European Commission Grants Orphan Status to Mesothelioma Drug

Once again the European Union has taken the lead in medical care by granting orphan status to a drug touted as bringing a new treatment option to patients with mesothelioma. On June 13, Verastem, Inc. announced its investigational drug VS-6063 received orphan medicinal product designation from the European Commission for use in mesothelioma.

Last year, the European Commission was applauded for bringing a new era to medicine in the Western world by approving gene therapy for the treatment of certain rare disorders. This approval paved the way for breakthrough treatments for diseases with a very high unmet medical need, such as mesothelioma.

Mesothelioma is a rare, highly aggressive cancer that develops over decades after exposure to asbestos. Designated a rare, or orphan, disease in both the United States and Europe, mesothelioma is diagnosed in close to 3,000 Americans and nearly 2,400 Brits each year. Britain has more deaths from asbestos-related diseases than any other country. Treatment for the disease is limited and currently, there is no cure for the disease.

Verastem, Inc., based in Cambridge, MA, is a clinical-stage biopharmaceutical company focused on discovering and developing drugs to treat cancer by the targeted killing of cancer stem cells. VS-6063 is indicated for use in mesothelioma patients that are lacking the Merlin protein. Merlin is believed to play a role in controlling cell shape, cell movement, and communication between cells. Merlin also functions as a tumor suppressor protein, which prevents cells from growing and dividing too fast or in an uncontrolled way.

According to Verastem, approximately 40-50% of mesothelioma patients lack Merlin. Studies by Verastem and others have shown that Merlin-low mesothelioma cells and tumors appear to be particularly sensitive to focal adhesion kinase inhibition, or FAK, which VS-6063 addresses.

Verastem held a mesothelioma briefing session with Dr. Dean Fennell at the American Society of Clinical Oncology meeting held in early June. According to the company’s press release, the discussion focused on the role of cancer stem cells and the tumor suppressor Merlin in mesothelioma progression. Verastem noted that the current mesothelioma treatments fall short when it comes to killing the cancer cells because they do not get to the heart of the cancer cells, the cancer stem cells, which VS-6063 targets. Various studies have shown that cancer stem cells can survive the effects of anti-cancer drugs and continue to grow and divide causing the cancer to metastasize.

“Mesothelioma is a devastating disease with limited treatment options,” said Dr. Joanna Horobin, Verastem Chief Medical Officer. “We are moving quickly to bring new treatment options to patients with mesothelioma.”

Verastem is working in conjunction with LabCorp to develop a biomarker test to identify those mesothelioma patients low in Merlin. In addition to the upcoming mesothelioma study, VS-6063 is currently being evaluated in a Phase 1/1b trial in combination with paclitaxel in patients with ovarian cancer.

Sources

Verastem, Inc
http://phx.corporate-ir.net/phoenix.zhtml?c=250749&p=irol-newsArticle&ID=1829497&highlight=
American Society of Clinical
http://www.cancer.net/cancer-types/mesothelioma/statistics

Mesothelioma Attorney Joseph Belluck Honored by New Yorkers for Patient & Family Empowerment

Joseph W. Belluck was one of the honorees of New Yorkers for Patient & Family Empowerment at its spring reception entitled, “Making Law Work for Health Consumers.” The event held on June 5 was hosted by Mark Green, a nationally known consumer rights advocate/author, and former NYC Public Advocate and Consumer Affairs Commissioner.

Belluck, a founding partner of the Belluck & Fox law firm, LLP, a nationally recognized law firm with attorneys who represent individuals with asbestos and mesothelioma claims, was honored for his legal work against tobacco and asbestos companies and for advocating for patient rights on a State Medicaid Redesign Team work group. Governor Cuomo established the Medicaid Redesign Team to bring together stakeholders and experts from throughout the state to work cooperatively to reform the system and reduce costs.

According to its website, New Yorkers for Patient & Family Empowerment, Inc (also known as “Patient & Family”), a not-for-profit organization, was formed to focus greater attention on patient safety and to counteract the factors that make it harder for patients and their loved ones to achieve greater safety in healthcare. The organization seeks to:

Empower patients and their loved ones in interacting with the healthcare system;
Strengthen public access to information on patient safety; and
Improve the quality and safety of healthcare in New York.

The other honorees Wednesday night were:

Elisabeth Benjamin – Community Service Society’s Vice President of Health Programs and founder of the Legal Aid Society’s Health Law Unit
Janet Foley – Director of Health & Safety at CSEA Local 1000 AFSCME; advocate for safe lifting & moving in healthcare (“Safe Patient Handling”)

Belluck is AV-rated by Martindale-Hubbell and is listed in Best Lawyers in America, New York Magazine’s “Best Lawyers in the New York Area” and in Super Lawyers.

Mesothelioma Patients Face Risk of Bankruptcy

Early last month we reported that doctors across the country have joined forces in calling for pharmaceutical companies to lower the cost of some cancer drugs. Also cited in that article were statistics from a study that found 30% of cancer patients reported that their medical expenses were a “significant burden,” and 11% called them a “catastrophic problem.” Mesothelioma and cancer patients are being strapped with higher and higher medical expenses, and sadly, they are susceptible to going bankrupt from those costs.

Mesothelioma patients are faced with many challenging issues after they are diagnosed with the incurable, asbestos-related cancer. Determining where to receive their medical care, what treatments to use to fight the disease and how to cope with the symptoms are stressful enough, but many patients quickly realize that the financial aspect of the disease will significantly add to their stress.

