Happy new year, friends! First off, thank you to everyone for your messages of love and support for Tyson, myself, and the boys that continue to pour in. Your words of encouragement truly lift us up and we feel so lucky to have all of you in our corner. This one took me a long time to write and I apologize for the wait. I have quite honestly been visualizing writing this particular entry for over two years. You’ll see why in just a moment.
Let me take you back briefly to June of 2015. We had recently returned home from a six week hospital stay where Tyson focused solely on recovering from a cardiac arrest he had suffered in late February of that year. The six weeks were meant for Tyson and I to both adjust, each in our own roles, to the new norm. Tyson was in critical but stable condition. His heart had stopped for 35 minutes as a result of respiratory failure due to muscle weakness. I had held his hand as he went into cardiac arrest in the ER and watched as a team of doctors worked to revive him. In the midst of it all, the CPR, the multiple chest compressions, et cetera, Tyson fought to stay. And even when they miraculously found his heartbeat, the doctors did not expect him to make it through the night let alone the weekend. But he did. They had no explanation as for why or how he survived. On top of that, the doctors had all but guaranteed that Tyson’s vital organs would be damaged due to his heart having stopped for over 30 minutes. Subsequent testing showed that this, in fact, did not happen! While in the ICU, some doctors gently encouraged me to place Tyson in hospice and/or palliative care. I fought this because I knew what Tyson and the stem cells were capable of. I knew Tyson just needed some help. I knew he had survived because of his incredible will, a WILL that was so strong, that it carried him through when he was unconscious and near death. I thought it was evident that the stem cells had played an extremely significant role, at the very least in protecting his heart, brain, and lungs. (For more on this, please see the March 2, 2015 entry.)
Once Tyson stabilized, he and I knew he needed more stem cell treatment. It was a non-negotiable for his recovery. In fact, we knew he needed the treatment months before his cardiac arrest and, if we were being honest, we knew that moving to India and committing to on-going stem cell therapy was what he truly needed. It was clear that the stem cells in combination with the Lyme antibiotics, physical therapy, and proper nutrition were what fueled his remarkable recovery at the start. Looking back, what contributed to his rapid decline midway through was the swiftness of the progression of the ALS symptoms caused by the Lyme bacteria that made it difficult for him to breathe coupled with not being able to address it exactly when we needed to. Regardless of that (and believe me, I am haunted by this every day…wishing we had found a way to pack up our lives and move to India) we knew we couldn’t dwell on it. We had to move forward. We knew we had to get back to India but now we would be attempting a 15 hour flight as a patient in critical condition with a full dependency to a ventilator. Not only would this be incredibly cost prohibitive, but Tyson and I were deeply concerned that he would not survive the flight.
I had stayed in touch with Dr. Shroff, our incredible doctor in India, and in typical fashion, she remained a constant source of support and encouragement during some of our darkest days. I emailed her one afternoon in June of 2015 and explained that I was extremely stressed trying to figure out just how I was going to get Tyson back to India for treatment. She responded by saying, “Give me a call as soon as you can, I have an idea!”. I called her right away. After asking how Tyson was doing, she said, “I’ve just discovered a program by the FDA called ‘Expanded Access’. It would allow us to import the stem cells to the U.S. for Tyson to continue his treatment!” I stammered in disbelief and said, “Oh my goodness! Are you serious? How? What do we need to do?” She said, “Find a doctor who would be willing to help you with the application as a sponsor and we’ll go from there”. Once we finished up our conversation, I ran to tell Tyson and then immediately emailed his primary care physician, Dr. Murphy. I asked if he would be willing to help and without hesitation, he said he would be happy to.
The next step was to research and familiarize myself with the details of FDA’s Expanded Access program so I could figure out how to get this done. According to the program description on the FDA’s website, Expanded Access, or Compassionate Use, is the use of an investigational drug, biologic, or device outside of a clinical trial; one that has not been approved by the FDA. This program is designed for patients with serious or life-threatnening illnesses to have access to treatment from anywhere in the world when they’ve exhausted all other alternatives here in the U.S. and are not eligible or don’t qualify for a clinical trial. The FDA must determine that there is sufficient evidence to support both the safety and efficacy of the proposed treatment and that the protocol/treatment plan is consistent with the FDA’s regulations. Additionally, it must be clear that the potential benefits of the treatment outweigh risks of the treatment and those risks of treatment are reasonable in the context of the disease or condition being treated.
