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Toferson
Good morning,
Did anyone participate in the Toferson clinical trial? If so, what has your experience been like so far? Do you believe it has helped or hindered your condition? Did you continue the treatment after the trial was completed?
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16 Replies
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Updated:
I was recently diagnosed, this past Friday, and this treatment was discussed because it targets the specific mutation that runs in my family. We’ve lost 15 family members to ALS, the last being this past February. I also have the mutation.
I’ve been volunteering for medical research since 2010; however when the clinical trials started for Toferson, I did not meet the criteria for the trial. I was asking so that I could gain insight and information to help me make decisions about my future health care.
Toferson was supposed to be reviewed by the FDA in January; however, Biogen submitted additional information to the FDA. The company put out a press release saying the the review date has been extended until the end of April.
I will have to apply for extended access in order to be considered for the treatment. I was hoping that some of our members could provide me with information. I have learned since positing that many times when people participate in clinical trials they are legally obligated not to share or discuss information until the drug has gone through the FDA process.
Amanda
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Amanda
I started as one of the last participants in the “closed” portion of the study and then was allowed on the extended access or open access portion of the study. I just received my 20th dose on Dec. 13th. Myself and my wife (an RN) are very happy with the results of the Tofersen drug. 20 doses ago (one every 4 weeks via Lumbar Puncture) I was unable to lift my right arm above my head and continued to lose strength in my lower extremities. I am very pleased with the results to date and actually started physical therapy at the Washington University Orthwein Center Sept 26th. My results have been good to very good with regaining strength and the ability to perform many more physical things without having to tremendously focus as before the doses. I do things physically without thought just as I did prior to my diagnosis. I am still not walking but continue to gain more and more strength across all body parts. In fact, my breathing capacity test increased to 48% from 38% over a 3 month period and tested on Nov. 13th or so. my 27 year old son who lives out of town is just in awe and what I can do …like transfer from my chair to a PT mat, or from my chair to a toilet/shower chair or even at night when I transfer myself from my chair to my recliner without any assistance from others. I feel stronger than I did 2 years ago. Really I do! I am very pleased with the results and will continue to go to PT 2X per week looking for some more tangible results in the next 3 months…targeting the 6 month mark or so to be able to walk on my own power using a walker to start! Get the drug , I believe it is worth the monthly LP until they come up with a preferred delivery method. Email me with any other thoughts or questions…[email protected]
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Hi my name is Les Wood I was diagnosed in August 2012.i am on the Toferson treatment I am just starting my seventh year on it.I am the first person in the UK to be on the trial,it is now classed as a treatment,I feel the drug has helped me a great deal,I have endured around 75 lumber punctures during this time as of course that is how the drug is administered.there are I believe ten of us on the treatment which is at Sheffield Hallamshire Hospital.the staff there are brilliant and very professional.If any one wants any imformation on tis subject I am only to happy to help all I can
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Les,
Thank you so much for the information. Has it been approved as a treatment there? Tofersen was supposed to be reviewed in January byt the FDA, here in the United States. Biogen provided additional information so now it is expected to be reviewed in late April. my doctor’s have applied for early access to the treatment on my behalf. They are hopeful that will be approved and I could expect to start the treatments in Jan/February.How do you feel the following couple of days after the treatments? Are you getting them every 4 weeks or what is the schedule.
I’m so happy for you and that you feel the treatments are helping you. I had no idea it had even been available anywhere for 7 years!! Any breakthroughs will help the medical professionals figure out how to help all pALS!
Amanda
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Hi Amanda,
I’m also interested in the tofersen treatment. Can I ask you what state you live in? Also where are you being treated at?
Tina
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Tina
Just to assist although the question was to Les…I am in StLouis and being treated at Washington University Medical Center where the creator of the drug Dr. Timothy Miller is the Chair of the department with his PI Dr Robert Bucelli who is Vice Chair!!
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Thank You, John
Sounds awesome what it has done for you.
I am 40 and walking with a cane, I can’t lift up my left arm very high anymore and now it’s starting in my right. I am also doing PT and exercising at home as much as possible.
I talked to my Neurologist and they put me on top of the list for Tofersen. I am just praying I am able to get it.
Thank you for posting!
Tina Johnson
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Tina
Any supplements as I do take (7) to help with body health!
My wife who is an RN-BSN did a bunch of research 2 1/2 years ago when I was diagnosed and found a good amount of info concerning what supplements showed any type of positive or non-harmful results.
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Hi John,
Would you be able to let me know what the supplements you take? Either on here or email?
My email is [email protected]
Thank you!
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Hi Tina,
I live in Florida and I get my treatments at the University of Miami ALS clinic. I live in the Fort Myers area. I just found out that someone that lives in my area also has the same mutation and has started his treatments here in Fort Myers!! What a small world.
The treatments should be available anywhere in the US. If you need names or phone numbers just let me know. Biogen has patient advocates that will help get you set up and help with the insurance approval.
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Amanda
i am so very very sorry that you have been diagnosed with ALS. Please know that I am extremely sad. I have been following your column sincd my husband was diagnosed about 3 years ago.
my father passed from ALS 6 years ago. Not familiar, but super lousy, just the same.
I suggest you contact Ron Hoffman, at CCALS.org. Please do this, to broaden awareness of patient, family, and caregiver care.
you must do as you wish, clinically, but as far as genuine care for you- and, your caregivers, please reach out to CCALS.
They have been the world to my PALS and I.
suzanne -
Hi fellow Toferson friends this is just an update or refresher if you like my name is John Wood though I am known as Les after my middle name Leslie also my father Les .Any way as I said earlier I was diagnosed in August 2012,though I I’m sure I had mild symptoms a few years before that.I am in the U Kand have my treatment still at Sheffield Royal Hallamshire Hospital every 28 days I am obviously the longest Toferson survivor in Britain and quite possibly in Europe due to the fact thr I was the first person in Britain to receive the drug I must have been on Toferson for around 8 years or so now.Like I said some time ago to begin with well certainly when I progressed to the maximum dose of 100 milligrams my health improved quite dramatically Evan appearing on the BBC national news.im now aged 70 and sorry to say that I believe the illness has overtaken the treatment.I can no longer walk,or even stand or weight bare without a wheelchair I am completely bed bound my only outing is my treatment day at the hospital and a occasional day at the horse racing to see one of my horses ( share of )race my wife drives me there and I get transferred to my wheelchair.im sorry to post such a negative review but I can’t see any point in glossing things over.Also no two persons are the same so some may benefit more than others If any of you want more detailed imformation on the Toferson treatment just reply to this message and I will endeavour to get back to you
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Les-Wood,
Thank you for your update, directness and honesty. I have heard there are some situations where a person’s body builds up a resistance to the medication and it no longer has the same impact. I’ve also heard an instance where someone had meningitis and had to stop for 3 months, and then start back up with half a dose to see how their body responded. As with everything, there are major risks. It is great to hear from someone with extended experience on Tofersen. Sharing your information is very helpful, so please continue to do so.
My doctors have always stressed it is a treatment, not a cure. They also stated that people will respond differently based on the SOD1 variant (rapid or slow) and how much damage has already occured. I know here in the US, it is still in clinical trials and was conditionally approved by the FDA. Hopefully they will continue to make strides in the area research for all kinds of ALS.
Les, thank you again for sharing. You really are a wonderful source of information.
Warmly,
Amanda
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Amanda, would you share an update on how the Toferson is helping you so far? We are cheering for you 🙂
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Tofersen update… I was at the University of Miami last Wednesday, Thursday and Friday. I also had my last ALS clinic visit in January with the entire multi disciplinary team. Both appointments brought great news and more hope!
