The International Myeloma Foundation (IMF) Black Swan Research Initiative (BSRI) is moving into high gear in the new year.
Building on remarkable progress made by the BSRI team during the last months of 2013, our game-changing approach to finding a cure for myeloma has hit key scientific goals ahead of schedule. Additionally, the work is attracting significant financial support from both industry and private partners.
BSRI is the IMF's signature research project - a multinational consortium of leading myeloma experts who are studying and harnessing new technologies and myeloma treatments. The linchpin to these studies is the quest for determining when zero Minimal Residual Disease (MRD-Zero) has been achieved and sustained. This is where the most thrilling breakthroughs have occurred.
Professors Alberto Orfao and Bruno Paiva from Spain presented results of an automated, ultra-sensitive technique for MRD testing at an investigator meeting held in New Jersey in October. The "multiple" in multiple myeloma refers to the frequency of myeloma occurring in different patches or areas of bone. Based on this, they came up with a cocktail of eight antigens that identify myeloma regardless of its state or location in the body.
To eliminate another common variable - the human observer - a computer software program was developed to analyze the read-out from the new test. The sensitivity of the test is one part in 100,000, and probably even lower.
We Can Analyze Hundreds of Thousands of Events
At a follow-up BSRI meeting held before last December's annual meeting of the American Society of Hematology (ASH) in New Orleans, excitement about the new test was palpable. With this new test, "we can analyze hundreds of thousands of events, maybe millions," said Vincent Rajkumar of the Mayo Clinic. "No one person can do that."
In early 2014, BSRI investigators will host a workshop in Spain to train others to run the Multicolor Flow Cytometry test, as the new technique is called. Once trained, those researchers will return to their own countries to teach others. The next step is achieving consensus on the test, then incorporating the flow cytometry testing into clinical trials so that the testing may be validated prospectively in a standardized fashion.
Dr. Rajkumar predicted that within two to three years the software will be able to be run anywhere globally. "It's why the Black Swan Research Initiative can change the world."
To some of the investigators on the team, it already has changed. Jesus San-Miguel asked a provocative question during the discussion of this new testing protocol at the meeting in New Orleans: "My question is--how many patients are already cured?" He was referencing the concept espoused by BSRI that finding the pathway to a cure requires systematic evaluation and validation every step of the way.
The new cytometry test may soon identify patients who have indeed been cured - which in turn would pave the way for successfully replicating their particular portfolio of treatments. Cross correlations will prove very important in establishing a more finalized MRD testing panel. A key aspect of MRD testing and validation is to compare the flow and molecular methods, such as the Sequenta DNA method. Imaging and molecular testing will round out BSRI's testing protocol.
Five Major Objectives to Achieve Our Goals
There are five major objectives to achieve our goals: 1) Establish a standardized definition for MRD-Zero accepted by the International Myeloma Working Group (IMWG); 2) Standardize the new MRD tests themselves; 3) Validate MRD-Zero in retrospective datasets; 4) Integrate standardized/automated/validated MRD-Zero testing into trials at different disease stages; and 5) Use MRD-Zero for treatment decisions to achieve cure.
Our timeline to accomplish all of this is approximately three years. But progress may be swifter than we imagine. Leading up to the next BSRI investigator meeting in spring 2014 the team is working to establish a chain of publications that will introduce the new definition of myeloma, including ultra-high risk.
We must also better understand the nature of resistant sub-clones to achieve MRD-Zero.
And, of course, alongside our scientific efforts we are cultivating the financial support for the costly clinical trials and labor-intensive research required to find answers to our questions. This is crucial if we are to accelerate BSRI's efforts to find a cure for myeloma.
Fortunately, support has been forthcoming--a sign we are on the right path.
Multi-Year Collaboration with Onyx Pharmaceuticals
Last December, the IMF was thrilled to announce the start of a multi-year collaboration with Onyx Pharmaceuticals, Inc., an Amgen subsidiary Onyx is BSRI's inaugural industry partner and we are grateful for their early and enthusiastic support.
The IMF is equally appreciative of the support given by generous individuals such as IMF Board of Directors member John O'Dwyer and his wife Dorothy. The couple are BSRI Founding Donors and, taking his commitment a step further, John has kindly agreed to serve in the important role of Chairman of the BSRI donor campaign. The O'Dwyers are also joined by Andrew and Laurie Kuzneski in the BSRI campaign.
We are indebted to Loraine Boyle, whose Peter Boyle Research Fund--named in honor of her late husband--turned its focus this year to Black Swan at the 2013 IMF 7th Annual Comedy Celebration. Private donors have committed more than $625,000 to the initiative so far. And local member fundraisers have begun earmarking their contributions for BSRI. The proceeds from October 2013's Miracles for Myeloma 5K run in New Jersey, for example, will fund Bruno Paiva's study of Minimal Residual Disease in myeloma.
This is all great. We want as many members of the myeloma community as possible to participate--researchers, patients, caregivers and friends. Anyone who can contribute whatever they can, be it research, financial support, or raising awareness of our work, is welcome. By pulling together, collaborating, and expanding our minds to consider possibilities never thought of before, I am certain we will find a pathway to achieving a cure.
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