'Gene Therapy' Cancer Drug That Costs $727,000 Per Treatment and Only Works 'Completely' 4% of the Time Granted 'Accelerated Approval' by FDA

Adaptimmune's "genetically modified" tumor drug TECELRA contains "short spans of genetic material that are identical to HIV."

The U.S. Food and Drug Administration (FDA) on Friday approved Adaptimmune’s first-of-its-kind gene therapy to treat a rare type of solid tumor cancer in the soft tissues typically affecting young men.

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The genetically modified drug, TECELRA® (afamitresgene autoleucel), is for the treatment of adults with unresectable or metastatic synovial sarcoma who have received prior chemotherapy.

“TECELRA is the first engineered cell therapy for a solid tumor cancer approved in the U.S., and the first new therapy option in more than a decade for synovial sarcoma, a rare, soft tissue cancer that most commonly impacts young adults,” a press release from Adaptimmune reads.

Synovial sarcoma most commonly develops in the extremities and impacts about 1,000 Americans each year, often affecting adult males in their 30s or younger.

The gene therapy is administered as a single intravenous dose.

The therapy is composed of a patient’s own T cells that have been genetically modified to express a T cell receptor (TCR) targeting the MAGE-A4 antigen.

The process is said to involve collecting T cells from the patient, modifying these cells to recognize and attack cancer cells expressing the MAGE-A4 antigen, and then reintroducing the modified T cells back into the patient.

The company says the treatment will launch at a list price of $727,000.

The FDA granted TECELRA “accelerated approval,” which is given to drugs for “serious conditions that fill an unmet medical need to be approved based on a surrogate endpoint,” according to the agency’s website.

Accelerated approval enables the FDA “to approve these drugs faster,” bypassing the regulator’s more rigorous full approval process.

Despite being the first gene therapy to be approved in the United States that uses a patient’s own immune response generating T-cells to fight cancer, the accelerated approval was based on a single seven-month study published in The Lancet.

And that study only included 44 patients.

“The approval of TECELRA was based on results of the SPEARHEAD-1 (Cohort 1) trial, which included 44 patients,” the press release reads.

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Moreover, the study confirmed only about a third of patients experienced any positive response to the treatment.

“Overall response rate was 37% (19 of 52; 95% CI 24–51) overall, 39% (17 of 44; 24–55) for patients with synovial sarcoma, and 25% (two of eight; 3–65) for patients with myxoid round cell liposarcoma,” the Lancet study says.

Significantly, only 4.5% experienced a “complete response.”

The study also revealed a potentially life-threatening systemic inflammatory response called ‘cytokine release syndrome’ occurs in most (71%) of patients.

The press release reads: “BOXED WARNING: Cytokine release syndrome (CRS), which may be severe or life-threatening, occurred in patients receiving TECELRA. At the first sign of CRS, immediately evaluate patient for hospitalization and institute treatment with supportive care. Ensure that healthcare providers administering TECELRA have immediate access to medications and resuscitative equipment to manage CRS.”

Following treatment, two percent of patients experienced ‘Immune Effector Cell–associated Neurotoxicity Syndrome’ (ICANS), a serious neurological side effect.

Infections occurred in a third (32%) of patients.

Some patients treated with TECELRA even developed “secondary malignancies or recurrence of their cancer.”

Under a section titled “Potential for HIV Nucleic Acid Test False-Positive Results,” Adaptimmune explains that TECELRA contains “short spans of genetic material that are identical to HIV” and that therefore “some commercial HIV nucleic acid tests may yield false-positive results in patients who have received TECELRA.”

The company advises patients should “not donate blood, organs, tissues, or cells for transplantation” for “at least 4 weeks” after receiving TECELRA.

Finally, TECELRA contains Dimethyl Sulfoxide (DMSO), a substance known to induce genetic mutations, which are permanent changes in an organism’s DNA.

DMSO may be toxic to blood, kidneys, liver, mucous membranes, skin, eyes, according to one safety data sheet.

“Repeated or prolonged exposure to the substance can produce target organs damage,” the document warns.

Adaptimmune is owned by the infamous asset manager BlackRock, an official partner of the World Economic Forum (WEF).

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