Sunday, August 23, 2015

Precision Medicine: Reaching New Levels

I’ve been writing a lot about the Precision Medicine Initiative because I am excited about this approach to cancer treatment, and the advancements being made.  The National Foundation for Cancer Research (NFCR) has supported cancer research for all cancers since 1973, and is taking a leadership role in educating the public about how cancer treatment has less to do with the location of the tumor, and more to do with the genetic abnormalities.  Yet today, it’s still the case in most medical care systems that cancers are classified mainly by the type of tissue or part of the body in which they presented—breast, lung, brain, colon, etc. But thanks to advances in scientific knowledge and DNA sequencing technology, things are changing, and researchers are discovering that cancers that arise in totally different parts of the body can sometimes have a lot in common. This is leading to rethinking how we approach clinical trials.

For example, The NCI-Molecular Analysis for Therapy Choice (NCI-MATCH) is a phase II clinical trial that will analyze patients’ tumors to determine whether they contain genetic abnormalities for which a targeted drug exists (that is, “actionable mutations”) and assign treatment based on the abnormality. NCI-MATCH seeks to determine whether treating cancers according to their molecular abnormalities will show evidence of effectiveness. For instance, a breast cancer patient may have the anaplastic lymphoma kinase (ALK) gene mutation often found in lung cancer. The NCI-MATCH Trial will look at treating the ALK mutation rather than the type of cancer. NIH Director Dr. Francis Collins has also commented “so it may be that somebody who has a lung cancer has more in common with somebody with a bladder cancer than two people with lung cancer.” In all, more than 20 different study drugs or drug combinations targeting specific gene mutation will be used in the NCI-MATCH Trial as treatment for each particular gene mutation.

These findings provide yet another example of how cancer research has been leading the way in precision medicine. Still, much more remains to be done. As part of the new Precision Medicine Initiative, researchers will explore fundamental aspects of cancer biology, and seek to understand the mechanisms of drug resistance. NFCR will continue to support research that will accelerate the design and testing of more precisely targeted cancer treatments, including combination therapies.  I am grateful for the continued public support for the important research being done by NFCR-supported cancer researchers.  #Gratitude

Saturday, August 15, 2015

Boston and Cambridge Innovation

Cancer hit close to home for me again this week.  A friend of mine in Washington, DC was diagnosed with Stage 4 lung cancer – age 44 non-smoker.  My heart is broken for him and his beautiful family.  At the same time, it reinforces my commitment to raising money for cancer research, including research that would help my friend.

It also reminds me of how fortunate I am to live in Massachusetts, and to participate in the scientific community of innovation in Boston and Cambridge.  The National Foundation for Cancer Research (NFCR) has supported cancer research in laboratories in Boston for many years, at Massachusetts General Hospital, Dana Farber, Beth Israel and MIT.  Greater Boston truly is one of the most important centers for innovation in the world, and I spend a lot of time here, exploring potential collaborations with leaders in the biotech, venture capital, and academic medical research communities.

Boston and Cambridge are home to so many innovative scientific organizations, too many to name.  One interesting group is LabCentral, “a 28,000 square-foot facility in the heart of the Kendall Square, Cambridge, biotech innovation hub, a first-of-its-kind shared laboratory space designed as a launchpad for high-potential life-sciences and biotech startups.”  I am also particularly impressed with people like Bob Urban at the Johnson & Johnson Innovation Center, and Dr. Laurence Cooper (pictured below) at Ziopharm Oncology. NFCR has supported Dr. Cooper’s research at MD Anderson since 2004, which helped lead to the license of his immunotherapy technology to Ziopharm.  I am grateful for playing a part in helping bring new technologies like these closer to the patient bedside.
I am excited and encouraged by the work being done in Boston and Cambridge.  Though I work in Bethesda, MD, I’ve lived in the Boston area for almost 30 years.  You will see me around town, and hear a lot more this fall about an exciting cancer research project I am working on at Massachusetts General Hospital.  This research in Boston will benefit patients around the world, including my friend in Washington, DC.