My biggest area of concern is my breathing. In Sept/October I was having difficulting saying a sentence without having to stop and catch my breath. When I was walking I was out of breath long before I was physically tired. My forced vital capacity dropped from the 80s to low 30s in what seemed to be overnight. Perhaps it was over a week or two, but it was quick and scary! In October my FVC was mid 30s, in January 39/40ish. Last week it was 43!! I still cannot walk and talk at the same time without getting out of breath, but I can speak much better and my breathing is less challenging. Also, at my January visit when they did the neuro exam – ALL my scores for muscle strength improved. ALL of them! I’m still not back to my pre-ALS self, and they have told me that will not happen. I’ll never be 100%; and I’m ok with that. I know that being eligible for Tofersen gives me hope and I am grateful for the hope and the improvements. They also said I can expect to see more improvements as time goes on.
Please understand that it is still obvious that I have ALS. I’ve fallen or tipped over a few times at work and needed help getting back on my feet. I’m slow and I don’t do as much public speaking as I use to do. However, I’m over a year into this journey and I’m still working, living independently, and enjoying most things. That’s a win in my book. That’s a win in OUR book!
I’ve had conversations with one of the doctors about my breathing. I was declining so quickly, over 50 points in less than a month. There isn’t enough room to have another drop like that. I’m convinced, and so is my doctor, that had I not started Tofersen in July, I would not have made it past Sept/October. I don’t say that to be morbid or negative; just the opposite. I’m a realist and I was preparing for what could happen. (paperwork, etc) I’m the first case in my family that has had ALS impact their diaphragm this early on. (I’m the 15th or 16th person in my family to have ALS…can’t keep up anymore). Typically, this is the last thing to happen. Let me also add that I am single and have no children. For anyone in my position, I’m sure you can understand the added stress of being in my shoes and the great need to be prepared. Every situation has it’s advantages and disadvantages. I just enjoy the advantages and do my best to prepare for any possibilities in my future.
I focus and try very hard to maintain a positive attitude. I focus on what I can do, and enjoy the memories of what I was lucky enough to do. I will share that when I was diagnosed I did ask for an anti-anxiety med. My doctor prescribed a med that works for both anxiety and depression. I think that it is only natural to experience a wide range of emotions – grief especially when you are facing a life altering diagnosis such as ALS. I’ve never been one to reach for prescription therapy, but I work in the mental health field and I know that when it’s needed, it is a game changer for many. So, I am trying to practice what I preach. This isn’t information I share with people outside of our circle. Mainly because it doesn’t come up. I also use a bipap machine nightly and often during the day when I get home from work. Dr. Granit said that was the best thing I could do to help with my breathing! I have no issues using it because I feel so much better when I do. I get a good night’s sleep – and I think it helps my body to recover.
I had an EMG on Friday and it showed where some nerves were spreading out to compensate for damaged nerves. That was pretty cool to hear. It didn’t seem to show any new damage since last fall from what I understood.
So, overall Tofersen is doing wonders for me. I am so grateful for all of you, the doctors, researchers and volunteers for the clinical trials. We all know that rare diseases do not get the same amount of attention and funding as other diseases. I am one of the few, the fortunate pALS that has the SOD1 mutation (I never thought I’d say that) that makes me a candidate for Tofersen. I don’t take this for granted for a second.
Please know that I am an open book and will happily talk to anyone on our forums or in our ALS Community about Tofersen and my journey. I will continue to advocate and support our community. Know that you can reach out to me with any questions or concerns and I will respond. If you don’t hear from me it is an oversight and just send me a private message.
There is research currently being conducted to find similar treatments for other mutations. I know that the C9orf mutation is a focus for much of this research. The technology is amazing, life altering and brings hope to our community. I believe that we are the generation that will continue to see treatments discovered that will make ALS a livable disease; hopefully a curable disease.
Cheers!
