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April 26, 2010

Exciting results from the MMRF-funded Genomics Initiative at AACR, Louise Perkins, PhD

Some of you may know that the MMRF-funded, MMRC Multiple Myeloma Genomics Initiative (MMGI) was designed to deeply explore the multiple myeloma tumor genome in more than 250 patient samples. What does that mean for folks who don't think about genomes every day? Briefly, the study was to understand the unique genetic alterations in patient tumor cells compared to their normal cells; those genetic changes are 'smoking guns' as to what has gone wrong and therefore what could be fixed to either destroy those myeloma cells or restore a more normal state to them. That's relatively easy to say (okay, for me, it is relatively easy to say) but this is difficult, time-consuming work requiring incredible technical expertise as well as patient tissue. We were blessed to be able to bring together the world-class researchers from the TGen and Broad Institutes together in a collaboration with tissue from our MMRC centers processed under standardized conditions at the MMRC Tissue Bank to undertake this 4-year, $10M project.

In terms of the time it takes to begin to see results, consider this analogy. If you have ever planted a garden, you know that careful planning and patience is needed. Many seeds are sewn and not all sprout. But when you see the first green sprouts and then the first blooms, you know that more is yet to come. The results presented at this year's American Association of Cancer Research (AACR) annual meeting are like some of those first flowers. The findings are extremely encouraging and more will come leading to new drugs and new treatments in the future. The AACR meeting, for those of you who may not know it, is one of the two largest cancer research meetings in the world bringing together scientists studying all types of cancers with attendance of 17,000 researchers.

The presentations fell into two main areas: Dr. Todd Golub of the Broad Institute disclosed results of our genome sequencing study on 38 patients’ tumor and matched normal samples and the second study was presented by Dr. Angela Baker of the TGen Institute who described genomics findings from 16 African American (AA) patient samples compared to patient samples from those of European descent.

Why are the results so exciting? First of all, the data revealed several new targets for which drugs are already at various stages of discovery and development including one target (BRAF) not previously known to be associated with MM. BRAF drugs have been created for a number of other cancers where its role in disease is clear. As compared to our sequencing study, nothing else at the AACR meeting described results on tumor genome sequences in as high a level of detail or on as many samples. In a complementary way, the findings from the AA patient study hint that other drugs in development might be particularly beneficial in AA patients. Those of us from the MMRF and MMRC are actively engaged with companies working on relevant drugs already in company pipelines and also ensuring that all of the findings are followed to lead to the next generation of drugs.

There really is a bright future here and with continued generous support we will be able to build on this momentum to benefit all patients with multiple myeloma!


April 21, 2010

AACR 2010: Cancer genomes keep coming

We are thrilled to see “live” coverage of data from Todd Golub’s presentation from our Genomics Initiative..."

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April 20, 2010

AACR 2010: Cancer genomes keep coming

Cancer genomes have been a hot topic at this year’s AACR. I stopped in to see a session hosted by Elaine Mardis, Washington University's genome maven whose been an author on most of the big cancer genome papers to date. In the session, we heard from Todd Golub of the Broad Institute, who gave preliminary results on the multiple myeloma genome, which hasn’t yet been published.

It looked like it has produced several interesting new potential cancer genes to look into. Though I won’t go into too much detail, here are some of the basic stats. They looked at cancers from 38 individuals sequencing both cancer cells and normal cells. They fully sequenced 23 of the individuals, and they did what’s called whole exome sequencing for 16 (in which they just sequence protein coding regions). One patient was sequenced by both methods.

The data produced a big list of mutations, but researchers have learned tricks for paring down such lists to find the so-called ‘drivers’ of cancer. By, for example, looking for mutations that appear frequently in cancer cells from different individuals. They came up with a short list of a dozen leading candidates. Four have been well characterized. Among the others, Golub found genes involved in regulating translation, the process by which RNA is made into protein, and even a gene implicated in susceptibility to Parkinson disease.

Posted by Brendan Maher on April 20, 2010.


April 11, 2010

Addressing Barriers to Drug Development MMRC-Style, Susan Kelley, MD

Last week, I was an invited speaker at two related meetings in Washington, DC. Both were attended by colleagues in academia, NIH, the pharmaceutical industry and advocacy so our presence was important and, from all accounts, impactful.

First, the ACRT/SCTS Joint Annual Meeting was the inaugural joint annual meeting of these two societies, and more than 2,500 attended. ACRT is the Association for Clinical Research Training SCTS is the Society for Clinical and Translational Science. Presentations focused primarily on research methodology and efficiencies, standardized approaches, and metrics; many people asked me about the metrics we use to track the speed and efficiency and member engagement in the MMRC. My talk was at the final plenary session. The session title was “ Innovative Models of Partnering For Innovative Medicines for Patients”, chaired by Queta Bond, retired as President of the Borroughs Wellcome Fund. My talk described the MMRC model and our collaborative efforts, as well as some of our metrics, directly related to our abstract published earlier this year in Clinical and Translational Science, Volume 3, April 2010, abstract O-040.

