Update on Mesothelioma: A CancerCare Teleconference

CancerCare Update on MesotheliomaOn Monday, June 30 from 4:30-5:30PM Eastern Time, CancerCare is hosting a free mesothelioma teleconference workshop. The teleconference, titled Update on Mesothelioma, will feature a panel of experts to discuss numerous topics related to the disease. Registrants will be able to listen in on the phone or through a live stream online.

The Update on Mesothelioma teleconference will cover topics including mesothelioma treatment choices; the role of clinical trials; managing side effects, discomfort and pain; communicating with your health care team; quality of life and life style concerns; and physical activity and nutrition issues and tips. Questions for the panel of experts will also be accepted.

The panel of experts consists of four individuals: Richard J. Gralla, MD, FACP, Professor of Medicine, Albert Einstein College of Medicine; Lee M. Krug, MD, Director of the Mesothelioma Program, Associate Attending Physician, Division of Thoracic Oncology, Department of Medicine, Memorial Sloan-Kettering Cancer Center; Mary Hesdorffer, MS, APRN-BC, Nurse Practitioner, Executive Director, Mesothelioma Applied Research Foundation; and Winfield Boerckel, MSW, MBA, Director of Social Service – Long Island, Lung Cancer Program Coordinator, CancerCare.

Mary Hesdorffer, MS, APRN-BC, is an expert nurse practitioner with over 16 years of experience in mesothelioma treatment and dozens of published articles in a variety of scientific journals. She is the executive director of the Meso Foundation and the first line of help for patients and caregivers faced with a mesothelioma diagnosis.

Lee M. Krug, MD, is an Associate Attending Physician in the Division of Thoracic Oncology, Department of Medicine at Memorial Sloan-Kettering Cancer Center in New York, New York, where he completed a fellowship and chief fellowship in medical oncology. Dr. Krug is the Director of the Mesothelioma Program at Memorial Sloan-Kettering Cancer Center. He is also the chair of the board of directors of the Meso Foundation.

To register for the free Update on Mesothelioma teleconference workshop, visit CancerCare.org.

Meet the Experts: Dr. Valerie Rusch, Memorial Sloan-Kettering Cancer Center

Meet the Experts: Dr. Valerie Rusch

The Meso Foundation would like to invite you to join us for the next installment of our Meet the Experts series: “Focused Discussion on a New Clinical Trial: GL-ONC1” on Monday, February 17 at 8 PM Eastern Time, featuring Dr. Valerie Rusch, Memorial Sloan-Kettering Cancer Center.

The teleconference will follow our usual format with Mary Hesdorffer, Nurse Practitioner, as the moderator. We encourage all listeners to submit questions through our Facebook page and email throughout the call.

The teleconference is free, but does require that you RSVP in order to receive the call-in information. You can RSVP here.

Dr. Valerie Rusch of the Memorial Sloan-Kettering Cancer Center is a thoracic surgeon with over 25 years of experience. Her focus is treatment of patients with cancers of the lung, airways (trachea, bronchi), esophagus, mediastinum, chest wall, and pleura (malignant pleural mesothelioma). Read more about Dr. Rusch here.

You can listen to previous Meet the Experts sessions, including our most recent session, “New Frontiers in Surgery” with Dr. Joseph Friedberg, on our Meet the Experts page.

What does the Government Shutdown mean for Mesothelioma?

Each week that the government is shutdown, the National Institutes of Health (NIH) will be turning away 200 patients. According to a piece in the Wall Street Journal, NIH director Francis Collins said, “About 200 patients who otherwise would be admitted to the NIH Clinical Center into clinical trials each week will be turned away.”1 An estimated 1000 patients have already been turned away in the past year due to the sequester.2 The Mesothelioma Applied Research Foundation estimates that the NIH sees 125 mesothelioma patients per year, which means that 2.4 mesothelioma patients are being turned away from clinical trials each week that the government is shutdown.

Lisa Gonneville

Lisa Gonneville speaks at the Meso Foundation’s Congressional Briefing on mesothelioma

With only one FDA approved treatment for mesothelioma, patients often turn to clinical trials conducted at NIH after they have exhausted all of their other options. Mesothelioma warrior Lisa Gonneville, who is currently participating in a clinical trial at the NIH, shared her experience with mesothelioma to Capitol Hill staffers on Mesothelioma Awareness Day. “I’ve endured all of the treatment options available for mesothelioma, which are very limited,” said Mrs. Gonneville, “my only hope at this point is clinical trials.”

