Advancing Precision Medicine with Patient Data
March 4, 2022
While advances have been made in the treatment of the rare blood cancer multiple myeloma, fundamental questions about how to optimize therapies for individual patients remain. The Multiple Myeloma Research Foundation launched CureCloud, an initiative to gather detailed genomic and health data from thousands of patients to both bring a precision medicine approach to the treatment of multiple myeloma and fuel the development of new breakthroughs. We spoke to Michael Andreini, president and CEO of the Multiple Myeloma Research Foundation, about the CureCloud initiative, the patient data it is gathering, and the potential to transform the treatment of multiple meyeloma with precision medicine.
Daniel Levine: Michael. Thanks for joining us.
Michael Andreini: Great to be with you, Danny. Thanks for having me.
Daniel Levine: We’re going to talk about multiple myeloma, the Multiple Myeloma Research Consortium, and an innovative effort to accelerate research called the MMRF Cure Cloud. Before we get to the Cure Cloud effort, though, I wanted to take a step back for listeners who may not be familiar with the Multiple Myeloma Research Foundation and discuss that a bit. Perhaps we can start with multiple myeloma. What is that?
Michael Andreini: Sure. Multiple myeloma is a rare blood cancer that affects the plasma cells and there’s about 140,000 patients in the United States. Essentially malignant plasma cells start to accumulate in the bone marrow and produce an abnormal protein called M protein. The result of the disease is it starts to cause anemia, bone damage, impaired immune function, and other organ damage as the disease progresses.
Daniel Levine: How well treated is the condition today?
Michael Andreini: So, there’s been a lot of advancement in the space, luckily, since the Multiple Myeloma Research Foundation was created back in 1998 and there are a lot of different modalities to treat patients. There’s stem cell transplant, there are immunomodulatory drugs, proteasome inhibitors, steroids, and immunotherapy agents that have started to come to become more available recently, including car T-cell therapy. Essentially, these drugs are used in various combinations and sequences to treat patients over the course of their disease. But the overall five-year survival rate is still only about 55 percent for these patients. In the United States, about 12,000 patients unfortunately succumb to the disease every year. And so, while there’s been a tremendous amount of progress, there still is a lot more work to do. Certainly, our organization, the Multiple Myeloma Research Foundation, our mission is really to accelerate a cure for this disease for all patients.
Daniel Levine: It’s not unusual today to see patient organizations playing a critical role in driving research into a specific disease. But I think MMRF has really been innovative here. What was the state of treatments for multiple myeloma when the organization was founded and how has the work its done really advanced research and the development of therapies into the condition.
Michael Andreini: Sure. When the organization was founded in 1998, there wasn’t a lot of ongoing research in this space so the organization was really created by our founder, Kathy Guisti, who was also a patient, to really help accelerate the rate of research and advance a cure for this disease. There were not many treatments available at that time and the five-year survival rate back then was only about 35 percent. Now the five-year survival rate has advanced to about 55 percent and there have been 15 new drugs approved over that period of time, many in which the MMF played a role in helping them advance.
Daniel Levine: As you look at the multiple myeloma research landscape today, are there critical questions that still need to be answered?
Michael Andreini: Absolutely, and with so many treatments that are now available for patients, and there are going to be more treatments that come to the market even this year for patients. But there still is a fundamental question about how can we optimize these treatments for every patient given their unique disease characteristics and stage of disease? That to us is essentially a precision medicine based question, right? It’s what are the right combinations and sequencing approaches of these treatments? When should we give these treatments to patients and for which patients? And so ultimately the goal is to make sure that all patients really maximize the efficacy from all the available agents for them.
Daniel Levine: Let’s talk about the MMRF Cure Cloud. What is the Cure Cloud?
