A Look at the Advancing Breakthrough Therapies for Patients Act

Mary at Friends of Cancer Researchby Mary Hesdorffer, NP, Executive Director, Mesothelioma Applied Research Foundation

The Advancing Breakthrough Therapies for Patients Act was first introduced in the Senate by Senators Bennet ( D-CO), Hatch (R-UT) and Burr (R-NC). Two months later, Congresswoman DeGette (D-CO) and Congressman Bilbray (R-CA) introduced the act in the House of Representatives. The bills, which received bipartisan support, were included as an amendment to the Food and Drug Administration’s Safety and Innovation Act (FDASIA), the latest iteration of the Prescription Drug User Fee bill, and in July of 2012, the breakthrough therapy designation was signed into law.

As a result of this law, a new drug may be designated as a breakthrough therapy if it is intended to treat a serious or life-threatening disease, and if preliminary clinical evidence suggests it provides a substantial improvement over existing therapies. Once a breakthrough therapy designation is granted, the FDA and drug sponsor work together to determine the most efficient path forward. In just two years, 178 requests for breakthrough designation have been submitted, 44 designations have been granted, and 6 drugs have been approved within the program.

On May 6 of this year, Mary Hesdorffer, the executive director of the Meso Foundation, and Melinda Kotzian, its chief executive officer, attended the congressional briefing titled Friends of Cancer Research, which focused on evaluating the progress of this program.

The panel agreed that the next task will be to streamline the process. This will eliminate applications that absolutely do not meet the criteria for breakthrough designation.

Janet Woodcock, MD, director, of the Center for Drug Evaluation and Research within Food and Drug Administration, stated that they “are not planning on moving drugs through this process that offer only incremental progress, but those that demonstrate a substantial benefit.”

Two representatives of the pharmaceutical industry shared their companies’ experience in gaining the first designation under this act. They noted that the time and commitment of all parties involved made this process work.

Dr. Woodcock cautioned that sequestration had an impact on the financing of this program. It will take more funding to continue operating the Breakthrough Therapies program at the present level, but she hopes that streamlining the process will allow for a more drugs to reach the patients in need.

First Congressional Cancer Hearing in Six Years a Success

Congressional Cancer Research Hearing

by Jessica Barker, Director of Government Affairs, Mesothelioma Applied Research Foundation

Wednesday afternoon, the Senate Special Committee on Aging held a hearing on cancer research featuring Dr. Harold Varmus, Director of the National Cancer Institute (NCI) and actress Valerie Harper, a survivor of Leptomeningeal carcinomatosis (LC), a rare complication of cancer in the brain. This was the first Congressional hearing with a dedicated focus on cancer in six years. The hearing, titled The Fight Against Cancer: Challenges, Progress, and Promise examined federal funding for cancer research and recent treatment improvements, and explored challenges facing survivors and scientists.

The hearing gave each of the five witnesses the opportunity to give testimony, which was followed by questions from the Members of the Committee. The witnesses included:

  • Harold E. Varmus, MD, Director, National Cancer Institute, National Institutes of Health
  • Valerie Harper, Actress and Cancer Survivor
  • Thomas Sellers, PhD, MPH, Director, H. Lee Moffitt Cancer Center and Research Institute
  • Mary Dempsey, Assistant Director and Co-Founder, The Patrick Dempsey Center for Cancer Hope and Healing
  • Chip Kennett, Advocate and Cancer Survivor

Each witness gave a powerful opening statement. Valerie Harper urged Members of Congress to think of cancer research dollars as an investment rather than spending. Ms. Harper also voiced a hopeful message for cancer patients, as she is positive and living life to the fullest despite her prognosis, stating “I am not going to the funeral until the day of the funeral!”

