Carlitos is a 6-year-old Bolivian boy. He lives with his parents, Mario and Miriam, and his younger brother, Daniel. In May 2008, their family life was rattled when Carlitos was diagnosed with Mucopolyssacharidosis II (MPS II). The disease, most commonly known as Hunter’s Syndrome, is a rare disease that affects 1 in 180,000 children – almost exclusively boys – and gradually leads to death if untreated. While symptoms vary by patient, the most common include: coarse facial features, enlarged head, joints of fingers, arms, and legs held in partial tension, airways obstruction, sleep apnea, enlarged abdomen due to enlarged liver and spleen, short stature, abnormal heart valves that may eventually lead to cardiac arrest, and others.
After their son was diagnosed with Hunter’s Syndrome, the family travelled from Bolivia to the United States to seek a second opinion. They first went to Miami and then Washington. In both cities, the diagnosis was confirmed. The family thus, initiated their quest for help in obtaining the treatment that could save their son. They learned that the only hope to cure Carlitos was to obtain a medicine that cost around $22,000 per month. The family could not afford to pay for the medication and focused their efforts on seeking help to obtain the medicine through other means.
While still in the United States, Carlitos’ parents learned that the manufacturer of this medicine would be conducting a clinical trial that was to be conducted in Porto Alegre, Brazil. In June 2008, they were told that the pharmaceutical was recruiting candidates for the investigation. Mario and Miriam then, full of hope, took their family back to Bolivia, sold their house, car, and other possessions to relocate to Porto Alegre.
Unfortunately, after Carlitos had already been accepted as a candidate, the study was suspended indefinitely leaving the family once again without the treatment for their child.
Mario, Miriam and other members of their family who joined in solidarity, began sending letters to many organizations and people, looking for new options that could help them with the provision of the medicine. Meanwhile, Carlitos’ health was deteriorating further and he had started showing frequent respiratory problems related to laryngitis and bronchitis. The family became more desperate as the days went by and they were unable to get access to the much-needed treatment for their child. In June 2009, AID FOR AIDS International (AFAI) received one of these letters. Familiar with the detrimental problems caused by lack of access to necessary treatment we, AFAI, took on the task of finding a solution for Carlitos’ case.
We contacted the pharmaceutical company, manufacturer of the medicine, explaining the facts surrounding Carlitos’ case and asking for help with the provision of the treatment. The first reply we were given was that they would review the case and let us know about their decision soon. During the ensuing weeks, we insisted with emails and phone calls that yielded little or no answer. Finally, on July 14th, AFAI received an email from the company — a negative response to the request. The drug maker had denied the medicine with the argument that Carlitos was living in Brazil, where he was not a citizen.
The pharmaceutical company refused to see that Carlitos and his family had moved to Brazil in order to receive the medication, and were willing to move anywhere in the world to help save their son. Shocked by the pharmaceutical company’s response, and armed with their commitment to save Carlitos’ life, the AFAI staff did not give up. Instead, we sent a powerful email message back to the pharmaceutical rebuking the answer that had been provided. We also got in contact with Patricia Janiot, senior anchor at CNN en Español who, informed us that the network was interested in doing a story on Carlitos’ case.
After sparking media attention and AFAI’s insistence, only a week past before we heard from the drug company again. They had reconsidered Carlitos’ case—AFAI’s efforts had paid off. The representative from the company stated that not only would the clinical trials would be re-activated in September, but also, that Carlitos would be eligible to participate in the trials.
Throughout the following weeks, AFAI’s staff continued tracking the progress of this case and the once-stalled clinical trials. Maintaining a constant line of communication with the parents, AFAI was notified that on Wednesday September 23, Carlitos started his treatment to help him combat this life-threatening disease. During these clinical trials, seven other kids will be treated too.
The AFAI team wishes Carlitos and his family the best and hopes for his recovery. Meanwhile, AID FOR AIDS International will continue working to help people, reaffirming its commitment to SAVING LIVES, ONE BY ONE.