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RESEARCH AND TREATMENT DEVELOPMENT UPDATES |
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RESEARCH AND DRUG DEVELOPMENT TIMELINE
The ADNP Kids Research Foundation believes that collaboration and transparency are key in accelerating treatment research and development in the quest to find viable treatments to help our children with ADNP Syndrome. We intend to keep families informed of the research and development on this page.
This page will be a working document with a living timeline and will be updated as changes are made. Clinical trials are complex but we believe that it is important that families are kept informed. Please check back frequently for updates.
SCROLL DOWN TO THE BOTTOM OF THE PAGE FOR THE LASTEST UPDATES
The ADNP Kids Research Foundation believes that collaboration and transparency are key in accelerating treatment research and development in the quest to find viable treatments to help our children with ADNP Syndrome. We intend to keep families informed of the research and development on this page.
This page will be a working document with a living timeline and will be updated as changes are made. Clinical trials are complex but we believe that it is important that families are kept informed. Please check back frequently for updates.
SCROLL DOWN TO THE BOTTOM OF THE PAGE FOR THE LASTEST UPDATES
NAP-peptide/CP201/Davunetide/NAP
(*The ADNP Kids Research Foundation does NOT support this project or associated companies)
CP201/NAP is a potential treatment for ADNP Syndrome derived within a peptide of eight amino acids called NAP. This peptide was discovered by Professor Illana Gozes and was run in human trials prior to the discovery of ADNP syndrome. To date, this peptide has not been in any human trials of children/patients with ADNP syndrome, which is a question that we are often asked. The foundation attempted to support this treatment in ADNP syndrome since it's inception in 2016 until 2021. However, the ADNP Kids Research Foundation no longer supports Professor Gozes and does not support ATED Therapeutics.
HISTORY:
In 2007, Professor Illana Gozes co-founded Allon Therapeutics where she began and ran large scale clinical trials in both Alzheimer’s, Schizophrenia and Progressive supranuclear palsy (PSP) patients. In 2012, following the unfortunate unsuccessful outcomes in both FDA Phase 3 clinical trials, this company dissolved.
In 2017 Dr. Gozes co-founded the company Coronis Neurosciences (Coronis). Coronis obtained ODD in 2018, the foundation attended the PRE-IND, but the drug failed to move into any clinical trials. In 2021 this company dissolved.
Between 2016 and 2021, the foundation made numerous attempts to assist in helping provide movement by offering to fund and perform a pilot study to look for efficacy and to provide much needed independent replication and validation, but these requests were turned down. The foundation also received the support of NCATS/NIH and facilitated meetings with the NIH and Mount Sinai Seaver Autism Center in an effort to assist Dr. Gozes and her biotech company forward.
In 2021 Dr. Gozes co-founded ATED Therapeutics her 3rd biotech startup, and the foundation has decided to end our relationship with Dr. Gozes and her new team. Any requests for information about how to fund their work should be sent directly to ATED.
(*The ADNP Kids Research Foundation does NOT support this project or associated companies)
CP201/NAP is a potential treatment for ADNP Syndrome derived within a peptide of eight amino acids called NAP. This peptide was discovered by Professor Illana Gozes and was run in human trials prior to the discovery of ADNP syndrome. To date, this peptide has not been in any human trials of children/patients with ADNP syndrome, which is a question that we are often asked. The foundation attempted to support this treatment in ADNP syndrome since it's inception in 2016 until 2021. However, the ADNP Kids Research Foundation no longer supports Professor Gozes and does not support ATED Therapeutics.
HISTORY:
In 2007, Professor Illana Gozes co-founded Allon Therapeutics where she began and ran large scale clinical trials in both Alzheimer’s, Schizophrenia and Progressive supranuclear palsy (PSP) patients. In 2012, following the unfortunate unsuccessful outcomes in both FDA Phase 3 clinical trials, this company dissolved.
In 2017 Dr. Gozes co-founded the company Coronis Neurosciences (Coronis). Coronis obtained ODD in 2018, the foundation attended the PRE-IND, but the drug failed to move into any clinical trials. In 2021 this company dissolved.
Between 2016 and 2021, the foundation made numerous attempts to assist in helping provide movement by offering to fund and perform a pilot study to look for efficacy and to provide much needed independent replication and validation, but these requests were turned down. The foundation also received the support of NCATS/NIH and facilitated meetings with the NIH and Mount Sinai Seaver Autism Center in an effort to assist Dr. Gozes and her biotech company forward.
In 2021 Dr. Gozes co-founded ATED Therapeutics her 3rd biotech startup, and the foundation has decided to end our relationship with Dr. Gozes and her new team. Any requests for information about how to fund their work should be sent directly to ATED.
