W

ASHINGTON — There’s a lot of hopeful talk here these days, from Republicans and Democrats alike, about boosting funding for medical research and speeding up the drug approval process to bring hope to millions of patients across America.

But hope doesn’t always mean cure.

Forty-four years after Richard Nixon first declared a “war on cancer,” researchers have made significant advances against many deadly diseases, thanks in part to $30 billion a year in federal spending on medical research through the National Institutes of Health. Yet there are no true cures on the horizon for cancer, heart disease, Alzheimer’s, Parkinson’s, or any number of chronic conditions.

advertisement

“When you’re dealing with patients or their families, the trick is not to say that we have a cure and not to say that we haven’t made any progress. Both are ridiculously off the mark,” said George Church, the Harvard Medical School geneticist who pioneered human genome sequencing.

“We have a revolution, and it’s a positive one. When and where that’s going to translate into cures for diseases is impossible to talk about,” he said.

President Obama talks in lofty terms about the potential of medical science in the near future. Last month, Vice President Biden called for a “moonshot” to cure cancer, saying “there are so many breakthroughs just on the horizon in science and medicine, the things that are just about to happen.”

But even top White House advisers speak with a greater sense of caution.

“The answers and the solutions come in very small packets, and that’s just the nature of science, that it’s more incremental than the concept of a major breakthrough,” said Jo Handelsman, the associate director for science at the White House Office of Science and Technology Policy.

“I rarely hear scientists talk about major breakthroughs. They have the next cool result, perhaps, but it’s the accumulation of all of that knowledge that leads to the next bigger step, which may be a treatment or a cure,” she said. “And then you add those up over time, and you can see, yes, we have made enormous progress in some of these very tough diseases.”

But incremental progress doesn’t help patients like Myriam Marquez, a former public defender in Seattle who has been battling early-stage Alzheimer’s for six years.

For her, the search for a cure is urgent — and not just for herself. Her relatives carry a gene that makes them vulnerable to the disease: Her father and seven of his siblings had it, she has it, and two of her three siblings have it.

She knows it’s a good bet that her kids and grandkids could get it, too.

“You know, the damage has been done to my brain. … If something could come along to stop the progression, that would be awesome for me,” Marquez said. “But for the rest of my family, I’m fighting for a cure.”

It’s not that medical science has been standing still.

Heart disease death rates have fallen dramatically — dropping 76 percent between 1963 and 2010 — thanks to new medical procedures, improved emergency care, and medications. New treatments for specific kinds of cancer, like prostate cancer and chronic myeloid leukemia — a type of blood cancer — have made them far less deadly than they used to be. More treatment options are available to reduce the symptoms of multiple sclerosis and Parkinson’s. And HIV is now a manageable disease.

And yet, millions of Americans are still living with illnesses that stubbornly resist a cure. More than 600,000 die of heart disease each year — still the leading cause of death despite all the progress. Cancer, the second-leading cause of death, kills nearly 600,000 Americans per year. Millions struggle with the devastating loss of function that comes with diseases like Alzheimer’s, Parkinson’s, or multiple sclerosis.

More than a million Americans are living with HIV. And millions more deal with manageable but never-ending illnesses like diabetes, asthma, and other chronic conditions — hoping all the while that, one day, they can just get rid of them.

Cancer is considered the most promising field for precision medicine, which seeks to tailor treatment to the genetic makeup of an individual’s tumors. That’s why the White House’s Precision Medicine Initiative has set aside $70 million just for research to identify molecular changes that are the most important causes of cancer, and to develop treatments that might work best on them. Researchers also have high hopes for the future of immunotherapy — treatments that focus on strengthening the body’s immune system to help it attack the cancer on its own.

To date, though, the war against cancer is still far from won.

Scientists have developed drugs that can treat the abnormal gene in chronic myeloid leukemia, so “almost everyone with this disease can live a normal lifespan,” said Harold Varmus, a former director of the National Cancer Institute. With pancreatic cancer, though, “we have no good therapy” to fight the mutation in genes, so most people die within one or two years. And lung cancer includes so many genetic mutations that there are effective drugs for only a third of them, Varmus said.

Even the success stories have qualifiers. The drop in the death rate from heart disease is undeniable, but it’s still the number-one killer in the country. And HIV may be a treatable condition now, but there’s still no cure — although it’s not out of the question, given the latest lines of research.

Leading medical researchers say there are good reasons to believe we’re about to start solving more of the remaining medical mysteries. MIT’s Edward Boyden, who’s working on techniques for enlarging brain tissue samples to make them easier to see, said that approach could help solve a basic problem that leads to high failure rates with new drugs: too much guesswork. “There are a lot of assumptions, and assumptions often turn out to be wrong,” he said.

And ultimately, Boyden said, medical science needs to understand the inner workings of diseases better. Otherwise, trying to find new cures and treatments, based on what we know now, would be like trying to land on the moon in 1600. People back then wouldn’t have tried to do it with a rocket, Boyden said — they would have attempted it with a hot air balloon.

“Medicine is a long game,” he said. “If we can create these maps on scales that have not been possible before, we can really identify the foundational drug targets.”

That’s reason enough to pay close attention to the next stages of research, medical scientists say — even if it doesn’t wipe out the toughest diseases tomorrow.

“Is this the revolution that’s going to end all disease? Probably not. I think we still have a few more revolutions to go,” said Antony Rosen, vice dean for research at Johns Hopkins University. “But this is going to have a big impact on many of the diseases that have vexed us for years.”

Leave a Comment

Please enter your name.
Please enter a comment.

Recommended Stories

Sign up for our biotech newsletter, The Readout

A guide to what’s new in biotech — delivered straight to your inbox every weekday morning.