People’s Pharmacy: Could a new treatment cure lethal blood cancer?
The blood cancer multiple myeloma is deadly. This disease is personal for us because one of our best friends and colleagues died from the complications of this malignancy in 2006.
Dr. Tom Ferguson got the best treatment available at the time and lived a lot longer than his doctors predicted. But like so many before him, his body ultimately surrendered to both the cancer and the aggressive treatment.
That’s why we were so excited to learn about a new clinical trial (Journal of Clinical Oncology, June 3). The scientists recruited 97 multiple myeloma patients who had stopped responding to other available therapies. Many of these patients had few, if any, options left except hospice.
The investigators offered these desperate people a kind of immunotherapy called CAR-T. That abbreviation stands for Chimeric Antigen Receptor T-Cell therapy, a complicated procedure in which the patient’s white blood cells are removed and engineered to recruit T-cells against the cancer. They are then reinfused to fight the malignant cells.
Until now, CAR-T approaches have mostly been developed for other blood cancers, such as leukemia or lymphoma. The new therapy, called ciltacabtagene autoleucel or cilta-cel for short, worked surprisingly well for multiple myeloma.
One-third of the patients who got the therapy five years ago are still cancer-free. The researchers note: “To our knowledge, our data provide the first evidence that cilta-cel is potentially curative in patients with RRMM.” (RRMM stands for relapsed or refractory multiple myeloma.)
The Food and Drug Administration has approved cilta-cel and it is sold under the brand name Carvykti. Oncologists are excited about the unprecedented results. Dr. Norman Sharpless, former director of the National CANCER Institute, told the New York Times, “This is the first time we are really talking seriously about cure in one of the worse malignancies imaginable.”
There are several challenges with treatments like CAR-T. In many cases, insurance companies and guideline committees reserve these approaches as a last resort. That’s partly because they come with serious side effects. Carvykti can trigger fever, infections, fatigue, headache, diarrhea, muscle pain, nausea, low blood pressure and neurological symptoms.
Another drawback of CAR-T is the price tag. Many insurance companies balk at paying over half a million dollars for this treatment, unless all other options have been exhausted. There may also be a co-pay depending upon coverage.
What this means is that many patients will be on death’s doorstep before they can get access. Some investigators hope that if the treatment were administered earlier in the disease, it might prove to be a cure for more patients.
Like many of the newest cancer drugs, Carvykti has received Orphan Drug Designation from the FDA. When this category was first established, it was for “significant drugs of limited commercial value.”
The expectation was that pharmaceutical companies would not charge excessively for medicines that would be taken by relatively few people.
Drug companies quickly saw an opportunity, though. Orphan drugs are now huge money-makers for the industry. Some treatments for rare genetic conditions can cost millions. Many of the latest cancer treatments are also incredibly expensive. How many people will be able to afford extraordinary advances from breakthroughs like Carvykti?
In their column, Joe and Teresa Graedon answer letters from readers. Write to them in care of King Features, 300 W. 57th Street, 41st Floor, New York 10019, or email them via their website: www.PeoplesPharmacy.com. Their newest book is “Top Screwups Doctors Make and How to Avoid Them.”