Thanks to the FDA, I can finally treat Gaucher’s and take the fight into my 80s

Last week, the FDA made two important decisions to secure our nation’s health. The first, a half-century in the making, allowed consumers to purchase, for the first time, a long-delayed drug developed at Stanford…

Thanks to the FDA, I can finally treat Gaucher's and take the fight into my 80s

Last week, the FDA made two important decisions to secure our nation’s health.

The first, a half-century in the making, allowed consumers to purchase, for the first time, a long-delayed drug developed at Stanford University to reverse harmful effects of gene therapy.

The other was to approve new classes of powerful medicines that will help people with cancer, immune deficiencies and chronic heart disease.

These approvals represent the latest steps in America’s ongoing struggle to improve the health of all Americans. Together, they’re an example of the FDA’s effort to unleash creativity, empower patients and boost the nation’s leading biomedical innovation.

Gene therapy, which is intended to treat inherited diseases like Duchenne muscular dystrophy, has been on the horizon for the past 50 years. Its time finally came in 2017 when Stanford researchers designed a protein to repair the genetic errors that cause muscular dystrophy. Their plan was to administer the therapy in a simple, two-pill-a-day pill by injecting the patient with a syringe directly into their muscle.

The regulator’s approval of a single-pill form, including the syringe, was a big step. It shows the FDA’s commitment to making sure that new medical products, including gene therapies, will reach their promise for patients in America.

Further FDA action is required to make sure the injections do what is promised. That means to fully commercialize the drug, scientists must continue developing the “cocktail therapy” to mimic the beneficial effects of the single-pill form.

Pfizer is currently doing preclinical testing on the cocktail therapy with the single-pill form and human data from that testing has been promising. From a physician’s perspective, it means more people with muscular dystrophy can get this good news and doctors with years of experience now have a new class of powerful medicines to help people suffering from conditions like cancer, heart disease and immune deficiencies.

But no one can improve health without increasing access to safe, effective medicines.

And that’s why last week, the FDA issued several new drug approvals, including a combination drug that delivers three powerful medicines to treat patients with chronic heart disease.

Pfizer is developing the combo in collaboration with the manufacturer Abbott Laboratories. This trio of medicines is designed to strengthen the heart with particular attention to the risks of dying from heart attacks.

In other FDA actions, the agency is also granting approval for Johnson & Johnson’s “MaxPrexy” Booster Shots to treat immune deficiencies. These medicines target Gaucher’s disease, a rare disease in which the immune system destroys skeletal muscle tissue.

In November, just before my 80th birthday, my wife and I were taken to the doctor, where it was revealed that a very rare illness had been triggered by such a problem in our children’s mouth. The family emergency line, Incripesia, had at that time only a single medication with which to treat it.

When I was a child, we would visit the dentist and he’d use his Saturday night time with me to conduct “chest x-rays.” Every time I visited him, I came home with a chest x-ray, right down to the size of the nipple. This caused me to laugh until my teeth and jaw hurt. It’s quite remarkable, but these x-rays were a kind of insurance policy.

No such insurance policy exists when people with Gaucher’s get a relapse, and very often that is fatal.

This medicine came as a much-needed lifeline. Although there is currently no cure for Gaucher’s, the MaxPrexy Booster Shots can reduce the likelihood of death by more than 80 percent. It can improve life expectancy by 10 to 30 years for some people. And it is the most well-known advance in the treatment of Gaucher’s disease in more than 100 years.

It’s also exciting. For the first time, the FDA has finally approved a two-drug combination for Gaucher’s, making this latest medicine the first available therapy with multiple protective factors.

That’s something I can get behind.

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