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An ALDH2 Deficiency Drug Mystery – What happened to the 'Miracle Cure' Alda-1?

An ALDH2 Deficiency Drug Mystery – What happened to the 'Miracle Cure' Alda-1?

In 2014, ALDEA Pharmaceuticals, a San Francisco – Bay Area drug company filed to begin studying a new drug to activate the mutated ALDH2 Enzyme. Alda-1, the name of the drug compound, was licensed from Stanford University in hopes of developing a new drug candidate that could restore the function of the ALDH2 Enzyme. What happened to Alda-1?


For the over 1 billion people globally with ALDH2 Deficiency, Alda-1 offered the hope of a drug that would fight acetaldehyde. ALDH2 Deficiency is a genetic disorder that leaves people with a mutated ALDH2 enzyme, which is responsible for breaking down acetaldehyde, a toxic carcinogen. Acetaldehyde accumulates in the blood in those with ALDH2 Deficiency from sources like alcohol, cigarettes, air pollution, coffee, fruits, and artificial flavors. Because of this, those with ALDH2 Deficiency are at significnalty increased risk of long-term diseases like cancer and liver disease. When drinking alcohol, acetaldehyde accumulates rapidly, causing facial flushing and other negative symptoms known as Alcohol Flush Reaction. Many thought it would give them the ability to drink without experiencing Alcohol Flush Reaction, and act as a miracle cure.

The company was hoping Alda-1 could help not only with ALDH2 Deficiency, but with Acute Alcohol Toxicity (alcohol poisoning), and a rare genetic disease called Fanconi anemia.

For Acute Alcohol Toxicty, Alda-1 would provide relief by helping the ALDH2 Enzyme to rapidly clear acetaldehyde from the blood, which builds up to dangerous levels when too much alcohol is consumed. This works because Alda-1 is not just an activator of the mutated ALDH2 Enzyme that causes ALDH2 Deficiency, but the healthy enzyme as well.

Fanconi anemia is characterized by bone marrow failure from damage from acetaldehyde and increased risk of cancer. Life expectancy is only 33 years in these patients. Alda-1 had the potential to decrease the damage done by acetaldehyde and improve life expectancy in these patients.In 2014 ALDEA Pharmaceuticals raised $24 million in series B financing. The investors included experienced Silicon Valley venture capital firms like Canaan Partners and Correlation Ventures. The money was raised to begin proof of concept and Phase II clinical trials for acute alcohol toxicity using intravenous injections of Alda-1 in emergency situations.

However, less than a year later, in 2015, the company hit a wall, funding was pulled, and no news about Alda-1 or the clinical trials has been heard since. Why? The data from these trials is not public, but the most likely reason these trials were stopped so quickly is they were either too toxic for use in humans or were having unintended side effects. We do know that the program was terminated and we likely will not be seeing Alda-1 reach the clinic.

While it would have been a great if Alda-1 could have benefitted those with acute alcohol toxicity and Fanconi anemia, it is unlikely it would have ever reached the hands of the public hoping to battle their ALDH2 Deficiency and Alcohol Flush? Why? Without an immediately life-threatening disease, the drug would have needed to have very low toxicity.

ALDH2 Deficiency increases risk for many diseases, like liver disease and cancer, but these are all long-term diseases, caused by years of exposure to carcinogens. No matter what, getting Alda-1 would require an emergency or a prescription unless there were no side effects. It is obvious from the abrupt end to testing that this was not the case. In addition, it is not clear that a viable, and low-toxicity, form of Alda-1 could be taken orally. The trials for acute alcohol toxicity were using intravenous injection.

When the program was spun out of Stanford and publicly announced, there was a lot of excitement from the public and from private investors for what Alda-1 could bring. Unfortunately, Alda-1 never got to the market and was pulled, likely due to side effects. Even if it did pass, it is unlikely it could have been the cure people were hoping for.

For those with ALDH2 Deficiency, the best option is still to continue to avoid sources of acetaldehyde and protect from acetaldehyde exposure.