News of Note—Reducing the risks of bone marrow transplant; an antisense approach to epilepsy

A bone marrow harvest in progress
A more selective approach to preparing the body for bone marrow transplants could avoid side effects like organ damage and infertility. (Wikipedia/public domain/Navy news service)

ASH: Magenta antibodies could lower risks of bone marrow transplants

One of the biggest risks to patients undergoing bone marrow transplants or stem cell gene therapy is that the preparation—chemotherapy plus total-body radiation—can cause infertility, organ damage or other toxic side effects. Magenta Therapeutics has developed antibody-drug conjugates (ADCs) that could sidestep these side effects by selectively removing specific cells prior to transplant. At the American Society of Hematology annual meeting in San Diego, Magenta presented preclinical results showing that its ADCs targeting the antigens CD117 and CD45, both of which are expressed on some types of leukemia cells, successfully depleted target cells in mouse models of acute myeloid leukemia and significantly improved survival. (Release)

Antisense approach shows promise in rare form of epilepsy

Most patients with a rare form of epilepsy called Dravet syndrome have a mutation in the gene Scn1a, causing insufficient production of a key protein. Now, researchers at Stoke Therapeutics and the University of Michigan have data from research in mouse models of the disease suggesting targeting the mutation with an antisense oligonucleotide could provide long-lasting relief. At the American Epilepsy Society 2018 conference in New Orleans, the company presented data showing that a single dose of its lead product candidate increased expression of the Scn1a gene in the animals for 14 weeks, reduced seizures and prevented the sudden death that can occur in patients. (Release)

Fred Hutch researchers discover 40 new gene variants for colorectal cancer

Investigators led by the Fred Hutchinson Cancer Research Center have discovered 40 genetic variants associated with colon cancer, validated 55 previously identified variants and found a rare variant that protects against sporadic forms of the disease—those that don’t run in families. They made the discoveries with the help of 130 institutions, using data and tissue samples from 125,000 patients, making this study nearly double the size of previous analyses. The researchers, who published their findings in the journal Nature Genetics, believe the results will improve both genetic screening for colon cancer risk and drug discovery. (Release)