While not mentioned in his testimony, Dr. Varmus was asked by Senator Kelly Ayotte (R-NH) about the Department of Defense’s (DoD) Congressionally Directed Medical Research Program (CDMRP). The CDMRP has been a great source of funding for mesothelioma, funding $9.3 million to date. Asked if the DoD and the NCI collaborated, Dr. Varmus emphasized that all of the federal agencies that do medical research, as well as all researchers around the country share their research through scientific meetings (such as the Meso Foundation’s Symposium) and through journals. When specifically asked if he thought the program should be in the DoD or under the umbrella of the National Institutes of Health (which houses the NCI), he stated that he does not have a problem with the CDMRP being housed at the DoD and that he welcomes co-funding.

Senator Sheldon Whitehouse (D- RI) asked Dr. Varmus about the Recalcitrant Cancer Act of 2012 and its progress. Dr. Varmus told the committee that he had completed the scientific framework for pancreatic cancer, and that he would be completing the scientific framework for small cell lung cancer by the statutory deadline of July 2014. This is good news for the mesothelioma community.

To view the hearing, please visit the Senate Special Committee on Aging website.

Dr. Ira Pastan of National Cancer Institute to Speak at Mesothelioma Symposium

Dr. Ira PastanThe Mesothelioma Applied Research Foundation announced that Dr. Ira Pastan, the Head of the National Cancer Institute’s Molecular Biology Section, will be the keynote speaker during its 11th annual International Symposium on Malignant Mesothelioma, taking place on March 5-7 in the Washington, DC metro area.

Alexandria, VA (PRWEB) February 28, 2014

The Mesothelioma Applied Research Foundation announced that Dr. Ira Pastan, the Head of the National Cancer Institute’s Molecular Biology Section, will be the keynote speaker during its 11th annual International Symposium on Malignant Mesothelioma, taking place on March 5-7 in the Washington, DC metro area.

Dr. Pastan is a world-renowned scientist known for his work in cancer research. Along with colleague Mark Willingham, he discovered mesothelin, a protein that is over-expressed in mesothelioma, as well as certain other cancers. This discovery has made it possible to develop an immunotoxin, SS1P, which targets the mesothelin antigen. SS1P has shown much promise in clinical trials by producing major tumor regressions lasting up to two years.

“It is an honor to have Dr. Pastan, one of those most imminent cancer researchers in this nation, joining us at our International Symposium on Malignant Mesothelioma,” said Mary Hesdorffer, NP, the executive director of the Mesothelioma Applied Research Foundation.

“Hearing about his promising work directly from Dr. Pastan will be a very inspiring experience for the mesothelioma patients in attendance, as well as others interested in curing mesothelioma,” she added.

Dr. Pastan will speak to the Symposium audience on Thursday, March 6 at 9:15 AM. The program will also be broadcast live on the web at http://www.curemeso.org/symposium.

Mesothelioma is a malignant tumor of the lining of the lung, abdomen, or heart known to be caused by exposure to asbestos. Medical experts consider it one of the most aggressive and deadly of all cancers. Approximately 3,500 Americans are diagnosed with mesothelioma every year and an estimated one-third were exposed while serving in the Navy or working in shipyards.

ABOUT THE MESOTHELIOMA APPLIED RESEARCH FOUNDATION
The Meso Foundation is the only 501(c)3 non-profit organization dedicated to eradicating mesothelioma and easing the suffering caused by this cancer. The Meso Foundation actively seeks philanthropic support to fund peer-reviewed mesothelioma research; provide patient support services and education; and advocate Congress for increased federal funding for mesothelioma research. The Meso Foundation is the only non-government funder of peer reviewed scientific research to establish effective treatments for mesothelioma and, ultimately, a cure for this extremely aggressive cancer. To date, the Foundation has awarded over $8.7 million to research. More information is available at http://www.curemeso.org.

Rep. Betty McCollum and Rep. Chellie Pingree to Receive Bruce Vento Hope-Builder Award

Congressional Briefing on Malignant MesotheliomaThe Mesothelioma Applied Research Foundation (Meso Foundation) announced that Congresswomen Betty McCollum and Chellie Pingree are the recipients of the Bruce Vento Hope-Builder Award, for sending a letter to Dr. Harold Varmus, Director of the National Cancer Institute, to urge the National Cancer Institute to further mesothelioma research.

Alexandria, VA (PRWEB) February 28, 2014

The Mesothelioma Applied Research Foundation (Meso Foundation) announced that Congresswomen Betty McCollum and Chellie Pingree are the recipients of the Bruce Vento Hope-Builder Award, for sending a letter to Dr. Harold Varmus, Director of the National Cancer Institute, to urge the National Cancer Institute to further mesothelioma research. They will be presented with the award during the International Symposium on Malignant Mesothelioma, on March 6.

Last month, U.S. Congresswomen Betty McCollum (D-MN) and Chellie Pingree (D-ME) sent a “Dear Colleague” letter to all members of the U.S. House of Representatives asking them to join them in urging the National Cancer Institute to create a scientific framework for mesothelioma. The Mesothelioma Applied Research Foundation (Meso Foundation) has long advocated for the National Cancer Institute to increase their focus on mesothelioma, and congratulates Representative McCollum and Representative Pingree for their efforts on behalf of the mesothelioma community, including patients and families, physicians, advocates, and researchers dedicated to eradicating the life-ending and vicious effects of mesothelioma.

The idea for this letter began when Representative Pingree’s constituent, mesothelioma survivor Lisa Gonneville spoke at the Capitol Hill briefing onMesothelioma Awareness Day (September 26).

Mrs. Gonneville, a mother of four and Dayton, ME resident, pleaded that the staffers do something to address this deadly cancer. “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.” Congresswoman Pingree was so moved by Ms. Gonneville’s story that she, too, wanted to help the mesothelioma community in her honor.

“Congresswomen McCollum and Pingree are leading a very important effort for everyone affected by mesothelioma by encouraging the National Cancer Institute to create a scientific framework to progress mesothelioma research,” said Meso Foundation’s executive director and nurse practitioner, Mary Hesdorffer.

“This is an incredible way to help thousands of patients affected by mesothelioma, and also honor our distinguished former board member and Congresswoman McCollum’s predecessor, Bruce Vento.”

Bruce Vento was a prominent member of Congress who, after nearly 24 years of service, succumbed to mesothelioma only eight months after diagnosis.

The Recalcitrant Cancer Research Act of 2012 was signed into law by President Obama on January 2, 2013 as part of the National Defense Authorization Act (Public Law No. 112-239), giving the Director of the National Cancer Institute (NCI) the ability to identify recalcitrant cancers for which to establish scientific frameworks that will guide research efforts.

For each recalcitrant cancer, NCI is to convene a working group of both Federal and non-Federal individuals to provide expertise and assistance in developing the scientific framework. The frameworks are to be completed within 18 months of enactment, then submitted to Congress and made publicly available on the HHS website within 30 days. The bill requires the progress of each scientific framework be reported in the National Institutes of Health (NIH) Biennial Report, with an assessment of progress made in improving outcomes for recalcitrant cancers. The bill further states that the NCI Director “shall consider” each relevant scientific framework when making recommendations for exception funding for grant applications.

Mesothelioma is a malignant tumor of the lining of the lung, abdomen, or heart known to be caused by exposure to asbestos. Medical experts consider it one of the most aggressive and deadly of all cancers. Approximately 3,500 Americans are diagnosed with mesothelioma every year and an estimated one-third were exposed while serving in the Navy or working in shipyards.

ABOUT THE MESOTHELIOMA APPLIED RESEARCH FOUNDATION
The Meso Foundation is the only 501(c)3 non-profit organization dedicated to eradicating mesothelioma and easing the suffering caused by this cancer. The Meso Foundation actively seeks philanthropic support to fund peer-reviewed mesothelioma research; provide patient support services and education; and advocate Congress for increased federal funding for mesothelioma research. The Meso Foundation is the only non-government funder of peer reviewed scientific research to establish effective treatments for mesothelioma and, ultimately, a cure for this extremely aggressive cancer. To date, the Foundation has awarded over $8.7 million to research. More information is available at http://www.curemeso.org.

