A potentially life-saving drug has been on the market for more than five months. But it wasn't until just two weeks ago any Michigan hospitals began administering it.

The disease is Spinal Muscular Atrophy (SMA), a genetic abnormality that results in a steady loss of muscle function and inevitably a dramatically shortened life span.

The drug is Spinraza, approved by the FDA on December 23, 2016.

7 Action News anchor Stephen Clark’s granddaughter Scarlet was the first patient in the world to be prescribed the drug and was given the first spinal injection on January 12, 2017. But that procedure wasn't done at a Michigan hospital. It was done at the Michigan Institute for Neurological Disorders in Farmington.

Since then, seven patients have been treated at the clinic while in the Detroit area. Only one hospital, U of M Mott Children’s Hospital, has offered Spinraza -- and on just one patient on May 31, 2017.

Helen DeVos Children’s Hospital in Grand Rapids is the only other Michigan hospital to administer Spinraza on three patients in the last couple weeks.

Why has it taken Michigan hospitals more than five months to do what a comparatively small clinic managed to accomplish in two weeks?

A director at the Michigan Institute for Neurological Disorders (MIND) doesn’t have an answer for that, but Sonda Rossman knows the hospitals are annoyed the clinic has moved so quickly.

“I think they’re a little bit irritated that we’ve been able to work through the process and get access to the patients and the families so much more quickly.” Rossman says, adding that she’s heard from families that some hospitals are using “deterring methods” such as suggesting if they get treated at the MIND clinic, they won’t be welcome back at the hospital.

Kristy Hetzel’s daughter Jessica went to the MIND clinic after getting such a warning from Beaumont Hospital.

“They want to have it done in the hospital setting and we were told if we started somewhere else we couldn't move somewhere else.” She says, adding that Beaumont does seem to have backed off that statement somewhat.

Still, Beaumont hasn’t begun offering Spinraza causing Hetzel to bring her daughter to the MIND clinic.

“We feel this is our only option and I’m not going to let her sit on the sidelines and wait for someone to say ‘lets go.’”

Jessica Hetzel, at 15 years old, has already lived longer than most SMA patients. In fact, 15 makes her too old to get treatment at U of M Mott, which won’t offer the drug to patients older than 14 despite the FDA’s broad approval for all age groups.

Jessica, a tenth grader and cheerleader at Dakota High, can barely grip her green, blue and white pom poms as she sits on the sideline in her wheelchair.

“Strength. And I’m not going get any worse, I’m only going to get better,” she says of her expectations shortly before her first dose of Spinraza.

The procedure by neuroradiologist Dr. Steve Seidman goes perfectly, despite the presence of titanium rods attached to Jessica’s spine to combat the curvature that comes with SMA.

Jessica is wide-awake and comforted by her family in the room.

Using fluoroscopy, Dr. Seidman guides a tiny needle into the spinal column to extract a small vial of spinal fluid, which he immediately replaces with an equal amount of Spinraza. An hour later, showing no adverse reaction to the drug, Jessica is sent home.

“We have state of the art equipment, we have a neuroradiologist, this is what he does all day, every day.” Sonda Rossman explains. “When we found out what this product could do and how it was administered, it was a no brainer. We would be able to do this and give it to patients…quickly.”

So, is all the fuss over Spinraza worth it? In a word, yes.

One day after her first injection, Jessica Hetzel began moving a foot that hadn’t moved in years.

Stephen Clark’s granddaughter Scarlet, after four treatments, is holding her head up, something she hasn’t been able to do since she was diagnosed with SMA at eight months of age.

She is now kicking legs that have been lifeless and regaining the ability to feed herself.

“I’m amazed at the improvement,” says Dr. Heather Little, a pediatric neurologist at the MIND clinic who has been monitoring Scarlet’s progress. “This is amazing. It's the kind of thing that makes you—it’s why you become a physician in the first place ….to see this kind of benefit from a treatment.”

But that progress for Scarlet and hundreds of SMA patients is in jeopardy.

The state of Michigan last week quietly adopted a policy that will only allow Medicaid coverage for Type 1 - the most severe form of the disease. Most SMA patients, including Scarlet and Jessica Hetzel, are classified as type 2.

Even older patients with type 3 and 4 SMA will benefit from Spinraza according to CureSMA, a national foundation dedicated to funding SMA research and education.

“Unencumbered access to SPINRAZA is critically important for all SMA patients.” Writes CureSMA president Kenneth Hobby in response the Michigan Medicaid’s policy. “For infants with type I SMA, SPINRAZA is essential for their survival; Medicaid coverage and reimbursement of the therapy will have a direct impact on their morbidity and mortality. Individuals with SMA types II and III are also critically dependent patients that require access to SPINRAZA; with beneficial results likely to increase the sooner the drug is administered post-diagnosis. Additionally, type IV patients are likely to see significant health and quality of life benefits from SPINRAZA.”

According to the Michigan Medicaid Board the recommendation to limit coverage came party following discussions from the “major Michigan centers expected to be administering Spinraza.”

In other words, Michigan hospitals, none of which had administered a single dose when the recommendations were made. The MIND clinic in Farmington, which has treated seven patients, was not consulted.

The hospitals also recommended, to ensure patient safety Spinraza only be administered in a hospital setting, That would exclude the MIND clinic, which administered the first commercial dose in America and three-quarters of the doses in Michigan so far..

In a statement to WXYZ.com, MIND’s Sonda Rossman writes: “Restricting patient access to this safe, effective, and potentially lifesaving treatment will only have a negative impact on families affected by SMA.”

After 7 Action News began asking questions about the state’s new Medicaid policy, and the hospital’s recommendation the state’s Medicaid director agreed to consider the MIND clinic’s safety protocols to “ensure that the safety of Medicaid beneficiaries is maintained at the highest level. “

RELATED DOCUMENTS:

From Michigan Medicaid board:

As of last week, Michigan Medicaid is covering Spinraza for individuals with Type 1 Spinal Muscular Atrophy (SMA). Our criteria is based on discussions with pediatric neurologists at the major Michigan centers expected to be administering Spinraza, the manufacturer Biogen, analysis of published clinical trial results and available clinical literature, as well as discussions with other state Medicaid programs and evaluating commercial insurance Spinraza policies. As the clinical literature and guidelines evolve around this new therapy, so too will our prior authorization criteria. Right now our current criteria is based on the best available published research, product information from the manufacturer, and discussions with clinical professionals. As it relates to the MIND clinic and other outpatient settings, because the procedure is invasive and requires post-procedural monitoring, our current criteria only allow this to be administered in a hospital or Ambulatory Surgical Center adjacent to a hospital that has protocols in place to address any complications, which these approved settings do provide.

MIND Statement:

MIND has a long standing commitment to taking care of adult and pediatric patients with all neurological conditions. Patient safety has always been and remains our highest priority. We will continue to provide access for patients to receive any life changing treatment as long as it can be done safely and effectively within our facility. Restricting patient access to this safe, effective, and potentially lifesaving treatment will only have a negative impact on families affected by SMA.

From SPINRAZA Manufacturer BIOGEN:

Based on the robust efficacy and favorable benefit-risk profile demonstrated in the clinical trials, the U.S. Food and Drug Administration approved SPINRAZA for a broad range of patients with spinal muscular atrophy (SMA), including individuals most likely to develop Types 1, 2 and 3. According to the label granted by FDA, “SPINRAZA is administered intrathecally by, or under the direction of, healthcare professionals experienced in performing lumbar punctures.” The U.S. label does not restrict the site of care, and SPINRAZA may be administered in either a hospital or clinic setting. Since the U.S. approval of SPINRAZA, we have not seen any safety issues that would alter its known risk/benefit profile or restrict its use in an outpatient clinical setting. Additionally, we are not aware of any private or public insurer policies in the U.S. that impose additional restrictions on the manner in which SPINRAZA is administered, beyond what is contained within the label. Biogen is committed to developing therapies for diseases with high unmet medical need. We are working closely with physicians, patient advocacy organizations, and regulatory and reimbursement agencies around the world to gain approval and help patients who may benefit from SPINRAZA have access to this important treatment option.

From Kenneth Hobby CureSMA:

While we recognize that there is a need in certain situations to ensure that there is careful post-procedure monitoring following intrathecal delivery of Spinraza, this is related to the specific stability of the patient rather than the actual nature of the route of administration. There are also situations when a SMA patient is more stable where the careful monitoring may not be necessary. As such, we believe that enforcing this as a requirement for all SMA patients will actually limit overall access to this therapy. Allowing for the ability of more stable patients to be seen at additional clinics and outpatient locations will allow for better access for more patients to this important new therapy. In addition, the FDA approved SPINRAZA with a broad label, including in its indications usage for the treatment of all four types of SMA and both pediatric and adult populations, with no restrictions. On behalf of all people born with SMA, we are writing to respectfully request that you provide access to SPINRAZA (nusinersen) for Medicaid beneficiaries with all types of SMA and of all ages. A phase III placebo controlled trial, CHERISH, was conducted for children likely to develop SMA type II and III and it showed SPINRAZA was effective in improving the motor milestone functions of children with later onset SMA, showing an almost 4 point increase on a scale measuring motor function. The children not receiving treatment showed deterioration throughout the trial period in comparison to the children receiving treatment, losing points on the same motor function scale. Unencumbered access to SPINRAZA is critically important for all SMA patients. For infants with type I SMA, SPINRAZA is essential for their survival; Medicaid coverage and reimbursement of the therapy will have a direct impact on their morbidity and mortality. Individuals with SMA types II and III are also critically dependent patients that require access to SPINRAZA, with beneficial results likely to increase the sooner the drug is administered post-diagnosis. Additionally, type IV patients are likely to see significant health and quality of life benefits from SPINRAZA.

Recommendations to State Medicaid Board from Michigan Hospitals: