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New Pathways and Approaches for MM Treatment

| Jim Omel

At ASH, I always look for new ideas and approaches for treating myeloma.  What is new?  What is coming, and what is close to being clinically useful for patients?  Every drug has its start in pre-clinical testing (cell lines), moves on to animal testing, and then makes the move to phase I safety and dosage evaluation in humans.  From there, of course, we become more and more interested as the agent moves through phase II and III testing.  A few of these compounds will make it to FDA acceptance.  We patients are just as excited at a pharma company’s success as they are.  To a company, success means financial viability.  For patients, success means a greater chance of surviving multiple myeloma.

There were some oral abstracts explaining new treatments such as Filanesib by Drs. Shah and Zonder.  New approaches outlining CAR-T cell therapy are very promising.  Most of the oral abstract presentations on myeloma, however, were a bit dated.  We learned, for instance, that phase I data on Ninlaro (ixazomib) is quite positive, as are early data on Darzalex (daratumumab) and Empliciti (elotuzumab).  These phase I trials are vital to any drug’s success, and they take time to accrue and ‘mature’ (or rather, to allow time for results to occur).  They are extremely important, and I certainly do not want to minimize their importance.  Without them, we would never have success and new product launches. However, it was hard to be really excited at oral presentations when we already know the outcome.  The speakers and their great presentations were the victims of their own success. Thank goodness all three drugs passed phases I, II, and III have been t zipped to formal FDA approval last month.

Chimeric Antigen Receptor (CAR)-T lymphocyte therapy is certainly promising for patients.  This totally new approach channels our own individual immune systems and has shown significant effectiveness in the very few cases where it has been used in myeloma. However, I seriously question the practicality of this amazing treatment scheme for the vast majority of patients.  Consider the word ‘individual’ because that is the way CAR-T cells must be made.  Each engineered cell created is specific to the person from whom it was extracted, grown, expanded in the lab, and re-infused back into the patient.  It is time and labor-intensive and enormously expensive.

Current CAR-T cell production can be thought of as the opposite of mass production, and I will use the analogy of producing automobiles to illustrate this point.  Before Henry Ford developed his assembly line, each auto was produced one at a time and each part and piece was manufactured individually for that vehicle.  It was not practical for the vast numbers of consumers wanting this new way to travel.  Ford’s mass production line changed everything, and his Detroit plant manufactured dozens (hundreds?) of cars every single day.  It made automobiles available and affordable for the average citizen.  Likewise, CAR-T cell therapy is at the same point of infancy as auto production pre-Henry Ford.   It holds promise for true CURE for one patient at a time.  It is a great innovative approach, and we all cheer for success.  While it is scientifically exciting, I wonder how practical it will be for any given patient diagnosed and facing treatment in the office of their community oncologist.

ASH posters and presentations provided optimism for the future.  We should all follow Dr. Durie’s cautious approach for any given treatment.  When HE is optimistic, then we patients can be too.  The best is definitely still ahead of us.

Follow Jim on Twitter: @IMFjimMYELOMA
http://www.grandisland.support.myeloma.org
The Central Nebraska Myeloma Support Group
Grand Island, NE

One Response to "New Pathways and Approaches for MM Treatment"
  1. Very well said, Jim. Thank you so much
    IMFHughMYELOMA

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