“CAR-T made it possible to cure incurable cancer”

cell and gene therapy, a new approach in the fight against cancer, is becoming increasingly widespread in the medical field. These are innovative forms of treatment that involve manipulating a patient’s own cells to combat a disease.honesty, they turn out to be more than just a promise. Having undergone rigorous clinical trials, they are now introduced into everyday medical practice. Spain recently took a major step in approving the use of treatments. AUTOMOBILE-Tone of these cell-based treatments, in some cases of blood cancer in the early stages of treatment.

The scientific weight of this cutting-edge therapy is reflected in its visibility at meetings and congresses in the sector. Last week, Valencia hosted the sixth European meeting dedicated exclusively to CAR-T, and Barcelona just hosted the seventh annual Cell and Gene Therapy Innovation Leaders Summit, an event organized by the consulting firm CGTI, which featured the relevant figures from this article . field took part.

Among them Warner Biddle, senior vice president and global chief commercial officer of Kite Pharma, a CAR-T biopharmaceutical company part of Gilead Sciences. We were able to talk with him about the current landscape of these treatments.

“We are going through a very interesting time right now; It’s amazing to see how far we’ve come in the last eight years since the first of these treatments became available,” explains Biddle. When the first therapy was launched in 2017, there were two big doubts: whether they could scale the treatment and go from treating hundreds of patients to thousands, and whether they would have a real impact on patients.

“Today the answer to both questions is yes. To date, we have already treated more than 19,500 patients with Kite Pharma therapy. “And if we look at the data in some cancers, we are showing breakthrough results for patients in terms of long-term survival that are significantly better than the old standard of care used in these cancers.”

An example of the importance it acquires is the Amsterdam plant. The 19,000 m² facilities are capable of providing CAR-T cell therapy to more than 235 eligible centers located in 20 countries in Asia and Europe, including Spain. The plant is capable of treating up to 4,000 patients per year.

New indications for CAR-T

Without a doubt, one of the current news in Spain is that two new indications for CAR-T therapy have recently been approved for use in the public health system, promoting their use. at earlier stages. This is one of the workhorses of the biopharmaceutical industry, which believes it is necessary to continue to advance along this path and ensure that at some point CAR-T becomes the first line of cancer treatment.

“If you look at the second line data from our Zuma-7 study, you will see significant reduction in risk of death and improved overall survival compared with stem cell transplantation, which has been around for 30 years. “This is the first time the company has been able to demonstrate this in a clinical trial setting,” says Warner Biddle, adding that the data is encouraging and gives them confidence that they will one day bring their CAR-T therapy to the forefront. line. “If you treat patients even earlier, you can get better results”.

This statement is based primarily on two ideas. The first is that as the use of CAR-T evolves, patients find themselves in health condition has not deteriorated so much allowing them to better benefit from treatment. Second, and no less important, is the ability to use T cells that are not damaged. “These cells develop during cancer treatment. “Many researchers believe that being able to access them when they have not been exposed to different types of treatments may actually improve CAR-T outcomes.”

To demonstrate this potential benefit, Kite Pharma launched new test, which is partly based on results obtained in previous studies. They had already demonstrated that response rates were better and that improved survival was achieved with early use of CAR-T. “But we don’t just want to conduct research to advance new treatments. “We also want to apply CAR-T therapy to other indications, such as solid tumors, and perhaps even in contexts other than cancer.”

Improved forecast

Without a doubt, one of the greatest successes of CAR-T therapy is that diseases with a very negative prognosis, even those considered incurable, have now radically changed their prognosis. “This is the fundamental point: the transformation that these treatments have made for the patients who are the main beneficiaries of this research,” adds Biddle. And this is hinted at by the case of a patient from Asturias, one of the first to receive CAR-T therapy. She would not be alive if she had not been given this medicine.

“For this reason, we are also investing in expanding our network of authorized treatment centers so that we can bring these treatments even closer to patients.” The vice president of Kite Pharma recalls that in SpainIn a very short time it has grown from nine authorized centers to the current 25.

Despite this, Warner Biddle also admits there is still a long way to go. “For example, we now have data that shows 45% overall survival for our CAR-T compared to the traditional standard of care. What about the other 57%? This is what motivates us to continue the investigation.” Biddle emphasizes that advancing treatment lines is a possible way, but it is not the only answer, and adds that work will need to be done to develop a new generation of CAR-Ts that will improve both outcomes while also reducing the side effect profile.

The challenge of reaching solid tumors

Although CAR-T therapy has been able to demonstrate its clinical benefit in the treatment of hematological cancers, without a doubt the big challenge for the future is the ability to do so in solid tumors, where they have faced major challenges so far. Biddle, while acknowledging the problem, is optimistic about it.

“We found that in solid tumors the tumor microenvironment is more complex and finding a suitable target is difficult, partly also because of its great heterogeneity. “But there are many companies around the world, including Kite Pharma, that are investing and looking at ways to bring this promise of CAR-T therapy to solid tumors, despite all this complexity.”

Biddle notes that in any case, it will take several more years to see the results of these treatments in clinical practice. “But we won’t give up.” Some avenues being explored to achieve this goal are the use of CAR-T therapies that target multiple targets or mechanisms that can influence these treatments to ensure they work more efficiently and effectively.

“There are many things that need to be studied, and it is likely that initial CAR-T therapy in solid tumors will focus on a small subsegment or high-risk tumor profiles. But hopefully then we can learn from this and expand it to reach more patients.”

Another major problem facing CAR-T therapy is its high price. Because these are personalized treatments, their development is currently costly and raises questions about how they might impact the sustainability of healthcare systems, although Biddle is clear that the impact these treatments have on survival also needs to be considered. “It is very important to understand that these treatments are one-time treatments and can actually lead to long-term overall survival. Some experts even believe that they have healing potential in some situations. So yes, the costs can be high, but the fact that you can achieve these results and do it with a one-time therapy is truly incredible.”

The Vice President of Kite Pharma also emphasizes how diverse economic assessments in countries such as the UK or Spain, where these treatments have been shown to be truly cost-effective. “This is often overlooked because of the initial price. But in many cases, if you don’t treat CAR-T patients, you may be treating them for years with various chemotherapy, biologic treatments, or targeted therapy, which also cost a lot of money, in addition to taking those patients from their family for a long time.”

Despite this, the industry is also working on various ways to try to reduce the costs involved. One way of research is to use allogeneic therapy, that is, from universal donors, and not from autologous ones or from the patient himself. In this way, cells would always be available to patients who need them, and the entire production process for each cell product would remain, as it is now, patient-specific. Although there are currently doubts about the potential effectiveness on this issue.

“We do not believe that allogeneic therapy currently has such a significant benefit in terms of efficacy or overall survival, which makes experts not very enthusiastic about the results of these first-generation treatments. But they may offer better results in the future, and at Kite Pharma we are also investing in our own allogeneic program to improve these results and make it easier for patients to access these treatments.

Another avenue also being explored to improve access to more patients is investing in manufacturing capacity to “speed up response times and make these treatments more accessible,” explains Warner Biddle.

“We know that reducing the time it takes to deliver CAR-T cell therapy will improve patient outcomes. But it’s also an interesting path because reducing production time will help us reduce system costs. “So we’re trying to do everything we can to make the treatment as cost-effective as possible.”