Disease Tab: Neuro

A Phase I Open Label Study to Evaluate the Safety and Tolerability of ISP-001 in Patients with Mucopolysaccharidosis Type 1

FIH Study of NRTX-1001 Neural Cell Therapy in Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy

The current therapies for drug-resistant epilepsy are only partially effective and have serious drawbacks.   One treatment that can significantly reduce seizure frequency is the removal of the affected part of the brain using surgical or laser ablation methods.  However, not surprisingly, removal of brain tissue can cause serious, irreversible damage, such as effects on memory, […]

CNS10-NPC-GDNF delivered into the motor cortex for the treatment of ALS

ALS is a neurodegenerative disease that results in the death of nerve cells in the brain and spinal cord, causing the muscles in the body to gradually weaken, leading to loss of limb function, difficulty breathing, paralysis, and eventually death.  There are medications that can slow down the progression of ALS, but unfortunately there is […]

Safety and Tolerability Study of Neural Stem Cells (NR1) in Subjects with Chronic Ischemic Subcortical Stroke

Gary Steinberg, M.D., Ph.D., and his team at Stanford University are conducting a clinical trial to test a therapy for motor disabilities caused by chronic ischemic stroke.  While “clot busting” therapies can treat strokes immediately after they occur (acute strokes), these treatments can only be given within a few hours of the initial injury.  There […]

The CuRe Trial: Cellular Therapy for In Utero Myelomeningocele Repair

UC Davis is conducting a clinical trial for in utero repair of myelomeningocele (MMC), the most severe form of spina bifida.  MMC is a birth defect that occurs due to incomplete closure of the developing spinal cord, resulting in neurological damage to the exposed cord.  This damage leads to lifelong lower body paralysis, and bladder and […]

A Phase 1b Safety Study for MRI guided delivery of AAV2-GDNF for the treatment of Parkinson’s disease

Parkinson’s Disease is a neurodegenerative disease that is thought to be the outcome of the gradual breakdown of dopaminergic neurons in the brain, which are a type of cell that produces a chemical in ones’ brain known as dopamine.  This decrease in the brain dopamine content can result in symptoms such as uncontrollable shaking of […]

MSC engineered to produce BDNF for the treatment of Huntington’s disease

A team at UC Davis plans to use bone marrow derived mesenchymal stem cells to deliver a growth factor, called BDNF, to patients’ brains in order to reduce the death of nerve cells that occurs in Huntington’s Disease (HD). In preparation for such a clinical trial study, the team completed a CIRM-funded observational trial in […]

A Phase I/IIa Dose Escalation Safety Study of AST-OPC1 in Patients with Cervical Sensorimotor Complete Spinal Cord Injury

Up to 12,000 Americans suffer a spinal cord injury (SCI) each year. This condition leads to a high level of permanent disability and decreased life expectancy. Currently, there are no approved therapies for patients with SCI. Asterias Biotherapeutics uses cells derived from embryonic stem cells to heal the spinal cord at the site of injury. They […]

A Double-Blind, Controlled Ph 2b Study of the Safety and Efficacy of Modified Stem Cells in Patients with Chronic Motor Deficit from Ischemic Stroke

Stroke is a major cause of long-term disability and there are no proven medical treatments for chronic stroke. SanBio is carrying out a Phase 2 clinical trial using mesenchymal stem cells (MSCs) to help people suffering from chronic disability following a stroke. The MSCs are isolated from the bone marrow of healthy adult donors, and […]

Progenitor Cells Secreting GDNF for the Treatment of ALS

ALS is a devastating neurodegenerative disease with no cure that specifically affect a patient’s motor neurons in the brain. A team at Cedars-Sinai is transplanting millions of genetically engineered stem cells into patients with ALS. When transplanted into the patient spinal cord, these cells become astrocytes, the support cells that keep nerve cells functioning. Due to […]