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To Your Health: NLM update Transcript

First gene therapy cancer treatment: 09/11/2017

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Greetings from the National Library of Medicine and MedlinePlus.gov

Regards to all our listeners!

I'm Rob Logan, Ph.D., senior staff, U.S. National Library of Medicine (NLM).

Here is what's new this week in To Your Health, a consumer health oriented podcast from NLM, that helps you use MedlinePlus to follow up on weekly topics.

The first gene therapy treatment for cancer recently was approved by the U.S. Food and Drug Administration after about 80 percent of the 63 recipients (young people with an aggressive form of leukemia) went into post-treatment remission.

The gene therapy treatment works by changing the genes in a young person (with B-cell acute lymphoblastic leukemia's under-functioning white blood cells) so they can better detect and negate cancer cells. Once transformed in a laboratory procedure, the newly therapeutic cells are re-injected as part of an overall treatment process, which could be a glimpse of future cancer care.

All the young persons who received the treatment had resisted standard care or their leukemia had returned.

In contrast with traditional medications, the treatment process (in this case) is sometimes referred to as using 'living drugs,' which genetically modify a patient's own cells (that have been removed from the body) to recognize and destroy cancer cells circulating in the blood.

In a press teleconference, the FDA called its approval 'historic' and noted other gene therapy treatments soon will be considered for clinical use.

Following the approval, Scott Gottlieb M.D., the FDA's commissioner told journalists (and we quote): 'We're entering a new frontier in medical innovation with the ability to reprogram a patient's own cells to attack a deadly cancer.... New technologies such as gene and cell therapies hold out the potential to transform medicine and create an inflection point in our ability to treat and even cure many intractable illnesses' (end of quote)

Interestingly, some of the initial coverage of the FDA's announcement of the therapy (which is manufactured by Novartis) did not mention the estimated costs of a new generation of custom-made 'living drugs.'

However, the New York Times and a few other news organizations reported the newly approved leukemia treatment may cost about $475,000. Some news stories published after the FDA's announcement suggested other future gene therapy and personalized gene therapy treatments may be even more expensive.

To provide some perspective about gene therapy's challenges, Francis Collins, M.D., NIH's director wrote in a recent blog (and we quote): 'many questions ... must be addressed before we herald immunotherapeutic approaches to cancer an unqualified success,' namely severe reactions to the therapy and also its very high price tag....' (end of quote).

Still, the FDA's approval of a living drug and gene therapy marks the beginning of a new era of personalized treatment in cancer care.

Meanwhile, some background information about gene therapy is provided by the Mayo Foundation for Medical Education and Research within the 'start here' section of MedlinePlus.gov's genes and gene therapy health topic page.

The American Society of Gene & Cell Therapy provides a website about genes and gene therapy for diverse diseases and genetic conditions within the 'specifics' section of MedlinePlus.gov's genes and gene therapy health topic page. The Nemours Foundation provides a website about gene therapy for children within the 'children' section of MedlinePlus.gov's genes and gene therapy health topic page.

MedlinePlus.gov's genes and gene therapy health topic page additionally provides links to the latest pertinent journal research articles, which are available in the 'journal articles' section. Clinical trials that may be occurring in your area can be found in the 'clinical trials' section. You can sign up to receive updates about gene therapy as they become available on MedlinePlus.gov.

To find MedlinePlus.gov's genes and gene therapy health topic page, please type 'gene therapy' in the search box on MedlinePlus.gov's home page, then, click on 'genes and gene therapy (National Library of Medicine).' MedlinePlus.gov also contains health topic pages devoted to immunotherapy and childhood leukemia.

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A disclaimer — the information presented in this program should not replace the medical advice of your physician. You should not use this information to diagnose or treat any disease without first consulting with your physician or other health care provider.

It was nice to be with you. Please join us here next week and here's to your health!