A Needle Worth Millions of Dollars.. "Shalafah" Reveals Treatment for Spinal Muscular Atrophy

Hebron - Afaq News - In light of the widespread interaction sparked by the campaign of the girl Basmalah, and the increasing questions about spinal muscular atrophy and the gene therapy that costs millions of dollars, Professor and Doctor Anas Shalalfa revealed details about the disease and the mechanism of treatment, confirming that the factor of time is a crucial difference in the results, and calling for the establishment of a permanent national and international fund to finance the treatment of those affected by rare diseases.

Shalalfa's remarks came during his appearance on the program "Talat Sabah," which is broadcast on the Ma'an satellite channel, the Ma'an radio network, and Radio Al-Rabia, hosted by media figure Adel Gharib, where he discussed in detail the nature of the disease and its genetic causes, the reality of the high-cost gene therapy, and the importance of early detection in improving the chances of benefiting from the treatment.

Shalalfa explained that spinal muscular atrophy is a rare genetic neurological disease that does not result from infection or virus, but rather is related to a genetic defect that affects the production of a protein necessary for the continued functioning of motor neurons responsible for transmitting signals to the muscles.

He pointed out that the main problem in the disease does not start from the muscles themselves, but from the motor neurons exiting the spinal cord, and as their function declines, the nerve signal does not reach the muscles normally, which gradually leads to their weakness and atrophy.

Genetics is at the heart of the disease.

Shallafah explained that spinal muscular atrophy is a recessive genetic disease, noting that if both parents are carriers of the genetic trait, the likelihood of having an affected child reaches 25% with each pregnancy.

He emphasized that this fact makes genetic testing, especially among families with a medical history, an important tool for prevention and early detection, calling for consideration of incorporating specialized tests into early screening programs for newborns.

He stressed that discovering the disease in the early days of a child's life can make a significant difference in treatment response, affirming that the most important rule in dealing with these cases is: the earlier the diagnosis and treatment, the greater the chance of preserving nerve function.

What is the truth about the injection that costs over two million dollars?

Regarding the treatment that has been increasingly discussed lately, Shallafah clarified that the injection in question is a gene therapy known as Zolgensma, which involves introducing a functional copy of the gene responsible for producing the protein necessary for the functioning of motor nerves.

He stated that the treatment is administered intravenously as a one-time dose, and its concept is to enable the body to produce the protein that the patient lacks due to the genetic defect.

Shallalfa pointed out that the price of the treatment reaches, according to what he mentioned during the interview, about 2.4 million dollars, explaining that the high cost is not only related to the substance contained in the dose, but also to a long system of scientific research, clinical trials, and the manufacturing and purification of the treatment, in addition to the limited number of patients benefiting from it due to the rarity of the disease.

Does the injection mean complete healing?

In one of the most prominent points that concern the families of patients, Shallalfa clarified that the treatment can support the motor neurons that are still alive and capable of regaining some of their functions, but it cannot restore the neurons that have been completely damaged and have actually died.

He added that improvement does not appear immediately after receiving the dose, but rather its results begin to appear gradually, stressing that delaying treatment means continuing to lose more nerve cells that cannot be recovered later.

He confirmed that the patient could achieve significant improvement in movement and functions if the nerves responsible for them are still intact and have not reached the stage of final damage.

He said that every day that passes before treatment can make a difference in the patient's condition and the desired final outcome.

The case of Basma brought the issue back to the forefront.

Shallafah addressed the campaign for the child Basma, which sparked widespread public interaction to raise funds for her treatment, praising everyone who contributed to the campaign and the sense of community solidarity that accompanied it.

However, he emphasized at the same time that the success of one campaign does not solve the root problem, questioning the fate of other children suffering from the same illness who do not receive media attention or have the ability to raise millions of dollars.

He confirmed that there are many families living the same pain while their voices do not reach the public, considering that relying solely on individual initiatives and donation campaigns cannot be a sustainable solution for a disease whose treatment costs exceed the capacity of most families.

Shallafah calls for the establishment of a national and international fund for rare diseases.

In light of these exorbitant costs, Shallafah proposed an initiative to create a specialized national fund for the treatment of rare diseases, which would operate under the supervision of specialized medical committees and with a clear and organized mechanism to ensure that support reaches those in need.

He revealed that discussions and communications have begun with personalities and health and executive entities inside and outside the country with the aim of pushing the idea towards an institutional and legal framework, ultimately leading to the possibility of developing it at the Arab and international levels.

He emphasized that dealing with rare diseases should not remain contingent on a family's ability to launch a campaign, or on the spread of a child's story on social media, stressing the need for a national database that identifies the number of rare cases, their needs, and treatment costs, which would help address international entities and institutions in a more organized manner.

Early detection.. a race against time

Professor Anas Shalalfa concluded by emphasizing that real investment should begin before reaching the stage of raising millions of dollars for treatment, by enhancing genetic testing for at-risk families, early detection in newborns, and building a healthcare system capable of monitoring rare genetic diseases from their onset.

The case of Basma is not just about one child or one expensive injection; it has opened the door to a larger file for families that may face the same disease in silence. Between a treatment measured in millions and time that may measure its impact in days, the question strongly posed by this file remains: Will the lives of patients with rare diseases continue to be hostage to a new fundraising campaign each time, or will Basma's story turn into a starting point for a national system that protects patients before time itself becomes part of the cost?