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Finding May Lead to New Treatments for Neurofibromatosis

Common symptoms of the genetic disorder neurofibromatosis type 1 (NF1), including skeletal fragility and the loss of bone mass, may be treatable with an existing anti-cancer drug, according to a study from researchers at Weill Cornell Medicine. The findings reveal the potential for an expanding array of therapeutic options for patients who have this difficult-to-treat condition.

Matthew B. Greenblatt, MD, PhD
Associate Professor of Pathology and Laboratory Medicine

The study, published Nov. 11 in Nature Communications, uncovered a signaling pathway in bone-making cells that helps drive the skeletal manifestations of NF1, and showed that a drug called ponatinib, which is already in use against certain forms of leukemia, can largely prevent these skeletal manifestations in mice with the disorder.

NF1 currently has almost no drug treatment options, and although doctors are beginning to use drugs called MEK inhibitors to treat the disease, the findings suggest that ponatinib or future novel drugs targeting the newly uncovered pathway may be able to treat NF1 more effectively and safely.

“Ponatinib may have some clinical value here, and we also can start screening for potential new drugs that work even better than ponatinib against this newly discovered pathway,” said senior author Dr. Matthew Greenblatt, an associate professor of pathology and laboratory medicine at Weill Cornell Medicine and a pathologist at NewYork-Presbyterian/Weill Cornell Medical Center.

NF1 afflicts about 1 in every 3,500 people, or roughly 100,000 people in the United States. Signs and symptoms, apart from skeletal manifestations, include tumors—mostly benign—on peripheral nerves, called neurofibromas, and learning difficulties in childhood.

Research Reveals Why Some Tumors Have Different Makeup of Cells

Molecular changes in cells called fibroblasts, which help provide support for tissues throughout the body, may explain why one type of colon cancer doesn’t respond to therapy, according to a team of researchers from Weill Cornell Medicine. Targeting these cells may be a way to make treatment more effective.

Jorge Moscat, PhD
Professor of Pathology and Laboratory Medicine

In a study published Nov. 17 in Developmental Cell the investigators examined cells called fibroblasts in CMS4, the most aggressive and difficult-to-treat form of colorectal cancer, to determine how these cancer-associated cells acquire traits that allow them to support malignancy in neighboring cells. CMS4 affects about a third of all colorectal cancer patients.    

“There are two important components to this study,” said co-senior author Dr. Jorge Moscat, Homer T. Hirst III Professor of Oncology in Pathology and Vice-Chair for Experimental Pathology. “First, we have shown in a mechanistic way how these cancer-associated fibroblasts acquire the characteristics that they have. Second, we confirmed that what we discovered in our lab models also applies to patients, which begins to suggest how these findings could be useful in the clinic.”

Tumors are made up not only of cancer cells, but many other kinds of cells as well. These other cell types can influence how a tumor behaves and can have a profound effect on whether it responds to therapy, including treatment with immunotherapy.

Dr. Massimo Loda Receives SBUR Meritorious Achievement Award

Massimo Loda, MD
David D. Thompson Professor & Chairman of Pathology and Laboratory Medicine

Congratulations to Dr. Massimo Loda for receiving the Meritorious Achievement Award from the Society for Basic Urologic Research (SBUR). 

Presented annually at the fall meeting, this award is to recognize a researcher (can be a clinician researcher) who has made significant contributions in the field of urological research. Accomplishments of note should include Publications, participation on distinguished panels, Advisory roles, overall contributions to the field of urology research.

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