Bioengineering Innovations Highlight the Future of Cancer, Hemophilia Treatment

A collection of studies presented at the 2016 ASH Annual Meeting and Exposition highlighted advances in biomedical engineering that could improve the delivery of treatments for patients with cancer and hematological-related illnesses. 

“All of these [innovations] build upon established science with bioengineering strategies that could make therapies significantly more effective if they are pursued and refined,” Armand Keating, MD, moderator of the press conference and professor of medicine and biomedical engineering at the University Health Network in Toronto, Canada, said in a press release. “I believe each of these has the potential to change practice.”

The press conference highlighted studies that examined the efficacy of innovative methods, designed to carry treatments directly to the areas affected by cancers and hematological illnesses. These novel methods—such as nanotherapies and engineered drug delivery vehicles—could provide significant advantages over traditional methods.

Instant Blood Transfusions

Among the studies presented, Allan Doctor, MD, presented data on the ErythroMer, a nanoscale biosynthetic artificial red cell that can emulate vital functions of natural red blood cells in order to provide an alternative to blood transfusion in emergency situations. 

“One key goal is to advance field resuscitation of civilian trauma victims in remote settings and soldiers who are wounded in austere environments without access to timely evacuation,” Dr Doctor said in a press release. “ErythroMer would be a blood substitute that a medic can carry in his or her pack and literally take it out, add water, and inject it. There are currently no simple, practical means to bring transfusion to most trauma victims outside of hospitals. Delays in resuscitation significantly impact outcomes; it is our goal to push timely, effective care to field settings.”

However, clinical testing for the ErythroMer is in the very early stages and human testing has yet to be conducted. The researchers estimate that the ErythroMer could be ready within 10 to 12 years.

Gene Therapies

Another study focused on an experimental gene therapy—SPK-9001 (Spark Therapeutics/Pfizer)—that researcher say could jumpstart production of the blood-clotting protein factor IX in patients with hemophilia B. 

“One of the potential innovations with a gene therapy for hemophilia B, compared to factor IX infusions, is that once an individual establishes a stable factor activity level, then they may remain at that level for an extended time,” Katherine A High, MD, chief scientific officer at Spark Therapeutics in Philadelphia, said in a press release. “At the factor IX levels seen in this study, most normal activities of daily living should be open to people with hemophilia. It could be a potential paradigm shift in the treatment of hemophilia.”

According to Dr High, factor IX is a protein essential to the body’s process of preventing uncontrolled bleeding. The experimental drug uses inactive virus to inject a small snippet of DNA into a patient’s cells. Once the DNA is stabilized in the liver, the body begins to produce factor IX. 

Study results have revealed that factor IX levels would remain sufficient for patients to engage in normal day-to-day events after just a single infusion of SPK-9001. Of the nine study participants, seven have maintained factor IX levels of 12% to 46%—compared to 50% in healthy adults—at 12 weeks postinfusion. The researchers noted that 12% factor IX levels is the minimum threshold considered necessary for adult patients. 

Dr High and colleagues also found that use of SPK-9001 reduced factor consumption by 543,589 IU—equivalent to $1,182,298.

Advances in Chemotherapy

A novel chemotherapy delivery method could improve survival among older patients with leukemia who receive bone marrow transplants, according to data presented by Jeffrey E Lancet, MD, of the department of malignant hematology at the H Lee Moffitt Cancer Center. 

Dr Lancet stated that the experimental process, known as CPX-351, is more effective than the standard “7+3” chemotherapy regimen. The CPX-351 delivery method combines the drugs cytarabine and daunorubicin into a single delivery, as opposed to “7+3” which delivered that drugs separately. 

“The two drugs together are delivered to the cell in the proper synergistic ratio that optimizes the cell-killing ability of these two drugs,” Dr Lancet said in a press release. “We think that by doing this, we can improve delivery to the cancer cells at the proper ratio.”

Among high-risk patients aged older than 60 with acute myeloid leukemia, the researchers found that there were 53% fewer deaths in the CPX-351 group within 100 days of transplantation, when compared to the “7+3” group. 

“These results suggest that CPX-351 may provide an effective bridge to successful transplant for a very poor-risk subgroup of acute myeloid leukemia patients,” they concluded. —David Costill