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With a compound annual growth rate (CAGR) of 34.2%, the global CAR-T cell therapy market is projected to hit $15.4 billion by 2028. This hypergrowth is largely driven by the incredible promise of CAR-T cell therapy as an effective, lasting way to fight cancer. Historically, however, that growth has been hindered by obstacles to developing a fast, affordable and low side-effect version of the treatment.
Avalon GloboCare (NASDAQ: AVCO), a clinical-stage biotech company focused on immunotherapy, diagnostics and therapeutics, may have found the breakthrough researchers have been looking for the past decade with its leading drug candidate, AVA-011, an mRNA CAR-T platform that solves the key obstacles blocking the revolutionary cell therapy from becoming more widely available.
Heres why CAR-T therapy has taken the biotech industry by storm, and why Avalon is optimistic that AVA-011 could finally offer a safe, affordable version of the treatment to patients around the world.
What Is CAR-T Cell Therapy?
CAR-T cell therapy is an exciting cancer treatment thats made huge waves in the biotech community over the last 10 years. Using a blood sample from a patient, developers can isolate the patients T cells, reengineer them with chimeric antigen receptors (CAR) and then reintroduce those cells into the patients bloodstream through an infusion.
T cells are the workhorses of our immune system. They systematically destroy foreign substances that could cause harm. The reengineered CAR-T cells offer two benefits on top of what T cells already do. First, the CAR molecule acts as a kind of molecular GPS guiding the T cell to the tumor site. Second, the CAR molecule itself is an added weapon that can bind to the signature tumor it was engineered to target and trigger an immune-driven cancer killing effect.
When the infusion is made it acts as a living drug, multiplying and remaining in the body to suppress any recurrence of the cancer it was engineered to target.
Story continues
In human trials of various versions of CAR-T cell therapy, more than 80% of patients saw a complete remission or a partial response for their cancer. While long-term studies are scarce because the treatment is still so new, one 5-year study found that every single patient who showed a full or partial response to the CAR-T cell therapy maintained that response five years later meaning that those with a complete response were still cancer-free and those with a partial response saw no progression of the disease to worse stages.
While the results of CAR-T cell therapy are nothing short of impressive, key challenges have prevented it from becoming more widespread. Above all, theres a high risk of adverse reactions. Cytokine Release Syndrome (CRS) is the most common reaction, causing fever and hypotension in 13% to 49% of patients, depending on which treatment was used.
Moreover, designer CAR-T cells like those used in this powerful new treatment are difficult and expensive to produce. The conventional protocol takes 14 days from vein to vein that is, from the time its extracted from the patient to the time its reengineered and delivered to the patient again via infusion. It also requires a disarmed viral vector to deliver the CAR molecule to the T cell. This adds additional cost and time to the manufacturing of the treatment. On average, a single infusion produced using this conventional protocol costs $500,000 for the patient.
Avalons FLASH-CAR Platform Addresses Key Challenges in CAR-T Cell Therapy
To overcome these obstacles, Avalon developed its proprietary FLASH-CAR platform. Using messenger RNA (mRNA), the platform is able to bypass the need for a viral vector to introduce the CAR molecule to the T cell. That bypassing shortens the vein to vein time to just 1 to 2 days and significantly reduces the cost of manufacturing.
Moreover, the mRNA used is modified to come with a safety switch. If a patient experiences CRS or another adverse reaction, Avalon has developed an FDA-approved antidote that can target those CAR-T cells and deactivate them, stopping the adverse reaction. This built-in safety switch is a game changer that gives patients and their healthcare providers more control over their cancer treatments.
The mRNA also enhances the living drug-effect by signaling a more rapid proliferation of CAR-T cells. This could potentially make the treatment more effective and offer even more long-term protection for patients.
Finally, mRNA contains functional units that allow manufacturers to activate other immune systems cell types as well, including natural killer cells and dendritic cells. This ability to engineer more than just T cells expands the potential of the cell therapy beyond the domain of blood cancers the only kinds of cancers the T cell-based infusions have currently been able to target successfully.
AVA-011 Prepares for First Human Clinical Trials
Avalons leading FLASH-CAR drug candidate is AVA-011, which is indicated to treat B-cell acute lymphoblastic leukemia and non-Hodgkin lymphoma (both blood cancers). With the potential of the FLASH-CAR platform to engineer other immune cell types, Avalon is working to develop other drug candidates that could target solid tumors. The promising new treatment has completed preclinical testing and slated to begin its first human clinical trials in 2022.
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2021 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.
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