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      Repurposing G-CSF (Filgrastim) in Long-Time Bone-Marrow Stem Cell Mobilization for Treating Neurodegenerative Disorders like ALS



            Velvio therapeutics aim at CNS reconstitution to renew stem cell populations in bone marrow, muscle and CNS, to reactivate normal cell functions (anti-senescence), to reactivate autophagy and normalize immune communication, to enhance stem cell friendly milieu (extracellular matrix), and to reverse scarring and fibrosis. Velvio has developed selective biomarkers to monitor regained functionalities and to bridge the time gap between therapy and recovery - these include MRI imaging, inflammatory cyto- and chemo/kines, and stem cell markers in peripheral blood. ALS (Amyotrophic Lateral Sclerosis) is a grave disease with still limited treatment options and a median survival of approx. 16 to 24 months after diagnosis. Long-time pulsed G-CSF - BM stem cell mobilization is safe 1 and highly effective 2 in ALS: Clinical pilot data in N=36 ALS patients revealed significant slowing of disease progression and significantly increased survival. Disease modelling and a biomarker panel identified and validated 15 long-time survivors with median OS of 3.8 yrs from start of treatment and a very limited disease progression of -0,12 ALS-FRS-R loss / month with G-CSF therapy in contrast to -0,9 ALS-FRS-R loss / month with best standard care. Among many other modes of action for G-CSF, it was nicely shown 3 that bone marrow cells drive central nervous system regeneration, here after radiation injury. With this impressive therapeutic potential in mind, we want to initiate a prospective pivotal adaptive clinical trial of G-CSF to achieve regulatory approval for ‘early’ ALS patients. The architecture of such a trial and the related expenditure will be discussed in the light of the high unmet medical need and the opportunity of using a ‘repurposed’ therapeutic compound employed over decades in hemato-oncology and intensive care medicine. Velvio believes that healthcare investors and regulatory authorities should not miss the opportunity for such a shortcut to establish a very efficacious new treatment option for desperate ALS patients.


            Author and article information

            RExPO24 Conference
            3 May 2024
            [1 ] Velvio GmbH, Am Biopark 11, 93055 Regensburg, Germany;
            [2 ] Deptm. Neurology, Div. Neuro-Oncology, Massachusetts General Hospital, Harvard Medical School, Boston, MA02114, USA ( https://ror.org/002pd6e78)
            [3 ] Department of Hematology and Oncology, Comp. Brothers Hospital St. Elisabeth, 94315 Straubing, Germany ( https://ror.org/02e560b93)
            [4 ] BGM Associates, Winkler Str 11, 10664 Berlin, Germany;
            [5 ] Dept. Radiology, Section Neuroradiology, TUM Munich, Ismaningerstr.22, 82675 Munich, Germany ( https://ror.org/02kkvpp62)
            [6 ] Berufsgenossenschaftliche Unfallklinik Murnau, Prof.-Küntscher-Str. 8, 82418 Murnau;
            Author notes
            Author information

            This work has been published open access under Creative Commons Attribution License CC BY 4.0 , which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. Conditions, terms of use and publishing policy can be found at www.scienceopen.com .

            Munich, Germany
            3-5 July 2024
            : 3 May 2024


            Funded by: funder-id , BMBF, Berlin;

            The datasets generated during and/or analysed during the current study are available from the corresponding author on reasonable request.
            Neurodegeneration, ALS - Amyotrophic Lateral Sclerosis,GCSF/Filgrastim,Hematopoietic Stem Cell Mobilization,Biomarker Panel,Bioinformatics,Disease Modelling,Individualized Therapy in Neurodegeneration


            1. Grassinger Jochen, Khomenko Andrei, Hart Christina, Baldaranov Dobri, Johannesen Siw W., Mueller Gunnar, Schelker Roland, Schulte-Mattler Wilhelm, Andreesen Reinhard, Bogdahn Ulrich. Safety and feasibility of long term administration of recombinant human granulocyte-colony stimulating factor in patients with amyotrophic lateral sclerosis. Cytokine. Vol. 67(1):21–28. 2014. Elsevier BV. [Cross Ref]

            2. Johannesen Siw, Huie J. Russell, Budeus Bettina, Peters Sebastian, Wirth Anna M., Iberl Sabine, Kammermaier Tina, Kobor Ines, Wirkert Eva, Küspert Sabrina, Tahedl Marlene, Grassinger Jochen, Pukrop Tobias, Schneider Armin, Aigner Ludwig, Schulte-Mattler Wilhelm, Schuierer Gerhard, Koch Winfried, Bruun Tim-Henrik, Ferguson Adam R., Bogdahn Ulrich. Modeling and Bioinformatics Identify Responders to G-CSF in Patients With Amyotrophic Lateral Sclerosis. Frontiers in Neurology. Vol. 12:2021. Frontiers Media SA. [Cross Ref]

            3. Dietrich Jorg, Baryawno Ninib, Nayyar Naema, Valtis Yannis K., Yang Betty, Ly Ina, Besnard Antoine, Severe Nicolas, Gustafsson Karin U., Andronesi Ovidiu C., Batchelor Tracy T., Sahay Amar, Scadden David T.. Bone marrow drives central nervous system regeneration after radiation injury. Journal of Clinical Investigation. Vol. 128(1):281–293. 2017. American Society for Clinical Investigation. [Cross Ref]


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