Funding Opportunities

Part the Cloud (PTC), a program through the Alzheimer's Association, announces a targeted challenge funding program focused on increasing early phase human trials (Phase 1 and Phase 2) of potential therapies tailored to the genetic underpinnings of Alzheimer’s disease and related dementias internationally. A number of factors and processes likely contribute to the development of brain diseases such as Alzheimer's disease, and these may not be the same for every individual. More than 100 genes have been associated with the development of Alzheimer’s as either risk or protective factors, each one influencing biological changes in the brain. Because of this variation in disease processes between individuals, there is a significant opportunity for personalized gene editing and therapy in Alzheimer’s. Evidence from the trials that led to the recent approvals for two new therapies targeting the abnormal accumulation of beta-amyloid “plaques” suggest potential subpopulations of responders and non responders. The complex interplay of factors and processes leading to the development of neurodegenerative disease points towards combination therapy of two or more drugs with behavioral interventions that target distinct biological processes as the likely future of treatment for Alzheimer’s and the other brain diseases that cause dementia. Personalized medical approaches to address an individual’s biological circumstances is the future of treatment for Alzheimer’s throughout the disease continuum. Gene targeting therapies have existed for some time, but only recently have they become viable for clinical use. Other new technologies such as small interfering RNA (siRNA), antisense oligonucleotides (ASOs), and CRISPR are being evaluated in the clinic for a range of diseases like sickle-cell anemia and heart disease. In early 2023, the U.S. Food and Drug Administration (FDA) approved ASO approaches for treatment of amyotrophic lateral sclerosis (ALS) and in late 2023, the FDA approved a CRISPR based therapy for sickle cell anemia, moving these approaches to care in real time. These recent approvals demonstrate the timeliness of leveraging approaches that build on the technological advances of ASOs, siRNA and CRISPR, as well as other approaches for gene modifications for Alzheimer’s and other brain diseases that cause dementia. The Alzheimer’s Association and Part the Cloud are already leading the way in these therapeutic approaches for Alzheimer’s disease and other related dementias. Previous Part the Cloud awards include an ASO therapy targeting tau, a gene therapy targeting brain derived neurotrophic factor (BDNF), and an adult stem cell therapy. The BDNF gene therapy trial has shown that a single injection can lead to prolonged increases in brain BDNF levels. While these advances are pushing the field forward, a greater emphasis on these approaches, or combinations of these approaches with other therapies, could lead to unprecedented acceleration in the treatment of Alzheimer’s. Slowing or preventing Alzheimer’s disease is a major public health need, and it is essential that the field begin to examine these promising new approaches. While many academic investigators and companies have successfully discovered candidate therapeutics, and have succeeded in the non-clinical stages of development, funding for the next phase to transition into human testing can be a major stumbling block. Most government and non- government grant mechanisms support preclinical development or mid to later clinical trials testing drug efficacy in humans, but there are few mechanisms for supporting the critical earlier Phase I trials in humans or have restrictions on funding that limit the forward momentum for therapeutic approaches.