The Hackensack Meridian John Theurer Cancer Center (JTCC) at Hackensack Meridian Hackensack University Medical Center played an integral role in the recent phase 2 KarMMA trial leading to the approval of the U.S. Food and Drug Administration (FDA) of a novel CAR-T therapy known as idecabtagene vicleucel (ide-cel), or Abecma. Representing the first patient-specific cell therapy approved by the world’s Gold Standard regulator, the novel CAR-T therapy targets a protein called B-cell maturation antigen (“BCMA”) found in malignant plasma cells. Now authorized by the FDA as a one-time infusion, the treatment is customized or tailored to the patient’s own T-cells, which are collected and genetically modified, and then infused back into the patient. Truly a personalized medicine for some cancer patients, once the care team injects the re-engineered immune cells back into the patient, they seek and destroy BCMA-containing cells. Part of the immuno-oncology revolution, biomedical researchers here in New Jersey applied this breakthrough technology to multiple myeloma. The KarMMA trial was led here in New Jersey by one of the nation’s foremost authorities on multiple myeloma—David S. Siegel, MD, PhD. Dr. Siegel directs the Multiple Myeloma Institute at John Theurer Cancer Center.
A type of blood cancer impacting the plasma cells located in the bone marrow, multiple myeloma is a type of blood cancer that impacts the body’s immune system. The disease can manifest in myriad ways, from broken bones and anemia to kidney problems and infections. The number of patients with this condition has steadily grown over the last thirty years in America and worldwide. Researchers think about half of the growth of the number of cases is attributable to the aging population. Over 30,000 Americans develop multiple myeloma cancer each year. It represents a growing problem and the goal of biomedical research in the field of immuno-oncology is, of course, to find treatments and hopefully someday a complete cure.
The Study Results
As TrialSite shared on the first of April, the FDA approved Abecma for adult patients with multiple myeloma who haven’t responded to, or whose disease has returned after, at least four prior lines (different types) of therapy.
The safety and efficacy was this novel CAR-T therapy was established in a multicenter study of 127 patients with relapsed myeloma (myeloma that returns after completion of treatment) and refractory myeloma (myeloma that does not respond to treatment), who received at least three prior anti-myeloma lines of therapy. About 88% of patients in the study group had received four or more prior lines of anti-myeloma therapy.
Hackensack Meridian Health recently reported that the study results were reported earlier this year in the New England Journal of Medicine, with Dr. Siegel as a co-author in the report titled, “Idecabtagene vicleucel in Relapsed and Refractory Multiple Myeloma.“ The study results found that out of 128 patients who received idecabtagene vicleucel, 94 (73%) had a positive response with 42 (33%) of those patients having a complete response.
Treatment with Abecma has the potential to cause severe side effects. The label carries a boxed warning for, cytokine release syndrome (CRS), hemophagocytic lymphohistiocytosis/macrophage activation syndrome (HLH/MAS), neurologic toxicity, and prolonged cytopenia, all of which can be fatal or life-threatening. CRS and HLH/MAS are systemic responses to the activation and proliferation of CAR-T cells causing high fever and flu-like symptoms, and prolonged cytopenia is a drop in the number of a certain blood cell type for an extended period of time. The most common side effects of Abecma include CRS, infections, fatigue, musculoskeletal pain, and a weakened immune system. Side effects from treatment usually appear within the first one to two weeks after treatment, but some side effects may occur later. Patients with multiple myeloma should consult with their health care professionals to determine whether Abecma is an appropriate treatment for them.
Principal Investigator Point of View
Dr. Siegel at this point can be considered one of the top experts on CAR-T cell therapy involving multiple myeloma and whether for patients and their families or the pharma sector, an important person to connect with on this topic. In the recent Hackensack Meridian Health news entry, he is quoted, “Patients with multiple myeloma are in critical need of new therapies after attempting traditional treatments unsuccessfully.” Dr. Siegel continued, “The approval of idecabtagene vicleucel as the first CAR-T cell therapy in Multiple Myeloma means we can offer hope to patients, many of whom have no other option.”
The Trial Site
This study was made possible thanks to the ongoing commitment and investments in this field by the Hackensack Meridian Health JTCC. The Hackensack Meridian Health Center has its roots in this region going back to 1888 representing Bergen County’s first hospital.
Fast forward today and the major non-profit, 770-bed research and teaching hospital is located as the bird flies just seven miles from Manhattan. Representing New Jersey’s largest provider of inpatient and outpatient services and the fourth largest hospital in the United States based on admissions—and the largest hospital in the overall sprawling health system that’s affiliated with the New Jersey Medical School of Rutgers University and Hackensack Meridian School of Medicine. Note that the JTCC is part of the National Cancer Institute (NCI)-designated Georgetown Lombardi Comprehensive Cancer Center based in Washington DC. This means that patients have access to all the latest advances to cancer fighting investigational therapies, leading the clinical research as a care option movement.
Led by Andrey Goy, M.D., Physician-in-chief of oncology services, Hackensack Meridian Health and Chair of John Theurer Cancer Center, and the Chief Innovations Officer Andrew Pecora, The JTCC has 14 specialized divisions and Radiation Oncology experts. Key divisions include:
- Blood and Marrow Stem Cell Transplantation Program
- Lymphoma Division
- Multiple Myeloma Division
- Leukemia Division
- Neuro-Oncology Division: Brain and Spine Institute
- Skin and Sarcoma Division: Cutaneous Malignancy Program
- Gastrointestinal Oncology Division
- Geriatric Oncology Division
- Head and Neck Oncology Division
- Thoracic Oncology Division
- Radiation Oncology Department
- Urologic Oncology Division
- Breast Oncology Division
- Gynecologic Oncology Division
- Research Division
Andre Goy went on the record on this accomplishment: “This approval opens a new chapter for patients with relapsed or refractory myeloma.” Dr. Goy continued, “At JTCC, we are grateful to be at the forefront of novel therapies development such as ide-cel. This not only expands treatment options in a very difficult situation for myeloma patients who have exhausted all other therapies, but we are also looking in ongoing trials at incorporating ide-cel including earlier in the course of the disease. We are very proud that our myeloma program at JTCC, together with the best institutions in the country, has been leading the efforts of all novel therapies approved in myeloma for the last three decades!”
Call to Action: If you or a loved one has been diagnosed with multiple myeloma and haven’t found a therapy that has worked as of yet, consider looking into this research and, if possible, connecting with JTCC. See the link for Dr. Siegel’s appointment tool. TrialSite is building Patient Communities including groups and should enough people seek a multiple myeloma group, the TrialSite’s news algorithm can provide targeted updates on multiple myeloma research.