New hope for spinal cord injuries: 17 patients receive a pioneering therapy in Madrid



A total of 17 patients with spinal cord injurynine o'clock Madrid's community and eight arrived from other parts of Spain, have begun to receive NC1 cell therapy, a drug developed entirely at the Hospital Majadahonda Iron Gate. It is the first of advanced therapy and non-industrial manufacturing authorized for hospital use by the Spanish Agency for Medicines and Health Products (AEMPS) in our country. The drug is the result of more than 20 years of research under the direction of Dr. Jesús Vaquero, head of the center's Neurosurgery Service, who died in the midst of the coronavirus pandemic in 2020.

Every year they are produced in Spain between 800 and 1,000 spinal cord injuries. Traffic accidents (also sports accidents), falls or, now in summer, dives are some of the most common causes of these injuries with devastating repercussions for those who suffer from them. The news for the approach of these injuries, are every day more hopeful. Just a few days ago he met that three people who had been left paraplegic managed to get back on their feet and walk with a technique developed by a team of Swiss researchers that is part of a clinical trial that is still ongoing, but has caused enormous expectation. The news was welcomed with "enormous joy" at National Hospital for Paraplegics of Toledo, as explained to The Newspaper of Spain the doctor Angel Gil Agudo, head of the rehabilitation service of the Castilian-Manchego health center.

Now, after a break forced by the coronavirus pandemic, the Madrid hospital Puerta de Hierro de Majadahonda finally begins the treatment with NC1 cell therapy, a drug that he had been researching for two decades. The medicine has a cost of more than 20,000 euros and the idea is that patients from all over Spain can be treated at the center. The treatment comes after 20 years of research and the publication of four clinical trials led by Dr. Cowboy.

Paraplegia or tetraplegia

Spinal cord injury is a disorder of the spinal cord, due to trauma, by disease (for example tumor) or congenital origin (spina bifida). Depending on the degree of damage of that injury it can be complete or partial. Depending on the level at which the damage occurs, the consequences can be more or less serious: tetraplegia or paraplegia. Traumatic spinal cord injury can cause physical disability and complications long-term complex: approximately 60% of chronic patients depend from a wheelchair and more than 97%, if the injury is complete at the time of the trauma.

Of all those who have started treatment, 12 have already received the full course of the drug

The new treatment that the Puerta de Hierro has begun to administer adds to the latest scientific advances for the approach to spinal cord injury. A new hope for the 40,000 injured in Spain. With nuances, because the hospital itself warns that if a person is a candidate for the therapy, they receive the treatment and are included in a long-term follow-up program to generate additional evidence on its efficacy and safety since, today, it is too early to deliver solid results.

Three months between the three doses

The first dose of NC1, as detailed from the Puerta de Hierro, has already been administered to 17 patients. According to the technical file, three months must pass between the administration of the first and second doses. Of all those who have started treatment, 12 have already received the full regimen. So far, all these patients have tolerated it well and no symptoms have been observed. serious adverse reactions in none of them. Between now and June 2022, the Puerta de Hierro Majadahonda Hospital will treat a total of 40 people with the full schedule of the drug, in accordance with the provisions of the AEMPS authorization conditions.

In fact, in November 2019, the Ministry of Health approved the financing of this first cell therapy developed by the National Health System itself. The cost of the drug was 21,577 euros and was intended to target adults with partial spinal cord injuries. During that year, the researchers estimated to apply it to a hundred patients, but the coronavirus crisis delayed that treatment that is now reactivated.

Increase the number of candidates

The goal of Puerta de Hierro is to gradually increase the number of candidates each year, and always in accordance with clinical results that maintain a favorable risk/benefit balance. To date, the region's Advanced Therapies Unit (UTA) has received a total of 78 requests for this treatment, 42 of them from other autonomous communities.

The center explains that all requests are reviewed and evaluated by a Selection Committee made up of doctors from Puerta de Hierro who are specialists in neurosurgery, clinical pharmacologyrehabilitation, neurology, urology and internal medicine, as well as members of the production room of Cellular therapytransplant coordination, nursing, psychology, social work or pharmacy, as well as a professional from outside the institution.

Currently, the researchers point out, it is still too early to offer solid results of efficacy in real clinical practice

When it is confirmed that a person is a candidate for therapy, they receive the treatment and, in addition, are included directly in a long-term multidisciplinary follow-up program that aims to generate additional evidence on the efficacy and safety of the drug. Currently, the researchers point out, it is still too early to offer solid results of efficacy in actual clinical practice.

Two phases of treatment

Treatment with this therapy, whose extensive research has been supported by the Community of Madrid, the Mapfre Foundation, the Rafael del Pino Foundation and the APYNME Association, is divided into two phases. In the first, the patient's own mesenchymal stem cells are extracted through a bone marrow tap. In the second phase, therapy is administered through an injection into the cerebrospinal fluid or the exact site of the spinal cord injury.

Advanced therapies are one of the main milestones achieved in recent years in dealing with diseases that have few treatment options.

The patient's stem cells are treated in a cell production room -Clean Room- where they are grown and expanded until the necessary dose is obtained to start the process. In the last year, the Puerta de Hierro has undertaken a series of refurbishment works in the center's White Room for Cellular Therapy in order to expand its production capacity and be able to reach thus to a larger number of patients.

Puerta de Hierro has a Multidisciplinary Unit of Advanced Therapies (UMTA). They are products for human use that are based on genes (gene therapies), cells (cell therapy) or tissues (tissue engineering) and, remember, they are one of the main milestones achieved in recent years in dealing with diseases that, to date, have few treatment options.



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