Navigation Links
NIH grant for the move toward clinical trials targeting the lysosomal storage disease MPSIIIB
Date:5/25/2011

Investigators at Nationwide Children's have received a grant from the National Institutes of Health (NIH) to help move a therapy for MPS IIIB that has been shown effective in mice toward clinical trials in humans.

Mucopolysaccharidosis (MPS) IIIB, also known as Sanfilippo Syndrome B, is a lysososmal storage disease caused by deficiency in the essential enzyme NAGLU. Children with MPS IIIB appear normal at birth, but develop severe, progressive developmental delay and neurological disorders by 2 years of age. MPS IIIB is a fatal disease and there is currently no treatment available.

"To date, the greatest challenge in developing therapies for MPS IIIB has been the presence of the blood-brain barrier, which prevents therapeutics from entering the central nervous system," said Haiyan Fu, PhD, and the project's lead investigator.

For more than a decade, Dr. Fu's team in the Center for Gene Therapy in The Research Institute at Nationwide Children's Hospital has been focusing on developing gene delivery approaches to efficiently restore the central nervous system NAGLU activity, which is missing in MPS IIIB patients. Using a single intravenous injection of a recently characterized viral vector, AAV9, which has the unique ability to cross the blood-brain barrier, Dr. Fu's team has achieved the best long-term therapeutic benefits to date in adult MPS IIIB mice. This strategy has resulted in correction of cognitive and motor function and extended survival in these mice, which like humans with MPS IIIB lack the NAGLU enzyme.

The NIH funding, awarded to Dr. Fu and co-investigator Kevin Flanigan, MD, will allow the team to complete necessary preclinical studies and to submit an investigational new drug application to the United States Food and Drug Administration for a Phase I/II AAV9 gene therapy clinical trial in patients with MPS IIIB. "Importantly, the intravenous rAAV9 gene delivery procedure is minimally invasive and is therefore a clinically relevant approach," said Dr. Fu.

"The Center for Gene Therapy at Nationwide Children's Hospital has been a leader in bridging gene therapy trials from the bench into the clinic, and we are excited about bringing that expertise to bear on this devastating disorder," added Dr. Flanigan.

Dr. Fu's MPS IIIB gene therapy project has received generous support since 2003 from the MPS III patient community through Ben's Dream The Sanfilippo Research Foundation. A research grant from the foundation was critical in supporting Dr. Fu's team while they were pursuing the NIH grant application.

"We believe the quote that 'Hope sees the invisible, feels the intangible and achieves the impossible,'" said Jennifer Siedman, president and secretary of Ben's Dream  the Sanfilippo Research Foundation. "Dr. Fu's recent research breakthrough and its recognition by the NIH with the awarding a U01 grant, brings to the Sanfilippo community the hope that a cure is on the horizon. The grant is not only a tribute to Dr. Fu's dedication to the field, but also to all the families and friends of Ben's Dream who have worked together for over a decade to fund this research."


'/>"/>
Contact: Erin Pope
Erin.Pope@NationwideChildrens.org
614-355-0495
Nationwide Children's Hospital
Source:Eurekalert

Related medicine news :

1. MetLife Foundations grant bolsters media coverage for aging issues
2. UTMB researcher receives $3 million NIH grant to study aging in Mexico
3. NIH awards Salk Institute $5.5 million grant to study Williams syndrome
4. Grant extends funding for the Broads Stanley Center for Psychiatric Research
5. Cedars-Sinais mobile medical clinics receive $500,000 grant from California Community Foundation
6. Immigrants Eat American Junk Food to Fit In: Study
7. Gates Foundation awards Ben-Gurion U. researcher grant for malaria detection using cell phones
8. Grant funds next phase in bringing healthier sorghum closer to underserved communities
9. K-RITH awards grants to foster teamwork among South African TB/HIV scientists
10. $4.5 million grant creates transdisciplinary program to train scholars in child obesity prevention
11. Fatting in: Immigrant groups eat high-calorie American meals to fit in
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:8/17/2017)... (PRWEB) , ... August 17, 2017 , ... ... care professionals, today announced that it has raised $1.675 M in seed funding ... Rethink Education, Lumina Foundation, and Techstars Venture Capital Fund are the company’s primary ...
(Date:8/17/2017)... ... , ... Pot Valet is a leading provider of premium medical-grade cannabis in ... and soon, every state in the country, the company offers patients safe, legal, and ... , According to Pot Valet, all California patients have the legal right ...
(Date:8/17/2017)... ... ... industry leader in digital marketing for medical practices, has been recognized as one of the ... 5000 for the second time in two years. Shawn Miele, Chief Executive Officer of Advice ... second year in a row. It’s extremely difficult to make this list once. The fact ...
(Date:8/17/2017)... , ... August 17, 2017 , ... ... Employers , an international competition, were announced today. The awards recognize the world’s ... suppliers who help to create and drive great places to work. , A ...
(Date:8/17/2017)... , ... August 17, 2017 , ... ... Business Development Group, a Florida-based oncology business advisor to oncology practices, announced today ... the dollar amount of attributable savings from resolving critical clinical issues that would ...
Breaking Medicine News(10 mins):
(Date:7/28/2017)... July 28, 2017 EnvoyHealth, a Diplomat company, has ... program for CleverCap LITE, a technology designed to improve ... deliver innovative health technology solutions and services that help ... CleverCap LITE offers medication monitoring and ... cover: Records date ...
(Date:7/27/2017)... Services, Inc. (NYSE: WST ) today announced ... financial guidance for the full-year 2017. ... Reported net sales of $397.6 million, a record high, ... constant currency (organic) grew by 3.9%. ... in the prior-year quarter. Second-quarter 2017 adjusted diluted EPS ...
(Date:7/26/2017)... Medical Aesthetics, a leading medical aesthetic clinic in Singapore ... several skin conditions from the inside. The natural process ... The skin becomes more transparent due to the thinning of the ... from the sun contributes to aging skin, causing age spots and ... ...
Breaking Medicine Technology: