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published on September 19, 2017 - 3:47 PM
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A new medication is being used on muscular dystrophy patients in the Central Valley and despite its high cost, its prospects are promising.

Approved last December by the US Food and Drug Administration to combat spinal muscular atrophy (SMA), the drug, Spinraza, was first administered by Dr. Terry Hutchinson at Community Regional Medical Center shortly after. Renee De La Rosa, family support and clinical care coordinator for the Muscular Dystrophy Association, said that the treatment was the result of years of work, which included research funding from their organization.

“MDA was very excited to hear about it. I think we’re just thrilled that the treatment is available to our SMA families,” De La Rosa said. “And I think it kind of just… showed all the hard work and dedication that MDA has done.”

Spinraza has also been given to eight patients at Valley Children’s Hospital since June, with five more awaiting approval. Dr. Raymund David, the director of the Muscular Dystrophy Clinic at Valley Children’s Hospital, stated that the drug has been able to greatly reduce the progression of the disease and improve the lives of patients.

“They’re not able to sit, they were not able walk,” Dr. David said, “but when they received this medication, they were able to reach some of these milestones.”

Spinal muscular atrophy is a disease that causes the loss of motor neurons, the cells in the spinal cord that affect the muscles and glands. This causes the progressive degeneration of muscles in the patient. This include respiratory muscles, which can lead to the necessity of a ventilator. There are four types of spinal muscular atrophy with SMA1 (also known as Werdnig-Hoffman disease) being the most severe, appearing during infancy.

“The problem is that there is a lack of proteins in the motor neurons,” Dr. David said. “The Spinraza acts like a patch and you are able to produce a protein, so there’s not a complete degeneration of the motor neurons.”

Developed by Biogen, Inc., Spinraza is given to patients via injection into the spinal canal (an intrathecal injection). First, patients receive four loading doses — heightened doses of the drug — which they receive every two weeks, with the fourth administered 30 days after the third. After this, the patients will be given a dose of the drug once every four months for the rest of their lives.

There are, however, some side effects to be expected with Spinraza. According to RxList.com, an online pharmaceutical database, common problems include respiratory infections, constipation, teething, congestion, ear infections and scoliosis.

There are also financial difficulties that come with the treatment. Dr. David stated that the drug is priced at $125,000 for one vial, and that a total cost of $750,000 can be expected for the first year of treatment.

The reason for the high cost, De La Rosa said, is because SMA is a rare disease.

Nonetheless, Dr. David expressed that the benefits to Spinraza outweigh the costs. The mother of one patient at Valley Children’s Hospital, age 9, reported that he has been able to pull up his own pants and get into the car, things which he was previously unable to. However, Dr. David cautioned that this patient is still in the loading stage of treatment, and these milestones could very well be the result of a placebo effect.

De La Rosa witnessed the treatment of another patient in April. Coming to Fresno from Hawaii, she was given Spinraza at Community Regional Medical Center.

“Just last night, the mom posted a video of the girl that I got to see get the treatment done, and she was never able to pick up a cup of water,” De La Rosa said. “And last night at dinner, she was able to pick up the cup of water and give the water to herself.”

De La Rosa stated that Spinraza is reason to believe that more breakthroughs in fighting muscular dystrophy are on the way as well.

“I think after that got approved, it definitely blew the fire underneath some people. We have been able to get two medications for Duchenne muscular dystrophy approved, and we had another medication for ALS — also known as Lou Gehrig’s disease — that also got approved by the FDA,” she said. “So I think that in the last five years, we’ve had more treatments be approved by the FDA than over the last 50 years.”


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