New drug can save your life, on the cheap

Written by O’Laurie Kellman, CNN

It looks much like an immunotherapy injectable chemotherapy drug — and costs about a quarter of the US$102,500 that it takes to gain approval from the US Food and Drug Administration.

But it has far-reaching impact, helping reduce the death toll by a third. At a drug fair held at the Hyatt Regency in Washington, DC, the 13-day immunotherapy treatment was on the floor like anything else.

The technology, dubbed AMT-04, was developed by the Bill & Melinda Gates Foundation and Ann C. Rothman, a blood cancer researcher at Einstein Medical Center in New York.

AMT-04 works by inserting the gene TP53 into the immune system’s T cells — a team of white blood cells that fight off infections.

“If you get the right disease that in the past for whatever reason has eluded you, but today you have a chance to find them, you’ll be like an alligator on the surface of the water,” says Craig McCourt, the director of medical and scientific affairs for the Gates Foundation and a researcher at Georgetown University Medical Center.

Other promising treatments in development work in a similar way, giving treatment that could reduce the sometimes torturous process of cancer diagnosis, treatment and many deaths from blood cancers.

Trial proves prowess

The system that Amgen and Biogen received FDA approval for last November, known as an antibody-drug conjugate (ADC), is being hailed as a cure-all. It inhibits the enzyme tumour necrosis factor (TNF), that drives cancer growth and survival. When in tumor cells, the cancer cells are destroyed; however, when the ADC is introduced into normal cells, the drug attack is effectively halted.

“Adamivir” is another drug that can help fight cancer — but only in certain types of lymphoma or leukaemia.

Attracted by strong results, Pfizer recently licensed the drug. Other promising treatments work in a similar way to AMT-04 and can be used to combat hundreds of different cancers, including breast, lung, colorectal, pancreatic, prostate, renal, and larynx.

The swift adoption of AMT-04 has been attributed to the fact that the cancer-specific approach, without requiring extensive side effects, could be used to treat multiple diseases at once.

One stark contrast

But AMT-04 could also be rolled out in the future to treat immune-system disorders other than cancer — with at least one recent trial testing AMT-04 as a treatment for multiple sclerosis (MS).

The concept of using antibody-drug conjugates to fight multiple diseases at once is pioneering. Most immunotherapy drugs are first tested for one condition, either a specific genetic mutation or several such genetic mutations, which have all been confirmed in multiple clinical trials.

Now that AMT-04 can be safely used in multiple conditions, perhaps one day it will help prevent disease altogether.

That’s where the miracle for AMT-04, and much of immunotherapy in general, could come in.

AMT-04 can safely be used in multiple conditions, perhaps one day it will help prevent disease altogether. O’Laurie Kellman, CNN

Irene Nestler, a clinical associate professor at the Icahn School of Medicine at Mount Sinai in New York, says the program could eventually offer an objective way of predicting which patients will benefit.

“If it is proven to be safe, it could change the way we identify appropriate treatment for various diseases,” she says.

She explains that normally patients diagnosed with multiple blood cancers would get the same treatment, regardless of their genetic status — and if they didn’t, that would be a no-go for that patient.

“AMT-04 can differentiate between risk and control,” she says.

Only time will tell if AMT-04 really saves lives in the future, but right now, the program is largely appreciated by those who have been using it to treat patients with blood cancers.

The data show in other trials that it reduced the severity of tumour cells, increases the survival rate of those with blood cancers, and reduces side effects. And that’s how a program as new as this could be a game-changer.

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