Navigation Links
Researchers report success in treating autism spectrum disorder
Date:7/2/2012

CINCINNATIUsing a mouse model of autism, researchers at the University of Cincinnati (UC) and Cincinnati Children's Hospital Medical Center have successfully treated an autism spectrum disorder characterized by severe cognitive impairment.

The research team, led by Joe Clark, PhD, a professor of neurology at UC, reports its findings online July 2, 2012, in the Journal of Clinical Investigation, a publication of the American Society for Clinical Investigation.

The disorder, creatine transporter deficiency (CTD) is caused by a mutation in the creatine transporter protein that results in deficient energy metabolism in the brain. Linked to the X chromosome, CTD affects boys most severely; women are carriers and pass it on to their sons.

The brains of boys with CTD do not function normally, resulting in severe speech deficits, developmental delay, seizures and profound mental retardation. CTD is estimated to currently affect about 50,000 boys in the United States and is the second-most common cause of X-linked mental retardation after Fragile X syndrome.

Following CTD's discovery at UC in 2000, researchers at UC and Cincinnati Children's led by Clark discovered a method to treat it with cyclocreatinealso known as CincY, and pronounced cinci-whya creatine analogue originally developed as an adjunct to cancer treatment. They then treated genetically engineered mice as an animal model of the human disease.

"CincY successfully entered the brain and reversed the mental retardation-like symptoms in the mice, with benefits seen in nine weeks of treatment," says Clark, adding that no harmful effects to the mice were observed in the study. "Treated mice exhibited a profound improvement in cognitive abilities, including recognition of novel objects, spatial learning and memory."

As a repurposed drug (originally developed for another therapy), CincY has already been through part of the U.S. Food and Drug Administration (FDA) approval process. It is taken orally as a pill or powder.

UC's Office of Entrepreneurial Affairs and Technology Commercialization has reached agreement with Lumos Pharma, a privately held Austin, Texas, startup company based on UC technology, to develop and commercialize CincY. Lumos Pharma was created with technology licensed from UC's Office of Entrepreneurial Affairs and Technology Commercialization. Its CEO is Rick Hawkins, a 30-year biotech industry veteran. Jon Saxe is its chairman.

"It has taken many years to get here and I am happy that our efforts have led to this translational effort to make a therapy available to those afflicted with CTD," says Clark. "We look forward with commitment and hope to the day when those patients will benefit from our work."

The collaboration gained momentum when Lumos Pharma submitted a proposal based on Clark's technology to the National Institutes of Health and was selected as a drug development project partner by the National Center for Advancing Translational Sciences' Therapeutics for Rare and Neglected Diseases (TRND) program. Under TRND's collaborative operational model, project partners form joint project teams with TRND and receive in-kind support from TRND drug development scientists, laboratory and contract resources.

Lumos Pharma plans to initiate a TRND-supported preclinical development plan, with TRND support continuing through the filing of an Investigational New Drug (IND) application with the FDA prior to beginning a clinical trial. Such a trial would be about three years away, Clark says.

In addition to Clark, study team members are Yuko Kurosawa, PhD; Ton de Grauw, MD, PhD; Diana Lindquist, PhD; Victor Blanco, PhD; Gail Pyne-Geithman, DPhil; Takiko Daikoku, PhD; James Chambers, PhD; and Stephen Benoit, PhD.


'/>"/>

Contact: Keith Herrell
keith.herrell@uc.edu
513-558-4559
University of Cincinnati Academic Health Center
Source:Eurekalert

Related medicine news :

1. NIH awards $20 million over 5 years to train next generation of global health researchers
2. Researchers develop a new cell and animal model of inflammatory breast cancer
3. Researchers uncover a viable way for colorectal cancer patients to overcome drug resistance
4. Researchers Find Gene Mutations That May Be a Key to Autism
5. Researchers find evidence of banned antibiotics in poultry products
6. NJ stroke researchers report advances in spatial neglect research at AAN Conference
7. Autism by the numbers: Yale researchers examine impact of new diagnostic criteria
8. Researchers Map Brain Regions Linked to Intelligence
9. Researchers ID Genes That May Determine Mental Illness
10. Researchers Develop Blood Test for Depression
11. University of Cincinnati researchers win $3.7M grant from US Department of Defense
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:1/17/2017)... Phoenix, AZ (PRWEB) , ... January 17, 2017 ... ... new and improved supplement, Healthful-Flex. , The company, owned and operated by ... the public at a reasonable price with the highest level of customer service. ...
(Date:1/17/2017)... ... January 17, 2017 , ... SunView Software’s Service Smart ... Innovation of the Year. , Each year, Pink Elephant recognizes a new ... approach to address a specific business problem or opportunity. The award highlights original innovations ...
(Date:1/17/2017)... ... , ... Kevin Harrington (one of the original Sharks from mega-hit reality series ... topic of Beauty and Personal Care. , Everyone makes New Year’s ... commit to these changes than beginning with personal care and beauty products? AsSeenOnTV.pro will ...
(Date:1/17/2017)... Grove Village, IL (PRWEB) , ... January 17, ... ... for musicians and music enthusiasts at the National Association of Music Merchants (NAMM) ... January 19 – 22, 2017. , Etymotic’s ER•4® has long been the gold ...
(Date:1/17/2017)... ... January 17, 2017 , ... Metro Development Group has ... high speed technology into the fabric of an entire community. A highlight of ... and wellness in a yet-to-be-named, health focused campus. Leading this initiative in partnership ...
Breaking Medicine News(10 mins):
(Date:1/17/2017)... the company that brought a full line of equine first aid ... line of companion animal pet first aid kits.  EquiMedic ... has developed two sizes of small pet first aid kits under ... Featuring a small and a large companion pet first aid kit, ... a newcomer to working with small animals either. Corporate owners, mother ...
(Date:1/17/2017)... , Jan. 17, 2016  Attorney Advertising -- Bronstein, ... of purchasers of Anthera Pharmaceuticals, Inc. ("Anthera" or the "Company") ... obtain additional information and assist the investigation by visiting the ... whether Anthera and certain of its officers and/or directors have ... of 1934. ...
(Date:1/17/2017)... 2017 Research and Markets has announced the ... Generic), Manufacturer (Captive, Merchant), Synthesis (Synthetic, Biotech), Product (mAb, Hormone) Drug ... 2021" report to their offering. ... The global active pharmaceutical ingredients market is ... Billion in 2016, growing at a CAGR of 6.3% from 2016 ...
Breaking Medicine Technology: