HSP clinical trial program update September 2018

Posted - September 2018 in HSPRF News

Progress in clinical trial planning and preclinical studies

Prof Carolyn Sue

Clinical Trial

A detailed plan and budget for an HSP clinical drug trial has now been developed by members of the clinical trial team over the past quarter. It was a substantial undertaking with multiple sources of input, review and revision, with 10 drafts in all being produced along the way.

Thanks is due to the dedicated team of investigators, especially Prof Carolyn Sue who made an enormous contribution while shouldering a staggering workload.

When funded, the trial will span three years and has a budget of $1.5 million. The challenge now is to explore additional avenues for funding to make this happen.

Dr Gautam Wali

Blood based biomarker for HSP

The goal of this study headed up by long-term HSP Research Program and clinical trial team member, Dr. Gautam Wali, is to identify a blood based biomarker that:

* reflects disease-specific defects, and

* can be used to quantify the effects of drug treatment.

Dr Wali reports that collecting has now begun with an intake of four HSPers having gone through the process and given blood, and with one of their number so far being assessed on the Spastic Paraplegia Rating Scale (SPRS). The bulk of the past three months has been spent operationalising the plan for this study, which required significant amounts of planning and organising between the three units involved – the HSP clinic, the pathology services and the research laboratory.

A strict protocol had to be developed that effectively integrated these three separate functions and insured interchangeability and repeatability with possible changes in personnel. Standard forms had to be modified and customised; personnel to manage and implement the different parts of the study had to be selected and communication and coordination activities developed and refined.

Dr Wali has also approached the University of Technology Sydney to explore and negotiate accessing a high throughput microscope with which to help perform the laboratory analyses. This has been a multistep process and appears set for a positive resolution in the near future. It is expected that the first round of sample collections from HSPers will be completed on schedule by the end of the calendar year.

Associate Prof Michelle Hill

Drug dose modelling study

This study will measure drug concentrations in the blood, brain and spinal cord of mice at different drug dosage levels. The aim is to learn more about various factors that affect the calculation of the drug dose needed to achieve target levels of the active ingredient in the neurons of HSPers in clinical trials.

The drug dosage experiments with the animals were completed in early May with blood, brain and spinal cord tissues collected and preserved for analysis. Around the same time, work began in the laboratory that will perform the analyses, to develop and validate measurement techniques and methodologies.

Associate Professor Michelle Hill of the Precision & Systems Biomedicine laboratory at QIMR Berghofer Medical Research Institute in Brisbane heads up this study. She recently reported that the study is on track with progress being made in developing the test and measures to be used in the tissue analysis. Completion date for the development and validation of the test is October, and for the analyses, December.

Comments on this story

Nasser posted at 8:45 pm on 2 September 2018Reply

Hello

I am an HSPer for about 4 years now. According to my own experience, mental issues such as positive thinking, happiness and good mood have a direct effect on HSP severity. Also crying has the same effect (recently I lost my grandpa and my limping got so much better, but it is temporary).

I think this information may lead to a new idea or something 😉

thank you all doctors who put their time and knowledge to solve our problem.

Nasser
Jay posted at 1:44 am on 3 September 2018Reply

Hello, I’m an HSP patient from China. I’m concerned that the drug has already started clinical trials. Would you like to consult if there is any chance for the experiment to be carried out in China?

China has a large population, so rare diseases are not uncommon. There are a lot of HSP patients around, who are eager to be treated. How can I keep in touch with you at any time? 😛 😛 😛
claire posted at 5:50 am on 3 September 2018Reply

I just don’t know what to say … Im over the moon!! This is fantastic news and the progress you have made is such good news … Our family has HSP (male side) and I hope this will bring positive results for those who have this nasty diagnosis. Such great news for my 11 year old son – your updates are something new I can relay back to our UK Consultants.
As you know – Clinical Trials.. we are more than happy to help at that particular phase as Im sure lots of other people will.
Best of luck for the future and cannot wait to read the next stage to the progress that has been made.
Milind posted at 4:02 pm on 3 September 2018Reply

Dear editor, is the drug used in clinical trial noscapine? Or will there be other brain chemicals with it?
And when the drug will be given to people to try, that is, when will the clinical trials start?
- Editor posted at 4:00 pm on 5 September 2018Reply
  
  Editor’s Note: The drug candidate to be tested in the clinical trial is Noscapine. It will not be combined with any other active compounds. The clinical trials can start as soon as there is firstly funding and secondly regulatory approval.
Jeannette posted at 1:33 am on 5 September 2018Reply

Could I be a part of the clinical trials? Would they take info from Canada? There are Canadian universities in Australia could we communicate through them?
- Editor posted at 4:06 pm on 5 September 2018Reply
  
  Editor’s Note: The initial clinical trial is planned for Australia only. Multiple clinical visits are necessary for participants and it is therefore impractical to have people in the trial not within reasonable ground transport distance of the trial site. It is possible that a subsequent Phase II clinical trial may be held in Canada, especially as there is already a large register of people with HSP through the CANHSP national program based at McGill University in Montréal.
Denis posted at 5:34 pm on 19 September 2018Reply

and the epothilone will no longer be used?
- Editor posted at 10:01 pm on 19 September 2018Reply
  
  Editor’s Note: Only one drug can be tested in the clinical trial because HSP is a rare disease and it will be challenging enough to scientifically prove or disprove the effectiveness of one drug with the limited number of eligible participants. Noscapine was chosen because it is no longer covered by patent and therefore readily available, relatively cheap and equally effective.
Chris posted at 11:58 pm on 19 September 2018Reply

I am a 45-year-old male living with spg4 late onset around 40. I started taking Noscapine a total of 90mg per day, in 3 batches of 30mg, spaced out around 4 hours apart (Half-Life). I have been taking now for 15 days. This pill and the dosage is making a big change in the way I walk and tension and pain in my feet and legs. This may be the answer to all our prayers. I was wondering the dosage that was going to use in the study? I had heard of people taking 400mg and 600mg per day as well.
- Editor posted at 11:27 am on 20 September 2018Reply
  
  Editor’s Note: The question of dosage/dose range to be used in the clinical trial is being decided in a year-long study using mice. This dose modelling study is due for completion by the end of calendar 2018. Until the data from that study is available, the required dosage and dose frequency is just guesswork.
- Denis posted at 2:38 pm on 20 September 2018Reply
  
  Hello Chris. where do you buy noscapine and in what form? is this cough remedy? I’m from Russia and found so far only the Japanese cough remedy, which includes noscapine. I ordered and wait. I do not know how legal noscapine is in Russia.
  - ben posted at 6:40 am on 21 September 2018
    
    Chris ~ this is so amazing to hear! Those of us HSPers would love to hear about how you were able to get this medication. Is it being prescribed by a medical doctor? Why 90mg a day with 30 mg 3x a day?
    
    I have so many more questions but do not want to overwhelm. Just looking for some hope!~!
  - John posted at 1:58 pm on 21 September 2018
    
    Noscapine has been sold as an effective cough suppressant since 1940, now sold in 20 countries. Russia, Australia, and USA are NOT on the list. I know all 20 countries. It is sold either as a low dose (15mg – 50mg) tablet or in liquid form (makes your tired). Noscapine has also been tried for many years in larger doses for certain types of cancer (including prostate and colon). Although Noscapine is not yet medically approved for cancer treatment, many people report great things like lowering PSA, killing cancer cells, and stopping good cells in the body from dying or getting sicker. This last quality of Noscapine is what led to testing it in the lab to stop the progression of HSP. It appears that Noscapine can improve the transmission of proteins through the nerve axon fibers connected to your muscles, leading to slightly improved mobility and balance. Since the half life of Noscapine is 3-4 hours, this will be tested in the upcoming clinical trial, as well as the dose. It may be more beneficial to take it more than one per day for this reason.
  - Chris posted at 3:37 am on 24 September 2018
    
    I have a friend in Holland that mails in tablets. 15mg tablets, Roter is the Manufacturer.
    
