The target is expressed in several tumor types: it has been shown that several tumors express N-glycolil-GM3, such as non-small cell lung cancer (van Cruijsen et al., 2009), breast cancer (Vázquez et al., 1995; Moreno et al., 1998), melanoma (Alfonso et al., 2002), and several pediatric tumors of neuroectodermal origin (Scursoni et al., 2011, 2012). From the industry's perspective this is interesting because it allows a broad range of potential indications. Particularly in the case of non-small cell lung cancer, the expression of the target is greater than 70% (van Cruijsen et al., 2009). This provides two additional advantages: (1) the potential for combination of Racotumomab with other therapies used in more selected patient populations (patient with specific mutations or histological types) without the need of prior evaluation of the presence of the target in the tumor and (2) even if the detection of the target ganglioside (N-glycolil-GM3) were needed, the fact that it is an immunohystochemical evaluation, makes it technically easy to perform, of low cost and widely accessible.

It has an innovative mechanism of action (Figure 1): anti-idiotype antibodies are a useful strategy to elicit an immune response toward a ganglioside, which is a scarcely immunogenic molecule in itself.

It is highly immunogenic (Figure 2) and shows clinical benefit: Racotumomab is a mAb, but is used as a vaccine. Only a small quantity of Racotumomab (1 mg) is needed per dose. It has been shown that Racotumomab is able to elicit a strong humoral and cellular immune response (Alfonso et al., 2002; Díaz et al., 2003; Guthmann et al., 2004, 2006; Hernández et al., 2008, 2011) and that this response has a positive impact in patient survival (Guthmann et al., 2004; Alfonso et al., 2007; Neninger et al., 2007). In a proof of concept trial, patients with NSCLC treated with Racotumomab had a longer overall survival in comparison to a placebo group (final results submitted for publication).

It can be used in a broad target population: despite the fact that more than 65% of all malignancies and more than 70% of the deaths associated to cancer occur in patients beyond 65 years old (Lynn et al., 2003), elderly patients continue to be underrepresented in clinical trials. The incidence of cancer is 10 times larger and death rate is 16 times larger in this group than patients below 65 years of age (Lynn et al., 2003). When the treatments evaluated in younger patient populations are approved and then used in the clinical setting and in elderly patients frequently the results obtained are not the same. Due to comorbidities and polimedication the risk of side effects, toxicity and complications is increased, and the tolerance to onco-specific treatments is reduced. Several studies have shown that patients more than 65 years old tend to have a greater risk of bone marrow depletion, neutropenia, infections, and neurotoxicity with the use of chemotherapy (Balducci, 2001, 2003; Balducci and Repetto, 2004; Belani, 2005; Gridelli, 2008). Cancer treatment in pediatric population presents a similar challenge. In addition, traditional cancer agents have impact in developing organs and systems and are likely to produce irreversible, negative changes.

Well-tolerated and safe products such as Racotumomab continue to be very much needed in these specific patient populations. This is also very important for diseases with late diagnosis and no chance of cure, because in this context patients and their families tend to recur to alternative therapies which may be costly, ineffective and unsafe.

The schedule of vaccination is comfortable and simple: it consists of five intradermal doses (1 every 2 weeks) followed by subsequent monthly intradermal doses.

The administration of the vaccine is quick and easy: the route of administration of Racotumomab and the fact that this product does not require the safety precautions that are needed when manipulating chemotherapeutic agents, nor costly processing or time consuming infusions makes this treatment interesting, since it can be delivered by a nurse at home, at the pharmacy or at the clinic. Although in the clinical trials it is recommended that the patients remain 1 h in observation after the first 2 doses, no threatening immediate reactions have been observed so far in more than 700 treated patients and no further surveillance is required if the vaccine was well-tolerated. All these factors favor patient compliance to therapy and have an impact on quality of life.

Good tolerance and feasibility of combination with standard therapies: preservation of the patient's quality of living is extremely important, especially when there are no chances of cure and palliative therapies are the only option. Racotumomab is mostly associated with mild to moderate injection-site reactions (local erithema, induration, and pain), which disappear within 24–48 h. Systemic reactions, such as flu like symptoms and chills are less frequent, reversible, and self-limited. A favorable safety profile allows administration of monthly booster doses during a long period of time to maintain the immune response beyond disease progression, possibilities of treatment combinations with a broad range of standard therapies (radiotherapy, chemotherapies, etc.,) and use in special populations such as elderly and pediatric patients. Especially in NSCLC Racotumomab could play a role in maintenance therapy, with an acceptable and easy administration schedule and a favorable safety profile, alone or in combination with other agents.