KETAMINE - CURRENT INFORMATION:
In early 2019 Sandra Sermone and Matt Davis, after investigating and collecting a large amount of clinical data, brought the idea of low dose ketamine as a potential treatment option for ADNP Syndrome. Dr. Alex Kolevzon and the Seaver team then thoroughly investigated that clinal data which suggests that low dose Ketamine increases ADNP expression and determined that it looks promising as a possible treatment for ADNP Syndrome.
Sermone and Kolevzon announced at the November 2019 ADNP Syndrome Family Conference, that Mount Sinai will be moving forward into a clinical trial study for Ketamine.
In early 2019 Sandra Sermone and Matt Davis, after investigating and collecting a large amount of clinical data, brought the idea of low dose ketamine as a potential treatment option for ADNP Syndrome. Dr. Alex Kolevzon and the Seaver team then thoroughly investigated that clinal data which suggests that low dose Ketamine increases ADNP expression and determined that it looks promising as a possible treatment for ADNP Syndrome.
Sermone and Kolevzon announced at the November 2019 ADNP Syndrome Family Conference, that Mount Sinai will be moving forward into a clinical trial study for Ketamine.
- This will involve testing safety in pediatric patients with ADNP Syndrome and assess preliminary efficacy signals.
- This study would be independent of the CP201 study and the Seaver team is currently working on the protocol and regulatory submission of Ketamine as a repurposed drug for treatment with the FDA.
- Dr. Kolevzon plans on submitting to the FDA by the end of the year, with a couple month turn around time with the FDA, and hopes to start the study by March of 2020.
- If the FDA comes back sooner, the trial can start sooner.
- The study duration is planned as 8-12 weeks. The study design and timeline could change upon regulatory review.
ADDITIONAL INFORMATION:
In regards to these study proposals, timelines and cost for clinical trials; the Seaver team is still working on the clinical protocols and will share more detailed information as soon as possible and we will update this page when we receive that information.
In general, they intend to enroll 20-30 patients in each placebo-controlled study. Each patient will ultimately receive the study drug either as part of the cross-over design or in the open-label extension.
In regards to these study proposals, timelines and cost for clinical trials; the Seaver team is still working on the clinical protocols and will share more detailed information as soon as possible and we will update this page when we receive that information.
In general, they intend to enroll 20-30 patients in each placebo-controlled study. Each patient will ultimately receive the study drug either as part of the cross-over design or in the open-label extension.
COST:
It is difficult to estimate the overall cost of drug development through regulatory approval for any of these treatments until the Seaver team has preliminary efficacy signal in ADNP children. Depending on a multitude of factors, the path to approval could differ as well as the cost. The Seaver team will provide cost estimates for the studies described above as soon as the design and timelines are finalized. When we receive those estimates we will update that information on this page.
It is difficult to estimate the overall cost of drug development through regulatory approval for any of these treatments until the Seaver team has preliminary efficacy signal in ADNP children. Depending on a multitude of factors, the path to approval could differ as well as the cost. The Seaver team will provide cost estimates for the studies described above as soon as the design and timelines are finalized. When we receive those estimates we will update that information on this page.
UPDATE: 1/1/2020
After multiple requests from the foundation and other academics, the Gozez Lab at Tel Aviv University refuses to share their validated ADNP mouse model. This is highly surprising to all in the research community and rather than waiting, the foundation has just paid Mount Sinai to begin a new mouse model through a company called OZGENE. One the line is developed, Mount Sinai will confirm/validate the mouse model and then the line will be stored at JAX Labs and shared with ALL collaborative researchers interested in ADNP. (update summer 2020, due to the pandemic, the project has come to a stop and will start as soon as the lab is back up and running)
After multiple requests from the foundation and other academics, the Gozez Lab at Tel Aviv University refuses to share their validated ADNP mouse model. This is highly surprising to all in the research community and rather than waiting, the foundation has just paid Mount Sinai to begin a new mouse model through a company called OZGENE. One the line is developed, Mount Sinai will confirm/validate the mouse model and then the line will be stored at JAX Labs and shared with ALL collaborative researchers interested in ADNP. (update summer 2020, due to the pandemic, the project has come to a stop and will start as soon as the lab is back up and running)
UPDATE: 1/22/2020
Today marks a significant step forward on the road to finding effective treatments for ADNP Syndrome. The FDA has just given the team at Mount Sinai approval to begin the Phase 2 study on Ketamine. Press release is attached below and more information should be out in the near future about the cost and trial design. We enthusiastically welcome the first clinical trial for children with ADNP syndrome.
PRESS RELEASE: https://www.adnpfoundation.org/ketamine.html
Today marks a significant step forward on the road to finding effective treatments for ADNP Syndrome. The FDA has just given the team at Mount Sinai approval to begin the Phase 2 study on Ketamine. Press release is attached below and more information should be out in the near future about the cost and trial design. We enthusiastically welcome the first clinical trial for children with ADNP syndrome.
