4 Promising Myeloma Treatments Bring Hope to Patients

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Day 2 of the 58th Annual ASH Meeting included exciting results from several studies of new therapies being examined in clinical trials; the MMRC, the clinical network of the MMRF, has played a critical role in accelerating a number of these agents forward. The overarching takeaway from all of the data was that myeloma is complex but highly treatable. Excitingly, several of the new treatments are for patients who have specific genomic features. The MMRF has been at the forefront of promoting this precision medicine approach, identifying the many molecular subtypes of myeloma and helping patients receive the right treatment for their own disease. Today’s talks highlighted the benefit of this approach.

Selinexor
Patients: Relapsed/refractory myeloma
How the drug is given: Oral
Given with: Low-dose dexamethasone
Key results: 21% ORR

This phase 2 study was conducted through the MMRC; the MMRF has supported the development of this treatment since before it entered clinical trials, having seen the promising preclinical data for this novel treatment. It analyzed the ability of selinexor to treat myeloma in patients who are refractory to four standard therapies: Velcade, Kyprolis, Revlimid and Pomalyst (or “quad refractory”). Some patients were also refractory to an anti-CD38 antibody, predominantly Darzalex, (or “penta refractory”). Patients had received an average of seven previous treatment regimens, selinexor showed impressive effectiveness in this highly refractory patient population. Side effects were primarily low blood counts and GI-related. This is the first study to show an effective therapy in the “penta refractory” group of patients, and the trial is enrolling for this group.

Venetoclax (Venclexta)
Patients: Relapsed/refractory myeloma
How the drug is given: Oral
Given with: Alone as a single drug
Key results: 40% ORR in patients with a translocation of chromosomes 11 and 14 (t11;14)

The drug venetoclax (Venclexta) recently received FDA approval to treat a specific type of chronic lymphocytic leukemia (CLL). Led by Dr. Shaji Kumar from the Mayo Clinic, the MMRC participated in this phase 1 trial in patients with advanced, relapsed or refractory multiple myeloma. Overall results showed that venetoclax significantly reduced the tumor size in 21% of patients, but that patients with a specific genomic change, specifically the translocation of chromosomes 11 and 14, t(11;14) responded best. 40% of these patients had a reduction in tumor size. The most common side effects were GI-related; the most serious were hematologic, primarily thrombocytopenia (low platelet count) and anemia. Trials are now being planned that will specifically target t(11;14) patients, underscoring the potential for precision medicine approaches.

Pembrolizumab (Keytruda)
Patients: Relapsed/refractory myeloma
How the drug is given: IV infusion
Given with: Pomalyst (pomalidomide)) and dexamethasone
Key results: 56% of patients had a significant reduction of myeloma cell counts

Pembrolizumab (Keytruda) is an immunotherapy that is FDA approvd to treat other types of cancer, including lung cancer and melanoma. It is one of the checkpoint inhibitors that help the immune system recognize and kill the myeloma cells (as opposed to the myeloma cells hiding from the immune system). This phase 2 study analyzed the combination of pembrolizumab, Pomalyst, and dexamethasone in patients who had received an average of three prior lines of therapy. It reported promising results in patients who had received a median of three prior treatments, and many of whom had high-risk disease features. Side effects were primarily low blood counts, as well as shortness of breath and dizziness. Several patient required a lower dose of Pomalyst. It is currently being evaluated in a phase 3 clinical trial in the combination vs. a control of Pomalyst and dex.

Nelfinavir (Viracept)
Patients: Advanced, refractory myeloma
How the drug is given: Oral
Given with: Velcade and dexamethasone
Key results: 65% of patients responded to the treatment (also called the objective response rate, or ORR)

The drug nelfinavir, currently being used to treat HIV, was studied in combination with Velcade and dexamethasone (NVd) in patients with advanced myeloma that was refractory to proteasome inhibitors, ie, no longer responded to proteasome inhibitors like Velcade and Kyprolis. Nelfinavir targets may targets a pathway to help resensitize myeloma cells to proteasome inhibitors so they can work better. This phase 2 study examined whether the three-drug combination (NVd) worked to kill myeloma cells over six 21-day cycles and how safe these drugs were. Researchers found that even in patients who had already received many types of therapy (five or more), these drugs were effective in treating most advanced, refractory disease. Side effects were found to be manageable, primarily low blood counts and increased risk of infections.

Check here for daily updates from MMRF during ASH to stay updated on the very latest myeloma research.

Posted: December 05, 2016

6 thoughts on “4 Promising Myeloma Treatments Bring Hope to Patients

  1. Sharon Ben-Meir says:

    Are any new therapies particularly effective for high risk MM with 17p deletion? Thank you for your life-saving work!


    1. MMRF says:

      Hi Sharon – While we direct your question to the appropriate resource, I recommend you contact one of our dedicated nurse specialists who should be able to provide you with information. They can be reached five days a week at 1-866-603-MMCT(6628)


  2. Lance Line, MD says:

    As a 54 year old resistant MM patient who did not achieve deep remission after autologous SCT, has not responded to Velcade, Revlimid, Dex combo, and is looking down the barrel of an unwanted allogeneic MUD transplant, I am very interested to know if I should stay the course of immunotherapy agents and risk my MM becoming even more stubborn or surrender to the advised allogeneic procedure. I am otherwise healthy, in great shape generally, have IGA nephropathy which has not yet been an issue in treatment, and a positive attitude. Would love to hear some arguments pro and con, immunotherapy or transplant.


    1. Rick Pahl says:

      I to am relapsed 9 months after ASCT and am wondering if an allotransplant is best for me. I’ll consult with my Oncologist. There are a number of treatment options but how to choose is the real bind. I will seek inclusion in UofMs molecular profiling initiative which will yield clinical grade sequencing in 10 days with clinical[y actionable results.


    2. MMRF says:

      Hi Lance – We are sure that you would find some interesting perspectives on both sides. If you would like to hear another perspective, we recommend you contact one of our nurse specialists who will be able to provide you with a little more information. They can be reached five days a week at 1-866-603-MMCT(6628)


  3. Joseph Iuzzolino says:

    As an 86 2/3 year old. Diagnosed about 5 years ago with MM, I have been doing great with immunotherapy which started once a week X 8 sessions, every other week X 8 sessions, once a month
    X 8 sessions, now in monthly treatment, indefinately.
    Drugs: I start by taking two Tylenol orally, then, IV Darzalex, Decadron, Benadryl.
    The worst side effect that I’ve experienced is that I can’t sleep the first night only. My Oncologist and I are extremely happy with the results.


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