When Don was first diagnosed, my initial response was that we had a problem and needed to solve it.  We would not passively accept this disease.  As I looked for treatments, I quickly found they fell into three categories:  Treatment to help with symptoms, "snake oil" sold without any scientific backing, and homeopathic remedies.  As I continued to search, I became aware of clinical trials.  Unfortunately, all of clinical trials for Corticobasal Syndrome (CBS) were observational.  At that time, it seemed to me that it was a cruel way to "observe" how people die and how fast they do it.  

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As I continued to dig, I realized Corticobasal Syndrome fell under the category of 4-repeat tauopathies (4-R tau).  There is interest in tauopathies because of the link to Alzheimer's disease.  Another tau disease is Progressive Supranuclear Palsy (PSP).  There were treatment studies for PSP, but they all excluded other tauopathies.  

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It seemed as if there were no options...then, in the Fall of 2018, Dr. Bordelon called and said there was a clinical trial in San Francisco that was a Phase 1B treatment test for safety.  Don is committed to science and thought the trial might help future patients, and he was willing to do it.  I worried that the drug would hasten his death.  We did not anticipate any change in health.

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Basket Trials

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Conventional clinical trials study the effects of a drug on one disease.  "Basket" trials group patients with different forms of a disease that have a common underlying biomarker.  In cancer research, this trial method has produced surprising and unexpected responses in cancers for which the drug was not originally intended.  

 

Dr. Adam Boxer, at the University of California, San Francisco (UCSF), was optimistic that this approach applied to neurodegenerative diseases would provide quicker and more efficient identification of beneficial new treatments.  Dr. Boxer started a TauBasket trial of four rare tauopathies, including CBS and, interestingly, patients with Chronic Traumatic Encephalopathy (CTE), linked to military veterans with blast exposure and/or concussive injury and football head injuries.  The drug that he used was Biogen's BIIB092, Gosuranemab.  The drug is given through an IV (intravenous) infusion every 28 days.  To be in this trial, you needed to have an amyloid PET scan proving you did not have Alzheimer's Disease.

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In January 2019, Don entered the TauBasket clinical trial walking on a cane, with swallowing difficulties, poor cognition, balance issues, in pain, and very stiff.  He was in bad shape.  They have a score to rate your condition using the PSP rating scale.  Unfortunately, I didn't have that done before the trial started, and the score was unavailable to me after the trial began. Nevertheless, the improvement was immediate; the downward progression of the disease didn't just slow; it dramatically reversed.  Within hours, Don could wiggle his neck, catch his backpack mid-air from the Uber driver, and even broke out into a spontaneous "floss" dance on the elevator. By mid-March, he was confident enough in his balance to repair a roof and trim trees using a Sawzall.  We were amazed!  By June 2019, Don was balancing on a hoverboard in the Caribbean of Cozumel.  Dr. Boxer was excited enough to request and was granted an open-label extension. Life was full of hope and dreams!  On December 11, 2019, Don was examined by five neurologists and two neuropsychologists in San Francisco.  They could not believe he was the same man who entered the trial.  Then, on December 13, 2019, our world came crashing down. Biogen had stopped another trial with the drug and would no longer provide it for the TauBasket trial.  We were devastated.  December 11, 2019, was his last infusion under the TauBasket Clinical Trial.  Don's unanticipated success with BIIB092 appears to validate Dr. Boxer's optimism in this trial method for studying neurodegenerative diseases.

 

We know BIIB092, Gosuranemab, benefitted other patients in the trial.  It appeared to benefit those involved in exercises that raised their dopamine levels.  Don has been on Carbidopa/Levodopa since 2016 to help with rigidity.  We believe there is a synergistic connection between Gosuranemab and dopamine.

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Without Medication

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No one knew what changes Don would experience without the medication.  Before we visited with Dr. Bordelon on December 16, 2019, she was on the phone with Biogen to see if he could have continued access.  Biogen emphatically said no.  Optimistically, she hoped he had been on the medication long enough to see continued benefits and scheduled another appointment for March 2020.

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There have been so many twists and turns throughout this process, but one thing I found hard to accept was the lack of scientific curiosity from UCSF.  Not once have we been contacted to see how Don was doing.  I felt like Don was used as a lab rat with complete disregard for him as a person.  Don, forever the scientist, was more forgiving.  Not me; I was angry and frustrated.

