The U.S. Food and Drug Administration today approved Cuvposa (glycopyrrolate) Oral Solution to treat chronic severe drooling caused by neurologic disorders in children ages 3 years to 16 years.

Drooling is normal in infants. But a significant proportion of the developmentally disabled population experiences drooling caused primarily by neuromuscular dysfunction that makes it hard to swallow. Cuvposa reduces drooling by lowering the volume of saliva produced.

Glycopyrrolate was approved decades ago to treat peptic ulcers and reduce salivation in patients under anesthesia. Until now, glycopyrrolate has been used on an off-label basis to treat drooling in the developmentally disabled population, but in a different dosage form than the approved product. A drug is said to be used off-label when a physician prescribes its use in a different way than described in the FDA-approved drug label.

In 2001, the FDA held an advisory committee meeting to discuss how best to develop products for drooling with ethically and scientifically sound trials in children who have neurological disorders. Utilizing the advice provided, the FDA has been able to move forward in addressing the needs of this population.

“Cuvposa provides an important therapy for controlling salivation in patients with neurologic disease,” said John Jenkins, M.D., director of the Office of New Drugs in the FDA’s Center for Drug Evaluation and Research. “FDA approval not only ensures that the product meets modern standards for safety, effectiveness, quality and labeling; but, also results in a more suitable dose form for this patient population.”

The FDA drug approval process provides a review of product-specific information that is critical to ensuring the safety and efficacy of a finished drug product. For instance, the applicant must demonstrate that its manufacturing processes can reliably produce drug products of expected identity, strength, quality, and purity. FDA’s review of the applicant’s labeling ensures that health care professionals and patients have the information necessary to understand a drug product’s risks and its safe and effective use.

When used off label, oral tablets of glycopyrrolate had to be crushed to treat drooling in children with neurological disorders. Cuvposa is a flavored oral solution that is easier to administer and provides the optimal dose for each patient.

In clinical trials of Cuvposa glycopyrrolate oral solution, 78 percent of the children on the drug reached clinical improvement in drooling compared with 19 percent of those given an inactive substance (placebo).

Common adverse reactions reported with glycopyrrolate are dry mouth, constipation, flushing, and urinary retention.

Cuvposa (glycopyrrolate) Oral Solution is marketed by Shionogi Pharma Inc. of Osaka, Japan.

Media Inquiries: Elaine Gansz Bobo, 301-796-7567; elaine.bobo@fda.hhs.gov
Consumer Inquiries: 888-INFO-FDA

The NIH has a new program entitled the NIH Blueprint for Neuroscience Research. This program is designed to turn discoveries into new drugs for nervous system diseases by providing a “virtual pharma” with resources, expertise and funding to advance promising compounds from chemical optimization through Phase 1 clinical testing. The network is structured so that the PI can retain ownership of the intellectual property for drugs developed within it. Disorders of interest include, but are not limited to, neurological, psychiatric and developmental disorders, dementias of aging, diseases and disorders of the eye or ear, and drug and alcohol dependence and addiction. The ultimate goals of this Neurotherapeutics Grand Challenge are to produce at least one novel and effective drug for a nervous system disorder that is currently poorly treated and to catalyze industry interest in novel disease targets by demonstrating early-stage success.
Entry requirements are the following:

• At least one small molecule compound with strong evidence of biologic activity in a nervous system disease assay.
• A robust, moderate throughput assay of biological activity suitable for testing compounds generated in an iterative medicinal chemistry effort.
• Secondary bioassays and models sufficient to evaluate the potential of a drug candidate for the intended indication.

To apply, go to
http://grants.nih.gov/grants/guide/rfa-files/RFA-NS-11-002.html
The deadline is August 10, 2010

Contact Dr. Rebecca Farkas at farkasr@ninds.nih.gov or (301) 496-1779
for more information regarding this new initiative

The Cerebral Palsy International Research Foundation (CPIRF) Board of Directors is pleased to welcome and announce the appointment of Mr. Glenn R. Tringali as Chief Executive Officer effective April 13, 2010. Since 1975, Mr. Tringali has spent his entire professional career in the nonprofit community, having held senior administrative and fund development positions with some of the country’s most prominent health agencies, including the March of Dimes, the Juvenile Diabetes Research Foundation, and Autism Speaks. “We are delighted to have the expertise and experience of Glenn to help lead our efforts at CPIRF going forward” said Paul Volcker, Chairman of CPIRF.

