In a recent press release, Catalent and BrainStorm Cell Therapeutics Inc. (“BrainStorm”) announced a partnership to manufacture NurOwn for patients with amyotrophic lateral sclerosis (ALS). For over 85 years, Catalent has worked to develop and manufacture health-related products, from drugs to gene therapy. Meanwhile, BrainStorm develops autologous stem cell therapies for patients with neurodegenerative disorders. Together, the pair is uniquely poised to bring NurOwn to market and improve patient outcomes.

NurOwn

Developed by BrainStorm, NurOwn is a proprietary platform that can create and produce autologous MSC-NTF cells. Usually, with stem cell therapies, you will hear them be described as either autologous or allogeneic. But what is the difference? The Dana-Farber Cancer Institute explains that:

An autologous transplant uses a person’s own stem cells. An allogeneic transplant uses stem cells from a donor whose human leukocyte antigens (HLA) are acceptable matches to the patient’s.

Autologous transplants require doctors to collect and freeze stem cells directly from patients. Next, the cells are treated outside of the body. Finally, the stem cells are returned to the body, prompting healing. In this case, NurOwn uses mesenchymal stem cells (MSCs). MSCs are usually derived from bone marrow. EuroStemCell explains that MSCs:

are ‘multipotent’, meaning they can produce more than one type of specialized cell of the body, but not all types. For example, they can differentiate − or specialize  −  into cartilage cells (chondrocytes), bone cells (osteoblasts) and fat cells (adipocytes)

NurOwn prompts MSCs to secrete neurotrophic factors (NTFs), small proteins that help protect neuron growth, development, and survival. Once returned to the body, NurOwn helps slow disease progression and reduce neuroinflammation and damage. Currently, autologous MSC-NTF cells are being evaluated in a Phase 2 and Phase 3 clinical trial. This treatment has also received Fast Track, Orphan Drug, and Orphan Medicinal Product designations.

Amyotrophic Lateral Sclerosis (ALS)

An estimated 5-10% of all cases of amyotrophic lateral sclerosis (ALS) are familial, or inherited via genetic mutations. However, that means 90-95% of diagnoses still exist without a known cause. Researchers believe that frontotemporal dementia or toxin exposure may contribute to the development of ALS, which is also known as Lou-Gehrig’s disease. ALS is a progressive neurodegenerative disorder. It is characterized by nerve cell death in the brain, spinal cord, and brain stem.

As ALS progresses, patients experience increasing muscle weakness and a loss of voluntary movement control. Generally, ALS is more prevalent in white males ages 60 to 69 than in other groups. ALS affects males more than females. Symptoms vary. However, these may include:

• Anxiety and depression
• Muscle cramps, stiffness, and rigidity
• Slow or slurred speech
• Difficulty speaking or swallowing
• Weakness in the arms, hands, and legs
• Poor posture
• Frequent tripping or falling
• Difficulty with daily activities or movement
• Inability to move muscles

Learn more about ALS.