This year will be considered a turning point for the treatment of glioblastoma, a rare and competitive form of brain cancer that affects an average survival time of about 15 to 18 months, said Carl June, a professor at the University of Pennsylvania’s Perelman School. of Medicine and renowned immunologist.
“It’s essentially a death sentence,” says June, who led a team that pioneered the first CAR-T mobile cure to treat some blood cancers. Pediatricians are “essentially saying, ‘Go home, we can’t do anything. ‘” But recent studies, including one conducted in June, have shown promise in the CAR T-mobile infusion box for treating glioblastoma.
Few people on the planet have replaced the global as profoundly as Carl June. I recently told you about new advances in mobile CAR-T therapy, which works through some of the patient’s immune cells, modifying them in the laboratory to recognize and attack their cancer and injecting the cells back into the frame to perform the task. . made.
June treated the first adult patients with experimental CAR T mobile therapy at UPenn in 2010, and the first pediatric patient in 2012, with amazing results. Five years later, in 2017, the FDA approved T-mobile’s first treatment drug. There are currently six of these drugs approved on the market and approximately 34,000 patients with blood cancers have been treated.
“Dr. June’s cutting-edge studies and technological contributions have shown that CAR T cells can induce remission and even cure complex cancers, particularly the quality of human life and pave the way for a more equitable and sustainable world for generations in the long term,” said Bruce Levine, a fellow UPenn immunologist who has worked intensively with him for decades. “June’s innovative paintings have not only revolutionized the care of patients with hematologic malignancies, but have also stimulated the emergence of a new industry in the field of CAR T cells. therapy. “
Here are five desirable concepts that June shared with me in our discussion:
Treatment features for patients with glioblastoma are limited and no significant innovations have been seen in decades. But a recent trial conducted through a Stanford research organization showed that repeated infusions of CAR T cells directly into the brains of pediatric patients had benefits. Three other study organizations, they added, have since demonstrated the benefits of direct infusions of CAR-T cells into the brains of adult glioblastoma patients, whose tumors are more confounding than those of children.
“We published six patients about a month and a half ago in an ongoing trial with a dual CAR delivered into the brain,” June said, referring to a T cell treatment that targets two primary antigens expressed through glioblastoma. , to triumph over evasion mechanisms. “And all six patients showed noticeable image adjustments very quickly. ” And she added: “I think in five years we will have FDA-approved CARs for glioblastoma. “
Solid cancerous tumors, which are notoriously more difficult to treat with CAR T treatment than blood cancers. This is because counterfeit tumors have a microenvironment that suppresses immune cells and depletes their potency. In June, hopes are seen in the so-called “shielded CAR T cells”, which are engineered cells that secrete recombinant proteins to modulate the tumour microenvironment or attack tumour antigens. “You can have a local benefit, but without the systemic responsibilities,” June explained. He is excited about this type of next-generation technique and believes it will allow CAR T cells to attack fake tumors such as pancreatic cancer or glioblastomas.
In February of this year, a team from a German hospital published knowledge on 15 patients who had been treated with CAR T treatment to treat their lupus, myositis and scleroderma. Patients experienced a 100 percent reaction rate, with remissions lasting an average of 15 months after follow-up.
More than 10 years ago, “we tried to do it at Penn,” June said, “and we couldn’t do it because our regulatory framework is not Germany’s. “There, a hospital could treat patients on a case-by-case basis with the approval of the local regulatory board, rather than requiring permission from their country’s federal oversight agency.
Since the publication of the German hospital’s remarkable insights, enthusiasm has grown and there are now around 44 international trials recruiting autoimmune patients for CAR T therapy. “It’s going to happen,” June said confidently. “This exercise is out of season. “
There are two types of mobile therapies. “Autologous” refers to immune mobiles that are extracted directly from a patient’s body and then custom manufactured with artificial modifications that will allow their own mobiles to combat their expressed disease. “Allogeneic,” on the other hand, refers to immune cells that come from a donor. They have pros and cons, and many corporations focus on one technique or another.
Proponents of autologous mobile treatment point out that they have a strong history of protection, must be rejected through the patient’s body, and have the ability to persist long-term, at least a decade, acting as a living medicine. However, they are expensive and difficult to manufacture, making them difficult to scale.
Proponents of allogeneic mobile therapy are positive about “commercially available” treatments that can treat patients more quickly and cheaply, even if they carry a higher risk of requiring immunosuppressants. Additionally, they necessarily persist as long as an autologous mobile, which can be a smart thing to do for the right patient.
June says we’re going to want either approach in the long run and that they will have independent uses. Cancer treatment, for example, requires the long-term patience of CAR T cell phones, making autologous cell phones a better option. But in the case of autoimmune diseases, allogeneic phones would probably be better, since the framework only needs them for a few months to restore the immune system: “I don’t think we really want an allogeneic phone to last long term, because it is rather an allogeneic mobile security risk.
Ex vivo refers to the engineering of a patient’s mobiles outside their body, which is how all the mobile treatments that exist on the market lately are manufactured. Although production is complex and expensive, June says that ex vivo treatment has a very important advantage: the ability to carry out multiplex genetic engineering on mobiles with base editing and other techniques, which allows scientists to detect at least 15 genes at a time and metabolically reconnect an entire T-mobile.
“I think this will occur in some fake tumors,” June said. “And I don’t see that happening in my lifetime with in vivo engineering. “
That said, in vivo engineering, in which a treatment modifies the patient’s body’s internal immune systems, is faster, more effective and more affordable. In fact, the first in vivo mobile treatment trial was just conducted in patients in Australia through Interius BioTherapeutics.
Capstan Therapeutics, which was co-founded in June and secured my team’s investment in Leaps, is another pioneer in the in vivo mobile treatment of RNA and a proprietary lipid nanoparticle, key technologies in Covid-19 vaccines.
Based on promising preclinical data, Capstan is advancing its lead candidate program toward early clinical mechanistic evidence.
“It is Dr. June’s vision and major advances in nanotechnology and RNA-based medicines that have led to existing efforts to open this new breach in medicine,” said Adrian Bot, chief clinical officer of Capstan. (Disclosure: June owns shares of Capstan, as a co-founder and clinical advisory board member. )
We are fortunate in an era of immediate medical innovation, where our complicated equipment has the potential to turn devastating diseases into manageable conditions. Beyond autoimmune diseases and false tumors, the prospects for CAR treatment extend even to regenerative medicine, with theoretical programs to combat anti-aging therapies, inflammatory dementias and Alzheimer’s disease.
It’s clear that the mobile treatment revolution is just beginning.
Thanks to Kira Peikoff for reporting more and more on this article.
A community. Many voices. Create a free account to share your thoughts.
Our network aims to connect other people through open and thoughtful conversations. We need our readers to share their perspectives and exchange ideas and facts in one space.
To do so, please comply with the posting regulations in our site’s terms of use. Below we summarize some of those key regulations. In short, civilians.
Your message will be rejected if we notice that it appears to contain:
User accounts will be blocked if we become aware that users are engaged in:
So, how can you be a user?
Thank you for reading our Community Guidelines. Read the full list of publishing regulations discovered in our site’s terms of use.