Amanda Sifford
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I received a text from one of my doctors telling me my NfL dropped from 17 to 2. That’s within the normal range for someone without ALS.
Below is information explain the importance and potential of using NfL. The web address for the full article is https://www.als.net/news/neing neurofilament-light-chain/’#:~:text=Because%20NfL%20is%20a%20structural,biomarker%20in%20ALS%20clinical%20trials.
“<strong style=”background-color: var(–bb-content-background-color); font-family: inherit; font-size: inherit; color: var(–bb-body-text-color);”>Neurofilament light chain (NfL) is a member of a family of proteins called neurofilaments that contribute to the structure of neurons in the brain and spinal cord. Research has shown that when neurons experience injury or damage, these proteins are shed and find their way into the <strong style=”background-color: var(–bb-content-background-color); font-family: inherit; font-size: inherit; color: var(–bb-body-text-color);”>cerebrospinal fluid (CSF) and bloodstream. While even healthy neurons shed a certain amount of NfL, elevated levels of the protein in blood or CSF are associated with several conditions that affect the central nervous system.
This has made NfL attractive to researchers as a potential biomarker in several diseases – including amyotrophic lateral sclerosis (ALS). A biomarker is a kind of biological “fingerprint” – something measurable about a living thing that can provide doctors or scientists with information about that organism. Biomarkers have many potential functions in diseases, including:
- Helping doctors diagnose a disease
- Measuring the progression of a disease
- Helping researchers see whether a drug has reached its intended target in the body
- Providing evidence that a drug is changing the course of a disease
Some common biomarkers associated with diseases include blood glucose levels for diabetes or LDL cholesterol for heart disease.
A lack of reliable biomarkers for ALS is one of the biggest challenges in this disease. For example, ALS diagnosis is based on the clinical presentation of symptoms combined with tests like EMGs, while eliminating other possible diagnoses. There is also much interest in developing and using biomarkers as surrogate endpoints in clinical trials. To date, ALS trials have generally relied on measuring a person’s survival, which can lead to lengthy trials, or changes in motor function through the ASLFRS-r survey, which has been criticized for not being sensitive or completely objective. As a surrogate endpoint, a biomarker would be used to show that a drug is having a reasonably likely effect on a disease.
The Potential of NfL in ALS
NfL is often called a nonspecific biomarker because it can increase not only in ALS, but in many other conditions, including traumatic brain injuries, multiple sclerosis, and Alzheimer’s disease. Because of this, NfL has limited potential as a diagnostic biomarker for ALS. However, an observed increase in the level of blood NfL has been found to be a potential indicator of the onset of ALS symptoms for people with ALS-related genetic mutations. It is showing promise as a biomarker for risk of onset of the disease in that subpopulation.
The level of NfL in the blood and CSF at diagnosis has been shown to correlate with the speed and severity of ALS progression, which has indicated that it may be a prognostic biomarker for ALS. While all people with ALS appear to have some degree of elevated NfL in earlier stages of the disease, those with higher NfL levels early on have been found to experience faster disease progression than those with lower NfL levels.
NfL in Clinical Trials
Currently, researchers are also focused on NfL’s potential as a biomarker of drug response in clinical trials. Because NfL is a structural component of neurons that is released when they are damaged, an experimental treatment that decreases levels of NfL in a clinical trial could indicate a possible slowing in neurodegeneration. This means that NfL has potential as a surrogate biomarker in ALS clinical trials.
In one recent case, Qalsody, a treatment for SOD1-related genetic ALS, was granted accelerated approval based on results demonstrating lowered levels of blood NfL in trial participants. A second trial of the drug for asymptomatic carriers of fast progressing SOD1 mutations is currently ongoing. This trial is also following participants’ NfL levels, in this case to determine when to start dosing the drug. A rise in NfL levels in asymptomatic carriers could indicate that the onset of weakness is likely approaching in the next 6-12 months. By administering the drug at this early time, researchers hope to delay the disease onset.
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