 

No longer on the medication, Don began to deteriorate by about 5% per month.  We contacted the FDA about Compassionate Use and were told, "We are currently reviewing the Right to Try Act and working to implement it in a manner consistent with Congressional intent and the FDA's health mission."  No pharmaceutical company is obliged to produce or give away its medication. This makes perfect intellectual sense, but it did not sit well with me as a wife.  

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We have been blessed with many people who have helped us along the way.  One of my former coworkers and friend took on the task of research and letter writing. Another friend developed a web page to tell Don's story.  And yet another family friend, a physician, contacted numerous medical people.  She contacted Dr. Christine Grady, Chief of the Department of Bioethics at the National Institute of Health, who referred us to Dr. Allison Bateman-House, an ethicist with renowned NYU Langon Health.   We even tried the political aspect with Congressman Kevin McCarthy's office.  But, unfortunately, all answers were a resounding "no." 

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It cannot be stressed enough; we were not grasping at straws about a drug we had just heard about. Don had taken BIIB092 for a year under medical supervision with unexpectedly successful results.  

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Single Patient Use

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Finally, we had a phone conversation in June 2020 with Dr. Bordelon and Biogen.  Dr. Bordelon presented her information regarding Don's case, and they said they had already presented 70% of what was said, and the answer was still "no."  I begged them to present the rest of the information they now had, and a week later, Don was granted Single Patient Use. We did not know at the time what swayed their opinion, but I believe it was due to four factors:

One - Don had already participated in a clinical trial with Gosuranemab.  Two - his results were well documented by me in videos and medically by Dr. Bordelon.  Three - UCLA  was one of the sites where Gosuranemab had been used in another trial, and they were equipped to handle the regulatory process needed to reinstate the medication. Four - they were still manufacturing a large quantity of the medication for an Alzheimer's Clinical Trial.

 

We were elated with the news of Don's continued treatment, but like with so many aspects, we were naive to the reality.  There had to be FDA approval, UCLA and Biogen needed to complete contractual paperwork, and the drug had to be manufactured in Italy and shipped to the United States.  The euphoria of July started to turn to panic by mid-August.  Don had lost 40% of his improvement.  He did not have the strength or coordination to do one jumping jack or push-up. Don's pain was returning with a vengeance, and his balance was beginning to be impaired, but Don is a man of science and faith.  He has been on the same medication since 2018; and was not going to take one extra pain pill or change medication.  He was committed to the scientific learning that may take place.  For Don, music and nature are places for his brain to relax.  I created playlists of his Christian music and the sounds of his Minnesota Loons.  I begged Dr. Bordelon and Biogen to hurry up the process. My fear was all-consuming.  I believed he would die before he could get an infusion.

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96 Hours After Don's First Infusion

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Finally, in October 2020, Don received his first infusion under Single Patient Use.  Dr. Bordelon and her team were there to cheer him on.  Fall is the time of year for Don to go to Minnesota and spend time with his extended family, and it is an annual tradition.  He believed this would be his last trip, and 48 hours after his infusion, he was on a plane to Minnesota.  My short observation of Don was that his body was "looser," not so rigid, and he seemed to be feeling better, but I didn't have the time to really "check" him out.  Ninety-six hours after that first infusion, our grandson sent me a video of him peeling potatoes with a kitchen knife.  He had smooth, fluid movement and was chatting away.  I was excited!  Could it be that Gosuranemab would work again?  I called our grandsons and had them do all the tests I had done on Don before the infusion.  I couldn't believe it!!  He could immediately do eight jumping jacks and five push-ups. It was as if jet fuel had been added to his body.  He could go from sitting in a chair to standing up with his arms crossed unassisted.  He could balance on each leg.  His body seemed to crave movement. He swung an axe over his head to cut firewood and spent the afternoon walking on various terrains.  My husband was "back!"

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We had been very transparent about the success Don was having with Gosuranemab.  We had several people who had reached out to us to try and gain access for their loved ones. Unfortunately, it would take us months to realize that they would not be given Single Patient Use because they had not been in the clinical trial. It has been absolutely heartbreaking, so we spent most of 2020 and 2021 sharing only with close friends and family.  

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Dr. Bordelon evaluated Don with the PSP rating scale before his October 2020 infusion. He scored 39.  The higher the number, the worse shape you are in.  I believe he was at a significantly higher number in December 2018 before he started the TauBasket Trial, but that score is unavailable to us.  In neurodegenerative diseases, you progressively get worse.  Success is measured by either a cure (which doesn't exist) or a slower rate of progression.  By January 2021, Dr. Bordelon re-evaluated him with the PSP rating scale, and he had improved to a score of 19, a twenty-point improvement!  This medication not only slows the rate of degeneration but can potentially improve patients! 

