High dose genistein aglycone therapy is safe in patients with mucopolysaccharidoses involving the central nervous system

Molecular Genetics and Metabolism

Volumn 109, Issue 4, 2013, Pages 382-385

  Kim, Katherine H ^a,b   Dodsworth, Charlotte ^a   Paras, Andrea ^a,b   Burton, Barbara K ^a,b  

^a CHILDREN'S MEMORIAL HOSPITAL   (United States)

^b NORTHWESTERN UNIVERSITY   (United States)

Author keywords

Genistein; Mucopolysaccharidosis; Sanfilippo; Treatment

Indexed keywords

AGLYCONE; GENISTEIN; GENISTEIN AGLYCONE; GLYCOSAMINOGLYCAN; HEPARAN SULFATE; UNCLASSIFIED DRUG;

ADULT; AGGRESSION; ALLERGIC REACTION; APPETITE; ARTICLE; BLEEDING; BREAST DEVELOPMENT; CARPAL TUNNEL SYNDROME; CENTRAL NERVOUS SYSTEM; CLINICAL TRIAL; COGNITION; CONJUNCTIVITIS; CONSTIPATION; DEEP VEIN THROMBOSIS; DIARRHEA; DISABILITY; DRUG EFFICACY; DRUG INDUCED HEADACHE; DRUG MEGADOSE; DRUG SAFETY; DYSPHAGIA; DYSPNEA; EAR INFECTION; EPISTAXIS; FEMALE; GALLSTONE; GASTROENTERITIS; HEARING IMPAIRMENT; HEART VENTRICLE HYPERTROPHY; HUMAN; HYPERACTIVITY; LIVER DISEASE; MALE; MENSTRUATION DISORDER; MUCOPOLYSACCHARIDOSIS; MUSCLE TONE; PERITONITIS; PHENOTYPE; PHYSICAL EXAMINATION; PRIORITY JOURNAL; RATING SCALE; RECTUM POLYP; RESPIRATORY TRACT INFECTION; SCOLIOSIS; SEIZURE; SIDE EFFECT; SLEEP DISORDER; SLEEP DISORDERED BREATHING; SPEECH DISORDER; SUBDURAL HEMATOMA; URINARY EXCRETION; VALVULAR HEART DISEASE; VIRUS PNEUMONIA; VOMITING; ADOLESCENT; ADVERSE DRUG REACTION; ANIMAL; BRAIN; CELL LINE; CHILD; DRUG EFFECTS; FIBROBLAST; INFANT; MAXIMUM TOLERATED DOSE; METABOLISM; MOUSE; MUCOPOLYSACCHARIDOSES; PATHOLOGY; PRESCHOOL CHILD;

MUS;

ADOLESCENT; ANIMALS; BRAIN; CELL LINE; CENTRAL NERVOUS SYSTEM; CHILD; CHILD, PRESCHOOL; DRUG-RELATED SIDE EFFECTS AND ADVERSE REACTIONS; FEMALE; FIBROBLASTS; GENISTEIN; GLYCOSAMINOGLYCANS; HUMANS; INFANT; MALE; MAXIMUM TOLERATED DOSE; MICE; MUCOPOLYSACCHARIDOSES;

EID: 84880794642     PISSN: 10967192     EISSN: 10967206     Source Type: Journal    
DOI: 10.1016/j.ymgme.2013.06.012     Document Type: Article

References (16)

