Multiple myeloma is personal for me. That’s because my best friend, Dr. Tom Ferguson, died from this blood cancer on April 14, 2006. Every year about 35,000 people are diagnosed with multiple myeloma and over 12,000 die. Tom was the father of the medical self-care movement.
To quote Wikipedia, he
“…was an American medical doctor, educator, and author. He was an early advocate for patient empowerment, urging patients to educate themselves, to assume control of their own health care, and to use the Internet as a way of accomplishing those goals.”
Tom and I authored two books together and I miss him every day. The photo is of Doc Tom. It sits on my computer so I can see him every day. He had the best treatment available at that time. Now, though, there is a new treatment for multiple myeloma and it might have saved his life if it had been available then.
CAR-T vs. Multiple Myeloma:
The Food and Drug Administration has just approved an exciting new treatment for multiple myeloma. It is called cilta-cel and will be sold under the brand name Carvykti. It was developed by Janssen, a subsidiary of Johnson & Johnson and Legend Biotech, a China-focused drug development company.
This CAR-T immunotherapy was tested in both China and the US. It involves the removal of a patient’s own immune-system T cells. These cells are then treated in the laboratory so that they learn to recognize and attack the proteins that the multiple myeloma cells produce. When re-introduced into the patient’s body, these T cells then mount a defense against the cancer.
The results have been promising. In one trial of 97 patients, 83 percent had a complete response. These patients had all tried at least three other therapies before Carvykti. After 22 months, more than half of these individuals were still alive and their disease had not progressed. That is impressive.
When Tom’s cancer returned, he had a bone marrow transplant. I remember trying to protect him from infection because his immune system had been decimated by heavy-duty chemotherapy. The photo below is Tom at the University of Arkansas Medical Center where he had recently undergone his bone marrow transplant. You can see the ports that were implanted. He recovered and did well for quite a while. Tom lived a lot longer than most multiple myeloma patients at that time. Carvykti might have changed the outcome.
The Downsides of CAR-T for Multiple Myeloma:
There are downsides to Carvykti. One is a serious potential side effect, neurotoxicity. Beyond that, cytokine release syndrome can threaten organ systems throughout the body. Another disadvantage for patients is the very high price tag, expected to be around $465,000.
Please do not get me started on the high cost of cancer drugs! Should you wish to read my thoughts on this topic, here is a link.
Finally, there have been supply chain problems that may make it difficult for patients to access this novel therapy, even at the high cost.
How Well Does CAR-T Work?
Initial results have been promising against blood cancers such as lymphomas and leukemias. Investigators have also reported on studies that have used CAR-T to treat people with multiple myeloma.
The studies are small, but the results are impressive. The volunteers had previously been given standard multiple myeloma treatments but their cancer had gotten worse. After the CAR-T therapy, almost every patient responded.
Many went into complete remission and some are more than a year out of treatment with no sign of disease. One patient, however, had the cancer worsen. The results with Carvykti are even more promising.
What Are the Side Effects?
Side effects of CAR-T therapy include low blood pressure, fever and difficulty breathing as a result of the immune system being overwhelmed. These reactions may require hospitalization but they usually respond to treatment.
To Learn More:
We discussed this as a cutting-edge cancer therapy in Show 1029: How to Mobilize the Immune System to Fight Cancer.