Trailblazing a cure, out of the box

Published 6:26 pm Saturday, January 16, 2016

Dr. Emil Freireich was a young researcher in 1955. Emil grew up in Depression-era Chicago as the son of a single mother working 18 hours a day in a sweatshop, learning early on to be tough and think for himself. Now, working at the National Cancer Institute, he was tasked by the director of the institute with curing childhood leukemia.

It was a bold assignment, and perhaps the director asked somewhat tongue-and-cheek. But Freireich took the assignment to heart.

At the time, expected survival for childhood leukemia was a mere eight weeks. It was any parent’s — or doctor’s — worst nightmare. Freireich would remember the horror of watching 3-, 4- and 5-year-old children bleed to death in that hospital unit. No one knew how to stop the bleeding.

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Chemotherapy was seen as a last-resort treatment. It felt like a death knell. Parents were often advised instead to just let their child pass away as comfortably as possible.

But where others saw hopelessness, Freireich was ready to fight. He first had to stop the horrific bleeding that was killing children before drug treatments even had a chance. He became convinced the problem was insufficient platelets, the tiny discs that enable clotting. The idea contradicted conventional wisdom, but when Freireich mixed platelets from his own blood with blood taken from a leukemic patient, he was proved right.

Now, he had time to start tinkering with a new approach to treatment. Until that point, cancer was treated with a single drug. If one drug didn’t work, you moved on to the next.

Instead of searching for a cancer “super-drug,” Freireich thought outside the box. He flipped the question, asking, “What can we do with what we have? How can we think differently about the options in front of us?”

Freireich wondered if combining several chemotherapy treatments simultaneously would increase the rate of remissions. Over the course of several trials, Freireich, along with a colleague, discovered a multi-drug treatment for leukemia. Their discovery led to cures that today save the lives of 75 percent of children with this cancer, along with providing more effective treatments for other cancers.

Freireich changed the course of history for cancer treatment. And he did it by challenging the status quo, thinking outside the box, and not being afraid to innovate.

Our healthcare system should focus on not only treating diseases, but also on curing and preventing them. The federal government should prioritize innovation, not create red tape. It should incentivize discovery, not create barriers to research. It should reward — not discourage — thinking outside the box.

That’s why I’ve introduced and co-sponsored legislation encouraging this kind of innovation in medical research:

The Patients First Act, for example, asks us to think differently about medical stem cell research. Adult stem cell research is an area where we see some agreement; adult stem cells are non-embryonic and noncontroversial, taken from live human beings through umbilical cord blood, bone marrow and other tissues, and used to regenerate and repair tissues and organs.

This bill, which I introduced, prioritizes funding stem cell research that has the most promise for curing and treating patients.  It provides scientists and researchers and physicians an opportunity to think differently.

The Childhood Cancer Survivorship, Treatment, Access, and Research Act, which I’ve cosponsored, expands opportunities for childhood cancer research and improves efforts to track childhood cancer through a national childhood cancer registry.

Even after beating childhood cancer, many survivors suffer from late effects of their disease or treatment, including secondary cancers and organ damage. This bill asks, “How can we improve quality of life for children who survive cancer?” and establishes a program to explore innovative models of care for them.

The Independent Innovator and Repurposing Act encourages developing new medical treatments by repurposing existing drugs to address other diseases, including cancer, Parkinson’s, Alzheimer’s, epilepsy, depression, sleep disorders and post-traumatic stress disorder. Drug repurposing is a critical avenue for discovering groundbreaking ways to combat — and ultimately cure — these devastating diseases.

Dr. Emil Freireich trailblazed ways to a breakthrough cure. I know we can do the same again today.

Congressman J. Randy Forbes represents Virginia’s Fourth District, which includes Suffolk, in the U.S. House of Representatives. Visit his website at forbes.house.gov.