As the world’s top blood experts gather, 5 key questions to watch

SAN DIEGO — The world’s experts on all things blood will convene here starting Friday to discuss the latest advances in treating disorders ranging from hemophilia to sickle cell anemia to cancers like leukemia and lymphoma.

Academics will share their latest research. So will scientists from biotech startups and global drug companies. Here, five questions we’ll be seeking answers to at the American Society of Hematology meeting, which runs through Tuesday.

1. How safe are the latest cancer immunotherapies?

There will be a lot of buzz about CAR-T immunotherapy, and for good reason. It’s been a rough year for the intricate cellular science, which involves extracting cancer patients’ own immune cells and engineering them to attack their tumors.

Juno Therapeutics, one of the leaders in the field, stunned the research community with news that five patients died due to side effects from a CAR-T therapy it developed with an experimental drug called JCAR015. Clinical trials for that drug are now on hold.

Juno executives will give more details both on the JCAR017 trial and its investigational work on another therapy, JCAR018, which has not experienced the same problems. They’ll seek to settle swirling concerns from investors, patients, and physicians about the safety of CAR-T — which has great promise, but also holds serious risks.

Two other startups working on CAR-T will also give presentations. Bluebird Bio will share early data from a clinical trial that has gone very well so far: Tumors shrank in the six multiple myeloma patients who received a high dose of Bluebird’s CAR-T therapy, and two of them had no traces of cancer after several months. It’s still early in the trial, but cancer doctors will be eager to hear more.

At one of the last sessions of the conference, we’ll hear from Kite Pharmaceuticals, which is widely expected to be the first to get a CAR-T therapy to market. Expectations are running high for Kite’s pivotal Phase 2 ZUMA-1 study, which is testing a CAR-T therapy in lymphoma patients. We’ll see the data on Tuesday.

2. Are we finally making progress against sickle cell?

Sickle cell anemia — which can cause intense pain, infections, and even organ failure — has been stubbornly hard to treat for decades.

We might finally get some good news this weekend.

Selexys Pharma, a small biotech based in Oklahoma, will be presenting the results of a trial of a drug to treat sickle cell. It uses an antibody called SelG1 to try to cut down on episodes of “sickle cell-related pain crises,” in which the malformed blood cells obstruct circulation in blood vessels.

One hint that the results will be positive: Selexys was recently acquired by Novartis in a $665 million deal.

3. Can we do transfusions with artificial blood cells?

Early stage research led by Washington University in St. Louis suggests that, one day, we may not have to go through the hardship of transporting blood to hard-to-reach locations, such as battlefields.

The concept behind these synthetic blood cells is simple: just add water. In theory, they’re then ready to transfuse into a patient.

The product, called ErythroMer, is still in very early, preclinical stages — but it’ll be interesting to hear more. A solid proof-of-concept could have some intriguing applications.

4. Are we entering a new era for lymphoma care?

A trio of Phase 3 studies seems to indicate that the standard of care to treat lymphoma could be shaken up.

Next-generation biologic drugs, coupled with chemotherapy, are helping some cancer patients live longer without their tumors growing substantially.

One of the drugs up for discussion is from Genentech. Marketed as Gazyva, it seems to be more effective in treating follicular lymphoma than Rituxan — another widely used Genentech drug.

5. Can we stop the body from rejecting cell therapies?

As we hurtle toward the era of cell therapies, such as CAR-T, one key question is how to scale up such highly personalized treatments — and how to do it in a cost effective way.

Right now, cell therapies usually involve extracting a patient’s own cells and turning them into a biologic drug to infuse back into the patient. That’s called an autologous cell transplant. Some companies are trying instead to develop a one-size-fits-all cell therapy, called an allogeneic transplant. In theory, such a treatment would be quicker and cheaper to develop than one customized to each individual patient’s cells.

But it’s been a hard road, because the immune system tends to attack any foreign body — like an unfamiliar cell — in what’s known as “graft vs. host disease.” (That’s why patients who receive organ transplants have to take powerful immune system suppressants to ward away organ rejection.)

At the ASH conference, we’ll get a peek at several intriguing new approaches to treat graft vs. host disease. Specifically, it looks like the cancer drug ibrutinib shows promise in preventing the immune system from flaring up against allogeneic transplants. We’ll be eager to see more data.