Can HSP really be treated with cough medicine?

Hereditary Spastic Paraplegia, otherwise known as HSP, is a genetic neurological disorder that most people have never heard of.

It has even been described by some as “The rare disease that no one talks about”.

HSP actually refers to a group of inherited diseases whose main symptom is impaired walking due to weak and stiff leg muscles. It can also cause difficulty with balance, urinary urgency, and fatigue.

There are over 80 different types of HSP, with SPG4 being the most common form of it.

Imagine talking to someone on a corded phone, when the cord is old and frayed. What you get is crackled communications, and periods of no communication at all. The upper motor neurons are like the phone cord. They transmit the brain’s signals for voluntary movements to the limbs.

When someone has HSP, a gene abnormality causes deterioration of the upper motor neurons in the brain and spinal cord, which prevents normal communication from reaching the motor neurons and muscles in the lower part of the body.

The condition can develop at any point in one’s life. It is usually a progressive disease, with the symptoms getting worse over time as the upper motor neurons become more damaged.

The severity and progression speed of the HSP symptoms vary greatly between sufferers (even between those with the same type of HSP). Patients with severe HSP symptoms may lose the ability to walk independently and must rely on mobility aids or wheelchairs. Others with more mild symptoms will not need to use mobility aids. While HSP seriously affects quality of life, it does not affect one’s life expectancy. All ethnic groups are equally affected by HSP.

Treatment for HSP mainly focuses on improving the physical and emotional well being of patients by using methods such as orthotics, physical therapy, and muscle relaxants.

While these treatments can help to reduce symptoms, they are by no means a cure and they do not stop the progression of the disease.

Professor Alan Mackay-Sim, world-renowned bio-molecular scientist and winner of the prestigious Australian of the Year award in 2017, is working to change this.

After a hang-gliding accident in his late 20’s left him wheelchair-bound for over six months, he declared it his life mission to repair spinal cord injuries, hereditary spastic paraplegia, and similar medical conditions. He has spent decades researching how to regenerate the nervous system.

His research paved the way to the first clinical trial where stem cells from the noses of paraplegic patients were transplanted into their damaged spinal cord in order to repair the injury by reviving the damaged nerve cells. This breakthrough was described as “the scientific equivalent of the moon landing.”

Professor Alan Mackay-Sim currently leads the HSP research team at Griffith University in Australia where they are investigating effective treatments for HSP and preparing to begin clinical trials.

According to the HSP Research Foundation, “Noscapine has now been identified as the preferred drug candidate for clinical trials based on its effectiveness on HSP stem cells, safety data, toxicology data, patent status, availability, and future affordability to HSPers.”

Noscapine has a well-established safety record, having been used for decades as a cough suppressant.

“This “repurposing” of approved drugs can be important for rare neurological disorders, like HSP, as it reduces the time and costs involved in developing a new drug, making it unprofitable for pharmaceutical companies.”

Professor Mackay-Sim’s studies reveal that Noscapine passes through the blood-brain barrier, which makes it capable of reaching the motor neurons and axons of the brain. It regenerates and repairs the function of stem cells and prevents cell death in HSP patients.

Professor Mackay Sim and his research team hope that Noscapine will prove successful in completely stopping muscle spasticity and the progression of HSP.

Noscapine has already been safely used in medicine for over 100 years.

However, it is because of this fact that as Noscapine now has no patent protection, there is no incentive for pharma companies to develop it as they have little chance of seeing a return on their investment. Instead, the pharma industry is currently trying to synthetically modify noscapine and create a “new” drug out of it that can be patented.

Clinical trials to treat this medical condition are expected to begin once funding is completed. In the meantime, many HPS patients do not wish to wait and have begun to take action. At the 18th annual conference of the Spastic Paraplegia Foundation Dr. Corey Braastad, Vice President of Genomics at Covance Drug Discovery disclosed that Noscapine is already being used informally by several dozen HSP patients in the USA.

Noscapine is available worldwide through specialty medication pharmacies or online suppliers.

More information about Noscapine can be found by reading the following research article and pharmacological report from the NCBI.