The second meeting was entitle Clinical Research Foundation: Transitions and opportunities in clinical Research. The Clinical Research Forum is comprised of 60 leading academic medical centers. The forum membership meets once a year with industry, private foundations and government to discuss ways to advance the mission of health care and research in the US. This is a small and powerful group of approximately 35 academics, 10 advocacy representative and 5 from NIH and the pharmaceutical industry. My talk was during the session (again chaired by Queta Bond) called "the Critical Role of Building Partnerships Bridging Academia and Patient Group's". The gaps identified by the panelists, representing the different groups at the meeting, were:

  • Approach to indirect costs
  • How to demonstrate ROI
  • How to build academic clinical trial infrastructure that actually delivers and when they should leave it to a CRO and industry
  • How to partner with foundations that are more “radical” in our demand to control data and standards of care, including the expectation that data will be shared and centers of excellence may be recognized and promoted (CFF and MMRF were the examples of radical by the way)
  • How to leverage the ties to industry

Once again, the MMRF and MMRC were recognized as having a highly progressive non-profit model for accelerating drug development, and it was rewarding to see all of the constituencies come together, recognizing that the current system can be improved and that we will only achieve progress with meaningful communication and collaboration.


January 26, 2010

Perspectives on Recent Clinical Trials Development, Susan Kelley, MD

Recently, two new drugs have entered into phase III clinical trials, representing the final stages of clinical development intended to create a data dossier for submission to health regulators, such as the US FDA as part of the approval process for new drugs. These new trials will include patients with relapsed (recurrent) MM from the US and around the world. One trial, sponsored by Keryx and AEterna Zentaris, evaluates the combination of Velcade (bortezomib) and KRX-0401 (perifosine), a novel inhibitor of key signaling pathways involved in tumor cell growth. The second new phase III trial evaluates the combination of Velcade with panobinostat, an inhibitor of histone deacetylase, which is thought to influence gene messaging in tumor growth processes (Novartis). Myeloma patients should be encouraged by the increasing numbers of myeloma clinical studies and the transition of new agents into late stage drug trials. The challenge now is to ensure that the clinical trials that are being conducted can be enrolled rapidly. MMRF and MMRC will continue to work closely with the sponsors and clinical investigators on these trials to improve patient enrollment to these and other ongoing trials.

There have also been several announcements of new study results confirming the importance of “maintenance” therapy administered after initial chemotherapy and stem cell transplant. This maintenance therapy frequently consists of 1-2 drugs, including an IMID, such as Revlimid (lenalidomide), administered for up to 2-3 years after initial response is documented. Preliminary data were presented by Professor Palumbo from the Celgene MM 015 trial at ASH. Then, during later December and early January, press releases were issued by Celgene with the IFM (France) and by the US NCI (cooperative group CALGB) about two additional studies (one in Europe and one in US), where patients receiving maintenance with Revlimid had a longer time until myeloma recurrence than patients who were not receiving long-term maintenance treatment.

Furthermore, several new clinical trials are evaluating the possible role of treatment with new or existing drugs for patients with Smoldering (Stage I) MM, and preliminary positive data were presented at ASH in December, from a trial evaluating Revlimid and dexamethasone, with very positive outcome, but it was an analysis on only 40 patients so it is premature to conclude that smoldering patients should receive treatment.

Finally, data from phase I-II (early clinical development) trials facilitated by the MMRC were also presented at ASH. The new drugs/studies presented included updates on the ongoing trials with carfilzomib, elotuzumab, Torisel, NPI-0052, pomalidomide and combined treatment using Revlimid, Velcade, Doxil and dexamethasone. There was an important session on advances in genomics studies and preliminary findings from the ongoing MMRF Genomics Initiative were presented.

So, what should patients take away from these recent data? The data from several of these important “maintenance” protocols will be released for greater scrutiny by the medical community during 2010. It is important that all patients currently receiving treatment for newly diagnosed MM speak with their hematologists/oncologists about the role of maintenance therapy and whether they should be considered for maintenance treatment starting some time after completion of stem cell transplant (if transplanted) or after attaining the maximal benefit from induction chemotherapy. As always, please ask your physicians about clinical trials which might suit your stage of disease, whether you are a newly diagnosed patient or a patient whose MM has recurred after initial therapy. There are important new drugs emerging and increased trial participation rates will mean that the important results from these trials can become available sooner.


December 9, 2009

Research Update from ASH - December 2009, Louise Perkins, PhD

Over the course of the last two days of the ASH meeting in New Orleans, there were quite a few scientific sessions and considerable excitement. In addition, a face-to-face meeting of the project team and Scientific Advisors to the Multiple Myeloma Genomics Initiative (MMGI) was held to discuss the current progress, challenges and plans for the project as it continues to develop a variety of data on the myeloma tumor genome. At the Presidential Symposium which wrapped up the meeting involved a presentation by one of the key contributors to the MMGI, Dr. Todd Golub of the Broad Institute as well as Dr. Louis Staudt of NCI who is both an advisor to the MMGI and a member of the MMRF Technology Board. Dr Staudt was also honored today by ASH as the recipient of the Dameshek prize for his contributions to the understanding of lymphoma. During this symposium, it was gratifying to hear Dr. Golub summarize some of the current achievements of the MMGI which now includes the successful sequencing of 25 myeloma patients’ tumor genomes. This project and the MMRF itself were featured on his first slide and he graciously recognized our organization for supporting this project. Dr. Timothy Ley from Washington University in St Louis also discussed leukemia genomics. He pointed out that sequencing of patient genomes is going to change medical care for patients and drive a future for personalized medicine – I wholeheartedly agree! New approaches to more sensitively detecting a patient's myeloma burden were also presented and these suggest that in the future it will be possible to use such readouts to determine whether a modified therapeutic approach will confer benefit by routinely aiming for so-called molecular responses. This is just one example of how we learn from other diseases and again exemplifies how personalized approaches are coming faster and faster to patients.


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