Aztec native shares $3 million science prize

• The drug Bennett helped develop is used to treat spinal muscular atrophy.
• SMA involves the loss of neurons in the spinal cord that control muscle movement, including breathing and swallowing.
• The condition eventually leads to paralysis and is often fatal.

FARMINGTON — An Aztec native has received a prestigious award for his work in developing the only Federal Drug Administration-approved treatment for a condition that is the leading genetic cause of death in infants younger than 2.

Pharmacologist Dr. C. Frank Bennett and his collaborator, Dr. Adrian Krainer, were awarded the 2019 Breakthrough Prize in Life Science. The two will be sharing the $3 million prize.

The award comes after the two scientists helped develop a drug, Nusinersen, which is marketed as Spinraza. The drug is used to treat spinal muscular atrophy, also known as SMA.

SMA involves the loss of neurons in the spinal cord that control muscle movement, including breathing and swallowing. The condition eventually leads to paralysis and is often fatal. Bennett said it occurs when a child does not inherit a certain gene from his or her parents.

The gene that is not inherited is necessary for the production of survival of motor neuron protein, Bennett explained. This protein is essential for the neurons that control muscle movement.

According to Cure SMA, about one in every 11,000 infants is born with spinal muscular atrophy and an estimated one in every 50 Americans is a genetic carrier for the condition. Symptoms can begin in infancy or later on in life, including in adulthood.

If symptoms appear when the patient is an infant, it is usually fatal.

Bennett’s team worked using antisense technology to develop a drug that is injected into the fluid surrounding the brain to treat the condition.

“We’ve been interested in applying the technology in treatment for a variety of diseases,” he said.

Antisense drugs involve injecting small pieces of synthetic genetic material into the patient. The genetic material then binds to the ribonucleic acid, better known as RNA. The purpose of the of the genetic material is to fix certain errors in genes. RNA is responsible for the production of proteins like SMN.

In 2004, Bennett began working to develop the antisense drug that would increase SMN protein production and keep the motor neurons healthy.

The work was partially funded by a grant from Cure SMA. Between 2003 and 2006, Cure SMA provided more than $500,000 to help develop the treatment.

It took more than a decade of work before the drug received its approval.  

“It wasn’t one discovery,” Bennett said. “It was actually a series of novel discoveries that kept me going.”

On Dec. 23, 2016 — the Friday before Christmas — Bennett and his team got a call at 4 p.m. from the FDA telling them their product had been approved to treat spinal muscular atrophy.

Bennett said the treatment not only has the ability to stop the impacts of the disease, it also can help patients regain functions they’ve lost or never possessed. If the treatment is given to infants within a few weeks of their birth, Bennett said it has a profound effect.

Cure SMA has developed a map with which patients can find sites that administer Spinraza in the United States. The map lists the University of New Mexico Hospital in Albuquerque as a site that is administering or preparing to administer Spinraza to patients of all ages.

Bennett said he was surprised when he received the news that he had won the prize. He accepted it during a ceremony earlier this month, which can be viewed on National Geographic's YouTube channel.

Bennett graduated from Aztec High School in 1974 and from UNM in 1980. He later earned a doctorate from Baylor University. Bennett still has family members who live in Aztec.

Bennett credits his mentors in high school and at UNM for helping inspire him to pursue his interest in science. He now works as senior vice president of research for Ionis Pharmaceuticals, which focuses on drugs targeting RNA. Bennett cofounded Ionis Pharmaceuticals, which is based in California.

Bennett grew up at Miss Gail’s Inn, which his family owned, in downtown Aztec. At the time, the business was known as the Aztec Hotel. He became interested in a science career while exploring the hills of northwest New Mexico.

“My natural curiosity sort of led me down the path to a science career,” he said when reached by phone Wednesday.

His mentor at UNM steered him toward a career in pharmacology, which interested him because it combined pharmacy and chemistry.

He encouraged young people to follow their dreams and passions.

“If you are passionate about what you work on, then you’ll be successful,” he said.

Hannah Grover covers government for The Daily Times. She can be reached at 505-564-4652 or via email at hgrover@daily-times.com.