This shutdown hurts researchers as well. The NIH has already suspended intramural (in-house) research projects, and will stop accepting new patients and enrolling patients in any of the clinical trials it is conducting. If Congress remains at a stalemate, NIH-funded research at universities will continue although researchers could face funding delays. Also, some government-run databases may have problems, as support staff is furloughed. Approval of new NIH extramural grants to researchers in a university setting may be delayed as well.

The NCI Office of Advocacy Relations sent an email out yesterday confirming that no new research would begin, stating “In terms of intramural research, doctors will continue to see patients at the NIH Clinical Center; however, no new research may begin.”

Some other NIH Activities that will cease under a shutdown:

  • Initiation of new protocols at the NIH Clinical Center
  • Basic research conducted by NIH scientists
  • Translational research conducted by NIH scientists that develops clinical applications of scientific knowledge
  • Training of graduate students and postdoctoral fellows at NIH facilities
  • Scientific meetings at NIH facilities
  • Travel of NIH scientists to scientific meetings
  • NIH scientific equipment services
  • Almost all NIH administrative functions
  • NIH mail, cafeterias, and most visitor services

The Mesothelioma Applied Research Foundation will continue to follow the government shutdown and update the community.

View the Washington Post blog here.


Footnotes

[1] The shutdown could prevent kids with cancer from getting treatment
[2] According to the American Cancer Society

BREAKING NEWS: New Clinical Trial Opening

New press release daily newspaper headlinePRESS RELEASE

Contact Verastem, Inc.
Brian Sullivan, 617-252-9314
bsullivan@verastem.com

CAMBRIDGE, MA – Sep. 10, 2013 – Verastem, Inc. (NASDAQ: VSTM), focused on discovering and developing drugs to treat cancer by the targeted killing of cancer stem cells, today announced the initiation of COMMAND (Control Of Mesothelioma with MAiNtenance Defactinib). COMMAND is a registration-directed trial of lead cancer stem cell inhibitor defactinib (VS-6063) in patients with malignant pleural mesothelioma, a highly aggressive form of lung cancer with a high percentage of cancer stem cells.

COMMAND is a registration-directed, double-blind, placebo-controlled trial with Progression Free Survival (PFS) and Overall Survival (OS) as the primary endpoints. The study incorporates the opportunity to enrich for patients with tumors low in the biomarker, merlin. Preclinical and early clinical research has demonstrated that low merlin levels are predictive of increased effectiveness of FAK inhibitors such as defactinib. The COMMAND study will stratify patients to evaluate the effect of defactinib in both the overall patient population and the subgroup that are merlin low.

“FAK inhibition holds significant potential in malignant pleural mesothelioma, a cancer whose aggressiveness and treatment resistance proves all too often to be devastating for patients,” said Dr. Joanna Horobin, Verastem Chief Medical Officer. “Development of a drug that preferentially kills cancer stem cells is a promising approach, as many standard-of-care treatments have been shown to either have no effect on, or actually enrich the population of, these chemoresistant cells.”

COMMAND is expected to enroll approximately 350-400 patients at clinical sites in 11 countries, including the US, UK, Australia, Canada, South Africa, New Zealand and countries in mainland Europe. Eligible patients who had a partial response or stable disease following standard first-line therapy with platinum/pemetrexed will be stratified to merlin low or high and then randomized 1:1 to receive either placebo or 400 mg of defactinib twice daily.

“The incidence of mesothelioma, among the most aggressive and lethal cancers, is increasing worldwide, underscoring an urgent need to expand the very limited treatment options for patients fighting this disease,” said Professor Dean Fennell, Chair of Thoracic Medical Oncology at the University of Leicester, incoming President of the International Mesothelioma Interest Group (iMig) and Coordinating Investigator for COMMAND in the UK. “This well-designed study is expected to deliver a one-two punch to the tumor by treating with defactinib following first-line therapy that achieves initial disease control, but has not eliminated the cancer stem cells nor historically stopped disease progression, in an attempt to prolong clinical response. There is significant enthusiasm within the mesothelioma community for the COMMAND study.”