    If you find a place to purchase please share it with me. My friend feels uncomfortable mailing these pills.
    
    Thanks
    Chris
  - Denis posted at 3:14 pm on 24 September 2018
    
    John, In which countries are you allowed to sell noscapine? You can reply to me on email
Denis posted at 3:04 am on 20 September 2018Reply

both of these substances have been known for a long time. and about their medicinal properties. but we still do not accept it. this is somewhat absurd. but then there are some problems associated with this. This is money? or nothing can be done about this and it remains only to wait? I just want to be sure that I did my best. it is necessary to begin the clinical trials.
Tara posted at 12:42 pm on 20 September 2018Reply

I am also from Canada and would love to participate in this trial!
Ashraf posted at 5:43 am on 23 September 2018Reply

I am from india , where can i get noscapine.
Please reply
Editor posted at 2:03 pm on 24 September 2018Reply

Editor’s Note: Caution is urged in self-medicating. A clinical trial is the only way that the questions around dose and effectiveness can be answered. For people thinking about participating in the clinical trials, any trace of the candidate drug found during pre-testing would disqualify people from participating in the trials.
Denis posted at 2:51 pm on 24 September 2018Reply

People from Russia cannot think about clinical trials. So we can test it on ourselves. But it is so difficult to find noscapine.
Milind posted at 10:10 pm on 24 September 2018Reply

Dear Editor, is the noscapine for a cure or for just to stop progression.
Secondly I Know dose studying is going on, but in cancer a dose up to 1 to 3000 mg. is given, so in the case of hsp axon generation, will the dose be more than that or would it be less than 1000 mg? I am just asking as per dose research study done until now? And as per cell study, how much longer will it take to show an effect on good mobility???
- Editor posted at 9:13 am on 25 September 2018Reply
  
  Editor’s Note: The potential effects of the drug in humans is not known. The possibilities are – no effect; slow down progression; stop progression; or restore functionality to a greater or lesser extent. The appropriate dose level is not known at this point. Hopefully the results of the dose modelling study indicate the dosing range for the clinical trial. Your third question about how long it may take to positively impact mobility is also unknown. If the drug can rescue neurons from axonal degeneration and stimulate axon regrowth, this could positively impact mobility, however the timeframe is unknown – it may well be some years as axons grow slowly – and it may well be that musculoskeletal retraining or rehabilitation is also required to fully realise the potential of restored function in the neurons.
Debbie posted at 10:26 am on 25 September 2018Reply

will i qualify for trial, live in florida have been diagnosed with hsp as a teenager, i’m now in my 50’s and use wheelchair
Alivia posted at 10:49 am on 25 September 2018Reply

Hello!!
The HSP community has been waiting for someone to help!
My daughter is 5 and was diagnosed 3 weeks ago. Age three it was CP then finally the diagnosis was HSP4. Diagnosed at UCSF Benioff California. I’m thankful for trials for all HSP patients alike. I would travel the world in a heartbeat to make something help her and many more……find a cure.
All my thanks to all for the hard work the team has persevered and dedicated to HSP!
Thank you,
Allies biggest fan – her mama
Brock posted at 3:11 pm on 25 September 2018Reply

My name is Brock and I live in Indiana. I was born with HSP. My dad has it too. Runs in males, but no one else in the family has been affected going back multiple generations, even though everyone carries the recessive gene and could easily pass it on to their kids.

Not gonna lie, I need this. I went to a really small school where I got bullied nonstop and had no friends. I wasn’t even invited to my high school reunion, even though I graduated. there were 62 of us in my graduating class. It’s not gonna be fun when you’re the guy who can’t walk. I got told from a young age that I was a loser and that I would die a virgin or that I would never have a good career even with a P.h.d. because you can’t respect a “cripple “. I got told no one would ever marry me because they wouldn’t want their kids to have my condition. And i guess i slowly starting believing it until it broke me down and made me hollow inside.

People made fun of me in class, in the cafeteria, in the neighborhood, etc. You would think it would stop when we grow up and become adults, but it actually got worse.

