Amazing Team Research at the California Stem Cell Program
By Don C. Reed
As high as $20 million each, the disease team grants are the most spectacular research projects offered by the California stem cell program.
The challenge is enormous: to succeed, each team of scientists must bring a therapy to human trials within just four years. At every step of the way milestones of progress must be met, or the funding stops—and every disease these teams will challenge has been declared chronic: incurable. A victory here would change medical history.
How are they doing? On March 19th, the next public meeting of the California stem cell program’s board of directors, there will be an update on the 25 teams in action now.
Here are the teams: may they all succeed.
1. ALZHEIMER’S DISEASE: Alexandra Capela, of Stem Cells Inc., wants to transplant neural stem cells directly into the hippocampus portion of the brain, attempting to “markedly restore memory and thus, quality of life, for patients.”
2. BLINDNESS: Mark Humayun, USC, estimates that “by 2020, over 450,000 Californians will suffer from vision loss or blindness due to age-related macular degeneration…the loss of a layer of cells at the back of the eye…this can be overcome by transplanting (new) cells derived from embryonic stem cells…”
3. BLINDNESS: Henry Klassen, UC Irvine, hopes to use cells to treat retinitis pigmentosa, (RP) a severe disease of the eye. His approach is to “save the light-sensing cells of the eye… an application will be made to the FDA… following approval, a small number of patients with severe RP will be injected with cells in their worse-seeing eye.”
4. BRAIN TUMORS: Karen Aboody, City of Hope, has built a stem cell bank to make her prospective attempt at destroying brain tumors widely available. Her techniques may also remove “solid tumors throughout the body”…” applicable to other deadly growths.
5. CANCER: Dennis Slamon, UCLA, points out that “ a minor population of cancer stem cells drives the growth of an entire tumor…” These trigger-like cells can bring back the cancer which appeared to be gone. Fortunately, says Slaymon, “our lead drug can inhibit the growth of cancer stem cells.”
6. CANCER: Judith Shizuru, Stanford, hopes to “enable chemotherapy free transplants”, with the use of a selected antibody. The same technique might also save the lives of children born with an immune-system defect, Severe Combined Immune Deficiency (SCID), who often die before the age of two.
7. CANCER: Antoni Ribas, UCLA, seeks to reprogram stem cells, hoping to “redirect the patient’s immune response to specifically attack the cancer….”
8. CRITICAL LIMB ISCHEMIA: John Laird, UC Davis, hopes to benefit two million Americans currently at risk of leg amputation or death. Since a key problem in this condition is low oxygen level in the affected limbs, Laird has shown that stem cells can “migrate into the areas of lowest oxygen…wrap around the damaged…blood vessels…and secrete (helpful) factors where needed most…” A stem cell hug of life!
9. DIABETES: ViaCyte, Inc. is challenging diabetes, using embryonic stem cells to build “beta cells, which have been shown to cure experimental diabetes in mice and rats… could become the most significant stem cell-based medical treatment of the coming decade…”
10. HEART ATTACK: Rachel Smith, of Capricor, Inc., wants to use stem cells to turn heart attack scars back into healthy tissue. As scarring makes the next heart attack more likely, Smith’s method may save lives.
11. HEART ATTACK: Eduardo Marban of Cedars-Sinai Medical Center, says: “The adult human heart contains small numbers of cardiac stem cells that are able to partially repair the heart following a heart attack…we have developed a way to isolate these cells and grow them to large numbers…” Clusters of these cells could be injected into the heart– even in the middle of a heart attack.
12. HEART FAILURE: Robert Robbins, Stanford, intends to use “embryonic stem cell-derived (heart cells) for patients with end stage heart failure…transplanting cells rather than a whole heart….”
13. HIV/AIDS: Irving Chen, UCLA, believes that the process which can change the color of a flower (RNA interference) may “block the spread of HIV infection…”. Right now, surviving with AIDS means requires several dozen pills a day. But the Chen method could mean “a therapy that will require only a single treatment…”
14. HIV/AIDS: John Zaia, City of Hope, may have found a way to duplicate a “cure” of an AIDS patient who received a bone marrow transplant with a mutation called delta-32. This mutation apparently blocks entry of the AIDS virus. It may be possible to alter a patient’s cells so that they can also block the virus.
15. HUNTINGTON’S DISEASE: Vicki Wheelock, UC Davis, hopes to fight the fatal disease with stem cells as a delivery vehicle: bringing a nerve-growth factor called Brain Derived Neurotrophic Factor, (BDNF) to the at-risk nerve cells.
16. LEUKEMIA: Catriona Jamieson and Dennis Carson of UCSD are fighting an after-treatment problem with the deadly disease; it often leaves small amounts of leukemia stem cells still alive, to come back stronger later. The Carson team hopes to make anti-leukemia drugs available soon.
17. LEUKEMIA: Irv Weissman, Stanford. The body’s protective cells, macrophages, should “eat” the leukemia invaders—but they don’t. A marker cell called CD-47 on the leukemia cells acts like a “don’t eat me” sticker. But if the marker could be removed….
18. LOU GEHRIG’S DISEASE: Larry Goldstein, UCSD, hopes to prevent the progression of the fatal disease (amyotrophic lateral sclerosis, ALS, or Lou Gehrig’s Disease) by using astrocytes (nerve support cells) developed from embryonic stem cells.
19. LOU GEHRIG’S DISEASE: Clive Svendsen, Cedars-Sinai Medical Center, wants to use nerve “fertilizer” (Glial Derived Neurotrophic Factor, GDNF) to treat the deadly disease. Svendsen wants to use neural progenitor cells as “Trojan horses”, to carry the GDNF to the nerve cells, where it is needed.
20. MUSCULAR DYSTROPHY: Stanley Nelson, UCLA, is tackling the devastating and muscle-wasting disease. Nelson’s technique brings repairs “directly inside each muscle cell”… and is “predicted to lessen the disease severity…”
21. OSTEOPOROSIS: Nancy Lane, UC Davis, wants to strengthen the bones of the elderly, and thereby lessen the number of bone fractures they suffer. In osteoporosis, bones can grow almost transparently thin and accordingly weak. Lane has developed a small molecule that directs stem cells to grow the skeleton stronger.
22. SICKLE CELL DISEASE: Donald Kohn, UCLA, offers a stem cell therapy for the agonizing and often fatal disease. The blood cells change shape into a c-shape like a sickle, said to cause pain like broken glass in the veins. Kohn’s approach will transplant “adult cells that are genetically corrected by…a gene that blocks sickling…”
23. SKIN DISEASE: Alfred Lane, Stanford, hopes to use induced Pluripotent Stem Cells (iPSC)s to combat a hideous disease, dystrophic epidermolysis bullosa, in which the skin literally rips apart. His method may treat other skin disorders as well.
24. SPINAL CORD INJURY: Nobuko Uchida, Stem Cells Inc., wants to transplant neural stem cells into the spine to ease chronic cervical spinal cord injury. His method has restored motion in animal models, and “could potentially reverse paralysis and improve the patient’s quality of life.” For paralyzed people, even a small improvement is hugely positive: perhaps the difference between needing an attendant and not.
25. STROKE: Gary Steinberg, Stanford, calls stroke “the number one cause of disability, the second leading cause of dementia, and the third leading cause of death in adults…” His team will use embryonic stem cells– to make neural stem cells– to “augment the body’s natural repair process after stroke.”