India's Blood Cancer Gene Therapy Shows 73% Response Rate in Clinical Trials

India's Blood Cancer Gene Therapy: According to results of clinical trials published in The Lancet Haematology journal, an indigenously developed gene therapy for certain blood cancers has shown a response rate of 73 percent among patients in India.

In this CAR T-cell therapy, T-cells, which are immune cells, are genetically engineered to attack cancer. While this study enrolled patients with leukemias involving the bone marrow and lymphomas of the lymphatic system.

The researchers involved Indian Institute of Technology-Bombay and Tata Memorial Hospital, Mumbai, who said that low- and middle-income countries, even in patients with 'B-cell' tumours, with tumor growth after a period of remission (relapse) or not responding to treatment (refractory) suffer poor outcomes due to lack of effective therapies.

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B-cells, another subgroup of white blood cells, play a significant defensive role against infections by producing antibodies.

"The clinical trials of India's first gene therapy for cancer offer hopes of another chance to live among these patients, that there is one more drug that doctors can try," lead author Rahul Purwar, professor at IIT-Bombay and founder of ImmunoACT, told PTI.

ImmunoACT, or Immuno-Adoptive Cell Therapy Private Limited, is a gene-modified cell therapy company that was formed as a spinoff from IIT Bombay.

"CAR T-cells, just like normal T-cells, persist in the body for a long time. These cells help to prevent a relapse," first author Dr Hasmukh Jain, professor of medical oncology, Tata Memorial Hospital, said.

Purwar said that CAR T-cell therapy was also a cost-effective alternative compared to that available in the developed world.

"We developed it over a period of 11 years, starting with drug design and lab work, which was then translated into animal studies, before progressing to clinical trials," he said.

The publication claims that 'talicabtagene autoleucel' is now approved in India and "available for USD 30,000, less than one-tenth of the price of other approved CD19 CAR T-cell therapy products marketed worldwide."

In a linked commentary article, authors from the University of Pennsylvania in the US-not involved in the study-said the approved CAR T-cell products cost between USD 373,000 and 475,000, while clinical care and relocations essentially take the very total treatment cost beyond USD 1 million.

"Therefore, access to CAR T-cell therapy is an important limitation to the success of this approach in not only high-income countries but particularly in low-income and middle-income countries," the research paper said.

In Phase 1 trials in India, fourteen patients aged 18 years or older with relapsed or refractory B-cell lymphoma received talicabtagene autoleucel by intravenous infusion. In Phase 2 trials, the drug was given to 50 patients aged 15 years and older with relapsed or refractory B-cell leukaemia or B-cell lymphoma.

The average age of the total study cohort was 44 years, with 49 male and 15 female participants among the 64 patients studied.

Generally, Phase 1 trials were put to the testing of safety with a small number of volunteers (20-100). Phase 2 trials came after Phase 1, with participant numbers usually ranging from 100 to 300 and with a focus on new drug effectiveness.

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"In the 51 patients studied, the overall response rate was found to be 73%," said the paper.

There were two deaths related to the treatment, and common toxicities included neutropenia (low neutrophil levels) in 55 out of 57 patients, while thrombocytopenia (low platelet levels) was in 37 patients, the team said.

Anaemia was found to present in 35 patients.

"Talicabtagene autoleucel had a manageable safety profile and induced durable responses in patients with relapsed or refractory B-cell malignancies," according to the research article.

"This therapy addresses an important unmet need for patients with relapsed or refractory B-cell malignancies in India," it said.

Jain said, the findings "give us an opportunity to test the therapy in the earlier settings and in combination with other immunotherapies".

These trials are currently going on in the Tata Memorial Centre, he informed.

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