One of those patients, Emily Whitehead, now 12 and the first child ever given the altered cells, was at the meeting of the panel with her parents to advocate for approval of the drug that saved her life. In 2012, as a 6-year-old, she was treated in a study at the Childrens Hospital of Philadelphia. Severe side effects raging fever, crashing blood pressure, lung congestion nearly killed her. But she emerged cancer free, and has remained so.
We believe that when this treatment is approved it will save thousands of childrens lives around the world, Emilys father, Tom Whitehead, told the panel. I hope that someday all of you on the advisory committee can tell your families for generations that you were part of the process that ended the use of toxic treatments like chemotherapy and radiation as standard treatment, and turned blood cancers into a treatable disease that even after relapse most people survive.
The main evidence that Novartis presented to the F.D.A. came from a study of 63 patients who received the treatment from April 2015 to August 2016. Fifty-two of them, or 82.5 percent, went into remission a high rate for such a severe disease. Eleven others died.
Its a new world, an exciting therapy, said Dr. Gwen Nichols, the chief medical officer of the Leukemia and Lymphoma Society, which paid for some of the research that led to the treatment.
The next step, she said, will be to determine what we can combine it with and is there a way to use it in the future to treat patients with less disease, so that the immune system is in better shape and really able to fight. She added, This is the beginning of something big.
At the meeting, the panel of experts did not question the lifesaving potential of the treatment in hopeless cases. But they raised concerns about potentially life-threatening side effects short-term worries about acute reactions like those Emily experienced, and longer-term worries about whether the infused cells could, years later, cause secondary cancers or other problems.
Oncologists have learned how to treat the acute reactions, and so far, no long-term problems have been detected, but not enough time has passed to rule them out.
Patients who receive the treatment will be entered in a registry and tracked for 15 years.
Treatments involving live cells, known as biologics are generally far more difficult to manufacture than standard drugs, and the panelists also expressed concerns about whether Novartis would be able to produce consistent treatments and maintain quality control as it scaled up its operation.
Another parent at the meeting, Don McMahon, described his son Connors grueling 12 years with severe and relapsing leukemia, which started when he was 3. Mr. McMahon displayed painful photographs of Connor, bald and intubated during treatment. And he added that chemotherapy had left his son infertile.
A year ago, the family was preparing for a bone marrow transplant when they learned about the cell treatment, which Connor then underwent at Duke University. He has since returned to playing hockey. Compared with standard treatment, which required dozens of spinal taps and painful bone marrow tests, the T-cell treatment was far easier to tolerate, Mr. McMahon said, and he urged the panel to vote for approval.
A third parent, Amy Kappen, also recommended approval, even though her daughter, Sophia, 5, had died despite receiving the cell treatment. But it did relieve her symptoms and give her a few extra months. Sophias disease was far advanced, and Ms. Kappen thought that if the treatment could have been given sooner, Sophia might have survived.
We hope that more families have a longer time with their children fighting this evil disease, and our children deserve this chance, she said.
The treatment was developed by researchers at the University of Pennsylvania and licensed to Novartis.
Use will not be widespread at first because the disease is not common. It affects only 5,000 people a year, about 60 percent of them children and young adults. Most children are cured with standard treatments, but in 15 percent of cases like Emilys and Connors the disease does not respond, or it relapses.
Analysts predict that these individualized treatments could cost more than $300,000, but a spokeswoman for Novartis, Julie Masow, declined to specify a price.
Although the figure may seem high, people with cancer often endure years of expensive treatment and repeat hospital stays that can ultimately cost even more.
Because the treatment is complex and patients need expert care to manage the side effects, Novartis will initially limit its use to 30 or 35 medical centers where employees will be trained and approved to administer it, the company said.
As to whether the treatment, known as CTL019 or tisagenlecleucel (pronounced tis-a-gen-LEK-loo-sell), will be available in other countries, Ms. Masow said by email: Should CTL019 receive approval in the U.S., it will be the decision of the centers whether to receive international patients. We are working on bringing CTL019 to other countries around the world. She added that the company would file for approvals in the European Union later this year.
By late November 2016, 11 of the 52 patients in the study who went into remission relapsed. Twenty-nine were still in remission. Eleven others had further treatments, like bone marrow transplants. One patient was not available for assessment. Three who had relapses died, and one who did not relapse died from a new treatment given during remission. The median duration of remission is not known because it has not been reached: Some patients were still well when last checked.
Researchers are still debating about which patients can safely forgo further treatment, and which might need a bone marrow treatment to give the best chance of a cure.
The treatment requires removing millions of a patients T-cells a type of white blood cell often called soldiers of the immune system and genetically engineering them to kill cancer cells. The technique employs a disabled form of H.I.V., the virus that causes AIDS, to carry new genetic material into the T-cells to reprogram them. The process turbocharges the T-cells to attack B-cells, a normal part of the immune system that turn malignant in leukemia. The T-cells home in on a protein called CD-19 that is found on the surface of most B-cells.
The altered T-cells are then dripped back into the patients veins, where they multiply and start fighting the cancer.
Dr. Carl H. June, a leader of the University of Pennsylvania team that developed the treatment, calls the turbocharged cells serial killers. A single one can destroy up to 100,000 cancer cells.
Because the treatment destroys not only leukemic B-cells but also healthy ones, which help fight germs, patients need treatment to protect them from infection. So every few months they receive infusions of immune globulins.
In studies, the process of re-engineering T-cells for treatment sometimes took four months, and some patients were so sick that they died before their cells came back. At the meeting, Novartis said the turnaround time was now down to 22 days. The company also described bar-coding and other procedures used to keep from mixing up samples once the treatment is conducted on a bigger scale.
Michael Werner, a lawyer and expert on gene and cell technologies and regulation, and a partner at Holland and Knight in Washington, said that results so far proved that T-cell treatment works.
The fact that it can be done means more people will go into the field and more companies will start developing these products. He added, I think were in for really exciting times.
Katie Thomas contributed reporting.
A version of this article appears in print on July 13, 2017, on Page A1 of the New York edition with the headline: F.D.A. Panel Urges New Living Drug To Fight Cancer.
See the original post here:
- Bitcoin Price Forecast and Analysis – September 19, 2017 - September 20th, 2017
- Litecoin Price Forecast and Analysis – September 19, 2017 - September 20th, 2017
- Ripple Price Forecast and Analysis – September 19, 2017 - September 20th, 2017
- Ripple Price Forecast and Analysis – September 18, 2017 - September 20th, 2017
- Litecoin Price Forecast and Analysis – September 18, 2017 - September 20th, 2017
- Ethereum Price Forecast and Analysis – September 18, 2017 - September 20th, 2017
- This Cryptocurrency Could Be the Next Bitcoin - September 20th, 2017
- Ripple Price Forecast and Analysis – September 15, 2017 - September 20th, 2017
- Ethereum Price Forecast and Analysis – September 15, 2017 - September 20th, 2017
- Ethereum Price Forecast and Analysis – September 19, 2017 - September 20th, 2017
- First genetic engineering therapy approved by the FDA for leukemia - Ars Technica - August 30th, 2017
- genetic engineering | Definition, Process, & Uses ... - August 24th, 2017
- Genetic Engineering Advantages & Disadvantages - Biology ... - August 24th, 2017
- Genetic engineering - Wikipedia - August 22nd, 2017
- genetic engineering | Definition, Process, & Uses ... - August 22nd, 2017
- Genetic Engineering: What is Genetic Engineering? - August 21st, 2017
- genetic engineering | Definition, Process, & Uses ... - August 21st, 2017
- Gene therapy - Wikipedia - August 21st, 2017
- Genetic Engineering Advantages & Disadvantages - Biology ... - August 21st, 2017
- Genetic engineering - Biology-Online Dictionary - August 21st, 2017
- History of genetic engineering - Wikipedia - August 21st, 2017
- ADRIAN Kibbler wonders whether genetic engineering may be used in the future to prevent illness - Ludlow Advertiser - August 21st, 2017
- ADRIAN Kibbler wonders whether genetic engineering may be used in the future to prevent illness - Ludlow Advertiser - August 19th, 2017
- Listening for the Public Voice - Slate Magazine - August 16th, 2017
- The Impossible Burger wouldn't be possible without genetic engineering - Grist - August 12th, 2017
- Genetically Engineering Pigs to Grow Organs for People - The Atlantic - August 11th, 2017
- When genetic engineering is the environmentally friendly choice - GreenBiz - August 10th, 2017
- Gene Editing Might Mean My Brother Would've Never Existed - TIME - August 10th, 2017
- It's Time to Stop Asking Whether Human Genetic Engineering Should Happen and Start Planning to Manage it Safely - HuffPost - August 10th, 2017
- Global: Engineering the Future of Our Food - STRATFOR - August 10th, 2017
- Genetic Engineering with Strict Guidelines? Ha! - Discovery Institute - August 9th, 2017
- When genetic engineering is the environmentally friendly choice - Genetic Literacy Project - August 8th, 2017
