FDA approves breakthrough treatment for dire cancers

Getty Images

Image credit: Mskcc.org " This is a brand new way of treating cancer", Dr. Stephen Grupp of Children's Hospital of Philadelphia, who treated the first kid with CAR-T cell therapy, told ABC News.

The treatment, called Kymriah, was approved for patients up to 25 years of age who have relapsed or not helped by prior treatment for B-cell acute lymphoblastic leukemia (ALL).

"We're entering a new frontier in medical innovation with the ability to reprogram a patient's own cells to attack a deadly cancer", FDA Commissioner Scott Gottlieb said in a news release following the approval.

To better ensure patient safety, the FDA is requiring Novartis to offer CAR-T therapy only through medical centers specially trained and certified to handle the complicated treatment.

The treatment is not a pill or a form of chemotherapy.

In 2012, Novartis and Penn launched their global collaboration to further research, develop, and commercialize vehicle T-cell therapies, including Kymriah, for the investigational treatment of cancers. It progresses quickly, and is the most common childhood cancer in the United States, affecting an estimated 3,100 patients aged 20 years and younger annually.

Novartis Pharmaceuticals announced the price for its one-time infusion would be $475,000, but noted there would be no charge for patients who didn't show a response within a month of receiving the treatment.

Narendra Modi launches Sports Portal on National Sports Day
Born in Uttar Pradesh, Chand earned three Olympic gold medals in 1928, 1932 and 1936 in field hockey during India's most dominant phase in the sport.

Kymriah treatment also has the possibility of serious side effects, including cytokine release syndrome (CRS), which consists of high fever and flu-like symptoms and can be life-threatening, and neurological events, which are also life-threatening.

Eleven patients who had CR or CRi relapsed after tisagenlecleucel prior to data cutoff and before any new cancer therapy.

The FDA is including a boxed warning for CRS and, in an unusual move, also approved a new indication for the rheumatologic drug tocilizumab (Actemra) as a treatment for CRS.

But, Novartis is supposedly working with Medicare-Medicaid on a unique plan where reimbursement would only be made if patients responded to treatment within one month. Firms developing CAR-T therapies say their expense is justified because they replace management of otherwise chronic or fatal conditions. But already scientists are studying whether vehicle T cells can be effective in other blood cancers and lymphomas, as well as in solid cancers like breast, prostate, ovarian and lung cancers.

The company did say that it's made an agreement with the USA government to pay for the drug only when pediatric or young adult patients with the cancer respond to treatment by the end of their first month. Also called Kymriah, the treatment isn't actually a drug in the traditional sense, but a collection of the patient's own cells that have been removed from the body and genetically engineered to recognize and destroy cancer cells circulating in the blood.

Manufacturing and delivery are more complex in CAR-T therapies than for a typical drug.

Novartis representatives said they calculated a "cost-effective price" for the therapy that fell between $600,000 and $750,000.

Related News:

  • US Open Tennis 2017 Schedule

    US Open Tennis 2017 Schedule

    French eighth seed Jo-Wilfried Tsonga made light work of Marius Copil 6-3, 6-3, 6-4. He also became the oldest male player to win Wimbledon in the Open era.
    Veteran Houston Police Officer Drowns While Responding to Harvey

    Veteran Houston Police Officer Drowns While Responding to Harvey

    During the conference, Acevedo emotionally reflected on Perez, stating, "Steve is one of the sweetest persons I've ever met". Perez lost his life because he tried to make it happen, he tried to get at his post.that's the ultimate sacrifice", he said.
    Rauner Axes Illinois Attorney General's Student Loan Plan

    Rauner Axes Illinois Attorney General's Student Loan Plan

    Religious leaders, including Cardinal Blase Cupich of the Archdiocese of Chicago, have lobbied for the credit. The CTU has been a major critic of the plan that will earmark up to $75 million for scholarship tax credits.
  • US Open player profile: Simona Halep

    US Open player profile: Simona Halep

    They will have another chance to cheer the comeback Wednesday, when Sharapova faces Timea Babos of Hungary in the second round . After leading by a set and 4-1 in the second, Sharapova showed some fatigue and rust, dropping five games in a row.
    'Get out now!!' Evacuations ordered as Houston flood worsens

    'Get out now!!' Evacuations ordered as Houston flood worsens

    He said the county is trying to determine where the water will go, specifically from the north end of the Addicks reservoir. You've heard a lot about Harvey's impact on Houston, but the storm has also been devastating in other areas.
    Yankees To Give Slumping Aaron Judge 'Couple Days' Off

    Yankees To Give Slumping Aaron Judge 'Couple Days' Off

    While Judge does have 37 home runs and 82 RBIs this season, most of that damage occurred from mid-April to the beginning of July. Judge said it is not, but Girardi pointed out, "It's my job to watch him and to do what I think is best".
  • BSE soars 258 points; Reliance, Adani, HDFC top gainers

    BSE soars 258 points; Reliance, Adani, HDFC top gainers

    Reliance Industries , Ultratech Cement , Ambuja Cements, HDFC and Yes Bank were the other major gainers in the index. However, the Nifty50 could not retain that level and closed at 9,884.40 points - up 88.35 points or 0.90 per cent.
    Amazon Echo Unavailable, Rumors Of New Version To Rival Apple HomePod

