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:5/22/2017)... ... May 22, 2017 , ... ... welcoming new patients with periodontal plastic surgery treatments, including crown lengthening ... Dr. Green provides these esthetic and functional procedures to help patients improve their ...
(Date:5/22/2017)... ... 2017 , ... “Homeostasis: The Journey of a Manic-Depressive” tells the story in ... Rodriguez takes readers on a rollercoaster ride that includes a midnight trek to the ... a dark place where Donald considers suicide. Readers learn how Donald finally achieves ...
(Date:5/22/2017)... ... May 22, 2017 , ... WeightZone Factor a popular ... Factor members to track their exercise patterns, monitor behavioral changes, employ biometric monitoring, ... their mobile phones. It also provides social networks for members with similar interests ...
(Date:5/21/2017)... ... 19, 2017 , ... HealthSmart, the largest independent benefits administrator ... as Chief Information Officer (CIO). He will be joining HealthSmart at the end ... performance. He defines strategic roadmaps by reimagining business processes—bridging the gap between information ...
(Date:5/21/2017)... ... , ... Pot. Reefer. Grass. Mary Jane. No matter what people call it, ... never before. More than a map, new social marketplace Weedable goes beyond ... and for the first time – each other. A social marketplace like no other, ...
Breaking Medicine News(10 mins):
(Date:5/10/2017)... 2017  The Corporate Whistleblower Center says, "We ... therapy clinics to call us anytime at 866-714-6466 if ... involved in a substantial scheme to overbill Medicare. ... an employee of a medical equipment company if ... provide medical practice groups with extra generous incentives to ...
(Date:5/10/2017)... 2017 CSSi, the global leader in patient ... industry, is proud to announce the launch of its ... features both enriched content and a customized layout that ... company,s already well-established position as the top global patient ... many months of hard work, we are delighted to ...
(Date:5/9/2017)... -- Demonstrating its commitment to representing research- based biopharmaceutical ... Research and Manufacturers of America (PhRMA) today approved ... have to meet new research and development (R&D) ... PhRMA. "By putting in place new ... message that being a member of PhRMA means ...
Breaking Medicine Technology: