Biotech companies are navigating new trends in addressing rare diseases, conditions effecting 200,000 or fewer Americans. Jeremy Springhorn (Alexion Pharmaceuticals), Andrew Curtis (Pfizer), Marc Beer, (Aegerion Pharmaceuticals) and Susan Kahn (National Tay-Sachs & Allied Disease Association), shared their perspectives on the future of new treatments for rare diseases during a panel discussion at the International BIO Convention in Washington D.C. Alexion Pharmaceuticals invented Soliris, the first and only therapy for treating Paroxysmal Nocturnal Hemoglobinuria (PNH), a rare, progressive and life-threatening disease defined by hemolysis, the destruction of red blood cells. Pfizer created a new research unit in 2010 with the goal of leveraging existing scientific experience in rare diseases, such as hemophilia, to discover novel, life-saving medicines for patients with large unmet medical need. Aegerion Pharmaceuticals is developing a new drug to treat Homozygous Familial Hypercholeterolemia (HoFH), a rare genetic lipid disorder resulting in an accumulation of low density lipoprotein (LDL-C) often referred to as bad cholesterol in the blood. The National Tay-Sachs & Allied Disease Association is directly funding research to treat and cure Tay-Sachs, Canavan and related genetic disease and supporting affected families and individuals. Tay-Sachs disease is a rare progressive neurological genetic disorder that is caused by the absence or insufficient level of a vital enzyme called Hexosaminidase (Hex-A). All children with classic Tay-Sachs disease die early in childhood, usually by the age of 5, and today there is no cure or effective treatment. Jeremy Springhorn summed up the sentiments of the panelists: “To commit to putting the resources it takes to developing treatments for rare diseases, our focus is and will always remain on the patient.”
Thursday, June 30, 2011
Tuesday, June 28, 2011
CHI was joined by the U.S.-Korea Business Council, the Silicon Valley U.S. Export Assistance Center, Sunnyvale Mayor Melinda Hamilton and other key stakeholders at a medical technology industry roundtable held June 24 with Jong hyun Choi, Minister of Economic Affairs, Embassy of South Korea at Accuray in Sunnyvale, Calif. The meeting focused on the support of the California medical technology community for the U.S.-Korea Free Trade Agreement (FTA), the first FTA to include an entire chapter with commitments to openness and transparency in medical technology regulation and payment policies. CHI members Mark Deem, partner of the medical technology incubator, The Foundry, and Tom Loarie, executive chairman of Mercator MedSystems, shared their experiences bringing medical technology innovations to market and highlighted the need for open markets and fair regulatory and payment schemes worldwide to support development of technologies that can improve the lives of patients. Fred Kinder, a former CyberKnife patient, spoke of the need for access to medical technologies for all patients. Korea is one of the world’s largest and fastest growing markets for medical technology, with expected growth of 10-15 percent per year over the next several years. Following the meeting Accuray CEO Euan Thomson, Ph.D., gave a demonstration of the CyberKnife System to highlight how the technology is used to treat tumors in patients with cancer. Watch NBC News coverage of the event.
Monday, June 27, 2011
Attendees at the Annual International BIO Convention kicked off the meeting Monday morning with a healthy 5K run hosted by the Diabetes Advocacy Alliance at Bluemark Park just outside the nation’s Capitol. I was joined by CHI member Sarah Nordstrom from Novo Nordisk and other biotech runners who came out to support the Partnership for a Healthier America, a partner to First Lady Michelle Obama’s Let’s Move initiative to combat childhood obesity. I was still on California time (4:00 a.m.!), so it was early to get up and run, but well worth the effort to raise awareness for a great cause! Watch the video.
Monday, June 20, 2011
As a volunteer for the Rolling Readers program, I began each Wednesday morning at Ocean Beach Elementary School, a community-oriented school that values education and learning through traditional and creative means. CHI allowed me the opportunity to bring my love of reading to the school each week, where I spent time on the colored floor mat of Ms. Tanner’s classroom – a lively bunch of students eager to read and quick with questions.
Rolling Readers connects volunteers to underprivileged classrooms, where we delve into classic tales (Curious George and Shel Silverstein books) and modern fantasies (John Lithgow’s I’m a Manatee).
The organization’s mission is a simple one: to inspire children to love reading. Children who love reading are more likely to become lifelong learners, so the saying goes.
I found inspiration through these staggering Rolling Readers statistics:
• In low income neighborhoods, the ratio of books to children is 1:300—which means that, on average, there is one book for every 300 children.
• By age four, children who live in poor families will have heard 32 million fewer words than children living in professional families.
Since every child deserves a book of their own, I was especially excited to participate in the annual Rolling Readers book drive. Each student in the classroom received a special edition of the popular Dr. Seuss book The Cat in the Hat (appropriately written in La Jolla).
The students erupted with joy when they saw the book. It is satisfying to know that they will be taking the book home to share, which may encourage reading among their friends and family.
Theodor Seuss Geisel, himself an advocate for elementary literacy, once said: "The more that you read, the more things you will know. The more that you learn, the more places you'll go.”
CHI-Advancing California biomedical research and innovation
Wednesday, June 8, 2011
Rep. McCarthy was interested in the group’s perspective on what policies are stifling innovation and U.S. competitiveness. The U.S. Food and Drug Administration (FDA) is not keeping pace with U.S. biomedical innovation. The Agency-industry partnership is strained by unexplained regulatory delays, by a lack of clear standards for what clinical data are necessary for product approval, and by a bureaucracy whose communications are neither consistent nor predictable. In this regulatory environment, companies are launching products first abroad forcing jobs and investment offshore. NIH funding must also be preserved to ensure a healthy pipeline of new research for product development. Rep. McCarthy is optimistic on American ingenuity and innovation and wants to make better government to advance it: “We have the framework to turn it around, but do we have the willpower?”
CHI-Advancing California biomedical research and innovation