Advancing Cures for Cancer by Realizing the Potential of CAR T

Nearly 60 years ago, scientists discovered that immune cells could kill cancer cells in mice – forever changing the course of cancer treatment. Today, there are more than 120 companies working on developing CAR T therapies, and over 200 ongoing or completed clinical trials. While we’re at the tipping point of harnessing the curative potential of CAR T, there are critical barriers we still need to overcome before cell therapy will reach its full potential. Addressing these challenges requires a heavy dose of healthy competition, but it’s critical that we compete for the right reasons – to advance these innovative therapies and ensure access to the patients who need them most.

CAR T offers hope when other treatment options have failed, so shouldn’t we strive to ensure that every eligible patient is aware of this option?

Currently, only a small percentage of eligible patients are actually referred to receive CAR T therapy. And, by the time those patients get access, most have already exhausted all other treatment alternatives and are extremely ill, so CAR T becomes their final lifeline. It pains me to think of the individuals who don’t even know CAR T is an option, especially after hearing the stories of so many who have benefitted. Take Chuck, for example, who was diagnosed with stage 4 follicular lymphoma when his daughter was just six months old. After two rounds of chemotherapy, his cancer did not respond. Then, Chuck was fortunate enough to be referred for a CAR T clinical trial, and just recently reached his three-year cancer-free anniversary. For Chuck, and so many others, CAR T offers hope when other options have failed, so shouldn’t we strive to ensure that every eligible patient is aware of this option?

Community oncologists whose patients will be transferred to other doctors in order to undergo CAR T will sometimes fear losing touch with them and their care. To address this, we developed an online portal that allows doctors to remain connected with their patients throughout their CAR T treatment journey. Increasing awareness and improving continuity of care will help drive increased patient access to CAR T therapy.

Even one patient who loses the chance to receive a potentially curative treatment due to manufacturing complications is one too many.

Manufacturing concerns among stakeholders still persist due to the highly complex process of engineering immune cells. CAR T is a definitive personalized treatment, and there are literally hundreds of steps involved in the manufacturing process – from drawing a patient’s blood (leukapheresis) to separating out the T cells, to sending those T cells to a manufacturing facility, to engineering those T cells, to reinfusion of the final CAR T-cell therapy. We’re extremely proud of our impressive 96% manufacturing success rate and our global manufacturing network at Kite, meaning there is rarely a need to put a patient through leukapheresis a second time. However, the manufacturing complexities related to autologous treatments mean that there can sometimes be delays in delivering treatments to patients – or, for a variety of reasons, patients may not receive treatment at all – a challenge that everyone in our industry is constantly working to improve.

Even one patient who loses the chance to receive a potentially curative treatment due to manufacturing complications is one too many. This is why we must continue to focus on finding solutions for manufacturing challenges. Kite’s manufacturing turnaround time in the U.S. is 16-17 days, and we’re investing in automation and allogeneic (off-the-shelf) therapies to further alleviate manufacturing strain with the potential to significantly shorten the delivery time of the CAR T cells to patients. 

We must educate patients, doctors and payers about the difference between cumulative and upfront cost to show the true value of this transformative, one-time treatment.

CAR T is a one-time treatment with curative potential. While the high up front price of CAR T can seem overwhelming, multiple independent cost-effectiveness models have shown that cell therapy is cost-effective based on commonly cited U.S. thresholds (costs and life years gained) compared to salvage chemotherapy. Getting cell therapies that could potentially lead to durable remission to patients in earlier lines of therapy could reduce the cumulative cost of treatment by preventing the need for subsequent treatment. Take diffuse large B-cell lymphoma (DLBCL) for example, where the total lifetime direct medical cost of treatment can reach and even surpass $1 million in relapsed/refractory patients. If patients could receive CAR T earlier, it could potentially offset the need for further treatment, driving down this overall cost significantly. 

It’s equally important to make sure all eligible patients can access CAR T, regardless of insurance type. Reimbursement must not be the driver of therapy choice – patient care needs to come first. Last year, the Centers for Medicare & Medicaid Services (CMS) finalized a proposal to create a new Medicare Severity Diagnosis-Related Group (MS-DRG) for CAR T. This change will increase payment predictability for CAR T inpatient therapy, a positive first step in helping Medicare patients get access. However, continued dialogue with CMS is needed on this topic as there is still room for improvement for Medicare reimbursement for CAR T patients.

We must lead with equal parts empathy and strategy and compete with compassion.

These are just a few of the many challenges our industry is facing, and we’ve also made a lot of progress for patients of which we should be extremely proud. We must lead with equal parts empathy and strategy, and compete with compassion in order to sustain this momentum. If we don’t continue to boldly drive scientific breakthroughs forward, we’ll never reach our goal of a future with cures for cancer.