Verastem was recently granted orphan drug designation for defactinib in mesothelioma by the U.S. Food and Drug Administration’s Office of Orphan Drug Products.  The Company received a similar designation from the European Commission in June of this year. Adding to an intellectual property portfolio including composition of matter for defactinib, these designations provide eligibility for seven- and ten-year periods of market exclusivity, respectively, after product approval, as well as other development incentives.

“Timely initiation of this study marks yet another important milestone in our development of defactinib, and highlights the diligent execution of our clinical strategy by Verastem’s research and development team,” said Robert Forrester, Verastem President and Chief Executive Officer. “We will provide an update on the progress of this study in our year-end update in the first quarter of 2014.”

In addition to defactinib in mesothelioma, Verastem recently announced the completion of the Phase 1 stage and initial data from an ongoing Phase 1/1b study of defactinib in combination with weekly paclitaxel for patients with ovarian cancer. Verastem expects to initiate additional studies of defactinib including a Phase 2 trial in KRAS-mutated Non-Small Cell Lung Cancer and a Phase 1 study in Japan during the third quarter of 2013. The Company also announced the recent initiation of a Phase 1 study of FAK inhibitor VS-4718 in patients with advanced cancer, and that it expects to enter the dual mTORC1/2 and PI3K inhibitor VS-5584 into the clinic later this year.

About Malignant Pleural Mesothelioma

Malignant pleural mesothelioma is an aggressive form of cancer that occurs in the mesothelium, the thin layer of tissue that covers the lungs. Mesothelioma is associated with exposure to asbestos in most cases. According to the World Health Organization, a total of 59,000 deaths occur worldwide each year due to mesothelioma. Most mesotheliomas begin as one or more nodules that progressively grow to form a solid coating of tumor surrounding the lung leading to eventual suffocation and death. A high percentage of mesotheliomas contain cancer stem cells which are generally resistant to the currently available treatment options for advanced mesothelioma.

About Defactinib

Defactinib (VS-6063) is an oral compound designed to target cancer stem cells through the potent inhibition of focal adhesion kinase (FAK). Cancer stem cells are an underlying cause of tumor resistance to chemotherapy, recurrence and ultimate disease progression. Research by Robert Weinberg, Ph.D., scientific cofounder and chair of Verastem’s Scientific Advisory Board, and Verastem has demonstrated that the FAK pathway is critical for the growth and survival of cancer stem cells.  Defactinib is currently being studied in a registration-directed COMMAND trial in mesothelioma and a Phase 1/1b study in ovarian cancer. A Phase 2 trial in KRAS-mutated Non-Small Cell Lung Cancer and a Phase 1 study in Japan are expected to begin in the third quarter of 2013.  Defactinib has been granted orphan drug designation in the U.S. and E.U. for use in mesothelioma.

About Verastem, Inc.

Verastem, Inc. (NASDAQ: VSTM) is discovering and developing drugs to treat cancer by the targeted killing of cancer stem cells. Cancer stem cells are an underlying cause of tumor recurrence and metastasis. Verastem is developing small molecule inhibitors of signaling pathways that are critical to cancer stem cell survival and proliferation: FAK, PI3K/mTOR and Wnt. For more information, please visit www.verastem.com.

Forward-looking statements:

This press release includes forward-looking statements about the Company’s strategy, future plans and prospects, including statements regarding the development of the Company’s compounds, including VS-6063, VS-4718 and VS-5584, and the Company’s FAK, PI3K/mTOR and diagnostic programs generally, the timeline for clinical development and regulatory approval of the Company’s compounds, the expected timing for the reporting of data from ongoing trials, the structure of the Company’s planned or pending clinical trials and estimates of the Company’s ability to fund operations. The words “anticipate,” “appear,” “believe,” “estimate,” “expect,” “intend,” “may,” “plan,” “predict,” “project,” “target,” “potential,” “will,” “would,” “could,” “should,” “continue,” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Each forward-looking statement is subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statement. Applicable risks and uncertainties include the risks that the preclinical testing of the Company’s compounds and preliminary data from clinical trials may not be predictive of the results or success of later clinical trials, that data may not be available when we expect it to be that the Company will be unable to successfully complete the clinical development of its compounds, including VS-6063, VS-4718 and VS-5584, that the development of the Company’s compounds will take longer or cost more than planned, and that the Company’s compounds will not receive regulatory approval or become commercially successful products. Other risks and uncertainties include those identified under the heading “Risk Factors” in the Company’s Annual Report on Form 10-K for the year ended December 31, 2012 and in any subsequent SEC filings. The forward-looking statements contained in this presentation reflect the Company’s current views with respect to future events, and the Company does not undertake and specifically disclaims any obligation to update any forward-looking statements.