Now I have serious mental health issues that I’ve numbed out with alcohol and even alot of illegal drugs. I’m sober now, for 37 days but it’s hard when you feel like a freak and like you don’t matter. i’ve been to rehab 18 times no joke. I had over a year clean and then my girlfriend left me. And i used again. I guess i wonder if maybe it was horrible trying to sleep with someone who can barely use their legs or maybe she was embarrassed by me, i don’t know. It just crushed me. It was like the final nail in the coffin.

I’ve tried to kill myself numerous times. I’ve gone through big rehab and other government programs, but no one wants to hire the guy in the wheelchair, so I live on disability and I’ve been homeless multiple times.

I need this so bad I don’t know if I can live without it. I don’t wanna be Quasi Modo anymore. I just want to be me and have a life and feel like I belong somewhere. I need this so bad. I’d sell my soul for it, but sometimes I don’t even believe in God anymore.

I check this page all the time. I really hope this is it. Thank you for all that you’ve done already. Sometimes that gives me a little bit of hope that things will change. I really do come from a place of unconditional love, even if I don’t always get it back. No matter what from now on I’m gonna take the high road and be a lighthouse for other people caught out in the storm. I have faith things are gonna get better.

Sincerely,

Brock
Patrice posted at 4:20 pm on 26 September 2018Reply

Hello to you all from the Netherlands. My husband (50years old) was diagnosed HSP9. They have told us he is the only or the 2nd HSP9’r in the Netherlands.
I would like to ask the research team if they know Noscapine will be positive for HSP9 patients. Will the reseach triall be done with different patients like HSP4 HSP9 HSP11 etc.
- Editor posted at 6:01 pm on 26 September 2018Reply
  
  Editor’s Note: The clinical trial will be restricted to SPG4 HSP in order to have sufficient statistical power to produce a definitive result on the question of the effectiveness of the candidate drug. The level of effectiveness is neither known nor predictable for any form of HSP at this point. This is why a clinical trial is necessary. It is worth remembering that the mission and vision of the Foundation includes finding effective treatments for all forms of HSP. SPG4 type HSP, which is responsible for around 40% of all people affected by HSP – many times more than any other type, is just the first target. Other work is already going on in other places to find treatments for various HSP types.
Ashraf posted at 7:20 pm on 26 September 2018Reply

Sir i am a hsp patient, but i don’t know which type of hsp i have
So help me how would i know which type of hsp i have.
Milind posted at 1:02 am on 28 September 2018Reply

Dear editor I am taking a tablet conos 25 mg that is noscapine I.p 25 mg tablets from past 5 months a dose is 500 mg a day but I am confused about results.sometimes I walk Better and sometimes I gets confused while walking my toes are dragging badly so after taking for 5 months still I have not seen any big improvement.
What should I do. My case is pure hsp.hope so 4 but I have not tested for Gene. But mri shows thinning of dorsal spine.which is common in hsp
What to do?
Is epothilone d or taxol will work better than noscapine because I researched lot on microtubules stabilizing drugs.
Whats your suggestion???
- Editor posted at 1:00 pm on 28 September 2018Reply
  
  Editor’s Note: We all want an effective treatment for HSP and we want it as soon as possible. The prospect of a remedy creates hope, expectations and, sometimes anxiety. There are multiple questions and issues that currently have no answer. This is why a clinical trial is necessary, but Clinical trials are demanding to implement, they take a long time and they cost a lot of money. The choice to self-medicate or not is an individual one, but even if someone is self-medicating, there are potential benefits in achieving and maintaining correct body weight; undertaking a regular program of movement and exercise (preferably individually designed by a physiotherapist with neurological training); and taking good care of mental health, including reducing stress and learning to relax, and seeking professional help for anxiety or depression. Acceptance and patience are valuable attributes while we wait for answers.
Denis posted at 10:13 am on 8 November 2018Reply

When I taking noscapine I feel something like goosebumps in my head and feel much better. Thank you so much :*