NGF Blog


What People With Gaucher Disease Should Know About COVID-19 Vaccines

As of early October 2020, the world has been grappling with the novel coronavirus disease 2019 (COVID-19) pandemic for about eight months. Globally, more than 35 million cases of the coronavirus have resulted in more than 1 million deaths.

In the current wave, 99% of cases are mild, while 1% of people with COVID-19 are seriously or critically ill. For people with Gaucher disease, the outcomes appear to be the same, if not better.

We spoke to two experts in Gaucher disease, vaccines, and the immune system for insights into how COVID-19, and possible vaccines, affect people living with Gaucher disease.

  • Pramod K. Mistry, MD, PhD, FRCP, is the director of the National Gaucher Disease Treatment Center and professor of pediatrics and medicine at Yale School of Medicine. A board-certified internal medicine doctor, he is also a medical advisory board member of the National Gaucher Foundation. He is a member and board member of several groups studying Gaucher disease and liver function in the U.S. and globally.
  • Maricar Malinis, MD, FACP, FIDSA, FAST, is an associate professor of medicine and the medical director, Transplant and Oncology Infectious Diseases, Section of Infectious Diseases, at Yale School of Medicine. Her research focuses on clinical outcomes of transplantation in older adults and people living with HIV. She is the primary investigator of the HOPE Act study at Yale and several multicenter clinical trials of novel antiviral agents for immunocompromised hosts.

COVID-19 and Lysosomal Disorders Like Gaucher Disease

Lysosomal storage disorders, like Gaucher disease, occur when people lack the enzymes that break down specific lipids (fats) or carbohydrates (sugars) in cells.

“The lysosome is also important in how COVID-19 engages cells in the body,” explains Dr. Mistry. It works like this:

  • When lysosomes build up in Gaucher disease, the body tries to get rid of them. One mechanism is to convert them into a lysolipid, a more soluble form, so that the body can remove it from the cells.
  • A lysolipid can activate two types of immune cells, particularly T cells and B cells.
  • Some of these immune cells produce substances in the blood called cytokines.
  • In people living with Gaucher disease, their cytokine level tells healthcare providers about their level of inflammation.

“We found an impressive similarity in the level of elevated cytokines in people with Gaucher disease and people with severe COVID-19,” continues Dr. Mistry. Early on, “we were scared that COVID-19 might cause a major health crisis in our Gaucher disease population.”

How Does COVID-19 Affect People With Gaucher Disease?

The crisis Dr. Mistry initially feared has not materialized. Instead, he says, “COVID-19 doesn’t seem to affect patients with Gaucher disease any differently than the general population.”

In fact, people living with Gaucher disease may be less likely to suffer severe effects from a COVID-19 infection.

Most people with Gaucher disease who contract COVID-19 have a flu-like infection and then recover. That may be because people with Gaucher disease often lack secondary risk factors, such as diabetes, obesity, or fatty liver disease, that increase the chances of poor outcomes from the coronavirus.

Some doctors and researchers are exploring whether Gaucher disease might even blunt the effect of a COVID-19 infection. Researchers are studying two possible reasons:

  1. A sluggish lysosomal system (due to low enzyme levels) may stunt the coronavirus so that it can’t complete its full life cycle.
  2. With Gaucher disease, the immune system continuously stimulates B cells. Perhaps these active B lymphocytes can quickly make effective antibodies when they encounter a viral infection (like COVID-19).

What Are the Coronavirus Vaccine Candidates?

As of October 2020, researchers were developing eight vaccines for SARS-CoV-2—the virus that causes COVID-19—through Operation Warp Speed (OWS). OWS is a partnership among several government entities and the private sector to speed the process of safely bringing a vaccine to the public. The vaccine or vaccines that will most likely be available in the U.S. are probably among these eight.

“These SARS-CoV2 vaccines focus on developing an immune response to the virus’ spike protein,” says Dr. Malinis. “The spike protein of COVID-19 allows viral attachment to a receptor found in the respiratory tract and in many of our organs and facilitates viral entry to the cell.”

All vaccines work to help the body build an immune response to a specific virus. To fight COVID-19, researchers are exploring three different vaccine types:

  • Virus-like vaccine: Uses an inactivated version of the SARS-CoV-2 virus
  • Viral vector vaccine: Uses a deactivated virus (such as an adenovirus, the common cold) as a Trojan horse to carry the COVID-19 virus protein into the body
  • mRNA vaccine: Encodes parts of the virus’s genetic material into human cells

[h2] Promising Vaccine News for People With Gaucher Disease

One of the vaccine candidates, from Moderna, is an mRNA vaccine. Researchers recently completed a small study on this vaccine in adult patients older than 55 years old.

