Displaying posts published in

September 2023

ELECTIONS Comments turned off

America Is Desperate for a New Beginning Nearly two-thirds of voters believe the system is ‘broken’ if it’s Trump vs. Biden. By William A. Galston

By on September 27th, 2023

https://www.wsj.com/articles/america-desperate-new-beginning-2024-election-voter-sentiment-polls-gop-primary-biden-trump-3fde70cd?mod=opinion_featst_pos2

We don’t need polls to tell us that confidence in our political institutions is at a low ebb, but they do help clarify what Americans are feeling.

In a recent poll from the Pew Research Center, 10% of Americans reported that thinking about U.S. politics made them feel hopeful, and 4% were excited. By contrast, 55% said they were angry, and 65% were exhausted.

This isn’t the first poll to note a pervasive sense of exhaustion, and I suspect it won’t be the last. Americans are tired of partisan quarrels that rarely reach a resolution. Issues like immigration reform linger for decades, and the Supreme Court has brought new ones such as abortion back into the arena.

Joe Biden was elected, in part, to calm this turbulence. Historians will debate whether he could have done so had he pursued a different agenda, but clearly his administration hasn’t reduced division, whether over economics, culture or foreign policy.

Americans blame both parties about equally for this situation. According to Pew, 60% of Americans have an unfavorable view of the Democratic Party, and 61% have an unfavorable view of the Republican Party.

A recent CBSpoll found that 54% of respondents regard the Republican Party as “extreme,” one of the favorite epithets of Democrats describing the GOP. But the same percentage also regards the Democrats as extreme, and only minorities think that either political party is “reasonable.”

Continue reading →
MEDICINE AND HEALTH Comments turned off

The FDA’s See-No-Data Approach A study showed promise in treating a rare disease. The agency wouldn’t even look at the results. By Stephen Cederbaum and Emil Kakkis

By on September 27th, 2023

https://www.wsj.com/articles/fda-food-and-drug-admin-arg1-d-pegzilarginase-aeglea-biotherapeutics-arginine-deficiency-healthcare-c9e8c21f?mod=opinion_lead_pos6

We’ve spent most of our medical careers investigating and treating rare genetic conditions, including some afflicting only a few dozen Americans. Recently, scientists developed a treatment for one such condition—arginase 1 deficiency, or ARG1-D, which causes the amino acid arginine to accumulate in the blood, harming the brain and causing seizures, stunted growth and intellectual disability. The Food and Drug Administration has refused to consider the therapy. Its decision ignores the best available data and indicates a lack of understanding of rare-disease research.

Discovering treatments for rare diseases is a daunting task. Recruiting even a few dozen people for a clinical trial requires doctors and drug companies to identify a large share of the patient population. And since the market for such therapies is necessarily small, it’s nearly impossible to attract investment. So when news emerged aboutAeglea BioTherapeutics’ ARG1-D therapy pegzilarginase, we could hardly believe it. Pegzilarginase is an enzyme engineered to lower the body’s levels of arginine. The randomized placebo-controlled study of pegzilarginase included 32 patients with ARG1-D.

The results speak for themselves. The amount of arginine present in blood plasma declined by 80% for patients on pegzilarginase. After only six months, 90.5% of patients who received pegzilarginase had normal arginine levels, and this was sustained over time. The data also suggested progressive improvements in motor function compared with a placebo. And most patients tolerated the therapy quite well.

These numbers were jaw-dropping. Which is why the FDA’s decision is incomprehensible.

Continue reading →