It's a Breakthrough Cancer Drug-And It'll Cost You

"This is the first-ever true gene therapy treatment made available to the USA population and will help accelerate the speed at which we will see even more gene-based therapies come to fruition".

The US Food and Drug Administration (FDA) has just approved a pioneering cancer treatment that reprogrammes the patient's own white blood cells to hunt down cancer. The FDA itself called the approval a "historic action".

"We've never seen anything like this before and I believe this therapy may become the new standard of care for this patient population", said Dr. Stephan Grupp, director of cancer immunotherapy at Children's Hospital of Philadelphia, which spearheaded this research.

"While both external and Novartis' quantitative assessments of these values indicate that a cost-effective price could be $600,000 to $750,000, we recognize the importance of this paradigm-shifting therapy and are setting the price at $475,000 for this one-time treatment", Dana Cooper, a spokesman for Novartis, said in an interview with OncLive.

An advocacy group called Patients for Affordable Drugs recently met with officials at Novartis to ask it to set a "fair" price for the drug, whose early development was supported by $200 million in federal research grants.


Novartis plans additional filings for Kymriah in the United States and EU later this year, including applications with the FDA and European Medicines Agency (EMA), for the treatment of adult patients with r/r diffuse large B-cell lymphoma (DLBCL). Switzerland-based Novartis, which makes the innovative therapy, announced that the drug will cost nothing if patients fail to benefit in the first month.

Treatment with Kymriah has the potential to cause severe side effects. A multicenter clinical trial of 63 pediatric and young adult patients with relapsed or refractory B-cell precursor ALL found that the overall remission rate within 3 months of treatment was 83%.

The treatment, called CAR-T, is made by the drug company Novartis.

The treatment process, known as vehicle T-cell therapy, will be used to fight the recurrence of the B-cell where cancer has not responded to treatment or returned after initial treatment, something that occurs in approximately 15-20% of patients.

It is not without risks, however, as CAR-T therapies can cause serious side effects such as cytokine release syndrome which can be life-threatening.


"Patients who get this therapy basically have no chance to survive", he said.

"The efforts involved in being able to provide this novel therapy to patients is unlike any other therapy now offered", says Colleen Dansereau, MSN, RN and CPN, who is director of clinical research nursing and gene therapy program manager at Dana-Farber/Boston Children's.

For one, the treatments would have to treat more types of cancer than the one Kymriah was approved for on Wednesday. Instead of getting a better, updated therapy for a disease every decade or so, we might begin to see second-generation cell therapies in a few years.

Shares of Bluebird Bio Inc, which is developing a promising CAR-T treatment, were up 11.1 percent at $113.73.

With the treatments available now, fewer than 10 percent of patients with the relapsed, hard-to-treat leukemia are alive five years after diagnosis.



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