Draft:Mohammad Arif Hossain

Dr. Mohammad Arif Hossain, also known as Dr. Hossain, is a research scientist renowned for his contributions to the field of lysosomal storage disorders (LSDs). His research has led to advancements in diagnostic techniques and therapeutic approaches, including the development of chemical chaperone therapies and novel biomarker-based methods.

Hossain's research investigates lysosomal storage diseases (LSDs) and other Inborn errors of metabolism (IEMs), with a focus on improving diagnosis and treatment. His work has demonstrably contributed to understanding Krabbe disease, galactosialidosis, Fabry disease, and others. He has utilized patient-specific motor neurons derived from induced pluripotent stem cells (iPSCs) to study metachromatic leukodystrophy (MLD) and analyzed caregiver perspectives on treatment for mucopolysaccharidosis II (MPS II). Hossain has developed cellular models using patient-specific iPSCs to study Niemann-Pick disease type C (NPC) and established a diagnostic approach for X-linked adrenoleukodystrophy (X-ALD) in Japan. His research has yielded valuable insights into various LSDs and IEMs, including the therapeutic potential of genistein in mucolipidosis II and the characterization of enzymes for neuronal ceroid lipofuscinosis (NCL) types 1 and 2.

Hossain has authored more than 26 peer-reviewed articles. in scientific journals. His efforts in uncovering the mechanisms and treatments for LSDs have earned him global recognition. He has been honored with the JSIMD Young Investigator Award in 2019, the JHG Young Scientist Award in 2017, and the Kunihiko Suzuki Award in 2017.In 2020, he was awarded the World Symposium Young Investigator Award

Since 2021 Hossain working as a medical advisor at JCR Pharmaceuticals Ltd. (JCRファーマ) to focus on developing enzyme replacement therapy (ERT) for various LSDs.

Personal life
Hossain, was raised in a large Bangladeshi family with a strong emphasis on education, excelled academically. He pursued medicine, encountering diagnostic limitations in pediatrics that fueled his research drive. After a Ph.D. in Japan, his research focused on LSDs, leading to a novel therapeutic approach for Krabbe disease and Galactosialidosis.

Beyond science, Hossain's life reflects deep faith and a commitment to public health. He previously led an anti-smoking organization and actively promotes child health through media and online platforms. Currently residing in Japan with his family, he balances research with his role as a devoted husband and father.

Education
• FRCP, Royal College of Physicians and Surgeons, Glasgow, UK September 2021

• FRCPCH, Royal College of Pediatrics and Child Health, London, UK November 2020

• FRCP, Royal College of Physicians, Edinburgh, UK September 2020

• Clinical Fellowship, Osaka University Hospital, Japan March 2014

• Ph.D . (Medical Science), Osaka University Graduate School of Medicine, Japan March 2014

• MBBS, Rajshahi University, Rajshahi Medical College, Bangladesh April 2004

Professional career
Hossain's medical career began with roles as a Medical Officer at the Islami Bank Medical College, Rajshahi (IBMCR), and a DCH (Diploma in Child Health) Physician at Rajshahi Medical College Hospital (RMCH). In December 2008, he joined IBMCR, Bangladesh, as a registered and specialist physician in the Department of Pediatrics. Just four months later, Hossain transitioned to Khwaja Yunus Ali Medical College, Bangladesh, as a consultant paediatrician and assistant professor in the Department of Pediatrics.

Hossain's research journey on LSDs began during his Ph.D. studies at Osaka University (October 2009 - March 2014). He focused on various lysosomal storage diseases (LSDs), including Krabbe disease, Galactosialidosis, and Mucolipidosis. His doctoral thesis specifically explored Krabbe disease, investigating its pathophysiology (how it develops) and leading to the discovery of promising new therapeutic approaches for Krabbe and Galactosialidosis. Following graduation, Hossain spent two years (2014-2016) in Saudi Arabia working as a Specialist Paediatric Doctor and Co-investigator at the King Abdullah Specialized Children’s Hospital and Research Center. He also maintained a collaborative relationship with Professor Norio Sakai at Osaka University, which allowed him to continue publishing research initiated during his Ph.D. studies.

