Robin, a member of the Myasthenia Gravis Association, participated in the Cartesian Car-T clinical trial over a year ago and is currently being reinfused after 17 months of positive results. Her experience provides a detailed look into the challenges and triumphs faced by MG patients seeking new treatment avenues. This article captures Robin's journey, from her decision to participate in the trial to the impact it has had on her life.
Discovery and Motivation
Robin discovered the Car-T clinical trial through an email the same week that she received a call for a cancellation of her upcoming neuromuscular follow up. She was told that her beloved neurologist of 14 years had passed away. While on the call, she recalled seeing the email and decided to take a second look at it. The clarity and comprehensiveness of the informational video attached to the email prompted her to consider this new direction.
Robin weighed the pros and cons of participating in a clinical trial, because trials do come with their risks. The severe complications with her existing treatments were a strong persuader in her decision and motivation to participate. Robin was stable with IVIG and other medications but faced challenges such as loss of vein access, incorrect port placement, clotting issues, among other problems caused by repeated biweekly infusions. Her journey from 2010 to 2023 was marked by setbacks, including nonmelanoma skin cancer from Imuran and vascular disease. After her port failed and she had to undergo numerous angioplasty and stenting, she urgently needed an alternative. The trial offered a potential lifeline.
Enrolling in the Trial
Robin described the team as incredibly supportive and professional, ensuring she was well-informed and comfortable throughout the process. She does note that there have been some updates as the trial has progressed. For example, she is required to be within 15 miles of the clinical trial site for 24 hours after receiving the Car-T infusion. That was not required during the first round of infusions. Also, the initial enrollment included the chance of receiving a placebo. The reinfusion stage is being done as an open label, which means no chance of placebo.
The enrollment process was thorough and manageable but has changed since she first applied. During the enrollment experience, Robin applied to all available clinical trial sites, ranging from Washington, California, Florida, among other sites. She ensured all her medical records were provided and met the trial's criteria. After a comprehensive physical exam and blood work in South Florida, she qualified and was one of three participants at that site. Currently there is a central enrollment process with Cartesian directly, eliminating the need to apply to multiple sites.
Treatment Experience
The process starts with collecting your blood cells via apheresis. They are then sent to the lab and re engineered to fight the MG antibodies. Once the cells were ready, Robin received one dose a week for six weeks. The infusion process involved 15 minutes of treatment, followed by 1 hour of observation before heading home. The consent form Robin signed prior to participating divulged numerous possible side effects, mostly flu like symptoms , such as fever, chills and nausea. Participants had 24/7 contact with the clinic and specific instructions for emergencies. She was to report any abnormal or unexpected symptoms to her care team via a 24-hour phone number. Robin compared the experience to what she imagines chemotherapy is like, with a range of side effects.
Robin experienced nausea, aches, fever and chills. The third hour post-infusion was particularly challenging, with severe chills and pain moving through different parts of her body. She described it as if the treatment was traveling through her body, fighting against her cells at different body parts and when finished with one fight, they moved to another part of the body to fight. Despite the discomfort, she was able to manage these side effects with Tylenol and Benadryl. The side effects mostly subsided within 4-6 hours.
Health and Symptoms
Robin's MG-ADL score improved dramatically from 12 to 1, indicating a significant positive response. While receiving treatment in the trial and the following months, Robin faced severe symptoms not related to the clinical trial. The symptoms presented to doctors as an allergic reaction, but after listening to her own body and knowing that she was not having an allergic reaction she was finally diagnosed with a bacterial infection in her port. This would be triggered any time her port was accessed and used for various treatments. Although she saw great results from the Car-T therapy, she imagines that she would have had more positive and longer lasting results without having to fight a bacterial infection. The trial allowed her to avoid IVIG for 17 months, relying only on Imuran and Mestinon as needed. Despite the bacterial infection potentially impacting results, she experienced notable improvement.
Robin was able to engage in social activities and travel after participating in the Car-T Trial. Her life no longer revolves around treatments, giving her a sense of normalcy and independence. This newfound freedom of not being confined to her treatment schedule was liberating. In addition to the side effects of IVIG, which left Robin with more bad days than good, she expressed that she would rather have a few weeks of bad from the Car-T therapy, in return for a solid year without repeated treatments. She is excited for what this next round will bring, hoping she will feel comfortable enough to plan a trip to Europe, which she had long ago given up on.
Monitoring and Follow-up
Follow-ups were meticulously scheduled at the same time each day. There were seven follow-ups, starting with the first three months of weekly appointments. Then they began to space out every two weeks, four weeks, eight weeks, her last appointment being at the one year mark. . Each follow-up appointment included regular blood work, physical exams, and evaluations of her MG symptoms. This information was collected and examined to monitor her progress and any side effects. Some preliminary results to this part of the trial have recently been published by the sponsor.
Challenges and Support
The primary challenge was the initial severe side effects and travel to and from the study site. During the immediate side effects post-treatment the trial coordinator and doctor provided support and guidance on managing these symptoms. The clinical trial coordinator was always available, offering support and resources as needed. Additionally, Robin connected with other participants for mutual support. Interacting with fellow participants provided Robin with a sense of community and shared experience, along with some symptom management tips, which was invaluable.
Overall Impression and Future Considerations
Robin is incredibly pleased with her progress. The improvement in her quality of life has been remarkable and she would recommend considering the trial to others that are also comfortable in taking that risk. Despite the challenges, the potential benefits far outweigh the difficulties, offering a chance at better management of MG symptoms.
She hopes to continue seeing improvements and exceeds her current level of symptom management. Robin's primary hope is that the trial results lead to broader availability of effective treatments for MG. She is optimistic about future advancements and improvements in managing this condition.
Conclusion
Robin's participation in the Cartesian clinical trial has been transformative. From overcoming severe medical challenges to achieving significant symptom relief, her journey offers renewed hope and a path toward a better quality of life for MG patients. For those considering this trial, Robin's experience underscores the potential benefits and the importance of exploring new treatment options
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