The more I read, the more confident I felt about receiving an approval for our request. It was clear that Tyson was an excellent candidate. He fit all of their criteria. So, we began the application process right away. Bear in mind, this FDA program is rarely used, and Dr. Murphy had never gone through the process so we were learning as we went along. To put it lightly, the application process was a bit more complicated than what we had originally thought and when we inevitably hit stumbling blocks and had questions along the way, there really was no where to turn to for help. Naturally there were forms to be filled out, letters to be written and so forth, but we also learned through trial and error on our part, that we would ultimately be required to draft a treatment plan. To start, we had applied for an “emergency” approval because we felt he should be considered an emergency, as he was listed in critical but stable condition. All I knew was that when I looked at Tyson, the only thing I could think of was getting him help immediately. However, despite that, after several weeks, the FDA informed us that our application had been denied. They did not consider ours to be an emergency case. My guess is that it had something to do with him not being hospitalized at the time. This was upsetting, but of course our option at this point was to simply submit a “regular” individual patient expanded access request. We learned from FDA that with our request, we would be required to first get an approval from an IRB, or Institutional Review Board. The IRB’s role would be to review our application, focusing on the protection and welfare of the patient. If we could get the approval of the IRB, the FDA would then likely approve.
We tried to use a local IRB and after ELEVEN!! months of having our application, they informed us in May of 2017 that they would not be able to help us and that we should seek the assistance of an IRB that had a background in stem cell research. They also told us that our application wasn’t in shape to be reviewed but didn’t go so far as to indicate what was missing, and from here, we weren’t sure where to turn. This was extremely devastating. We had worked so hard for this approval and without it, we knew we wouldn’t be able to move forward with the FDA. Keep in mind, too, that all the while, Tyson was home fighting to remain stable and hang onto whatever bits of strength he had left and despite my strong sense of urgency to get this treatment for him, others did not operate with the same drive which was extremely challenging to accept. For the next week or so, I researched IRB’s in the U.S. that had some experience with stem cell projects. I contacted a university nearby and kindly asked if they would be willing to lend their expertise to help us tighten up our proposal and/or accept our application for review. They declined both requests and to top it off, told me it was highly unlikely we’d be able to get anyone to approve this. We had been through almost two years of all sorts of denials, misleading paths, dead-ends, and endless frustration in our attempts to get this treatment approved, so hearing this at this stage of the game didn’t shake us. It was irritating and upsetting, but I KNEW beyond a shadow of a doubt that Tyson was deserving of this treatment and that if given a chance to review, the FDA would be inclined to agree.
So, one morning as I sat at my desk trying to research a way through all of this, I began a general search for independent IRB’s in the United States. There are hundreds of IRB’s all over the country at various universities and hospitals. I figured we would be able to find ONE who could help us. On a whim, I typed “independent IRB and Expanded Access” and right away found a website for the WCG Foundation (www.wcgfoundation.org). A snapshot of their company’s description said that part of their mission was to help patients and physicians navigate procedural obstacles in the Expanded Access process. I laughed to myself because if there were two words I’d choose to describe our struggles of the past year, it would be “procedural obstacles”. I was a little nervous and intimidated to get on the phone with them because I was afraid I wouldn’t be able to sufficiently explain what we needed. But I tossed my fear aside and gave them a call. The woman who answered my call was named Dr. Marjorie Speers. She could not have been sweeter or shown more compassion as she listened to a three minute synopsis of what Tyson had been through followed by an explanation what I was trying to get approved for him through Expanded Access. She told me that they collaborated with an independent IRB in the state of Washington and that she would contact them once we got off the phone. She also said I should expect to hear from the IRB by the end of the day. I could hardly believe my ears.
I want to stop right here for a moment and point out that along the way in this nearly two year endeavor, we met people who were truly interested and enthusiastic to help and we also met others who weren’t. It felt like for the past year and a half that I was begging people to hear our story and to help us get Tyson the treatment he needed in order for him to have any chance at surviving and for the most part, it was all falling on deaf ears. We had tremendous support from our friends at the Global Lyme Alliance when we needed to connect with Senator Blumenthal’s office to request his assistance. As a result, this conversation I had with his associates lead to the start of a dialogue with the FDA which we so desperately needed at the time. And the women I spoke with at the FDA were kind, compassionate, and helpful. But beyond that, we had very little support and endless roadblocks along the way, so to have met this wonderful woman at this incredible foundation at such a critical point in our journey was an enormous blessing. It was evident that she cared so very much for Tyson and ensuring that he get the treatment he needed. She became my new power partner 🙂
So sure enough, I received a phone call from the WIRB and spoke with another amazing woman who asked me a series of questions about Tyson, his medical history, and our journey thus far. I remember she said to me, “Of course I cannot guarantee anything but based on what I’m hearing from you, we should have no problem getting this approved for you.” Choked up, I thanked her. She went on to explain the next steps regarding how we needed to submit our application to them (our application included several different files and documents) and by the end of the call, I had set her up with Dr. Murphy’s office to continue the conversation. We had been waiting nearly a year for a response from the previous IRB and here we were moments away from having our application reviewed by one of the top IRB’s in the country. And, on May 31, 2017, TEN DAYS after our initial conversation, we learned that the IRB had APPROVED our application!
