authorNazem Atassi MD
Neurological Clinical Research Institute, Massachusetts General Hospital, 165 Cambridge Street, Suite 600, Boston, Massachusetts, 02114 USA
Search for more papers by this authorCorresponding Author
Sabrina Paganoni MD, PhD
Neurological Clinical Research Institute, Massachusetts General Hospital, 165 Cambridge Street, Suite 600, Boston, Massachusetts, 02114 USA
Spaulding Rehabilitation Hospital, Boston, Massachusetts, USA
Correspondence to: S. Paganoni; e-mail: [email protected]Search for more papers by this authorMohamad J. Alshikho MD
Neurological Clinical Research Institute, Massachusetts General Hospital, 165 Cambridge Street, Suite 600, Boston, Massachusetts, 02114 USA
Athinoula A. Martinos Center for Biomedical Imaging, Department of Radiology, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA
Search for more papers by this authorSarah Luppino RN
Neurological Clinical Research Institute, Massachusetts General Hospital, 165 Cambridge Street, Suite 600, Boston, Massachusetts, 02114 USA
Search for more papers by this authorJames Chan MA
Massachusetts General Hospital Biostatistics Center, Boston, Massachusetts, USA
Search for more papers by this authorLindsay Pothier BA
Neurological Clinical Research Institute, Massachusetts General Hospital, 165 Cambridge Street, Suite 600, Boston, Massachusetts, 02114 USA
Search for more papers by this authorDavid Schoenfeld PhD
Massachusetts General Hospital Biostatistics Center, Boston, Massachusetts, USA
Search for more papers by this authorPatricia L. Andres DPT
Neurological Clinical Research Institute, Massachusetts General Hospital, 165 Cambridge Street, Suite 600, Boston, Massachusetts, 02114 USA
Search for more papers by this authorSuma Babu MD
Neurological Clinical Research Institute, Massachusetts General Hospital, 165 Cambridge Street, Suite 600, Boston, Massachusetts, 02114 USA
Search for more papers by this authorNicole R. Zürcher PhD
Athinoula A. Martinos Center for Biomedical Imaging, Department of Radiology, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA
Search for more papers by this authorMarco L. Loggia PhD
Athinoula A. Martinos Center for Biomedical Imaging, Department of Radiology, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA
Search for more papers by this authorRobert L. Barry PhD
Athinoula A. Martinos Center for Biomedical Imaging, Department of Radiology, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA
Search for more papers by this authorSilvia Luotti MS
IRCCS Mario Negri Institute for Pharmacological Research, Milan, Italy
Search for more papers by this authorGiovanni Nardo PhD
IRCCS Mario Negri Institute for Pharmacological Research, Milan, Italy
Search for more papers by this authorMaria Chiara Trolese MS
IRCCS Mario Negri Institute for Pharmacological Research, Milan, Italy
Search for more papers by this authorSerena Pantalone MS
IRCCS Mario Negri Institute for Pharmacological Research, Milan, Italy
Search for more papers by this authorCaterina Bendotti PhD
IRCCS Mario Negri Institute for Pharmacological Research, Milan, Italy
Search for more papers by this authorValentina Bonetto PhD
IRCCS Mario Negri Institute for Pharmacological Research, Milan, Italy
Search for more papers by this authorFabiola De Marchi MD
Neurological Clinical Research Institute, Massachusetts General Hospital, 165 Cambridge Street, Suite 600, Boston, Massachusetts, 02114 USA
Search for more papers by this authorBruce Rosen MD, PhD
Athinoula A. Martinos Center for Biomedical Imaging, Department of Radiology, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA
Search for more papers by this authorJacob Hooker PhD
Athinoula A. Martinos Center for Biomedical Imaging, Department of Radiology, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA
Search for more papers by this authorMerit Cudkowicz MD
Neurological Clinical Research Institute, Massachusetts General Hospital, 165 Cambridge Street, Suite 600, Boston, Massachusetts, 02114 USA
Search for more papers by this authorNazem Atassi MD
Neurological Clinical Research Institute, Massachusetts General Hospital, 165 Cambridge Street, Suite 600, Boston, Massachusetts, 02114 USA
Search for more papers by this authorABSTRACT
Introduction: RNS60 is a novel immune-modulatory agent that has shown neuroprotective effects in amytrophic lateral sclerosis (ALS) preclinical models. RNS60 is administered by weekly intravenous infusion and daily nebulization. The objective of this pilot open-label trial was to test the feasibility, safety, and tolerability of long-term RNS60 administration in ALS patients. Methods: The planned treatment duration was 23 weeks and the primary outcomes were safety and tolerability. Secondary outcomes included PBR28 positron emission tomography (PET) imaging and plasma biomarkers of inflammation. Results: Sixteen participants with ALS received RNS60 and 13 (81%) completed 23 weeks of RNS60 treatment. There were no serious adverse events and no participants withdrew from the trial due to drug-related adverse events. There were no significant changes in the biomarkers. Discussion: Long-term RNS60 administration was safe and well-tolerated. A large, multicenter, phase II trial of RNS60 is currently enrolling participants to test the effects of RNS60 on ALS biomarkers and disease progression. Muscle Nerve 59:303–308, 2019
Supporting Information
| Filename | Description |
|---|---|
| mus26385-sup-0001-Figs1.tifTIFF image, 657 KB | Supplementary figure 1. Study Schema |
| mus26385-sup-0002-Figs2.tifTIFF image, 1.3 MB | Supplementary figure 2. ALSFRS-R (panel A and B) and SVC (panel C and D). |
| mus26385-sup-0003-TableS1.docxWord 2007 document , 14.7 KB | Supplementary Table 1. Adverse Events (AEs). |
| mus26385-sup-0004-supinfo.docxWord 2007 document , 13.2 KB | Supproting Informations |
Please note: The publisher is not responsible for the content or functionality of any supporting information supplied by the authors. Any queries (other than missing content) should be directed to the corresponding author for the article.
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