In a recent study from the Fred Hutchinson Cancer Research Center in Seattle, researchers set out to understand the relationship between receiving a cancer diagnosis and filing for bankruptcy. The impact medical expense-related debt has on cancer patients was revealed:

“The researchers found that cancer patients were 2.65 times more likely to go bankrupt than people without cancer. Bankruptcy rates were two to five times higher among younger cancer patients compared to cancer patients age 65 years or older, which indicates that Medicare and Social Security may mitigate bankruptcy risk for the older group.”

The researchers, led by Scott Ramsey, M.D., Ph.D, analyzed medical, personal, legal, and bankruptcy records covering the Western District of Washington State in U.S. Bankruptcy Court from 1995 to 2009. They concluded that “employers and governments may have a policy role to play in creating programs and incentives that could help people cover expenses in the first year following a cancer diagnosis.”

According to a separate study conducted by researchers at the National Cancer Institute, the cost of cancer care in the United States is expected to rise from an estimated $125 billion in 2010 to $158 billion by the year 2020. Some patients are forced to end treatments early due to their inability to afford further care. Others continue to build debt until they are left wondering how they will cover the bills.

Know more about mesothelioma and how you can deal with it.

Mesothelioma Survivors Celebrate Life on National Cancer Survivors Day

Mesothelioma survivors are encouraged to join the nearly 14 million other Americans, and millions more around the world, who have survived cancer on Sunday, June 2, 2013, to observe the 26th annual National Cancer Survivors Day.

The day is a CELEBRATION for those who have survived, an INSPIRATION for those recently diagnosed, a gathering of SUPPORT for families, and an OUTREACH to the community, according to the National Cancer Survivors Day Foundation who sponsors the day. We encourage all mesothelioma survivors to take the time out to celebrate with your family and others.

“When most people hear the word ‘cancer,’ they automatically think the worst,” says Foundation spokesperson, Laura Shipp in a press release announcing the day. “But the truth is that more people are living longer and better quality lives after cancer than ever before. National Cancer Survivors Day is an opportunity for these cancer survivors to come together and celebrate this new reality in cancer survivorship. There is life after cancer. And that’s something to celebrate.”

The American Cancer Society reports the average survival time for people with mesothelioma, a rare cancer caused by past asbestos exposure, is between 4 and 18 months. However, between 5% and 10% of mesothelioma patients will live at least 5 years after diagnosis. The five-year survival rate for lung cancer is 16%, according to the National Lung Cancer Partnership.

The National Lung Cancer Partnership celebrated its ten-year anniversary last year, and to mark its next decade it announced its bold, new vision: double the overall five-year lung cancer survival rate, currently at 16%, to 32% in the United States by 2022.

We honor these special mesothelioma survivors who have battled the cancer and have been able to move on with their lives despite the challenges they face every day: Jan Egerton, a nearly 15-year survivor from England, Lou Williams, 10-year survivor of Australia, and Don Smitley, 18-month survivor from Pennsylvania. You can read more about these mesothelioma warriors through “Faces of Mesothelioma.”

Women cancer survivors are asked to attend the National Women Survivors Convention, which will take place this year in Nashville, TN, August 22 – 24. It’s a fabulous opportunity to learn, network and celebrate with other women survivors.

To find out more about National Cancer Survivors Day visit the Foundation’s website.

Part II: Chris Knighton Promised to Fight for Mesothelioma Victims

After she lost her husband to mesothelioma, Chris Knighton formed the Mick Knighton Mesothelioma Research Fund to help find a cure for the asbestos-caused disease. In Part II of this series, see how Chris’s efforts earned her a Royal award.

Support Groups and The Snowdrop Memorial

In addition to funding research, the MKMRF offers support groups for anyone who has been touched by mesothelioma. The Mesothelioma Self-help/Friendship Groups provide information and support to sufferers and their families. The groups meet once a month and welcome all whose lives have been affected by mesothelioma.

“We understand how important it is to meet others, and to share experiences, as well as having some fun along the way,” said Chris.

Another way to bring families together has been through the Snowdrop Fund. Chris created the fund to raise money, but also as a lasting tribute to loved ones. Families can set up Snowdrop Funds in memory of a loved one through the MKMRF website, and friends and families can make donations to MKMRF in memory of that person.

Often described as looking like three drops of milk hanging from a stem, snowdrop flowers, according to Chris, “work hard through the depth of winter; then come spring they bloom, bringing hope and promise for a better future.”

As part of the launch of the Snowdrop Memorial Fund, North Tyneside Council and Gateshead Council agreed to plant 5,000 snowdrops along the banks of the Tyne River in North East England, where thousands of people were previously exposed to asbestos.

Treasured Experience

Chris’s resolve to bring an end to deaths from mesothelioma earned her one of the highest honors for an English citizen – Member of the Order of the British Empire (MBE) Award. This is awarded for a significant achievement or outstanding service to the community. An MBE is also awarded for local ‘hands-on’ service which stands out as an example to other people.

“This is truly a treasured experienced,” said Chris upon receipt of the award by Her Majesty the Queen at Buckingham Palace last November.

Although humbled by the honor, Chris has no plans to give up on her quest to help find a cure for mesothelioma.

“For the first time ever, there is much to be positive about in terms of mesothelioma research; therefore MKMRF will continue to raise much needed funding in the hope of delivering real advances into treatment and understanding of the disease.”

Make a donation or find out more about Chris and the Mick Knighton Mesothelioma Research Fund.

See Part I of Chris Knighton’s fight for Mesothelioma Victims posted last week.

Author: Nancy Meredith