Navigation Links
Researcher test first-in-class compound for neuroprotection, hope of stopping MS disease progression
Date:11/20/2013

A $500,000 drug development grant from the National Multiple Sclerosis Society (NMSS) was awarded to a partnership between a multiple sclerosis research team at the Icahn School of Medicine at Mount Sinai and Karyopharm Therapeutics Inc., a clinical stage pharmaceutical company. Dr. Patrizia Casaccia, MD, PhD, Professor in the Departments of Neuroscience and Genetics and Genomics, at Icahn School of Medicine at Mount Sinai, will be the academic lead. She will test the effectiveness of a novel Karyopharm compound that can be orally administered and aimed at stopping the progressive phase of the disease. With the 14-month grant, Dr. Casaccia also hopes to gather information that will help design future clinical trials for MS treatments.

Karyopharm specializes in the synthesis of Selective Inhibitors of Nuclear Export, also known as SINE compounds. These compounds are thought to prevent the cause of irreversible damage to neurons, by blocking the early stages of neurodegeneration. Dr. Casaccia's laboratory first identified nuclear export as an important mechanism related to the initial events occurring in neurons and eventually leading to neurodegeneration. As inhibitors, these novel compounds target the nucleus in neurons, and block the accumulation of toxic substances in the axons. Axons are coated with myelin, and they can be damaged because myelin is destroyed or because they can be directly attacked by toxic factors that accumulate during the MS disease process. Neurodegenerative symptoms result from loss of myelin. Electrical signals are transmitted from the cell body of the neuron down an axon to other nerve cells, muscles, and other cells. Signal transmission slows down and progressive disability results from damage to the axons and loss of neurons, due to neurodegeneration.

Dr. Casaccia underscored the new strategy in MS drug development. "What's unique about this work is that SINE compounds target and prevent nuclear export, which is critically important for the neurodegenerative phase of the disease," she said. Preliminary experiments in Dr. Casaccia's laboratory have been encouraging. In mouse models, oral administration of the new compound to mice with paralysis of the tail and hindlimb, allowed them to walk again.

"The idea of rebuilding the nervous system and protecting it from ongoing MS damage was just a dream a few years ago," said Timothy Coetzee, Chief Advocacy, Services and Research Officer at the National MS Society. "Now, because of efforts by the research community as well as focused investments by the Society, we can see a future where people with MS will have treatments that could restore what's been lost."

In partnering with Karyopharm Therapeutics Inc., Dr. Casaccia will test these oral compounds in preclinical models and unravel their mechanism of action. The work would not be possible if the National MS Society did not invest $500,000 with Karyopharm through Fast Forward, as part of a comprehensive approach to MS research and treatment focusing on accelerating commercial development of promising research discoveries.,

"We look forward to collaborating with Dr. Casaccia, who has dedicated herself to advancing research in multiple sclerosis and other important diseases," said Karyopharm Founder, Chief Scientific Officer, and President of Research and Development, Sharon Shacham, PhD, MBA.

Fred Lublin, MD, Saunders Family Professor of Neurology and the Director of the Corinne Goldsmith Dickinson Center for Multiple Sclerosis at Mount Sinai Medical Center also applauded this research. "Developing novel approaches to treating the neurodegenerative component of MS is critically important for our efforts at halting this disease and then reversing the damage. Existing medications for MS only aim to reduce the number of relapses. They are not restorative to the nervous system."


'/>"/>

Contact: Press Office
newsmedia@mssm.edu
212-241-9200
The Mount Sinai Hospital / Mount Sinai School of Medicine
Source:Eurekalert

Related medicine news :

1. NSU researchers receive $4.1 million grant from DOD to investigate Gulf War illness
2. Bedroom access to screen-based media may contribute to sleep problems in boys with autism, MU researchers find
3. Researchers Identify a New Genetic Risk Factor for Severe Psychiatric Illness
4. New Mobile Reporting Platform from OneWhitePixel Links Scientific Researchers With Field Reporters
5. Researchers find protein that regulates the burning of body fat
6. Water and Eye Health Researcher Offers Tips on Coping with Winter “Flu Eyes”
7. Johns Hopkins heart researchers develop formula to better calculate bad cholesterol in patients
8. U of M researchers find HIV protein may impact neurocognitive impairment in infected patients
9. Nanotech researchers 2-step method shows promise in fighting pancreatic cancer
10. Company co-founded by UH researcher wins Nanomedicine Award
11. Get Out and Play! Radio Flyer is Searching for Innovative New Ride-On Toys for Kids to Inspire Outdoor Play as Researchers Recommend Limiting Kids' Screen Time
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:6/25/2016)... ... June 25, 2016 , ... Austin residents seeking Mohs surgery services, can ... Surgery and to Dr. Russell Peckham for medical and surgical dermatology. , Dr. Dorsey ... cancer. The selective fellowship in Mohs Micrographic Surgery completed by Dr. Dorsey was under ...
(Date:6/25/2016)... ... June 25, 2016 , ... ... non-athletes recover from injury. Recently, he has implemented orthobiologic procedures as a method ... —Johnson is one of the first doctors to perform the treatment. Orthobiologics are ...
(Date:6/24/2016)... (PRWEB) , ... June 24, 2016 , ... June 19, ... the dangers associated with chronic pain and the benefits of holistic treatments, Serenity ... who are suffering with Sickle Cell Disease. , Sickle Cell Disease (SCD) is a ...
(Date:6/24/2016)... ... ... Comfort Keepers® of San Diego, CA is excited to announce they are ... drive cancer patients to and from their cancer treatments. Comfort Keepers provides quality ... and ongoing independence. Getting to and from medical treatments is one of the ...
(Date:6/24/2016)... ... June 24, 2016 , ... Puradigm® & Innovative ... has initiated cultivation and processing operations at its production facility, and opened its ... Puradigm is the manufacturer of a complete system of proactive air and surface ...
Breaking Medicine News(10 mins):
(Date:6/24/2016)... -- Dublin ... the " Global Markets for Spectroscopy Equipment" ... This report focuses on the global market ... its applications in various applications. The report deals with ... main industries: pharmaceutical and biotechnology, food and beverage, and ...
(Date:6/24/2016)... Calif. , June 24, 2016  American Respiratory Labs (ARL), ... is now able to perform sophisticated lung assessments in patients, homes, ... , Inc. Patients are no longer limited to ... EasyOne PRO ® , ARL patients like Jeanne R. of ... in the comfort of her own home. ...
(Date:6/24/2016)... , Belgium , June ... MKT: VNRX), today announced the appointment of Dr. ... of Directors as a Non-Executive Director, effective June ... Company,s Audit, Compensation and Nominations and Governance Committees.  ... Dr. Futcher will provide independent expertise and strategic ...
Breaking Medicine Technology: