Biological basis for efficacy of activin receptor ligand traps in myelodysplastic syndromes
Amit Verma,1 Rajasekhar N.V.S. Suragani,2 Srinivas Aluri,1 Nishi Shah,1 Tushar D. Bhagat,1 Mark J. Alexander,2 Rami Komrokji,3 and Ravi Kumar2
1Albert Einstein College of Medicine, Montefiore Medical Center, Bronx, New York, USA.
2Acceleron Pharma, Cambridge, Massachusetts, USA.
3Moffitt Cancer Center, Tampa, Florida, USA.
Address correspondence to: Amit Verma, 1300 Morris Park Avenue, Albert Einstein College of Medicine, Montefiore Medical Center, Bronx, New York 10461, USA. Phone: 718.430.8761; Email: amit.verma@einstein.yu.edu.
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1Albert Einstein College of Medicine, Montefiore Medical Center, Bronx, New York, USA.
2Acceleron Pharma, Cambridge, Massachusetts, USA.
3Moffitt Cancer Center, Tampa, Florida, USA.
Address correspondence to: Amit Verma, 1300 Morris Park Avenue, Albert Einstein College of Medicine, Montefiore Medical Center, Bronx, New York 10461, USA. Phone: 718.430.8761; Email: amit.verma@einstein.yu.edu.
Find articles by Suragani, R. in: JCI | PubMed | Google Scholar
1Albert Einstein College of Medicine, Montefiore Medical Center, Bronx, New York, USA.
2Acceleron Pharma, Cambridge, Massachusetts, USA.
3Moffitt Cancer Center, Tampa, Florida, USA.
Address correspondence to: Amit Verma, 1300 Morris Park Avenue, Albert Einstein College of Medicine, Montefiore Medical Center, Bronx, New York 10461, USA. Phone: 718.430.8761; Email: amit.verma@einstein.yu.edu.
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Aluri, S.
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1Albert Einstein College of Medicine, Montefiore Medical Center, Bronx, New York, USA.
2Acceleron Pharma, Cambridge, Massachusetts, USA.
3Moffitt Cancer Center, Tampa, Florida, USA.
Address correspondence to: Amit Verma, 1300 Morris Park Avenue, Albert Einstein College of Medicine, Montefiore Medical Center, Bronx, New York 10461, USA. Phone: 718.430.8761; Email: amit.verma@einstein.yu.edu.
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1Albert Einstein College of Medicine, Montefiore Medical Center, Bronx, New York, USA.
2Acceleron Pharma, Cambridge, Massachusetts, USA.
3Moffitt Cancer Center, Tampa, Florida, USA.
Address correspondence to: Amit Verma, 1300 Morris Park Avenue, Albert Einstein College of Medicine, Montefiore Medical Center, Bronx, New York 10461, USA. Phone: 718.430.8761; Email: amit.verma@einstein.yu.edu.
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Bhagat, T.
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1Albert Einstein College of Medicine, Montefiore Medical Center, Bronx, New York, USA.
2Acceleron Pharma, Cambridge, Massachusetts, USA.
3Moffitt Cancer Center, Tampa, Florida, USA.
Address correspondence to: Amit Verma, 1300 Morris Park Avenue, Albert Einstein College of Medicine, Montefiore Medical Center, Bronx, New York 10461, USA. Phone: 718.430.8761; Email: amit.verma@einstein.yu.edu.
Find articles by Alexander, M. in: JCI | PubMed | Google Scholar
1Albert Einstein College of Medicine, Montefiore Medical Center, Bronx, New York, USA.
2Acceleron Pharma, Cambridge, Massachusetts, USA.
3Moffitt Cancer Center, Tampa, Florida, USA.
Address correspondence to: Amit Verma, 1300 Morris Park Avenue, Albert Einstein College of Medicine, Montefiore Medical Center, Bronx, New York 10461, USA. Phone: 718.430.8761; Email: amit.verma@einstein.yu.edu.
Find articles by Komrokji, R. in: JCI | PubMed | Google Scholar
1Albert Einstein College of Medicine, Montefiore Medical Center, Bronx, New York, USA.
2Acceleron Pharma, Cambridge, Massachusetts, USA.
3Moffitt Cancer Center, Tampa, Florida, USA.
Address correspondence to: Amit Verma, 1300 Morris Park Avenue, Albert Einstein College of Medicine, Montefiore Medical Center, Bronx, New York 10461, USA. Phone: 718.430.8761; Email: amit.verma@einstein.yu.edu.
Find articles by Kumar, R. in: JCI | PubMed | Google Scholar
First published January 21, 2020 - More info
J Clin Invest. 2020;130(2):582–589. https://doi.org/10.1172/JCI133678.
© 2020 American Society for Clinical Investigation
Signaling by the TGF-β superfamily is important in the regulation of hematopoiesis and is dysregulated in myelodysplastic syndromes (MDSs), contributing to ineffective hematopoiesis and clinical cytopenias. TGF-β, activins, and growth differentiation factors exert inhibitory effects on red cell formation by activating canonical SMAD2/3 pathway signaling. In this Review, we summarize evidence that overactivation of SMAD2/3 signaling pathways in MDSs causes anemia due to impaired erythroid maturation. We also describe the basis for biological activity of activin receptor ligand traps, novel fusion proteins such as luspatercept that are promising as erythroid maturation agents to alleviate anemia and related comorbidities in MDSs and other conditions characterized by impaired erythroid maturation.
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