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PBS and HHMI Tangled Bank Studios Announce THE GENE DOCTORS: The Dawn of a New Era in Medicine

29 September 2017 Howard Hughes Medical Institute

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, at PBS.org/genedoctors and for broadcast on local PBS stations, takes viewers to the front lines of this medical revolution, unfolding in real time, exploring new gene therapies and groundbreaking technologies on the horizon, like CRISPR.

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 on October 12, the FDA’s Cellular, Tissue and Gene Therapies Advisory Committee (CTGTAC) will meet in an open session to discuss and make recommendations on the safety and effectiveness of it.  This therapy—featured in the film—could become one of the first-ever gene therapies 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.

http://www.pbs.org/genedoctors

Attached files

  • The Gene Doctors Press Release AlphaGalileo


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