It’s aliiive! And it’s approved!

Last week, the FDA approved Novartis’ Kymriah—a “living drug” that works by making immune cells realize they should get rid of those pesky leukemia cells making a mess of things. We’ve been following this story since June, because it’s really cool for a couple reasons. First, it marks the first time a gene therapy has been approved for use in the US, although more CAR-T treatments are in the pipeline. Second, the treatment is designated for the most prevalent form of childhood cancer in the US—acute lymphoblastic leukemia (ALL). There is a bit of worry that the treatment is prohibitively expensive, which is what sunk the first gene therapy approved in the EU, but maybe competition will help drop prices.

Now don't be stingy. Share this.Share on FacebookShare on Google+Tweet about this on TwitterShare on LinkedIn

Safe T first

Yay, we get to write another article about fewer needles in the lives of diabetics! Researchers in London are working on an immunotherapy approach to slow down the progression of Type I Diabetes, which would ideally result in a future where those diabetics won’t have to inject insulin daily. The disease works by attacking insulin-producing cells, so the scientists decided to try to get the lazy part of the immune system that wasn’t stopping that—regulatory T cells—to stop playing video games and get a job! (Sorry. Too close to home?) The scientists proved the safety of this approach in a recent Phase I study, so it will be a while before anything is marketed to the general public, but we just think this immunotherapy stuff is the bee’s knees.

Now don't be stingy. Share this.Share on FacebookShare on Google+Tweet about this on TwitterShare on LinkedIn

FDA panel ALL in on CAR-T cancer therapy

An FDA advisory board voted unanimously for the approval of a CAR-T cancer therapy for acute lymphoblastic leukemia, or ALL. This could be the first gene therapy—where one’s own genes are altered to create “killer T-cells” to attack cancerous cells—approved in the US. CAR-T cell therapy is a one-time treatment and shows promise in its ability to knock out ALL where other cancer drugs have failed. Novartis, the drug developer, reported 83% of the patients achieved complete remission within three months and a high probability of being relapse-free at 12 months. The new treatment isn’t without safety concerns, but traditional treatments are more toxic than CAR-T, must be taken long term and eventually stop working. Take THAT, childhood cancer!

Now don't be stingy. Share this.Share on FacebookShare on Google+Tweet about this on TwitterShare on LinkedIn

1. Clinical trials in black and white (but mostly white, if we’re honest)

Clinical trials are complex. They are costly. They take too long. They favor white people. Wait. What? According to an article in the New York Times, many cancer clinical trials are heavily weighted towards the white population. Why? They cite several reasons: most top cancer centers are not in rural locations; trials sponsored by industry do not have to have a specific mix of races; many minorities have chronic diseases that may exclude them from cancer trials. But the fact remains that cancer trials are weighted toward Caucasians. Maybe they’re easier to enroll, but should that matter? Maybe. Check out the FastPoll™ below and tell us what you think.

Now don't be stingy. Share this.Share on FacebookShare on Google+Tweet about this on TwitterShare on LinkedIn

2. Hot, hot, hot then not, not, not

Immunotherapy. Got your attention? Heat Biologics (HTBX), a biotech developing treatments for lung cancer using immunotherapy, went on a stock market roller coaster over the past few days, which is a lesson in just how fragile the drug development process can be. The last seven days of trading have seen HTBX’s share price move as follows: $1.53 to $1.91 to $2.49 to $3.23 to $2.98 to $2.87 to $1.03. The climb: anticipation around the release of Phase II data. The fall: compound HS-410 did not meet its primary endpoint. Now the fun part, HTBX is presenting data from another compound (HS-110) this Wednesday in Europe. Might want to check the ticker that day and hold onto your hat. Drug development is a wild ride.

Now don't be stingy. Share this.Share on FacebookShare on Google+Tweet about this on TwitterShare on LinkedIn

2. Is that a Cuban cigar in your pocket?

We all have a guy or know a guy who has a guy who has connections at the airport. While his services are typically reserved for smuggling cigars, you might want his help to get your hands on Cuban-developed and produced Cimavax, an immunotherapy-based therapeutic vaccine developed to halt cancer growth and keep it from recurring in patients with non-small-cell lung cancer. It is not FDA-approved, but it will soon be part of a clinical trial in the US based out of the Roswell Park Cancer Institute in Buffalo NY. FYI, it turns out Cuba may have a bona fide healthcare and pharmaceutical development system and the Buffalo-based trial could take years, so grab your backpack and head to Havana.

Now don't be stingy. Share this.Share on FacebookShare on Google+Tweet about this on TwitterShare on LinkedIn

5. Copycats or cost controls?

Has risk reduction negatively impacted cancer drug developments? Dr. Richard Pazdur, head of the FDA’s office of oncology products, expressed this concern during the American Society of Clinical Oncology meeting last week regarding the number of similar drugs in development, all focused on the PD-1 protein that cancerous tumors use to evade the immune system. While immunotherapy has shown promise even with the deadliest cancers, these therapies are currently priced in the $150k per year range, prompting not just “me-too” drugs, but me-too desires for the profits generated by these drugs. Many in the industry responded with the importance of PD-1 candidates for future combination therapies and the potential benefit to patients in the form of lower drug prices when there is competition in the marketplace.

Now don't be stingy. Share this.Share on FacebookShare on Google+Tweet about this on TwitterShare on LinkedIn