CAR-T (chimeric antigen receptor T-cell) therapy is a relatively new blood cancer treatment that alters the genes inside a patient’s T cells so these cells can destroy cancer cells. As CAR-T cells can stay active in the body for a long period of time, some patients only need one round of therapy, making it less invasive than other blood cancer drugs. And, in some cases, the therapy has cured patients where other therapies have failed. This year, two ground-breaking pieces of news have emerged in the CAR-T therapy space.
- The first human CAR-T therapy patients are cancer-free, 10 years after undergoing the treatment.
- The clinical-stage biopharmaceutical company Arcellx has raised $124 million in IPO (initial public offering) financing to advance CAR-T therapy.
Scientists from around the world use the community site RegMedNet to keep up with cell therapy developments and news. RegMedNet has covered both of these progressions in CAR-T therapy on its website.
Researchers from the University of Pennsylvania and Novartis Institutes for Biomedical Research have published a pioneering study that documents the recovery of the first two cancer patients to receive CAR-T therapy. These patients underwent the therapy as the initial treatment for their chronic lymphocytic leukemia (CLL) 10 years ago. The findings, which have appeared in the journal Nature, confirm that the patients are now cancer-free.
In 2010, Doug Olson and Bill Ludwig were battling CLL, which is the most common type of leukemia in adults. Both had undergone several treatments since their respective diagnoses in 1996 and 2000. As remaining treatment options dwindled, both volunteered to become the first participants in a clinical study of CAR-T therapy, which involves genetically modifying a patient’s T cells so these cells can kill cancerous cells, at the Abramson Cancer Center and the Perelman School of Medicine at the University of Pennsylvania. Little did they know that the therapy would eradicate their end-stage leukemia and launch a new era of highly personalized medicine.
Both patients achieved complete remission within a year. Since then, Olson has taken up distance running and completed six half-marathons. He fundraises for the Leukemia and Lymphoma Society and supports newly diagnosed patients. Meanwhile, Ludwig enjoyed travelling with his wife in their motor home and was able to enjoy several family milestones. He sadly passed away in 2021 due to COVID-19 complications.
Although durable remissions have been demonstrated in relapsed, refractory B cell malignancies with CD19-specific CAR-T cells, until now, little research has been conducted into the long-term stability and potential of infused cells. During this study, the researchers analyzed the evolution of CAR-T cells over time. A highly activated CD4+ cell population emerged and became dominant in both patients. Data indicated that there were two distinct phases of CAR-T therapy responses in the patients. The killer T cells dominated the initial phase, and the CD4+ T cells controlled the long-term remission.
Over the following years, the CD4+ cells continued to demonstrate tumor-cell-killing characteristics. The study revealed that the CAR-T cells were still detectable at least a decade after infusion in both patients, marking the longest persistence of CAR-T therapy against CLL recorded to date.
In 2017, the U.S. Food and Drug Administration (FDA) approved the therapy that Ludwig and Olson received. This treatment, which is now known as Kymriah®, is the first CAR-T product that the FDA has approved to treat acute lymphoblastic leukemia (ALL) in pediatric and young adult patients. In 2018, the FDA then approved the treatment for certain types of lymphoma.
10 years ago, few scientists had explored CAR-T therapy’s therapeutic potential. Many considered the therapy a fringe approach that was unlikely to work. This new study demonstrates that the therapy can offer a solution for patients who have cancer that isn’t responding to chemotherapy.
Science in the regenerative medicine sector continues to advance quickly and drive investor interest. In 2021, developers raised a record-breaking $22.7 billion, climbing 14% from 2020’s previous record of $19.9 billion. Overall, investments in regenerative medicine have more than tripled since 2017.
Gene therapies and cell-based immuno-oncology (IO) developers make up the majority of these investments and have generated $10.2 billion and $10.1 billion respectively, marking 12% and 26% year-on-year increases. Gene therapy typically generates the largest amount of investment, which was the case in 2021. That said, cell-based IO companies are now seeing a much higher level of investment, a level that is outpacing gene therapy financing. And, in 2021, companies active in gene editing raised approximately one-third of total regenerative medicine funding.
As a result of these developments, IPOs in the regenerative medicine field have risen by 30% to a record number of 26 (totalling at $4.8 billion) between 2020 and 2021. While Sana Biotechnology raised $675 million in IPO financing, Lyell Immunopharma raised $425 million, CARsgen Tx raised $400 million, and Instil Bio raised $368 million.
Another company that has raised impressive IPO financing is Arcellx, which raised $124 million to develop its CAR-T therapeutic candidate for clinical studies. In its bid to reimagine cell therapy through the development of revolutionary immunotherapies for cancers and other incurable diseases, the biotechnological company offers deimmunized synthetic binding scaffolds, which are smaller than those available in the CAR-T therapy industry at the moment.
Arcellx’s preclinical research has demonstrated that the smaller size of these scaffolds enables higher transduction efficiency, higher expression of binders on the surface of T cells, and lower signaling that can cause off-target activation of T cells.
In February 2022, Arcellx closed its initial IPO of 9,487,500 shares of its common stock, which includes the exercise in full of the underwriters’ option to buy 1,237,500 additional shares of its common stock. Underwriters could buy these shares at a public offering price of $15.00 each. The aggregate gross proceeds from the offering totaled at $142.3 million, before deducting commissions, underwriting discounts, and other expenses.
Arcellx hopes to develop a range of therapeutic options that incorporate different cell modalities, mechanisms, and targets to treat solid tumors, hematologic cancers, and indications outside of oncology, like autoimmune diseases.
RegMedNet is a community site for regenerative medicine specialists and other research professionals to access free, high-quality resources like webinars, news pieces, and journal articles, each of which examines the latest in cell therapies’ development, manufacture, regulation, and commercialization. These specialists also use RegMedNet’s sister journal, Regenerative Medicine, to keep their knowledge up to date. This award-winning journal covers topics from biologics, biomaterials, and cell and gene therapies to small molecule drugs and tissue engineering.
The progressive scientific and medical publisher Future Science Group is home to both RegMedNet and Regenerative Medicine. The independent publisher curates 34 peer-reviewed, open-access journals and provides platforms for clinicians, researchers, and decision-makers to collaborate and communicate.
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