As 2021 comes to a close, a generous donor has offered to match contributions to support research in laboratory with 3 dollars for every one dollar. Our team has spent the last 25 years trying to create new therapies for ALS patients. Once diagnosed, these patients face an inexorable decline that begins with mild weakness and ends with full body paralysis, sparing only the muscles that move the eyes. Despite being completely unable to move, they can feel everything, and their brains remain sharp. On average, patients can lose the ability to breath independently by three years from their diagnosis. Sadly, most choose hospice and death over further decline into dependence and paralysis on a ventilator.
Please join our fight to help these patients by selecting the “Hopes and Dreams ALS” account in the drop-down menu. The progress made with your donations gives the gift of hope of more dignity, more quality time with loved ones in the face of this terrible disease. Thank you.
Boulis Laboratory Updates:
The Boulis laboratory has developed a new way to administer the drug riluzole to patients. Riluzole is the only drug proven to slow progression to that moment of reconning where the patients must choose between hospice and a ventilator. However, on average, Riluzole only gives patient an extra three months and has unpleasant side effects on thinking and emotions that lead many patients to quit the drug. The new method of delivery can increase the amount of drug getting to the dying motor cells by tenfold and eliminates the side effects. Moreover, the new method can overcome the tolerance that undermines the protective effect of the drug as disease progresses. Dr. Boulis and his team feel that these improvements could change the 3-month survival benefit by a year. That means an extra year of time spent with loved ones with more quality time. WE HAVE ALREADY TREATED TWO ALS PATIENTS, and are working with the FDA to approve trials that will prove the safety and efficacy of the approach.
In addition, we have also pioneered stem cell transplant into the spinal cord for ALS ten years ago and developed a new approach to the use of stem cells in ALS which may preserve the ability of these patients to breath for years. The laboratory has used the Nobel Prize winning method of Yamanaka (induced pluripotent stem cells: IPSCs) to make the motor neuron cells which die in ALS. By transplanting these cells into the nerve that patients use to breath close to the point where the nerve meets muscle, these neurons rapidly replace the lost innervation of the diaphragm. By stimulating these connections, the diaphragm can be activated allowing the patient to continue breathing. This method may ultimately allow ALS patients to go on breathing without a ventilator indefinitely.