President Droupadi Murmu with Maharashtra Governor Ramesh Bais, IIT Bombay Director Subhasis Chaudhuri, Tata Memorial Centre Director Sudeep Gupta and others during the launch of the first indigenous CAR-T cell therapy, in Mumbai on April 4, 2024.
| Photo Credit: PTI

President Droupadi Murmu on April 4 launched India’s first indigenously-developed CAR T-cell therapy for treatment of cancer, hailing it as a “major breakthrough” that provides a “new hope for the humankind” in the battle against the disease.

During the launch event held at the Indian Institute of Technology (IIT) Bombay located in Powai in Mumbai, she also said the development of this therapy is an example of the ‘Make in India’ initiative.

Developed by the IIT Bombay and the Tata Memorial Centre, this gene-based therapy will help in curing different types of cancer. This NexCAR19 CAR T-cell therapy is the country’s first ‘Made in India’ CAR T-cell therapy, which will significantly bring down the cost of cancer treatment.

Speaking on the occasion, President Murmu said the CAR T-cell therapy is considered as one of the phenomenal advances in medical sciences.

“The development of this therapy is also an example of the ‘Make in India’ initiative and speaks volume about Indian scientists and physicians,” she said.

“The launch of India’s first gene therapy is a major breakthrough in our battle against cancer. As this line of treatment, named CAR T-cell therapy, is accessible and affordable, it provides a new hope for the whole of humankind,” she said.

Sudeep Gupta, Director of the Tata Memorial Centre, said the CAR T-cell therapy has been an enormously expensive treatment which is out of the reach of an overwhelming majority of people.

“NexCAR19 has been rolled out at approximately one-tenth of the price at which it is available outside India,” he said.

IIT Bombay Director Prof. Subhasis Chaudhuri said the treatment costs approximately ₹4 crore abroad.

The low-cost CAR T-cell therapy is a huge achievement for our country and for cancer patients in India, and it places India firmly on the global map of cell and gene therapy, he said.

Mr. Gupta said the creation and rollout of NexCAR19 is a historic milestone in the field of cancer care and genetic engineering.

“This treatment is not only a scientific achievement of the highest order, but also has immense practical application. NexCAR19 will save many, many lives and wipe many, many tears,” he said.

Yuvan Thakkar, an Indian-origin teenager diagnosed with cancer, says he is now able to enjoy the things he loves after life-changing treatment thanks to a fund set up by the U.K.’s state-funded National Health Service to make innovative therapies accessible to thousands of patients.

According to NHS England, 16-year-old Thakkar from Watford near London was the first child in the U.K. to benefit from a pioneering CAR T therapy called tisagenlecleucel (Kymriah) thanks to its Cancer Drugs Fund (CDF).

It comes as the National Health Service (NHS) marks a milestone this weekend of 100,000 patients benefitting from early access to the latest and most innovative treatments with the help of CDF. The undisclosed cost of such treatments is covered by the fund.

“My life has changed so much since I received the CAR T therapy,” said Thakkar, who thanked Great Ormond Street Hospital (GOSH) in London for the “incredible” care he received.

“I remember I had to take so many trips to hospital and had long periods out of school… They have helped me recover to a state where I am able to enjoy so many things I love doing, such as playing snooker or pool, meeting friends and family, and going on wonderful holidays. It’s hard to imagine how things would have been if the treatment wasn’t available,” he said.

Thakkar, diagnosed with a form of leukaemia aged six, received a treatment which modifies a person’s immune cells to recognise and attack cancer cells.

His treatment began in 2019, when he was 11 years old after he relapsed following other treatments such as chemotherapy and a bone marrow transplant. His mother Sapna said the family had received a “second chance” at life since the success of the treatment. Without the fast-track access available through the CDF, the 45-year-old said there may have been no other way for her son to receive the life-saving treatment.

“It felt like our prayers were finally answered. We still feel so grateful for this chance that’s been given to us and not a single day passes by when we haven’t felt thankful for all the doctors and nurses that have helped us through this long and difficult journey,” said Sapna Thakkar.

The CDF, which opened in its current form in July 2016, is used by NHS England to provide fast-tracked access for patients to all new cancer treatments approved by the National Institute for Health and Care Excellence (NICE), in addition to gathering further evidence of long-term effectiveness for promising drugs. It allows faster access to more than 100 drugs to help improve, extend or – in some cases – save their lives.

