Recent FDA Designation Puts Promising Engineered Cellular Therapy that Reduces Complications of Lymphoma Treatment on Accelerated Path to Market Approval

In November 2020, the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation for a promising engineered cellular therapy called AB-205. AB-205 acts promptly to repair injured vascular niches of organs to prevent or reduce severe toxicities in patients who have Hodgkin lymphoma (HL) or non-Hodgkin lymphoma (NHL) undergoing high dose chemotherapy and autologous stem cell transplantion.

AB-205 represents a new approach to repairing damaged organs and tissues and consists of proprietary universal human engineered cord endothelial (E-CEL®)cells.  This novel approach was based on groundbreaking research started in 2005 by Jason Butler, Ph.D., a research scientist at Hackensack Meridian’s Center for Discovery and Innovation (CDI).

“The CDI is at the forefront of pioneering research that translates to improved care and quality of life for patients at Hackensack University Medical Center, and around the world,” said Robert C. Garrett, FACHE, CEO, Hackensack Meridian Health.

AB-205’s RMAT designation is based on the drug’s Phase 1b/2 clinical trial results.

The John Theurer Cancer Center (JTCC) at Hackensack University Medical Center participated in the in the Phase 2 AB-205 trial that led to the RMAT designation. Two JTCC patients were enrolled in the study.

“At Hackensack University Medical Center, our patients have access to some of the most promising, groundbreaking clinical trials that are advancing the field of cancer care,” said Ihor S. Sawczuk, M.D., FACS, northern market regional president at Hackensack Meridian Health and the chief research officer of the network. “AB-205 is a perfect example of how our researchers are helping to bring treatments from the lab to the clinic for the benefit of our patients.”

According to a press release from Angiocrine Bioscience Inc., the clinical-stage biotechnology company that is developing the drug, the RMAT designation is an important step in accelerating the development of AB-205 toward its first market approval.

“The FDA RMAT designation is awarded to regenerative medicine therapies that have the potential to address serious unmet medical needs and provides AB-205 the opportunity for an accelerated review with the FDA,” said Scott Rowley, M.D., of the Stem Cell Transplantation and Cellular Therapy Program, at Hackensack University Medical Center’s John Theurer Cancer Center and principal investigator on the AB-205 trial. “Only a small number of novel products  receive this designation each year.”

AB-205 , is is administered intravenously on the same day that the patient’s own blood stem cells are infused, after a week of high-dose chemotherapy and radiation to eradicate cancer cells.  By repairing vascular niches in healthy organs, AB-205 cells aid in the restoration of the body’s natural ability to heal itself following myelosuppressive therapies including chemotherapy and radiation. Examples of vascular niches, which have significant  restorative capabilities, include the bone marrow and the gastrointestinal (GI) tract.

“The GI tract replenishes its lining approximately every 7 days and can be very sensitive to chemotherapy,” said Dr. Rowley.

When the vascular niche in the GI tract is injured by chemotherapy, it can result in nausea, vomiting and diarrhea. Harmful bacteria may also escape from the gut and cause serious infections such as endotoxemia, bacteremia, or sepsis. These complications are known as severe regimen-related toxicities (SRRT).

High-dose chemotherapy and stem cell transplantation are the standard therapy for patients with aggressive systemic HL and NHL. However, SRRT can occur in up to 50 percent of people with lymphoma who receive the standard treatment, with increased frequency and severity of SRRT occurring in older people. AB-205 helps repair vascular niches by restoring the natural process of tissue renewal, preventing or reducing SRRT.

“With AB-205, patients have fewer side effects from cancer treatment, they are discharged more quickly, and have a better quality of life,” said Dr. Rowley. “The product has so far shown to be very safe.”

Hackensack University Medical Center also plans to participate in Angiocrine’s planned Phase 3 clinical trial for AB-205, which is expected to begin in 2021.

Dr. Rowley and Dr. Butler are also looking forward to applying this research to other cancers — particularly multiple myeloma (MM) through Hackensack University Medical Center’s robust MM program.

“This could be a very major advancement for patients with MM as well as lymphoma,” said Dr. Rowley.

Dr. Butler is also looking for ways to use these types of cells to repair the cardiovascular system.

“Cardiovascular disease is the biggest killer of people around the world,” said Dr. Butler. “This would be very helpful for patients with co-morbidities that attack the cardiovascular system, such as diabetes.”

“Our CDI researchers and clinical teams are continually building on research successes and finding new ways to apply scientific advancements to improve public health,” said Mark D. Sparta, FACHE, president and chief hospital executive, Hackensack University Medical Center.