    Amazon Echo Unavailable, Rumors Of New Version To Rival Apple HomePod

    Amazon Music Unlimited (for students) is available in the USA starting today for the low-low price of $4.99 per month. Currently, people can only use it with music-streaming services Amazon Music , TuneIn, iHeartRadio, and Pandora.
    Gold rush seals SEA Games medals victory for Malaysia

    Gold rush seals SEA Games medals victory for Malaysia

    Indonesia won its second and last gold medal in the sport in the 1991 Games after beating host Philippines in a penalty shootout. A chant of " Singapore dogs " in the match against their neighbours was also condemned by SEA Games organisers.
  • Transgender service members file lawsuit against Trump for military ban

    Before Mattis spoke, Trump had tweeted: "The U.S. has been talking to North Korea, and paying them extortion money, for 25 years". The department will carry out the president's policy direction, in consultation with the Department of Homeland Security.
    Cavaliers, Celtics deal possible even without significant asset added

    Cavaliers, Celtics deal possible even without significant asset added

    Thomas initially injured the hip in mid-March, and he reaggravated the ailment against the Wizards in Game 6 of the East semis. While Brown or Tatum are unlikely to be inserted, there is a chance the Celtics buckle and throw in additional draft pick (s).
    Pliskova wins at soggy US Open as Nadal, Federer wait

    Pliskova wins at soggy US Open as Nadal, Federer wait

    Guaranteed to face unseeded players in her next two matches, Osaka's draw is not imposing. Not since Svetlana Kuznetsova lost in the first round in 2005 had the defending U.S.


Most liked

EU's Barnier tells United Kingdom again to clarify Brexit positions
He called Mrs May's approach to Brexit "unsatisfactory", and criticised her refusal to put a figure on how much she is willing to hand over.

Centre extends Aadhaar deadline to 31 Dec as SC defers hearing PILs
If the details do not match, it causes a problem and the user is advised to get the details amended in his/her Aadhaar or PAN . Next thing to do is, to enter your 12 digit Aadhaar card number, then verify captcha and then click on " Link Aadhaar " button.

Cummins to build all-electric HGV engines from 2019
It can haul a 22-ton trailer, and is meant for urban delivery or for transferring cargo to, from, and around ports and terminals. If the charging power is generated from 100 percent renewable sources , then the truck can be described as fully carbon neutral.

Target markets Taylor Swift's new album with print glossies
Viewers enjoyed the video , with one writing on Twitter that "no on can roast Taylor Swift better than Taylor Swift herself". The scene in Taylor Swift's new video where she raids the bank vault of a music streaming company is coming true.

YouTube rolls out its new look with a brand new icon
And in the coming months, users will be able to swipe directly to their next video, Tinder-style. These new changes to YouTube comes a year after its mobile app received an interface makeover.

Liverpool prepare £60m bid for Monaco midfielder
Liverpool's offer has dwarfed the proposals their rivals have submitted for the accomplished dribbler, who also poses a threat from set-pieces.

Vance McDonald traded to the Steelers
The health setback limited him to only 11 games, though he recorded 24 receptions for career-bests 391 yards and four touchdowns. It's reasonable to expect the guys who are here will respond positively in the right way to his presence with their play.

Merkel demands release of Germans unduly imprisoned in Turkey
In the television interview, he accused Ms Merkel's chancellery of blocking collective lawsuits against the auto industry. She said: "We're a democracy and everyone can freely express themselves in public the way they want".

Islamabad refutes USA claim, says why would Taliban hide in Pakistan
He assured America's continued support for the Afghan government and military in fighting the Taliban in the field. In context to Trump's South Asia policy, Asif does not foresee a military role for India in Afghanistan .

Israeli lawmakers visit Al-Aqsa Mosque compound after two-year ban
It is the first time Guterres has visited the West Bank and the Gaza Strip since he took over from Ban Ki-moon on January 1.

Ford self-driving cars to deliver Domino's pizzas in US
Whether the sci-fi-esque food future the company is building now will leave a similar bitter aftertaste, only time will tell. We need to understand if a customer's experience is different if the auto is parked in the driveway versus next to the curb.

World 'blind and deaf' to Rohingya muslims plight, says Erdogan
Human Rights Watch says satellite data show fires have razed 100km of land in Rakhine state in the wake of crackdown . Some 3,000 people have attempted to flee to Bangladesh but Myanmar has tightened its borders.

New Mexico shooting incident leaves two dead, injures four
A shooting inside a public library that killed two people and wounded four has deeply shaken an eastern New Mexico community. He said the two who died were both female, but he declined to release their names until authorities could notify families.

Arsenal fans unimpressed at last-ditch attempt to sign Man City transfer target
However, they would be willing to negotiate if winger Raheem Sterling is included in any deal, as well as if City offered them a huge sum of money.

Elon Musk teases Tesla Model S in his tunnel under LA
I say another, because in July Elon Musk tweeted that he has a green light to build a hyperloop between NY and Washington D.C. And unlike some forms of transportation, hyperloop pods need to be kept straight if they are going to avoid derailment.