When Sylvan Menezes was diagnosed with amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease, in 2004, he refused to give up. ALS is a disease that attacks nerve cells in the brain and spinal cord, leading to muscle weakening, twitching, and the inability to move the arms, legs, and body. The former teacher, social worker, business owner, and marine mechanic has become involved with the ALS Therapy Development Institute (ALS TDI), attending annual seminars, supporting the ALS TDI "A Cure is Coming" awareness walk, and attending the ALS Association's Advocacy Days. Thanks to ALS TDI, Menezes has been able to build relationships with other ALS patients. Although the disease has since affected Menezes' upper body, weakening his neck muscles, arms, and tongue and limiting his ability to breathe, he tries to remain positive. "My family give me strength," he says. "Their support and the support of my friends has made this a tolerable experience. My outlook has been to do as much as possible while I am still able. My wife and I were able to travel extensively and make many memories over the last few years. I try to maintain a positive attitude and take each day as it comes."
While most of Griffin Lynch's classmates were celebrating their last few months as high school seniors, the North Carolina student was fighting for his life. Being diagnosed with a malignant tumor in the left ventricle of his brain put Griffin's life on pause. Surgery successfully removed 80% of the tumor, but left him unable to swallow, speak, or move his right side. Complications caused him to go legally blind. After intensive rehabilitation, it became necessary to remove the last 20% of the tumore through eight weeks of proton beam radiation therapy. Griffin and his mom, Eileen O'Flaherty, came to Massachusetts General Hospital, one of the few facilities to offer this treatment. Due to the nature of the treatment, the family had to seek housing nearby. Christopher's Haven, established by cancer survivor Dan Olsen, offers families comfortable and affordable living space right near Mass General. The proximity of other families provided the Lynch's with much needed emotional support and comfort. After the successful eight week therapy, Griffin and his mother returned home. He is now a Freshman at UNC-Chapel Hill. "The team [at Christopher's Haven] continues to follow him, emotionally and socially," says O'Flaherty. "They're just gifted, loving people."
Former Massachusetts Governor Paul Cellucci was diagnosed with amyotrophic lateral sclerosis (ALS), or Lou Gehrig's disease in 2011. This degenerative disorder attacks nerve cells in the brain and spinal cord, which leads to muscle weakness and loss of voluntary movement. Under the care of Dr. Robert H. Brown, one of the world's leading ALS researchers, Cellucci was inspired to both spread hope and fuel research surrounding the disease. He is spearheading the UMass ALS Champion Fund, an initiative to raise $10 million that will go directly to Dr. Brown and his team towards ALS research and breakthroughs. "My hope", says Cellucci, "is that we can get breakthroughs that will slow or stop ALS progression and ultimately lead to a cure -- the sooner, the better."
Beth Pond understands all too well the challenges of living with X-linked Hypohidrodic Ectodermal Dysplasia (XLHED). Her brother, Steve, and two of her sons, Nolan (age 16) and Philip (age 15), are affected by the rare orphan disease which causes a range of symptoms, including an absence of sweat glands, poor temperature control, respiratory problems, hair loss, and missing and malformed teeth. Individuals affected by XLHED are at an especially high risk during their early years of life, as their inability to sweat often leads to hypothermia. Pond had a second brother who likely succumbed to those complications when he was only six weeks old. “He died on a hot, summer day while taking a nap,” said Pond. “Doctors said it was SIDS, but we think that he had XLHED and overheated. It’s important to understand that XLHED can be life-threatening and deadly, not just a host of medical and dental needs.” Because of her proximity to the disease, Pond has become an outspoken advocate for XLHED awareness, serving as a regional family liaison for the National Foundation for Ectodermal Dysplasias (NFED), where she works to create a network of communications for families affected by the disease. “I know a lot of families who, after their first child is born with HED, don’t have any more children and that child is all alone,” said Pond. “Families find support and friendship from other families who are going through the same thing.” Through her involvement with the foundation, she was introduced to Cambridge-based Edimer Pharmaceuticals—a company dedicated to delivering a significant and durable improvement in the health and quality of life to future generations affected by XLHED. Currently, Edimer is in the process of developing an innovative therapy known as EDI200, which has been shown in animal studies to substitute for Ectodysplasin-A1—a protein that is crucial to the development and formation of skin and teeth. EDI200 is expected to go into clinical trial in late 2011/early 2012. “It’s heartbreaking to me that it won’t help my brother or sons, but it does give hope to future generations,” said Pond. “All of Philip and Nolan’s daughters will be carriers, so hope is all we’ve got.”
