The world’s first patient to receive Moderna’s mRNA genetic disease therapy

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The world’s first patient to receive Moderna’s mRNA genetic disease therapy

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This 20-year-old girl became the world’s first patient to receive Moderna’s mRNA genetic disease therapy.

Scientists discovered and recognized the role of mRNA as early as 1960, and since then, mRNA has been studied and planned for use in vaccines or disease treatments.

But until the end of 2020, two mRNA COVID-19 vaccines were approved for marketing. In the past 60 years, most people have never heard of the concept of mRNA vaccines or mRNA drugs.

Before the COVID-19 pandemic, Moderna had been investigating the use of mRNA for the treatment of glycogen storage disease type Ia ( GSD-Ia) , a severe genetic disorder of metabolism in which patients due to homozygous mutations in the G6PC gene lead to glucose- 6-phosphatase is inactivated, so the liver cannot decompose glycogen into glucose, resulting in hypoglycemia in the body.

In addition, it will be accompanied by hepatomegaly, acidosis, hyperlipidemia, hyperuricemia, hyperlactatemia, coagulation A series of clinical symptoms such as dysfunction and developmental delay are life-threatening.

One such unfortunate individual was Loveah Hernandez of Dallas, Texas , who was diagnosed with glycogen storage disease type Ia ( GSD-Ia) in the hospital at the age of 6 months when she suddenly developed unresponsive symptoms .

Since then, her life has been difficult in order to survive – a feeding tube is inserted from her nose to her stomach so that she can get the blood sugar level she needs by naso-feeding cornstarch, which is able to build up in the intestines.

Long stay and slow release glucose. At age 3, the tube was removed and replaced with a gastrostomy tube through which cornstarch was supplemented for nutrition.

Like other patients with glycogen storage disease type Ia ( GSD-Ia) , she requires regular cornstarch supplementation every 3-4 hours to stay alive.

This meant she would never be able to sleep even a single night, and her mother had to go to school on time every day to ensure her study and safety.

Because of this rare disease, she was unable to participate in the school’s group activities, and her classmates did not dare to call her to participate in gatherings. Hospitalization was a common occurrence due to physical reasons.

Still, Loveah Hernandez did well, graduating high school in fourth place, and now at 20, she’s studying biology in college and plans to become a physician assistant after graduation.

Loveah Hernandez

After learning that Moderna was launching a clinical trial of an mRNA therapy for GSD-Ia, Loveah Hernandez has worked hard to become a trial participant and has spent the past two years making sure she is eligible to participate in the clinical trial.

The new therapy, code-named mRNA-3745, is designed to determine the safety of mRNA in patients with GSD-Ia and whether it may help correct the defect that causes GSD-Ia and restore normal function in patients.

If the trial is successful, mRNA-3745 will help liver cells effectively break down glycogen, improve symptoms of hypoglycemia, and eliminate the need for patients to regularly consume starch.

Preparations for this clinical trial between Moderna and UConn Children’s Hospital began four years ago and will officially launch in July 2021.

After a year of planning and preparation, on June 1, 2022, Loveah Hernandez became the first patient in the world to receive mRNA therapy for glycogen storage disease type Ia (GSD-Ia) , which she received through an intravenous infusion in her arm.

Lipid nanoparticle (LNP) delivery of G6PC gene mRNA. ” Being the first to receive this investigational treatment was really cool, ” says Loveah Hernandez .

A nurse is measuring blood pressure for Loveah Hernandez (right)

Juan C. Salazar , chief physician at the University of Connecticut Children’s Hospital, who is in charge of the clinical trial, said that this clinical trial is exciting and historic , and the mRNA COVID-19 vaccine has made us realize the effectiveness of mRNA.

And now we’re using the same technology platform to treat a completely different disease, which could be a life-changing treatment for children and their families around the world a feat.

Finding a suitable patient is difficult because many are reluctant to be the first to receive this new treatment, said Shaylee King , director of pediatric clinical research at the University of Connecticut Children’s Hospital .

As the first patient to participate in this clinical trial, Loveah Hernandez ‘s selflessness and bravery provide a new treatment option for other glycogen storage disease type Ia (GSD-Ia) ,

The Phase 1 clinical trial is designed to determine whether the investigational mRNA-3745 therapy is safe and well tolerated to create the patient’s missing liver in the patient’s liver, said endocrinologist Karen J. G6PC enzyme, and its ability to maintain blood sugar levels during fasting.

The goal is to enable patients to improve their quality of life by eliminating the need for regular cornstarch intake.

References :
https://today.uconn.edu/2022/06/breaking-barriers-the-first-patient-in-the-world-receives-moderna-mrna-trial-infusion-for-glycogen-storage-disease-at- uconn-health/

The world’s first patient to receive Moderna’s mRNA genetic disease therapy

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