Protein substitution therapy for neurodevelopmental diseases.

Scientists at the Georg-August-Universität Göttingen Stiftung Öffentlichen Rechts, Universitätsmedizin, developed a modified MeCP2 protein. This biotechnologically produced protein, which might be used for a substitution therapy of the RETT-Syndrome, crosses the blood-brain-barrier. Thus it reaches the brain as the therapeutical target. First preliminary results could show the success of this strategy in a mice model. A patent application for the novel protein and its uses has been applied for. The corresponding IP rights of the University of Göttingen have been successfully and exclusively licensed to the Swiss biotechnological company IPT Pharma AG, Zürich. The University receives financial payments, which will benefit the scientists as well as their research departments. The agreement has been negotiated by the MBM ScienceBridge GmbH, the technology transfer organization of the Georg-August-Universität Göttingen. The RETT-syndrome is the second most common cause of mental retardation. At least 90% of the cases are due to mutations in a specific gene. The consequence is an inability to produce enough functional MeCP2 protein and the subsequent abnormal brain development is the cause for the RETT-syndrome. The cause and course of this disease including brain atrophy and progressive dementia is yet not fully understood. Clinical manifestations include decelerating head growth, emotional withdrawal, loss of learned skills, truncal and gait ataxia/apraxia, stereotypical hand movements, dementia, and motor deterioration. Young girls show first symptoms after the 18. month. The course of the disease is progressive and results in a lifelong disability and in need of care. Alone in Europe and in the USA there are aprox. 1000 newborns yearly having RETT-syndrome. No successful treatment is yet known for patients suffering the RETT-syndrome. In the last years amazing therapeutical success could be achieved through novel protein therapies for a few genetical diseases. Hence with a biotechnologically produced modified MeCP2 protein a therapeutical or preventive treatment for neurodevelopmental diseases could be tested in coming years, particularly for the RETT-syndrome. Further diseases might include attention deficit disorder (ADD) or the Angelman-syndrome. Due to its expert knowledge with recombinant proteins and development of biopharmaceuticals the licensee IPT Pharma AG selected PharmedArtis GmbH from Aachen as its technological partner. Moreover, the PharmedArtis will provide IPT Pharma its experience developing novel immunotherapeutics for the treatment of cancer and inflammatory diseases, including support with regulatory affairs. The development, GMP-production, realization of the pre-clinical phase and the prearrangements for clinical phases has been assigned to the PharmedArtis. First relevant pre-clinical results are expected for end of 2009. A certified GMP-development of the protein is aimed for the first quarter of 2010. The Universitätsmedizin of the Georg-August-Universität Göttingen includes the school of medicine and its hospital. It is one of the leading educational and research centers for medical doctors in Germany and in Europe and one of the universities in Germany awarded with the title “University of Excellence” www.med.uni-goettingen.de MBM ScienceBridge GmbH is a fully owned subsidiary of the University of Göttingen and is the technology transfer organization for several German Universities and Research facilities. The IP portfolio managed by MBM ScienceBridge covers innovation in the areas of Life Sciences, Chemistry, Physics, Agriculture and Medical Technologies. www.sciencebridge.de / info@sciencebridge.de / +49-(0)551-30724151 The IPT Pharma AG which is located in Zürich/Switzerland is a recently founded biotechnological company. IPT Pharma develops innovative therapies based on recombinant proteins for the therapy, prevention and diagnosis of neurodegenerative and neurodevelopmental diseases. The leading project of IPT Pharma focuses on the development of a protein substitution therapy for the treatment of RETT-syndrom, with the aim of a registration approval. www.ipt-pharma.ch / mail@ipt-pharma.ch PharmedArtis GmbH is a biotechnology company founded in 2004 by experienced scientists of the Fraunhofer Institute for Molecular Biology and Applied Ecology (IME) in Aachen, Germany. Being a specialist in recombinant technologies and Protein Production PharmedArtis is a one-stop-shop offering a wide range of services from gene to product including state-of-the-art GMP facilities. Its knowledge includes production systems in E. coli, yeasts, fungi, mammal cells and plant cells. Besides these services PharmedArtis develops based on four patent families novel immunotherapeutics for the treatment of cancer, inflammatory diseases and autoimmune diseases. www.pharmedartis.de / mail@pharmedartis.de Ihre Ansprechpartnerin: Barbara Fink, M.A. , Öffentlichkeitsarbeit, MBM ScienceBridge GmbH, Hans-Adolf-Krebs-Weg 1, 37077 Göttingen, info@sciencebridge.de Tel.: (0551) 30 724 151 Fax: (0551) 30 724 155

Learning about Music Therapy and Rett Syndrome

When there seems to be a developmental disorder in your child, you want to do everything you can to help them. This is why the link between music therapy and Rett Syndrome has gotten so much press. By combining this effective and side effect free therapy with more traditional developmental therapies, music therapy and Rett Syndrome are working hand in hand to help children move through the world in a more constructive way – even as their bodies are trying to defy them. Before you can learn about music therapy and Rett Syndrome, it will help to learn the basics of music therapy. In short, music therapy is the practice in which music is used as a tool to help a patient reach certain goals. For some patients, music might be used to encourage them during a particularly hard physical therapy session. For others, music might be used to help with mental disorders by allowing them to speak more freely about their troubles through lyrics, song sharing, etc. And in the case of Rett Syndrome, music seems to helping improve motor coordination, which can allow children to lead a more normal life. The other side of the music therapy and Rett Syndrome discussion is the disorder itself. Characterized by jerking movements of the body, Rett Syndrome is a developmental disorder the presents itself in early childhood. Sometimes it can be as sever as to not allow the child to move their muscles properly, while in other cases, the child may be in extreme pain in trying to move against what their body is trying to do. This is generally a gradual onset disorder and a child may appear to be developing at a normal rate for the first few years of their life. But as the syndrome progresses, the child may begin to show autistic signs – toe walking, coordination troubles, and breathing troubles. Caused by a genetic defect, Rett Syndrome does not have any cure as of yet, but it does appear to respond to concentrated therapy – like physical therapy. This can give the child more mobility while also helping them learn to use their body in a productive manner. By teaching children how to communicate through rhythm and music, the child can feel as though they are a part of their world as well as having the parent feel as though their child can 'speak' to them. These children who use music therapy also seem to be able to make decisions on their own as well as lessening the jerky movements that their body uses. The link between music therapy and Rett Syndrome progress is easy to see in the many research studies that have been done around the world.

Pediatric Lokomat® Walking Therapy

The Rehabilitation Institute of Chicago provides robot-assisted walking therapy using the Lokomat® to help pediatric and adult patients improve their ability to walk. Featured on the Today Show, RIC offers Pediatric Lokomat therapy, or robot-assisted walking therapy, for children with neurological conditions such as cerebral palsy (CP) to help improve their ability to walk. Robot-assisted walking therapy may not be appropriate for everyone, and certain medical conditions may prohibit your participation in this activity. To learn more about our program please review the information below and also see Questions & Answers About Robot-Assisted Walking Therapy About Lokomat TherapyRobot-assisted walking therapy is a form of physical therapy that uses a robotic device to help a person improve his or her ability to walk. The patient is suspended in a harness over a treadmill, and an exoskeleton robotic frame, attached by straps to the outside of the legs, moves the legs in a natural walking pattern. A computer controls the pace of walking and measures the body’s response to the movement. The pediatric model also has an interactive gaming interface which, through cartoon characters and challenges, motivates children and provides them instruction. Without the Lokomat, this type of physical therapy is conducted with the aid of two or more therapists who manually move the patient’s legs in a walking pattern. The strenuous nature and variability of the manual method can limit the frequency and duration of therapy. With the Lokomat, the robotic device does most of the heavy work, the pattern and pace are consistent throughout the session, and the exercise can be sustained over longer periods of time, making it more effective. The Science Behind the TreatmentRIC has been examining and tracking the effects of this therapy for adults for years and continues to focus on new research and clinical trials that provide more data on this treatment. In fact, RIC was the