The ALS Therapy Development Institute Partners with Regenesance BV to Investigate Potential Treatments for Lou Gehrig's Disease

The ALS Therapy Development Institute Partners with Regenesance BV to Investigate Potential Treatments for Lou Gehrig's Disease

Complement Pathway Modulation at Center of Collaboration

CAMBRIDGE, Mass. and AMSTERDAM, May 7, 2012 /PRNewswire-USNewswire/ -- The ALS Therapy Development Institute (ALS TDI) announced today that it has entered into a partnership with Regenesance to investigate a potential treatment for ALS (aka Motor Neurone or Lou Gehrig's disease).  The collaboration will enable ALS TDI, the world's largest ALS drug development center, to screen novel compounds against a crucial pathway implicated in the development and progression of ALS.

"This partnership is an example of how important collaboration is to defeat a disease like ALS," said Steve Perrin, Ph.D., CEO & CSO of ALS TDI.  "We are excited to be working with Regenesance on this program and look forward to the results."

The compounds developed by Regenesance target a highly specific aspect of the immune system, called the complement pathway.  The complement pathway is a central part of the immune system and activation occurs in neurodegenerative disorders.  Regenesance has shown that complement activation can cause nerve damage and may profoundly impair neuroregeneration.  ALS TDI will use tightly controlled preclinical models of ALS to evaluate whether the compounds developed by Regenesance prevent nerve damage and have an effect on the onset or progression of ALS.

"We have shown in preclinical tests that inhibition of the complement pathway accelerates nerve regeneration and we are pleased to work together with ALS TDI to evaluate our compounds for potential treatment of this disease as they are world renowned experts in this field," said Prof. Frank Baas, CSO of Regenesance.

About ALS
Amyotrophic lateral sclerosis (ALS, Lou Gehrig's disease) is a progressive neurodegenerative disease that leads to paralysis, due to the death of motor neurons in the spinal cord and brain. There is no known cause, cure or effective treatment for the disease. About 5,000 people in the US are diagnosed with ALS each year; the incidence is similar to Multiple Sclerosis. However, with no effective treatment and an aggressive and rapid disease progression, the average patient survives only 2-5 years following their diagnosis. There are about 30,000 US citizens diagnosed with the disease at any given time. The worldwide population of ALS patients is estimated at 400,000.

About the ALS Therapy Development Institute (ALS TDI)
The mission of the ALS Therapy Development Institute (ALS TDI) is to develop effective therapeutics that slow or stop amyotrophic lateral sclerosis (ALS, Lou Gehrig's disease) as soon as possible for patients today. Focused on meeting this urgent unmet medical need, ALS TDI executes a robust target discovery program, while simultaneously operating the world's largest efforts to preclinically validate potential therapeutics; including a pipeline of dozens of small molecules, protein biologics, gene therapies and cell-based constructs. The world's first nonprofit biotech institute, ALS TDI has developed an industrial-scale platform, employs 30 professional scientists and evaluates dozens of potential therapeutics each year. Built by and for patients, the Cambridge, Massachusetts based research institute collaborates with leaders in both academia and industry to accelerate ALS therapeutic development, including Biogen Idec, UCB, Aestus Therapeutics, MDA and RGK Foundation. For more information, please visit us online at

About Regenesance BV
Regenesance BV is a biotech company located in Amsterdam, The Netherlands, focused on creation and development of innovative therapeutics for neurodegenerative diseases and acute nerve trauma.  The Company has a lead compound in preclinical development for chronic neurological indications as Myasthenia Gravis and Multiple Sclerosis, and has a research program for treatment of acute neurological disorders such as Guillain Barre and Traumatic Brain Injury. The Company focuses on clinical development of its products in orphan neurological indications. For more information, see

Media Contacts:  Mari Sullivan, ALS Therapy Development Institute, Public Relations Manager, [email protected], 617-441-7220
Dr. Manja Bouman, Regenesance BV, Chief Executive Officer, [email protected], +31 6 236 09002

SOURCE ALS Therapy Development Institute