Joost takes part in exclusive international drug trial

In this 21 June 2015 image from Twitter Joost van der Westhuizen encourages others to give a thumbs up to #thumbsupformndcure
In this 21 June 2015 image from Twitter Joost van der Westhuizen encourages others to give a thumbs up to #thumbsupformndcure

The Medicines Control Council of South Africa has granted Joost van der Westhuizen rare Section 21 approval to gain access to the motor neuron disease (MND) drug GM604 on compassionate grounds.

The drug, manufactured by California-based Genervon Biopharmaceuticals, has shown some promise in promoting the survival, growth and maintenance of neurons, which die in MND sufferers.

Read: Understanding motor neuron disease

According to an article in the 24 December 2015 issue of You magazine Joost – referred to as Patient 104 – is not allowed to divulge details about the treatment, including the cost.

In a press release, the makers of Genervon explain why Joost was chosen to take part in the trial.

"Patients from around the world were selected to receive GM604 treatment first because they are all influential, vocal, and strong advocates for curing ALS. Some of these patients are celebrities in their field. We want all of them to experience first-hand the curing power of GM604 to strengthen their conviction." has published updates on how the former rugby star is responding.

Genervon report on Patient 104

Patient 104 is a 44-year-old male from South Africa. His ALS onset was more than 5 years ago, and he was diagnosed with MND in May 2011.

He exhibited speech difficulty as early as May 2011, which is a sign of bulbar, cervical, and lumbosacral motor neuron deterioration. Patient 104 had some mobility in his legs, but he could not stand up or walk. He was able to move his fingers slightly.

He had very little strength in his arms and hands, and he could not hold or pick up objects. His speech was barely audible, and he had difficulty chewing and swallowing. His neck was extremely weak, and he was unable to hold his head up for any length of time.

After his 5th dose the patient's Progress Report notes:

"Patient says [he is] able to swallow a little bit better. Doctor observes what seems to be a marginal improvement in [arm] strength. Friends think his speech is slightly better. Patient did not experience any adverse side effects."

After his 7th dose, the patient's Progress Report notes:

"Patient reports that: his speech is slightly easier... his ability to swallow is better and sucking through a straw is, he says, 'a lot' better. He says his legs are slightly stronger and that he is drooling less. He has not experienced any adverse side effects. He has gained 2 kg and believes something is working. The changes have been slight and gradual. He says he definitely hasn't deteriorated since the start of the treatment programme."

They conclude that, at this stage, common improvements reported by patients treated with GM604 are an increase in energy and strength, improved swallowing and body weight, improved range of motion and breathing, and improvement in mood with GM604 treatments.

In February this year Genervon approached the US Food and Drug Administration (FDA) for accelerated approval of the drug after a small trial at Columbia University's Massachusetts General Hospital on patients with amyotrophic lateral sclerosis (ALS), aka Lou Gherig's disease (a form of MND) was performed.

The 12-week trial ended in 2014 and involved 12 ALS sufferers of which eight received the treatment and four were given placebos.

Genervon described the results as "robust and dramatic". They argue that by accelerating the drug they can save many ALS sufferers' lives.

Under normal circumstances it would take three years for the drug to be approved and thus become available to all, including under medical or health insurance plans.

In an exclusive interview with You magazine, Joost echoed ALS sufferer Nick Grillo's stance when he said he'd put his body through anything to try to help others:

Nick, who is 54 years old and lives in San Fransisco, said: "Personally I'd rather die a guinea pig and contribute to research than sit at home and waste away." 

Progress regarding the drug can be followed on Hope Now for ALS Facebook page.

Read more:

The ALS challenge raises cold cash

Common cause of ALS found

Joost undergoes stem cell therapy

Joost's treatment is failing (2011)

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