I spent last weekend covering the American Society of  Hematology (ASH) annual meetings in Atlanta.  ASH is the largest gathering of hematologists in the world each December.

I was covering the conference on behalf of the International Myeloma Foundation (IMF), so most of my focus for five jam-packed days was multiple myeloma research.  But I did have a chance to follow other ASH news as well.  Since this is more of a lifestyle site, I don’t post much here about conventional medical advances unless they are possible game-changers.  I would like to share news about several impressive new blood cancer drugs coming out of ASH.  Watch for my reports over the next few days.

To start, a new leukemia drug, ponatinib (Iclusig), was approved today by the FDA for use against “chronic myeloid leukemia (CML) and Philadelphia chromosome-positive acute lymphoblastic leukemia (ALL) in adults.”

According to Med Page Today, “Approval was based on a single phase II trial, results of which were reported last week at the American Society of Hematology’s annual meeting.”

Here is an excerpt from today’s Med Page Today article about ponatinib:

In the 449-patient study, called PACE, rates of major cytogenetic and hematologic responses to daily oral treatment with ponatinib ranged from 33% to 70%, depending on the disease subtype. Toxicities were generally mild, except for a few patients who experienced serious pancreatitis.

The drug was designed to get around one of the most common mutations that confer resistance to tyrosine kinase inhibitors (TKIs), the “gatekeeper” T315I substitution that blocks binding for all three currently approved drugs: imatinib (Gleevec), dasatinib (Sprycel), and nilotinib (Tasigna).

Ponatinib is considered a pan-BCR-ABL inhibitor because it is effective against native and all tested mutant forms of the BCR-ABL protein produced by the Philadelphia chromosome, which gives rise to CML and Ph-positive ALL.

Patients in the trial had previously failed other tyrosine kinases, with most showing resistance or intolerance to dasatinib or nilotinib.

But the FDA’s announcement of the approval did not say that patients should not receive ponatinib as first-line treatment.

Response rates in the PACE trial were highest in patients with chronic- and acute-phase CML, ranging from 50% to 70%. For patients with blast-phase CML or Ph-positive ALL, response rates were in the 33% to 35% range…

Those are some pretty impressive numbers for any cancer, let alone stubborn, refractory cases of CML and ALL.

CLICK HERE to read more of Senior Editor John Gever’s article, including a side effect profile.

Tomorrow, I will share some practically miraculous results from a new immunotherapy drug for children.

Feel good and keep smiling!  Pat


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