Jun 05 2016

Progress Gets Personal

by at 10:52 pm under Uncategorized

The flood of exciting results coming out of this weekend’s ASCO meeting is a powerful reminder that research and discovery are making a difference in the lives of people with cancer. Lombardi is well represented, as always. Immunotherapy continues to be a star of the show. For example, the combination of nivolumab (anti-PD1) and ipilumumab (anti-CTLA4) has important anti-tumor activity in small cell lung cancer, which has seen no major advances in over 25 years. An anti-CD38 antibody shows significant clinical activity in multiple myeloma. There is progress in molecularly targeted therapies as well, and evidence is beginning to emerge that the overall strategy indeed improves outcomes. There is even progress in adenocarcinoma of the pancreas, one of the diseases that gives cancer a bad name. Simply combining the standard agents gemcitabine and capecitabine yields significantly better survival in the adjuvant setting compared with gemcitabine alone.

Over the past several months, that progress has been demonstrated in a very personal manner. One of my elderly relatives was diagnosed with myelofibrosis, which is caused by JAK/STAT or calreticulin mutations, and leads to progressive fibrosis of the bone marrow. This causes anemia, pancytopenias and until recently was a very difficult to treat, accelerated chronic illness with a relatively short life expectancy. Individuals afflicted with this disease are typically tortured by relentless pruritus (itching) that is extremely difficult to treat. Lest you think that pruritus is a trivial toxicity, think about itching that robs you of sleep and sanity. It is no fun.

When my relative’s pruritus worsened, even as his hemoglobin rose and stabilized after treatment with recombinant erythropoietin, he commenced therapy with low, twice-daily oral doses of ruxolitinib, a JAK/STAT inhibitor. One week later his pruritus had nearly resolved. He is likely to do well for quite a while, and his disease is likely to remain controlled so he can maintain his active lifestyle. This is a small miracle – a reprieve – that could not have happened without the fundamental and translational research that led to the development of an important drug that relieves suffering and prolongs survival.

ASCO is a reminder that our work has broad impact that manifests itself in very particular ways, one person at a time. It reminds me of Lombardi’s Vision Statement of having local focus and global impact. Keep up the great work, and have a wonderful week.


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