There was plenty to celebrate at that point, although I knew we were only half way there. We still needed the FDA’s approval but we reminded ourselves that we were told that with an IRB approval, the FDA would likely approve, so we were still feeling very confident. We handed everything over to the FDA in the beginning of June, including the approval letter from the IRB. They sought clarification on several points of our proposal and requested multiple adjustments to our documents, which Dr. Shroff addressed accordingly, but at the end of four weeks of review, the FDA contacted Tyson’s physician and indicated their intention to deny approval.
Dr. Shroff and I were absolutely floored. I was angry and in utter disbelief. This was the hardest blow for us. I called Dr. Speers at the WCG Foundation and told her the news. She immediately shared in our frustration and said, “We will figure this out.” And that’s exactly what we set out to do. We couldn’t understand why he would be denied access to a treatment that he already underwent several times with proven efficacy. It didn’t make sense. The FDA informed Tyson’s physician that they would be sending a letter explaining their reasoning within 30 days. So in the meantime, with advice from Dr. Speers, I began researching alternatives, e.g., possibly traveling to Canada or Mexico to see if either of those countries would allow him access to the treatment, contacting any and all government officials we could, utilizing the Right to Try law that had recently been passed in Connecticut, just to name a few. We didn’t come up with anything really substantial and by the 30 day mark, the FDA had reached out to our doctor. They requested that we address the small changes they highlighted in our proposed treatment plan and then informed him that we could proceed! WE DID IT! It was officially approved on August 15th when we received the IND number (official proof of approval). I am so grateful to FDA for reconsidering Tyson’s case and ultimately approving the expanded access request. It was always our intention to seek treatment with FDA approval and not find a way around FDA.
Dr. Shroff and I spoke just about every day during these couple of months of agony and consistently throughout the two year period. I am so grateful to her for her encouragement, her confidence, and her strength, all of which had a ripple effect down to me and Tyson. She helped us keep our faith and press on even when we were at our breaking point. And so, when we got the approval, she called me up right away and we basically screamed on the phone to each other with incredible excitement and relief!!
The next step was to figure out the logistics of securely shipping the cells from India to the United States. Of course this part wasn’t easy either and we ran into several roadblocks along the way but despite all of that, we were finally able to have them shipped here toward the end of October. I am elated to tell you that we have BEGUN treating Tyson with the stem cells as of the start of November. Tyson is already feeling some slight relief and improvement in his eyes; his vision is less blurry and his eyes feel stronger, his leg muscles are stronger, and he is beginning to breathe over the ventilator slightly. We know that stem cells take a long time to regenerate and repair the damage in his body and we are working hard at remaining focused on our daily tasks and staying positive.
Gratitude is something that is talked about nearly every day in our house because we know we just have so much to be thankful for and because we need to be able to counter the seemingly unavoidable and overwhelming feelings of sadness and frustration that we’re faced with every single day. When I look at Tyson, I am so upset to see him so tired and weak and I am furious that despite my efforts, I wasn’t able to get him help sooner. This process to get the stem cells here to the U.S. didn’t need to take this long but we can’t control all of that. We are acutely aware that all that matters right now is what’s in front of us which is a long road to recovery. Here’s where the gratitude kicks in–we know we are fortunate to even be in this fight. We now have access to the very thing that fueled his recovery and allowed him to make such tremendous strides in his improvement five years ago and we made history doing it! Tyson is the first patient in the United States to receive these types of stem cells and the magnitude of that is not lost on us. We are hopeful that one day these stem cells will be available to patients all over the United States who are suffering as Tyson is. ❤ We are all exhausted, but we know the most important thing is that he now has the greatest opportunity to continue on his road to recovery and we will make the MOST of this moment.
As you know by now, the physical therapy aspect of his treatment has ALWAYS been a fundamental component as it has helped him to remain stable and avoid things like pneumonia and blood clots which can be fatal for an individual in Tyson’s condition. Now that we finally have the stem cells back at center stage of his treatment plan, it is critical that he increase his physical therapy sessions in order to maximize the productivity of the stem cells. The cells, in a sense, need to be shown where to go and that is done in large part by the physical therapy. Many of you have been asking what you can do to help and we hope you know how grateful we are for your generous support over the years. What we need the most right now is help with physical therapy costs. If you feel inclined to share Tyson’s story and to give toward his rehabilitation, know that you will be making a lasting impact on Tyson’s life.
Thank you to all of you for your thoughts and prayers. This is undoubtedly a difficult journey for us but your friendship and endless support makes it an easier one. ❤