South Middlesex Times

Rutgers Health researchers are set to enroll patients with primary or secondary progressive multiple sclerosis (p-MS) in a study to determine if an engineered immune cell therapy can halt the progression of the autoimmune disease.

Multiple sclerosis is characterized by mistargeted attacks by the body’s immune system and active central nervous system (CNS) inflammation. As CNS damage accumulates, patients typically experience increasing difficulty swallowing, balancing, breathing, coordinating movements, generating strength, and controlling their bladders and bowels. Cognitive impairment may also increase.

The cellular therapy being tested involves altering a specific subset of immune cells taken from each patient: regulatory T cells (Tregs). Tregs typically suppress inflammation and autoimmune disease while promoting repair. The engineering process enables the modified Tregs to respond specifically to CNS inflammation. After administration, these engineered Tregs will circulate through the body and activate upon finding inflamed CNS tissue to suppress inflammation, reset immune tolerance, limit tissue destruction, and enable repair.

“The goal is to reprogram each patient’s Tregs to eliminate the inflammation within the central nervous system, limiting neuronal injury and restoring healthy nerves,” said Vikram Bhise, a professor of neurology at Rutgers Robert Wood Johnson Medical School who will oversee the trial in New Brunswick. “If treatment reverses disease progression, it will constitute a true breakthrough because existing treatments prevent relapses but don’t work for patients with p-MS.”

Approximately 1 million Americans have one of four types of multiple sclerosis: clinically isolated syndrome, relapsing-remitting, secondary progressive, and primary progressive. The phase I trial is open to patients with either secondary or primary p-MS without relapse activity who have a particular genetic signature and evidence of CNS inflammation on MRI.

Abata Therapeutics is developing the therapy and financing the study to test its safety and efficacy on at least a dozen patients at participating centers across North America. Abata recently announced clearance of their investigational new drug application by the U.S. Food and Drug Administration (FDA), allowing them to initiate the phase I trial. Bhise hopes to recruit between three and five patients at Robert Wood Johnson University Hospital.

Researchers at Abata Therapeutics will harvest Tregs from patients’ blood samples, activate them to multiply, and genetically edit them in the laboratory before reinjecting them into patients.

This strategy could help p-MS patients by preventing or stopping aggressive immune cells from attacking nerves. The Tregs can also break down clusters of inflammatory cells and secrete nerve repair factors.

Robert Wood Johnson’s selection as a trial site was influenced partly by Rutgers Health's extensive operations capable of supporting such research with both a neurology team and a comprehensive cancer center — Rutgers Cancer Institute — that already provides related types of cell therapies like CAR T cell therapy.

Participation in this study will require seven hospital visits over approximately 12 months, including several days of inpatient care around the Treg cell infusion.

“We’re telling people upfront that it’s a vigorous process requiring genuine investment,” Bhise said. “There are spinal taps and high-strength MRIs with a risk of side effects. It’s not easy but it’s potentially life-changing.”