As the president and scientific director of Microtest Laboratories, Steve Richter has dedicated his career to saving lives, but when it came to saving his own, he can thank Rituxen, an antibody co-marketed by industry pioneers, Genentech and Biogen Idec. At the age of fourteen, Richter was diagnosed with lupus—a chronic, inflammatory autoimmune disorder affecting the skin, joints, kidneys, and other organs. This diagnosis provided the spark that motivated Richter to pursue a career in the life sciences, eventually founding the Agawam-based Microtest in 1984. Though the lupus remained asymptomatic for many years, Richter was later diagnosed with two more debilitating autoimmune disorders—rheumatoid arthritis and idiopathic thrombocytopenic purpura (ITP), a bleeding disorder in which the immune system destroys platelets necessary for normal blood-clotting. Steve began a four-month treatment of immunosuppressive agents to reduce the clones of B-cells that had begun inadvertently attacking healthy cells and tissues in his body. He was in remission until last spring, when his platelets fell to dangerously low levels. Rather than undergoing a splenectomy, Richter opted to begin a program consisting of weekly Rituxen treatments in conjunction with targeted chemotherapy. After four Rituxen treatments, his platelets started returning to normal levels. “I wasn’t bruising anymore; I could even play sports,” said Richter. “I had more energy and just felt better overall. It definitely improved my quality of life.” He currently serves on the boards of both MassBio and MassBioEd and remains dedicated to advancing the work of the life sciences. “It’s an amazing industry, helping so many people like myself,” said Richter. “To be involved is incredibly exciting.”
According to a World Health Organization estimate, pneumococcal disease is the leading cause of vaccine-preventable death worldwide in children younger than five years old. Thanks to Pfizer’s new pediatric vaccine, Prevnar 13®, that could all change. Michael Hodgdon, a senior supervisor at Pfizer’s Andover facility, played an important role in the vaccine’s development, which took over four years. “That’s the longest I’ve ever been on a project, but it’s by choice,” said Hodgdon. “It’s exciting what we’re doing. No matter what your day is like, you can go home thinking, ‘I just helped thousands of kids around the world.’” Approved by the U.S. Food and Drug Administration in February, Prevnar 13 was developed for children between the ages of six weeks and five years, which makes it especially poignant for Hodgdon, who has two young granddaughters of his own, Morgan and Brady. Just a few months ago, they both received the vaccination. “My daughter told them, ‘Grandpa made it with love,’” he said. “We take pride in what we do here and strive for safety first. I know first-hand how it’s made and about our rigorous safety standards. I would never hesitate to have my grandbabies vaccinated.” Prevnar 13 helps prevent invasive pneumococcal disease caused by some of the most common strains of the bacteria threatening young children today. Invasive pneumococcal disease includes bacterial meningitis, which is an inflammation of the covering of the brain and spinal cord. “I love this product,” said Hodgdon, who has been working in the biotech industry since 1988. “You can’t overestimate the value it will have, especially in the world’s poorest countries where children die of pneumonia all the time.”
Although you would not know it by looking at him, six-year-old Will Doggett of Franklin, Mass., once experienced severe complications from the seasonal respiratory syncytial virus (RSV). Three-and-a-half weeks after being delivered early via cesarean section, Will was rushed to Children’s Hospital in Boston where he was intubated, placed on a ventilator and diagnosed with RSV. When his condition did not improve, Will was placed on the heart-lung bypass system extracorporeal membrane oxygenation (ECMO) for 12 days and spent a total of three months in the hospital – a record for the longest stay at Children’s for an RSV patient. Will’s mother, Ariel Doggett, said the MedImmune prescription medication Synagis®, a shot administered monthly during RSV season, was not offered to Will because he “didn’t qualify.” She said had her son been given Synagis at the time, he probably would’ve been spared a lot of his experience on ECMO. However, Will was given the shot the following two RSV seasons and he has not contracted the disease since. Ariel is now an advocate for Synagis because she wants parents of children coping with the disease to have greater knowledge than she and her husband had. “It’s important for the industry and the public to see the other side of it – how really, really sick these kids can get,” she said. “As a parent, you don’t like to imagine it. But you have to be your own advocate. Nobody knows your child like you do.”
William Johnson, Athletic Director of Silver Lake Regional High School in Kingston, Mass., enjoyed spending time in the sun when he was young. But Johnson's infrequent use of sunscreen began to affect his skin over the years, and led to a diagnosis of actinic keratoses (AKs). Thanks to Wilmington, Mass., based DUSA Pharmaceuticals, Inc., Johnson overcame AKs with the help of the company's Levulan® Photodynamic Therapy (Levulan PDT), one of the fastest growing procedures in dermatology. Levulan PDT uses an advanced light-activated drug therapy to destroy AKs. Johnson was pleased with the treatment's results, as nearly all of his AKs were destroyed during the four-month treatment period, and only a minimal amount has appeared since. Johnson has been able to get on with his life, saying "It would be impossible for me to stay out of the sun." Johnson, who remains a fixture on the Silver Lake sidelines, said it gives him "more confidence that this is something that can at least be treated."