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2021 was filled with continued good health.  Every day is not perfect, not for anyone, but overall his strength and coordination remain steady.  Don enjoys being outdoors and challenging himself with all sorts of fun activities, like walking in a slanted fun house, balancing on a boat to go snorkeling, and trekking through the woods.  In 2017, his swallow study showed he had a significant problem and was in danger of aspiration, and the recommendation was plain food with no variety in texture.  Now he eats a variety of foods.  We even had the opportunity to dine in a setting with moving pictures on the table, music, and various food.  He enjoyed every minute without any problems.

 

We have become big fans of Biogen and hope for the best for their drug pipeline. In June 2020, Biogen was granted FDA approval for Aducanumab, a drug that reduces Amyloid-beta in Alzheimer's patients.  We were thrilled and hoped they would combine it with Gosuranemab to reduce the tau protein.  Unfortunately, they did not get the results they wanted in the Alzheimer's trials and ceased production of Gosuranemab.  However, they generously gave Don enough medication to get him through June 2023.

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Veterans and Agent Orange Presumptives

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While looking for clinical trials, I looked into the Veteran's Administration (VA).  They appeared to have Parkinson's as agent orange presumptive with research centers, but Don's claim was denied because Corticobasal Syndrome is a Parkinsonism.  In June of 2021, we received a letter from the VA stating they were adding Parkinsonism to the agent orange presumptive list.  We appealed his case under Public Law (P.L.) 116-283, the William M. Thornberry National Defense Authorization Act.  We were hopeful this would allow Veterans to receive treatment with Gosuranemab, and we waited for one full year to hear back from the Veterans Administration.  

 

We know that no pharmaceutical company will make a medication for one patient, and I don't see how you entice them to manufacture it for a rare disease.  Still, we thought the Veterans Administration might be able to help. Just as we were beginning our summer of 2022, I received a letter from the VA denying his claim because he was stationed in Udorn, Thailand, and not Vietnam. Don and I have a great marriage, and I believe part of the reason is that we don't keep secrets from each other, but I did not have the heart to tell him this news.  I was devastated and resolved to make 2022 an amazing summer and ignore the dark cloud hanging over our heads.

 

 June 2022 was our summer to celebrate Don's good health with each of his children and our grandchildren.  Every moment was planned with activities to fill a lifetime of memories.  He took his daughters on a personal history tour of his beloved Minnesota.  The youngest grandson was allowed to go to the deer cabin in the northern woods and spent a week fishing with all the "men folk" and enjoying all their goofy antics.  We took two granddaughters to Florida, where we taught one to snorkel and let them swim with the dolphins.  Three grandchildren got to go to our favorite vacation spot - Cozumel Island.  We spent nine full days in the ocean snorkeling, swimming, deep-sea fishing, and walking in the humidity.  We took a kazillion pictures and videos filled with happy memories.

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PACT-Act

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August 2022 was the first I heard of the PACT-Act.  With a large bipartisan majority, Congress passed, and the President signed the PACT Act, a new law that expands VA health care and benefits to veterans exposed to toxic substances.  One of the sections of the Act, Title V, provides funding for research.

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One of the initial signs of agent orange exposure was cancer, and it took years for neurodegenerative diseases to develop.  We believe there is the same potential with veterans exposed to burn pits.  We also know many of our current veterans suffer from traumatic brain injury due to blast injuries and concussions, which will lead to neurodegenerative disorders.  

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We want to emphasize that Don has had no ill or adverse effects from Gosuranemab, only therapeutic benefits.  Don's success in a cutting-edge clinical trial exemplifies American ingenuity.  Gosuranemab, as an essential component of a care plan for Parkinsonism, has enhanced Don's quality of life and could be valuable to other veterans.  Unfortunately, our Vietnam-era veterans have been vilified and ignored for far too long.  It is time to rectify this situation.

 

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Our Call to Action

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We are reaching out to veterans, veteran organizations, neurologists and other physicians, organizations that advocate for patients with neurodegenerative diseases, Biogen researchers, Biogen's team of veterans, and policymakers to put Gosuranemab back into production and explore this medication fully.

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Veterans have a long history on the battlefield with the Soldier's Code, "Until they are home, NO MAN LEFT BEHIND."  We believe this applies to the health of our surviving Warriors and Veterans, and Gosuranemab, with a comprehensive care plan, is the best current option for our veterans battling Corticobasal Syndrome and possibly CTE and other neurodegenerative disorders.

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