Drug Shows Promise as First Definitive Treatment for Rare Anemia

Medically reviewed by Rachael Grace, MD In the mid-1960s, David G. Nathan, MD, president emeritus of Dana-Farber and, at that time, a hematologist at Boston Children’s Hospital, published some of the first reports on a rare, inherited type of anemia caused by the breakdown of red blood cells because of a lack of a key … Continued

Resistance to Targeted Leukemia Drug Lurks in Cells’ “Powerhouse”

Medically reviewed by Catherine J. Wu, MD Within every human cell, a fateful balance prevails. The mitochondria — where nutrients from food are converted into fuel for the cell — serve as a kind of jury box where pro-survival proteins contend with proteins that favor cell death. In the ebb and flow of these proteins, … Continued

Chemical Toolkit Aids Study of Cancer Drug Resistance

Medically reviewed by Jun Qi, PhD Cancer is extremely clever at finding ways to survive, so even the most successful new drugs tend to become less effective over time as the tumors develop resistance. Often, cancer cells become resistant because of changes in the cells’ genetic code, allowing them to counteract or sidestep the drugs’ … Continued

Basic Science Discovery Leads to Clinical Trial for Patients with Chemotherapy-Resistant Form of Ovarian Cancer

Dana-Farber scientists recently uncovered a potential vulnerability in a form of ovarian cancer notoriously resistant to chemotherapy. Now they’ve opened a clinical trial involving a drug that targets that susceptibility in patients with the disease. The impetus for their research is a type of ovarian cancer with excess copies of the cyclinE1 gene (abbreviated CCNE1). … Continued

CAR T-Cell Therapy for Pediatric Patients: The Latest Updates

Since it was approved by the U.S. Food and Drug Administration (FDA) in the fall of 2017, a form of the powerful and promising therapy known as CAR T-cell therapy has been used to treat certain young patients with B-cell acute lymphoblastic leukemia (ALL) who have relapsed or didn’t respond to standard regimens. Today, researchers … Continued

Research Clears Up Mystery About Most Common Cancer Gene

Medically reviewed by Benjamin Ebert, MD, PhD In the rogue’s gallery of cancer mutations, the Most Wanted are found in TP53, the most frequently mutated gene in cancer and in some ways the most ominous. In patients with acute myeloid leukemia (AML) or a myelodysplastic syndrome (MDS), for example, the presence of a TP53 mutation … Continued

Making the Immune System Work Against Cancer: A Pioneering Researcher’s Journey

Bone marrow transplantation, which was first developed in the 1970s, was conceived as a way of dealing with the effects of high-dose chemotherapy for leukemia, lymphoma, and other blood-related diseases. The large doses killed diseased blood cells throughout the body but also destroyed the bone marrow, birthplace of new blood cells. By transplanting bone marrow … Continued

Grandfather Gets Back to Life and Nature after CAR T-Cell Therapy for Lymphoma

Jerry Jalbert is always up for new adventures. He did not expect that non-Hodgkin lymphoma (NHL) would be one of them, but with the help of CAR T-cell therapy that keeps his cancer at bay, the 73-year-old professional photographer-turned-baker-turned-forest checkpoint operator is continuing to enjoy life’s many journeys alongside his wife, Ethel. “We are not … Continued

Immunotherapy for Pediatric Solid Tumors: What’s the Latest?

Medically reviewed by Natalie Collins, MD, PhD New treatments that spur the immune system against cancer have entered the clinic to combat some forms of pediatric blood cancers, such as acute lymphoblastic leukemia (ALL). One form of immunotherapy, CAR T cells, has been approved for children and young adults with ALL. In treating solid tumors … Continued

For Father with Non-Hodgkin Lymphoma, CAR T-Cell Therapy Saves Memories and More

In May 2018, Tyler Goodwin underwent CAR T-cell therapy for follicular non-Hodgkin lymphoma (NHL). While he was having preparatory chemotherapy for the procedure, a surprise visitor to his hospital bedside provided a powerful reminder of why this promising new cancer treatment was so important to him. “It was my daughter Theresa, all made up for … Continued

New Study Reports “Curative Potential” of a Combination Therapy for Some Leukemia Patients

Medically reviewed by Matthew Davids, MD, MMSc Chemoimmunotherapy combined with a targeted drug given for two years has achieved undetectable minimal residual disease (MRD) for a high proportion of younger patients with chronic lymphocytic leukemia (CLL), Dana-Farber scientists report. The phase 2 clinical trial results are so favorable that they represent a step toward “a … Continued

Study Implicates Failure of DNA Damage Repair in BRCA1 Breast Cancer

Medically reviewed by David Livingston, MD It’s been known for decades that a mutated BRCA1 gene in a woman’s breast cells sharply increases her lifetime risk of breast cancer. What has eluded scientists, however, is a detailed understanding of how the loss of BRCA1 function leads normal breast cells down the path to malignancy. Now, … Continued

Single-Cell Sequencing Reveals Glioblastoma’s Shape-Shifting Nature

This post was originally published on Discoveries, the blog of Boston Children’s Hospital. Glioblastoma, a cancer that arises in the brain’s supporting glial cells, is one of the worst diagnoses a child can receive. The grade IV, highly malignant tumor aggressively infiltrates healthy brain tissue, and most children die of the disease within one to … Continued

Scientists Identify Genes Tied to Increased Risk of Ovarian Cancer

Medically reviewed by Alexander Gusev, PhD A team of Dana-Farber scientists and their associates has identified 34 genes associated with an increased risk of developing earliest-stage ovarian cancer. The findings, published in the journal Nature Genetics, will both help identify women who have the highest risk of developing ovarian cancer and pave the way for identifying … Continued

Double Strike Against Tumor Protein Shows Promise in Non-Small Cell Lung Cancer

Medically reviewed by Pasi A Jänne, MD, PhD Imagine inserting a key to shut off an engine only to find that it no longer fits — that the configuration of the lock has been changed without notice. Scientists developing targeted therapies to treat cancer often face a similar conundrum. Targeted therapies derive their effectiveness from … Continued