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May 28, 2014

Knowledge Is Power



With every holiday, every gathering, I meet fellow cancer patients. Not just myeloma patients, but people with all types of cancers. They know about my journey and reach out to me as both a knowledgeable supporter and fellow survivor.

Their stories can inspire, but many are heartbreaking. Perhaps the hardest part of my job is hearing stories like the one I heard this Memorial Day weekend. “Kathy, I have been battling ovarian cancer for many years, but I am seeing the end in front of me. My doctor says I may have years, but they have just run out of options.”

Years ago I might have gone along with this, but today one of the greatest revolutions in oncology is taking place in the form of advances in genomics and immune therapy. It is becoming more important that cancer patients know the right questions to ask and the right steps to take to be proactive with their disease. There are so many new therapies emerging and new technologies that are accelerating cures. Patients can play an important role in their own treatment along with their doctors.

With the 50th American Society of Clinical Oncology (ASCO) annual meeting coming up this week, there is no better time for patients to embrace this education. ASCO takes place once a year and provides the latest information on research and treatment across many cancers.

While the information is complex, many foundations and the press break it down to simpler terms. In addition, patients can go to the ASCO website directly to see updated treatment information, new clinical trials and leading centers and experts who conducted the work.

Fortunately for multiple myeloma patients, our team will be attending ASCO, and we will be feeding critical information to you via www.themmrf.org/asco with blogs, through our social channels and webcasts. Thanks to great teamwork, superior scientists, and cutting edge companies, progress is happening fast in myeloma and other cancers. Together we are transforming the world of cancer research as we know it. Please stay informed so you can make a difference for yourself and others on this journey of hope and promise.




February 25, 2014

Impatience is a Virtue





Anne Quinn Young, MPH, Marielena Mata, PhD, Principal Scientist at Janssen, Kathy Giusti, and Peter Lebowitz, MD, PhD, Oncology Global Therapeutic Head of Janssen


When Janssen Pharmaceutical Companies of Johnson & Johnson asked me to speak at their all-employee oncology meeting, I debated the best message to deliver. They had explained this was a diverse group of scientists and business leaders from all over the world.

Once again, it was the words of a patient that reminded me of the most critical message I could deliver to this enthusiastic, hard–working team: Urgency. Janssen has a long history with the MMRF in myeloma. They handle the international sales and distribution of Velcade, oversee Doxil, support our CoMMpass trial and work with us on new diagnostic approaches.

But most critical is their pipeline. We continually study every company’s pipeline searching for new drugs that might work against specific targets. Janssen has several drugs in early testing in myeloma; drugs the MMRF and our patients are following closely, such as daratumab, ibrutinib and other targeted compounds. And while they look promising, the question we answer most is, “how soon will it be available” and “can I get it?”

To me, the most important part of our job at the MMRF is to work with amazing partners in the industry to understand how we can accelerate their efforts. It may be testing their compound in our clinical network, the MMRC. It may be identifying and confirming new targets with our tissue bank and genomic efforts. It may be identifying patients for precision-based trials. Whatever we can do, we are “In”, because the words of my dear friend and fellow patient always ring in my ear when she says to me, “The greatest frustration I’ve faced is not the fact that I’ve never been in remission, it is not the side effects I’ve endured, it is knowing that a great new drug is out there and I won’t see it in time.”

We can’t let that happen. So, I closed my speech to this wonderful and engaged group of MMRF partners by reminding them that “every month, every week, and every minute” their passion and commitment makes a difference. I urged them to remain as “impatient” as we are about finding a cure and let them know how important it is that by working together we can bring this pipeline of therapies to the thousands of patients who desperately need hope and help. This is progress. This is how cures are found.




November 18, 2013

24 Hours…



Last night I spoke with a patient and thought, ‘Wow, we are getting it right.’ I was talking with a young myeloma patient who made it to a top myeloma center, saw a leading myeloma clinician and was entered into our MMRF CoMMpassâ„  Study. He had made all the right moves, taken the perfect first steps; his genomics were sequenced and subtype identified. He learned that he had a high risk form of myeloma called deletion 17 (Del17p) and his medical team would be watching him carefully, adjusting his treatment and evaluating new drugs and trials that might help him down the road.

This morning I was meeting with our MMRF team who is working tirelessly to identify new treatments for this unique subtype and talking with any and all pharmaceutical companies that would consider developing a drug for such an uncommon cancer like myeloma. Unfortunately, our lead company told us they didn’t have the funding or support to conduct a trial for a mutation that affects less than 15 percent of myeloma patients. It’s a crushing blow, but understandable since in some circles myeloma is still considered an orphan cancer and this particular subtype (Del17p) makes the market that much smaller.

Clearly, if we are going to provide drugs to small groups of patients with rare subtypes, we are going to have to pay for the trials ourselves, or at least a good portion of it. The cost of managing a portfolio like this runs in the millions, at a time when raising dollars seems to be the hardest thing we do.

I once believed, but am no longer as optimistic, that we could begin to open precision based trials in 2014.

Late this afternoon I received an email from a family we have been helping over the past number of months. It was titled, “Out of time.” Sadly, this patient with a known subtype has run out of options. We’ve done everything we can, but in our world sometimes that’s just not enough. We know too well the need is enormous. I’ve seen it with my own eyes patient upon patient where the heartbreak of this disease is indescribable. And her husband’s email broke our hearts once again.

Driving home this evening I’m thinking: We move fast at the MMRF. We know what to do and we know how to do it. Building models, driving results—we do it every day. I can’t believe that the critical path is now the funding. If only there were more people to ask. If only there were more hours in a day…




July 8, 2013

“YOU” are the Champions!





“If physicists and mathematicians can do it, and archeologists can do it, why can’t biomedical research do it?”
Biomedical Researcher, White House, “Open Science Champions of Change”


Do what? Share data for the benefit of the people who need it most – those battling diseases like multiple myeloma. But thanks to you – our champions – all of our partners, staff and investors -- we are leading the way. In fact, we were recognized recently at the White House during the Open Science Champions of Change ceremony on June 20th for helping make “open” the “default” in scientific research.

During the event, nearly every other honoree, particularly those working in biomedical research, acknowledged that the current incentive structures in academia and industry do not reward researchers for sharing data. Yet, they also uniformly agreed that opening up and sharing data is critical to making progress, and that scientists need outlets to facilitate data sharing and collaboration, like our MMRF Researcher Gateway.

The collection of a patient’s clinical and genomic data over time is the most important thing we can do to understand which drugs work and why. Unfortunately, many in the life-sciences industry are not sharing data, and even many patients don’t realize how important it is to share this information in an open environment.

It was a true honor to represent the MMRF as one of 13 recipient leaders from the many disciplines that spanned biomedical research to archeology, and from the humanities to astronomy, who was given this prestigious award in recognition for using “open scientific data to accelerate progress and improve our world”. The MMRF was recognized for our CoMMpass study, the landmark program designed to uncover the molecular segments and variations in multiple myeloma.

We at the MMRF applaud the White House “Champions of Change” for shining a spotlight on how a more collaborative, open-science-based approach to research, facilitated by data-sharing, will radically improve our understanding and treatment of cancer. That knowledge can then be used to develop individualized treatments that extend lives and offer the promise of lifesaving cures.

Of course none of this work would be possible without the staunch support of our friends and partners. Thank you for your ongoing, lifesaving engagement.

I leave you with this quote from a fellow researcher at the White House. It reinforces our mission and confirms the path we have chosen.


“The concept that patients are primarily voyeurs is an odd thing when you’re trying to work on disease.  You have to recognize that crowds, working as groups, almost always beat the individual lone wolf.”
White House, “Open Science Champions of Change”



June 20, 2013

Democratizing the Science, Accelerating the Cure

Multiple myeloma is a fatal blood cancer with a five-year survival rate of only 41 percent – one of the lowest of all cancers. As an organization, we have worked tirelessly to remove barriers slowing research into the disease and the progress we have helped make – namely six new treatments in the span of 10 years – has had a meaningful impact on patients. Despite the progress, however, all multiple myeloma patients will inevitably relapse, necessitating new lines of therapy that effectively treat the disease.

Significant challenges exist. Chief among these is multiple myeloma’s staggering genetic diversity. Advances in basic science have taught us that multiple myeloma, like most cancers, is not a single disease; rather, it is comprised of several distinct sub-types, each one defined by abnormal genes and disruptive proteins that allow cancer cells to thrive. Developing and matching patients to treatments that precisely neutralize these abnormalities is key to achieving longer-lasting remission and cures.

In 2011, we took a giant step forward to this goal with the launch of the MMRF CoMMpassâ„  study, a landmark, $40 million study that brings together competitors from pharmaceutical and biotech companies, academic institutions, and community cancer centers to work as one to define multiple myeloma’s subtypes and what drugs work best for different sub-types. 1,000 newly diagnosed multiple myeloma patients will be followed longitudinally over five years and their tissue samples, genetic information and various disease and clinical outcomes will be extensively analyzed. We believe that CoMMpass data will allow us to precisely match patients, based on their sub-type, with treatments that offer the best chance of long-lasting remissions with fewer effects and cures.

Importantly, all data from CoMMpass will be placed into a publicly available, open source, IP-free data ecosystem, together with data from other MMRF-driven initiatives and other datasets, to create one mega dataset that is unprecedented in its depth and breadth. We encourage anyone interested in using or contributing to this robust dataset to be part of our search for a cure. The MMRF Researcher Gateway, scheduled to be launched in September 2013, draws upon the wisdom and creativity of the crowd to advance discovery. Basic analytic tools make data accessible to a diverse group of users—scientists, pharmaceutical companies, health care organizations, physicians, and even patients—without requiring strong IT know-how. As a complement to this, we also developed the MMRF Community Gateway, also to be launched in September, that engages patients in the research process. The Community Gateway will create a dynamic, online community of myeloma patients that will allow patients to better understand their disease and treatment options, share their data together with their experiences, and take action by choosing to share their data with research studies and joining clinical trials.

On behalf of the entire MMRF team and our partners in CoMMpass, we thank the White House for recognizing the promise and significance of our approach and are honored to have been named an Open Science Champion of Change.




April 19, 2013

TEDMED 2013

Serendipity seated me next to Shirley Bergin, TEDMED COO, at a Forbes Healthcare conference. She was a force of nature with intense passion and that “connection” gene that compels her to learn your story and connect you with others who play in your field. A few months later, I was fortunate to join Jay Walker, TEDMED Curator and Chairman, and other healthcare leaders at the Walker Library and capture firsthand his unbelievable ability to tell a story. So I was not surprised to feel the energy of the entire team on the TEDMED stage as I attended my first meeting. As a patient, and as an entrepreneur, these are the meetings that inspire us to move forward, to continue challenging the system and finding new models and partners that can help lead to cures. So why should patients care that #TEDMED is here?

TEDMED provides context. #Cancer patients are unbelievably smart and courageous. We know our doctor, we know our treatment, we know the journey is often grueling; we know the toll it takes on our families. We don’t know how broken the system is, how unbelievably difficult it is to break it down and fix it. Hearing that the “publish or perish” mentality started 350 years ago helps us to know why this remains a huge challenge in academia. Knowing the funding limitations for start ups and NIH provides perspective-so does hearing firsthand the burnout of the physicians and medical teams. Today’s system is not easy for anyone. But I am confident that the more we know, the less patient we will be.

TEDMED accelerates solutions. So many of the TEDMED delegates are innovators looking for that “unmet need” where they can help break down the system. With new technologies in #genomics, #big data, #open access, mobile mobile, and new opportunities are opening up every day-ones that can place the patient at the center. Changes in the system are going to come from these entrepreneurs finding the partners and capital they need to grow quickly.

TEDMED puts the patient first. Whether hearing from a colon cancer patient who became a comprehensive source of data as n of 1, an oncologist who sees data as a “non-depletable resource” or the head of UCSF who is changing culture, it is clear: our journeys as patients are not just our own isolated journeys –they are sources of important data that must be shared. I will keep you updated as we unveil our groundbreaking open access data platform as described in this month’s issue of Fast Company in the coming months.