Md Suhail Alam Research Assistant Professor

Pathogenesis and Treatment of Rare Genetic Diseases
Md Suhail Alam

Research Interests:

I utilize interdisciplinary approach consisting of molecular biology, biochemistry, cellular and animal models to understand mechanisms of neurodegenerative disorders and develop novel interventions for their treatment. My research is focused on rare monogenetic disorders that lack treatments. In addition, I use monogenetic disease models as portals to better understand more prevalent diseases such as Alzheimer’s, Parkinson’s disease etc. A major part of my research focuses on re-purpose existing clinically FDA-approved drugs with an aim to develop therapies that could potentially progress quickly from lab to clinic. Towards this, my goal is to utilize components of epigenetic pathways as targets to develop therapies for treating neurodegenerative diseases. Epigenetic mechanisms play important roles in several neurodegenerative disorders. Histone deacetylases (HDAC) and their inhibitors (HDACi) that affect chromatin structure and cell survival pathways have been recognized for their potential to treat multiple neurological disorders. We developed a chronic HDACi-based therapy to treat Niemann-Pick Type C (NPC), a lysosome storage disorder. The technology has been licensed to a company for its further advancements. Current efforts are directed to expand its applicability to additional diseases such as Kabuki Syndrome, Non-Ketotic Hyperglycinemia (NKH) etc. Additional interests include developing suitable cell and animal models of genetic disorders as well as investigating gene-, chaperone- and stem cell-based therapeutic strategies for their treatment.



  • Research Assistant Professor 2017-Present
  • Director of External Programs for Boler-Parseghian Center for Rare and Neglected Diseases 2016-Present
  • Research Scientist, University of Notre Dame 2014-2015
  • Postdoctoral Research Associate, University of Notre Dame 2009-2014
  • Ph.D. (Molecular Biology and Biochemistry) from Institute of Microbial Technology, Chandigarh, India
  • M.S in Biotechnology from Hamdard University, New Delhi, India


Recent Papers:

  • Alam MS, Cooper B, Farris JD, Haldar K (2018). Tolerance of chronic HDACi treatment for neurological, visceral and lung Niemann-Pick Type C disease in mice. Scientific Reports, Mar 1;8(1):3875. doi: 10.1038/s41598-018-22162-7.
  • Collins CJ , Loren B, Alam MS, Mondjinou Y, Skulsky JL, Chaplain CR, Haldar K and Thompson DH (2017). Pluronic based β-Cyclodextrin polyrotaxane formulation for treatment of Niemann-Pick Type C disease. Scientific Reports, Apr 28;7:46737. doi:10.1038/srep46737.
  • Alam MS, Getz M and Haldar K (2016). Chronic administration of an HDAC inhibitor treats both neurological and systemic Niemann-Pick type C disease in a mouse model. Science Translational Medicine, 8(326): 326ra23
  • Alam MS, Getz M, Yi S, Kurkewich J, Safeukui I and Haldar K (2014). Plasma signature of neurological disease in the monogenetic disorder Niemann-Pick Type C. Journal of Biological Chemistry 289: 8051-8066.
  • Alam MS, Getz M, Safeukui I, Yi S, Tamez P, Shin J, Velázquez P and Haldar K (2012). Genomic expression analyses reveal lysosomal, innate immunity proteins, as disease correlates in murine models of a lysosomal storage disorder. Plos One, 7(10): e48273. (doi:10.1371/journal.pone.0048273).
  • Kumar S, Badireddy S, Pal K, Sharma S, Arora C, Garg SK, Alam MS, Agrawal P, Anand GS, Swaminathan K. (2012). Interaction of Mycobacterium tuberculosis RshA and SigH is mediated by salt bridges. Plos One, 7(8): e43676. (doi:10.1371/journal.pone.0043676)
  • Konar M, Alam MS, Arora C and Agrawal P (2012). WhiB2 Rv3260c, a cell division-associated protein of Mycobacterium tuberculosis H37Rv, has properties of a chaperone, FEBS J, 279: 2781-2792
  • Pal K, Kumar S, Sharma S, Garg SK, Alam MS, Xu HE, Agrawal P, Swaminathan K (2010) Crystal structure of full-length Mycobacterium tuberculosis H37Rv glycogen branching enzyme: insights of N-terminal beta-sandwich in substrate specificity and enzymatic activity. Journal of Biological Chemistry, 285: 20897-20903
  • Alam MS, Garg SK, Agrawal P (2009) Studies on structural and functional divergence amongst seven WhiB proteins of Mycobacterium tuberculosis H37Rv. FEBS J, 276: 76-93.
  • Garg SK*, Alam MS, Radha Kishan KV. Agrawal P. (2009). Redox Biology of Mycobacterium tuberculosis H37Rv: protein-protein interaction between GlgB and WhiB1 involves exchange of thiol-disulfide. BMC Biochemistry, 10:1 (Two first authors). This paper was assigned as highly accessed paper.