Some of the logistics, manufacturing and pricing details in the aftermath of Novartis gaining FDA approval of CAR-T

Wow, where to start? So much has been written about CAR-T while I was away this past week. The stories below just scratch the surface. I’m sure I’ll have more in the next few days. So, in no particular order…


Details of Novartis’ manufacturing process:

 


Kite’s manufacturing process (Kite Pharmaceuticals anticipates being next to receive FDA approval for their CAR-T therapy):

 


From Novartis, a video of its manufacturing process:

 


About that $475,000 price tag … This was guaranteed to stir controversy. Novartis’ rationale is that CAR-T can supplant an allogeneic stem cell transplant (SCT), which can cost $550 – 800K, with greater mortality risk and probably less curative potential.  Also controversial is Novartis’ decision not to refund the cost if the patient has achieved a Complete Remission (CR) in the first month, when many patients may relapse afterwards.  A 3-month or even 6-month CR would be more indicative of a potential cure.

 


Speaking of costs, the twittersphere is buzzing about this quote from Dr. Carl June at U. Penn back in 2012:

Dr. June said that producing engineered T-cells costs about $20,000 per patient — far less than the cost of a bone-marrow transplant. Scaling up the procedure should make it even less expensive, he said, but he added, “Our costs do not include any profit margin, facility depreciation costs or other clinical care costs, and other research costs.”

 


A profile of Carl June and his immunotherapy research in the Wall Street Journal:

 


Some of CAR-T’s success can be attributed to insights gained by the publicly-funded National Institutes of Health (NIH):

 


From Memorial Sloan-Kettering – from transplants to breakthroughs such as Steven Rosenberg at the NIH, etc…

 


This didn’t get quite as much press, but the FDA simultaneously “approved an expanded indication for Actemra to treat CAR T cell-induced severe or life-threatening CRS in patients ages two and older on Wednesday”. Actemra is an IL-6 inhibitor typically used to treat Rheumatoid Arthritis.  It was famously used as a last resort to reduce the CRS and thereby save the life of CAR-T’s first patient, 6-year-old Emily Whitehead, at U. Penn in 2012.  I’m now planning to ask my own oncologist, during my next visit in January, whether it was ever considered to control my CRS after my CAR-T infusion in July 2015.  (I spent five days in ICU after my infusion.)

 


I know I’ve posted about this before.  The Fred Hutchinson Cancer Center in Seattle is attempting to bypass the entire T-cell collection and manufacturing process by modifying the patient’s T cells in situ:

 


From journalist Michelle Cortez at Bloomberg News… it details some other attempts to bypass the $$ T-cell collection and manufacturing process, including equipping the hospitals and cancer centers to conduct it themselves:

 


Hmmm…

 


This Science Magazine article contains links to other interesting articles…

 


From AP News (links to other CAR-T demo videos):

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FDA has just approved the first CAR-T therapy!

Truly a momentous day in the long and winding history of this life-saving therapy.  It’s initially approved for Acute Lymphoblastic Leukemia (ALL).  Can approval for Lymphoma be far behind?

Press Announcements > FDA approval brings first gene therapy to the United States

Novartis wins a landmark FDA approval with first CAR-T cancer drug

EP Vantage – World’s first CAR-T approval sweeps away more cell therapy doubts

Support LLS, via a dear friend…

Not exactly "CAR-T news" per se, but…

A dear friend of ours, Laura Housley, was diagnosed with leukemia 17 years ago, and was saved by an allogeneic stem cell transplant (SCT).  In fact, she indirectly saved my life as well.  Almost five years ago, Laura recommended that I begin seeing the transplant specialist at the John Theurer Cancer Center of Hackensack Hospital.  It was he who in 2014 first recommended my enrollment in a CAR-T trial, over his own SCT treatment.  (I'd never even heard of CAR-T the first time he mentioned it.)  I owe Laura and him my life.

Since her recovery, Laura has gone on to do a lot of fundraising for The Leukemia & Lymphoma Society, and was nominated and named their Woman of the Year, for her role as a spokeswoman and fundraiser for the Society. She still offers support to patients about to undergo a SCT.  You can read her blog post for LLS here:

In November, Laura's daughter Sara, who is now in college, will be running for The Leukemia & Lymphoma Society's team, in the New York City Marathon.  If you're interested in donating, please see Sara's fundraising page:

(LLS is truly a worthy cause for present and future leukemia and lymphoma patients.  Donations to LLS contributed to CAR-T's development, and they have a very informative, helpful CAR-T page here.)

