FDA Panel Recommends Approval for Gene-Altering Leukemia Treatment
Source: NY Times
A Food and Drug Administration panel opened a new era in medicine on Wednesday, unanimously recommending that the agency approve the first-ever treatment that genetically alters a patients own cells to fight cancer, transforming them into what scientists call a living drug that powerfully bolsters the immune system to shut down the disease.
If the F.D.A. accepts the recommendation, which is likely, the treatment will be the first gene therapy ever to reach the market. Others are expected: Researchers and drug companies have been engaged in intense competition for decades to reach this milestone. Novartis is now poised to be the first. Its treatment is for a type of leukemia, and it is working on similar types of treatments in hundreds of patients for another forms of the disease, as well as multiple myeloma and an aggressive brain tumor.
To use the technique, a separate treatment must be created for each patient their cells removed at an approved medical center, frozen, shipped to a Novartis plant for thawing and processing, frozen again and shipped back to the treatment center.
A single dose of the resulting product has brought long remissions, and possibly cures, to scores of patients in studies who were facing death because every other treatment had failed. The panel recommended approving the treatment for B-cell acute lymphoblastic leukemia that has resisted treatment, or relapsed, in children and young adults aged 3 to 25.
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.
Read more: https://www.nytimes.com/2017/07/12/health/fda-novartis-leukemia-gene-medicine.html?hp&action=click&pgtype=Homepage&clickSource=story-heading&module=first-column-region®ion=top-news&WT.nav=top-news
broadcaster90210
(333 posts)nt
Major Nikon
(36,827 posts)We are rapidly approaching 3rd world status where only a small minority will have access to modern medicine while the masses will only have eastern medicine style snake oil remedies. Best to line up your preferred witch doctor or faith healer now and avoid the rush.
broadcaster90210
(333 posts)nt
customerserviceguy
(25,183 posts)there may be ways to use economies of scale to make it more accessible. And it may well wind up on the list of things that "medical tourism" will provide.
WhoWoodaKnew
(847 posts)iluvtennis
(19,863 posts)raging moderate
(4,306 posts)So much for Sarah Palin and her cohorts.
Kudos to you, brave researchers and brave doctors. Especially you warriors against cancer.