Site Selection for Cancer Immunotherapy Studies

Rapid advances in cancer immunotherapy treatments have fueled a tremendous investment in this promising area of research.

While sponsors and investigators rush to get immunotherapy treatments (such as checkpoint inhibitors, adoptive cell transfer, therapeutic antibodies and immune system modulators) into the clinic, it’s important not to overlook the operational considerations associated with cancer immunotherapy studies.

As we all know, cancer studies are more complex than studies in most other therapeutic areas.

And furthermore, cancer immunotherapy studies are more complex than most typical cancer studies.

This complexity requires an additional level of detailed planning to properly address the nuances of an immunotherapy study. Failure to do so could cause you to prematurely end a study – even though the therapy is actually working – or end up with extended timelines, bad data and cost overruns.

Site Selection – The First Critical Operational Decision

cancer immunotherapy site sectionProper site selection is critical to the success of any oncology study. It’s even more critical in the rapidly expanding area of cancer immunotherapy.

To get the right data within your timelines, you need:

  1. The investigators who have access to the patients your study requires
  2. The right mix of sites (academic, government and community-based) that are able to get patients enrolled within your timeline
  3. Sites that are skilled and experienced in handling the type of treatment being administered
  4. Sites that understand how to deal with the differences in how the immune system reacts to these types of treatments

These variables directly affect your study cost and data quality.

In addition, if your study is phase I, you will also have to evaluate their dose-escalation experience (and we address that here).

Investigational Product Handling

Several of the immunotherapy studies we’ve managed recently have required us to follow the NIH BioSafety Level 2 (BSL-2) Guidelines for the handling of therapies utilizing microorganisms associated with human diseases.

Biosafety levels, or BSLs, are designations to classify the level of danger of a biological agent in an enclosed laboratory facility.

The designations and risks associated are as follows:

Level 1 – The lowest level, this applies to agents that pose minimal threat to lab workers and the environment.

Level 2 – This includes work with agents that are associated with human disease – pathogenic or infectious organisms posing a moderate health hazard.

Level 3 – Indigenous or exotic agents that cause serious or lethal disease via aerosol transmission.

Level 4 – Extremely dangerous agents that pose risk of life-threatening diseases for which there are no vaccines or cures.

If your treatment is BSL-2, it will affect your site selection. Not all sites have the processes or infrastructure to handle these types of agents, which require the use of surgical gowns, gloves, goggles, safety glasses and possibly a Class II biological safety cabinet.

Additionally, the local investigational biosafety committee (IBC) must approve the site’s level of preparation to handle these types of agents. This was a challenge for Medelis during our first cancer immunotherapy studies (back in 2008) because at that time, not all sites had an IBC. Thus, we had to work with the sites to help them establish one. As Level 2 immunotherapy agents become more prominent, institutions are becoming better prepared, but many sites still require assistance with infrastructure issues.

Biosample Collection

A key goal of cancer immunotherapy studies is to collect and utilize specimens from patients for immuno monitoring, and for developing and credentialing biomarkers that will assess the pharmacodynamic effects or that can serve as predictors of response.

Thus, it’s critical to plan for the logistics of sample collection, processing and shipping during trial planning. Consider:

  • Containers – Do you require specialized containers?
  • Transport – What requirements should be in place for the couriers? Most immunotherapy treatments require temperature control during transport and storage.
  • Customs – If you’re running a global study, what are the implications of samples passing through customs? This may impact the countries you select.

In the Aduro Biotech GVAX Pancreas Prime study, we handled this by working closely with the drug distributor to coordinate just-in-time delivery with the vaccine and dry nitrogen shippers. We also worked closely with the site to ensure that there were no patient treatment delays.

Product Storage

Many of the new immunotherapy treatments require specialized storage procedures. Review the site’s pharmacy procedures for monitoring the temperature of stored products, along with how they report deviations.

For many therapies, an ultra-low temperature freezer is required, and in recent years, vendors have started producing this type of freezer in sizes that fit in a typical site’s pharmacy.

Serious Adverse Events (SAEs and irAEs)

The evaluation and handling of SAEs in cancer immunotherapy studies are critical to the outcome of the study. The majority of the events will be autoimmune-related, since the body will start attacking itself.

A failure to clearly understand how the immune system responds to your treatment could cause an improper evaluation of response data and cause you to prematurely end your trial… when the immunotherapy treatment is actually working!

This is such a critical area, we’ll address it in full detail in a future article.

Experience Is the Key

New scientific breakthroughs always come with a learning curve. It’s no different with cancer immunotherapy research.

To avoid the risk of misinterpreting your data or missing trial deadlines, work with the sites and vendors that have experience in immunotherapy studies and can help you avoid the numerous potential pitfalls.

Connect with us if you’d like assistance with site selection or with planning the details of your upcoming immunotherapy study. We’ve managed 15 of them recently and have the resources, vendors and experience to handle the challenges you might encounter.

If you’d like a more comprehensive review of the strategic aspects of an immunotherapy study, check out our webinar – How Immune-Related Response Criteria is Changing Immunotherapy Treatments.