Mass General researchers are exploring the genetic mechanisms of ALS in their quest to develop effective treatments.

Clotilde Lagier-Tourenne MD, PhD, and colleagues in her lab at Massachusetts General Hospital are exploring the development and progression of amyotrophic lateral sclerosis (ALS) in an effort to develop effective therapies to slow, halt and ultimately, prevent this disease, which currently has no cure.

ALS, the disease made famous by the Ice Bucket Challenge in 2014, causes progressive deterioration of the nerve cells in the brain and spinal cord that control body movement.

Collaborating across neurological disease areas is saving both time and money.

Dr. Lagier-Tourenne is a researcher at The MassGeneral Institute for Neurodegenerative Disease (MIND).

The recent discovery of a connection between ALS and fronto-temporal dementia (FTD), evidenced by progressive paralysis in ALS and language and behavioral dysfunction in FTD, has allowed a more focused investigation of the points of overlap to identify effective therapeutic targets.

Collaborating across neurological disease areas is saving both time and money as MIND researchers bring their expertise to bear on the challenges that occur when neurons malfunction.

Developing Therapeutic ALS Strategies

By analyzing skin biopsies from individuals with ALS, the Mass General researchers have been able to screen more than 5,000 drugs for potential therapeutic value. They are also able to differentiate skin cells into neurons and generate mouse models of ALS to develop therapeutic strategies.

The researchers are most hopeful about the potential impact of antisense oligonucleotides (ASO).

The researchers are most hopeful about the potential impact of antisense oligonucleotides (ASO), which turn off the action of the gene that makes it malfunction.

The first clinical trial to determine the safety of ASO therapy in patients was led by Merit Cudkowicz, MD, chief of Neurology at Mass General, with the help of academic-industry partnerships. An ASO therapy to treat a pediatric form of motor neuron disease was approved by the FDA in December, 2016.

To learn more about how you can support ALS research at Mass General, please contact us

MIND: Collaborating for a Cure

The MassGeneral Institute for Neurodegenerative Disease (MIND) is committed to finding treatments and cures to improve the lives of patients with neurodegenerative disorders including Alzheimer’s, Amyotrophic Lateral Sclerosis (ALS), Huntington’s and Parkinson’s disease.

Founded in 2001, MIND unites scientists and physician-scientists across disciplines with the common goal of translating laboratory findings into therapies that can reach patients in the clinic. Researchers in MIND laboratories work collaboratively to achieve the most promising results.

To learn more about MIND, please visit our website.