The Miracle Cancer Test


Cancer may be the most frightening word in the English language. As a physician, I know that if I have to approach that word with my patients, I must be prepared for their frightened reaction. In fact, I am so careful to broach the subject of cancer with my patients that I am even careful when I tell them they don’t have cancer, lest they mistake what I’m saying to mean the opposite!

We all know detecting cancer early is very important to the prognosis. Many types of cancer are curable if detected early, such as breast, prostate, colon, kidney, and skin cancers. Unfortunately, many cancers are nearly always detected late when treatment is rarely curative, such as pancreatic, lung, and brain cancers.

Just imagine, what if you could take a simple blood test to detect 50 different types of cancer early enough to be cured? Sounds like a fantasy – but it isn’t. In fact, such a blood test has already been developed. But alas, it is stuck in regulatory purgatory and may take years to reach availability.

Regular readers of The Wall Street Journal know the Saturday edition always has a section called The Weekend Interview. This week Allysia Finley interviewed Francis deSouza, CEO of a company called Illumina. She tells us that eight years ago a scientific breakthrough occurred by serendipity. Meredith Halks-Miller, a pathologist at the genetic-screening company Illumina, stumbled on something unusual while running prenatal blood tests for fetal chromosomal abnormalities. In some blood samples, the fetal genes were normal but the maternal DNA wasn’t.

Illumina alerted pregnant women’s doctors to the finding. After further investigation, all the women were diagnosed with cancer, though none had symptoms when their blood was drawn. This led to the development of a blood test that can now detect 50 types of cancer and has the potential to save tens of thousands of lives a year if it becomes widely available. But currently, regulators are slowing down the process.

The Federal Trade Commission last month wrapped up an administrative trial in which it seeks to block Illumina’s $8 billion acquisition of Grail, the company that makes the blood test. Illumina founded Grail in 2015 and spun it off a year later. The Menlo Park, California -based startup is named Grail because scientists have long been on a quest for a blood test that can diagnose cancer early – the Holy Grail!

“We know that cancer kills about 10 million people a year – about 600,000 in the U.S. And we know that even for some of the deadliest cancers, your odds of survival are much higher if you catch the cancer in Stage 1 or Stage 2,” says Illumina CEO Francis deSouza. “So a blood test that can catch cancer early can truly be life-changing.”

Currently there are only recommended screenings for five cancers – breast, colon, prostate, cervical, and lung in high-risk smokers. While these tests can be very helpful, they are also prone to false positives – suggesting cancer that isn’t really there. Cancer is absent in about 70% of patients with an elevated PSA test for prostate cancer detection and 7 to 12% of suspicious mammograms. The Grail blood test has a false positive rate of less than 1%.

To be sure, the Grail blood test is not perfect. But it can detect the 12 deadliest cancers with 60% accuracy. But Grail estimates that adding the blood test to existing screening could reduce late-stage cancer diagnoses by more than half among patients ages 50 to 79, which translates into a 26% overall reduction in five-year cancer mortality.

Why, you might ask, did Illumina spin off Grail after the discovery of this blood test? After Dr. Halks-Miller’s discovery, more research, clinical testing and investment were needed before a test could launch to the public. Illumina spun off Grail so that it could raise more money for studies. Grail ultimately raised $1.9 billion in capital, while Illumina maintained a 12% stake. With a commercially viable product nearly in hand, Grail in September 2020 began exploring an initial public offering (IPO). That’s when Illumina made its $8 billion offer.

Why is the FTC holding up the Illumina acquisition of Grail?

Illumina is the leading manufacturer of gene-sequencing machines, were the first to identify the novel coronavirus in late 2019, and more than 70 countries are using them to find new variants. Moderna and Pfizer also relied on DNA sequencing from Illumina machines to develop their mRNA vaccines.

