Sorting out the most important news from the ASH Meeting in Atlanta this past weekend is like trying to drink from a fire hose. There were too many trials with too many new drugs to cover in any detail, so I'll just give a broad overview from my perspective.
It is interesting how my interests have changed since the 2011 ASH Meeting. Then I was most concerned about the MLN9708 trials and other drugs for treating newly-diagnosed patients, as well as questions about when or if to undergo an autologous stem cell transplant (ASCT). With all that now behind me, I have turned my attention to what progress is being made in treating relapsed/refractory MM (RRMM), a situation I will no doubt be facing one of these days. One thing remains constant: I am still looking for news on treatments and prognosis for high-risk patients, such as myself.
While there were no dramatic breakthroughs announced at the meeting regarding a potential cure for MM, excellent clinical trial results were presented for Kyprolis (carfilzomib) and pomalidomide. Not only were these results as good or better than for the now-standard Velcade and Revlimid, both as initial therapy and after relapse, but they both also worked for patients who had become refractory to either or both of them. This is good news for RRMM patients in the future, most of whom have been treated by one or both of these. For myself, now that I am on a long-term Revlimid maintenance therapy, it is comforting to know that if that stops working, pomalidomide may be available to take up the slack.
I was pleased to note that the MLN9708 trials are being very successful for both induction therapy and for RRMM patients. These positive results indicate that it may be approved by the FDA as a standard treatment for MM within the next two years or so. Along with Kyprolis, MLN9708 has a real prospect of displacing Velcade in the future as a drug of choice for treating both newly-diagnosed and RRMM patients. Both of these drugs minimize the periperal neuropathy (PN) associated with Velcade, and the MLN9708 has the advantage of being given orally. Great news! I'm so glad I was able to participate as one of the first clinical trial volunteers for MLN9708 as a newly-diagnosed patient.
There are numerous other exciting new drug therapies being tested in clinical trials. A lot of promising results were reported at this ASH Meeting, but I'll spare you the details for now. (and, if you're lucky, I never will). Unfortunately, the initial trials for these drugs usually involve patients who have had multiple relapses and have become refractory to most or all of the available regimens. They are desperate for some kind of salvage therapy.
The first step in a new drug is to see whether it has an anti-MM effect when taken alone, so a trial such as this may be a last resort for some of these heavily-treated patients. A promising new drug may have an Overall Response Rate (ORR), which means Partial Response or better, of 20% to 30% when taken alone. This may not sound very good, but a drug needs to show some anti-MM benefit on its own, which will enable it to move to the next stage, where it will be combined synergistically in follow-on trials with other known MM drugs, such as Velcade, Revlimid, dexamethasone, or others. In the meantime, what happens to the 70% to 80% who didn't respond? Their options may have just run out.
When reading the statistics, it's easy to ignore the human side of all of this. Many of these heavily-treated patients are sacrificing their lives to advance the science for those who will follow them. It's really sad when you think about it. The hope is that with all the research and new treatment options becoming available, there will be fewer and fewer patients in this boat.
Tomorrow, we are attending an all-day Patient Symposium at Dana Farber, where the ASH results will be addressed in detail. I hope to come back with a more complete sense of what progress is being made in the fight against MM. I'll try to summarize anything interesting that I learn, once I've had a chance to digest it.