Breakthrough drugs raise worries over higher costs

Potential breakthroughs in medical treatment for serious diseases are turning what was once “the stuff of science fiction” into “real life science,” in the words of former FDA Commissioner Scott Gottlieb. 

New areas of treatment include methods like replacing defective genes with working ones or even editing genes. 

But the worry is that these new breakthroughs will also have enormous price tags. One new gene therapy this year became the world’s most expensive drug, with a cost of $2.1 million. 

Here are five new areas of treatment that could provide breathtaking new cures, but also at potentially stunning costs. 

Cell therapy

Instead of a standard pill, what if living cells were injected into a patient to help treat a disease? That, in a nutshell, is the idea behind cell therapy. 

Cells can either come from the patient’s own body or from a donor. 

Treatments currently in development that replace damaged cells with new ones could help repair retinas, replace brain cells lost to Parkinson’s disease and inject healthy skin cells to help heal wounds or burns, according to the Harvard Stem Cell Institute. 

The Harvard Stem Cell Institute says its scientists are working on ways to make it easier to use cells from other people in patients’ bodies. That would allow for broader use of such treatments instead of forcing doctors to make tailored treatments for each individual using just their own cells. 

“If cell therapy is ever going to be available to large numbers of people, we will need disruptive breakthroughs in academic, commercial, and industrial research and development,” the Harvard Institute writes. 

Both the advancements are not without controversy. Some have raised ethics concerns over types of cell therapy, like stem cells that come from human embryos.

Gene therapy

People normally do not inject viruses into their body, but that’s exactly what can happen with gene therapy. 

A working gene is delivered to a cell via a virus modified to be harmless. That working gene can replace or deactivate a mutated one that is causing a disease, according to the National Institutes of Health (NIH). 

One breakthrough example is Zolgensma, the new treatment for the deadly childhood disease spinal muscular atrophy, which also comes with a massive price tag. 

But gene therapies are in the early stages and remain risky. 

“Although gene therapy is a promising treatment option for a number of diseases (including inherited disorders, some types of cancer, and certain viral infections), the technique remains risky and is still under study to make sure that it will be safe and effective,” the NIH states. “Gene therapy is currently being tested only for diseases that have no other cures.”

DNA editing

Instead of inserting functioning genes into a patient, a new technology called CRISPR allows researchers to edit DNA, cutting it and making changes. 

The technology has exciting implications for curing disease, but also daunting ethical risks. 

Earlier this month, researchers announced that they had used gene editing to remove HIV from mice. 

“The possibility exists that HIV can be cured,” Howard Gendelman, a researcher at the University of Nebraska Medical Center who worked on the study, told USA Today. “It’s going to take a little bit of time, but to have the proof of concept gets us all excited.”

The darker side of the technology came in November, when a Chinese scientist named He Jiankui announced that he had created the first “CRISPR babies”: a pair of twins with edited genes. 

Francis Collins, the NIH director, tweeted that the move “is profoundly disturbing & tramples on ethical norms.” He called for an “international consensus” on limits on the technology. 

CAR T-cell therapy

In one of the most exciting developments in recent cancer treatments, researchers are now working to train a patient’s own immune system to attack cancer.

A type of cell therapy, called CAR T-cell therapy, has “captured the attention of researchers and the public alike because of the remarkable responses they have produced in some patients — both children and adults — for whom all other treatments had stopped working,” the National Cancer Institute writes. 

One example of the treatment, called Kymriah, was approved in 2017 with a price tag of $475,000. 

CAR T-cell therapy works by drawing blood from a patient, separating out the T-cells (part of the immune system) and modifying them to be able to attack tumor cells. 

Sickle cell anemia treatment

Sickle cell anemia, a disease that causes extreme pain from misshaped blood cells, has no real cure. But that could change through a gene therapy being developed at the NIH. 

The gene therapy involves removing cells from a patient and adding a functioning gene instead of the defective one in people with sickle cell disease, according to the NIH. 

CBS’s “60 Minutes” recently reported on a patient with sickle cell disease who had seen her condition improve considerably after receiving the gene therapy. 

But Collins, the head of the NIH, told “60 Minutes” there are still many unknowns and risks with the area of treatment. 

“Make no mistake, we’re talking about very cutting-edge research where the certainty about all the outcomes is not entirely there,” Collins said. “We can look back at the history of gene therapy and see there have been some tragedies.”

Another concern: The advocacy group Patients for Affordable Drugs is worried that once it reaches the market, the treatment for sickle cell could cost more than $1 million.