As part of the GM1 & GM2 Tanganil Health Initiative, TREND analyzed the conversations shared in a private Facebook group created as an open forum for parents of GM1 and GM2 afflicted children to share their ideas, thoughts, concerns, and results of Tanganil. Following is our report.
Prepared by Sarah Inoue, PhD
This report is a first look at the information shared on the Facebook group: Tanganil for Tay-Sachs and Sandhoff created as an open forum for parents of GM1 and GM2 afflicted children to share their ideas, thoughts, concerns, and results of Tanganil. It summarizes the conversations that that took place on the Facebook group over the last year (starting in October 2017). The group has 131 members as of September 14, 2018. These members posted 74 times since October 2017 with 525 comments on those posts. Most of the information in this report comes from the comments on the posts. 200 of those comments were kind words to the original poster or someone else commenting, and the rest of the posts and comments had something to say about Tanganil or the disease burden of the person/patient posting.
The report will start with the good news and a list of all of the improvements noted when taking Tanganil. Many people reported improvements, and they may have reported the improvements more than once. Because we looked at anonymized data, we don’t know if the reports are repetitions or new reports, so the number of improvements is not as important as the list of all the improvements noted. We will also note the few times that people were not happy with Tanganil, and their reasons for that.
The main question that came up over and over was about dosing, so after listing the improvements, we will report on the discussions of dosing.
We will then give the information we have on the age of patients, length of time on Tanganil, the diseases mentioned, and other medications taken.
Sadly, three comments or posts mentioned that a child had died. Our hearts go out to the families of those lost, and we hope that this information collected will help in the search for treatments and a cure for these diseases.
The researchers who prepared this report are not doctors, are not providing medical advice and are only reporting what was said in the conversations online.
IMPROVEMENTS WITH TANGANIL
This is a list of the improvements noted while taking the drug. We have tried to classify them based on age. Some of the improvements are clearly observed in infants and some are clearly in older people. In general, the main improvement seems to be better physical strength and quality of life. Balance and smiling/giggling were mentioned frequently when discussing improvements.
- Alertness 8
- Laugh (giggling) 5
- Smile 4
- Interacting 2
- Pointing 2
- Swallowing 2
- Delaying progression 2
- Physically active 1
- Looking around 1
- Stronger 1
- Eating Baby food 1
- Wiggly 1
- Trying to lift head 1
- Screaming lungs out 1
- Excited 1
- Sleeping better 1
- Stretch more 1
- Cooing 1
- Increased motor stability 1
Juvenile and LOTS:
- Quality of life back 1
- More stable 1
- Increased strength 1
- Coordination of legs 1
- Improvement with writing 1
- Increase in coordination and balance 1
- Change own shirt 1
- Decreased toe-walking 2
- Improvement in Speech 2
- Increased Motor stability 1
- Speech and handwriting improved 1
- Balance improved 1
- Reduced nystagmus 1
- Alert and happier 1
- Balance and stability better 1
Increased hand tremors were mentioned twice. On Tanganil, the trembling in someone or two people’s hands increased to the point where they had to stop taking the medicine and were scared to try it again. Another person remarked that there was no improvement with Tanganil.
All of the improvements were noted after people began taking the drug, but we have little information about how long they were taking the drug when they noticed the improvements. Three people noted that it took a month for improvements to be seen, others mentioned almost instantaneous improvements. 18 people mentioned the length of time they had been on the drug, although we suspect that one of those people was repeating the information, as time passed. People mentioned taking the drug from 2 weeks to 16 months.
Tanganil is not yet approved for these diseases, and no scientific research has determined the correct dose yet. Thus, the question of what the right dose is and what dose will be the most effective is an open one with no clear answer.
One of the questions that also came up around dosing was the issue of taking a break from the medicine or not taking it for a period of time before resuming. This question arose nine times. One person described clearly taking a break every 6-8 weeks. In five months of taking the drug, she or he stopped twice and found this the most effective for improvement of symptoms. Others mentioned not taking a break and finding this the most effective. We have no evidence either way, but we do note that this is an area for further research.
One question asked specifically about a relapse when taking a break from Tanganil. One person mentioned that as far as she knew everyone who paused in taking Tanganil relapsed. This type of break seemed to be longer than the two weeks of a dose break, but it’s not completely clear from the conversation.
Much information was shared about the patients and people in the group. This is a summary of that information.
Not everyone in the group mentioned the disease they have or are caring for, but we found mention of a disease thirty times. Some people may have been repeating information, but because the data is anonymized, we don’t know if they are. This is just to give some sense of the diseases in this community and under discussion.
- LOTS 9
- GM1 8
- Infant Tay-Sachs 7
- GM2 4
- Juvenile Tay-Sachs 1
- Juvenile Sandhoff 1
Age of Patient
Again, not everyone mentioned the age of the patient or their own age. People also grew older during the time frame that we were listening to the conversation. Age was mentioned 24 times. 9 months old and 10 months old had the most mentions (seven combined mentions). Two infants were 11 months and 15 months. Six were two years to 2.5 years old. Two were 3.5 – 4 years old. Then, they stepped up in age 8 years, 9 years, 14 years, 16 years old, and finished with 2-3 people who are 29 years old.
A few people mentioned other medications that are taken to relieve symptoms. Those included four mentions of Zavesca, and one each of Omaprazola, Ayurvedic, Keppra, Onfi, Cannabis oil, Antibiotics, Cord Blood Transplant, Gene Therapy, and Infergen.
A number of conversations mentioned the need for more studies of Tanganil. Everyone, as mentioned earlier, wants to know the best dose of the drug for the maximum effect. A few times, the group had the chance to talk to doctors or researchers and this usually was appreciated. Posts about these conversations received many comments (26 in one case).
Become a Citizen Scientist by sharing your experiences with Tanganil on TREND Community. We will help you turn your anecdotes into evidence that can benefit your entire community.
How it works:
- Join the GM1 & GM2 Tanganil Health Initiative on the TREND Community platform (participation is free). Go to www.trend.community, scroll to the bottom and request an invite.
- After receiving the email invite, follow the link to set up an account.
- Sign an electronic informed consent agreeing to share your data in order to participate.
- Join an exclusive and private discussion group within TREND Community for the GM1 & GM2 Tanganil Health Initiative participants to share and support.
- Answer regular survey questions.
- Keep an online journal. You can review these entries over time to discover your own trends.
- Receive TREND Community updates and reports on the Health Initiative progress and trends.
- Share with family members, your medical team and researchers to help provide both you and your child with a potentially better quality of life.