First indigenously developed CAR-T cell therapy could be the safest ever

India’s first indigenously developed Chimeric Antigen Receptor (CAR)-T Cell T therapy, a cutting- edge treatment for specific types of cancer patients, has shown promising results and could be the safest therapy in this category so far, researchers have said.

The therapy was tested on six paediatric patients of Acute Lymphocytic Leukaemia and 10 adults suffering from B-cell lymphoma as part of phase one clinical trials by researchers associated with the Tata Memorial Hospital in Mumbai and the Indian Institute of Technology, Bombay.

“Phase one clinical trial data demonstrates that India’s first indigenously developed novel CAR-T Cell therapy is safe and shows promising early sign of efficacy in treating lymphoma, a type of blood cancer,” said IIT Bombay after findings from the trial were presented in the annual symposium of cell and gene therapy at the Christian Medical College, Vellore.

How does it work?

CAR-T cells are a patient’s own immune cells that are genetically engineered in the laboratory to fight cancer. As part of the treatment, a specific type of white blood cells called T cells are changed in the lab so they can find and destroy cancer cells.

It is also sometimes referred to as a type of cell-based gene therapy because it involves altering genes inside T cells to help them attack cancerous cells.

Promising results

As part of the clinical studies, the participants received autologous HCAR19 therapy (humanised CAR 19 therapy, the product developed by scientists associated with IIT Bombay) and it was observed that there were no dose-limiting toxicities and only low grade Cytokine Release Syndrome (CRS) was seen in 40 percent of participants.

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None of the participants had immune effector cell-associated neurotoxicity syndrome. While three out of 10 participants had a complete response post-CAR-T cell therapy and none of the participants required ICU admission, there were no CAR-T treatment related deaths.

“Overall, the novel humanised HCAR19 tested in phase one clinical trials for adult lymphoma was found to be safe and has shown promising early signs of activity,” said IIT Bombay.

It added that the clinical trials will now enter phase two clinical trials after approvals from the Central Drugs Standards Control Organisation.

Safe and effective

Rahul Purwar, associate professor at IIT Bombay, who is the chief developer of the HCAR19 therapy, said that the primary objective of the first phase of the trial was safety and it established that there was no high-grade cytokine-release syndrome, no death on the trial, and no ICU admission for severe toxicity.

“The secondary end point was efficacy and we saw three complete responses which means patients became cancer free, one partial response and one stable disease; so overall response is 50 percent, which is huge,” Purwar told Moneycontrol.

He also said that most of the studies - using the therapy developed in other developed countries - have shown some neurotoxicity and ICU admission.

“We did not see these severe toxicities in our phase one study and therefore we can say this is the safest CAR so far,” Purwar, who is the founder and CEO of IIT, Bombay spin-off company ImmunoACT, said.

Therapy in India

ImmunoACT along with Immuneel Therapeutics - backed by Biocon Biologics and top oncologist Dr Sidhartha Mukherjee - are two startups currently working to make CAR T cell therapy available in India.

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Both of these companies have said the therapy would be available in the country at a fraction of cost charged for the treatment in the developed countries like the US where it can cost about Rs 2-3 crore.

CAR T cell therapy is used as third or second-line treatment for patients of specific types of cancers of blood and lymph nodes. In India, those willing to undergo this treatment have to travel to other countries, mostly the US.

Purwar said that the therapy would most likely be available commercially in India from early 2024.