Michael Andreini: Sure. The Cure Cloud is an innovative direct to patient registry platform that’s essentially capturing longitudinal genomic data from a targeted gene panel from peripheral blood samples and clinical data from electronic health record information. And what’s really unique about this platform is that we’ve done this all in a clinical grade environment. So, we can actually return the result of this genomic testing back to patients and the treating clinicians to inform their overall care. And we actually match patients to potential clinical trials associated with the genomic alterations or identified through the test. So we do this report, obviously there’s a clinician facing port, but we’ve also partnered with a genomic counseling service to help us create a patient friendly version of that report so they can more easily digest the information. And again, the idea is to be able to use this information to inform care, have better discussions with their clinician.
Daniel Levine: What’s the range of data you’re seeking to gather?
Michael Andreini: It’s a great question. Right now, we’re capturing genomic data, again from that targeted gene panel of about 70 genes, and then we’re collecting clinical data from the electronic health record information. By clinical data, that’s treatment information, that’s lab data, that’s outcome data. We really think bringing those two pieces of data together are really going to be helpful in informing the next era of precision medicine breakthroughs that’s going to help us really be able to identify new targets for drug discovery, new biomarkers to inform care. Ultimately the idea is to help inform care pathways overall to make sure that patients are having the best possible options given this broad armamentarium for their treatment.
Daniel Levine: What’s the consent process like? Are you doing this electronically?
Michael Andreini: Yeah. The consent process is all done online electronically. It just takes a handful of minutes to complete. Essentially, patients can sign up, express their interest. They answer a few upfront questions to ensure the patient has active disease. That’s really the criteria for enrolling in the study. Tthen they review that consent form and sign it digitally. They also sign an authorization form for the MMRF to go out and procure their electronic health record information. Then also, patients have to have their clinician sign a blood draw order form to obtain the blood sample for that sequencing test.
Daniel Levine: And what demands are made on patients to participate? Are there other things they need to do once they consent?
Michael Andreini: We really tried to design this to ensure that we lowered as many barriers to participation as possible. We wanted to make this a seamless process and we wanted to make sure the burden on the patient was as low as we possibly can make it. Really the main onus on the patient is to go through and sign the consent and ensure that their treating clinician signs off on the blood draw order form. After that point, really the onus is then on the MMRF to make sure we’re doing the sequencing test, we’re delivering that back and procuring all of the electronic health record information for the patient.
Daniel Levine: And what happens to the research findings? Are they shared with the individual patients or are they shared with the community in any way?
Michael Andreini: Absolutely. In real time, because we’ve done this in the clinical grade fashion, we are returning results back to the individual patient that they can use for their own care, but also we’re aggregating and de-identifying this data and we’re sharing it a couple of different ways. One is we are sharing the data back to patients through various visualizations. One of the visualizations is essentially of the overall journey that that patient went on with their disease. The other is more of an aggregated kind of view of all of the data so patients can kind of explore the data set broadly and be able identify who are other patients that look like them that have the same kind of disease characteristics, how were they treated, what were their outcomes? We think that’s a helpful tool for patients to be able to understand the overall landscape of disease, and be able to have more informed decisions and discussions with their clinician about their care.
Daniel Levine: Do you expect those results to actually inform individual patient decisions, treatment decisions at this time?
Michael Andreini: Absolutely, with the clinical grade genomic report that we’re returning we are presenting patients and their treating clinician with clinical trials that they’ve matched to, based on a handful of genomic alterations that are actionable at this time. So, certainly those are options that they’ll need to evaluate with their clinician, but it’s something that is an option for their care in the immediate.
Daniel Levine: And who owns the data and who is the data being made available to?
Michael Andreini: Sure. In terms of real ownership over the data, myself and the broader MMRF, we really believe that patients ultimately own their own data. But certainly, as part of this research study, we at the MMRF become stewards of that data for research purposes. So, in terms of sharing it more broadly, we do intend to share the aggregated and de-identified data with researchers to help advance new discoveries. It’s important to underline that we would never share any identifiable information about patients. But certainly, the aggregated and de-identified data has a tremendous amount of value to the broader ecosystem in terms of helping to advance new scientific discoveries in pursuit of, again, our highest level mission, which is to accelerate a cure for this disease.
