Rooting Out Leukemia

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Just one day before leaving for a week-long trip to Hawaii together with his family, Joel Rutstein found out he has Myelodysplastic syndrome (MDS).  During a routine check-up, his doctor noticed some irregularities with the hemoglobin levels and decided to do some more investigations.  The bone marrow biopsy confirmed the terrible diagnosis.

MDS is a condition where blood-forming cells in bone marrow become mutated so instead of making healthy blood cells, they produce defective or immature ones, called blasts, which are dangerous and end up accumulating within blood vessels.  They often die prematurely and the body also destroys a good number of them, effectively leaving the person without enough normal blood cells.  In about 1 in 3 patients, MDS can progress to a rapidly growing cancer of bone marrow cells called acute myeloid leukemia (AML).

The standard course of treatment involves chemotherapy and, if necessary, a bone marrow transplant.  Unfortunately, most patients over 60, like Joel, cannot handle this intensive treatment.  Despite being in great physical shape, as he swims most days, he was still considered an imperfect candidate for this treatment.

When I was diagnosed, we heard about other people we know who had MDS”, said Joel.  “One of them was a woman my brother knew, whom we had met, who was living in the Boston area.  She started getting chemotherapy.  Her MDS morphed into leukemia and she eventually died”.

While effective in many cases, chemotherapy has very limited success against cancer stem cells.  It can eradicate most of the run-of-the-mill cancer cells, but the “root” of the problem, the leukemia stem cells, more often than not return, bringing back the disease.

He did not know it at the time, but being ineligible for that treatment allowed Joel to take part in a groundbreaking research program at the Anschutz Medical Campus.

Craig T.  Jordan, Ph.D., Chief of the Division of Hematology and one of the main investigators at Colorado University explained that “the way leukemia stem cells make energy is different than how normal stem cells make energy.  And we found that a drug called venetoclax stops them from making energy in this way, without harming the mechanism that normal stem cells use to make energy”.

As the low-intensity chemotherapy with Azacitidine was used to kill the bulk of the cancer cells, Venetoclax was administered to block the leukemia stem cells’ ability to make energy.  After the short course of chemotherapy ended, Joel stayed on venetoclax.

Blood counts recovered, blast counts went down.  Eventually, by early fall, blood counts and even hemoglobin were back to normal”, told Joel.  “Since I can live a normal life with this drug, I don’t have any interest in going off of it”.

Among the 33 patients enrolled in the trial, all past 65 years and unsuited for the standard treatment, 91% of patients achieved an overall response.  All had a poor prognosis, but now many continue to be in a durable remission.

My story isn’t a real exciting one.  There’s no car chase, no femme fatal.  I don’t know why I happened to get diagnosed when I did or why this treatment happened to come around when it did.  I just know that if I weren’t on this drug program, I wouldn’t be here” noted Joel.

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