first hospital in the U.S. to obtain this technology and began clinical trials with the Lokomat® when it was first approved by the Food & Drug Administration in March 2002. In addition to its research studies, RIC now makes robot-assisted walking therapy available in the clinical setting for adult inpatients and outpatients as well. In an early study conducted in Europe [1] examining the use of Lokomat therapy for children with CP, results indicated an improvement in walking speed and in gross-motor function. The therapy was also rated “excellent” in providing motivation for carrying out therapy among a majority of children, while there was also a very high level of approval from the therapy team and parents. QuestionsWhat are the criteria for using the Pediatric Lokomat?All patients must undergo an evaluation by an RIC physician and physical therapist before being recommended for the Lokomat. The following information can be used as a guideline for patients and family members interested in RIC’s services: Age: Minimum age typically 4 years; Lokomat is available for older children and adults Diagnoses: Central gait impairment secondary to cerebral palsy or malformations, spinal cord injury, stroke, traumatic brain injury or Guillain-Barre’ Syndrome Over Ground Walking: Achieving over ground walking ability; presents some stepping pattern in supported gait Cognition: Ability to follow single step commands and to signal fear, pain or discomfort Femur Length: Femur length must be 21 centimeters Are there conditions that would prevent a patient from using the Lokomat (contraindications)The following issues may prevent a patient from being eligible for services in the Lokomat: Spasticity: Severe spasticity requires a trial test Lokomat session Cardiovascular issues: Cardiovascular instability or thromboembolic disease are contraindications Bone Issues: Fractures, osseous instabilities, osteoporosis, scoliosis greater than 20 degrees are contraindications Skin: Unhealed skin lesions in the trunk or lower extremities are contraindications Musculoskeletal issues: Weight-bearing restrictions are a contraindication Neurological comorbidities: Acute or progressive neurological disorders are a contraindication Ventilators: Ventilator dependency is a contraindication Intravenous Drip: Intravenous drip is a contraindication Behavior: Aggressive or self harming behavior is a contraindication How much does robot-assisted walking therapy cost?The cost of robot-assisted walking therapy will vary according to how often and how long it is required to achieve the maximum benefit for each individual. Many healthcare insurance providers cover robot-assisted walking therapy; you should consult your insurance provider for details. Where can I participate in robot-assisted walking therapy?RIC offers robot-assisted walking therapy only in the inpatient, outpatient and research centers of our flagship hospital, which is located at 345 E. Superior Street in Chicago. How often does a person need to participate in robot-assisted walking therapy?The effectiveness of robot-assisted walking therapy varies from person to person, so patients should be able to commit to a minimum of 60 minutes of therapy per day, three days per week, for four weeks. Periodic evaluations will be conducted to determine if more sessions would be helpful in achieving the maximum benefit. How do I schedule an appointment for a physician evaluation for robot-assisted walking therapy?Based upon the information supplied to RIC, clinical staff will make a preliminary determination of the appropriateness of robot-assisted walking therapy. You may call RIC at 1-800-354-REHAB (7342) or 1-312-238-1000 and ask for Pediatric Lokomat Patient Line to leave your information. You also may submit an request online for a physician consultation . You will be contacted to determine how best to proceed, which may include scheduling a physician evaluation. Are the results of robot-assisted walking therapy guaranteed?No, the results of robot-assisted walking therapy or any other type of therapy are not guaranteed. However, during the course of therapy, periodic evaluations will be conducted to measure improvement and determine if more sessions may help to achieve the maximum benefit. Read further questions and answers about Lokomat therapy . -------------------------------------------------------------------------------- [1] The Rehabilitation Centre for Children and Young People (Affoltern am Albis , Switzerland ); Dr. von Haunerschen Kinderspital (Munich ) and Hocoma, 2007 Source: Rehabilitation Institute of Chicago