Daniel Levine: At the same time, would this help identify potential participants in a clinical trial?
Michael Andreini: Certainly, in terms of clinical trials that they are matched to from their genomic report. Certainly, in presenting those clinical trial options for patients that will present those options and potentially enroll patients in those trials. So absolutely, that’s certainly one of the benefits of the data.
Daniel Levine: And do you see any opportunity to leverage this data by layering it with other data sets?
Michael Andreini: Yes, we’ve started with this genomic data and the clinical outcomes data from electronic health records, but we really intend to expand the data that we’re collecting as part of this research study to be able to answer new and different research questions. So, one of the things that we’re actually planning to do this year is add patient reported outcomes data to the overall study—that survey data from patients on a variety of different aspects of their disease. It’s a nice tool to better understand individual patient journey and how they’re feeling, given certain treatments and where they are in their overall journey.
Daniel Levine: How is all this being funded?
Michael Andreini: This is funded primarily from philanthropy from donors and most of our donors and philanthropists within the foundation are our patients or patient families. We’ve also reached out to our partners in industry and asked for sponsorship funding to help pursue this overall study as well.
Daniel Levine: And where does the data sit? Is this on an existing platform? Is this something that MMRF has built specifically for this?
Michael Andreini: Sure. So, this was really built in a very bespoke way. And when I say, we at MMRF helped to build this, it’s really a large partner network that helped us do this—upwards of 10 various partners that helped us put this together. We essentially had to build our own architecture for this research project and integrate a sequencing lab, genomic counselors, molecular pathology lab EHR abstraction and curation, and mobile phlebotomy service, and stitch that all together to be able to execute this research initiative.
Daniel Levine: Is there any opportunity to leverage that work so other organizations might be able to benefit from it?
Michael Andreini: Absolutely. We’ve also been very active in making sure we’re proactively sharing our models for research at the MMRF. We’ve been engaged with several ongoing institutions that are looking to spin up other types of registry efforts that are similar to this. So certainly, we’ve been playing a role in helping to share and advise other organizations to do similar types of efforts.
Daniel Levine: As you think about this project, how does it fit into MMRF’s broader philosophy about patient driven research?
Michael Andreini: Sure. At the MMRF we’re big believers in taking risk driving innovation and being patient focused, being really patient-centric in our overall approach. So, this Cure Cloud initiative really fits into that. It hasn’t been done in the exact way that we’ve done this today. There was a risk in bringing that forward. We really do believe that this is an innovative model and because it’s direct to patient it is very patient-centric in the overall approach. Also, because we’ve been an organization that’s focused in advancing precision medicine, one of the core things you need to deliver on is data. You need to have a lot of data, deep, high dimensional data to be able to drive the right types of insights that are going to inform precision medicine approaches, whether that’s new target discovery or biomarkers or advancing care pathways. So, we’ve always made a lot of investment in generating very robust data assets, and then sharing them with the larger research community to drive as much insight and innovation and impact as possible.
Daniel Levine: Just from a broader vantage point, how do you see these types of research efforts changing the way research is done and really the potential for a patient organization like yours to have an impact?
Michael Andreini: It’s a great question. Nonprofit research organizations and patient centered researcher organizations have a very unique role to play because we’re unbiased third parties and not encumbered by a lot of different constraints that do impact other organizations like having to focus on profits in the industry, having to focus on publications and grant funding and academia. So, because we’re a little bit free from some of those constraints, or these types of organizations are maybe unique in having more access to patients than at any one other research institution, and can more freely share the data with other researchers. That’s one of the more common impediments that we think to advancing research—data is fractured and in lots of different silos. We think having larger, more robust data sets that can be accessed by more researchers is really important to driving progress.
Daniel Levine: Michael Andreini, president and CEO of the Multiple Myeloma Research Foundation. Michael, thanks so much for your time today.
Michael Andreini: Thanks for having me.
This transcript has been edited for clarity and readability.
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