- When genetic engineering is the environmentally friendly choice - Genetic Literacy Project - August 7th, 2017
- The Call-In: Genetic Engineering - NPR - August 6th, 2017
- Experts Call on US to Start Funding Scientists to Genetically Engineer Human Embryos - Gizmodo - August 5th, 2017
- What is genetic engineering? | Facts | yourgenome.org - August 4th, 2017
- Genetic Engineering with 'Strict Guidelines?' Ha! - National Review - August 4th, 2017
- A Blueprint for Genetically Engineering a Super Coral - Smithsonian - August 4th, 2017
- Don't fear the rise of superbabies. Worry about who will own genetic engineering technology. - Chicago Tribune - August 3rd, 2017
- When genetic engineering is the environmentally friendly choice - Ensia - August 2nd, 2017
- We Need to Talk About Genetic Engineering | commentary - Commentary Magazine - August 1st, 2017
- Understanding the basics of Genetically-Modified Organisms - NIGERIAN TRIBUNE (press release) (blog) - August 1st, 2017
- Can genetic modification turn annual crops into perennials? - Genetic Literacy Project - August 1st, 2017
- Genetic engineering creates an unnaturally blue flower - Engadget - July 31st, 2017
- 'True blue' chrysanthemum flowers produced with genetic ... - Nature - Nature.com - July 31st, 2017
- When genetic engineering is the environmentally friendly choice - Ensia - July 29th, 2017
- Should genetic engineering be used as a tool for conservation? - chinadialogue - July 29th, 2017
- Scientists Give a Chrysanthemum the Blues - New York Times - July 29th, 2017
- Human Genetic Engineering Begins! | National Review - National Review - July 27th, 2017
- True Blue Chrysanthemum Flowers Produced with Genetic Engineering - Scientific American - July 27th, 2017
- Pancreas in a Dish Tells Story of How Metastatic Cells Turn Back Time - Genetic Engineering & Biotechnology News (press release) - July 27th, 2017
- 'True blue' chrysanthemum flowers produced with genetic engineering - Nature.com - July 26th, 2017
- Ghana mulling genetic engineering to combat armyworm crop damage - Genetic Literacy Project - July 26th, 2017
- Genetically Engineering Nature Will Be Way More Complicated Than We Thought - Gizmodo - July 21st, 2017
- Should Genetic Engineering Be Used as a Tool for Conservation? - Yale Environment 360 - July 21st, 2017
- DARPA funds $65 million for safer genetic engineering and technology to fight bioterrorism - Next Big Future - July 21st, 2017
- A Super-algae to Save our Seas - Laboratory Equipment - July 21st, 2017
- McCaskey grad writes new book on CRISPR and genome engineering - LancasterOnline - July 20th, 2017
- Chris R. Badman - Lincoln Journal Star - July 20th, 2017
- China unveils technology to create SUPER-HUMANS via hyper-muscular test-tube dogs - Express.co.uk - July 18th, 2017
- 32 genetic engineering incidents since 2011 revealed in regulator's ... - The Canberra Times - July 17th, 2017
- Genetically Modified Rice Stacked With Antioxidants - Asian Scientist Magazine - July 17th, 2017
- This Is Why Investors Will Need to Learn a New Acronym: CRISPR - Madison.com - July 17th, 2017
- China unveils gene technology to create SUPERHUMANS with hyper-muscular test-tube dogs - Express.co.uk - July 16th, 2017
- Boston author turns real life into Hollywood-ready stories - Boston Herald - July 16th, 2017
- I poured out my hot sauce - The Telegraph - July 14th, 2017
- Genetically engineered salmon is coming to America - The Week Magazine - July 12th, 2017
- Are Pink Pineapples Safe to Eat? - Observer - July 12th, 2017
- Can Genetic Engineering Put an End to Diamondback Moth Plague ... - Growing Produce - July 11th, 2017
- Writing the human genome - The Biological SCENE - July 10th, 2017
- Stanford's Final Exams Pose Question About the Ethics of Genetic Engineering - Futurism - July 10th, 2017
- America's First Free-Roaming Genetically Engineered Insects Are ... - Gizmodo - July 8th, 2017
- Genetically modified food is too advanced for its out-of-date regulations - The Hill (blog) - July 8th, 2017
- New Molecular Scalpel Acts as GPS to Improve Genetic Editing - Bioscience Technology - July 7th, 2017
- Stop GM mustard release as it will harm farmers: scientists to PM - Livemint - July 6th, 2017
- IARPA seeks tech to ID bioengineered life forms - FCW.com (blog) - July 5th, 2017
- 'Woolly' Breathes New Life Into A Scientific Saga - NPR - July 5th, 2017
- The race to revive woolly mammoths using ancient DNA - CBS News - July 2nd, 2017
- Scientific finding paves way for rice genetic engineering to develop efficient water storage - InterAksyon - June 30th, 2017
- Biotechnology confusion: Differences among GMOs, gene editing and genetic engineering - Genetic Literacy Project - June 29th, 2017