In 2016, Hossain returned to Japan to join the Advanced Clinical Research Center as a laboratory chief scientist. There, he worked alongside Prof. Yoshikatsu Eto.

Eto's guidance opened doors for Hossain to explore a broader range of LSDs, including Fabry disease, Pompe disease, Metachromatic Leukodystrophy (MLD), Adrenoleukodystrophy (ALD), Neuronal Ceroid Lipofuscinosis (NCL), and Niemann-Pick disease type C (NPC). This diverse experience allowed him to make a significant contribution by uncovering a novel pathophysiological mechanism specifically affecting females with Fabry disease.

Since 2021, Hossain has been a medical advisor at JCR Pharmaceuticals Ltd. where their focus is on developing a next-generation enzyme replacement therapy (ERT) for various lysosomal storage diseases (LSDs). Current ERT treatments have limitations, as they are unable to reach the central nervous system (CNS) effectively and, therefore, cannot address neurocognitive impairment, a common symptom of many LSDs. JCR Pharmaceuticals is pioneering a new form of ERT (J-Brain Cargo), specifically designed to target both the CNS and somatic organs. This advancement has the potential to significantly improve the lives of LSD patients by not only addressing their physical symptoms but also helping to maintain average growth and development. Hossain's expertise in LSDs allows them to play a vital role in this groundbreaking effort.

In addition to his research achievements, Hossain has been recognized as a valuable speaker and mentor in the field of LSDs. Notably, he has been invited as a guest speaker at the International Symposium on Data Science in NAIST and served as a mentor at the WORLDSymposium, the largest international conference on LSDs.

Honors and Awards

 * Monbu-kagaku-shō also known as MEXT Scholarship (Japanese Government Scholarship) October 2009- March 2014


 * Young Investigator Award, 2011, Asian Congress for Inherited Metabolic Diseases (ACIMD), Tokyo, Japan


 * Young Scientist Travel Award 2012, Society for the Study of Inborn Errors of Metabolism (SSIEM), Birmingham, UK


 * Young Investigator Award 2014 (Eto Prize), 56th Annual Meeting of the Japan Society of Inherited Metabolic Diseases and Asian Symposium of Inherited Metabolic Diseases, Sendai, Japan


 * Young Scientist Award 2017, Journal of Human Genetics (JHG), Nature


 * Kunihiko Suzuki Award, Japan Society of Lysosomal Diseases


 * JSIMD Young Investigator Award, Japan Society of Inherited Metabolic Diseases (JSIMD)


 * Young Investigator Award, 2020, World Symposium, Orlando

Professional affiliations and licenses
Member, Society for the Study of Inborn Errors of Metabolism (SSIEM), Europe

Member, Japan Pediatrics Society

Member, Japanese Society of Human Genetics

Member, Japanese Society of Inherited Metabolic Diseases

Life Member, Bangladesh Pediatrics Association

Licenses

 * 1) Permanent medical license by Bangladesh Medical and Dental Council (BMDC) : Reg No. A43074
 * 2) Permanent Medical license by Saudi Commission for Health Specialties (SCFHS) : Reg No.  15-R-M-0015484

