Historic FDA Approval Makes First Gene Therapy Available in US

Novartis was the first to receive FDA approval for a CAR-T drug to treat cancer

In this type of therapy, patients' own immune cells are modified ex vivo to include a gene for the vehicle protein, which directs them to attack leukemia cells carrying the CD19 antigen. Then the genetically-modified cells are sent back to be re-inserted in the patient. Firms developing CAR-T therapies say their expense is justified because they replace management of otherwise chronic or fatal conditions. Kymriah will carry a boxed warning for cytokine release syndrome, a potentially lethal systemic response to the activation and proliferation of CAR-T cells, causing high fever and potential for neurological problems. It is the first gene therapy to be allowed in the United States - a "historic" approval, the agency said.

Grupp is Emily Whitehead's doctor - she was the first patient to receive the cell therapy, and though she was as close to 48 hours from organ failure when first enrolled in the experimental treatment, Whitehead has now been cancer-free for five years.

In a key study of 63 advanced patients, 83% went into remission, although it is not clear how long the benefit lasts: Some patients did relapse months later.

What's perhaps most exciting about CAR-T is that it operates similarly to a vaccination, providing the patient with lifetime protection against the targeted cancer.

Dr. Kenneth Anderson is president of the American Society of Hematology, which focuses on blood cancers.

Among concerns, sometimes leukemia can develop resistance, and sometimes patients worsen while waiting for their new cells, said Sekeres, who directs the Cleveland Clinic's leukemia program and wasn't involved with CAR-T testing. The others still are being tracked to see how they fare long-term.

Rescues continue in Houston; storm called 'disaster of historic proportions'
Total rainfall could reach 50 inches as rain continues through Wednesday. "Texas has never seen an event like this". Rainfall amounts of 5 to 10 inches are expected in southeast Louisiana and coastal MS and Alabama.

My Favorite Jud Heathcote Quote
And over the course of the time in some of the champion games I was able to attend and tournaments and that type of thing. North Dakota coaching legend Jud Heathcote has passed away at the age of 90.

Best Buy Earnings Exceed Expectations
Excluding one-time charges, the company earned 69 cents per share , compared with a forecast profit of 63 cents per share. Meag Munich Ergo Kapitalanlagegesellschaft Mbh stated it has 1.01% of its portfolio in Best Buy Co Inc (NYSE: BBY ).

"Unfortunately leukemia grows so rapidly that it can evade even the smartest of our technologies", he added.

Novartis did not immediately disclose the therapy's price, but it is expected to cost hundreds of thousands of dollars.

The drug Kymriah was developed by Novartis Pharmaceuticals and the University of Pennsylvania. Most of these symptoms should appear within three weeks of infusion with Kymriah, the FDA said.

"There has been an urgent need for novel treatment options that improve outcomes for patients with relapsed or refractory B-cell precursor ALL", Novartis, the drug company that makes Kymriah, said in a statement. But the customized treatment is also being tested for a range of diseases from non-Hodgkin lymphoma and multiple myeloma to solid tumors, and its approval signals a new chapter in treating cancer by mobilizing the body's own immune system.

Other genetic therapies for cancer are also in the research pipeline.

Related News:

  • Amazon Alexa and Microsoft Cortana team up to take on Google

    Amazon Alexa and Microsoft Cortana team up to take on Google

    For Echo and Android owners, the cooperation between Alexa and Cortana will not immediately result in a very practical solution. It's likely both Bezos and Nadella had Apple and Google at the top of their minds when they agreed to the partnership.
    President Trump visits Springfield, Mo. to speak about tax reform

    President Trump visits Springfield, Mo. to speak about tax reform

    President Donald Trump's fall push for the Republican tax reform effort kicks off on Wednesday in Springfield, Missouri. The president will speak at a manufacturing company in Springfield, but is not expected to reveal key policy details.
    Mattis 'Freezes' Trump's Transgender Military Ban

    Mattis 'Freezes' Trump's Transgender Military Ban

    Tammy Duckworth, a military veteran who lost both her legs during combat, also criticized Trump's ban on transgender troops. Mattis had requested six more months to study the issue when Trump tweeted about barring transgender service members.
  • U.S.  strikes ISIS militants heading toward evacuees

    U.S. strikes ISIS militants heading toward evacuees

    State news agency SANA said 10 people were killed and 28 wounded when the vehicles they were traveling in drove over mines. The coalition nevertheless said in a statement that it is not bound by the evacuation agreement.
    Mumbai Rains updates: Bad Weather affecting millions might end today

    Mumbai Rains updates: Bad Weather affecting millions might end today

    Five more teams of NDRF are enrout to Mumbai as a precautionary measure in addition to Five teams already deployed there. There are fewer people on Mumbai's roads this morning with schools and colleges closed and many staying home.
    Johanna Konta crashes out of the US Open in first round

    Johanna Konta crashes out of the US Open in first round

    Nevertheless, she had gone into this match having not beaten a top 10 opponent since the 2014 US Open . Asked how she would deal with the defeat, Johanna said: "I think not to catastrophise is important".
  • Taylor Swift: Look what you made me read

    Taylor Swift: Look what you made me read

    She reportedly felt like that the spotlight was stolen from her by Taylor Swift who allegedly meant to hurt her nemesis with the premiere.
    FDA Reveals Stem Cell Policy Plans Amid Clinic Crackdowns

    FDA Reveals Stem Cell Policy Plans Amid Clinic Crackdowns

    They can divide and then morph into other kinds of cells - red blood cells, muscle cells, whatever our bodies need. The article was written by doctors unconnected with the clinic who treated the women for the disastrous results.
    Bawana bypoll: Its candidate 3rd, Cong finds solace in vote

    Bawana bypoll: Its candidate 3rd, Cong finds solace in vote

    On the other side BJP who was struggling to be the second choice finally managed to finish as runner-up in the Bawana By election. The shift in favour of the Congress no doubt came from the "minorities" vote that had backed Parrikar so convincingly in 2012.
  • Human West Nile Cases In Simcoe Muskoka

    Human West Nile Cases In Simcoe Muskoka

    Lynch said there have been cases of infected mosquitos on the west, east and south sides of town. Health officials say the infection rate is moderate this year.
    Facebook Debuts Trailer for its Ball Family Reality Show

    Facebook Debuts Trailer for its Ball Family Reality Show

    The show, produced by MTV reality show vets Bunim/Murray Productions, certainly looks the part of the Real World style format. After the debut episodes, the show will air regularly on Sundays starting September 10.
    EU's Brexit negotiator tells United Kingdom  to speed up and 'get serious'

    EU's Brexit negotiator tells United Kingdom to speed up and 'get serious'

    British public are overwhelmingly opposed to the government agreeing a large Brexit divorce bill with the EU . The UK wants trade talks to take place at the same time as discussions on separating the UK from the bloc.


Most liked

Hasbro: Calling All Star Wars Fans
Having Luke Skywalker wear black feels rather ominous, though that's all he wore for the entirety of Return of the Jedi as well. In a new photo from the upcoming flick, we see a contemplative Rey prepare for some serious (and solitary) Jedi training.

Master P criticizes Kevin Hart's Hurricane Harvey challenge
J-Lo and her boyfriend, A-Rod donated $25,000 each to the Red Cross , and offered inspiring words to get others to help out, too. Around 13 million people from Corpus Christi to New Orleans are under flood watches and warnings as of Monday morning.

Bangladesh on top in 1st test, Australia struggling at 123
David Warner was the only batsman in the Australian lineup who stood between Bangladesh and their maiden Test win over Australia. Josh Hazlewood was caught leg before by Taijul Islam to seal the contest in front of Bangladesh Prime Minister Sheikh Hasina.

Changes in Signals Identified: Chico's FAS, Inc. (CHS), Bill Barrett Corporation (BBG)
Keating Invest Counselors holds 0.75% of its portfolio in Altria Group Inc (NYSE:MO) for 23,390 shares. Since March 2, 2017, it had 1 insider purchase, and 4 selling transactions for $1.14 million activity.

BRC warns of food shortages if no Brexit customs deal agreed
The deflation rate for prices of non-food products specifically was 1.3 per cent, the slowest rate of deflation since April 2013. In contrast, the inflation rate of Ambient Food prices accelerated to 1.9 per cent in August from 1.6 per cent in July.

Don't panic, Hurricane Harvey could send gas prices up
The national average price for a gallon of gasoline measured across 150,000 service stations is $2.38, DeHaan said. Mississippians won't see skyrocketing gas prices because of Tropical Storm Harvey according to an analyst .

Gas prices could spike amidst Hurricane Harvey devastation
Hurricane - now Tropical Storm - Harvey has caused almost 100 oil and gas production platforms in the Gulf of Mexico to temporarily shut down.

Trump Declares Emergency for Louisiana as State Braces for Hurricane Harvey
The number could grow as the slow-moving storm remained a threat after spending days over Houston. Louisiana lawmakers, including Gov.

P Chidambaram slams RBI over note ban
A collateral damage as a result of rise in printing and other cost was dividend RBI pays to the government. According to the RBI , 2,56,324 Rs 1,000 notes valued at Rs 25.63 crore were detected during FY17.

WWE 'SmackDown' Rumors: AJ Styles To Boost Shelton Benjamin's Return To WWE?
Kingston kicked Jimmy then missed a splash and Jey went for a samoan drop but Kingston reversed it with a dragon sleeper. Out came SmackDown commissioner Shane McMahon to counter Owens' claims and ensure English's match went on as planned.

Texans-Cowboys preseason game canceled so players can be with families
So I think it's extremely important that we get home as quickly as we possibly can. "Now we just have to adjust". But it was a shout of joy when we announced we were going home. "We have people back home to worry about".

Orton plants Nakamura with RKO
Owens decides to enter the ring and take the shirt off of the referee to put on himself, showing everybody "how it's done". However, Styles was able to make a strong comeback and defeat Dillinger with the Calf Crusher for the submission victory.

The Markston International LLC Decreases Stake in Anadarko Petroleum Corporation (APC)
It improved, as 40 investors sold WAB shares while 153 reduced holdings. 207 funds opened positions while 479 raised stakes. The Company's product OMONTYS is for the treatment of anemia due to chronic kidney disease in adult patients on dialysis.

Eminem goes off on Donald Trump at United Kingdom concerts: 'F
Eminem also appeared on rapper Big Sean's " No Favors " and took Trump to task over racism in America and police brutality. Eminem also wore a shirt that read " Fack Trump " during a show in Glasgow, Scotland on August 24.

Google Maps Will Now Help You Find Parking In Some Cities
Paul, New York City, Orlando, Philadelphia, Phoenix, Pittsburgh, Portland, San Diego, Sacramento, San Francisco, Seattle, St. These difficulty icons will allow users to see how hard it might be to park in certain places.