What are the upcoming Potential Drugs for MND/ALS?

Masitinib

Masitinib is a tyrosine kinase inhibitor (TKI) being developed by AB Science to treat symptoms of amyotrophic lateral sclerosis (ALS).

The treatment is advancing in clinical trials as a possible therapeutic for numerous disorders, from cancer and inflammatory diseases to those of the central nervous system, such as ALS. Trial results expected in 2021

Masitinib for ALS

Ibudilast

In late 2018, it was found that a drug called Ibudilast used for Asthma treatment in Japan showed very positive results for ALS patients. MGH Boston started phase 3 trial with selected patients in December 2018 and will publish results sometime later this year 2019. In the mean time FDA has already approved this drug on a fast track approval on April 17, 2019. Although there is no real cure for ALS, So far FDA had approved 3 drugs, labelled as treatment for ALS, namely Riluzole(Rilutek), Radicut(Edaravone) and Ibudilast(Ketas).

If you want to get this medicine, it is available in Japan,  and can also be ordered through the web.  One such location a friend of mine was able to order is

https://www.mimaki-family-japan.com/item/list?keyword_pc=ibudilast&x=0&y=0

 

In India to order you will need a Credit Card that allows international purchases.  In India, it is preferable to have a import license which is  Form 12A which can be obtained from the site

https://cdscoonline.gov.in/CDSCO/homepage

To obtain this license you will need to attach a doctor’s prescription. Indicate while ordering the import license that it is for personal use and this life saving medication is not available in India

 

 

 

https://alsnewstoday.com/2018/07/11/phase-2-als-trial-shows-ibudilast-rilutek-improves-function/

Telbivudine

In December 2018, it was found that a drug called Telbivudine used for Hepatitis B treatment in Canada showed very positive results for ALS patients.  University of Alberta is starting a phase 3 trial with selected patients

https://www.sciencedaily.com/releases/2018/12/181218123120.htm

CuATSM

 

New ALS drug slows disease progression in groundbreaking clinical trial

Work on the drug, known as CuATSM, began over 15 years ago. At first it was thought that an excess of copper in the brain was the problem, but the researchers soon realized the opposite may be true.

“The initial focus of our work was on Alzheimer’s disease where it was thought that copper drove the formation of amyloid plaques,” Professor Kevin Barnham, co-developer of CuATSM said back in 2017. “We reasoned if you could just remove this copper you could eliminate plaques, and you’d have the solution. Then we thought, perhaps it’s the other way around? Maybe cells are expelling the copper and so we’ve actually got a situation of copper deficiency. So, what happens if you deliver the copper back?”

And that’s just what the new drug does. CuATSM is a compound that can penetrate the blood-brain barrier and deliver copper to the cells that need it. After tests in vitro and in animal models showed promise, a Phase 1 human clinical trial began in late 2016 to find the right dosage.

I have been following this research for a while and tried contacting professor Kevin Barnham and Peter Crouch in Melbourne, Australia.

The main genes that seem to cause ALS that they are investigating are SOD1, TDP-43, FUA and C9Orf72. Although there is tremendous hope for success for this drug, Unfortunately, they indicated that the drug will not be available for public for at least 3 to 4 years.

Professor Kevin Barnham (Florey) and Associate Professor Peter Crouch (The University of Melbourne) take us behind the scenes of the recent news about the clinical trial of CuATSM (Cugtsm)for motor neuron disease

https://www.youtube.com/watch?v=Ezj5iJCU5K4

 

TEFERSEN from BIOGEN

https://www.pmlive.com/pharma_news/biogens_tofersen_shows_promise_in_slowing_down_als_1286593

Tofersen is being developed for ALS patients who carry the SOD1 mutation, which accounts for just 2% of ALS cases, but nevertheless plays an important part in the drug’s mechanism.

During the phase 1/2 study, those dosed with tofersen over a three-month period resulted in a statistically significant reduction of SOD1 protein levels in the cerebrospinal fluid and a numerical trend towards slowing of clinical decline as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R).

 

https://newatlas.com/als-mnd-drug-clinical-trial-success/58026/?amp=true

 

Ropinirole Hydrochloride

TeT

Japan university’s clinical tests to use Parkinson’s drug for ALS