In Gene Sequencing Treatment for Leukemia, Glimpses of the Future

Second Chance: Lukas Wartman, a leukemia doctor and researcher, developed the disease himself. Facing death, his colleagues sequenced his cancer genome. The result was a totally unexpected treatment.

Thanks to Vinod Khosla who tweeted the URL for this article. This is an important case – we know that cancer is definied by its DNA and RNA – not by the invaded organ or tissue. Lukas Wartman was lucky to be at one of the top cancer institutes, Washington University, and part of a team who had the knowledge and facilities to do whole genome sequencing and analysis. The team found via his RNA sequence a rogue gene that was responsible for the high rate of cancer cell production. Wartman’s luck was further enhanced when the team identified a drug approved for kidney cancer that might shut down the malfunctioning gene.

Here is a snippet of the first paragraphs:

ST. LOUIS — Genetics researchers at Washington University, one of the world’s leading centers for work on the human genome, were devastated. Dr. Lukas Wartman, a young, talented and beloved colleague, had the very cancer he had devoted his career to studying. He was deteriorating fast. No known treatment could save him. And no one, to their knowledge, had ever investigated the complete genetic makeup of a cancer like his.

So one day last July, Dr. Timothy Ley, associate director of the university’s genome institute, summoned his team. Why not throw everything we have at seeing if we can find a rogue gene spurring Dr. Wartman’s cancer, adult acute lymphoblastic leukemia, he asked? “It’s now or never,” he recalled telling them. “We will only get one shot.”

Dr. Ley’s team tried a type of analysis that they had never done before. They fully sequenced the genes of both his cancer cells and healthy cells for comparison, and at the same time analyzed his RNA, a close chemical cousin to DNA, for clues to what his genes were doing.

The researchers on the project put other work aside for weeks, running one of the university’s 26 sequencing machines and supercomputer around the clock. And they found a culprit — a normal gene that was in overdrive, churning out huge amounts of a protein that appeared to be spurring the cancer’s growth.

Even better, there was a promising new drug that might shut down the malfunctioning gene — a drug that had been tested and approved only for advanced kidney cancer. Dr. Wartman became the first person ever to take it for leukemia.

And now, against all odds, his cancer is in remission and has been since last fall. While no one can say that Dr. Lucas is cured, after facing certain death last fall, he is alive and doing well. Dr. Wartman is a pioneer in a new approach to stopping cancer. What is important, medical researchers say, is the genes that drive a cancer, not the tissue or organ — liver or brain, bone marrow, blood or colon — where the cancer originates.