Most recently, Mr. Tringali was the Chief Development Officer at the Cancer Research Institute and from 2001-2008, previously served the autism community, first as CEO of the National Alliance for Autism Research prior to leading the organization’s successful merger with Autism Speaks in February, 2006 where he was named Executive Vice President.

“I am incredibly honored and excited to be joining the Cerebral Palsy International Research Foundation. The national and global research and education programs we are funding are critically important. With an estimated 1 million children and adults with CP in our country, the need for supporting our efforts has never been greater”.

CPIRF is the nation’s preeminent private organization dedicated to the prevention and treatment of Cerebral Palsy and related developmental disorders. In its fifty-five year history, CPIRF has provided more than $50 million to support research and educational activities in the biomedical and clinical sciences.

Dr. John Hsu is the 2010 winner of the Weinstein-Goldenson Medical Science Award. This award is presented annually to a clinician-scientist for outstanding contributions in medical research which enhance the lives of individuals with cerebral palsy and their families.

Dr. Hsu is an Emeritus Clinical Professor in the Department of Orthopedics, at the University of California, Keck School of Medicine. He is the attending orthopedic surgeon at the Muscle Disease Clinic, Rancho Los Amigos National Rehabilitation Hospital and at the Children’s Fracture Clinic at the Orthopedic Hospital in Los Angeles, CA.  He is a member of the American Orthopedic Association, the American Academy of Orthopaedic Surgeons, the American College of Surgeons and the Pediatric Rehabilitation Association among others. Dr. Hsu has over 120 publications and written numerous book chapters in the field of orthopedic surgery interventions for neuromuscular disorders such as cerebral palsy and Duchenne muscular dystrophy. Dr. Hsu has had a long and distinguished career in caring for children and adults with cerebral palsy. He will be presented with the 2010 Weinstein-Goldenson Medical Science Award at the Transformational Technology Workshop to be held on September 2-4, 2010 in Downey, CA.

Dr. Terrance Sanger is the 2010 winner of the Isabelle and Leonard H. Goldenson Technology and Rehabilitation Award. This award is presented annually to a scientist for outstanding contributions in the development and use of technology and rehabilitation methodologies that enhance the quality of life for individuals with cerebral palsy and other disabilities and their families.

Departments of Biomedical Engineering, Neurology, and Biokinesiology and on the medical staff at the Children’s Hospital of Los Angeles, Department of Neurology. He graduated from Harvard Medical School Division of Health Sciences and Technology with a medical degree in 1993 and from the Massachusetts Institute of Technology with a doctorate in Electrical Engineering and Computer Science.  Dr. Sanger’s primary research goal is to understand and prevent adverse effects of childhood brain disorders on motor development. His major research areas in computational neurosciences and neural networks include the mathematical description of dystonia and abnormal human movement; mathematical models of abnormal basal ganglia function and neural networks of normal and disordered learning. His major clinical research areas include tools for quantification and diagnosis of dystonia in children, the role of botulinum toxin in the modification of learned motor patterns, and feedback retraining of multiple-muscle patterned movement. He is currently funded by the NIH to use high-speed programmable logic devices to simulate the development of long and short-latency reflexes and the evolution of spasticity and dystonia in children with early brain injury. He has had past research funding from NIH, CPIRF, the Lucile Packard Children’s Hospital, the Allergan Corporation,  the Pfizer Pharmaceuticals Group and  Elan Pharmaceuticals. He has published 52 papers in peer reviewed journals and has been invited to present at scientific meetings and conferences on well over 32 occasions.  Dr. Sanger will be presented with the Isabelle and Leonard H. Goldenson Technology and Rehabilitation Award at the 2010 American Academy of Cerebral Palsy and Developmental Medicine Annual Conference in Washington DC on September 24th in Washington, DC.

CPIRF is proud to co-sponsor a two-day workshop entitled ‘Themes in Neurorehabilitation: CP Across the Lifespan’ at the Kennedy Krieger Institute in Baltimore, MD on November 5 and 6^th 2010. Dr. Mindy Aisen, CPIRF’s Medical Director, will be giving the keynote address. Come learn about the best-evidence practices for the treatment of the medical, rehabilitative and psychosocial conditions that may occur in individuals with cerebral palsy at each stage of life. Educators, nurses, physical therapists, occupational therapist, speech & language pathologists, community program coordinators, psychologists, social workers, counselors, parents and individuals with disabilities are encouraged to attend.

READ THE EVENT FLYER HERE

Save the date
November 5 – 6, 2010

Themes in Neurorehabilitation:
Cerebral Palsy Across the Lifespan

Presented by Maryland Center for Developmental Disabilities at Kennedy Krieger Institute and Kinetic Connections Johns Hopkins Medical Institutions • Baltimore, Maryland

Learn about the current best evidence-based practices for the treatment of medical, rehabilitative and psychosocial conditions that may occur in individuals with cerebral palsy at each state of life.

Keynote Speakers:
Janice Brunstrom-Hernandez, MD, Director, Pediatric Neurology Cerebral Palsy Center, St. Louis Children’s Hospital
Mindy Aisen, MD, CEO, Cerebral Palsy International Research Foundation

Registration information available in mid-May at www.resourcefinder.kennedykrieger.org
Who Should Attend:
Community program coordinators, counselors, educators, nurses, nutritionists/ dietitians, occupational therapists, parents and individuals with disabilities, physical therapists, psychologists, social workers, and speech & language pathologists.

Conference Sessions:

November 5
• Augmentative Communication Strategies

• Coping with a New Diagnosis/Accessing Care

• Education Issues

• Neuroimaging as a Guide to Diagnosis

• Nutrition/Feeding

• Strategic Planning for Therapeutic Interventions

November 6
• Community Integration/Socialization

• Disability & Sexuality

• Quality of Life

• Transition Issues

• Wellness/Medicine/Aging

Cable News Network (CNN) will be airing a story on the use of Robotics in both Cerebral Palsy and Stroke Rehabilitation. They interviewed Dr. Joelle Mast of Blythedale Children’s Hospital who is a past recipient of a research award from CPIRF to study the effectiveness of robot-assisted physical therapy in children with hemiplegic Cerebral Palsy.
The piece will be aired at the following times

CNN
Saturday, March 27 2-3PM
Sunday March 28 6-7PM
Wednesday March 31 6-7AM

CNN Headline News
Saturday, March 27 2:30-3PM
Sunday March 28 2:30-3PM

CPIRF is pleased to co-sponsor the 2010 Association of Children’s Prosthetic-Orthotic Clinics’ Annual Meeting at the Sheraton Sand Key Resort in Clearwater Beach, FL June 2^nd through the 5^th. Click here for meeting agenda. CPIRF’s Medical Director Dr. Mindy Aisen and orthopedic surgeons Drs. Laura Tosi and Kevin Murphy will hold a symposium entitled ‘ . Cerebral Palsy: Pediatric to Adult Transition’ on Friday June 5^th.

To learn more about the Association of Children’s Prosthetic-Orthotic Clinics (ACPOC) go to their website at www.acpoc.org

If you want to attend the upcoming annual meeting call (847)698-1937 or email at raymond@aaos.org

The American Academy of Neurology, an international professional association of more than 21,000 neurologists and neuroscience professionals, has just published new clinical guidelines on the treatment of spasticity in children and adolescents with Cerebral Palsy.  Click here to read their press release. These guidelines were developed after a rigorous review of the medical literature by top neurologists and scientists. Basically the consensus of the workgroup found that Botulinum Toxin A is an effective treatment for spasticity in children and adolescents with Cerebral Palsy but does carry some risk. The link to the new clinical  guidelines and tools is:

http://www.aan.com/guidelines

You can find their full press release here.

Many thanks to Heather Fawcett for organizing both a 5k and a kids race on September 9, 2009 at Sunset Park in Pennsylvania to benefit CPIRF.  Heather belongs to a National community service organization called Alpha Phi Omega that helped her put the event together. In all, they had 45 runners and raised over $300 for cerebral palsy research.

Heather, a student at Penn State, has a form of mild cerebral palsy.  She is very grateful to have had wonderful doctors, physical therapists, and family members who have supported her  throughout her life. When she was younger, she  had trouble doing even fairly simple tasks, but thanks to her family and therapists, she completed her first marathon last year in a time of 4 hours and 26 minutes (10:13 min per mile pace).

In Heather ‘s words, ‘I just feel so blessed for all that has been given to me and I know that not all other individuals with Cerebral Palsy are as lucky as I am. That’s why I hope that someday we will be able to make the lives of those with much more severe disability be easier, so they can lead normal, happy lives. “

You too, can help promote CP awareness, by getting involved and organizing local events to benefit CP research and educational activities.  CPIRF can help by providing T-shirts, pamphlets and educational materials.

You can view the Stride for Cerebral Palsy Awareness Race website by going to: http://sites.google.com/site/strideforawareness or just www.stride4awareness.org

The New York Times recently ran a story of a 31 year –old with cerebral palsy and his remarkable response to dance lessons and muscle relaxation techniques. AOL picked up the story and interviewed our Medical Director, Dr. Mindy Aisen, about its potential usefulness as a therapy to improve function in individuals with CP.  Now, Good Morning America has done a piece on this same remarkable story of Gregg Mozgla and how he reduced his symptoms of cerebral palsy through ballet.

http://www.aolhealth.com/condition-center/cerebral-palsy/overcoming-cerebral-palsy

Here’s a link to the GMA piece: http://abcnews.go.com/video/playerIndex?id=9600885

Is Dance an Effective Therapy for Cerebral Palsy?

By Justine van der Leun

Gregg Mozgala, a 31-year-old actor, used to feel inhibited by his cerebral palsy, a neurological condition that occurs when a child’s brain is damaged before the age of two and afflicts a million Americans — most often in the form of poor coordination, weak muscles, and compromised posture. But with a load of determination and the help of an unconventional choreographer, Mozgala is now set to star in an hour-long dance piece in New York City. “I have felt things that I felt were completely closed off to me for the last 30 years,” Mozgala told The New York Times. “The amount of sensation that comes through the work has been totally unexpected and is really quite wonderful.” While there is no cure for cerebral palsy, Mozgala’s success suggests that a change in approach to the condition can translate into a change in the lives and capabilities of sufferers.

Mozgala’s journey began in 2008 when he met choreographer Tamar Rogoff (pictured above doing body work with Mozgala). After seeing Mozgala play the lead in “Romeo and Juliet,” produced by Theater Breaking Through Barriers, a group composed of disabled and non-disabled actors, Rogoff was inspired to create a dance piece for him. At first, both Mozgala and Rogoff imagined a 10-minute performance: Mozgala, who then walked on his toes with his upper body thrown back, assumed he could not manage much more, and Rogoff figured she would create some basic choreography for him. But as they began to work together, her imagination and his capacities began to expand.

Rogoff, who knew little about cerebral palsy, taught Mozgala techniques to release muscular tension. She helped him locate areas of his body over which he had previously exercised no control. In agonizing and illuminating sessions, they worked together to increase his range of movement, employing dance and stretching techniques, and finding his true physical limits. Soon enough, Mozgala was able to stand up straight, to place both feet on the floor, as well as to feel his Achilles tendon, which he had never before done. He called these revelations “eureka moments” in the New York Times interview.

“There are pre-existing structures in the brain that are very receptive to music, rhythm, and moving to music, which is why at a rock concert, everyone is swaying,” explained Mindy L. Aisen, MD, medical director of The Cerebral Palsy International Research Foundation. “The innate pleasure we get from music acted as a reinforcement for getting [Mozgala's] body re-engaged and for forging new pathways in his brain.”

Mozgala had been to physical therapists for over a decade, but his dance training was different: While before, the therapists had moved his body for him, now he learned how to move his own body. Rogoff identified some of the physical patterns he had been stuck in and gave him specific instructions on how to overcome them, both in the studio and out. His daily life has changed: His balance and strength are so improved that he rarely falls; his gait is steadier, and he is subject to fewer stares on the street. Most important, he no longer feels mentally constrained by cerebral palsy. As he told the Times: “Everybody told me there was nothing I could do,” he said. “That’s just what you hear, from the time you’re five to adulthood. Tamar gave me an option.”

According to Aisen, Mozgala’s story supports an open-minded, patient-centered approach to Cerebral Palsy and other neurological impairments. CPIRF is considering funding a dance therapy program, and at their Washington, D.C. Center, they have begun to use robotics and fun, motivational virtual games to help children use afflicted muscles. “Just as musicians have to practice to hone a motor skill, a brain that’s never had a chance to develop in areas needs the opportunity,” says Aisen. “We think we can help anyone with cerebral palsy reshape their nervous system in a way. It’s not a cure, but it is going towards a cure.”