Patients with rare cancers often face late diagnoses and few—if any—treatment options. Recently, in partnership with more than 20 rare cancer foundations, the Broad Institute of MIT and Harvard launched an international effort to help turn the tide on rare cancer research and discovery.
Though the treatment of childhood cancers has improved in recent decades, some forms of the disease remain difficult to crack. Among the most notorious are diffuse intrinsic pontine gliomas (DIPG)—aggressive brain tumors diagnosed in 300 children a year in the U.S. No therapy currently exists for DIPG and average survival after diagnosis is nine months.
Alison Anderson thought she had beaten cancer. Diagnosed with early-stage breast cancer in 2008 at the age of 38, she underwent the standard therapies for a woman under 40 with stage I disease and had done well.
Levi Garraway, an oncologist, used every technique in the precision medicine toolkit to treat his lung cancer patient. He sequenced the man’s tumor, identified a genetic mutation with a matching drug, and used the treatment to stabilize the patient’s condition. Eight months later, however, the tumor came roaring back to life.
Using a patient’s genetic information to tailor treatment is central to genomic medicine, and nowhere has genomics made a greater impact than in cancer. Thanks to advances in technology and affordable sequencing, oncologists are now able to customize treatments for some cancer patients based on the specific mutations of their tumors. However, limitations still exist: available drugs only work for a small number of patients and drug resistance often occurs.
MCL1 is one of the most common culprits in cancer. Multiple copies of the gene are often present in tumors, each encoding a protein that interrupts programmed cell death and fuels the growth of cancerous cells.