The U.S. Food and Drug Administration (FDA) has approved BrainStorm Cell Therapeutics‘ request to open a Phase 2 clinical trial testing the safety and effectiveness of its proprietary NurOwn mesenchymal stem cell (MSC) treatment in progressive multiple sclerosis (MS) patients.
The request was in the form of Investigational New Drug (IND) application, and BrainStorm plans to launch the study in early 2019.
“We are excited about the launch of early testing of this cell-based approach to treating progressive MS, for which there are few treatment options,” Bruce Bebo, executive vice president of research at the National MS Society, said in a press release. “We look forward to better understanding the potential benefits and safety of this and other cell-based therapies for MS.”
Stem cells have the ability to develop into different cell types, and to replace damaged or dying cells in the body. There are different types of stem cells, and therapies based on these cells are being investigated for several diseases, including MS.
NurOwn cells are MSCs obtained from a patient’s bone marrow — the spongy tissue inside bones — and grown in the lab under patented conditions in order to induce the cells to secrete high levels of neurotrophic factors (NTF), molecules that support the growth, survival, and differentiation of neurons. These MSC-NTF cells are then injected back into the patient intravenously or into the spinal canal.
Experiments with mice suggest that injected NurOwn stem cells do not themselves replace damaged cells. Instead, the lab treatment customizes MSCs, converting them into biological factories that secrete high levels of NTFs, which in turn encourage neural stem cells to replace damaged cells.
NTFs may also exert a therapeutic effect in progressive MS by other mechanisms. They can improve neuronal survival following injury, protecting cells from oxidative stress (an imbalance between the body’s production of potentially harmful reactive oxygen species and its ability to contain them), and they can modulate the immune response, shifting it from proinflammatory to anti-inflammatory, dampening the autoimmune damage done to myelin (the protective cover of neurons that is destroyed in MS).
Currently, no autologous cell therapies are FDA-approved to treat MS or other neurological diseases.
“We are excited to participate in this Phase 2 clinical trial and hope that this innovative cell therapy approach leads to a new treatment option for patients with progressive MS,” said Fred Lublin, MD, director of the Corinne Goldsmith Dickinson Center for Multiple Sclerosis at Mount Sinai Medical Center. “We are particularly pleased to see a scientifically rigorous approach to advance the science of stem cell therapeutics.”
NurOwn cell therapy is currently being evaluated for safety and efficacy in a Phase 3 clinical trial (NCT03280056) in up to 200 people with amyotrophic lateral sclerosis (ALS) at sites in the U.S. This double-blinded and placebo-controlled study, which is recruiting eligible adults, administers the therapy three times bi-monthly via lumbar puncture (intrathecal injection) and is expected finish in July 2019.
The NurOwn technology was developed in the laboratories of researchers at Tel Aviv University.