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Promising drug for pediatric leukemia in developing stages

Researchers had success with the drug in several studies over the last two years

Photo (c) tashatuvango - Fotolia
For young leukemia sufferers, the odds of survival aren’t very high, as the disease attacks their bone marrow and red blood cells.

However, hope is on the horizon.

Scientists from Northwestern Medicine have found that if they could stabilize the MLL protein -- the one responsible for the spread of the disease -- they could work to slow down the progression of leukemia in affected patients.

Making progress

Researchers at Northwestern’s Simpson Querrey Center for Epigenetics have spent over two decades -- led by Ali Shilatifard -- studying the MLL protein and its effect on the spread and evolution of leukemia.

“I’ve been working on this translocation for more than two decades, and we’re finally at the point where in five to 10 years, we can get a drug in kids that can be effective,” Shilatifard said. “If we can bring that survival rate up to 85 percent, that’s a major accomplishment.”

The researchers have focused their studies on two types of leukemia: acute lymphocytic leukemia (ALL) and acute myeloid leukemia (AML).

While all past studies have been conducted on mice, the researchers’ two main findings from prior research could propel the testing to a clinical trial with human patients, and hopefully inch closer towards Shilatifard’s survival rate goal.

The researchers previously determined special compounds that, when interrupted during a special genetic process known as “Super Elongation Complex” (SEC), can slow down the spread of leukemia. Additionally, they were able to identify the cells that are most often mutated in cancer patients.

Most recently, they found that stabilizing the MLL protein in leukemia patients is effective in slowing the advancement of the disease. For the researchers, the next step is taking this information to the next level; the goal is to turn the findings into a new drug that can be tested on human patients in the coming years.

The researchers are confident in the direction the study has taken, and they believe that a new drug would change cancer treatments, as it could be beneficial for more than just leukemia sufferers.

“This opens up a new therapeutic approach not only for leukemia, which is so important for the many children who are diagnosed with this terrible cancer, but also for other types of cancers that plague the population,” said first author Zibo Zhao.

Fighting childhood cancer

To help continue the fight against leukemia and other childhood cancers, researchers have developed a new gene therapy that would not only treat the disease but also work to cure it.

The treatment -- sold as Kymriah -- is designed for patients who either experienced a relapse or who didn’t have good results with traditional drugs or treatments.

“Kymriah is a first-of-its-kind treatment approach that fills an important unmet need for children and young adults with this serious disease,” said Dr. Peter Marks. “Not only does Kymriah provide these patients with a new treatment option where very limited options existed, but a treatment option that has shown promising remission and survival rates in clinical trials.”

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