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The US Is Critically Short of a Key Medicine Used to Treat Epilepsy And Cancer

HOLLY ROSENKRANTZ, THE WASHINGTON POST
6 NOV 2019

A severe shortage of immune globulin - a popular medicine used to treat epilepsy, cancer and immune disorders - is forcing doctors nationwide to cancel patients' lifesaving infusions, even as hospitals and treatment centers are resorting to rationing and dose-cutting.

 

Immune globulin helps protect patients from infections, and it is used to treat many medical conditions including seizures, leukemia, auto­immune diseases, organ transplants, acute muscle illnesses and nerve disorders.

As off-label use spreads for lifesaving niche drugs, supplies grow scarce.

"It is miraculous," said John Boyle, president of the Immune Deficiency Foundation, a national nonprofit group representing patients with immune disorders, many of whom receive immune globulin. "It is the one thing, if you have an antibody deficiency, that gives you gas in the tank. It is necessary to live."

Made from human blood donations, immune globulin contains infection-fighting antibodies derived from blood plasma, and it is typically administered to patients intravenously in a hospital or an infusion center.

The medicine is highly effective and has become popular in recent years among doctors who routinely prescribe it for off-label conditions.

Jerry Siegel, a clinical associate professor at Ohio State University College of Pharmacy, said doctors are using the treatment, known as IG therapy, as a medicine for ailments such as strep infections that have crossed the blood-brain barrier, infertility and even toe fungus.

 

"We don't always know why it works, but it works," Siegel said.

"When it was taking off in the '80s, we would see patients come into the hospital on a gurney so flaccid they were unable to walk. The next day, after their IG therapy, they were able to get up and walk out the door. When you see some really profound results like that, it is really convincing to prescribers."

Shortages of immune globulin have become more frequent in the past few years, spurred by demand for the medicine. Nevertheless, manufacturers say the current shortage is among the worst they have experienced since the treatment has been on the market.

In a statement in August, the Food and Drug Administration said that "despite increased supply of immune globulin products in recent years, the demand for IG products has also increased over the same time and there is an ongoing shortage."

While the agency does not have the authority to force an increase in production, FDA officials said they are trying to help mitigate the shortage by working with drug companies to boost supply.

 

Even so, the shortage is depriving patients of crucial treatment, and some have resorted to desperate measures. In Orlando, Laurel Smith gave the hospital bottles of immune globulin so that they could continue treating her son, Lowry, 7, who suffers from a rare form of autoimmune encephalitis, called GAD-65, which means his immune system attacks his brain.

Lowry is treated with aggressive medicines that dampen his body's immune response and leave him with no natural defenses against invading germs that can lead to illnesses and infections.

As a result, Lowry gets intravenous immune globulin (IVIG) treatments every month. Without the treatments, exposure to the outside world could send him into a coma.

"Lowry needs triple immune suppression to beat his disease, but he has become a bubble boy where a single infection could kill him without IVIG to give protection from day-to-day germs," his mom said.

In 2017, just after Lowry was diagnosed with limbic encephalitis, his doctor ordered home health IVIG infusions.

But doctors soon decided to give the infusions in a hospital as opposed to in the home along with other treatments, leaving his mother with two bottles of the immune globulin.

 

Knowing its value, she held on to it for over two years, assuring it was always kept properly in case of an emergency. To everyone's surprise, two years later, that emergency came when they were told that there were no more immune globulin supplies, the hospital could not obtain it, and Lowry's infusion was canceled.

Michael Ganio, director of pharmacy practice and quality at the American Society of Health-System Pharmacists, a trade group, said that doctors are responding to the shortage by reducing doses and lengthening the time between treatments.

He pointed out that, in the past few weeks, many medical offices have been calling patients to tell them that their infusion appointments were canceled.

"People are taking a hard look at who is receiving the medicine, and really, is it the right treatment," Ganio said.

This is where a tension point has been created among the different communities who use immune globulin. Some say that, given the shortage, certain illnesses should get priority. And the Immune Deficiency Foundation put out a statement in July saying doctors should find suitable alternative medicines.

"For our population, there is no substitute," said Boyle, the IDF president.

Many experts say that it will not be easy to prevent immune globulin shortages from continuing to be a problem. According to the Plasma Protein Therapeutic Association, which represents makers of plasma-based medicines, while collections continue to rise in the United States, supply is failing to meet the steady increase in demand.

Manufacturers operate collection centers throughout the country that pay people about US$30 for donated plasma.

Among the companies that manufacture the medicine are Takeda Pharmaceuticals, CSL Behring, Grifols and ADMA Biologics. Siegel said it can take seven to 12 months to produce and supply plasma-based medicines to customers.

It can take more than 1,000 plasma donations to make a one-year supply for a patient.

Katie Joyce, a spokeswoman for Takeda, said the company was making "efforts to optimize both plasma collecting and manufacturing capacity, opening more collection centers and more recently investing in a new manufacturing facility in the US."

CSL Behring also noted that it was working hard to manufacture more product "with ongoing investment in state-of-the-art manufacturing facilities around the world to increase manufacturing efficiency."

Brad Pick, a Grifols spokesman, said the company has plans to open more infusion centers for collecting plasma for the immune globulin. The company is building a new plant in Clayton, NC When it's operational in 2021, the company will have increased its production capacity by more than 35 percent compared with today, he said.

For patients, the shortage is frustrating because there is not a lot they can do to fix the problem. Families can't organize a blood drive to help collect plasma. The collected plasma is processed at a central manufacturing plant and then distributed, so donation rates at a collection center do not tie directly to availability of medicines for local patients.

"We just don't know when the supply will increase," Ganio said. One manufacturer, he said, is waiting on FDA approval to build a new plant. But he said there is "not a lot of transparency" from the manufacturers.

In Massachusetts and other areas, some hospitals have notified patients that immune globulin will not be available to them until next year.

Juli Hunt, whose son, Colton, 8, was airlifted to Children's National Hospital from Fredericksburg four years ago during an attack of anti-NMDA receptor encephalitis, has been traveling to the hospital in the District for monthly intravenous immune globulin.

Last month, she received a notice that he would no longer be receiving the IVIG treatments, and that the subcutaneous version is a solid option.

"I get that they are saving what they have for those who are critical," Hunt said. "But I am very worried about how we are going to do without it."

2019 © The Washington Post

This article was originally published by The Washington Post.