Mesothelioma Researcher Receives Pioneer Award

Dr. Michele Carbone

Dr. Michele Carbone spoke at the Meso Foundation’s 2013 International Symposium on Malignant Mesothelioma

The Mesothelioma Applied Research Foundation announced today that the 2014 recipient of its Pioneer Award is Michele Carbone, MD, PhD, for his remarkable achievements in mesothelioma research.

Michele Carbone, MD, PhD is a Professor of Pathology and the Director of the University of Hawaii Cancer Center, a National Cancer Institute designated Consortium Cancer Center affiliated with the University of Hawaii – Manoa. He has published over 150 peer-reviewed papers, most in the area of the pathogenesis of mesothelioma.

In addition to his impressive publication record, Dr. Carbone is known in the mesothelioma community for his tireless work in the Turkish region of Cappadocia, where mesothelioma causes 50% of all deaths. Dr. Carbone discovered that a combination of environmental exposures and genetic predisposition were the reason for such a high incidence of mesothelioma among the villagers. Taking his role as a scientist a step further, alongside famed Turkish professor Izzettin Baris, Dr. Carbone demonstrated his compassion by convincing the Turkish government to move and rebuild the affected villages, thus removing them from the environmental exposures.

Dr. Carbone’s studies in Turkey led him to a life-long quest to identify genes implicated in mesothelioma. In 2001, he published in “The Lancet” that predisposition to developing mesothelioma was transmitted as an in an autosomal dominant character in certain Turkish families.

Subsequently, back in the United States, Dr. Carbone and Joseph Testa, PhD led their research team, which included Drs. Giovanni Gaudino, Harvey Pass, Jianming Pei, Mitchell Cheung, and Haining Yang, to study US families with a high incidence of mesothelioma, and through this collaboration discovered the “BAP1 cancer syndrome.” They found this “syndrome” to be caused by germline mutations of the BAP1 gene, which is characterized clinically by a very high risk of developing mesothelioma, melanoma, and other cancers.

Dr. Carbone is known in his field as a generous mentor, and has helped several prolific scientists enter the field of mesothelioma research. Dr. Haining Yang, is one researcher whose work with Dr. Carbone resulted in a peer-reviewed grant award of $100,000 from the Mesothelioma Applied Research Foundation which helped her to develop the data that allowed her two years later to win her NCI-RO1 and DOD grants to study mesothelioma.

“I have worked closely with Dr. Carbone over the years and have been impressed by his kindness and availability,” said Mary Hesdorffer, NP, Meso Foundation’s executive director. “He has been a leader in the field of mesothelioma research, and every one of his many contributions brings us that many steps closer to life-saving treatments for mesothelioma patients.”

The Pioneer Award honors individuals “pioneering” scientific advances in the field of mesothelioma, with the goal of eradicating the life-ending and vicious effects of mesothelioma. The award will be presented during the Awards Dinner, at the International Symposium on Malignant Mesothelioma in Alexandria, VA, on March 5-7.

Mesothelioma is a malignant tumor of the lining of the lung, abdomen, or heart known to be caused by exposure to asbestos. Medical experts consider it one of the most aggressive and deadly of all cancers. Approximately 3,500 Americans are diagnosed with mesothelioma every year and an estimated one-third were exposed while serving in the Navy or working in Navy shipyards.

ABOUT THE MESOTHELIOMA APPLIED RESEARCH FOUNDATION
The Meso Foundation is the only 501(c)3 non-profit organization dedicated to eradicating mesothelioma and easing the suffering caused by it. The Meso Foundation actively seeks philanthropic support to fund peer-reviewed mesothelioma research; provide patient support services and education; and advocate Congress for increased federal funding for mesothelioma research. The Meso Foundation is the only non-government funder of peer reviewed scientific research to establish more effective treatments for mesothelioma and, ultimately, a cure for this extremely aggressive cancer. To date, the Foundation has awarded over $8.7 million to research.

More information is available at http://www.curemeso.org.