 

FROM THE FOUNDATION: Your Questions (and the Doctors’ Answers) from the 2012 Symposium — Part Two

The 2012 Symposium has brought together the best minds in mesothelioma research in order to highlight breakthroughs and options in asbestos cancer and mesothelioma treatment. This year, the Meso Foundation enabled a Live Stream in order to bring the Symposium online and allow you at home to interact.

Here is Part Two of the many questions the meso warriors and survivors sent in through the Live Stream.

Q: How can a patient boost his immune system by over-the-counter drugs?

A: There are many products advertised over the Internet and radio which are intended to boost the immune system. Without exception, they’ are all fraudulent. There is no evidence that any drug will generally boost your immune system.

What you need to do is maintain your health as best as possible by eating well and exercising regularly.

Q: Is there different treatment for someone who has sarcomatoid mesothelioma?

A: Unfortunately, the treatment for sarcoma mesothelioma is poor, and the survivals are poor as well. I know of no institution that has survival rates from diagnosis that exceed one year. Such patients should seek out major medical centers specializing in mesothelioma for experimental trials of new drugs.

Q: Are there any new advances in treatment of peritoneal mesothelioma?

A: There have been important advances in the surgical treatment with removal of known disease, combined with intraperitoneal local and regional chemotherapy given as an outpatient. There are a few centers that specialize in this. I would contact the Meso Foundation’s Nurse Practitioner Mary Hesdorffer at 703-879-3820 or mary@curemeso.org for more information.

Q: Is there any other cause of mesothelioma besides asbestos?

A: High doses of radiation are also known to cause mesothelioma. There may be other causes that have not yet been identified.

Q: To follow up on Sugarbaker comment where chemotherapy gives you one year of life and surgery three years of life.

A: Those comments reflect Dr. Sugarbaker’s feelings, emotions, and hopeful anticipation. You are correct that there is no such study which supports those assertions.

Q: Can MRI be as effective as CT scan for peritoneal mesothelioma follow-up?

A: It often can be more effective, especially in heavier patients with a higher body fat content. Different oncologists prefer different test modalities. CT scan itself is safe in this context and I would let your oncologist decide.

Q: Is there any thoughts among medical professionals that stress or other factors trigger mesothelioma?

A: While we generally think that mesothelioma, like other cancers, is caused by abnormalities in the genetic apparatus, stress and other factors in the patient already diagnosed certainly make matters worse.

Q: If younger patients of being diagnosed are there any thoughts That meso be caused by something other than asbestos.

A: This is not clear yet, but it certainly is a possibility. No cause other than asbestos or ionizing radiation has been identified yet, however.

Q: What is the shortest time from exposure to disease? And how many 911 first responders have been diagnosed with mesothelioma so far?

A: Older statistics suggested that less than 1% of mesothelioma’s had a latency time shorter than 15 years. More recently the estimate of latency time has shortened and may possibly be even less than five years.

I believe that there have been two cases of mesothelioma diagnosed in the 9/11 responders, but whether they were due to 9/11 exposure will never be clarified.

Q: What is the cause of mesotheliomas in young children? Is it a predisposition?

A: The cause in young children may be asbestos, and also may depend upon a genetic predisposition. None of this is really known at this point. There are not enough cases to form an opinion.

Q: Don’t you ever feel bad putting patients on placebo even in the trial?

A: Please understand that when doing a clinical trial the physician is not certain whether the drug will help you or hurt you. Otherwise he/she would not be doing the trial. If the drug is strong and there was a chance that the side effects would be dangerous, detrimental, shorten your life,  then he should have a control group that will get a placebo. If the physician is confident that the drug is likely to help you and there is little chance of it hurting you, then he should not administer a placebo; more to the point, he should not be doing the trial.

