PBS and HHMI Tangled Bank Studios Announces THE GENE DOCTORS: The Dawn of a New Era in Medicine
ARLINGTON, VA (August 22, 2017) — Every year more than one million babies are born worldwide with a disease caused by a single error in one of our many genes. These errors, or mutations, can cause genetic illnesses that are often severe and can rob people of sight, breath, movement and life. Now, for the first time, doctors can take aim at the root causes of these diseases. THE GENE DOCTORS, available to stream on Monday, October 2, 2017, on PBS.org and for broadcast on local PBS stations, takes viewers to the front lines of this medical revolution, unfolding in real time.
THE GENE DOCTORS, a one-hour documentary produced by HHMI Tangled Bank Studios, explores remarkable recent advances in genetic medicine through intimate stories of patients and families battling inherited blindness, cystic fibrosis, muscular dystrophy and fatal familial insomnia, and the doctors and scientists working to help them.
The film’s release in October comes at a time when gene therapies have made recent headlines as they move through the U.S. Food and Drug Administration’s (FDA) review process. A new gene therapy for inherited blindness, with the potential to restore vision, is currently under priority FDA review, and an update on its status is expected soon. This therapy—featured in the film—could become the first-ever gene therapy approved for use in the U.S.
“New gene therapies are transforming lives in ways previously thought impossible,” said John Rubin, executive producer of THE GENE DOCTORS. “We are moving from treating the symptoms of terrible inherited diseases to providing correct copies of faulty genes or repairing the havoc they cause. It’s an extraordinary time in medicine.”
In THE GENE DOCTORS, viewers meet Molly Troxel, an Omaha teenager who was declared legally blind as a child due to a defect in one of her genes. Molly joined a clinical trial for the blindness gene therapy, conducted by Dr. Jean Bennett at Children’s Hospital of Philadelphia and the University of Pennsylvania, undergoing a procedure in which her retinas were injected with a virus that delivered a correct version of the faulty gene. In what some describe as a miracle of science, Molly’s vision was restored.
Kimberly Ramirez, a teen from San Francisco, has cystic fibrosis, one of the most common fatal genetic diseases in the U.S., which affects the lungs and digestive system, making breathing difficult and lungs vulnerable to frequent infections. For 11 years, she and her family spent most Christmases in the hospital. As her doctors considered a lung transplant, she started taking a new drug designed to improve the function of a deformed molecule produced by the faulty gene responsible for her disease. For Kimberly, the impact of this new drug was dramatic. THE GENE DOCTORS chronicles the life-changing results.
THE GENE DOCTORS also follows the lives of Boston-area teen brothers Austin and Max Leclaire, who both suffer from Duchenne muscular dystrophy, which causes progressive muscular degeneration, confining most children to wheelchairs by the time they are teenagers. The brothers and their family participate in a trial for a promising new drug that guides patients’ cells to skip over the disease-causing genetic typo. The FDA recently granted approval of this drug, which has the potential to change the lives of thousands of others suffering from this debilitating disease.
Viewers also meet Sonia Vallabh, a Harvard-educated lawyer who, after watching her mother die from an extremely rare brain disease called fatal familial insomnia, received news that she had inherited the same disease-causing mutation. With no available treatment, Sonia is unlikely to live past age 50. However, she is determined not to stand by and let the disease take its toll. She and her husband quit their jobs and are working as biomedical researchers to find a cure.
“It did seem incredible that this one-letter change could be so dangerous,” said Sonia, now a PhD student in biology at Harvard Medical School in Boston and co-founder of the Prion Alliance. “At no other time in history could I have taken on my own illness, and today that is just what I am doing. Treating disease at its root is hard, but as we are seeing, remarkable progress is possible.”
These promising treatments are just the first wave. A revolutionary new technology called CRISPR promises the ability to edit and repair broken genes with pinpoint precision, letter-by-letter. This breakthrough technology was recently used to correct a dangerous gene mutation in human embryos and has the potential to cure a wide range of genetic diseases.
THE GENE DOCTORS tells the stories of the people and innovations driving the science changing the future of medicine. Additional information about the film is available now at PBS.org/genedoctors.
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Learn more about the diseases highlighted in the film
Cystic Fibrosis (CF)
Duchenne Muscular Dystrophy (DMD)
Fatal Familial Insomnia (FFI)
Leber Congenital Amaurosis (LCA)/Inherited Blindness
About PBS
PBS, with nearly 350 member stations, offers all Americans the opportunity to explore new ideas and new worlds through television and digital content. Each month, PBS reaches nearly 100 million people through television and nearly 28 million people online, inviting them to experience the worlds of science, history, nature and public affairs; to hear diverse viewpoints; and to take front row seats to world-class drama and performances. PBS’ broad array of programs has been consistently honored by the industry’s most coveted award competitions. Teachers of children from pre-K through 12th grade turn to PBS for digital content and services that help bring classroom lessons to life. Decades of research confirms that PBS’ premier children’s media service, PBS KIDS, helps children build critical literacy, math and social-emotional skills, enabling them to find success in school and life. Delivered through member stations, PBS KIDS offers high-quality educational content on TV – including a new 24/7 channel, online at pbskids.org, via an array of mobile apps and in communities across America. More information about PBS is available at www.pbs.org, one of the leading dot-org websites on the internet, or by following PBS on Twitter, Facebook or through our apps for mobile and connected devices. Specific program information and updates for press are available at pbs.org/pressroom or by following PBS Pressroom on Twitter.
About HHMI Tangled Bank Studios
HHMI Tangled Bank Studios is a production company established and funded by the Howard Hughes Medical Institute as an extension of its longstanding science education mission. Dedicated to the creation of original science documentaries for broadcast, theatrical and digital distribution, the company’s award-winning programs address important contemporary issues and capture compelling stories of discovery across all branches of scientific inquiry. For more information, please visit www.tangledbankstudios.org. Follow us on Twitter, Facebook and Instagram. HHMI is a science philanthropy whose mission is to advance basic biomedical research and science education for the benefit of humanity. The institute is the largest private supporter of science education in the country.
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For additional information, photos, interviews and more, contact:
Jennifer Farber
GMMB
Jen.Farber@GMMB.com
202.813.4878
Anna Irwin
HHMI Tangled Bank Studios
irwina@hhmi.org
301.215.8861