#Slide 1. Friedreich’s ataxia is an inherited neuro-degenerative disease, affecting the body gradually after its onset. The patient may experience a slurred speech, blurred vision, imbalanced body movements, drunken gait, poor co-ordination and other related problems including diabetes, heart ailments etc. which often gets worse over time and may also cause early death in certain cases.
#slide 2. Talking about FA in India – the concentration of the sufferers of this disease is very low when compared to other big countries like USA and UK. And may be because of this the awareness as well as the research on the disease is almost low.
#Slide 3. SAMAG(seek a miracle ataxia group) is a private organization that was started by Chandu, an FAer from Hyderabad India with its branches in various Indian cities like Indore, Pune. The main motive of this organization is to raise awareness of FA among people and help the FAers along with rare disease patients and their families. The group members participate and also help in organizing medical camps like the DNA camp in Indore that was recently organized by AIIMS.
#Slide 6. Although there is no cure for FA yet, but the researchers and scientists have been working for years to find a cure for FA very soon.
#Slide 7. Trials are now being conducted on patients, many of which are showing a positive result. This has increased the expectancy of patients of living a better life.
#Slide 8. Here we see the research pipeline of FARA (Freidreich’s Ataxia Research Alliance). Out of all the drugs, I would like to emphasize mainly on the EPO and EPI743 by Edison pharmaceuticals as these two have successfully crossed the phase I and Are about to step into the phase III of drug trials
#Slide 9. The abnormality in the frataxin protein levels is the main cause of this disease. The EPO (Erythropoietin) drug has been found to increase the activity of frataxin gene in FA patients, and hence the effects of the is great. On the other hand EPI 743 improves the mitochondrial dysfunction that is again one of the causes of FA. Both of these drugs are well tolerated by patients.
#Slide 10. I would also like to highlight the stem cell therapy, which is carried out around various private hospitals and clinics and have shown positive results in many ailments including FA. But, as all of the patients cannot afford the big fees of this therapy, I would also like to request the doctors of the govt hospitals to make arrangements of the same at a lower cost.
#Slide 11. We want these drug trials to be initiated by the doctors and researchers in India, but the rules and regulations of the Indian Government on the clinical trials on patients is a big hindrance on the hopes of patients
#Slide 12. On behalf of all the ataxia patients, I wrote a letter to the president of India, Dr. Pranab Mukherjee last year and requested him to remove the ban imposed on drug trials in India, but got no positive response.
#Slide 13. The views of the guardians and patients suffering with ataxia were also sent to the president along with the letter where all of them have appealed the govt officials to relax the restrictions on clinical trials.
#Slide 14. We patients expect firstly from the Indian govt. to remove the restrictions on clinical trials. Secondly, we request the researchers and doctors of India and abroad to collaborate and start with clinical trials, especially the trials which are on the phase 3. Thirdly, almost everyone of us would like to volunteer for the stem cell therapy and drug trials. We have waited for long and now expect them to be started as soon as possible.
Friedreich’s ataxia is an autosomal recessive inherited disease that progressively damages the nervous system of the sufferer. As a result, the FAer experiences slurred speech, blurred vision, imbalanced gait and body movements, poor muscle co-ordination, diabetes, heart ailments and many other problems that worsen over time. An early death can also be an outcome of FA.
When talking about FA in India, the concentration of people with this disease is comparatively lower than that in the USA and UK. Moreover, the awareness about this neuro-degenerative disease is pretty low among people. However, back in 2006, Mr. Chandu Prasad George (FA sufferer) founded SAMAG- Seek A Miracle Ataxia Group, with an ambition of spreading awareness about FRDA and also support the sufferers and their families.
As far as a cure for this disease is concerned, then sadly there is no sure shot treatment available yet. However, the scientists and researchers from all over the globe have begun to work for the development of an effective treatment to fight and prevent this progressive ailment. Clinical trials of various drugs and therapies are being conducted on patients with many showing positive results.
But, no kind of drug or therapy trials are even started in India, which is demoralizing for every sufferer. Although the doctors and researchers are willing to carry out clinical trials on the patients, but they are obstructed by the restrictions imposed on clinical trials on humans by the government of India.
Last year, on behalf of all FAer’s, a letter was written to the president of India, with a request to remove the ban and allow the clinical trials to be conducted on the volunteer patients in India. But, this effort hardly accomplished any positive result.
Today again, all we patients would like to make a request from the Indian government to grant the necessary permissions to the authorized doctors and researchers who are willing to carry out the drug and therapy trials, as all of us are seeking for a miraculous treatment.