The first patient has been enrolled in a Phase 2 multicenter clinical trial testing the use of NurOwn cellular therapy to treat progressive multiple sclerosis (MS), BrainStorm Cell Therapeutics announced.
The open-label trial (NCT03799718), titled Safety and Efficacy of Repeated Administration of neuron (MSC-NTF Cells) nin Participants With Progressive MS, will enroll and treat up to 20 adults with progressive MS at multiple investigational study sites. Both primary progressive MS (PPMS) and secondary progressive MS (SPMS) patients are eligible.
Autologous cellular therapy — the transplant of stem cells removed from a patient, grown or modified, and put back into that same patient — is emerging as a credible and practical treatment option for cancer and other highly debilitating diseases, including neurodegenerative diseases.
The idea behind BrainStorm’s NurOwn cellular technology is to take mesenchymal bone marrow stromal cells (MSCs) from patients with a neurodegenerative disease, and modify them to become able to produce neurotrophic factors — biomolecules that support the growth, survival, and maturation of brain cells.
These bone marrow-derived MSCs are expanded and modified outside the patient, referred to as MSC-NTFs or NurOwn cells. The company aims to use this therapy approach to target disease pathways important in neurodegenerative disorders, including MS, amyotrophic lateral sclerosis (ALS), and Huntington’s disease.
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Autologous MSC-NTFs have been shown to effectively deliver multiple neurotrophic factors and immunomodulatory signaling molecules directly to the site of damage. The goal in patients is to ultimately slow or stabilize disease progression.
“We are pleased to announce the first participant with progressive MS has been enrolled in our Phase 2 clinical trial,” Chaim Lebovits, president and CEO of BrainStorm, said in a press release.
“Progressive MS has deeply affected the lives of many and dramatically impacted family members, caregivers and others. Today, there is no U.S. FDA approved therapy addressing more than one progressive form of MS and we are pleased to deploy our NurOwn technology platform in this new indication,” Lebovits added.
The 20 progressive MS participants in the trial will undergo a bone marrow biopsy to collect patient MSCs. Participants will receive three intrathecal transplants — injections into the cerebrospinal fluid — of NurOwn within 16 weeks, and will be followed for 12 weeks to determine the treatment’s safety and effectiveness.
Researchers will evaluate the safety of the procedure by the incidence of treatment-emergent adverse events up to 28 weeks after treatment. To confirm that MSC-NTFs are delivering neurotrophic factors and immunomodulatory signaling molecules as expected, the team will look for an increase in the amount and type of these biomolecules in the cerebrospinal fluid following NurOwn transplant.
“The first participant enrolled in our Phase 2 trial represents an important milestone in our commitment to address the unmet medical need in progressive MS,” said Ralph Kern, MD, chief operating and chief medical officer of Brainstorm. “We plan to quickly initiate enrollment across all study sites and look toward top-line data by the middle of 2020.”
“Soon, we will announce additional sites where the clinical trials will be conducted,” Lebovits added.
For more information about the trial, please visit this link.