Thursday, August 6, 2015

Another Targeted Advance in Treating NSCLC

The U.S. Food and Drug Administration (FDA) recently approved Iressa (gefitinib) for the first-line treatment of patients with metastatic non-small cell lung cancer (NSCLC) whose tumors harbor specific types of epidermal growth factor receptor (EGFR) gene mutations.    This new approval extends only to patients whose tumors express specific mutations, originally identified by National Foundation for Cancer Research (NFCR)-supported scientist Daniel Haber, M.D., Ph.D. at Massachusetts General Hospital (MGH), which make them especially responsive to the drug. With this approval, metastatic non-small cell lung cancer patients whose tumors have EGFR mutations now have three personalized treatment options. 

It was Dr. Haber’s landmark research in 2004 that first identified the specific EGFR mutations that predict which patients will have a positive response to Iressa. In making its ruling, the FDA cites a recent clinical trial, which directly credits Dr. Haber’s research as instrumental in its success. One of the landmark achievements of the era of precision medicine was the effort to solve the puzzle of the drug Iressa. Why did only 13% of patients respond well to the drug? How could we screen for this small group of patients? These critical questions needed to be answered to unlock the true power of this drug – matching it with the patients who would most benefit. 

This breakthrough discovery not only helps to tailor Iressa to the right cancer patients, it also has profound indication in guiding the usage of other drugs, such as Tarceva, which also works by blocking EGFR. It is an illustration of targeted therapy in the new age of precision medicine. Research being done by scientists such as Dr. Haber and his team has expanded the work on identifying genetic lesions across multiple cancer types. Another example of how we are all in this together.


Sunday, August 2, 2015

Diagnostic Advances in Metastatic Breast Cancer

I continue to be encouraged by advances in the use of circulating tumor cell (CTC) analysis in cancer treatment, but so much work needs to be done.  In a recent study led by Martine Mazel from University Medical Centre in Montpellier, France, researchers have demonstrated the ability to gauge PD-L1 expression from liquid biopsies of metastatic breast cancer patients.  Immune checkpoint regulators are becoming increasing important and have given rise to the development of immunotherapies for cancer treatment. PD-L1 is an immune checkpoint regulator targeted by a number of approved and developmental oncology therapies.  

Using the CELLSEARCH® System developed by Janssen Diagnostics, researchers tested blood samples from 16 women with hormone receptor-positive, HER2-negative metastatic breast cancer and found 11 patients had a subpopulation of CTCs with weak or strong PD-L1 expression. These results indicate that CTC analysis for PD-L1 expression is feasible and when confirmed, could open up the opportunity to predict response to certain therapies in future, larger studies using a blood test rather than tissue biopsy. In a research setting, the CELLSEARCH® System offers the ability to capture CTCs via a routine blood test. 

"I believe we have demonstrated that CTC characterization for PD-L1 expression is feasible using a CTC platform. This paves the way for the utilization of a CTC/PD-L1 assay in future clinical trials to explore whether it can stratify patients according to response, and potentially predict the efficacy of immune checkpoint blockade," commented study investigator Catherine Alix-Panabieres, Ph.D., Director of the Laboratory of Rare Human Circulating Cells, University Medical Centre of Montpellier, University of Montpellier EA2415, Montpellier, France.  

CTCs have the potential to allow real-time, dynamic monitoring of tumor characteristics without the need for repeated invasive biopsies. It was demonstrated earlier that CTC expression of key biomarkers such as HER21, IGFR2, c-MET3 and other proteins can be monitored successfully. This publication in Molecular Oncology underscores the potential of CELLSEARCH® CTC testing to also become an important tool in cancer immunotherapy. 

Yet according to a recent article in MIT Technology Review, “new diagnostics can find the DNA that drives a tumor, but evidence that they help patients is missing”. “You have tests coming to market that are sometimes proven and sometimes unproven,” says Tycho Peterson, an analyst at J.P. Morgan who tracks the industry. “Commercial activity is increasing very quickly.” He estimates $20 billion a year in tests globally by 2020, up from about $100 million today.  But we are a long way from the liquid biopsy being used to detect cancer before symptoms arise. All the more reason that the National Foundation for Cancer Research continues to support the work of Dr. Daniel Haber, Director of the Massachusetts General Hospital in Boston, and his collaborators.  We have continuously supported this important research since 2004, and I believe this ultimately will help patients across ALL CANCERS.