— Ben

SaraAndLaura_IMG_0843

LauraHousleyWomanOfTheYear

CAR-T news — June 2017

“One of the leading CAR T-cells immunotherapy company, Cellectis dosed the first blood cancer patient with the allogeneic, “off-the-shelf” CAR-T therapy, UCART123 at Weill Cornell Medicine and NewYork-Presbyterian Hospital…”

Scientists Get Closer to First CAR T-Cells Based Immunotherapy for Humans

Cellectis’ off-the-shelf CAR-T Cancer Therapy has Entered the Clinic

Cellectis’ Press Release…

First in Human Administration of UCART123 in Cellectis’ AML Phase I Clinical Trial at Weill Cornell Medicine, NewYork-Presbyterian Hospital | Cellectis


A journalist from Bloomberg News is working on a story similar to the one below:

For experimental cancer therapy, a struggle to ensure supply keeps up with demand | Science | AAAS


“The sky-high cost of producing CAR T cells, and limitations on the availability and regulatory qualification of reagents can be barriers to making CAR T cell therapy accessible…”

Biotechin.Asia- Driving CAR T-Cell Therapy From Bench to Bedside

CAR-T Value Calculation In The Firing Line | Seeking Alpha


Work continues on applying CAR-T to solid tumors:

Unleashing CAR-T Therapies on Solid Tumors: Are We There Yet? | BIOtechNow

Buy Celyad And Get In On The CAR-T Space In Solid Tumors: Medicine’s Next Frontier – Celyad (NASDAQ:CYAD) | Seeking Alpha


A history of all reported deaths (clinically described as a “Grade 5 Adverse Event”) thus far from CAR-T:

EP Vantage – Spotlight – Putting a number on CAR-T deaths


Neurotoxicity needs separate treatment from CRS in CAR T-cell therapy  | (Free registration required)


Engineering CAR-T cells


CAR-T coverage in PC Magazine(!)

How Scientists Are Hacking Cancer | News & Opinion | PCMag.com

Upcoming CAR-T coverage at Bloomberg News?

For anyone who would like to share their stories of how they were able to gain access to CAR-T therapy, or whether they are still waiting to gain access…

I received the email below a few days ago from one of the lymphoma Yahoo Groups I am a member of. I’ve since shared my CAR-T story with Michelle, but my story may be less relevant than some of yours, since I did CAR-T way back in 2015. 🙂

Please read Michelle’s email below and see if you may be interested:

Hi. I’m a journalist at Bloomberg News, and I’m writing a story about Car-T therapy for DLBCL. I am hoping to find a patient who is willing to share their story and perspective about trying to get into one of the studies or is waiting for the therapy to be approved. We and others have written about the actual treatment and how that process occurs. I want to explore the issues that come with waiting for access, as that is likely to be a bigger issue once the approach is approved. I appreciate the help!

The story will have a US focus since the approval is supposed to come this year from the FDA. I am based in Minneapolis but can call anywhere to anyone, anytime. Thanks so much for any help!

Michelle Cortez
Email: mcortez@bloomberg.net
Phone: 612-991-8887

ICML 2017 – Prof. Stephen J. Schuster from the Abramson Cancer Center, Philadelphia, PA, US, discusses interim results from the phase II JULIET trial of CTL019 in adult patients with R/R DLBCL | LymphomaHub

Dr. Schuster details the entire process using Novartis’s CTL019 CAR-T at U. Penn.

http://www.lymphomahub.com/medical-information/prof-stephen-j-schuster-interim-results-of-the-juliet-trial-of-ctl019-in-rr-dlbcl-icml-2017

As a side note, my wife and I met with Dr. Schuster and his team for over 2 hours in February 2015. I found him to be a warm, down-to-earth doctor. I almost enrolled in his trial, before my wife happened to discover online that Memorial Sloan-Kettering in NYC, where I was already a patient, had just started enrolling Follicular Lymphoma patients in their CAR-T trial. (I’ve been told I’m fNHL patient #1 to complete CAR-T at MSK.)