St Jude is definitely one of the leaders in healthcare for children and our favorite! They are also finding new treatment, therapy and on the way to finding cures for most childhood diseases.  

About St. Jude Children's Research Hospital:

 St. Jude is leading the way the world understands, treats and defeats childhood cancer and other life-threatening diseases. 

• Families never receive a bill from St. Jude for treatment, travel, housing or food.
• We share our scientific discoveries with the world.

 What is ALS?

Amyotrophic Lateral Sclerosis, often called Lou Gehrig's disease, is tricky to understand and treat. It affects approximately one to two per 100,000 people in the United States annually. Nearly 95% of ALS cases are caused by unknown factors, with only around 5% being genetically inherited from parents. Although ALS usually strikes around age 50-60, it can affect people of any age. 

Amyotrophic Lateral Sclerosis (ALS) occurs when the body starts experiencing the death of neurons controlling voluntary muscles. ALS affects both upper and lower motor neurons, meaning patients with the disease begin to experience both involuntary spasticity and muscle weakening over time. This automatic spasticity results in worsening symptoms.

Stem cell therapy for Amyotrophic Lateral Sclerosis is a treatment approach that involves using stem cells, undifferentiated cells that can develop into various specialized cells, to replace or repair damaged or lost cells in the brain associated with ALS. The stem cells are administered into the body via intravenous injection and will find areas of inflammation and damage via homing mechanisms.

The primary mechanisms of stem cells are to reduce inflammation (including neuroinflammation) and modulate the immune system. Stem cell therapy aims to prevent disease progression for an extended period through immune modulation. A secondary benefit of stem cell treatment for ALS is to help prevent further loss of neurons to slow progression and improve motor symptoms, tremors, stiffness, and difficulty with movement.

‍Stem cell therapy for ALS

Mesenchymal stem cell therapy has already shown strong therapeutic potential in different clinical fields. In particular, mesenchymal stem cells (MSCs) may exert their action by differentiating toward a specific cell type or by releasing further growth and trophic factors. Clinical models have shown evidence that mesenchymal stem cells (MSCs) may represent a promising approach to treat ALS; MSC transplantation may delay the disease onset and progression and therefore increase lifespan.

Furthermore, the loss of motor neurons may also be reduced, resulting in a delay in motor function loss. The results obtained from preclinical studies have encouraged the administration of mesenchymal stem cells (MSC) in ALS patients. (3)

Stem cell therapy may be a viable treatment option for ALS. Stem cells may be feasible for sustaining and nurturing diseased motor neurons. (1)

According to a recent study conducted by Goutman et al.

“The premise of stem cell therapy for ALS aims to improve the diseased microenvironment. While stem cells are unable to replace diseased motor neurons directly, transplanted stem cells secrete neurotrophic factors and differentiate into supportive cells, such as astrocytes and microglia, generating a neuroprotective milieu that can slow degeneration of motor neurons.” (1) 

At present, there is no cure for ALS. However, there are several treatment options, including stem cell therapy, Riluzole, occupational therapy, and gene therapy, which may help manage symptoms and slow the progression of the disease, as well as ongoing research for potential cures and treatments.

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Nurown is a type of cellular therapy developed to treat ALS (Amyotrophic lateral sclerosis), also known as Lou Gehrig's disease. Nurown is a proprietary, autologous (self-donated) stem cell therapy comprising a patient's bone marrow-derived mesenchymal stem cells (MSCs) that are expanded in the lab and delivered back to the patient. The treatment is designed to slow the progression of ALS by promoting the survival and growth of motor neurons affected by the disease.

Nurown for ALS has been developed and manufactured by BrainStorm Cell Therapeutics. The therapy is still in the early stages of development and has undergone a Phase 2 clinical trial. The test results demonstrated a statistically significant improvement in the rate of decline in the primary endpoint of ALSFRS-R, a measure of ALS disease progression, as well as substantial improvements in secondary endpoints, including muscle strength and quality of life.

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