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May 19, 2009

Translational Research – Under Construction! Louise M. Perkins, Ph.D., Chief Scientific Officer

How should the next generation of medicines be discovered and advanced to clinical testing?  Who should pay for and conduct this work?  These were among the key topics at the Translational Medicine Alliance Forum that I attended in Philadelphia on May 14 and 15.  This forum brought together thought leaders from foundations, academia, government and the private sector to identify barriers, possible solutions and the role that each of these key stakeholders should play in this important field.

So, what is translational research and why is it so important?  It is the process of advancing new medical approaches from the research laboratory to patients– that is, translating research “from bench to bedside”.   The MMRF has long supported this type of research, and the MMRF Scientific Agenda charts strategies aimed at translating an understanding of the genomics and proteomics of myeloma to targets and then to treatments and personalized medicine.  This may sound easy on paper - but actually making it happen in an efficient way is the real challenge. 

What was clear from this meeting is that disease-focused foundations like the MMRF are viewed as critical players in this complex, expensive and time-consuming process.  I was privileged to be invited as a panelist discussing the role of non-profit foundations in translational research along with other venture philanthropy leaders, like Bob Beall - MMRF Board Member and head of the Cystic Fibrosis Foundation (http://www.translationalmedicinealliance.org/forum/agenda.cfm).

While the specific approaches of the different groups vary, the common thread is that leading non-profit foundations like the MMRF fund commercial as well as academic research to overcome the barriers related to bringing new treatments forward.  The MMRF and its sister organization the MMRC have already made tremendous advances, such as opening clinical trials 30%-40% more quickly than the industry standard.  But, despite these improvements, there are still significant needs.  For example, there is a growing urgency to devise and fund new solutions that incentivize clinical development of drugs in MM since financial constraints facing the entire pharma and biotech industry threaten continued innovation and progress. 

Commercial investors who provide funding for the development of innovative products were also at the meeting.  These venture capitalists uniformly acknowledged that programs like the MMRF Biotech Investment Award that support promising biotech drugs are not just a source of cash.  Importantly, they also represent a “seal of approval” that can trigger the influx of venture capital to those companies.  Why is this important?  Simply put, it means that MMRF donor dollars are complemented with additional money that might not otherwise come to the field of myeloma thereby extending our reach and enhancing the chance of success for those drugs.

All in all, these were an exciting two days at the Translational Medicine Alliance Forum and it was rewarding to see so many scientists and business people working diligently to develop increasingly optimized models to translate research findings to real-world therapies for all patients, including those with multiple myeloma.

April 19, 2009

Greetings from the American Association for Cancer Research (AACR): Chief Scientific Officer, Louise Perkins

Greetings from the American Association for Cancer Research (AACR) annual meeting in Denver, Colorado!   This annual gathering is the most important cancer research meeting in the US, bringing together tens of thousands of research scientists and clinical researchers from around the world, with expertise in many different types of cancer, including multiple myeloma.

In addition to the scientific updates that will be presented over the next few days, one critical aspect from this convocation is the chance to meet with colleagues to explore innovative ideas and new approaches.  Several of us from the MMRF and MMRC capitalized on this opportunity to solidify existing and build new partnerships with biopharma companies to accelerate more treatments into myeloma clinical trials in the fastest possible timeframe.

Why is it so important to meet and work with these industry colleagues? Because they have the new drugs that could represent the next generation of myeloma therapies!  So, to ensure the most promising are tested in multiple myeloma, several us from MMRF (Joan Levy, Dan Cortright, Daniel Auclair and I) met with 8 companies over the span of 15 hours on Saturday. What did we learn and what was achieved? The MMRF and MMRC are clearly recognized as leaders among cancer non-profit foundations.  This reputation set the stage for the meaningful discussions we had.  The resulting innovative ideas may leverage the core strengths of the MMRF/MMRC but also depend on new models to prioritize, fund, and implement trials in this evolving field.

It’s hard to describe the level of enthusiasm apparent from many of the company representatives we met. For the most part, they clearly understand why the MMRF/C is such a special organization and how we are so well suited to advance new treatments into clinical trials.  They know that we are working for our patients and that it is in everyone’s best interest to cooperate because speedy trials mean drugs get to patients, a common goal for all of us.

April 19, 2009

MMRF Founder, CEO Honored with AACR Centennial Medal, Kathy Giusti, CEO

What an incredible morning! It was an unbelievable honor to be recognized with the AACR Centennial Award for our efforts to advance the delivery of new treatments and, eventually, a cure for multiple myeloma. I was so proud to accept the award from AACR President Dr. Raymond DuBois and from AACR CEO Dr. Margaret Foti, who I deeply admire and consider a dear friend, and on behalf of the AACR, for all of their amazing work. I am truly humbled to follow in the footsteps of last year’s recipient Lance Armstrong, one of cancer’s greatest heroes, and to stand shoulder to shoulder with fellow honorees, the founders of Stand Up to Cancer and Nick Valvano of the V Foundation.

When I started the MMRF and, later, the Multiple Myeloma Research Consortium (MMRC), I knew we had a lot of work ahead of us and many challenges that would slow our progress. But from the start, I had amazing support. This award is a truly testament to the willingness of everyone to work together to break down barriers slowing multiple myeloma research and to do whatever it takes to bring patients the treatments they urgently need.

I am so thankful for the MMRF team’s phenomenal dedication and our extraordinary donors, who inspire us with their generosity and who are behind us every step of the way. I would like to thank Drs. Ken Anderson, Bill Dalton, Rafael Fonseca, and Keith Stewart, who were instrumental in helping to create the MMRC, and the many other talented researchers who share our commitment to innovation, speed, and results. Thanks, too, to Celgene and Millennium for their extraordinary commitment to develop new and better treatments. Finally, I am grateful to the many wonderful clinicians and nurses, whose compassionate care has made a world of difference for myeloma patients, and especially to the patients themselves, who advance research by participating in clinical trials and donating tissue to our Tissue Bank, and who inspire us to work harder, smarter, and faster.

Together, we have come so far.  The AACR Centennial Award not only validates our work but help continue to bring awareness to the disease so that we can continue our work to bring patients the next generation of treatments and combination therapies.

April 13, 2009

Golf Pro Kenny Perry Raises Multiple Myeloma Awareness with MMRF Bracelet

Congratulations to Kenny Perry for an outstanding game at the Masters Golf Tournament and many thanks for his efforts to raise multiple myeloma awareness. Kenny wore the MMRF’s signature teal bracelet throughout the tournament to raise multiple myeloma awareness in honor of his mother, Mildred, who is living with the disease.

On behalf of families like Kenny’s, we will continue our urgent work to bring new treatments to patients and to stand as the premier educational and informational resource for patients and caregivers living with the disease.

Click here to see pictures of Kenny during the Masters Golf Tournament.

Kenny Perry Photo #1

Kenny Perry Photo #2

March 1, 2009

Live Report from IMW - Day 3: Chief Medical Officer, Susan Kelley

The proceedings on Day 3 of this 4 day meeting were just as energetic as the prior two days. The general scientific sessions and abstract presentations started at 6:30 in the morning and ran through 6:30 in the evening. It is quite gratifying to see over 1,200 scientific researchers and clinicians discussing data, sharing research results and challenging each other to ensure that the most important questions to be addressed in multiple myeloma receive attention. Among the important scientific sessions today, was a presentation by the Consensus Panels of the International Myeloma Working Group. These three Consensus groups reached out to a total of over 150 experts in multiple myeloma worldwide, through a series of meetings and teleconferences. Three areas of myeloma diagnosis and treatment were addressed and three reports have been generated. The reports are available on the IMW website and will be published in a major medical journal later this year.

Guidelines for the Uniform Reporting of Clinical Trials in Myeloma: this report presents a standard approach for clinical trials to evaluate new treatments in myeloma, including standard definitions of patient populations, types of clinical data to be collected on a trial, and how to present the results of a trial, so that the findings across important trials may be interpreted consistently. View guidelines http://www.mw-delhi09.com/spargoDocs/Consensuspanelone.pdf

· Guidelines for Risk Stratification in Myeloma: The committee members stressed that, currently, to mandate definitive treatment according to cytogenetic abnormalities is premature. Additional randomized prospective studies are needed to validate the relationship of particular risk factors to treatment outcome or to assign patients with specific cytogenetic (chromosomal) abnormalities to specific treatment regimens. Instead, these guidelines provide a suggested set of risk factors that can be evaluated to help assess prognosis. View guidelines http://www.mw-delhi09.com/spargoDocs/Consensuspaneltwo.pdf

· Guidelines for Standard Investigative Work Up in Myeloma (Diagnosis and Staging): The standard procedures (laboratory and clinical tests) that should be conducted at the time of initial diagnosis and at subsequent relapse or disease progression have been agreed and outlined. View guidelines http://www.mw-delhi09.com/spargoDocs/Consensuspanelthree.pdf

In another session there was a presentation of viewpoints from myeloma experts in the US, Italy and France regarding the approaches to timing and type of bone marrow transplant or stem cell transplant in myeloma. Dr. M. Cavo from Italy reminded the audience of the favorable 20-30% Complete Response rates being reported with use of the newer therapies (lenalinomide and/ or bortezimib combination therapy) in the treatment of newly diagnosed myeloma. There are important questions to be answered in upcoming studies, regarding the timing of ASCT (stem cell transplant) in patients who may derive good, durable response after 4-5 cycles of treatment with the newer treatment regimens.

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