Pediatric Lokomat® - Functional locomotion therapy for children

The Pediatric Lokomat allows intensive locomotion therapy now also for small children with cerebral palsy or other neurological disorders. The reduced size robotic gait orthosis offers the same benefits as the Lokomat for adults. Special harnesses and cuffs ensure precise fit also for small children

RIC Introduces Pediatric Lokomat Therapy to Enhance Ability to Walk in Neurologically Impaired Child

The Rehabilitation Institute of Chicago is now offering the pediatric version of the Lokomat robot-assisted walking therapy. 05.19.2009 — The Rehabilitation Institute of Chicago (RIC) announced it’s offering Pediatric Lokomat therapy, or robot-assisted walking therapy, for children with neurological conditions such as cerebral palsy(CP), to help improve their ability to walk and achieve their life goals and dreams. | Cerebral palsy is a neurological condition affecting the motor control portion of the brain often accompanied by secondary orthopaedic conditions affecting a child’s voluntary muscle activation, tone, reflexes, coordination, balance and ability to walk and be independent. CP is the leading cause of disability in children, and approximately 800,000 adults and children in the U.S. currently live with CP. An additional 10,000 babies born this year are estimated to develop this condition[2]. “RIC is committed to discovering new treatments that maximize the abilities of kids with cerebral palsy allowing them to achieve their goals in life,” said Deborah Gaebler-Spira, MD, director of the cerebral palsy program at RIC. “There is an immense need for research in this area, and RIC is dedicated to examining the vast opportunities and discovering new treatments that advance ability for those who are living with CP.” About Lokomat Therapy Robot-assisted walking therapy is a form of physical therapy that uses a robotic device to help a person improve his or her ability to walk. The patient is suspended in a harness over a treadmill, and an exoskeleton robotic frame, attached by straps to the outside of the legs, moves the legs in a natural walking pattern. A computer controls the pace of walking and measures the body’s response to the movement. The pediatric model also has an interactive gaming interface which, through cartoon characters and challenges, motivates children and provides them instruction. Currently, this type of physical therapy is conducted with the aid of two or more therapists who manually move the patient’s legs in a walking pattern. The strenuous nature and variability of the manual method can limit the frequency and duration of therapy. In the Lokomat, the robotic device does most of the heavy work, the pattern and pace are consistent throughout the session, and the exercise can be sustained over longer periods of time, making it more effective. The Science Behind the Treatment RIC has been examining and tracking the effects of this therapy for adults for years and continues to focus on new research and clinical trials that provide more data on this treatment. In fact, RIC was the first hospital in the U.S. to obtain this technology and began clinical trials with the Lokomat® when it was first approved by the Food & Drug Administration in March 2002. In addition to its research studies, RIC now makes robot-assisted walking therapy available in the clinical setting for adult inpatients and outpatients as well. In an early study conducted in Europe [3] examining the use of Lokomat therapy for children with CP, results indicated an improvement in walking speed and in gross-motor function. The therapy was also rated “excellent” in providing motivation for carrying out therapy among a majority of children, while there was also a very high level of approval from the therapy team and parents. Natalie’s Story Natalie Davis, 10, from Plano , Ill. , has one goal – to play soccer. Davis, who mainly uses a wheelchair to move around because of the weakness and abnormal coordination in her legs, has experienced ongoing treatment and therapy to manage the effects of CP her whole life. She has had regular physical therapy, botox treatments and even surgery to continuously improve the physical effects of CP. In January, Natalie underwent a surgery to lengthen her hamstrings and loosen the tension and spasticity in her leg muscles in hope of increasing her ability to walk. Once her casts were removed, Natalie was admitted to RIC as an inpatient for rehabilitation to strengthen her muscles, and improve her balance and endurance with the goal of getting her back on her feet. Natalie has been participating in Lokomat therapy three times a week to help retrain her step patterns and build her strength, coordination and endurance. It is believed that Lokomat therapy, in combination with her the surgical intervention and ongoing care, will improve her ability to walk. “As a parent, I want Natalie to work to her full potential,” said Christine Davis, Natalie’s mother. “The walker had become quite a challenge, so we hope that the Lokomat will help improve her walking. So far, we have seen an improvement in her leg strength, and her endurance has gotten better.” Candidates for Robot-Assisted Walking Therapy on the Lokomat Children with neurological conditions who are about four years old or older (or who have a femur length of at least 21 cm) and who are evaluated by a physician to determine if they are medically appropriate for robot-assisted walking therapy are eligible. If you are interested in having your child evaluated for Lokomat therapy, please call 1-312-238-6100 or make an appointment online for an evaluation with Dr. Deborah Gaebler-Spira or Dr. Gadi Revivo. About The Rehabilitation Institute of Chicago The Rehabilitation Institute of Chicago (RIC) is making a difference in the world for people with disabilities. RIC provides world-class care to patients from around the globe for a range of conditions from acute brain and spinal cord injury to chronic arthritis, pain and sports injuries. RIC, founded in 1954, has been designated the “#1 Rehabilitation Hospital in America” by U.S. News & World Report every year since 1991 and attributes its leading standard of care in part to its innovative research and discovery, particularly in the areas of bionic medicine, robotics, neural regeneration, pain care and better outcomes. RIC operates its 165-bed, Flagship hospital in downtown Chicago , as well as a network of 30 sites of care located throughout the city and surrounding suburbs that provide additional inpatient care, day rehabilitation and outpatient services. RIC also maintains strategic alliances with leading healthcare providers throughout the state of Illinois and Indiana. [1] U.S. News & World Report has ranked RIC the “#1 Rehabilitation Hospital ” every year since 1991. [2] Centers for Diseases Control (CDC), 2008 data [3] The Rehabilitation Centre for Children and Young People (Affoltern am Albis , Switzerland ); Dr. von Haunerschen Kinderspital (Munich ) and Hocoma, 2007 Robotics Trends would like to thank the Rehabilitation Institute of Chicago for permission to reprint this article. The original article can be found at http://www.ric.org/aboutus/mediacenter/press/2009/0210.aspx.

Rett syndrome Awareness meeting

An Awareness meeting for Indian Rett parents, Doctors, Scientists and all caregivers of Rett children who want to know more about Rett syndrome. Please let your Indian Friends and others Know about this and tell them to attend this for the cause of raising more awareness in India. Date and Venue of the meeting: Sunday, October 11, 2009 Time: 10:00am - 2:00pm Location: Lecture theater 2, Teaching block, 2nd floor All India Institute of Medical Sciences Ansari Nagar New Delhi-110029, India Phone: +919999343421 Email: info.rett@yahoo.com