CLINICAL NEWS
Scientific Program Co-Chairs
Andrew W. Roberts,
MBBS, PhD
Professor
Walter and Eliza Hall
Institute of Medical
Research
The Royal Melbourne Hospital
Parkville, Australia
Akiko Shimamura,
MD, PhD
Professor of Pediatrics/
Hematology-Oncology
Adjunct Professor of
Medicine/Hematology
University of Washington
Fred Hutchinson Cancer Research Center
Seattle, WA
In your opinion, what are the events
and sessions in this year’s Scientific
Program that attendees need to add
to their agenda?
Dr. Shimamura: This year’s Special Scientific Symposia focus on the translation of
cutting-edge scientific findings to clinical
care. “The Special Symposium on Targeting
the Epigenome,” for instance, will focus on
recent insights into epigenetic mechanisms
of malignant transformation and the development of epigenetic-directed therapeutics.
The “Special Scientific Symposium on Genomically Engineered Stem Cells: A Brave
New World for Therapeutics” will explore
the exciting possibilities for the development
of novel cellular therapies that genomic
engineering of stem cells has opened up.
I believe many of the general issues with
developing and operationalizing individualized therapies are broadly applicable to
non-malignant hematology, as well.
Dr. Roberts: The Ham-Wasserman, E. Donnall Thomas, and Ernest Beutler lectures
are always must-sees for hematologists who
enjoy hearing from pioneers about how the
great advances in hematology came about.
This year’s line-up is no different.
NOW ENROLLING
Which areas of research do you think
will have the most exciting implications for clinical practice?
Dr. Roberts: As always, a number of
practice-changing clinical trials will be
reported in the Oral sessions,
Plenary Scientific session, and
Late-Breaking Abstract session.
Dr. Shimamura: The potential
diagnostic and clinical implications
of genome-wide analyses in guiding risk stratification and informing
treatment decisions are vast. This
technology is advancing at a rapid
pace – leading to decreased costs
and faster turnaround – and the
implications cut across malignant
and non-malignant hematology, as well as
hematopoietic stem cell transplantation.
How does this year’s program
differ from previous years?
Dr. Shimamura: A strong theme emerged
while we were developing this year’s Scientific Program: Advances in genomics
and genomic engineering are poised to
change hematology science and practice.
Many presentations will highlight the
implications for basic mechanistic understanding of hematology, opportunities to
develop novel therapeutics, and decisionmaking in clinical care.
Dr. Roberts: Also, our capacity to target
epigenetic regulators with drugs and to alter the genetic makeup of primary human
cells will be showcased in several areas.
What are some of the special sessions
designed for early-career hematologists and trainees? For educators?
Dr. Shimamura: The Continuing Conversations with the Speakers series will
provide attendees with the opportunity
to meet with experts in the field for a
small-group discussion. This format is
designed to foster dialogue and discussion, rather than didactic lectures. The
Scientific Spotlight Sessions, which
open with a short presentation by two
leading experts in the field followed by
interactive discussions with the audience, are also designed to encourage
in-depth dialogue about these topics. ●
The STORM Trial (KCP-330-012)
(Selinexor Treatment of Refractory Myeloma)
A Phase 2b, Open-Label, Single-Arm Study of Selinexor (KPT-330)
plus Dexamethasone in Patients with Multiple Myeloma Exposed to
Bortezomib, Carfilzomib, Lenalidomide and Pomalidomide and
Refractory to an IMiD and a Proteasome Inhibitor.
• Smoldering MM or Plasma cell leukemia
DIAGNOSIS AND MAIN CRITERIA FOR INCLUSION:
• Documented systemic amyloid light chain amyloidosis.
• Male and female patients, ages ≥ 18 years, with measurable progressive
MM according to the International Myeloma Working Group (IMWG)
Uniform Response Criteria.
• Active CNS MM.
• Patients must have received ≥ 4 prior regimens including (alone or in
combination) an alkylating agent, bortezomib, carfilzomib, lenalidomide,
pomalidomide, and a glucocorticoid, and that their MM is refractory to at
least one IMiD and one PI.
MAIN CRITERIA FOR EXCLUSION:
• MM that does not express M-protein or FLC (i.e., non-secretory MM
is excluded; plasmacytomas without M-protein or FLC are excluded).
• Pregnancy or breastfeeding.
• Radiation, chemotherapy, or immunotherapy or any other anticancer
therapy ≤ 2 weeks prior to Cycle 1 Day 1, and radio-immunotherapy 6
weeks prior to Cycle 1 Day 1.
• Not adequately recovered from the side effects of previous
antineoplastic agents prior to dosing.
• Refractory is defined as ≤ 25% response to therapy, progression during
therapy, or progression within 60 days after completion of therapy.
• Active graft versus host disease after allogeneic stem cell transplantation.
• At least 25% of the patients included must have MM refractory to an
anti-CD38 monoclonal antibody therapy.
• Major surgery within four weeks prior to Cycle 1 Day 1.
• Non-hematological toxicities (if any) from