The Discovery and Approval of Myeloma Therapy

Sumedha Maddali '24

The start of the beginning. The commencement of the journey to cure cancer, a disease that claims the lives of an estimated 9.5 billion people worldwide [1]. 


A few weeks prior, the second cell-based and cell-targeted therapy for multiple myeloma, a type of blood cancer, was approved by The Federal Drug Administration (FDA) in the United States. After the upstanding approval, Johnson and Johnson, a pharmaceutical industrial giant, gained rights to the drug and conducted multiple studies that proved the effectiveness of the therapy [2]. But, in order to truly comprehend the significance of this action, the two root questions that must be answered are: 


  1. What is Multiple Myeloma? 


Multiple Myeloma is defined as a blood cancer in plasma cells. Plasma cells are primarily found in the bone marrow, which is a soft tissue where blood cells are made. The bone marrow plays a vital role in the body’s immune system. Overarchingly, the immune system comprises a variety of cells that can help fight infections and other diseases. Plasma cells are a significant part of this system as they make immunoglobulins to kill germs. Occasionally, when these plasma cells grow exponentially out of control, they make an abnormal protein known as monoclonal immunoglobulin. Ultimately, this malady is known as multiple myeloma.  Some major issues involved with the diagnosis of this disease are low blood counts, bone and calcium issues, infections and kidney problems [3]. Currently, about 160,000 people are diagnosed with this condition per year [4]. 


  1. What was the 1st therapy that was approved in 2021? 


On the 26th of March, 2021, the FDA approved a type of immunotherapy named the CAR T-cell therapy for patients with multiple myeloma. This treatment was particularly catered towards patients who had not responded to or relapsed after 4 or more past cancer treatments. Indeed, previous therapies exist but have proven to be ineffective as they merely work for a few months, often leading to relapses. The drug’s approval was based on a small-scale study that found this treatment to completely or partially shrink tumors in 72% of patients. Within the process to carry out this therapy, a patient’s T-cells are collected and with the extensive aid of genetic engineering, are transformed to be able to better identify and “kill” cancer cells. However, James Kochenderfer, M.D., a senior investigator in NCI’s Center for Cancer Research states that, “…we still have a long way to go because very few patients, if any, are cured. So we need to continue research to improve the treatment to try to permanently cure patients of multiple myeloma”. Unfortunately, some examples of side effects this treatment can have are brain-related, overactive immune responses, skyrocketing expenses and many more [5]. 


Building on these new innovations, a second therapy was approved in February, 2022 by the FDA. As previously mentioned, Johnson and Johnson has acquired the rights to this drug. Historically, this is the first drug to be used in the United States initially manufactured in China. This drug, coined “Carvykti”, is solely meant for older patients who have not responded well to previous treatments. In one of J&J’s studies, it was discovered that among the 97 participants, 98% had a significant reduction in the formation of the abnormal proteins and 83% had no detection of cancer cells at all. This was administered over a period of 22 months [6]. 


Nevertheless, this drug has potential drawbacks as well. The complex processes involved in the manufacturing of this drug and the expenses in administering it have hindered the wide usage of it. This therapy is priced at almost $400,000 per patient [7]. But, most medicare insurance does indeed cover these expenses. In the past, most CAR T-cell therapies had research conducted in the Western World but J&J took the risk of collaborating with a Chinese company, the Legend Biotech Corp. As a result, a few selected FDA officials have raised concerns on the quality of the research and testing and the congruence of those results with an American population [6]. 


With this discovery, the world is hoping to see light at the end of the tunnel to save countless lives. Hopefully, an increased numbers of treatments can be formed for other types of cancers through the successes of the Myeloma Therapy.