1. Valstar M.J., Ruigter G.J.G., van Digglen O.P., Poorthuis B.J., Wejburg F.A., Ruigter G.J.G., van Digglen O.P., Poorthuis B.J., Wejburg F.A. Sanfilippo syndrome: a mini review. J. Inherit. Metab. Dis. 2008, 31:240-252.
2. Wraith J.E., Scarpa M., Beck M., et al. Mucopolysaccharidosis II (Hunter syndrome): a clinical review and recommendations for treatment in the era of enzyme replacement therapy. Eur. J. Pediatr. 2008, 167:267-277.
3. Wraith J.E. The first 5years of clinical experience with laronidase enzyme replacement therapy for mucopolysaccharidosis I. Expert. Opin. Pharmacother. 2005, 6:489-506.
4. Piotrowska E., Jakobkiewicz-Banecka J., Baranska S., Tylki-Szymanska A., Czartoryska B., Wegrzyn A., Wegrzyn G. Genistein-mediated inhibition of glycosaminoglycan synthesis as a basis for gene-expression-targeted isoflavone therapy for mucopolysaccharidoses. Eur. J. Hum. Genet. 2006, 14:846-852.
5. Jakobkiewicz-Banecka J., Piotrowska E., Narajczyk M., Baranska S., Wegrzyn G. Genistein-mediated inhibition of glycosaminoglycan synthesis, which corrects storage in cells of patients suffering from mucopolysaccharidosis, acts by influencing an epidermal growth factor-dependent pathway. J. Biomed. Sci. 2009, 16:1-9.
6. Arfi A., Richard M., Gandolphe C., Scherman D. Storage correction in cells of patients suffering from mucopolysaccharidoses types IIIA and VII after treatment with genistein and other isoflavones. J. Inherit. Metab. Dis. 2010, 33:61-67.
7. Malinowska M., Wilkinson F.L., Bennett W., Langford-Smith K.J., O'Leary H.A., Jakobkiewicz-Banecka J., Wynn R., Wraith J.E., Wegrzyn G., Bigger B.W. Genistein reduces lysosomal storage in peripheral tissues of mucopolysaccharide IIIB mice. Mol. Genet. Metab. 2009, 98:235-242.
8. Friso A., Tomanin R., Salvalaio M., Scarpa M. Genistein reduces glycosaminoglycan levels in a mouse model of mucopolysaccharidosis type II. Br. J. Pharmacol. 2010, 159:1082-1091.
9. Malinowska M., Wilkinson F.L., Langford-Smith K.J., Langford-Smith A., Brown J.R., Crawford B.E., Vanier M.T., Grynkiewicz G., Wynn R.F., Wraith J.E., Wegrzyn G., Bigger B.W. Genistein improves neuropathology and corrects behavior in a mouse model of neurodegenerative metabolic disease. PLoS One 2010, 5:e14192.
10. Wegrzyn G., Jakobkiewcz-Banecka A., Piotrowska E., Malinowska M., Maryniak A., Tylki-Szymanska A., et al. Gene-expression targeted isoflavone therapy (GET IT) for mucopolysaccharidosis type III (Sanfilippo disease): a pilot clinical trial. J. Inherit. Metab. Dis. 2007, 30(Suppl. 1):116.
11. Piotrowska E., Jakobkiewicz-Banecka J., Tylki-Symanska A., Libereck A., Marynaik A., et al. Genistein-rich soy isoflavone extract in substrate reduction therapy for Sanfilippo syndrome: an open-label, pilot study in 10 pediatric patients. Curr. Ther. Res. 2008, 69:166-179.
12. Marucha J., Tylki-Szymańska A., Jakóbkiewicz-Banecka J., Piotrowska E., Kloska A., Czartoryska B., We{ogonek}grzyn G. Improvement in the range of joint motion in seven patients with mucopolysaccharidosis type II during experimental gene expression-targeted isoflavone therapy (GET IT). Am. J. Med. Genet. A 2011, 9:2257-2262.
13. Delgadillo V., O'Callaghan M., Trtuch R., Montero R., Pineda M. Genistein supplementation in patients affected by Sanfilippo disease. J. Inherit. Metab. Dis. 2011, 34:1039-1044.
14. de Ruijter J., Valstar M.J., Narajczyk M., Wegrzyn G., Kulik W., Ijilst L., Wagemans T., van der Wal W.M., Wijburg F.A. Genistein in Sanfilippo disease: a randomized controlled crossover trial. Ann. Neurol. 2012, 71:110-120.
15. Meyer A., Kossow K., Gal A., Muhlhausen C., Ullrich K., Braulke T., Muschol N. Scoring evaluation of the natural course of mucopolysaccharidosis type IIIA (Sanfilippo syndrome type A). Pediatrics 2007, 120:e1255-e1261.
16. Ullmann U., Bendik I., Fluhmann B. Bonistein ™ (synthetic genistein), a food component in development for a bone health nutraceutical. J. Physiol. Pharmacol. 2005, 56(Suppl. 1):79-95.