Fourth study published over two-year span that analyzes key leukemia protein
Northwestern Medicine scientists have discovered two successful therapies that slowed the progression of pediatric leukemia in mice, according to three studies published over the last two years in the journal Cell, and the final paper published Dec. 20 in Genes & Development.
When a key protein responsible for leukemia, MLL, is stabilized, it slows the progression of the leukemia, the most recent study found. The next step will be to combine the treatments from the past two years of research into a pediatric leukemia “super drug” to test on humans in a clinical trial.
The survival rate is only 30 percent for children diagnosed with MLL-translocation leukemia, a cancer that affects the blood and bone marrow. Patients with leukemia have a very low percentage of red blood cells, making them anemic, and have approximately 80 times more white blood cells than people without cancer.
30%Current survival rate for pediatric leukemia patients.
“These white blood cells infiltrate many of the tissues and organs of the affected individuals and is a major cause of death in leukemia patients,” said senior author Ali Shilatifard, the Robert Francis Furchgott Professor of Biochemistry and Molecular Genetics and Pediatrics, the chairman of biochemistry and molecular genetics and the director of Northwestern’s Simpson Querrey Center for Epigenetics. “This is a monster cancer that we’ve been dealing with for many years in children.”
There are several types of leukemia. This research focused on the two most common found in infants through teenagers: acute myeloid leukemia (AML) and acute lymphocytic leukemia (ALL).
For the past 25 years, Shilatifard’s laboratory has been studying the molecular function of MLL within its complex known as COMPASS (Complex Proteins Associated with Set1). Most recently, it was demonstrated that COMPASS components are one of the most frequently identified mutations in cancer.The next step of this work will be to bring the drug to a clinical trial setting, which Shilatifard said he hopes will happen in the next three to five years.
“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.”
Earlier work from Shilatifard’s laboratory published in Cell in 2018 identified compounds that could slow cancer growth by interrupting a gene transcription process known as “Super Elongation Complex” (SEC). It was the first compound in its class to do this.
This MLL stabilization process discovered in the most recent paper could potentially work in cancers with solid tumors, such as breast or prostate cancer, said first author Zibo Zhao, a postdoctoral research fellow in Shilatifard’s lab.
“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,” Zhao said.
“The publication of these four papers and the possibility of a future human clinical trial could not have happened if it weren’t for the cross-disciplinary collaboration at Northwestern,” Shilatifard said.
The Latest on: Pediatric leukemia
via Google News
The Latest on: Pediatric leukemia
- Atrium Health Levine Children’s begins FDA-approved CAR T-cell therapy treatmenton April 30, 2021 at 9:00 pm
Juan Bravo, 10, was diagnosed with leukemia in 2019 and relapsed in May 2020. With the support of his pediatric care team at Atrium Health Levine Children’s, family and community, Juan had undergone ...
- Pediatric nursing at its beston April 30, 2021 at 12:15 pm
The Alex Stuetze Memorial Pediatric Nursing Award was created to honor pediatric nurses who excel in caring for our children.
- Platelet function and bleeding at different phases of childhood immune thrombocytopeniaon April 30, 2021 at 10:49 am
Immune thrombocytopenia (ITP) is believed to be associated with platelet function defects. However, their mechanisms are poorly understood, in particular with regard to differences between ITP phases, ...
- Naugatuck Family Battles Leukemia For 2nd Time: Reporton April 30, 2021 at 7:11 am
The Brice family's two young daughters contracted the disease; one overcame it, the other is in the midst of the fight.
- A clinical transcriptome approach to patient stratification and therapy selection in acute myeloid leukemiaon April 30, 2021 at 3:21 am
Several genomic features have been found for acute myeloid leukaemia (AML) but targeted clinical genetic testing fails to predict prognosis. Here, the authors generate an AML prognostic score from RNA ...
- 'There's hope' | Naugatuck family demonstrates love and strength as another daughter battles leukemiaon April 29, 2021 at 8:27 pm
I tell other mothers - I want you to know this is our second time battling cancer- because there is hope. There’s hope.“ ...
- What to know about acute myeloid leukemia in childrenon April 28, 2021 at 6:00 pm
Acute myeloid leukemia can cause a fever, fatigue, shortness of breath, and easy bruising or bleeding. Learn more about this type of cancer in children.
- 5-year-old fighting leukemia grants wish to give back to other sick kids through fun beddingon April 26, 2021 at 5:48 pm
But 5-year-old Grayson at St. Louis Children’s Hospital hopes his wish will help other kids like him. Grayson was granted a wish from the Make-A-Wish Foundation of Missouri & Kansas after getting ...
- Ashley Cain's 8-Month-Old Daughter Dies of Leukemia—What Are the Signs of Infant Leukemia?on April 26, 2021 at 3:44 pm
Ex-UK soccer player and reality TV star Ashley Cain shared heartbreaking news on social media on Sunday. His baby daughter, Azaylia Diamond Cain, died on Saturday at 8 months old. At only 8 weeks old, ...
- FDA Grants Priority Review to SH-111 for Pediatric T-Cell Leukemiaon April 23, 2021 at 8:00 am
The FDA has granted a priority review designation to SH-111, a sterile injectable therapy, for the treatment of pediatric patients with T-cell leukemia.
via Bing News