Precision Medicine and Myeloma

I recently attended the Cancer Moonshot Summit held at Howard University in Washington, DC.  A lot of talk at the summit revolved around Precision Medicine. Last year, President Obama announced the launch of the Precision Medicine Initiative (PMI) – an effort to accelerate a new era of medicine, focused on delivering more tailored health care. We are already using precision medicine in some areas of healthcare.  If you need a blood transfusion your blood is typed prior to the transfusion and you are given the exact match based on that result.  If you need to wear glasses you are given a prescription based on your current eyesight.

I am one of the few old-timers that still wear hard contact lenses. My prescription is not only based on my eyesight, but my eye care practitioner measures the exact shape of my cornea and prescribe lenses with the curvature, size, and corrective power that suit MY particular eyes. But in cancer, until recently, treatments were generic.  The PMI is hoping to make the one size fits all approach to cancer treatment a thing of the past.

Precision Medicine and Myeloma

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Precision Medicine in cancer is about giving the right drug or combination of drugs, to the right person, in the right amount at the right time based on a shared decision made by the patient and doctor. But how do we know who is the right person and what is the right treatment?  The answer may lie in Big Data. Big Data refers to extremely large data sets that may be analyzed computationally to reveal patterns, trends, and associations. Until recently this was not able to be done in cancer research because the technology needed to accomplish such a task did not exist. We now have the ability to sequence a genome in hours instead of days at an affordable price, the capability to store and share the enormous amounts of data that is being produced and computers such as IBM’s Watson that have complex computing algorithms to quickly interpret the volume and variety of the stored data. The time is now right- we have the perfect storm.


What “Big Data” is needed from the myeloma patient?  Tissue samples, bone marrow biopsies, and the results to any genetic testing such as Fluorescence in Situ Hybridization (FISH), Gene Expression Profile Scores (GEP), and Next Generation Sequencing (NGS) is some of the meaningful data that should be entered into the computer network. Information about the patient’s socio-demographics, preexisting medical conditions, lifestyle and family history should also be added to the data set. That same computer network then is used to keep track of the patients’ treatment protocols, side effect profiles, patient reported outcomes (PRO) and clinical response to treatment. Once the database becomes big enough by accruing thousands of patients the computer can begin to recognize patterns and may be able to predict who will respond to what therapy based on genomic characteristics and other analyzed information.

Does this sound familiar? Studies using big data are already being done in myeloma. They include the Multiple Myeloma Research Foundation’s (MMRF) CoMMpass Study for 1000 newly diagnosed myeloma patients and Celgene’s  ConnectMM- The Multiple Myeloma Disease Registry of 3000 newly diagnosed myeloma patients. A new study by Takeda entitled INSIGHT is about to open. INSIGHT is an observational study for 5000 patients which will included both newly diagnosed and relapsed refractory arms. After accrual INSIGHT will follow a patient’s treatment and outcomes for 5 years.


A key to finding answers faster is to have very large sets of data.  This can be accomplished in a timely fashion by designing a system that encourages its member to share their data on an open network. Shared data could speed up innovations and therapies for patient.  At the Cancer Moonshot Summit Vice President Biden implored all present “to break down the silos that keep research away from the world and from one another.” Collaboration is essential. Vice President Biden said, “We need to improve the way we work together in medicine.” The Vice President stated that all other areas of science already work in collaboration. We must change the system that rewards competition. He also stressed we need to move forward with a sense of urgency.

In response to the Moonshot’s emphasis on data sharing, the American Society of Hematology (ASH) committed to develop an international Multiple Myeloma Data Sharing Platform.  ASH invited myeloma experts from the Multiple Myeloma Research Foundation, the Mayo Clinic, the Dana-Farber Cancer Institute, the University of Arkansas Myeloma Institute (MIRT), and international partners to take the first step toward designing this data sharing infrastructure. ASH hopes that this multiple myeloma data sharing concept could be an example of what can be done in all cancers.

President-elect of ASH Dr. Ken Anderson, a myeloma specialist from Dana-Farber, will lead the myeloma data sharing effort. Dr. Anderson said, “By freeing the knowledge that is trapped in the databanks of individual research teams, universities, countries, and continents, we will gain a deeper understanding of the ways that specific diseases respond to minute genetic variances. Through collaboration, cancer researchers can come together across specialties and really get a grasp of what truly works, how it works, and when we can best utilize existing and experimental treatments to conquer cancer in untold numbers of patients.”

We as patients can help too!  By freely sharing our de-identified data in cancer registries and by asking about and enrolling in clinical trials when they are a viable option we can help advance the goal of the Cancer Moonshot Initiative which is to double the rate of cancer research and progress. We are all in this together. The Cancer Moonshot Mission will only be accomplished if ALL stakeholders including patients collaborate. If we could land a man on the moon, we surely can end cancer sufferings as we know them. We are on mission for the cure.  Let the countdown begin.

“Alone we can do so little, together we can do so much.” –Helen Keller

Cindy Chmielewski Precision Medicine Advocate

Cindy Chmielewski Precision Medicine Advocate

Cynthia Chmielewski was diagnosed with multiple myeloma, a blood cancer, in 2008. Cindy’s induction therapy stopped working after a few cycles and she proceeded with a stem cell transplant which failed to put her into remission. Depressed and scared she continued her fight using newly FDA-approved targeted therapies which eventually put her in remission. Cynthia continues treatment with a maintenance protocol.  Cynthia is using her passion for education to teach a new group of “students” – myeloma patients, their caregivers and others interested in myeloma.  She is a trained mentor, advocate and Patient Ambassador.