Monday, October 16, 2017

How Much are Gene-Therapy Treatments Likely to Cost?

An article about the potentially high costs for cures for genetic diseases reveals a subtle shift in how pharmaceutical companies intend to justify their high prices.

In the past, the usual argument was that high drug costs were justified by the high costs of drug development. The standard argument was that since it costs upwards of a billion dollars to take a drug all the way from initial research through Phase I, II, and III clinical trials, drug companies justifiably needed to charge high prices to recoup their costs. In some cases, drug companies needed to recoup losses incurred by drugs that never made it to market at all.

The paradigm has shifted with the advent of genetic therapy. Now, cells can be removed from a patient with a specific genetic mutation, modified by gene therapy techniques for that patient (and only for that patient) and then returned to the patient, affecting a complete cure. Since these cures are specific to individual patients, the FDA has begun to approve these new gene therapy techniques when they have been shown to work on just a few patients (perhaps several dozen). Gone are those lengthy Phase II and Phase III clinical trials, with their thousands of patient at dozens of medical centers. And gone, too, most of the costs of drug development.

As a result, drug companies are being forced to come up with new arguments to justify their high prices. And their new argument is shaping up to be something along the lines of "What's it worth to you?" How much will you or your insurance company pay for what one drug company spokesperson calls "long-term transformative benefits"?

Potential prices ranging from about $475,000 to $900,000 have been floated by some companies. At some point we may have wrestle with the question, "What is the value of life"?

Wednesday, October 11, 2017

The First Gene Therapy to Cure a Fatal Brain Disease

Gene therapy has now been used to cure a fatal brain disease called adrenoleukodystrophy (ALD), a genetic disorder that occurs in approximately one in 20,000 boys. Even more startling is that the vectors used to deliver the normal form of the ALD gene to the boy's cells are disabled viruses similar to the AIDS virus, called lentiviruses.

In the new technique, hematopoietic (blood-forming) stem cells are removed from the patient's bone marrow and exposed to lentiviruses that contain normal copies of the ALD gene. (Lentiviruses are used because like the AIDS virus, they are better than most other viruses at inserting genes into cells.) The stem cells are then returned to the patient's bone marrow, where they grow and multiply.

Now for the really interesting part. Some of the corrected stem cells make their way to the brain, where (apparently because of the environment there) they develop into neural supporting cells called glial cells, correcting the original defect in glial cell formation.

The timing of treatment is critical, however. It takes about a year between initial diagnosis and the time the treatment is effective. Unfortunately, ALD can progress so fast that patients who already have obvious symptoms of the disease may not be able to be saved. The best candidates for a cure are patients who (by virtue of hereditary history) are thought to be at risk for the disease, and whose only signs of the disease so far are changes in their brain scans.

Monday, October 9, 2017

HPV Vaccination Rates are (Finally) Rising

Vaccination rates against the HPV virus responsible for most cases of cervical cancer have been rising (finally), according to a recent CDC report.   Currently, more than 60 percent of teens have received at least one of the two recommended doses of the vaccine; that compares favorably to the 30% who had received the vaccine just ten years ago.

In 2016, the Advisory Committee on Immunization Practices (ACIP) updated its vaccinations recommendations to include a two-dose schedule instead of the previous three, making it easier for teens to be fully vaccinated.  Nearly 50% of all 17-year-old girls and 38% of all boys are now fully vaccinated, according to the new two-dose schedule.

Health officials are encouraged, but there is still room for improvement in vaccination rates. More than 40% of Americans aged 18-59 are infected with genital HPV.  There are nearly 40,000 new cases of cancers each year in areas of the body where the HPV virus is found (cervix, vagina, vulva, penis, anus, rectum, and oropharynx), according to the CDC.  Nearly 80% of these cancers could be prevented by vaccination.

Thursday, September 14, 2017

Allergan Seeks to Preserve a Drug Patent

Here's a novel idea for protecting your drug patent: sign it over to a Native American tribe. Allergan is trying to do just that with its blockbuster drug Restasis, according to a news release from the company.

According to the news release, Allergan has transferred its patents for Restasis to a Native American tribe in New York. The deal stipulates that Allergan will pay the tribe royalties of about $15 million a year in exchange for exclusive rights to the patents. For its part, the tribe will argue to the United States Patent Trademark Office that they have sovereign immunity, and hence the patent cannot be challenged by the U.S. government.

The move, if it works, would shake up the drug industry and perhaps give Native American tribes a significant new source of revenue. But make no mistake; it'll be the drug companies that will be the big winners. Restasis had sales of more than $330 million in just the past three months.

Monday, September 11, 2017

H7N9 Bird Flu is Still Around

Have you forgotten about bird flu? Well, don't, because it's still around. In fact the 2017 flu season was the worst ever, according to the CDC. From Oct. 2016 to the summer of 2017, the H7N9 strain of bird flu infected 759 people. About a third of them - 281 people - died. That's nearly as many as the total number of deaths in the four previous years combined.

CDC officials consider H7N9 to "the influenza virus with the highest potential pandemic risk", according to a weekly report from the agency. Fortunately, the virus has not yet evolved to be easily transmitted between humans; nearly all cases to date have been the result of contact with infected birds. But if the virus ever does mutate to become more easily transmissible between humans, watch out!

Health officials continue to monitor the virus closely for any signs that it is changing in ways that would make it a worldwide threat. But so far, all cases have been in China, Hong Kong, and Macao.

Friday, September 8, 2017

Innate Fitness May Affect Cancer Risk

It's been known for several years now that being physically fit reduces a woman's risk of breast cancer. But how? Is there something about actively engaging in an exercise regimen that reduces risk, or is it just a function of being naturally fit (called "innate" fitness)? Or both?

To try to address this question, researchers took advantage of groups of rats that had been bred to be either genetically "fit" or genetically "not fit". The two populations were created over many generations by exercising rats on a treadmill; those that could run long distances were bred to other rats that also ran long distances; those that were poor exercisers were bred to other poor exercisers. Over time, the groups diverged in terms of innate fitness.

In recent experiments, then, female offspring of the "fit" and "not fit" groups were exposed to a known carcinogen. None of the rats in either group engaged in exercise after birth, so any differences between the two groups must be attributable to innate fitness, rather than exercise per se. The results were strikingly different - the incidence of breast cancer was reduced by more than 70% in the "fit" rats, compared to the "not fit" rats.

It's not known yet whether an active exercise regimen would reduce the risk of breast cancer in the "not fit" rats. Undoubtedly that will be tested next. But for now, the new information is that there's something about the metabolism of innately fit rats (and perhaps humans) that protects against breast cancer, even if they're not routinely exercising.

The researchers looked only at breast cancer as their measured endpoint in these experiments, but perhaps fitness affects the risks other cancers as well.

Wednesday, September 6, 2017

Childhood Leukemia Treatment to Cost $475,000.

Remember that new gene-based therapy for childhood leukemia that was approved by the FDA this past summer? (See this blog, July 15, 2017). Novartis, the company responsible for developing the therapy, has finally put a price to it; $475,000 for a full treatment regimen, according to an article in The Guardian.

Consumer groups are worried that prices such as this will usher in a whole new era of expensive medical treatments. At some point we have to ask - is the price for a health- or life-saving procedure just too high? How will we pay for such treatments? If health insurance companies cover the treatment, then surely they'll recover their costs by raising the price of health insurance for us all.

The company claims that the price is justified by its need to earn a return on its investment. Hmmm....