“Treating 100,000 cancer patients in England with innovative treatments through the Cancer Drugs Fund is a fantastic milestone for the health service to reach, and testament to the hard work of oncologists and their teams across the country,” said Professor Sir Stephen Powis, NHS national medical director.

“This vital fund is helping ensure patients get access to the most promising drugs far quicker than would otherwise be the case, helping people with cancer like Yuvan receive a life-changing intervention that sets a path for a longer, healthier life spent with family and friends,” he said.

The fund benefits people with common cancers, such as breast, lung, colorectal and prostate, as well as those with less common cancers, such as ovarian, cervical, kidney, skin, myeloma, lymphoma and leukaemia, and rare cancers, including thyroid and biliary tract.

The current CDF budget of GBP 340 million is 70 per cent more than the previous CDF and is used alongside NHS England’s Innovative Medicines Fund of GBP 340 million, which the health service said means a total of GBP 680 million is ringfenced for fast-tracking new medicines.

When Michel Sadelain began his decades-long quest to genetically modify immune cells to fight cancer, his peers dismissed his ideas as absurd and even his mother grew concerned for his career.

On Thursday, the French and Canadian scientist was announced as a winner of the prestigious Breakthrough Prize for his pioneering work in CAR T-cell therapy, a new form of treatment that has shown exceptional efficacy against blood cancers.

“Over the years, I can’t tell you how many times I’ve heard this won’t work, can’t work, even if it works it has no future,” the 63-year-old told AFP in an interview.

He was passed up for grants, promotions became uncertain, and graduate students steered clear of joining his lab.

“One thing I have to do is to throw a big party with all those who contributed,” Sadelain said, laughing. He will split $3 million with American immunologist Carl June, who also led groundbreaking research into the field independently of his co-winner.

The Breakthrough Prize awards “the world’s most brilliant minds” in fields including life sciences, fundamental physics and mathematics, styling itself as the Silicon Valley-backed answer to the Nobels. Founding sponsors include Sergey Brin, Priscilla Chan and Mark Zuckerberg.

Living drugs

Sadelain studied medicine in Paris, then immunology in Canada, before taking up postdoctoral research at the Massachusetts Institute of Technology in 1989.

At the time, there was great interest in developing vaccines to train the immune system to recognize and destroy cancer cells, in the same way it can be taught to tackle foreign invaders such as bacteria and viruses.

“But I started thinking that perhaps we should learn how to directly instruct the fighters of the immune system, in particular the T-cells,” he said, with his early work focused on mice.

After moving to the Memorial Sloan Kettering Cancer Center in New York, Sadelain developed a way to use a disabled virus to genetically reprogram human T-cells, so that they grew claw-like structures called antigen receptors, allowing the T-cells to target specific cancer cells.

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Beyond recognizing the cancer, these Chimeric Antigen Receptor (CAR) T-cells, as Sadelain named them, were also given genetic instructions to enter a killing mode and to multiply, growing an army inside the body to eliminate the enemy.

Thanks to the groundwork laid by June and Sadelain, there are now half a dozen US approved CAR-T cell therapies, with hundreds more trials underway.

Patients’ own T-cells are collected, modified outside the body, then infused back into the blood, creating a so-called “living drug.”

In a trial against multiple myeloma, a cancer that develops in plasma cells, 72% of patients responded to treatment, with total disappearance of the disease seen in 28%, among whom 65% had sustained eradication for 12 months.

High costs

The treatment comes with serious side effects — including in some cases death — from the release of inflammatory molecules called cytokines. Doctors have learned to recognize and manage this better over time.

Another risk is nervous system impairment, with symptoms such as deep confusion or inability to talk, although these clear up within days.

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Sadelain is excited for what the future could hold: from improving the T-cells so they tackle solid cancers, to treating autoimmune conditions such as lupus, to fighting currently intractable infections such as HIV.

One area he acknowledges must improve is the sky high cost, with price tags upwards of $500,000. In the United States, private and government-subsidized insurers pick up most of the tab for those who qualify, as do health systems in Europe.

“Researchers were aghast when we saw what was charged for these very first therapies,” he said. “The cost has to come down,” he added, something he expects to happen as the pharmaceutical industry improves its processes, and as scientists continue to innovate.

For example, his own lab is preparing to publish a study showing that improvements to CAR-T cells’ efficiency vastly reduces the number needed for treatment.

Other researchers are looking at using stem cells to manufacture CAR-T cells, creating a more economical “off the shelf” solution instead of relying on patients’ cells. Clinical testing is underway.