The company’s dominance in the DNA-testing market, however, attracted regulatory scrutiny. In March the FTC sued Illumina and Grail to block the acquisition, arguing that it would “lessen competition in the U.S. multi-cancer early detecting test market by diminishing innovation and potentially increasing prices.” Mr. deSouza pushes back from this analysis, saying, “Today, there is nobody who is even starting the studies to develop a 50-cancer test like Grail, and once you start the study, it’s still a few years before you actually get the test. We think there will also be blood tests for single cancers, for colorectal cancer and other cancers. Those won’t compete with Grail. They will be complementary to Grail.”

Illumina promised the FTC it wouldn’t thwart Grail’s potential competitors and would give its clinical oncology customers contractual guarantees of ‘equal and fair access.” But the FTC wasn’t satisfied. Although it asked the federal judge overseeing the case to dismiss its complaint in June, it’s still alive as an administrative proceeding. Frustrated by the slow process, Illumina and Grail closed the deal in August despite the risk that it could be undone if it loses in U.S. or European courts. As for the U.S., the Supreme Court likely wouldn’t hear a case until 2025, so Illumina faces the risk that the deal could be blocked for years.

It’s frustrating to imagine the number of lives lost by the delays before this breakthrough, exciting scientific development becomes a reality. But the future is nevertheless bright. Mr. deSouza says, “I truly believe that, within our lifetime, we can make a significant dent in cancer survival rates, and a lot of that will come through the work that’s done in genomics.”

Vaccines for Young Children?


Do young children need to be vaccinated? That is the question on the minds of millions of parents as pharmaceutical companies push for authorization of vaccines for the very young.

Pfizer has announced plans to seek emergency use authorization (EUA) from the FDA soon for vaccines for youngsters ages 5 to 11. The FDA has promised to move quickly on the application and expects to make a decision by the end of the month. Previous FDA approval was given first to those over age 16, and later for those ages 12 to 15. Is it time to begin vaccinating children as young as age 5?

Although Covid is generally mild in children, the latest data shows the Delta variant has resulted in more than 30,000 children being hospitalized since August. According to the American Academy of Pediatrics, nearly 5.9 million Americans younger than 18 have been infected with the coronavirus. Of the roughly 500 Americans under 18 who have died, about 125 were ages 5 to 11. That’s a tiny fraction of the numbers affecting older Americans, but it is still a significant number, especially if it is your child.

“It really bothers me when people say kids don’t die of Covid,” said Dr. Grace Lee, an associate chief medical officer at Stanford Children’s Health who also leads a key advisory committee to the C.D.C. “They die of Covid. It’s heartbreaking.”

Pfizer anticipates giving young children one-third of the adult dose of the vaccine. Just as in adults, two shots will be necessary, spaced three weeks apart. The Wall Street Journal says school districts and public-health officials have begun preparations for vaccinations, though the work remains in early stages. Health and vaccine experts expect the vaccines to be administered at certain schools, pediatrician offices and some pharmacies.

Schools and public-health departments are experienced with childhood vaccines unrelated to Covid-19 because they routinely administer them, although the cold-storage requirements of the Pfizer-BioNTech vaccine may pose some challenges, according to the experts.

Pediatrician offices and other administration sites won’t be able to vaccinate the children with existing supply on hand because doses for children are smaller and prepared differently than for adults. Pfizer said it would start shipping the pediatric doses, if authorized, as soon as it is cleared to do so by U.S. health authorities.

Many parents are eager to have their children vaccinated so they can resume many activities that have been restricted due to the pandemic. Others parents are wary of exposing their children to a new vaccine. According to a recent survey conducted by the Kaiser Family Foundation, roughly a third of parents of children between ages 5 and 11 said they would wait and see before allowing their children to receive the shot.

Dr. Walt A. Orenstein, an epidemiologist at Emory University and a former director of the U.S. immunization program, said that given the competing pressures on the F.D.A. to make vaccine decisions quickly but carefully, public discussion was essential. He said many parents were wavering between fear of Covid-19 and fear of side effects from a pediatric vaccine. If they were less worried about the consequences of coronavirus infection, he said, concerns about possible side effects would be their top priority. If they were more worried, the vaccine’s effectiveness would matter more. As with other vaccines, Dr. Orenstein said, pediatricians would play a critical role in easing parental anxiety.

I am old enough to remember when vaccinations for young children first became available for poliomyelitis. Polio is a devastating disease that crippled many children and killed some. The polio fatality rate is 2 to 5% in young children and 15 to 30% in adolescents. The introduction of the polio vaccine in 1955 changed all that. The graph below shows the impact of the polio vaccine.

By contrast, recent data in young children indicates 5.9 million have tested positive for Covid 19 according to the American Academy of Pediatrics. There have been 520 deaths recorded in children. Of these, only 125 under the age of 12 have died – and there is no crippling effect as was commonly seen in polio. The AAP reports a fatality rate of 0.01% of all child Covid 19 cases, but this includes children up to age 18. The fatality rate for young children, under age 12, is only 0.002%. So, polio takes the life of 2 to 5 children in every 100, while Covid-19 takes the life of about 2 in every 100,000. That means the chances of your child dying from Covid are more than 1000 times less than polio. As a parent, you’ll have to weigh these figures compared to the risks of a serious vaccine side effect in your child. That data is currently unavailable.

There is no doubt Covid-19 is not the scourge that polio was in the 1950s. Whether or not it is serious enough to have your child vaccinated is a question every parent of small children will soon have to decide.

Covid Pill Should be Coming Soon

Home treatment of Covid may be easier soon.  Instead of hospitalization, many people suffering from Covid may be able to take a pill to treat their infection.

This exciting news comes from Merck and Ridgeback Biotherapeutics that have worked together to produce this new Covid drug called molnupiravir. These companies just released data that showed molnupiravir reduces hospitalizations by about half. They will soon apply to the Food and Drug Administration for emergency use authorization (EUA), which hastens the usually slow process of approval.

The Wall Street Journal editorial board says this potential wonder drug should have been available much sooner. Since the beginning of the pandemic, doctors have been hoping for an oral antiviral that could prevent recently infected patients from getting sicker. The FDA approved Gilead’s remdesivir for emergency use in hospitalized patients last spring, but the intravenous drug is not available to those not sick enough to be hospitalized.

The National Institutes of Health (NIH) prioritized development of monoclonal antibodies, which have helped many patients, including President Trump. But they are difficult to produce and distribute. Since demand exceeded supply this summer, the feds have rationed treatments (a taste of socialized medicine early). But the FDA and the NIH missed the chance to accelerate antivirals like molnupiravir, which creates errors in the machinery of the virus copying code.

Early clinical trials this spring showed molnupiravir rapidly reduces the amount of virus in patients. The Biden administration signed a $1.2 billion contract for 1.7 million courses this past June. While some critics of the pharmaceutical industry complained about the cost, Merck is spending to develop the treatment and scale up production in anticipation of high demands at its own risk. Merck has also signed licensing agreements with generic manufacturers to accelerate the pill’s availability world-wide. The technology to produce the pills in low-income countries is sufficiently simple to make this possible, unlike with the Covid vaccines. This makes molnupiravir a perfect solution for third-world countries.

Yet the FDA has already shown reticence to approve the drug without Phase 3 trials. It seems the FDA believed this summer that vaccines were sufficient for the immediate future, but the many unvaccinated have proven this strategy a failure. WSJ says molnupiravir would have been very helpful during the Delta variant surge this summer if it were only available. Preliminary results from the Phase 3 trials available, showed 7.3% of at-risk patients who received molnupiravir were hospitalized, and none died within 29 days of treatment. By contrast, 14.1% of placebo recipients were hospitalized or died.

Even better news is that the drug seems to be effective against different variants and is unlikely to produce viral resistance. This makes this a great drug now and in the future, not only in America, but world-wide, especially in those countries where vaccines may be unavailable. It may also reduce transmission and the severity of breakthrough infections.

This new drug highlights two facts about our current drug innovation system. One, we need to do everything possible to encourage R & D in developing new drugs. This highlights what I wrote recently in Undermining Drug Innovation. Two, we need a faster system for approval of these drugs by the FDA. The FDA can make up for the lost time by approving molnupiravir as soon as possible.