Jeffrey Bardsley knows no limits when it comes to pursuing his dreams. After being diagnosed with Hunter syndrome when he was four years old, the 2009 graduate of the University of Richmond was given a second chance when he was one of 12 patients selected to participate in a clinical trial at the University of North Carolina at Chapel Hill. Bardsley was treated with ELAPRASE, an enzyme replacement therapy developed by Transkaryotic Therapies Inc. (TKT), and acquired by the Cambridge-based Shire Human Genetics Therapies Inc. (Shire HGT). Although he still receives weekly infusion treatments since there is no cure for Hunter syndrome, Bardsley would like to become more involved with lobbying for additional research funding for companies like Shire upon completing grad school. "I've been very impressed with Shire's attitude and motivation to make improvements in patients' lives," he said. "It's exciting, working together to try to find solutions."
Steven Schatzkin, a consultant, husband and father of two, was diagnosed with stage III non-Hodgkin's lymphoma in his spine in December 2004. Schatzkin participated in a clinical trial of Mozobil, a drug developed by Genzyme to help mobilize stem cells in non-Hodgkin's lymphoma and multiple myeloma patients. As a result of the successful treatment, Schatzkin was able to ride in the Pan-Mass Challenge, the 140-mile bike-a-thon for cancer research and treatment. He has also served as an online mentor to non-Hodgkin's lymphoma patients, offering advice and sharing his personal story on several message board sites. "It changed my life in every way," he said. "When you go through this process, just to witness the kindness, love and support - even from complete strangers and all the doctors and nurses - it was incredible. Now it's my turn to give it back."
Maddie Hunter, a New Jersey-based business consultant and coach, loving mother and daughter, has triumphed over multiple myeloma thanks in part to Velcade, a proteasome inhibitor developed by the Cambridge biopharmaceutical company Millennium Pharmaceuticals, Inc. The cancer drug was launched in May 2003 for patients with relapsed and refractory myeloma, which was a perfect fit for Hunter. After two cycles of Velcade spanning two months, Hunter went back into complete remission. The treatment's incredible result led Hunter to form a strong relationship with the company. Last year, Hunter was selected to speak at Millennium's annual meeting where she and fellow myeloma patients shared their stories. "It's not an accident I've become such a strong supporter of Millennium," said Hunter. "They're helping me to stay alive. But it's not just that; it's a very special company. They have a strong culture of connection and affirmation of one another that's very unique and that shows up in the quality of what they do in the world."
"MS is my motivational spirit." That is not usually a phrase you expect someone living with the disease to say, but Tara Hillstrand-Lane is determined to overcome relapsing multiple sclerosis. Hillstrand-Lane, a coach and figure skating and skiing enthusiast from Colorado, was diagnosed in March 2000, just weeks after her wedding day. She immediately took hold of her MS, as she found the right physician for her needs and participated in an evidence study that compared two drugs approved for relapsing MS in a head-to-head trial. Hillstrand-Lane remains committed to her original treatment plan and serves as an MS LifeLines Amassador for EMD Serono, which has allowed her to reach out to people who have face similar challenges. "By talking to others and letting them know they're not alone, it gives them hope and encouragement. To have someone give you a hug and say thank you, that's pretty powerful. I think the people I've met have had as much of an impact on me as I've had on them."
Arkansas native Greg Jenkins was diagnosed with a tumor in the fall of 2008. Despite undergoing a craniotomy and having 100 percent of the tumor removed, it returned shortly thereafter and was diagnosed as being malignant, meaning it would return without radiation treatment. In combating the challenge, Jenkins spent eight weeks at the AstraZeneca Hope Lodge Center in Boston. A relief from the burden of hotel and transportation bills was important to Jenkins, a lifelong resident of Arkansas who works for an independent insurance agency. He and his wife, Amy, a teacher, have a six-year-old son and a baby daughter on the way. "I can't imagine a better program than [Hope Lodge]," said Jenkins. "Staying somewhere else for two months, I would have been looking at $6,000-7,000 out of my pocket. It truly would have been a financial hardship. Plus, in a hotel, you're trapped in your room all day by yourself. Here, there was a constant stream of people and I developed several friends just in a short time. It allowed me to maintain a high quality of life."
MassBio Member News
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- February 20, 2015 Prominent Proteomics Business Development Team Joins Nuclea
- February 19, 2015 Clinigen Group Adds New Named Patient Program to Global Access Portfolio
- February 18, 2015 Seeding Labs Recognized by Fast Company for its Contribution to Global Science
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