According to European Pharmaceutical Review, results from a Phase 1/2a clinical trial support the use of AstroRx for patients with amyotrophic lateral sclerosis (ALS). The clinical trial, headed by Kadimastem Ltd. (“Kadimastem”), analyzed the safety, efficacy, and tolerability of the astrocyte cell treatment.
AstroRX
Designed by Kadimastem, AstroRx is a cell therapy which uses astrocytes derived from human embryonic stem cells (hESC). Neuroscientifically Challenged defines astrocytes as:
star-shaped glial cells that have a number of functions, including support of the blood-brain barrier, provision of nutrients to neurons, repair to nervous tissue following injury, and facilitation of neurotransmission.
Basically, astrocytes support healthy nervous system function and may help to prevent neurodegeneration. In this case, AstroRx works to address cellular malfunction, prevent ALS disease progression, improve quality of life (QOL), and help patients to live longer. The FDA granted Orphan Drug designation to AstroRx for the treatment of ALS.
During the clinical trial, researchers evaluated the safety, efficacy, and tolerability of AstroRx in Cohort B. 5 patients enrolled in the trial. These patients received 250×106 AstroRx cells and also participated in a 6-month follow-up. Cohort A, which had undergone treatment earlier in 2020, received 100×106 cells. In Cohort B, AstroRx significantly reduced disease progression by up to 45%. These results were sustained over the follow-up period. Additionally, the treatment was found to be relatively safe and well-tolerated, with no serious adverse reactions reported. This data falls in line with what researchers discovered using Cohort A.
Now, Kadimastem hopes to continue the clinical development of AstroRx and hold additional clinical trials moving forward.
Amyotrophic Lateral Sclerosis (ALS)
Also known as Lou Gehrig’s disease, amyotrophic lateral sclerosis (ALS) is a progressive neurological disease which causes brain stem, spinal cord, and brain nerve cell death. While patients with familial ALS have specific genetic mutations leading to their condition, approximately 90-95% of ALS diagnoses have unknown causes. As the nerve cells die, muscles begin to weaken, causing loss of voluntary movement. In many cases, ALS is fatal as patients lose their ability to breathe. Generally, ALS is more common in white males between ages 60 and 69. Symptoms vary, but may include:
- Anxiety and depression
- Frequent tripping and falling
- Difficulty speaking or swallowing
- Changes in speech (slurring, slowed speech)
- Muscle pain, cramping, and weakness
- Poor posture
- Difficulty walking or performing small movements