PRESS RELEASE: https://www.adnpfoundation.org/ketamine.html
UPDATE: 2/4/2020
Ketamine Trail Update: The team at Mount Sinai is hopeful that we will begin the ketamine study by May 1st. The enrollment and visit details will follow as soon as the Mount Sinai IRB is approved.
The Phase 2 study is estimated to cost approximately $130,000, but a hard number will be available soon. As soon as we get the final information from Mount Sinai, we will be launching a global fundraiser. If anyone finds any grant opportunities, please forward the information to [email protected].
Ketamine Trail Update: The team at Mount Sinai is hopeful that we will begin the ketamine study by May 1st. The enrollment and visit details will follow as soon as the Mount Sinai IRB is approved.
The Phase 2 study is estimated to cost approximately $130,000, but a hard number will be available soon. As soon as we get the final information from Mount Sinai, we will be launching a global fundraiser. If anyone finds any grant opportunities, please forward the information to [email protected].
UPDATE: 5/7/2020
Our team at Mount Sinai just received the IRB approval to start recruiting for the Ketamine study. Very soon we will post information about the study but our team is working from home so we anticipate it taking a week or two. The plan is to open the trial for recruitment while launching a fundraising campaign.
Our team at Mount Sinai just received the IRB approval to start recruiting for the Ketamine study. Very soon we will post information about the study but our team is working from home so we anticipate it taking a week or two. The plan is to open the trial for recruitment while launching a fundraising campaign.
UPDATE: 5/14/2020
Ketamine Trail Update: Enrollment
Enrollment will be posted soon on clinicaltrials.gov - study to begin August 2020
Ketamine Trail Update: Enrollment
Enrollment will be posted soon on clinicaltrials.gov - study to begin August 2020
UPDATE 7/1/2020
Ketamine Trial Update: "WARRIOR FUN RUN" Fundraiser
The amazing and supportive families of children with ADNP Syndrome participated in our 1st ever virtual fun run, and raised almost $150,000 to fund the Phase 2 ketamine study in one month!
Thank you so much to all of the participants and generous donors. Because of your, the first ever drug trial for ADNP Syndrome will begin!
Ketamine Trial Update: "WARRIOR FUN RUN" Fundraiser
The amazing and supportive families of children with ADNP Syndrome participated in our 1st ever virtual fun run, and raised almost $150,000 to fund the Phase 2 ketamine study in one month!
Thank you so much to all of the participants and generous donors. Because of your, the first ever drug trial for ADNP Syndrome will begin!
UPDATE 9/10/2020
We are incredibly happy to announce that the very first ADNP patient has received the drug for our low dose ketamine phase 2 drug trial today at the Seaver Autism Center at Mount Sinai in New York. The family will come back for 4 weeks for evaluations and then the team will see the next patient. We are hopeful for a smooth clinical trail that will result in helpful information about the safety and efficacy of low dose ketamine as treatment for ADNP syndrome.
We are incredibly happy to announce that the very first ADNP patient has received the drug for our low dose ketamine phase 2 drug trial today at the Seaver Autism Center at Mount Sinai in New York. The family will come back for 4 weeks for evaluations and then the team will see the next patient. We are hopeful for a smooth clinical trail that will result in helpful information about the safety and efficacy of low dose ketamine as treatment for ADNP syndrome.
UPDATE 2/1/2021
Ketamine Trial Update: HALFWAY MARK!
We are halfway through our Ketamine trial at the Seaver Autism Center at Mount Sinai in New York, led by Dr. Alex Kolevzon. The trial is running smoothly and we would like to recognize and thank the 10 families who have enrolled their children in this very important study.
Ketamine Trial Update: HALFWAY MARK!
We are halfway through our Ketamine trial at the Seaver Autism Center at Mount Sinai in New York, led by Dr. Alex Kolevzon. The trial is running smoothly and we would like to recognize and thank the 10 families who have enrolled their children in this very important study.
UPDATE 4/30/2021
ADNP Kids Research Foundation has fully funded an important iPSC study costing $95,000.
The study aims to:
ADNP Kids Research Foundation has fully funded an important iPSC study costing $95,000.
The study aims to:
- Phenotypic characterization of human glutamatergic neurons derived from isogenic ADNP mutation iPSC's.
- Compound testing in rescuing morphology deficits of the ADNP-syndrome in vitro models.
- Synergistic effects of ketamine and NAP in rescuing morphology deficits in ADNP-syndrome in vitro models.
- Validation of ketamine in rescuing morphology deficits in neurons derived from iPSC lines from ADNP-syndrome patients.
