New FDA-Approved Medication Delays Type 1 Diabetes
A preventative therapy for type 1 diabetes
called Teplizumab-mzwv recently passed FDA approval and is already on
the shelves. It is the first medication capable of delaying the
progression of the disease. Listed below are the key facts about the new
medication and the way it can benefit type 1 diabetes patients.
How is type 1 diabetes unique?
Diabetes is an umbrella term for three separate conditions:
Type 1 diabetes - a hereditary autoimmune condition
Type 2 diabetes - the most widespread, may be acquired or inherited
Gestational diabetes - diabetes that appears in pregnancy.
Of these three conditions, type 1 diabetes is the most difficult to
treat. While gestational and type 2 diabetes can often be managed with
changes in diet, exercise, and a variety of medications, the only
available treatment option for type 1 diabetes is insulin.
In type 1 diabetes, the immune system
attacks and destroys the cells in the pancreas responsible for producing
insulin called beta cells. As a result, the pancreas is able to produce
less and less insulin, which makes blood glucose levels rise. Insulin
is a hormone produced in the pancreas that allows glucose to enter the
cells and be utilized as energy. When the levels of insulin drop,
glucose levels skyrocket and eventually damage the body.
Type 1 diabetes is usually diagnosed in children and young adults. A
person is at higher risk for type 1 diabetes if they have a relative
with type 1 diabetes. This is precisely the group of
patients Teplizumab-mzwv can benefit.
How does Teplizumab-mzwv work?
Teplizumab-mzwv is the first medication capable of delaying the
progression of type 1 diabetes. The treatment was approved by the US
Food and Drug Administration (FDA) on November 17, 2022, under the brand
name Tzield.
Tzield is a synthetic protein that works
like a monoclonal antibody produced by the immune system. This is a type
of antibody that instructs immune T cells to ignore the beta cells of
the pancreas - states ProventionBio, the manufacturer of
Teplizumab-mzwv. In addition, the medication urges the immune system to
produce more immune cells that protect the beta cells from T cells.
Who can benefit from Teplizumab-mzwv?
The treatment is designed to slow down the progression of the disease.
Therefore, it is beneficial for patients prior to the 3rd stage. Type 1
diabetes is formally divided into three stages that reflect the extent
of damage to the beta cells and symptoms.
Stage 1 is asymptomatic. It is characterized by elevated levels of
antibodies that attack the pancreas, but blood sugar levels remain
normal. Most often, it is diagnosed in children whose parents suffer
from diabetes, and it can last for years.
Stage 2 is also asymptomatic, but the
patient’s glucose levels start to rise, suggesting a higher degree of
beta cell damage. Stage 2 can last from months to years.
Stage 3 is the last. Patients notice symptoms like blurry vision,
increased thirst and urination, fatigue, and weight loss. The pancreas
can no longer produce enough insulin to sustain the body, and the
patient requires supplemental insulin.
Teplizumab-mzwv is approved for use in all patients with stage 2 type 1
diabetes who are eight or more years old. The medication was shown to
delay the advance to stage 3 for more than two years compared to a
placebo in a clinical trial. There was some variability among
participants, meaning some patients developed stage 3 later and others
sooner.
"The drug's potential to delay clinical diagnosis of type 1 diabetes may
provide patients with months to years without the burdens of disease,"
said Dr. John Sharretts of the FDA Center for Drug Evaluation and
Research in an official press release of the FDA.
The Medical Trial
Seventy-six participants aged 8-49 years
old were involved in the trial. All had stage 2 type 1 diabetes.
Forty-four of the participants received IV infusions of teplizumab-mzwv
for 14 consecutive days, and the remaining participants got placebo
infusions.
After four years and three months, 45% of patients who received
teplizumab-mzwv were diagnosed with stage 3 compared to 72% of the
placebo group. On average, placebo patients developed stage 3 type 1
diabetes twice as fast as those who got the treatment.
Common side effects of teplizumab-mzwv
A rash and headaches are the most common side effects of
teplizumab-mzwv. Patients who undergo the immunotherapy may be more
susceptible to infections because teplizumab-mzwv can temporarily lower
the body’s immune protection.
Some patients in the trial developed a temporary decrease in some immune
cells. According to ProventionBio, the number of these immune cells
started recovering a week after the treatment and fully bounced back a
couple of months later.
The manufacturer also cautions that
patients who take teplizumab-mzwv should be monitored by a professional
due to more serious potential side effects like vomiting or angioedema
(swelling under the skin). Sometimes, teplizumab-mzwv triggers something
called cytokine release syndrome - an acute syndrome in which T cells
cause a systemic inflammatory response that comes with a fever and
multiple organ dysfunction.
How much does teplizumab-mzwv cost?
The USA Today reports that a single vial of Teplizumab-mzwv costs
$13,850, so the full 14-day treatment is worth $193,000. This is a steep
price tag, but many people now have the potential to delay the disease
and the burden it creates every day, and for them, teplizumab-mzwv is a
life-changer.