Publications

 * 1) Nakamura K, Sakai N, Hossain MA, Eisengart JB, Yamamoto T, Tanizawa K, So S, Schmidt M, Sato Y. Analysis of caregiver perspectives on patients with mucopolysaccharidosis II treated with pabinafusp alfa: results of qualitative interviews in Japan. Orphanet J Rare Dis. 2024 Mar 7;19(1):104. doi: 10.1186/s13023-024-03112-1. PubMed PMID: 38454486; PubMed Central PMCID: PMC10921713.
 * 2) Hossain MA, Hasegawa-Ogawa M, Manome Y, Igarashi M, Wu C, Suzuki K, Igarashi J, Iwamoto T, Okano HJ, Eto Y. Generation and characterization of motor neuron progenitors and motor neurons using metachromatic leukodystrophy-induced pluripotent stem cells. Mol Genet Metab Rep. 2022 Jun;31:100852. doi: 10.1016/j.ymgmr.2022.100852. eCollection 2022 Jun. PubMed PMID: 35782608; PubMed Central PMCID: PMC9248224.
 * 3) Saito R, Miyajima T, Iwamoto T, Wu C, Suzuki K, Hossain MA, Munakata M, Era T, Eto Y. A neuropathological cell model derived from Niemann-Pick disease type C patient-specific iPSCs shows disruption of the p62/SQSTM1-KEAP1-NRF2 Axis and impaired formation of neuronal networks. Mol Genet Metab Rep. 2021 Sep;28:100784. doi: 10.1016/j.ymgmr.2021.100784. eCollection 2021 Sep. PubMed PMID: 34377675; PubMed Central PMCID: PMC8327345.
 * 4) Wu C, Iwamoto T, Hossaina MA, Akiyama K, Igarashi J, Miyajima T, Eto Y. A combination of 7-ketocholesterol, lysosphingomyelin and bile acid-408 to diagnose Niemann-Pick disease type C using LC-MS/MS. PLoS ONE 2020, 15(9), e0238624.
 * 5) Hossain MA, Wu C, Yanagisawa H, Miyajima T, Akiyama K, Eto Y. Future clinical and biochemical predictions of Fabry disease in females by methylation studies of the GLA gene. Mol Genet Metab Rep. 2019, 20, 100497.
 * 6) Yanagisawa H, Hossain MA, Miyajima T, et al. Dysregulated DNA methylation of GLA gene was associated with dysfunction of autophagy. Mol Genet Metab 2019, 126(4), 460-465.
 * 7) Hossain MA, Miyajima T, Akiyama K, Eto Y. A case of adult-onset Pompe disease with cerebral stroke and left ventricular hypertrophy. J Stroke Cerebrovasc Dis 2018, 27(11), 3046-3052.
 * 8) Itagaki R, Endo M, Yanagisawa H, Hossain MA, Akiyama K, Yaginuma K, Miyajima T, Wu C, Iwamoto T, Igarashi J, Kobayashi Y, Tohyama J, Iwama K, Matsumoto N, Shintaku H, EtoY. Characteristics of PPT1 and TPP1 enzymes in neuronal ceroid lipofuscinosis (NCL) 1 and 2 by dried blood spots (DBS)and leukocytes and their application to newborn screening. Mol Genet Metab 2018, 124(1), 64-70.
 * 9) Irahara-Miyana K, Otomo T, Kondo H, Hossain MA, Ozono K, Sakai N. Unfolded protein response is activated in Krabbe disease in a manner dependent on the mutation type. JHG 2018, 63(6), 699-706.
 * 10) *Okada J, *Hossain MA, Wu C, Miyajima T, Yanagisawa H, Akiyama K, Eto Y. Ten-year-long enzyme replacement therapy shows a poor effect in alleviating giant leg ulcers in a male with Fabry disease. Mol Genet Metab Rep 2017, 14, 68-72. *Equal contribution.
 * 11) Hossain MA, Yanagisawa H, Miyajima T, Wu C, Takamura A, Akiyama K, Itagaki R, Eto K, Iwamoto T, Yokoi T, Kurosawa K, Numabe H, Eto Y. The severe clinical phenotype for a heterozygous Fabry female patient correlates to the methylation of non-mutated allele associated with chromosome 10q26 deletion syndrome. Mol Genet Metab 2017, 120 (3), 173–179.
 * 12) Wu C, Iwamoto T, Igarashi J, Miyajima T, Hossaina MA, Yanagisawaa H, Akiyama K, Shintaku H, Eto Y. Application of a diagnostic methodology by quantification of 26:0 lysophosphatidylcholine in dried blood spots for Japanese newborn screening of X-linked adrenoleukodystrophy. Mol Genet Metab Rep 2017, 12, 115–118.
