Multiple Sclerosis Awareness Month 2025: Looking Back at Progress for Cell Therapy
In observance of Multiple Sclerosis Awareness Month, held annually in March, we took a look back at the past year's news in cell therapy for MS.
According to the Multiple Sclerosis Association of America, almost 1 million people in United States live with multiple sclerosis (MS). Although diagnosis can occur at any age, symptoms most commonly appear when people with MS are 20 to 50 years of age. The condition is more commonly diagnosed in women than men, and risk factors vary with regard to race, ethnic background, and place of residence.
An important area of interest for new therapeutic development in MS is cell therapy. A number of companies and academic institutions are now pursuing the development of such advanced treatments, with several clinical trials currently active. In honor of Multiple Sclerosis Awareness Month, observed annually in March by the patient and clinician communities, CGTLive® is taking a look back at the progress that has been made in cell therapy for MS over the past year. Click the "READ MORE" buttons for more details and information about each item.
Abata Therapeutics' Treg Therapy ABA-101 Garners FDA Fast Track Designation
September 1, 2024 — Abata Therapeutics’ ABA-101, an autologous regulatory T-cell (Treg) therapy for the treatment of patients with progressive MS, has received fast track designation from the FDA.
The cell therapy has demonstrated a tolerable safety profile in animal models with antigen-dependent Treg functionality, antiinflammatory cytokine production, suppression of the production of inflammatory cytokines, and therapeutic effect. The therapy is being developed for the treatment of patients with progressive MS who have imaging evidence of ongoing inflammatory tissue injury and who are HLA-DRB1*15:01 positive. About 45,000 people in the United States fit into this patient population.
"There are no effective treatments for progressive MS, and rapidly advancing new therapies is critical for patients and their families," Samantha Singer, MS, MBA, the president and chief executive officer of Abata, said in a statement. "We are very pleased that the FDA granted us Fast Track designation as it will enable us to expedite our efforts to bring ABA-101 to patients. We are focused on initiating our phase 1 study this year in patients and evaluating the potential impact of this important new therapy."
ImmPACT Bio’s CD19/CD20 CAR-T IMPT-514 Cleared for Phase 1 Trial in Multiple Sclerosis
August 22, 2024 — ImmPACT Bio’s IMPT-514, an investigational bispecific CD19/CD20-directed chimeric antigen receptor T-cell (CAR-T) therapy, has received clearance of an investigational new drug (IND) application from the FDA for a phase 1 clinical trial in patients with MS.
The planned study will take the form of a dose escalation trial and will seek to recruit adult patients with all forms of MS whose disease is suboptimally controlled even after previous treatment with high efficacy disease-modifying therapies. IMPT-514 is intended to slow or stop progression of accumulation of disease disability and is the only CD19/CD20 CAR-T therapy in development for MS to ImmPACT Bio’s knowledge. The company expects to launch the study during the first half of next year.
“IND clearance for our bispecific CAR T-cell therapy in MS marks an exciting achievement that further expands clinical development of our autoimmune program,” Sumant Ramachandra, MD, PhD, the chief executive officer of ImmPACT Bio, said in a statement. “As an intended one-time treatment, IMPT-514 has the potential to reset the immune system by depleting a broad range of autoreactive immune cells implicated in the pathogenesis of MS in patients. We expect to dose the first patient in the first half of 2025.”
TG Therapeutics Receives Clearance from FDA for Phase 1 Trial in Multiple Sclerosis for Allogeneic CAR-T Azer-Cel
August 14, 2024 — The FDA has cleared an IND application submitted by TG Therapeutics for azercabtagene zapreleucel (azer-cel), an investigational allogeneic CAR-T therapy, for a phase 1 clinical trial in patients with progressive MS.
Azer-cel was originally developed by Precision BioSciences, but licensed to TG Therapeutics for further development for the potential treatment of autoimmune diseases and other noncancer indications. In light of the IND clearance, TG Therapeutics now intends to initiate a phase 1 clinical trial before the end of the year.
“We would like to congratulate TG Therapeutics on receiving IND clearance for azer-cel in patients with progressive MS,” Michael Amoroso, MBA, the chief executive officer of Precision BioSciences, said in a statement. “We believe the expansion of allogeneic CAR-T into autoimmune diseases holds the potential to unlock new therapies for patients living with chronic disease. We look forward to TG Therapeutics initiating a clinical trial for azer-cel in autoimmune disease as we focus on the advancement of our own wholly owned in vivo gene editing pipeline, including our planned IND and/or clinical trial application submission for PBGENE-HBV for hepatitis B this year.”
Indapta to Evaluate g-NK Therapy in Trial for Progressive Multiple Sclerosis
August 7, 2024 — The FDA has cleared Indapta Therapeutics’ IND to evaluate its g-natural killer (g-NK) cell therapy IDP-023 in participants with progressive MS.
“I am excited to participate in this clinical trial because of the multiple potential mechanisms by which g-NK cells may impact the biology of MS,” Lawrence Steinman, MD, Professor of Medicine and Principal Investigator, Stanford, said in a statement. “In addition to being able to achieve B cell depletion by combining with a B cell directed monoclonal antibody, g-NK cells have the ability to kill HLA-E expressing autoreactive T and B cells. In addition, g-NK cells have potent anti-viral activity, and therefore may also address the Epstein Barr Virus reservoir that contributes to the disease pathogenesis.”
The clinical trial will be led by Stanford and University of California, San Francsico. Participants will receive IDP-023 in combination with ocrelizumab.
Eque-cel CAR-T Cleared for Multiple Sclerosis Trial
July 25, 2024 — The FDA has cleared IASO Bio’s IND application for its CAR T-cell therapy equecabtagene autoleucel (eque-cel) for the potential treatment of MS.
"In an investigator initiated trial (IIT) conducted in China, Eque-cel has shown promising efficacy in 6 autoimmune diseases. The IND approval of Eque-cel in the treatment of MS from the FDA is another strong evidence of IASO Bio's ongoing dedication and technological advancements in the treatment of autoimmune diseases. We will continue to adhere to the research and development philosophy that prioritizes clinical value to address unmet clinical needs and will place great importance on implementing a global strategy. Through close collaboration and in-depth exchanges with international clinical research institutions, we aim to accelerate the development and commercialization of more innovative drugs, bringing greater benefits to patients worldwide,” Yongke Zhang, PhD, Chief Scientific Officer, IASO Bio, said in a statement.
Eque-cel is an independently developed fully human antiBCMA CAR T-cell therapy initially investigated for treating multiple myeloma. IASO Bio has been exploring its use for treating central nervous system autoimmunity. The company recently reported on eque-cel's use in neuromyelitis optica spectrum disorder, myasthenia gravis, and immune-mediated necrotizing myopathy, and also received IND clearance for a trial in generalized myasthenia gravis in April 2024.
