Critics of experimental cancer therapy just don’t get it

Cancer killed my young son, Zach. He fought hard against it for four years. His experience, his bravery, and most of all his absence make me question some of the critics of CAR-T therapy, a cutting-edge form of cancer immunotherapy. They say it is dangerous. I say I wish it had been around for Zach.

CAR-T therapy involves modifying a cancer patient’s own T cells so they recognize and kill cancer. Critics have seized on several deaths in a CAR-T clinical trial and are blaming companies for moving too fast in a race to bring the first CAR-T product to market. They were quick to spotlight the harrowing details of patients who died or experienced harmful side effects. CAR-T therapy was referred to in a STAT article as “terrifying,” “Frankensteinian T cells” whose treatment costs could “be enormous, with potential bills running hundreds of thousands of dollars.”

Zach experienced horrendous side effects from his chemotherapy, died from treatment-related complications, and the cost was astronomical — but all of this was with “standard” cancer therapy. In my view, the critics of CAR-T therapy are taking a myopic view of this incredibly powerful and exciting form of cancer treatment, one that holds great hope for desperate patients and their families.

Zach’s story

Zach was diagnosed with acute myeloid leukemia (AML), an aggressive type of cancer, when he was just 5 years old. Although AML occurs far more often in adults, it is the second most common type of leukemia in children and one with a poor prognosis. Current treatments for AML are brutal, requiring multiple rounds of in-patient, pedal-to-the-metal chemotherapy that wipes out the immune system and leaves patients vulnerable to life-threatening infections. Treatment-related visits to the hospital and the intensive care unit are not uncommon.

I worked round-the-clock to comfort Zach as he suffered uncontrollable fevers, unrelenting nausea, hours-long nosebleeds, and skin burned from the inside-out — all side effects of the chemotherapy that was poisoning his healthy cells as well as his cancerous ones.

Zach struggled against AML for four years. He endured multiple rounds of intense chemotherapy, full-body radiation, and three bone marrow transplants. He died in an intensive care unit from transplant-related liver failure and blood loss. The cost of his four years of treatment was millions of dollars — not uncommon for young AML patients who receive one or more bone marrow transplants.

But know this: he died from a treatment option he had chosen to have. And he died with hope.

A chance at hope

Zach’s fight makes me passionate about speeding access to more targeted and curative cancer therapies. I, along with other patients and families running out of options, want scientists and companies to move faster. We understand that progress is not always pretty. But neither is cancer, nor many of the current treatments for it.

The harsh reality is that 10,000 Americans died from AML last year, and nearly 600,000 died from all cancers. Chemotherapy and radiation are highly toxic; bone marrow transplants, highly criticized in the early days, have notoriously high mortality and morbidity rates. And yet, over one million patients have received marrow transplants, and this Nobel Prize-winning therapy has saved countless lives and led the way in the discovery of immunotherapy.

Those who survive AML and other cancers can end up battling difficult and costly problems related to their cancer treatment as they get older, such as organ failure, infertility, graft vs. host disease, and even secondary cancers. This is especially problematic for childhood cancer survivors, who have years of life ahead of them.

CAR-T and other immunotherapies are a shining beacon of hope. Results in CAR-T clinical trials are promising, with complete remission often seen in some kinds of blood cancer. In childhood acute lymphoblastic leukemia, remission rates exceeding 90 percent have been reported. Many children and adults who were once facing certain death, like Emily Whitehead and Milton Wright, are alive today because of this new form of immunotherapy.

My family would have given anything for an option like CAR-T.

Zach participated in numerous clinical trials — he was a pioneer, of sorts. By its very definition, pioneering can be dangerous, and even deadly. We were always aware of the risks.

I believe that researchers and clinicians must be empowered to innovate and move full-speed ahead. When working at the cutting-edge of medicine, they need to follow strict safety measures and best practices, and certainly must be held accountable. But they also need to be given ample room to fail, learn, and refine. We accept injury and death as part of “standard” cancer treatments like the ones Zach endured. We need to do the same for experimental therapies such as CAR-T.

I’m not advocating for research run amok. Investigators, clinicians, cancer patients, and their family members must approach an experimental therapy responsibly, balancing risk versus reward. But patients and families are more than capable of weighing the risks of experimental therapies against one last chance at hope.

We also need investors to support firms developing next-generation cancer therapies. That means the media must provide balanced reporting. It’s fine to communicate safety issues in a clinical trial. But it’s also important to provide context. How many people has the treatment helped? What are the dangers and expense of the “standard” treatment?

And let’s not forget history. Countless patients, and even some researchers, were lost in the development of life-saving treatments like chemotherapy, radiation, and bone marrow transplant.

Here’s my bottom line: testing CAR-T therapies is giving hope to patients facing certain death. The idea that there are safer and less expensive alternatives often isn’t true. There will inevitably be stumbling blocks and risks associated with developing any new therapy — and some people will die. But with this risk comes the potential of incredible reward and new discoveries.

Sadly, there was simply no good path for Zach. He and our family chose to stand and fight, opting for a boundary-pushing, high-risk, 3rd bone marrow transplant. It didn’t save Zach, but we were grateful to have had the choice.

Despite the current safety concerns with CAR-T, I can say with confidence that Zach and my family would have chosen it had it been available then. That’s a choice thousands of Zachs are waiting for today.

Julie Guillot is a former IT/business operations executive who now advocates and raises funds for the Children’s Oncology Group and other organizations to accelerate less toxic, more curative treatments for pediatric cancer. The Children’s Oncology Group receives funding from the National Cancer Institute and pharmaceutical companies.