 * 13) Hossain MA, Obaid A, Rifai M, Alem H, Hazwani T, Shehri AA, Alfadhel M, Eto Y, Eyaid W. An early onset of Fazio–Londe Syndrome; first case report from Arab peninsula. Hum Genome Var 2017, 4, 17018.
 * 14) Alfadhel M, Benmeakel M, Hossain MA, Mutairi FA, Othaim AA, Alfares AA, Balwi MA, Alzaben A, Eyaid W. Thirteen year retrospective review of the spectrum of inborn errors of metabolism presenting in a tertiary center in Saudi Arabia. Orphanet Journal of Rare Diseases 2016, 11 (1),126.
 * 15) Hossain MA, Higaki K, Shinpo M, Nanba E, Suzuki Y, Ozono K, Sakai N. Chemical chaperone treatment for galactosialidosis: Effect of NOEV on β-galactosidase activities in fibroblasts. BRADEV 2016, 38 (2), 175–80.
 * 16) Hossain MA, Higaki K, Saito S, Ohno K, Sakuraba H, Nanba E, Suzuki Y, Ozono K, Sakai N. Chaperone therapy for Krabbe disease: potential for late-onset GALC mutations. JHG 2015, 60 (9), 539–45.
 * 17) Hossain MA, Otomo T, Seiji Saito, Kazuki Ohno, Hitoshi Sakuraba, Hamada Y, Ozono K, Sakai N. Late-onset Krabbe disease is predominant in Japan and its mutant precursor protein undergoes more effective processing than the infantile onset form. GENE 2014, 534 (2), 144–54.
 * 18) Otomo T, Hossain MA, Ozono K, Sakai N. Genistein reduces heparan sulfate accumulation in human mucolipidosis II skin ﬁbroblasts. Mol Genet Metab 2012, 105(2), 266-69.
 * 19) Kardas F, Uzak AS, Hossain MA, Sakai N, Canpolat M, Yıkılmaz A. A Novel Homozygous GALC Mutation: Very Early Onset and Rapidly Progressive Krabbe Disease. GENE 2013, 517 (1), 125- 27.
 * 20) Tokushige SI, Sonoo T, Maekawa R, Shirota Y, Hanajima R, Terao Y, Matsumoto H, Hossain MA, Sakai N, Shiio Y. Isolated pyramidal tract impairment in the central nervous system of adult-onset Krabbe disease with novel mutations in the GALC gene. Brain Dev 2013, 35 (6), 579-81.
 * 21) Rahman MS, Nagai Y, Popiel HA, Fujikake N, Okamoto Y, Ahmed MU, Islam MA, Islam MT, Ahmed S, Rahman KM, Uddin MJ, Dey SK, Ahmed Q, Hossain MA, Jahan N, Toda T. Genetic testing for Huntington’s disease in Parkinsonism. Mymensingh Med J 2010, 19(4), 510-14.
 * 22) Rahman AKMA, Begum N, Nooruddin M, Rahman MS, Hossain MA, Song HJ. Prevalence and risk factors of helminth infections in cattle of Bangladesh. Korean Journal of Veterinary Service 2009, 32 (3), 265-73.
 * 23) Uddin MJ, Rahman MS, Akter SH, Hossain MA, Islam MT, Islam MA, Park JH, Song HJ. Seroprevalence of brucellosis in small ruminants in selected area of Bangladesh. Korean Journal of Veterinary Service 2007,31 (4), 511-25.
 * 24) Uddin JM, Rahman MS, Hossain MA, Akter SH, Majumder S, Park JH, Song HJ. Relation between brucellosis and husbandry practices in goats in Bangladesh. Korean Journal of Veterinary Service 2007, 30 (2), 259-67.
 * 25) Rahman AKMA, Nooruddin M, Hossain MM, Rahman MS, Hossain MA, Song HJ. Pulmonary Diseases in Slaughtered Cattle 4. Pathology of Pulmonary Lesions. Korean Journal of Veterinary Service 2006, 29 (4), 483-87.
 * 26) Rahman MS, Uddin MJ, Park JH, Chae JS, Rahman MB, Islam MA. A short history of brucellosis: special emphasis in Bangladesh. Bangl. J. Vet. Med 2006, 4 (1), 01–06.

Books
Dr. Hossain has authored Chapter 6 of the book Krabbe Disease. Inherited metabolic diseases handbook (先天代謝異常ハンドブック). Published by Nakayama Shoten Co. Ltd. Volume 1, Issue 1 (2013). With Sakai N.