BY PAUL KLEUTGHEN It is becoming increasingly hard to keep up with the many advances in the treatment of blood cancers. In the case of multiple myeloma, we have seen the advent of several new FDA-approved treatment alternatives and several more are at various stages of clinical effort. It seems that the field of drugs used is gradually shifting to monoclonal antibodies (Mab) so it was interesting to see a recently published article in the Blood Cancer Journal that not only provides a new gene treatment target of our disease but also provides encouraging, pre-clinical results of two potentially ‘small molecule’ drugs. ‘Small molecule’ drugs that we may be familiar with are Velcade or Revlimid. The article gets right to the point from the start :
‘Outcomes for many myeloma patients have improved over the past two decades with the introduction of proteasome inhibitors, immunomodulatory drugs and, more recently, monoclonal antibodies. However, high-risk disease, characterised by ⩾1 adverse cytogenetic features (t(4;14), t(14;16), t(14;20), 1q+, 17p−) or distinct gene expression proﬁles (for example, UAMS GEP70 score) remains therapeutically intractable, with little evidence that currently available therapies have improved patient outcomes. New treatment strategies are therefore urgently required.’
The authors identified that overexpression of the EZH2 gene has a negative effect on outcomes of MM patients. The authors collected peripheral blood samples of over 1,500 MM patients who were enrolled in late stage clinical studies and were able to conclude the following :
And now for the good news: the authors tried two recently discovered potent and selective EZH2-inhibitors that are currently used in early Phase I clinical studies across different types. The article indicates that :
The fact that the two compounds mentioned above are already in clinical trials is positive news in that safety and dose ranging evaluations will have already progressed which will help expedite the development timeline of either or both these compounds to give us another treatment target and treatment option in the, not too distant, future. Of specific interest is also the in-vitro response seen in cell/cell linings of high-risk myeloma.
about the author
I am a patient diagnosed in 2014 with primary plasma cell leukemia (pPCL), a rare and aggressive variant of multiple myeloma and have been very fortunate to find successful treatment at the division of Cellular Therapy at the Duke University Cancer Institute. My wife, Vicki, and I have two adult children and two grandsons who are the ‘lights of our lives’. Successful treatment has allowed Vicki and I to do what we love best : traveling the world, albeit it with some extra precautions to keep infections away. My career in the pharmaceutical industry has given me insights that I am currently putting to use as an advocate to lower drug pricing, especially prices for anti-cancer drugs. I am a firm believer that staying mentally active, physically fit, compliant to our treatment regimen and taking an active interest in our disease are keys to successful treatment outcomes.