Legend Dies! Hawking’s Gone, but ALS Drug Development Should Continue

The legendary life of Hawking, the beloved great theoretical physicist, ended on March 14, 2018 by when Hawking fought against the well-known amyotrophic lateral sclerosis (ALS) for 55 years, which is 53 years more than the 2-year survival period diagnosed by the doctor, namely, his survival time is 27 times the diagnosed. However, we have to admit that the current ALS drug development still does not meet the expectations and has a long way to go.

ALS is a common neurodegenerative disease among rare diseases. 40-60 years old is the age bracket with high incidence; in terms of gender, males are more, accounting for about 60%; globally, the number of ALS patients in China is not small, reaching over 200,000 people. Celebrities like British actor David Niven, British musicologist Stanley Sadie, and U.S. baseball star Lou Gehrig also had the ALS.

According to research, there are only two ALS drugs marketed at present, separately Riluzole and Radicava. Riluzole, the first modifying drug approved for ALS, was born in 1995, specially approved to enter the Chinese market in 1999 because it is the first ALS drug, and included in the National Drug Catalog for Basic Medical Insurance, Work-Related Injury Insurance, and Maternity Insurance (2017 Edition) of China in 2017. However, Riluzole has very limited efficacy, no significant improvement of patients’ activity function and survival rate, and poor overall benefit, therefore, the drug has big limitations.

Fig. 1 Structural Formulas of Riluzole (left) and Radicava (right)

There was no new ALS drug marketed for a long time after Riluzole was marketed. Over 20 years later (2017), another ALS drug: Radicava under Japan Mitsubishi Tanabe was then approved by the U.S. FDA.

However, the current treatment situation of ALS is still not optimistic. According to an expert, most patients will die of respiratory tract infection and malnutrition, etc. in 3-5 years after being diagnosed with ALS. The current treatment situation of ALS is that there are drugs, but the effect thereof is not enough, let alone radical cure.

Fig. II Structural Formula of Masitinib

Therefore, the global big pharmaceutical enterprises and drug research institutions are actively developing ALS drugs, for example, masitinib developed by AB Science is a very promising ALS drug. As proved by the research, the combination of masitinib and riluzole in treating ALS has significantly better efficacy than riluzole alone. This drug received the U.S. FDA’s orphan drug designation in 2015, and received EMA’s orphan drug designation in 2016.

Besides, the Chinese pharmaceutical enterprises have put a good deal of effort into developing ALS drugs. The butyphthalide under CSPC has recently (March 11, 2018) received the U.S. FDA’s orphan drug designation.

The passing away of the "giant" Hawking is an enormous loss to the world scientific community. Unfortunately, drugs failed to save Hawking or have him stand again, or even substantially improve his life. Now, Hawking is gone, but there are millions of ALS patients in the world, and the drug development should continue.

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