Modern medical research may be inching closer to a cure for cancer, but the answer may lie in a very unlikely place. It seems crazy to think that any form of cancer could be treated by a disabled version of the HIV virus, which has killed more than 25 million people over the past 32 years. Nonetheless, scientists and pharmaceutical companies have been working together to do just that, with several success stories in clinical trials. Could a cure for cancer be on the horizon?
Emma Whitehead was just five-years-old when she was diagnosed with acute lymphoblastic leukemia (ALL), a cancer commonly diagnosed and successfully treated in children. Emma was different, though. She was not among the 85 percent of kids that were able to beat the cancer into remission with chemotherapy; in fact, her cancer kept coming back no matter how many things they tried. The Whiteheads had one last hope to save Emma’s life in a clinical trial that had never before been tried on a child, or anyone with ALL for that matter.
Blood was taken out of Emma’s body, stripped of its white blood cells, and put back in. The next step taken by trial pioneers from University of Pennsylvania sounds unthinkable. A modified version of HIV was used to genetically reprogram her white blood cells and put them back into her blood in hopes that they would attack the cancer. This particular method of using a disabled form of the HIV virus to genetically re-engineer a person’s immune system to fight against cancer is referred to as chimeric antigen receptor T-cells (CART) therapy.
The treatment almost killed her. She was back in the hospital within days of the treatment, with a one-in-1,000 chance of surviving. What happened next gave doctors and scientists the last piece in the puzzle: Emma was given a rheumatoid arthritis drug to calm the cytokine storm in her immune system flaring from the CART treatment. Emma woke up on her seventh birthday in recovery. Days later, the Whiteheads received a call that Emma was cancer free, and two years later as she wraps up the second grade, she still is.
An older patient named Doug Olson is another example of miracle survival. Olson, then 64, had suffered with chronic lymphocytic leukemia for 14 years, and his body had stopped responding to chemotherapy. He said in an interview with Forbes that he was basically just “waiting to die.” He was the first patient to receive the CART treatment, and the results were the same as Emma’s. Four years later, he is still cancer free.
“As far as I’m concerned I’m cured,” Olson said. “I don’t ever think about relapse. I don’t. It’s like all of a sudden you can breathe.”
This treatment has worked in dozens of cases, with a 90 percent remission rate with ALL patients in particular. Several other survival cases have emerged over the past couple of years in patients of all ages with various types of blood cancers, all citing the CART treatment developed at University of Pennsylvania as the procedure they hold responsible for their remission. The entire medical community is beginning to wonder whether CART therapy is a building block for a cure for cancer on the horizon.
For years, big pharmaceutical companies like Bristol-Meyers Squibb and Novartis have been investing in cell treatments which use the immune system as the weapon against blood cancers and even tumors. These treatments have been a beacon of hope in patients that have stopped responding to chemotherapy drugs and have nowhere else to turn. However, breakthrough treatments like the one that saved Emma have many hurdles to jump before they can be taken to market.
Nonetheless, big pharma is putting a lot of faith—and a lot of money—into this particular cancer treatment. Novartis has devoted $20 million to building a research center on Penn’s campus in hopes to eventually bring a drug to market using the CART treatment.
Among the questions that still need an answer is whether CART therapy will work for any other type of cancer besides forms of leukemia. Drugs like Novartis’ patented Gleevec are effective treatments for against blood cancers, but when it comes to tumors from cancers of the breast and lung, results are not as promising. CART drugs may or may not be more effective, but researchers are gearing up to study how CART therapy measures up against cancers like mesothelioma (a type of lung cancer) to start looking for answers.
There has become a bit of a research and development race, with other companies such as Juno Therapeutics testing different versions of CART therapies that have less harmful side effects than the early version which nearly killed its clinical trial patients’ immune systems.
Although modern medicine may have a cure for cancer on the horizon, it is still too early to tell how far away that horizon might be, and who might get there first. The important thing is that the world may be on the eve of a milestone in the battle against cancer.
By Erica Salcuni