“The encouraging result of this study is that there is a good immune response, and it’s well tolerated by older adults,” Dr. Malinis says.

“That’s important for the Gaucher patient population because about a third of people affected with Gaucher disease in the U.S. are older than 55 to 65 years old,” adds Dr. Mistry. “In the general population, age is a risk factor for the most severe type of COVID-19 infection.”

In other words, we have evidence that a vaccine can be effective not only in younger people but also in older adults. That’s good news for everyone, and it applies equally to people living with Gaucher disease.

How Are the Coronavirus Vaccines Tested?

There is great urgency to come up with a vaccine, given the severity of COVID-19. Yet researchers follow stringent protocols to ensure the vaccines are safe, Drs. Malinis and Mistry emphasize.

Vaccine development follows an established process of testing, approval, and validation. This process considers the importance of protecting the population, including vulnerable people.

“The FDA [Food and Drug Administration] has established this process to protect both safety and efficacy, and that process has not changed over time,” Dr. Mistry says. “The process ensures the public that scientists are studying these vaccines carefully and making sure that the benefits outweigh the risks.”

FDA vaccine testing includes three phases. These phases are the same for any FDA-approved medication or clinical treatment, including enzyme therapy and gene therapy for Gaucher disease:

  • Phase 1: Evaluating the drug’s safety in a small, healthy group
  • Phase 2: Exploring effectiveness in larger cohorts, including activating different doses to decide on the appropriate dose
  • Phase 3: Testing the vaccine in a much bigger population to assess safety rigorously

Will the Coronavirus Vaccine Be Safe for People With Gaucher Disease?

The short answer: Yes. Drs. Malinis and Mistry expect any FDA-approved vaccine for COVID-19 to be as safe for people with Gaucher disease as for anyone else.

“In terms of general vaccinations, we don’t regard Gaucher disease patients as immunosuppressed,” adds Dr. Mistry. “We encourage people with Gaucher disease to receive all regular vaccines.”

Dr. Malinis agrees, noting that anyone with Gaucher disease can receive any approved vaccine. She adds, “I know we are looking forward to having a COVID-19 vaccine soon. But we are also heading into flu season. Flu and COVID-19 are both respiratory viral infections that are difficult to distinguish from each other. People with Gaucher disease—and everyone—should definitely get the influenza vaccine this year.”

Doctors recommend the flu vaccine for everyone. Additionally, people who are close to someone with a possibly compromised immune system should be especially sure to get the flu vaccine, which is available now.

How Does Precision Medicine Play Into Coronavirus Vaccine Development?

“Precision medicine is a big topic in the Gaucher disease community,” says Dr. Mistry, who has led podcasts on the subject for NGF. “That’s in part because the disease can vary enormously across the patient population, from a child who is very sick to a 90-year-old with hardly any signs of the condition.

“Still, at present, we see no evidence that people with even the most severe Gaucher disease are at higher risk of SARS-CoV-2 infection, and that’s borne out globally,” Dr. Mistry says. “Even in places like Egypt, where children with Gaucher disease are so, so sick, our colleagues have not had a single person perish from COVID-19 infection.”

The results are similar in Spain and Italy, which COVID-19 has hit very hard. In those countries, people have Gaucher disease that is generally much more severe than what we see in the U.S., says Dr. Mistry.

“People in the U.S. should not be scared that if you have severe Gaucher disease, you have more risk of a bad COVID-19 infection. The take-home message is that COVID-19 appears to be no worse in Gaucher patients than in any others.”

As a result, Dr. Mistry sees no reason to justify undertaking an expensive immunological investigation to explore an individual’s COVID-19 risk based on precision medicine. “People with Gaucher disease should not get anxious that they need expensive immune profiling. It hasn’t been shown to be necessary,” he adds.

How Can People With Gaucher Disease Protect Themselves Against COVID-19?

Take steps to protect yourself and your loved ones from COVID-19. The most important are what Dr. Malinis calls the “pillars of fighting COVID-19”:

  • Maintain social distancing.
  • Wear a mask when you’re around other people, especially indoors.
  • Maintain good handwashing practices.
  • Get your flu shot and the COVID-19 vaccine, when it’s available.

You can also boost your immune system naturally or learn more about coronavirus tips for Gaucher patients.


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