Q:  What is the most promising new treatment benefitting meso patients now?

A:  In my opinion, the most promising new treatment benefitting meso patients right now is the advent of lung-sparing surgery for patients with pleural mesothelioma.  There is evidence from multiple centers in the U.S. and around the world that for patients with epithelial pleural mesothelioma that multimodality therapy involving lung-sparing radical pleurectomy in combination with post-operative chemotherapy +/- radiation therapy can engender survival benefits which appear equivalent to those historically provided by extrapleural pneumonectomy (EPP).

Q:  What should you do if you think you have been exposed to asbestos?

A:  Absolutely you should inform your doctor of this information, and your doctor can then initiate, if appropriate, a series of tests to assess whether you have been adversely affected by the asbestos.  It is relatively straightforward to determine if your lung function has been detrimentally affected by the asbestos exposure, as well as to whether you currently have lung cancer or mesothelioma.  What is more complicated is the issue of future screening for development of lung cancer or mesothelioma on the basis of asbestos exposure alone.  As there are no standard screening recommendations, the best option would be to consider enrolling on a clinical trial for screening of those who have been exposed to asbestos.

Q:  What is the percentage of people receiving chemotherapy  after surgical procedure such as EPP or P/D (radical pleurectomy-RP).

A: In general, if patients have not previously received neo-adjuvant (pre-operative) chemotherapy, they should receive adjuvant (post-operative) chemotherapy, as long as they have recovered sufficiently after the surgery to be able to tolerate the treatment.  The recommendation is for 4 (or more) cycles of Pemetrexed and Cisplatin (or Carboplatin).  If patients already have received chemotherapy prior to surgery, there is no standard recommendation for giving additional chemotherapy after EPP/RP, but this is an important topic for future clinical studies.

Q:  Is photodynamic therapy (PDT) available to those who have already had surgery?

A: PDT is not currently available for patients who have had prior EPP or RP/PD, but we are very interested in using this treatment to stimulate immune responses in patients who have had recurrences after definitive surgery.  For patients who have had a prior “diagnostic” surgery for biopsy, pleurodesis, partial pleurectomy, it is still possible for them to undergo RP followed by intraoperative PDT.

Q:  What is being done to educate medical students and/or general practitioners in order for them to diagnose patients in a timely manner.

A:  The best methods are the dissemination of evidence-based knowledge through medical journals, textbooks, and scientifically-based internet based resources, as well as including mesothelioma related topics in medical school curricula and in residency training for general internists and family physicians.

Q: With so few patients, are there other ways than randomized double-blind clinical trials?

A: Clinical trials are rare because they’re very expensive, and pharmaceutical companies do not wish to spend a great deal of money on treatment of a rare cancer.

So that doctors are doing exactly what you say, they are reporting small series of cases individually. Please be assured that if it’s a terrific “standout” treatment appearing in  a small series, it will be quickly tested in the largest and most appropriate series.

UPDATE, July 17, 3:52 p.m. — We discovered these missing questions in our Inbox. We’re bringing them to you today!

Q: Why doesn’t everyone get mesothelioma who has been in contact with asbestos.

A: Many cancers, including mesothelioma are multifactorial, that is, they only appear when an appropriate combination of factors comes together. For example, not everyone who smokes a lot of cigarettes gets lung cancer.

Q: Question what happens when you get only one specialist and have to take what you get?

A: You are a consumer of healthcare. Do not have to “take what you get.” If as a mesothelioma patient, you have seen only one specialist, you should get in touch with MARF to help you chart your course among the different providers, specialists, and institutions, so as to get the best treatment.

 

UPDATE, July 25, 10:37 p.m. — One more question discovered in our Inbox from the 2-012 Symposium…

Q: Is there research into chronic pain?

A: There is a great deal of ongoing research into chronic pain syndromes such as you described. Even if an anatomical cause is not identified, much can be done. Usually opiates such as morphine or fentanyl are needed, supplemented with adjuvant drugs such as gabapentin or baclofen. also very new drugs such as  antibody to the mu receptor and drugs targeting the NMDA receptor will soon be available. Many pain specialists at major medical centers can help.